The United States of America are not united. Politics have polarized the competing monologues and the policy making around vaccines, masks, children returning to school, what children are taught in school, and whether the federal government (or the National Football League) can or should create universal mandates enforcing one extreme of any of those policy disputes. Public health and health care have become so entangled in polarized politics that the role of science has often been pushed aside.

Polarization is not a novel event in the history of governments. The partition of India in 1947 divided most of its Hindu and Muslim inhabitants into separate countries, but that hasn’t stopped the recent resurgence of Hindu nationalism in India. The Thirty Years’ War in Europe sought to decide whether Catholics or Protestants would dominate Western Christianity. Those two sides decided in 1648 that coexistence was wiser than continuing into the abyss of mutual annihilation. Current conflicts between Israelis and Palestinians, between Shia and Sunni Arab states, between China and the Uyghurs, and within Sudan and Ethiopia together demonstrate that polarization to the point of genocide can occur regardless of religion, race, and nationality.

Abraham Lincoln, a lawyer in Illinois with a habit of losing elections, was nominated in 1858 to be the Republican nominee in the U.S. Senate race. His speech accepting the nomination spoke a truth that resonated across the nation and across time. It is known as the House Divided speech. He said: “A house divided against itself cannot stand. I believe this government cannot endure, permanently half slave and half free. I do not expect the Union to be dissolved – I do not expect the house to fall – but I do expect it will cease to be divided. It will become all one thing or all the other.”

The Republican Lincoln, supported by antislavery groups, lost that election to the Democrat Stephen A. Douglas, whose party espoused popular sovereignty and local decision-making about slavery. Lincoln’s acceptance speech propelled him 2 years later to be nominated for and elected President of the United States. Lincoln’s first inaugural address as the President of the United States on March 4, 1861, focused on the issue of division and secession. This time, Lincoln placed much more emphasis on preserving the Union. He specifically renounced any federal efforts to use force to abolish slavery in the states that permitted it. He declared: “I have no purpose, directly or indirectly, to interfere with the institution of slavery in the States where it exists. I believe I have no lawful right to do so, and I have no inclination to do so.”

President Lincoln’s approach might not meet muster in today’s cancel culture. He was facing a precariously divided nation not unlike the current day, so his speech contains insights and wisdom important for today. Lincoln saw government as “a majority held in restraint by constitutional checks and limitations.” I am loath to further quote out of context or paraphrase his masterful words. Go read the original, in its balanced entirety.

I have written previous columns about the importance of taking time to reflect on one’s life and one’s career. Reflection is both a wellness check and a moral compass check. Some call it mindfulness. I lean toward calling it thankfulness and gratitude. Hence, November is a convenient time for pediatricians if flu and respiratory syncytial virus seasons haven’t started.

The Gettysburg Address extols the virtue of dedication. Lincoln’s second inaugural address promotes mercy and forgiveness. His Farewell Address to Springfield in 1861 in a single paragraph captures grief, faith, and hope. Those speeches are my perennial favorites. But this year it is the two aforementioned addresses that must be mined for wisdom.

I advocate vaccine and mask mandates, but I am not enamored with the idea of President Biden using the unchecked power of the executive branch to promulgate a single federal regulation that overreaches into every moderate-size business nationwide. The 1861 inaugural address concurs. Lincoln’s prophecy that division will be solved when one side ultimately wins is not the model I seek. It hasn’t worked for gun control. It hasn’t worked for abortion as we approach the 50th anniversary of Roe v. Wade. The present 50+1 vote majority in the U.S. Senate does not have a mandate to overhaul society, especially when those majorities are transient. One should have the courage to seek change, but beware of creating large divisions with small majorities.

Facebook profits when you meditate in the echo chambers of large, outraged groups. Avoid that. Hebrew tradition has some reflection occurring in groups of two or three, rather than solo. Truth is revealed in community. Voltaire said: “Cherish those who seek the truth but beware of those who find it.” As a scientist, my experience is that humility, skepticism, and a dedication to finding truth have served me well for a lifetime.
 

Dr. Powell is a retired pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

The United States of America are not united. Politics have polarized the competing monologues and the policy making around vaccines, masks, children returning to school, what children are taught in school, and whether the federal government (or the National Football League) can or should create universal mandates enforcing one extreme of any of those policy disputes. Public health and health care have become so entangled in polarized politics that the role of science has often been pushed aside.

Polarization is not a novel event in the history of governments. The partition of India in 1947 divided most of its Hindu and Muslim inhabitants into separate countries, but that hasn’t stopped the recent resurgence of Hindu nationalism in India. The Thirty Years’ War in Europe sought to decide whether Catholics or Protestants would dominate Western Christianity. Those two sides decided in 1648 that coexistence was wiser than continuing into the abyss of mutual annihilation. Current conflicts between Israelis and Palestinians, between Shia and Sunni Arab states, between China and the Uyghurs, and within Sudan and Ethiopia together demonstrate that polarization to the point of genocide can occur regardless of religion, race, and nationality.

Abraham Lincoln, a lawyer in Illinois with a habit of losing elections, was nominated in 1858 to be the Republican nominee in the U.S. Senate race. His speech accepting the nomination spoke a truth that resonated across the nation and across time. It is known as the House Divided speech. He said: “A house divided against itself cannot stand. I believe this government cannot endure, permanently half slave and half free. I do not expect the Union to be dissolved – I do not expect the house to fall – but I do expect it will cease to be divided. It will become all one thing or all the other.”

The Republican Lincoln, supported by antislavery groups, lost that election to the Democrat Stephen A. Douglas, whose party espoused popular sovereignty and local decision-making about slavery. Lincoln’s acceptance speech propelled him 2 years later to be nominated for and elected President of the United States. Lincoln’s first inaugural address as the President of the United States on March 4, 1861, focused on the issue of division and secession. This time, Lincoln placed much more emphasis on preserving the Union. He specifically renounced any federal efforts to use force to abolish slavery in the states that permitted it. He declared: “I have no purpose, directly or indirectly, to interfere with the institution of slavery in the States where it exists. I believe I have no lawful right to do so, and I have no inclination to do so.”

President Lincoln’s approach might not meet muster in today’s cancel culture. He was facing a precariously divided nation not unlike the current day, so his speech contains insights and wisdom important for today. Lincoln saw government as “a majority held in restraint by constitutional checks and limitations.” I am loath to further quote out of context or paraphrase his masterful words. Go read the original, in its balanced entirety.

I have written previous columns about the importance of taking time to reflect on one’s life and one’s career. Reflection is both a wellness check and a moral compass check. Some call it mindfulness. I lean toward calling it thankfulness and gratitude. Hence, November is a convenient time for pediatricians if flu and respiratory syncytial virus seasons haven’t started.

The Gettysburg Address extols the virtue of dedication. Lincoln’s second inaugural address promotes mercy and forgiveness. His Farewell Address to Springfield in 1861 in a single paragraph captures grief, faith, and hope. Those speeches are my perennial favorites. But this year it is the two aforementioned addresses that must be mined for wisdom.

I advocate vaccine and mask mandates, but I am not enamored with the idea of President Biden using the unchecked power of the executive branch to promulgate a single federal regulation that overreaches into every moderate-size business nationwide. The 1861 inaugural address concurs. Lincoln’s prophecy that division will be solved when one side ultimately wins is not the model I seek. It hasn’t worked for gun control. It hasn’t worked for abortion as we approach the 50th anniversary of Roe v. Wade. The present 50+1 vote majority in the U.S. Senate does not have a mandate to overhaul society, especially when those majorities are transient. One should have the courage to seek change, but beware of creating large divisions with small majorities.

Facebook profits when you meditate in the echo chambers of large, outraged groups. Avoid that. Hebrew tradition has some reflection occurring in groups of two or three, rather than solo. Truth is revealed in community. Voltaire said: “Cherish those who seek the truth but beware of those who find it.” As a scientist, my experience is that humility, skepticism, and a dedication to finding truth have served me well for a lifetime.
 

Dr. Powell is a retired pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

The United States of America are not united. Politics have polarized the competing monologues and the policy making around vaccines, masks, children returning to school, what children are taught in school, and whether the federal government (or the National Football League) can or should create universal mandates enforcing one extreme of any of those policy disputes. Public health and health care have become so entangled in polarized politics that the role of science has often been pushed aside.

Polarization is not a novel event in the history of governments. The partition of India in 1947 divided most of its Hindu and Muslim inhabitants into separate countries, but that hasn’t stopped the recent resurgence of Hindu nationalism in India. The Thirty Years’ War in Europe sought to decide whether Catholics or Protestants would dominate Western Christianity. Those two sides decided in 1648 that coexistence was wiser than continuing into the abyss of mutual annihilation. Current conflicts between Israelis and Palestinians, between Shia and Sunni Arab states, between China and the Uyghurs, and within Sudan and Ethiopia together demonstrate that polarization to the point of genocide can occur regardless of religion, race, and nationality.

Abraham Lincoln, a lawyer in Illinois with a habit of losing elections, was nominated in 1858 to be the Republican nominee in the U.S. Senate race. His speech accepting the nomination spoke a truth that resonated across the nation and across time. It is known as the House Divided speech. He said: “A house divided against itself cannot stand. I believe this government cannot endure, permanently half slave and half free. I do not expect the Union to be dissolved – I do not expect the house to fall – but I do expect it will cease to be divided. It will become all one thing or all the other.”

The Republican Lincoln, supported by antislavery groups, lost that election to the Democrat Stephen A. Douglas, whose party espoused popular sovereignty and local decision-making about slavery. Lincoln’s acceptance speech propelled him 2 years later to be nominated for and elected President of the United States. Lincoln’s first inaugural address as the President of the United States on March 4, 1861, focused on the issue of division and secession. This time, Lincoln placed much more emphasis on preserving the Union. He specifically renounced any federal efforts to use force to abolish slavery in the states that permitted it. He declared: “I have no purpose, directly or indirectly, to interfere with the institution of slavery in the States where it exists. I believe I have no lawful right to do so, and I have no inclination to do so.”

President Lincoln’s approach might not meet muster in today’s cancel culture. He was facing a precariously divided nation not unlike the current day, so his speech contains insights and wisdom important for today. Lincoln saw government as “a majority held in restraint by constitutional checks and limitations.” I am loath to further quote out of context or paraphrase his masterful words. Go read the original, in its balanced entirety.

I have written previous columns about the importance of taking time to reflect on one’s life and one’s career. Reflection is both a wellness check and a moral compass check. Some call it mindfulness. I lean toward calling it thankfulness and gratitude. Hence, November is a convenient time for pediatricians if flu and respiratory syncytial virus seasons haven’t started.

The Gettysburg Address extols the virtue of dedication. Lincoln’s second inaugural address promotes mercy and forgiveness. His Farewell Address to Springfield in 1861 in a single paragraph captures grief, faith, and hope. Those speeches are my perennial favorites. But this year it is the two aforementioned addresses that must be mined for wisdom.

I advocate vaccine and mask mandates, but I am not enamored with the idea of President Biden using the unchecked power of the executive branch to promulgate a single federal regulation that overreaches into every moderate-size business nationwide. The 1861 inaugural address concurs. Lincoln’s prophecy that division will be solved when one side ultimately wins is not the model I seek. It hasn’t worked for gun control. It hasn’t worked for abortion as we approach the 50th anniversary of Roe v. Wade. The present 50+1 vote majority in the U.S. Senate does not have a mandate to overhaul society, especially when those majorities are transient. One should have the courage to seek change, but beware of creating large divisions with small majorities.

Facebook profits when you meditate in the echo chambers of large, outraged groups. Avoid that. Hebrew tradition has some reflection occurring in groups of two or three, rather than solo. Truth is revealed in community. Voltaire said: “Cherish those who seek the truth but beware of those who find it.” As a scientist, my experience is that humility, skepticism, and a dedication to finding truth have served me well for a lifetime.
 

Dr. Powell is a retired pediatric hospitalist and clinical ethics consultant living in St. Louis. Email him at [email protected].

Sudeep Taksali, MD, thought he’d won his battle to avoid a steep price tag on a medicine for his daughter. He was wrong.

In 2020, he’d fought to get insurance to cover a lower-priced version of a drug his then-8-year-old needed. She’d been diagnosed with central precocious puberty, a rare condition marked by early onset of sexual development – often years earlier than one’s peers. KHN and NPR wrote about Dr. Taksali and his family as part of the Bill of the Month series.

The girl’s doctors and the Taksalis decided to put her puberty on pause with a hormone-blocking drug implant that would be placed under the skin in her arm and release a little bit of the medication each day.

Dr. Taksali, an orthopedic surgeon, learned there were two nearly identical drug products made by Endo Pharmaceuticals, both containing 50 mg of the hormone blocker histrelin. One cost more than eight times more than the other. He wanted to use the cheaper one, Vantas, which costs about $4,800 per implant. But his insurer would not initially cover it, instead preferring Supprelin LA, which is approved by the Food and Drug Administration to treat central precocious puberty, and costs about $43,000.

Vantas can be prescribed off label for the condition, and after much back-and-forth dialogue, Dr. Taksali finally got the insurer to cover it.

Then this summer, it was time to replace the implant.

“I thought we would just get a Vantas replacement,” Dr. Taksali said. “In my mind, I was like: ‘Well, she got it the first time, and we’ve already kind of fought the battle with the insurance company and, you know, got it approved.”

But during a virtual appointment with his daughter’s doctor, he learned they couldn’t get Vantas. No one could. There was a Vantas shortage.

Endo cited a manufacturing problem. Batches of Vantas weren’t coming out right and couldn’t be released to the public, the company’s vice president of corporate affairs, Heather Zoumas Lubeski, said in an email. Vantas and Supprelin were made in the same facility, but the problem affected only Vantas, she wrote, stressing that the drugs are “not identical products.”

In August, Endo’s president and CEO, Blaise Coleman, told investors Supprelin was doing particularly well for the company. Revenue had grown by 79%, compared with the same quarter the year before. The growth was driven in part, Mr. Coleman explained, “by stronger-than-expected demand resulting from expanded patient awareness and a competitor product shortage,” he said.

What competitor product shortage? Could that be Vantas?

Asked about this, Ms. Zoumas Lubeski said Mr. Coleman wasn’t referring to Vantas. Since Vantas isn’t approved to treat central precocious puberty, it can’t technically be considered a competitor to Supprelin. Mr. Coleman was referring to the rival product Lupron Depot-Ped, not an implant, but an injection made by AbbVie, Ms. Zoumas Lubeski said.

Dr. Taksali was skeptical.

“It’s all very curious, like, huh, you know, when this particular option went away and your profits went up nearly 80% from the more expensive drug,” he said.

Then, in September, Endo told the FDA it stopped making Vantas for good.

Ms. Zoumas Lubeski said that, when Endo investigated its Vantas manufacturing problem, it wasn’t able to find “a suitable corrective action that resolves the issue.”

“As a result, and after analysis of the market for the availability of alternative therapies, we made the difficult decision to discontinue the supply of this product,” she said via email. “Endo is committed to maintaining the highest quality standards for all of its products.”

Dr. Taksali said he felt resigned to giving his daughter Supprelin even before the shortage turned into a discontinuation. Ultimately, he won’t pay much more out-of-pocket, but his insurance will pay the rest. And that could raise his business’s premiums.

The FDA cannot force Endo to keep making the drug or set a lower price for the remaining one. It doesn’t have the authority. That decision lies with Endo Pharmaceuticals. A drugmaker discontinuing a product isn’t anything new, said Erin Fox, who directs drug information and support services at University of Utah Health hospitals.

“The FDA has very little leverage because there is no requirement for any company to make any drug, no matter how lifesaving,” she said. “We have a capitalist society. We have a free market. And so any company can discontinue anything ... at any time for any reason.”

Still, companies are supposed to tell the FDA about potential shortages and discontinuations ahead of time so it can minimize the impact on public health. It can help a firm resolve a manufacturing issue, decide whether it’s safe to extend an expiration date or help a company making an alternative product to ramp up production.

“The FDA expects that manufacturers will notify the agency before a meaningful disruption in their own supply occurs,” FDA spokesperson Jeremy Kahn wrote in an email. “When the FDA does not receive timely, informative notifications, the agency’s ability to respond appropriately is limited.”

But the rules are somewhat flexible. A company is required to notify the FDA of an upcoming drug supply disruption 6 months before it affects consumers or “as soon as practicable” after that. But their true deadline is 5 business days after manufacturing stops, according to the FDA website.

“They’re supposed to tell the FDA, but even if they don’t, there’s no penalty,” Ms. Fox said. “There’s no teeth in that law. ... Their name can go on the FDA naughty list. That’s pretty much it.”

In rare cases, the FDA will send a noncompliance letter to the drugmaker and require it to explain itself. This has happened only five times since 2015. There is no such letter about Vantas, suggesting that Endo met the FDA’s requirements for notification.

Concerned about potential drug shortages caused by COVID-19 in March 2020, a bipartisan group of legislators introduced the Preventing Drug Shortages Act, which aimed to increase transparency around shortages. But the legislation gained no traction.

As a result of limited FDA power, the intricacies of drug shortages remain opaque, Ms. Fox said. Companies don’t have to make the reasons for shortages public. That sets the Vantas shortage and discontinuation apart from many others. The company is saying more about what happened than most do.

“Many companies will actually just put drugs on temporarily unavailable or long-term backorder, and sometimes that can last years before the company finally makes a decision” on whether to discontinue a product, she said. “It can take a long time, and so it can be frustrating to not know – or to kind of stake your hopes on a product coming back to the market once it’s been in shortage for so long.”

It’s hard to know exactly how many children will be affected by the Vantas discontinuation because data about off-label use is hard to come by.

Erica Eugster, MD, a professor of pediatrics at Indiana University, Indianapolis, said central precocious puberty patients weren’t her first thought when she learned of the Vantas discontinuation.

“I immediately thought about our transgender population,” she said. “They’re the ones that are really going to suffer from this.”

No medications have been FDA approved to treat patients with gender dysphoria, the medical term for when the sex assigned at birth doesn’t match someone’s gender identity, causing them psychological distress. As a result, any drug to stop puberty in this population would be off label, making it difficult for families to get health insurance coverage. Vantas had been a lower-cost option.

The number of transgender patients receiving histrelin implants rose significantly from 2004 to 2016, according to a study published in the Journal of Pediatric Endocrinology and Metabolism.

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Sudeep Taksali, MD, thought he’d won his battle to avoid a steep price tag on a medicine for his daughter. He was wrong.

In 2020, he’d fought to get insurance to cover a lower-priced version of a drug his then-8-year-old needed. She’d been diagnosed with central precocious puberty, a rare condition marked by early onset of sexual development – often years earlier than one’s peers. KHN and NPR wrote about Dr. Taksali and his family as part of the Bill of the Month series.

The girl’s doctors and the Taksalis decided to put her puberty on pause with a hormone-blocking drug implant that would be placed under the skin in her arm and release a little bit of the medication each day.

Dr. Taksali, an orthopedic surgeon, learned there were two nearly identical drug products made by Endo Pharmaceuticals, both containing 50 mg of the hormone blocker histrelin. One cost more than eight times more than the other. He wanted to use the cheaper one, Vantas, which costs about $4,800 per implant. But his insurer would not initially cover it, instead preferring Supprelin LA, which is approved by the Food and Drug Administration to treat central precocious puberty, and costs about $43,000.

Vantas can be prescribed off label for the condition, and after much back-and-forth dialogue, Dr. Taksali finally got the insurer to cover it.

Then this summer, it was time to replace the implant.

“I thought we would just get a Vantas replacement,” Dr. Taksali said. “In my mind, I was like: ‘Well, she got it the first time, and we’ve already kind of fought the battle with the insurance company and, you know, got it approved.”

But during a virtual appointment with his daughter’s doctor, he learned they couldn’t get Vantas. No one could. There was a Vantas shortage.

Endo cited a manufacturing problem. Batches of Vantas weren’t coming out right and couldn’t be released to the public, the company’s vice president of corporate affairs, Heather Zoumas Lubeski, said in an email. Vantas and Supprelin were made in the same facility, but the problem affected only Vantas, she wrote, stressing that the drugs are “not identical products.”

In August, Endo’s president and CEO, Blaise Coleman, told investors Supprelin was doing particularly well for the company. Revenue had grown by 79%, compared with the same quarter the year before. The growth was driven in part, Mr. Coleman explained, “by stronger-than-expected demand resulting from expanded patient awareness and a competitor product shortage,” he said.

What competitor product shortage? Could that be Vantas?

Asked about this, Ms. Zoumas Lubeski said Mr. Coleman wasn’t referring to Vantas. Since Vantas isn’t approved to treat central precocious puberty, it can’t technically be considered a competitor to Supprelin. Mr. Coleman was referring to the rival product Lupron Depot-Ped, not an implant, but an injection made by AbbVie, Ms. Zoumas Lubeski said.

Dr. Taksali was skeptical.

“It’s all very curious, like, huh, you know, when this particular option went away and your profits went up nearly 80% from the more expensive drug,” he said.

Then, in September, Endo told the FDA it stopped making Vantas for good.

Ms. Zoumas Lubeski said that, when Endo investigated its Vantas manufacturing problem, it wasn’t able to find “a suitable corrective action that resolves the issue.”

“As a result, and after analysis of the market for the availability of alternative therapies, we made the difficult decision to discontinue the supply of this product,” she said via email. “Endo is committed to maintaining the highest quality standards for all of its products.”

Dr. Taksali said he felt resigned to giving his daughter Supprelin even before the shortage turned into a discontinuation. Ultimately, he won’t pay much more out-of-pocket, but his insurance will pay the rest. And that could raise his business’s premiums.

The FDA cannot force Endo to keep making the drug or set a lower price for the remaining one. It doesn’t have the authority. That decision lies with Endo Pharmaceuticals. A drugmaker discontinuing a product isn’t anything new, said Erin Fox, who directs drug information and support services at University of Utah Health hospitals.

“The FDA has very little leverage because there is no requirement for any company to make any drug, no matter how lifesaving,” she said. “We have a capitalist society. We have a free market. And so any company can discontinue anything ... at any time for any reason.”

Still, companies are supposed to tell the FDA about potential shortages and discontinuations ahead of time so it can minimize the impact on public health. It can help a firm resolve a manufacturing issue, decide whether it’s safe to extend an expiration date or help a company making an alternative product to ramp up production.

“The FDA expects that manufacturers will notify the agency before a meaningful disruption in their own supply occurs,” FDA spokesperson Jeremy Kahn wrote in an email. “When the FDA does not receive timely, informative notifications, the agency’s ability to respond appropriately is limited.”

But the rules are somewhat flexible. A company is required to notify the FDA of an upcoming drug supply disruption 6 months before it affects consumers or “as soon as practicable” after that. But their true deadline is 5 business days after manufacturing stops, according to the FDA website.

“They’re supposed to tell the FDA, but even if they don’t, there’s no penalty,” Ms. Fox said. “There’s no teeth in that law. ... Their name can go on the FDA naughty list. That’s pretty much it.”

In rare cases, the FDA will send a noncompliance letter to the drugmaker and require it to explain itself. This has happened only five times since 2015. There is no such letter about Vantas, suggesting that Endo met the FDA’s requirements for notification.

Concerned about potential drug shortages caused by COVID-19 in March 2020, a bipartisan group of legislators introduced the Preventing Drug Shortages Act, which aimed to increase transparency around shortages. But the legislation gained no traction.

As a result of limited FDA power, the intricacies of drug shortages remain opaque, Ms. Fox said. Companies don’t have to make the reasons for shortages public. That sets the Vantas shortage and discontinuation apart from many others. The company is saying more about what happened than most do.

“Many companies will actually just put drugs on temporarily unavailable or long-term backorder, and sometimes that can last years before the company finally makes a decision” on whether to discontinue a product, she said. “It can take a long time, and so it can be frustrating to not know – or to kind of stake your hopes on a product coming back to the market once it’s been in shortage for so long.”

It’s hard to know exactly how many children will be affected by the Vantas discontinuation because data about off-label use is hard to come by.

Erica Eugster, MD, a professor of pediatrics at Indiana University, Indianapolis, said central precocious puberty patients weren’t her first thought when she learned of the Vantas discontinuation.

“I immediately thought about our transgender population,” she said. “They’re the ones that are really going to suffer from this.”

No medications have been FDA approved to treat patients with gender dysphoria, the medical term for when the sex assigned at birth doesn’t match someone’s gender identity, causing them psychological distress. As a result, any drug to stop puberty in this population would be off label, making it difficult for families to get health insurance coverage. Vantas had been a lower-cost option.

The number of transgender patients receiving histrelin implants rose significantly from 2004 to 2016, according to a study published in the Journal of Pediatric Endocrinology and Metabolism.

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Sudeep Taksali, MD, thought he’d won his battle to avoid a steep price tag on a medicine for his daughter. He was wrong.

In 2020, he’d fought to get insurance to cover a lower-priced version of a drug his then-8-year-old needed. She’d been diagnosed with central precocious puberty, a rare condition marked by early onset of sexual development – often years earlier than one’s peers. KHN and NPR wrote about Dr. Taksali and his family as part of the Bill of the Month series.

The girl’s doctors and the Taksalis decided to put her puberty on pause with a hormone-blocking drug implant that would be placed under the skin in her arm and release a little bit of the medication each day.

Dr. Taksali, an orthopedic surgeon, learned there were two nearly identical drug products made by Endo Pharmaceuticals, both containing 50 mg of the hormone blocker histrelin. One cost more than eight times more than the other. He wanted to use the cheaper one, Vantas, which costs about $4,800 per implant. But his insurer would not initially cover it, instead preferring Supprelin LA, which is approved by the Food and Drug Administration to treat central precocious puberty, and costs about $43,000.

Vantas can be prescribed off label for the condition, and after much back-and-forth dialogue, Dr. Taksali finally got the insurer to cover it.

Then this summer, it was time to replace the implant.

“I thought we would just get a Vantas replacement,” Dr. Taksali said. “In my mind, I was like: ‘Well, she got it the first time, and we’ve already kind of fought the battle with the insurance company and, you know, got it approved.”

But during a virtual appointment with his daughter’s doctor, he learned they couldn’t get Vantas. No one could. There was a Vantas shortage.

Endo cited a manufacturing problem. Batches of Vantas weren’t coming out right and couldn’t be released to the public, the company’s vice president of corporate affairs, Heather Zoumas Lubeski, said in an email. Vantas and Supprelin were made in the same facility, but the problem affected only Vantas, she wrote, stressing that the drugs are “not identical products.”

In August, Endo’s president and CEO, Blaise Coleman, told investors Supprelin was doing particularly well for the company. Revenue had grown by 79%, compared with the same quarter the year before. The growth was driven in part, Mr. Coleman explained, “by stronger-than-expected demand resulting from expanded patient awareness and a competitor product shortage,” he said.

What competitor product shortage? Could that be Vantas?

Asked about this, Ms. Zoumas Lubeski said Mr. Coleman wasn’t referring to Vantas. Since Vantas isn’t approved to treat central precocious puberty, it can’t technically be considered a competitor to Supprelin. Mr. Coleman was referring to the rival product Lupron Depot-Ped, not an implant, but an injection made by AbbVie, Ms. Zoumas Lubeski said.

Dr. Taksali was skeptical.

“It’s all very curious, like, huh, you know, when this particular option went away and your profits went up nearly 80% from the more expensive drug,” he said.

Then, in September, Endo told the FDA it stopped making Vantas for good.

Ms. Zoumas Lubeski said that, when Endo investigated its Vantas manufacturing problem, it wasn’t able to find “a suitable corrective action that resolves the issue.”

“As a result, and after analysis of the market for the availability of alternative therapies, we made the difficult decision to discontinue the supply of this product,” she said via email. “Endo is committed to maintaining the highest quality standards for all of its products.”

Dr. Taksali said he felt resigned to giving his daughter Supprelin even before the shortage turned into a discontinuation. Ultimately, he won’t pay much more out-of-pocket, but his insurance will pay the rest. And that could raise his business’s premiums.

The FDA cannot force Endo to keep making the drug or set a lower price for the remaining one. It doesn’t have the authority. That decision lies with Endo Pharmaceuticals. A drugmaker discontinuing a product isn’t anything new, said Erin Fox, who directs drug information and support services at University of Utah Health hospitals.

“The FDA has very little leverage because there is no requirement for any company to make any drug, no matter how lifesaving,” she said. “We have a capitalist society. We have a free market. And so any company can discontinue anything ... at any time for any reason.”

Still, companies are supposed to tell the FDA about potential shortages and discontinuations ahead of time so it can minimize the impact on public health. It can help a firm resolve a manufacturing issue, decide whether it’s safe to extend an expiration date or help a company making an alternative product to ramp up production.

“The FDA expects that manufacturers will notify the agency before a meaningful disruption in their own supply occurs,” FDA spokesperson Jeremy Kahn wrote in an email. “When the FDA does not receive timely, informative notifications, the agency’s ability to respond appropriately is limited.”

But the rules are somewhat flexible. A company is required to notify the FDA of an upcoming drug supply disruption 6 months before it affects consumers or “as soon as practicable” after that. But their true deadline is 5 business days after manufacturing stops, according to the FDA website.

“They’re supposed to tell the FDA, but even if they don’t, there’s no penalty,” Ms. Fox said. “There’s no teeth in that law. ... Their name can go on the FDA naughty list. That’s pretty much it.”

In rare cases, the FDA will send a noncompliance letter to the drugmaker and require it to explain itself. This has happened only five times since 2015. There is no such letter about Vantas, suggesting that Endo met the FDA’s requirements for notification.

Concerned about potential drug shortages caused by COVID-19 in March 2020, a bipartisan group of legislators introduced the Preventing Drug Shortages Act, which aimed to increase transparency around shortages. But the legislation gained no traction.

As a result of limited FDA power, the intricacies of drug shortages remain opaque, Ms. Fox said. Companies don’t have to make the reasons for shortages public. That sets the Vantas shortage and discontinuation apart from many others. The company is saying more about what happened than most do.

“Many companies will actually just put drugs on temporarily unavailable or long-term backorder, and sometimes that can last years before the company finally makes a decision” on whether to discontinue a product, she said. “It can take a long time, and so it can be frustrating to not know – or to kind of stake your hopes on a product coming back to the market once it’s been in shortage for so long.”

It’s hard to know exactly how many children will be affected by the Vantas discontinuation because data about off-label use is hard to come by.

Erica Eugster, MD, a professor of pediatrics at Indiana University, Indianapolis, said central precocious puberty patients weren’t her first thought when she learned of the Vantas discontinuation.

“I immediately thought about our transgender population,” she said. “They’re the ones that are really going to suffer from this.”

No medications have been FDA approved to treat patients with gender dysphoria, the medical term for when the sex assigned at birth doesn’t match someone’s gender identity, causing them psychological distress. As a result, any drug to stop puberty in this population would be off label, making it difficult for families to get health insurance coverage. Vantas had been a lower-cost option.

The number of transgender patients receiving histrelin implants rose significantly from 2004 to 2016, according to a study published in the Journal of Pediatric Endocrinology and Metabolism.

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Although genomic testing is useful when an interstitial lung disease diagnosis is uncertain, the testing results themselves aren’t sufficient to make the diagnosis, Daniel Dilling, MD, FCCP, said in a presentation at the annual meeting of the American College of Chest Physicians, which was held virtually.

The genomic classifier (Envisia, Veracyte) helps differentiate idiopathic pulmonary fibrosis (IPF) by detecting usual interstitial pneumonia (UIP), the hallmark pattern of this interstitial lung disease.

However, UIP is just one piece of the larger diagnostic puzzle, according to Dr. Dilling, professor of medicine in the interstitial lung disease program at Loyola University Medical Center in Maywood, Ill.

“Remember, it’s just a pattern, and not a diagnosis of IPF,” Dr. Dilling said in his presentation.

Genomic classifier results correlate well with both histologic and radiographic UIP pattern, studies show.

However, Dr. Dilling said the value of the genomic classifier is not in isolation.

“We don’t use this in a vacuum,” he said. “It increases our confidence and consensus, but it has to be incorporated into a multidisciplinary discussion group.”
 

Part of the diagnostic pathway

Dr. Dilling said the genomic classifier should be considered part of a diagnostic pathway in uncertain cases, particularly when the risk of surgical lung biopsy is high.

Current clinical practice guidelines recommend surgical lung biopsy for histopathologic diagnosis when clinical and radiologic findings are not definitive for IPF, the speaker said.

However, surgical lung biopsy carries some risk, and sometimes it can’t be done, he added.

In his presentation, Dr. Dilling cited a systematic review and meta-analysis of 23 studies looking at surgical lung biopsy for the diagnosis of interstitial lung diseases.

The postoperative mortality rate was 3.6% in that meta-analysis, published in 2015 in the Journal of Thoracic and Cardiovascular Surgery.

“The final decision regarding whether or not to perform a [surgical lung biopsy] must be based on the balance between benefits to establish a secure diagnosis and the potential risks,” authors wrote at the time.

Mortality risk is higher in immunocompromised and acutely ill patient populations, according to Dr. Dilling, who added that as many of 19% of patients will have complications from surgical lung biopsy.
 

Genomic classifier studies

In a proof-of-principle study, published in 2017 in the Annals of the American Thoracic Society, authors described how they used machine learning to train an algorithm to distinguish UIP from non-UIP pattern in tissue obtained by transbronchial biopsy (TBB).

The top-performing algorithm distinguished UIP from non-UIP conditions in single TBB samples with specificity of 86% and sensitivity of 63%, according to investigators, who said at the time that independent validation would be needed before the genomic classifier could be applied in clinical settings.

In a prospective validation study, published in 2019 in The Lancet Respiratory Medicine, the genomic classifier identified UIP in TBB samples from 49 patients with a specificity of 88% and sensitivity of 70%.

Excluding patients with definite or probable UIP as shown on high-resolution computed tomography, results show that the classifier had a sensitivity of 76%, specificity of 88%, and positive predictive value of 81%.

“The performance of the test is good, even in that scenario,” Dr. Dilling said.
 

Real-world results

Dr. Dilling also highlighted a “real-world” study, published earlier in 2021, demonstrating that UIP pattern recognized by a genomic classifier had encouraging sensitivity and specificity when combined with high-resolution CT and clinical factors.

That study included 96 patients who had both diagnostic lung pathology and a transbronchial lung biopsy for molecular testing with the classifier.

The classifier had a sensitivity of 60.3% and a specificity of 92.1% for histology-proven UIP pattern, investigators said in their report, which appears in the American Journal of Respiratory and Critical Care Medicine.

Local radiologists identified UIP with a sensitivity of 34.0% and specificity of 96.9%. But adding genomic classifier testing to local radiology testing increased the diagnostic yield, investigators said, with a sensitivity of 79.2% and specificity of 90.6%.

“This might suggest that the implementation of this into a local [multidisciplinary discussion] with your local radiology expertise might really improve your recognition of UIP,” Dr. Dilling said.

Dr. Dilling reported disclosures related to Bellerophon, Boehringer Ingelheim, Genentech, Nitto Denko, and Lung Bioengineering.

Although genomic testing is useful when an interstitial lung disease diagnosis is uncertain, the testing results themselves aren’t sufficient to make the diagnosis, Daniel Dilling, MD, FCCP, said in a presentation at the annual meeting of the American College of Chest Physicians, which was held virtually.

The genomic classifier (Envisia, Veracyte) helps differentiate idiopathic pulmonary fibrosis (IPF) by detecting usual interstitial pneumonia (UIP), the hallmark pattern of this interstitial lung disease.

However, UIP is just one piece of the larger diagnostic puzzle, according to Dr. Dilling, professor of medicine in the interstitial lung disease program at Loyola University Medical Center in Maywood, Ill.

“Remember, it’s just a pattern, and not a diagnosis of IPF,” Dr. Dilling said in his presentation.

Genomic classifier results correlate well with both histologic and radiographic UIP pattern, studies show.

However, Dr. Dilling said the value of the genomic classifier is not in isolation.

“We don’t use this in a vacuum,” he said. “It increases our confidence and consensus, but it has to be incorporated into a multidisciplinary discussion group.”
 

Part of the diagnostic pathway

Dr. Dilling said the genomic classifier should be considered part of a diagnostic pathway in uncertain cases, particularly when the risk of surgical lung biopsy is high.

Current clinical practice guidelines recommend surgical lung biopsy for histopathologic diagnosis when clinical and radiologic findings are not definitive for IPF, the speaker said.

However, surgical lung biopsy carries some risk, and sometimes it can’t be done, he added.

In his presentation, Dr. Dilling cited a systematic review and meta-analysis of 23 studies looking at surgical lung biopsy for the diagnosis of interstitial lung diseases.

The postoperative mortality rate was 3.6% in that meta-analysis, published in 2015 in the Journal of Thoracic and Cardiovascular Surgery.

“The final decision regarding whether or not to perform a [surgical lung biopsy] must be based on the balance between benefits to establish a secure diagnosis and the potential risks,” authors wrote at the time.

Mortality risk is higher in immunocompromised and acutely ill patient populations, according to Dr. Dilling, who added that as many of 19% of patients will have complications from surgical lung biopsy.
 

Genomic classifier studies

In a proof-of-principle study, published in 2017 in the Annals of the American Thoracic Society, authors described how they used machine learning to train an algorithm to distinguish UIP from non-UIP pattern in tissue obtained by transbronchial biopsy (TBB).

The top-performing algorithm distinguished UIP from non-UIP conditions in single TBB samples with specificity of 86% and sensitivity of 63%, according to investigators, who said at the time that independent validation would be needed before the genomic classifier could be applied in clinical settings.

In a prospective validation study, published in 2019 in The Lancet Respiratory Medicine, the genomic classifier identified UIP in TBB samples from 49 patients with a specificity of 88% and sensitivity of 70%.

Excluding patients with definite or probable UIP as shown on high-resolution computed tomography, results show that the classifier had a sensitivity of 76%, specificity of 88%, and positive predictive value of 81%.

“The performance of the test is good, even in that scenario,” Dr. Dilling said.
 

Real-world results

Dr. Dilling also highlighted a “real-world” study, published earlier in 2021, demonstrating that UIP pattern recognized by a genomic classifier had encouraging sensitivity and specificity when combined with high-resolution CT and clinical factors.

That study included 96 patients who had both diagnostic lung pathology and a transbronchial lung biopsy for molecular testing with the classifier.

The classifier had a sensitivity of 60.3% and a specificity of 92.1% for histology-proven UIP pattern, investigators said in their report, which appears in the American Journal of Respiratory and Critical Care Medicine.

Local radiologists identified UIP with a sensitivity of 34.0% and specificity of 96.9%. But adding genomic classifier testing to local radiology testing increased the diagnostic yield, investigators said, with a sensitivity of 79.2% and specificity of 90.6%.

“This might suggest that the implementation of this into a local [multidisciplinary discussion] with your local radiology expertise might really improve your recognition of UIP,” Dr. Dilling said.

Dr. Dilling reported disclosures related to Bellerophon, Boehringer Ingelheim, Genentech, Nitto Denko, and Lung Bioengineering.

Although genomic testing is useful when an interstitial lung disease diagnosis is uncertain, the testing results themselves aren’t sufficient to make the diagnosis, Daniel Dilling, MD, FCCP, said in a presentation at the annual meeting of the American College of Chest Physicians, which was held virtually.

The genomic classifier (Envisia, Veracyte) helps differentiate idiopathic pulmonary fibrosis (IPF) by detecting usual interstitial pneumonia (UIP), the hallmark pattern of this interstitial lung disease.

However, UIP is just one piece of the larger diagnostic puzzle, according to Dr. Dilling, professor of medicine in the interstitial lung disease program at Loyola University Medical Center in Maywood, Ill.

“Remember, it’s just a pattern, and not a diagnosis of IPF,” Dr. Dilling said in his presentation.

Genomic classifier results correlate well with both histologic and radiographic UIP pattern, studies show.

However, Dr. Dilling said the value of the genomic classifier is not in isolation.

“We don’t use this in a vacuum,” he said. “It increases our confidence and consensus, but it has to be incorporated into a multidisciplinary discussion group.”
 

Part of the diagnostic pathway

Dr. Dilling said the genomic classifier should be considered part of a diagnostic pathway in uncertain cases, particularly when the risk of surgical lung biopsy is high.

Current clinical practice guidelines recommend surgical lung biopsy for histopathologic diagnosis when clinical and radiologic findings are not definitive for IPF, the speaker said.

However, surgical lung biopsy carries some risk, and sometimes it can’t be done, he added.

In his presentation, Dr. Dilling cited a systematic review and meta-analysis of 23 studies looking at surgical lung biopsy for the diagnosis of interstitial lung diseases.

The postoperative mortality rate was 3.6% in that meta-analysis, published in 2015 in the Journal of Thoracic and Cardiovascular Surgery.

“The final decision regarding whether or not to perform a [surgical lung biopsy] must be based on the balance between benefits to establish a secure diagnosis and the potential risks,” authors wrote at the time.

Mortality risk is higher in immunocompromised and acutely ill patient populations, according to Dr. Dilling, who added that as many of 19% of patients will have complications from surgical lung biopsy.
 

Genomic classifier studies

In a proof-of-principle study, published in 2017 in the Annals of the American Thoracic Society, authors described how they used machine learning to train an algorithm to distinguish UIP from non-UIP pattern in tissue obtained by transbronchial biopsy (TBB).

The top-performing algorithm distinguished UIP from non-UIP conditions in single TBB samples with specificity of 86% and sensitivity of 63%, according to investigators, who said at the time that independent validation would be needed before the genomic classifier could be applied in clinical settings.

In a prospective validation study, published in 2019 in The Lancet Respiratory Medicine, the genomic classifier identified UIP in TBB samples from 49 patients with a specificity of 88% and sensitivity of 70%.

Excluding patients with definite or probable UIP as shown on high-resolution computed tomography, results show that the classifier had a sensitivity of 76%, specificity of 88%, and positive predictive value of 81%.

“The performance of the test is good, even in that scenario,” Dr. Dilling said.
 

Real-world results

Dr. Dilling also highlighted a “real-world” study, published earlier in 2021, demonstrating that UIP pattern recognized by a genomic classifier had encouraging sensitivity and specificity when combined with high-resolution CT and clinical factors.

That study included 96 patients who had both diagnostic lung pathology and a transbronchial lung biopsy for molecular testing with the classifier.

The classifier had a sensitivity of 60.3% and a specificity of 92.1% for histology-proven UIP pattern, investigators said in their report, which appears in the American Journal of Respiratory and Critical Care Medicine.

Local radiologists identified UIP with a sensitivity of 34.0% and specificity of 96.9%. But adding genomic classifier testing to local radiology testing increased the diagnostic yield, investigators said, with a sensitivity of 79.2% and specificity of 90.6%.

“This might suggest that the implementation of this into a local [multidisciplinary discussion] with your local radiology expertise might really improve your recognition of UIP,” Dr. Dilling said.

Dr. Dilling reported disclosures related to Bellerophon, Boehringer Ingelheim, Genentech, Nitto Denko, and Lung Bioengineering.

Should doctors seeking healthcare disclose that they are a doctor? 

The question drew spirited debate when urologist Ashley Winter, MD, made a simple, straightforward request on Twitter: “If you are a doctor & you come to an appointment please tell me you are a doctor, not because I will treat you differently but because it’s easier to speak in jargon.”

She later added, “This doesn’t’ mean I would be less patient-focused or emotional with a physician or other [healthcare worker]. Just means that, instead of saying ‘you will have a catheter draining your urine to a bag,’ I can say, ‘you will have a Foley.’ ”

The Tweet followed an encounter with a patient who told Dr. Winter that he was a doctor only after she had gone to some length explaining a surgical procedure in lay terms.

“I explained the surgery, obviously assuming he was an intelligent adult, but using fully layman’s terms,” she said in an interview. The patient then told her that he was a doctor. “I guess I felt this embarrassment — I wouldn’t have treated him differently, but I just could have discussed the procedure with him in more professional terms.” 

“To some extent, it was my own fault,” she commented in an interview. “I didn’t take the time to ask [about his work] at the beginning of the consultation, but that’s a fine line, also,” added Dr. Winter, a urologist and sexual medicine physician in Portland, Ore.

“You know that patient is there because they want care from you and it’s not necessarily always at the forefront of importance to be asking them what they do for their work, but alternatively, if you don’t ask then you put them in this position where they have to find a way to go ahead and tell you.”

Several people chimed in on the thread to voice their thoughts on the matter. Some commiserated with Dr. Winter’s experience:

“I took care of a retired cardiologist in the hospital as a second-year resident and honest to god he let me ramble on ‘explaining’ his echo result and never told me. I found out a couple days later and wanted to die,” posted @MaddyAndrewsMD.

Another recalled a similarly embarrassing experience when she “went on and on” discussing headaches with a patient whose husband “was in the corner smirking.”

“They told my attending later [that the] husband was a retired FM doc who practiced medicine longer than I’ve been alive. I wanted to die,” posted @JSinghDO.

Many on the thread, though, were doctors and other healthcare professionals speaking as patients. Some said they didn’t want to disclose their status as a healthcare provider because they felt it affected the care they received.

For example, @drhelenrainford commented: “In my experience my care is less ‘caring’ when they know I am a [doctor]. I get spoken to like they are discussing a patient with me — no empathy just facts and difficult results just blurted out without consideration. Awful awful time as an inpatient …but that’s another story!”

@Dr_B_Ring said: “Nope – You and I speak different jargon – I would want you to speak to me like a human that doesn’t know your jargon. My ego would get in the way of asking about the acronyms I don’t know if you knew I was a fellow physician.”

Conversely, @lozzlemcfozzle said: “Honestly I prefer not to tell my Doctors — I’ve found people skip explanations assuming I ‘know,’ or seem a little nervous when I tell them!”

Others said they felt uncomfortable — pretentious, even — in announcing their status, or worried that they might come across as expecting special care.

“It’s such a tough needle to thread. Want to tell people early but not come off as demanding special treatment, but don’t want to wait too long and it seems like a trap,” said @MDaware.

Twitter user @MsBabyCatcher wrote: “I have a hard time doing this because I don’t want people to think I’m being pretentious or going to micromanage/dictate care.”

Replying to @MsBabyCatcher, @RedStethoscope wrote: “I used to think this too until I got [very poor] care a few times, and was advised by other doctor moms to ‘play the doctor card.’ I have gotten better/more compassionate care by making sure it’s clear that I’m a physician (which is junk, but here we are).”

Several of those responding used the words “tricky” and “awkward,” suggesting a common theme for doctors presenting as patients.

“I struggle with this. My 5-year-old broke her arm this weekend, we spent hours in the ED, of my own hospital, I never mentioned it because I didn’t want to get preferential care. But as they were explaining her type of fracture, it felt awkward and inefficient,” said @lindsay_petty.

To avoid the awkwardness, a number of respondents said they purposefully use medical jargon to open up a conversation rather than just offering up the information that they are a doctor.

Still others offered suggestions on how to broach the subject more directly when presenting as a patient:

‘”Just FYI I’m a X doc but I’m here because I really want your help and advice!” That’s what I usually do,” wrote @drcakefm.

@BeeSting14618 Tweeted: “I usually say ‘I know some of this but I’m here because I want YOUR guidance. Also I may ask dumb questions, and I’ll tell you if a question is asking your opinion or making a request.’”

A few others injected a bit of humor: “I just do the 14-part handshake that only doctors know. Is that not customary?” quipped @Branmiz25.

“Ah yes, that transmits the entire [history of present illness],” replied Dr. Winter.

Jokes aside, the topic is obviously one that touched on a shared experience among healthcare providers, Dr. Winter commented. The Twitter thread she started just “blew up.”

That’s typically a sign that the Tweet is relatable for a lot of people, she said.

“It’s definitely something that all of us as care providers and as patients understand. It’s a funny, awkward thing that can really change an interaction, so we probably all feel pretty strongly about our experiences related to that,” she added.

The debate begs the question: Is there a duty or ethical reason to disclose?

“I definitely think it is very reasonable to disclose that one is a medical professional to another doctor,” medical ethicist Charlotte Blease, PhD, said in an interview. “There are good reasons to believe doing so might make a difference to the quality of communication and transparency.”

If the ability to use medical terminology or jargon more freely improves patient understanding, autonomy, and shared decision-making, then it may be of benefit, said Dr. Blease, a Keane OpenNotes Scholar at Beth Israel Deaconess Medical Center in Boston.

“Since doctors should strive to communicate effectively with every patient and to respect their unique needs and level of understanding, then I see no reason to deny that one is a medic,” she added.”

Knowing how to share the information is another story.

“This is something that affects all of us as physicians — we’re going to be patients at some point, right?” Dr. Winter commented. “But I don’t think how to disclose that is something that was ever brought up in my medical training.”

“Maybe there should just be a discussion of this one day when people are in medical school — maybe in a professionalism course — to broach this topic or look at if there’s any literature on outcomes related to disclosure of status or what are best practices,” she suggested. 

A version of this article first appeared on Medscape.com.

Should doctors seeking healthcare disclose that they are a doctor? 

The question drew spirited debate when urologist Ashley Winter, MD, made a simple, straightforward request on Twitter: “If you are a doctor & you come to an appointment please tell me you are a doctor, not because I will treat you differently but because it’s easier to speak in jargon.”

She later added, “This doesn’t’ mean I would be less patient-focused or emotional with a physician or other [healthcare worker]. Just means that, instead of saying ‘you will have a catheter draining your urine to a bag,’ I can say, ‘you will have a Foley.’ ”

The Tweet followed an encounter with a patient who told Dr. Winter that he was a doctor only after she had gone to some length explaining a surgical procedure in lay terms.

“I explained the surgery, obviously assuming he was an intelligent adult, but using fully layman’s terms,” she said in an interview. The patient then told her that he was a doctor. “I guess I felt this embarrassment — I wouldn’t have treated him differently, but I just could have discussed the procedure with him in more professional terms.” 

“To some extent, it was my own fault,” she commented in an interview. “I didn’t take the time to ask [about his work] at the beginning of the consultation, but that’s a fine line, also,” added Dr. Winter, a urologist and sexual medicine physician in Portland, Ore.

“You know that patient is there because they want care from you and it’s not necessarily always at the forefront of importance to be asking them what they do for their work, but alternatively, if you don’t ask then you put them in this position where they have to find a way to go ahead and tell you.”

Several people chimed in on the thread to voice their thoughts on the matter. Some commiserated with Dr. Winter’s experience:

“I took care of a retired cardiologist in the hospital as a second-year resident and honest to god he let me ramble on ‘explaining’ his echo result and never told me. I found out a couple days later and wanted to die,” posted @MaddyAndrewsMD.

Another recalled a similarly embarrassing experience when she “went on and on” discussing headaches with a patient whose husband “was in the corner smirking.”

“They told my attending later [that the] husband was a retired FM doc who practiced medicine longer than I’ve been alive. I wanted to die,” posted @JSinghDO.

Many on the thread, though, were doctors and other healthcare professionals speaking as patients. Some said they didn’t want to disclose their status as a healthcare provider because they felt it affected the care they received.

For example, @drhelenrainford commented: “In my experience my care is less ‘caring’ when they know I am a [doctor]. I get spoken to like they are discussing a patient with me — no empathy just facts and difficult results just blurted out without consideration. Awful awful time as an inpatient …but that’s another story!”

@Dr_B_Ring said: “Nope – You and I speak different jargon – I would want you to speak to me like a human that doesn’t know your jargon. My ego would get in the way of asking about the acronyms I don’t know if you knew I was a fellow physician.”

Conversely, @lozzlemcfozzle said: “Honestly I prefer not to tell my Doctors — I’ve found people skip explanations assuming I ‘know,’ or seem a little nervous when I tell them!”

Others said they felt uncomfortable — pretentious, even — in announcing their status, or worried that they might come across as expecting special care.

“It’s such a tough needle to thread. Want to tell people early but not come off as demanding special treatment, but don’t want to wait too long and it seems like a trap,” said @MDaware.

Twitter user @MsBabyCatcher wrote: “I have a hard time doing this because I don’t want people to think I’m being pretentious or going to micromanage/dictate care.”

Replying to @MsBabyCatcher, @RedStethoscope wrote: “I used to think this too until I got [very poor] care a few times, and was advised by other doctor moms to ‘play the doctor card.’ I have gotten better/more compassionate care by making sure it’s clear that I’m a physician (which is junk, but here we are).”

Several of those responding used the words “tricky” and “awkward,” suggesting a common theme for doctors presenting as patients.

“I struggle with this. My 5-year-old broke her arm this weekend, we spent hours in the ED, of my own hospital, I never mentioned it because I didn’t want to get preferential care. But as they were explaining her type of fracture, it felt awkward and inefficient,” said @lindsay_petty.

To avoid the awkwardness, a number of respondents said they purposefully use medical jargon to open up a conversation rather than just offering up the information that they are a doctor.

Still others offered suggestions on how to broach the subject more directly when presenting as a patient:

‘”Just FYI I’m a X doc but I’m here because I really want your help and advice!” That’s what I usually do,” wrote @drcakefm.

@BeeSting14618 Tweeted: “I usually say ‘I know some of this but I’m here because I want YOUR guidance. Also I may ask dumb questions, and I’ll tell you if a question is asking your opinion or making a request.’”

A few others injected a bit of humor: “I just do the 14-part handshake that only doctors know. Is that not customary?” quipped @Branmiz25.

“Ah yes, that transmits the entire [history of present illness],” replied Dr. Winter.

Jokes aside, the topic is obviously one that touched on a shared experience among healthcare providers, Dr. Winter commented. The Twitter thread she started just “blew up.”

That’s typically a sign that the Tweet is relatable for a lot of people, she said.

“It’s definitely something that all of us as care providers and as patients understand. It’s a funny, awkward thing that can really change an interaction, so we probably all feel pretty strongly about our experiences related to that,” she added.

The debate begs the question: Is there a duty or ethical reason to disclose?

“I definitely think it is very reasonable to disclose that one is a medical professional to another doctor,” medical ethicist Charlotte Blease, PhD, said in an interview. “There are good reasons to believe doing so might make a difference to the quality of communication and transparency.”

If the ability to use medical terminology or jargon more freely improves patient understanding, autonomy, and shared decision-making, then it may be of benefit, said Dr. Blease, a Keane OpenNotes Scholar at Beth Israel Deaconess Medical Center in Boston.

“Since doctors should strive to communicate effectively with every patient and to respect their unique needs and level of understanding, then I see no reason to deny that one is a medic,” she added.”

Knowing how to share the information is another story.

“This is something that affects all of us as physicians — we’re going to be patients at some point, right?” Dr. Winter commented. “But I don’t think how to disclose that is something that was ever brought up in my medical training.”

“Maybe there should just be a discussion of this one day when people are in medical school — maybe in a professionalism course — to broach this topic or look at if there’s any literature on outcomes related to disclosure of status or what are best practices,” she suggested. 

A version of this article first appeared on Medscape.com.

Should doctors seeking healthcare disclose that they are a doctor? 

The question drew spirited debate when urologist Ashley Winter, MD, made a simple, straightforward request on Twitter: “If you are a doctor & you come to an appointment please tell me you are a doctor, not because I will treat you differently but because it’s easier to speak in jargon.”

She later added, “This doesn’t’ mean I would be less patient-focused or emotional with a physician or other [healthcare worker]. Just means that, instead of saying ‘you will have a catheter draining your urine to a bag,’ I can say, ‘you will have a Foley.’ ”

The Tweet followed an encounter with a patient who told Dr. Winter that he was a doctor only after she had gone to some length explaining a surgical procedure in lay terms.

“I explained the surgery, obviously assuming he was an intelligent adult, but using fully layman’s terms,” she said in an interview. The patient then told her that he was a doctor. “I guess I felt this embarrassment — I wouldn’t have treated him differently, but I just could have discussed the procedure with him in more professional terms.” 

“To some extent, it was my own fault,” she commented in an interview. “I didn’t take the time to ask [about his work] at the beginning of the consultation, but that’s a fine line, also,” added Dr. Winter, a urologist and sexual medicine physician in Portland, Ore.

“You know that patient is there because they want care from you and it’s not necessarily always at the forefront of importance to be asking them what they do for their work, but alternatively, if you don’t ask then you put them in this position where they have to find a way to go ahead and tell you.”

Several people chimed in on the thread to voice their thoughts on the matter. Some commiserated with Dr. Winter’s experience:

“I took care of a retired cardiologist in the hospital as a second-year resident and honest to god he let me ramble on ‘explaining’ his echo result and never told me. I found out a couple days later and wanted to die,” posted @MaddyAndrewsMD.

Another recalled a similarly embarrassing experience when she “went on and on” discussing headaches with a patient whose husband “was in the corner smirking.”

“They told my attending later [that the] husband was a retired FM doc who practiced medicine longer than I’ve been alive. I wanted to die,” posted @JSinghDO.

Many on the thread, though, were doctors and other healthcare professionals speaking as patients. Some said they didn’t want to disclose their status as a healthcare provider because they felt it affected the care they received.

For example, @drhelenrainford commented: “In my experience my care is less ‘caring’ when they know I am a [doctor]. I get spoken to like they are discussing a patient with me — no empathy just facts and difficult results just blurted out without consideration. Awful awful time as an inpatient …but that’s another story!”

@Dr_B_Ring said: “Nope – You and I speak different jargon – I would want you to speak to me like a human that doesn’t know your jargon. My ego would get in the way of asking about the acronyms I don’t know if you knew I was a fellow physician.”

Conversely, @lozzlemcfozzle said: “Honestly I prefer not to tell my Doctors — I’ve found people skip explanations assuming I ‘know,’ or seem a little nervous when I tell them!”

Others said they felt uncomfortable — pretentious, even — in announcing their status, or worried that they might come across as expecting special care.

“It’s such a tough needle to thread. Want to tell people early but not come off as demanding special treatment, but don’t want to wait too long and it seems like a trap,” said @MDaware.

Twitter user @MsBabyCatcher wrote: “I have a hard time doing this because I don’t want people to think I’m being pretentious or going to micromanage/dictate care.”

Replying to @MsBabyCatcher, @RedStethoscope wrote: “I used to think this too until I got [very poor] care a few times, and was advised by other doctor moms to ‘play the doctor card.’ I have gotten better/more compassionate care by making sure it’s clear that I’m a physician (which is junk, but here we are).”

Several of those responding used the words “tricky” and “awkward,” suggesting a common theme for doctors presenting as patients.

“I struggle with this. My 5-year-old broke her arm this weekend, we spent hours in the ED, of my own hospital, I never mentioned it because I didn’t want to get preferential care. But as they were explaining her type of fracture, it felt awkward and inefficient,” said @lindsay_petty.

To avoid the awkwardness, a number of respondents said they purposefully use medical jargon to open up a conversation rather than just offering up the information that they are a doctor.

Still others offered suggestions on how to broach the subject more directly when presenting as a patient:

‘”Just FYI I’m a X doc but I’m here because I really want your help and advice!” That’s what I usually do,” wrote @drcakefm.

@BeeSting14618 Tweeted: “I usually say ‘I know some of this but I’m here because I want YOUR guidance. Also I may ask dumb questions, and I’ll tell you if a question is asking your opinion or making a request.’”

A few others injected a bit of humor: “I just do the 14-part handshake that only doctors know. Is that not customary?” quipped @Branmiz25.

“Ah yes, that transmits the entire [history of present illness],” replied Dr. Winter.

Jokes aside, the topic is obviously one that touched on a shared experience among healthcare providers, Dr. Winter commented. The Twitter thread she started just “blew up.”

That’s typically a sign that the Tweet is relatable for a lot of people, she said.

“It’s definitely something that all of us as care providers and as patients understand. It’s a funny, awkward thing that can really change an interaction, so we probably all feel pretty strongly about our experiences related to that,” she added.

The debate begs the question: Is there a duty or ethical reason to disclose?

“I definitely think it is very reasonable to disclose that one is a medical professional to another doctor,” medical ethicist Charlotte Blease, PhD, said in an interview. “There are good reasons to believe doing so might make a difference to the quality of communication and transparency.”

If the ability to use medical terminology or jargon more freely improves patient understanding, autonomy, and shared decision-making, then it may be of benefit, said Dr. Blease, a Keane OpenNotes Scholar at Beth Israel Deaconess Medical Center in Boston.

“Since doctors should strive to communicate effectively with every patient and to respect their unique needs and level of understanding, then I see no reason to deny that one is a medic,” she added.”

Knowing how to share the information is another story.

“This is something that affects all of us as physicians — we’re going to be patients at some point, right?” Dr. Winter commented. “But I don’t think how to disclose that is something that was ever brought up in my medical training.”

“Maybe there should just be a discussion of this one day when people are in medical school — maybe in a professionalism course — to broach this topic or look at if there’s any literature on outcomes related to disclosure of status or what are best practices,” she suggested. 

A version of this article first appeared on Medscape.com.

Endocrine-disrupting chemicals linked to a variety of health problems are abundant in fast foods sold in the United States, such as chicken nuggets, hamburgers, and cheese pizza, new research suggests.

Digital Vision./Thinkstock

The first-of-its-kind study, which measured concentrations of chemicals such as phthalates in foods and gloves from U.S. fast food chains, is also the first to detect the plasticizer DEHT in fast foods.

“We knew from prior research that fast food consumption is linked to higher levels of phthalates in people’s bodies, but our study was novel because we actually collected these food items from fast food places and measured them,” said study author Lariah Edwards, PhD, a postdoctoral research scientist at the Milken Institute School of Public Health, George Washington University, Washington.

“Our research added an additional piece of information to the puzzle,” Dr. Edwards said in an interview.

A class of chemicals used in food packaging and food processing equipment, phthalates such as DEHP and DnBP, can leach out of these items and interfere with hormone production, Dr. Edwards said. They are linked with a wide variety of reproductive, developmental, brain, and immune effects, as well as with childhood obesity, asthma, cancer, and cardiovascular problems.

Meanwhile, nonphthalate or replacement plasticizers have been used in place of phthalates, some of which have been banned in certain products. But these plasticizers aren’t well studied, Dr. Edwards said, making the detection of DEHT in fast foods particularly concerning.

“There’s very limited research out there to understand the human health effects” of DEHT in food, she said, “so we’re being exposed before we understand what it’s doing to our health. It’s almost like we’re setting ourselves up for a big experiment.”

The study was recently published in the Journal of Exposure Science & Environmental Epidemiology .
 

Fast foods containing meat had highest concentrations of chemicals

Dr. Edwards and colleagues obtained 64 food samples, including hamburgers, fries, chicken nuggets, chicken burritos, and cheese pizza, as well as three pairs of unused gloves from six different fast food restaurants in San Antonio.

Using gas chromatography–mass spectrometry, they analyzed the samples for 11 chemicals, including eight phthalates and three replacement plasticizers.

The researchers detected 10 of the 11 chemicals in fast food samples: 81% of foods contained DnBP (di-n-butyl phthalate), and 70% contained DEHP (di(2-ethylhexyl phthalate)). Meanwhile 86% of samples contained replacement plasticizer DEHT (di(2-ethylhexyl terephthalate)).

Overall, fast food samples containing meat — including chicken nuggets, chicken burritos, and hamburgers — contained higher levels of these chemicals, Dr. Edwards noted.

“We know fast food is not the most nutritious, and now we’re seeing these chemicals in it we shouldn’t be exposed to,” she said.

The results also create implications for health equity, Dr. Edwards said, as Black people in the United States report eating more fast foods than other racial and ethnic groups for many reasons, such as longstanding residential segregation.

Many advocacy groups are pushing for stronger regulations on phthalates in foods, she said, and the study can be used to fuel those efforts.

“We’re hoping our findings help people understand what they’re eating and what’s in food,” Dr. Edwards said. “If they want to reduce exposure to phthalates in fast food, they can choose foods without meat in them. But not everyone has the option of reducing fast food consumption — personal choice is important, but policy is what’s going to protect us.”

Dr. Edwards noted that the research was limited by small sample sizes gathered in one U.S. city. Limitations in extraction methods also meant the researchers were able to detect chemicals in gloves only at high concentrations.

“That being said, I do think our results are fairly generalizable,” she added, “because the way fast foods are prepared at these restaurants is fairly consistent.”

The study was funded by the Passport Foundation, Forsythia Foundation, and Marisla Foundation. Dr. Edwards has reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Endocrine-disrupting chemicals linked to a variety of health problems are abundant in fast foods sold in the United States, such as chicken nuggets, hamburgers, and cheese pizza, new research suggests.

Digital Vision./Thinkstock

The first-of-its-kind study, which measured concentrations of chemicals such as phthalates in foods and gloves from U.S. fast food chains, is also the first to detect the plasticizer DEHT in fast foods.

“We knew from prior research that fast food consumption is linked to higher levels of phthalates in people’s bodies, but our study was novel because we actually collected these food items from fast food places and measured them,” said study author Lariah Edwards, PhD, a postdoctoral research scientist at the Milken Institute School of Public Health, George Washington University, Washington.

“Our research added an additional piece of information to the puzzle,” Dr. Edwards said in an interview.

A class of chemicals used in food packaging and food processing equipment, phthalates such as DEHP and DnBP, can leach out of these items and interfere with hormone production, Dr. Edwards said. They are linked with a wide variety of reproductive, developmental, brain, and immune effects, as well as with childhood obesity, asthma, cancer, and cardiovascular problems.

Meanwhile, nonphthalate or replacement plasticizers have been used in place of phthalates, some of which have been banned in certain products. But these plasticizers aren’t well studied, Dr. Edwards said, making the detection of DEHT in fast foods particularly concerning.

“There’s very limited research out there to understand the human health effects” of DEHT in food, she said, “so we’re being exposed before we understand what it’s doing to our health. It’s almost like we’re setting ourselves up for a big experiment.”

The study was recently published in the Journal of Exposure Science & Environmental Epidemiology .
 

Fast foods containing meat had highest concentrations of chemicals

Dr. Edwards and colleagues obtained 64 food samples, including hamburgers, fries, chicken nuggets, chicken burritos, and cheese pizza, as well as three pairs of unused gloves from six different fast food restaurants in San Antonio.

Using gas chromatography–mass spectrometry, they analyzed the samples for 11 chemicals, including eight phthalates and three replacement plasticizers.

The researchers detected 10 of the 11 chemicals in fast food samples: 81% of foods contained DnBP (di-n-butyl phthalate), and 70% contained DEHP (di(2-ethylhexyl phthalate)). Meanwhile 86% of samples contained replacement plasticizer DEHT (di(2-ethylhexyl terephthalate)).

Overall, fast food samples containing meat — including chicken nuggets, chicken burritos, and hamburgers — contained higher levels of these chemicals, Dr. Edwards noted.

“We know fast food is not the most nutritious, and now we’re seeing these chemicals in it we shouldn’t be exposed to,” she said.

The results also create implications for health equity, Dr. Edwards said, as Black people in the United States report eating more fast foods than other racial and ethnic groups for many reasons, such as longstanding residential segregation.

Many advocacy groups are pushing for stronger regulations on phthalates in foods, she said, and the study can be used to fuel those efforts.

“We’re hoping our findings help people understand what they’re eating and what’s in food,” Dr. Edwards said. “If they want to reduce exposure to phthalates in fast food, they can choose foods without meat in them. But not everyone has the option of reducing fast food consumption — personal choice is important, but policy is what’s going to protect us.”

Dr. Edwards noted that the research was limited by small sample sizes gathered in one U.S. city. Limitations in extraction methods also meant the researchers were able to detect chemicals in gloves only at high concentrations.

“That being said, I do think our results are fairly generalizable,” she added, “because the way fast foods are prepared at these restaurants is fairly consistent.”

The study was funded by the Passport Foundation, Forsythia Foundation, and Marisla Foundation. Dr. Edwards has reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Endocrine-disrupting chemicals linked to a variety of health problems are abundant in fast foods sold in the United States, such as chicken nuggets, hamburgers, and cheese pizza, new research suggests.

Digital Vision./Thinkstock

The first-of-its-kind study, which measured concentrations of chemicals such as phthalates in foods and gloves from U.S. fast food chains, is also the first to detect the plasticizer DEHT in fast foods.

“We knew from prior research that fast food consumption is linked to higher levels of phthalates in people’s bodies, but our study was novel because we actually collected these food items from fast food places and measured them,” said study author Lariah Edwards, PhD, a postdoctoral research scientist at the Milken Institute School of Public Health, George Washington University, Washington.

“Our research added an additional piece of information to the puzzle,” Dr. Edwards said in an interview.

A class of chemicals used in food packaging and food processing equipment, phthalates such as DEHP and DnBP, can leach out of these items and interfere with hormone production, Dr. Edwards said. They are linked with a wide variety of reproductive, developmental, brain, and immune effects, as well as with childhood obesity, asthma, cancer, and cardiovascular problems.

Meanwhile, nonphthalate or replacement plasticizers have been used in place of phthalates, some of which have been banned in certain products. But these plasticizers aren’t well studied, Dr. Edwards said, making the detection of DEHT in fast foods particularly concerning.

“There’s very limited research out there to understand the human health effects” of DEHT in food, she said, “so we’re being exposed before we understand what it’s doing to our health. It’s almost like we’re setting ourselves up for a big experiment.”

The study was recently published in the Journal of Exposure Science & Environmental Epidemiology .
 

Fast foods containing meat had highest concentrations of chemicals

Dr. Edwards and colleagues obtained 64 food samples, including hamburgers, fries, chicken nuggets, chicken burritos, and cheese pizza, as well as three pairs of unused gloves from six different fast food restaurants in San Antonio.

Using gas chromatography–mass spectrometry, they analyzed the samples for 11 chemicals, including eight phthalates and three replacement plasticizers.

The researchers detected 10 of the 11 chemicals in fast food samples: 81% of foods contained DnBP (di-n-butyl phthalate), and 70% contained DEHP (di(2-ethylhexyl phthalate)). Meanwhile 86% of samples contained replacement plasticizer DEHT (di(2-ethylhexyl terephthalate)).

Overall, fast food samples containing meat — including chicken nuggets, chicken burritos, and hamburgers — contained higher levels of these chemicals, Dr. Edwards noted.

“We know fast food is not the most nutritious, and now we’re seeing these chemicals in it we shouldn’t be exposed to,” she said.

The results also create implications for health equity, Dr. Edwards said, as Black people in the United States report eating more fast foods than other racial and ethnic groups for many reasons, such as longstanding residential segregation.

Many advocacy groups are pushing for stronger regulations on phthalates in foods, she said, and the study can be used to fuel those efforts.

“We’re hoping our findings help people understand what they’re eating and what’s in food,” Dr. Edwards said. “If they want to reduce exposure to phthalates in fast food, they can choose foods without meat in them. But not everyone has the option of reducing fast food consumption — personal choice is important, but policy is what’s going to protect us.”

Dr. Edwards noted that the research was limited by small sample sizes gathered in one U.S. city. Limitations in extraction methods also meant the researchers were able to detect chemicals in gloves only at high concentrations.

“That being said, I do think our results are fairly generalizable,” she added, “because the way fast foods are prepared at these restaurants is fairly consistent.”

The study was funded by the Passport Foundation, Forsythia Foundation, and Marisla Foundation. Dr. Edwards has reported no relevant financial relationships.

A version of this article first appeared on Medscape.com.

During the month of September, Texans who weren’t vaccinated against COVID-19 were 20 times more likely to die from COVID-19 and related complications than those who were fully vaccinated, according to a new study from the Texas Department of State Health Services.

The data also showed that unvaccinated people were 13 times more likely to test positive for COVID-19 than people who were fully vaccinated.

“This analysis quantifies what we’ve known for months,” Jennifer Shuford, MD, the state’s chief epidemiologist, told The Dallas Morning News.

“The COVID-19 vaccines are doing an excellent job of protecting people from getting sick and from dying from COVID-19,” she said. “Vaccination remains the best way to keep yourself and the people close to you safe from this deadly disease.”

As part of the study, researchers analyzed electronic lab reports, death certificates, and state immunization records, with a particular focus on September when the contagious Delta variant surged across Texas. The research marks the state’s first statistical analysis of COVID-19 vaccinations in Texas and the effects, the newspaper reported.

The protective effect of vaccination was most noticeable among younger groups. During September, the risk of COVID-19 death was 23 times higher in unvaccinated people in their 30s and 55 times higher for unvaccinated people in their 40s.

In addition, there were fewer than 10 COVID-19 deaths in September among fully vaccinated people between ages 18-29, as compared with 339 deaths among unvaccinated people in the same age group.

Then, looking at a longer time period -- from Jan. 15 to Oct. 1 -- the researchers found that unvaccinated people were 45 times more likely to contract COVID-19 than fully vaccinated people. The protective effect of vaccination against infection was strong across all adult age groups but greatest among ages 12-17.

“All authorized COVID-19 vaccines in the United States are highly effective at protecting people from getting sick or severely ill with COVID-19, including those infected with Delta and other known variants,” the study authors wrote. “Real world data from Texas clearly shows these benefits.”

About 15.6 million people in Texas have been fully vaccinated against COVID-19 in a state of about 29 million residents, according to state data. About 66% of the population has received at least one dose, while 58% is fully vaccinated.

A version of this article first appeared on WebMD.com.

During the month of September, Texans who weren’t vaccinated against COVID-19 were 20 times more likely to die from COVID-19 and related complications than those who were fully vaccinated, according to a new study from the Texas Department of State Health Services.

The data also showed that unvaccinated people were 13 times more likely to test positive for COVID-19 than people who were fully vaccinated.

“This analysis quantifies what we’ve known for months,” Jennifer Shuford, MD, the state’s chief epidemiologist, told The Dallas Morning News.

“The COVID-19 vaccines are doing an excellent job of protecting people from getting sick and from dying from COVID-19,” she said. “Vaccination remains the best way to keep yourself and the people close to you safe from this deadly disease.”

As part of the study, researchers analyzed electronic lab reports, death certificates, and state immunization records, with a particular focus on September when the contagious Delta variant surged across Texas. The research marks the state’s first statistical analysis of COVID-19 vaccinations in Texas and the effects, the newspaper reported.

The protective effect of vaccination was most noticeable among younger groups. During September, the risk of COVID-19 death was 23 times higher in unvaccinated people in their 30s and 55 times higher for unvaccinated people in their 40s.

In addition, there were fewer than 10 COVID-19 deaths in September among fully vaccinated people between ages 18-29, as compared with 339 deaths among unvaccinated people in the same age group.

Then, looking at a longer time period -- from Jan. 15 to Oct. 1 -- the researchers found that unvaccinated people were 45 times more likely to contract COVID-19 than fully vaccinated people. The protective effect of vaccination against infection was strong across all adult age groups but greatest among ages 12-17.

“All authorized COVID-19 vaccines in the United States are highly effective at protecting people from getting sick or severely ill with COVID-19, including those infected with Delta and other known variants,” the study authors wrote. “Real world data from Texas clearly shows these benefits.”

About 15.6 million people in Texas have been fully vaccinated against COVID-19 in a state of about 29 million residents, according to state data. About 66% of the population has received at least one dose, while 58% is fully vaccinated.

A version of this article first appeared on WebMD.com.

During the month of September, Texans who weren’t vaccinated against COVID-19 were 20 times more likely to die from COVID-19 and related complications than those who were fully vaccinated, according to a new study from the Texas Department of State Health Services.

The data also showed that unvaccinated people were 13 times more likely to test positive for COVID-19 than people who were fully vaccinated.

“This analysis quantifies what we’ve known for months,” Jennifer Shuford, MD, the state’s chief epidemiologist, told The Dallas Morning News.

“The COVID-19 vaccines are doing an excellent job of protecting people from getting sick and from dying from COVID-19,” she said. “Vaccination remains the best way to keep yourself and the people close to you safe from this deadly disease.”

As part of the study, researchers analyzed electronic lab reports, death certificates, and state immunization records, with a particular focus on September when the contagious Delta variant surged across Texas. The research marks the state’s first statistical analysis of COVID-19 vaccinations in Texas and the effects, the newspaper reported.

The protective effect of vaccination was most noticeable among younger groups. During September, the risk of COVID-19 death was 23 times higher in unvaccinated people in their 30s and 55 times higher for unvaccinated people in their 40s.

In addition, there were fewer than 10 COVID-19 deaths in September among fully vaccinated people between ages 18-29, as compared with 339 deaths among unvaccinated people in the same age group.

Then, looking at a longer time period -- from Jan. 15 to Oct. 1 -- the researchers found that unvaccinated people were 45 times more likely to contract COVID-19 than fully vaccinated people. The protective effect of vaccination against infection was strong across all adult age groups but greatest among ages 12-17.

“All authorized COVID-19 vaccines in the United States are highly effective at protecting people from getting sick or severely ill with COVID-19, including those infected with Delta and other known variants,” the study authors wrote. “Real world data from Texas clearly shows these benefits.”

About 15.6 million people in Texas have been fully vaccinated against COVID-19 in a state of about 29 million residents, according to state data. About 66% of the population has received at least one dose, while 58% is fully vaccinated.

A version of this article first appeared on WebMD.com.

Initiating treatment of polyarticular juvenile idiopathic arthritis (polyJIA) with both a conventional synthetic disease-modifying antirheumatic drug and a biologic DMARD resulted in more patients achieving clinical inactive disease 2 years later than did starting with only a csDMARD and stepping up to a biologic, according to data presented at the virtual annual meeting of the American College of Rheumatology.

“The 24-month results support the 12-month primary results that suggested that the early-combination group was superior and that, at 24 months, more early combination CTP [consensus treatment plan] patients achieve CID [clinical inactive disease], compared to step up,” Yukiko Kimura, MD, division chief of pediatric rheumatology at HMH Hackensack (N.J.) University Medical Center, told attendees. “This suggests that starting biologics early in polyJIA may lead to better long-term outcomes in many patients.”

Dr. Kimura noted that polyarticular JIA patients are already at risk for poor outcomes, and initial therapy can especially impact outcomes. Further, little evidence exists to suggest when the best time is to start biologics, a gap this study aimed to address.

Diane Brown, MD, PhD, a pediatric rheumatologist at Children’s Hospital Los Angeles who was not involved in the study, was pleased to see the results, which she said support her own preferences and practice patterns.

“Starting sooner with combination therapy, taking advantage of the advances with biologics and our long history with methotrexate at the same time, gives better outcomes for the long run,” Dr. Brown said in an interview. “Having studies like this to back up my own recommendations can be very powerful when talking to families, and it is absolutely invaluable when battling with insurance companies who always want you to take the cheapest road.”
 

Study details

The findings were an update of 12-month results in the CARRA STOP-JIA study that enrolled 400 untreated patients with polyJIA and compared three Childhood Arthritis and Rheumatology Research Alliance (CARRA) CTPs. Overall, 49.5% of participants received biologics within 3 months of starting the study. For these updated results, 275 participants had complete data at 24 months for the three CTPs:

• A step-up group of 177 patients who started therapy with a csDMARD and added a biologic if needed at least 3 months later
• An early-combination group of 73 patients who started therapy with a csDMARD and biologic together
• A biologic-first group of 25 patients who started with biologic monotherapy, adding a csDMARD only if needed at least 3 months later.

The primary outcome was the percentage of participants who reached CID without taking glucocorticoids at 24 months. Since the participants were not randomized, the researchers made adjustments to account for baseline differences between the groups, including differences in JIA categories, number of active joints, physician global assessment of disease activity, and the clinical Juvenile Arthritis Disease Activity Score based on 10 joints (cJADAS10).

At 24 months in an intention to treat analysis, 59.4% of the early-combination group had achieved CID, compared with 48% of the biologic-first group and 40.1% of the step-up group (P = .009 for early combination vs. step up). All three groups had improved since the 12-month time point, when 37% of the early-combination group, 24% of the biologic-first group, and 32% of the step-up group had reached CID.

There were no significant differences between the groups in secondary outcomes of achieving cJADAS10 inactive disease of 2.5 or less or 70% improvement in pediatric ACR response criteria at 24 months. All groups improved in PROMIS pain interference or mobility measures from baseline. Most of the 17 severe adverse events were infections.
 

Moving from step-up therapy to early-combination treatment

Dr. Brown said that she spent many years in her practice using the step-up therapy because it was difficult to get insurance companies to pay for biologics without first showing that methotrexate was insufficient.

”But methotrexate takes so long to control the disease that you need a lot of steroids, with all of their side effects, at least temporarily, or you must simply accept a longer period of active and symptomatic disease before you get to that desired state of clinically inactive disease,” Dr. Brown said. “And during that time, you can be accumulating what may be permanent damage to joints, as well as increase in risk of contractures and deconditioning for that child who is too uncomfortable to move and exercise and play normally.”

Dr. Brown is also wary of using a biologic as an initial therapy by itself because the actions of biologics are so specific. ”I like to back up the powerful, rapid, and specific actions of a biologic with the broader, if slower, action of methotrexate to minimize chances that the immune system is going to find a way around blockade of a single cytokine by your biologic,” she said.

While patient preference will also play a role in what CTP patients with polyJIA start with, Dr. Brown said that she believes more medication upfront can result in less medication and better outcomes in the long run, as the findings of this study suggest. The results here are helpful when speaking with families who are anxious about “so much medicine” or “such powerful medicines,” she said. ”I hope it will also help ease the fears of other providers who share the same concerns about ‘so much medicine.’ ”

The study’s biggest limitation is not being a randomized, controlled trial, but Dr. Brown said the researchers demonstrated effectively that the disease burden remains similar across the groups at baseline.

”It would also be useful to have a clear breakdown of adverse events and opportunistic infections because an excess of opportunistic infections would be a key concern with early combination therapy,” she said, although she added that the study overall was a ”beautiful example of the value of registry data.”

Dr. Kimura emphasized that polyJIA remains a challenging disease to treat, with 40%-60% of participants not reaching CID at 24 months. The registry follow-up will continue for up to 10 years to hopefully provide more information about longer-term outcomes from different treatments.

The research was funded by a grant from Genentech to CARRA. Dr. Kimura reported royalties from UpToDate and salary support from CARRA. Dr. Brown had no disclosures.

Initiating treatment of polyarticular juvenile idiopathic arthritis (polyJIA) with both a conventional synthetic disease-modifying antirheumatic drug and a biologic DMARD resulted in more patients achieving clinical inactive disease 2 years later than did starting with only a csDMARD and stepping up to a biologic, according to data presented at the virtual annual meeting of the American College of Rheumatology.

“The 24-month results support the 12-month primary results that suggested that the early-combination group was superior and that, at 24 months, more early combination CTP [consensus treatment plan] patients achieve CID [clinical inactive disease], compared to step up,” Yukiko Kimura, MD, division chief of pediatric rheumatology at HMH Hackensack (N.J.) University Medical Center, told attendees. “This suggests that starting biologics early in polyJIA may lead to better long-term outcomes in many patients.”

Dr. Kimura noted that polyarticular JIA patients are already at risk for poor outcomes, and initial therapy can especially impact outcomes. Further, little evidence exists to suggest when the best time is to start biologics, a gap this study aimed to address.

Diane Brown, MD, PhD, a pediatric rheumatologist at Children’s Hospital Los Angeles who was not involved in the study, was pleased to see the results, which she said support her own preferences and practice patterns.

“Starting sooner with combination therapy, taking advantage of the advances with biologics and our long history with methotrexate at the same time, gives better outcomes for the long run,” Dr. Brown said in an interview. “Having studies like this to back up my own recommendations can be very powerful when talking to families, and it is absolutely invaluable when battling with insurance companies who always want you to take the cheapest road.”
 

Study details

The findings were an update of 12-month results in the CARRA STOP-JIA study that enrolled 400 untreated patients with polyJIA and compared three Childhood Arthritis and Rheumatology Research Alliance (CARRA) CTPs. Overall, 49.5% of participants received biologics within 3 months of starting the study. For these updated results, 275 participants had complete data at 24 months for the three CTPs:

• A step-up group of 177 patients who started therapy with a csDMARD and added a biologic if needed at least 3 months later
• An early-combination group of 73 patients who started therapy with a csDMARD and biologic together
• A biologic-first group of 25 patients who started with biologic monotherapy, adding a csDMARD only if needed at least 3 months later.

The primary outcome was the percentage of participants who reached CID without taking glucocorticoids at 24 months. Since the participants were not randomized, the researchers made adjustments to account for baseline differences between the groups, including differences in JIA categories, number of active joints, physician global assessment of disease activity, and the clinical Juvenile Arthritis Disease Activity Score based on 10 joints (cJADAS10).

At 24 months in an intention to treat analysis, 59.4% of the early-combination group had achieved CID, compared with 48% of the biologic-first group and 40.1% of the step-up group (P = .009 for early combination vs. step up). All three groups had improved since the 12-month time point, when 37% of the early-combination group, 24% of the biologic-first group, and 32% of the step-up group had reached CID.

There were no significant differences between the groups in secondary outcomes of achieving cJADAS10 inactive disease of 2.5 or less or 70% improvement in pediatric ACR response criteria at 24 months. All groups improved in PROMIS pain interference or mobility measures from baseline. Most of the 17 severe adverse events were infections.
 

Moving from step-up therapy to early-combination treatment

Dr. Brown said that she spent many years in her practice using the step-up therapy because it was difficult to get insurance companies to pay for biologics without first showing that methotrexate was insufficient.

”But methotrexate takes so long to control the disease that you need a lot of steroids, with all of their side effects, at least temporarily, or you must simply accept a longer period of active and symptomatic disease before you get to that desired state of clinically inactive disease,” Dr. Brown said. “And during that time, you can be accumulating what may be permanent damage to joints, as well as increase in risk of contractures and deconditioning for that child who is too uncomfortable to move and exercise and play normally.”

Dr. Brown is also wary of using a biologic as an initial therapy by itself because the actions of biologics are so specific. ”I like to back up the powerful, rapid, and specific actions of a biologic with the broader, if slower, action of methotrexate to minimize chances that the immune system is going to find a way around blockade of a single cytokine by your biologic,” she said.

While patient preference will also play a role in what CTP patients with polyJIA start with, Dr. Brown said that she believes more medication upfront can result in less medication and better outcomes in the long run, as the findings of this study suggest. The results here are helpful when speaking with families who are anxious about “so much medicine” or “such powerful medicines,” she said. ”I hope it will also help ease the fears of other providers who share the same concerns about ‘so much medicine.’ ”

The study’s biggest limitation is not being a randomized, controlled trial, but Dr. Brown said the researchers demonstrated effectively that the disease burden remains similar across the groups at baseline.

”It would also be useful to have a clear breakdown of adverse events and opportunistic infections because an excess of opportunistic infections would be a key concern with early combination therapy,” she said, although she added that the study overall was a ”beautiful example of the value of registry data.”

Dr. Kimura emphasized that polyJIA remains a challenging disease to treat, with 40%-60% of participants not reaching CID at 24 months. The registry follow-up will continue for up to 10 years to hopefully provide more information about longer-term outcomes from different treatments.

The research was funded by a grant from Genentech to CARRA. Dr. Kimura reported royalties from UpToDate and salary support from CARRA. Dr. Brown had no disclosures.

Initiating treatment of polyarticular juvenile idiopathic arthritis (polyJIA) with both a conventional synthetic disease-modifying antirheumatic drug and a biologic DMARD resulted in more patients achieving clinical inactive disease 2 years later than did starting with only a csDMARD and stepping up to a biologic, according to data presented at the virtual annual meeting of the American College of Rheumatology.

“The 24-month results support the 12-month primary results that suggested that the early-combination group was superior and that, at 24 months, more early combination CTP [consensus treatment plan] patients achieve CID [clinical inactive disease], compared to step up,” Yukiko Kimura, MD, division chief of pediatric rheumatology at HMH Hackensack (N.J.) University Medical Center, told attendees. “This suggests that starting biologics early in polyJIA may lead to better long-term outcomes in many patients.”

Dr. Kimura noted that polyarticular JIA patients are already at risk for poor outcomes, and initial therapy can especially impact outcomes. Further, little evidence exists to suggest when the best time is to start biologics, a gap this study aimed to address.

Diane Brown, MD, PhD, a pediatric rheumatologist at Children’s Hospital Los Angeles who was not involved in the study, was pleased to see the results, which she said support her own preferences and practice patterns.

“Starting sooner with combination therapy, taking advantage of the advances with biologics and our long history with methotrexate at the same time, gives better outcomes for the long run,” Dr. Brown said in an interview. “Having studies like this to back up my own recommendations can be very powerful when talking to families, and it is absolutely invaluable when battling with insurance companies who always want you to take the cheapest road.”
 

Study details

The findings were an update of 12-month results in the CARRA STOP-JIA study that enrolled 400 untreated patients with polyJIA and compared three Childhood Arthritis and Rheumatology Research Alliance (CARRA) CTPs. Overall, 49.5% of participants received biologics within 3 months of starting the study. For these updated results, 275 participants had complete data at 24 months for the three CTPs:

• A step-up group of 177 patients who started therapy with a csDMARD and added a biologic if needed at least 3 months later
• An early-combination group of 73 patients who started therapy with a csDMARD and biologic together
• A biologic-first group of 25 patients who started with biologic monotherapy, adding a csDMARD only if needed at least 3 months later.

The primary outcome was the percentage of participants who reached CID without taking glucocorticoids at 24 months. Since the participants were not randomized, the researchers made adjustments to account for baseline differences between the groups, including differences in JIA categories, number of active joints, physician global assessment of disease activity, and the clinical Juvenile Arthritis Disease Activity Score based on 10 joints (cJADAS10).

At 24 months in an intention to treat analysis, 59.4% of the early-combination group had achieved CID, compared with 48% of the biologic-first group and 40.1% of the step-up group (P = .009 for early combination vs. step up). All three groups had improved since the 12-month time point, when 37% of the early-combination group, 24% of the biologic-first group, and 32% of the step-up group had reached CID.

There were no significant differences between the groups in secondary outcomes of achieving cJADAS10 inactive disease of 2.5 or less or 70% improvement in pediatric ACR response criteria at 24 months. All groups improved in PROMIS pain interference or mobility measures from baseline. Most of the 17 severe adverse events were infections.
 

Moving from step-up therapy to early-combination treatment

Dr. Brown said that she spent many years in her practice using the step-up therapy because it was difficult to get insurance companies to pay for biologics without first showing that methotrexate was insufficient.

”But methotrexate takes so long to control the disease that you need a lot of steroids, with all of their side effects, at least temporarily, or you must simply accept a longer period of active and symptomatic disease before you get to that desired state of clinically inactive disease,” Dr. Brown said. “And during that time, you can be accumulating what may be permanent damage to joints, as well as increase in risk of contractures and deconditioning for that child who is too uncomfortable to move and exercise and play normally.”

Dr. Brown is also wary of using a biologic as an initial therapy by itself because the actions of biologics are so specific. ”I like to back up the powerful, rapid, and specific actions of a biologic with the broader, if slower, action of methotrexate to minimize chances that the immune system is going to find a way around blockade of a single cytokine by your biologic,” she said.

While patient preference will also play a role in what CTP patients with polyJIA start with, Dr. Brown said that she believes more medication upfront can result in less medication and better outcomes in the long run, as the findings of this study suggest. The results here are helpful when speaking with families who are anxious about “so much medicine” or “such powerful medicines,” she said. ”I hope it will also help ease the fears of other providers who share the same concerns about ‘so much medicine.’ ”

The study’s biggest limitation is not being a randomized, controlled trial, but Dr. Brown said the researchers demonstrated effectively that the disease burden remains similar across the groups at baseline.

”It would also be useful to have a clear breakdown of adverse events and opportunistic infections because an excess of opportunistic infections would be a key concern with early combination therapy,” she said, although she added that the study overall was a ”beautiful example of the value of registry data.”

Dr. Kimura emphasized that polyJIA remains a challenging disease to treat, with 40%-60% of participants not reaching CID at 24 months. The registry follow-up will continue for up to 10 years to hopefully provide more information about longer-term outcomes from different treatments.

The research was funded by a grant from Genentech to CARRA. Dr. Kimura reported royalties from UpToDate and salary support from CARRA. Dr. Brown had no disclosures.

A number of late-phase clinical trials in lymphoma and multiple myeloma (MM) have opened in recent months. Maybe one of your patients could benefit from being enrolled? 

Untreated peripheral T-cell lymphoma 

Adult patients with peripheral T-cell lymphoma who have received no therapy except corticosteroids are invited to join a phase 2 study testing duvelisib (Copiktra) added to usual chemotherapy. Duvelisib is currently used in relapsed/refractory patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or follicular lymphoma; this study explores first-line use in a different type of lymphoma, so it may be a potential new indication for the drug. All participants will receive a 5-month chemotherapy regimen of cyclophosphamide, doxorubicin (Adriamycin, Rubex), vincristine (Oncovin), prednisone, and etoposide (VePesid). One group will also take oral azacitidine (Vidaza) while the third (experimental) group has oral duvelisib. The primary outcome is complete remission rate; overall survival (OS) is a secondary outcome. Quality of life (QoL) is not measured apart from mood and fatigue. The study opened at Memorial Sloan Kettering Cancer Center on July 30 for up to 170 participants. 

Untreated CLL/SLL 

Patients with CLL/SLL, no 17p deletions, and no prior systemic therapy can join a phase 3 study of pirtobrutinib, an investigational oral tyrosine-kinase inhibitor. Pirtobrutinib targets Bruton's tyrosine kinase, an intracellular signaler that is crucial to the proliferation and survival of leukemic cells. The trial will involve treatment for up to 5 years, with either oral pirtobrutinib or a standard combination of intravenous bendamustine (Treakisym, Treanda, Ribomustin) and rituximab (Ruxience, Riabni, Truxima, Rituxan, MabThera). Investigators at the study site, the California Research Institute in Los Angeles, started recruiting on Sept. 23 hoping for 250 participants. Progression-free survival is the primary outcome, OS is a secondary measure, and QoL will not be tracked.  

Relapsed or refractory follicular lymphoma after one line of therapy

 Adult patients who have CD20-positive follicular lymphoma (grades 1-3A) who have received at least one prior systemic lymphoma therapy can join a phase 3 trial of investigational drug mosunetuzumab combined with lenalidomide (Revlimid, Linamide). Participants in the mosunetuzumab group will be treated with the drug combo for approximately 1 year then followed for 8 years. People in the comparator group will receive a rituximab-lenalidomide combination instead. The trial planned to start enrolling on Oct. 31, looking for a total of 400 people in 144 study locations worldwide, including in nine U.S. states. The primary outcome is progression-free survival. OS is a secondary outcome and, apart from fatigue, QoL parameters will not be assessed.  

Relapsed or refractory follicular lymphoma after two lines of therapy 

Adults with follicular lymphoma (grades 1-3A) despite two or more treatment regimens, including at least one anti-CD20 therapy, are eligible for a phase 2 study of loncastuximab tesirine (Zynlonta). The drug already has an FDA accelerated approval this year for a different lymphoma, relapsed/refractory large B-cell lymphoma, so this could be a new indication. In this trial, it will be compared with idelalisib (Zydelig), which is already approved for follicular lymphoma. Participants will get either an infusion of loncastuximab every 3 weeks or a twice-daily tablet of idelalisib for up to 30 months. Investigators started recruiting on Oct. 30 and hope for 150 participants in Nevada and New Jersey. Complete response rate is the primary outcome. OS and QoL are secondary outcome measures. 

Untreated multiple myeloma not eligible for autologous stem-cell transplant (ASCT) 

Adults with untreated multiple myeloma who are not eligible for stem-cell transplantation are sought for a phase 2 study testing the performance of selinexor (Xpovio) plus dexamethasone. (Prior treatment with emergency steroids and radiation therapy is allowed.) Selinexor plus dexamethasone was approved in 2019 for multiple myeloma after four prior therapies; the goal of this study is to assess its performance as frontline treatment. Participants will receive oral selinexor and dexamethasone for up to 3 years in addition to subcutaneous daratumumab (Darzalex) and capsules of lenalidomide. The study opened Sept. 10, aiming for 100 participants at sites in Arizona, Colorado, Maryland, New York, Oregon, Texas, and Virginia. OS is a secondary outcome measure; QoL will not be assessed.  

Newly diagnosed multiple myeloma where ASCT not planned 

Patients with newly diagnosed multiple myeloma who are not having ASCT as initial therapy are eligible for a phase 3 study of the investigational CAR T-cell therapy ciltacabtagene autoleucel (cilta-cel). This product targets B-cell maturation antigen (BCMA), which is expressed on the surface of mature B lymphocytes and malignant plasma cells; it is in late-stage clinical trials for multiple myeloma but has not yet been approved. In this study, the control-group participants will receive standard therapy for up to approximately 4 years - a regimen of bortezomib (Velcade), lenalidomide, and dexamethasone. Patients destined for cilta-cel will undergo apheresis to garner their T cells, which will then be genetically engineered to express the synthetic antigen receptor, duplicated, and re-infused. During the 6-month wait between apheresis and the cilta-cel infusion, the CAR T patients will receive similar treatment to the control group. Recruitment started for 650 patients across 12 U.S. states and 24 countries on August 19. The primary outcome is progression-free survival. OS and QoL are secondary measures and will be tracked for approximately 12 years. 

All trial information is from the National Institutes of Health U.S. National Library of Medicine.

A version of this article first appeared on Medscape.com

A number of late-phase clinical trials in lymphoma and multiple myeloma (MM) have opened in recent months. Maybe one of your patients could benefit from being enrolled? 

Untreated peripheral T-cell lymphoma 

Adult patients with peripheral T-cell lymphoma who have received no therapy except corticosteroids are invited to join a phase 2 study testing duvelisib (Copiktra) added to usual chemotherapy. Duvelisib is currently used in relapsed/refractory patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or follicular lymphoma; this study explores first-line use in a different type of lymphoma, so it may be a potential new indication for the drug. All participants will receive a 5-month chemotherapy regimen of cyclophosphamide, doxorubicin (Adriamycin, Rubex), vincristine (Oncovin), prednisone, and etoposide (VePesid). One group will also take oral azacitidine (Vidaza) while the third (experimental) group has oral duvelisib. The primary outcome is complete remission rate; overall survival (OS) is a secondary outcome. Quality of life (QoL) is not measured apart from mood and fatigue. The study opened at Memorial Sloan Kettering Cancer Center on July 30 for up to 170 participants. 

Untreated CLL/SLL 

Patients with CLL/SLL, no 17p deletions, and no prior systemic therapy can join a phase 3 study of pirtobrutinib, an investigational oral tyrosine-kinase inhibitor. Pirtobrutinib targets Bruton's tyrosine kinase, an intracellular signaler that is crucial to the proliferation and survival of leukemic cells. The trial will involve treatment for up to 5 years, with either oral pirtobrutinib or a standard combination of intravenous bendamustine (Treakisym, Treanda, Ribomustin) and rituximab (Ruxience, Riabni, Truxima, Rituxan, MabThera). Investigators at the study site, the California Research Institute in Los Angeles, started recruiting on Sept. 23 hoping for 250 participants. Progression-free survival is the primary outcome, OS is a secondary measure, and QoL will not be tracked.  

Relapsed or refractory follicular lymphoma after one line of therapy

 Adult patients who have CD20-positive follicular lymphoma (grades 1-3A) who have received at least one prior systemic lymphoma therapy can join a phase 3 trial of investigational drug mosunetuzumab combined with lenalidomide (Revlimid, Linamide). Participants in the mosunetuzumab group will be treated with the drug combo for approximately 1 year then followed for 8 years. People in the comparator group will receive a rituximab-lenalidomide combination instead. The trial planned to start enrolling on Oct. 31, looking for a total of 400 people in 144 study locations worldwide, including in nine U.S. states. The primary outcome is progression-free survival. OS is a secondary outcome and, apart from fatigue, QoL parameters will not be assessed.  

Relapsed or refractory follicular lymphoma after two lines of therapy 

Adults with follicular lymphoma (grades 1-3A) despite two or more treatment regimens, including at least one anti-CD20 therapy, are eligible for a phase 2 study of loncastuximab tesirine (Zynlonta). The drug already has an FDA accelerated approval this year for a different lymphoma, relapsed/refractory large B-cell lymphoma, so this could be a new indication. In this trial, it will be compared with idelalisib (Zydelig), which is already approved for follicular lymphoma. Participants will get either an infusion of loncastuximab every 3 weeks or a twice-daily tablet of idelalisib for up to 30 months. Investigators started recruiting on Oct. 30 and hope for 150 participants in Nevada and New Jersey. Complete response rate is the primary outcome. OS and QoL are secondary outcome measures. 

Untreated multiple myeloma not eligible for autologous stem-cell transplant (ASCT) 

Adults with untreated multiple myeloma who are not eligible for stem-cell transplantation are sought for a phase 2 study testing the performance of selinexor (Xpovio) plus dexamethasone. (Prior treatment with emergency steroids and radiation therapy is allowed.) Selinexor plus dexamethasone was approved in 2019 for multiple myeloma after four prior therapies; the goal of this study is to assess its performance as frontline treatment. Participants will receive oral selinexor and dexamethasone for up to 3 years in addition to subcutaneous daratumumab (Darzalex) and capsules of lenalidomide. The study opened Sept. 10, aiming for 100 participants at sites in Arizona, Colorado, Maryland, New York, Oregon, Texas, and Virginia. OS is a secondary outcome measure; QoL will not be assessed.  

Newly diagnosed multiple myeloma where ASCT not planned 

Patients with newly diagnosed multiple myeloma who are not having ASCT as initial therapy are eligible for a phase 3 study of the investigational CAR T-cell therapy ciltacabtagene autoleucel (cilta-cel). This product targets B-cell maturation antigen (BCMA), which is expressed on the surface of mature B lymphocytes and malignant plasma cells; it is in late-stage clinical trials for multiple myeloma but has not yet been approved. In this study, the control-group participants will receive standard therapy for up to approximately 4 years - a regimen of bortezomib (Velcade), lenalidomide, and dexamethasone. Patients destined for cilta-cel will undergo apheresis to garner their T cells, which will then be genetically engineered to express the synthetic antigen receptor, duplicated, and re-infused. During the 6-month wait between apheresis and the cilta-cel infusion, the CAR T patients will receive similar treatment to the control group. Recruitment started for 650 patients across 12 U.S. states and 24 countries on August 19. The primary outcome is progression-free survival. OS and QoL are secondary measures and will be tracked for approximately 12 years. 

All trial information is from the National Institutes of Health U.S. National Library of Medicine.

A version of this article first appeared on Medscape.com

A number of late-phase clinical trials in lymphoma and multiple myeloma (MM) have opened in recent months. Maybe one of your patients could benefit from being enrolled? 

Untreated peripheral T-cell lymphoma 

Adult patients with peripheral T-cell lymphoma who have received no therapy except corticosteroids are invited to join a phase 2 study testing duvelisib (Copiktra) added to usual chemotherapy. Duvelisib is currently used in relapsed/refractory patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) or follicular lymphoma; this study explores first-line use in a different type of lymphoma, so it may be a potential new indication for the drug. All participants will receive a 5-month chemotherapy regimen of cyclophosphamide, doxorubicin (Adriamycin, Rubex), vincristine (Oncovin), prednisone, and etoposide (VePesid). One group will also take oral azacitidine (Vidaza) while the third (experimental) group has oral duvelisib. The primary outcome is complete remission rate; overall survival (OS) is a secondary outcome. Quality of life (QoL) is not measured apart from mood and fatigue. The study opened at Memorial Sloan Kettering Cancer Center on July 30 for up to 170 participants. 

Untreated CLL/SLL 

Patients with CLL/SLL, no 17p deletions, and no prior systemic therapy can join a phase 3 study of pirtobrutinib, an investigational oral tyrosine-kinase inhibitor. Pirtobrutinib targets Bruton's tyrosine kinase, an intracellular signaler that is crucial to the proliferation and survival of leukemic cells. The trial will involve treatment for up to 5 years, with either oral pirtobrutinib or a standard combination of intravenous bendamustine (Treakisym, Treanda, Ribomustin) and rituximab (Ruxience, Riabni, Truxima, Rituxan, MabThera). Investigators at the study site, the California Research Institute in Los Angeles, started recruiting on Sept. 23 hoping for 250 participants. Progression-free survival is the primary outcome, OS is a secondary measure, and QoL will not be tracked.  

Relapsed or refractory follicular lymphoma after one line of therapy

 Adult patients who have CD20-positive follicular lymphoma (grades 1-3A) who have received at least one prior systemic lymphoma therapy can join a phase 3 trial of investigational drug mosunetuzumab combined with lenalidomide (Revlimid, Linamide). Participants in the mosunetuzumab group will be treated with the drug combo for approximately 1 year then followed for 8 years. People in the comparator group will receive a rituximab-lenalidomide combination instead. The trial planned to start enrolling on Oct. 31, looking for a total of 400 people in 144 study locations worldwide, including in nine U.S. states. The primary outcome is progression-free survival. OS is a secondary outcome and, apart from fatigue, QoL parameters will not be assessed.  

Relapsed or refractory follicular lymphoma after two lines of therapy 

Adults with follicular lymphoma (grades 1-3A) despite two or more treatment regimens, including at least one anti-CD20 therapy, are eligible for a phase 2 study of loncastuximab tesirine (Zynlonta). The drug already has an FDA accelerated approval this year for a different lymphoma, relapsed/refractory large B-cell lymphoma, so this could be a new indication. In this trial, it will be compared with idelalisib (Zydelig), which is already approved for follicular lymphoma. Participants will get either an infusion of loncastuximab every 3 weeks or a twice-daily tablet of idelalisib for up to 30 months. Investigators started recruiting on Oct. 30 and hope for 150 participants in Nevada and New Jersey. Complete response rate is the primary outcome. OS and QoL are secondary outcome measures. 

Untreated multiple myeloma not eligible for autologous stem-cell transplant (ASCT) 

Adults with untreated multiple myeloma who are not eligible for stem-cell transplantation are sought for a phase 2 study testing the performance of selinexor (Xpovio) plus dexamethasone. (Prior treatment with emergency steroids and radiation therapy is allowed.) Selinexor plus dexamethasone was approved in 2019 for multiple myeloma after four prior therapies; the goal of this study is to assess its performance as frontline treatment. Participants will receive oral selinexor and dexamethasone for up to 3 years in addition to subcutaneous daratumumab (Darzalex) and capsules of lenalidomide. The study opened Sept. 10, aiming for 100 participants at sites in Arizona, Colorado, Maryland, New York, Oregon, Texas, and Virginia. OS is a secondary outcome measure; QoL will not be assessed.  

Newly diagnosed multiple myeloma where ASCT not planned 

Patients with newly diagnosed multiple myeloma who are not having ASCT as initial therapy are eligible for a phase 3 study of the investigational CAR T-cell therapy ciltacabtagene autoleucel (cilta-cel). This product targets B-cell maturation antigen (BCMA), which is expressed on the surface of mature B lymphocytes and malignant plasma cells; it is in late-stage clinical trials for multiple myeloma but has not yet been approved. In this study, the control-group participants will receive standard therapy for up to approximately 4 years - a regimen of bortezomib (Velcade), lenalidomide, and dexamethasone. Patients destined for cilta-cel will undergo apheresis to garner their T cells, which will then be genetically engineered to express the synthetic antigen receptor, duplicated, and re-infused. During the 6-month wait between apheresis and the cilta-cel infusion, the CAR T patients will receive similar treatment to the control group. Recruitment started for 650 patients across 12 U.S. states and 24 countries on August 19. The primary outcome is progression-free survival. OS and QoL are secondary measures and will be tracked for approximately 12 years. 

All trial information is from the National Institutes of Health U.S. National Library of Medicine.

A version of this article first appeared on Medscape.com

He always dressed the same at conferences: dark suit, white shirt, bright red bow tie.

Courtesy of Beck Institute for Cognitive Behavior Therapy

For all his fame, he was very kind, warmly greeting those who wanted to see him and immediately turning attention toward their research rather than his own. Aaron Beck actually didn’t lecture much; he preferred to roleplay cognitive therapy with an audience member acting as the patient. He would engage in what he called Socratic questioning, or more formally, cognitive restructuring, with warmth and true curiosity:

• What might be another explanation or viewpoint?
• What are the effects of thinking this way?
• Can you think of any evidence that supports the opposite view?

The audience member/patient would benefit not only from thinking about things differently, but also from the captivating interaction with the man, Aaron Temkin Beck, MD, (who went by Tim), youngest child of Jewish immigrants from the Ukraine.

When written up in treatment manuals, cognitive restructuring can seem cold and overly logical, but in person, Dr. Beck made it come to life. This ability to nurture curiosity was a special talent; his friend and fellow cognitive psychologist Donald Meichenbaum, PhD, recalls that even over lunch, he never stopped asking questions, personal and professional, on a wide range of topics.

It is widely accepted that Dr. Beck, who died Nov. 1 at the age of 100 in suburban Philadelphia, was the most important figure in the field of cognitive-behavioral therapy (CBT).

He didn’t invent the field. Behaviorism predated him by generations, founded by figures such as John Watson and B.F. Skinner. Those psychologists set up behaviorism as an alternative to the reigning power of Freudian psychoanalysis, but they ran a distant second.

It wasn’t until Dr. Beck added a new approach, cognitive therapy, to the behavioristic movement that the new mélange, CBT, began to gain traction with clinicians and researchers. Dr. Beck, who had trained in psychiatry, developed his ideas in the 1960s while observing what he believed were limitations in the classic Freudian methods. He recognized that patients had “automatic thoughts,” not just unconscious emotions, when they engaged in Freudian free association, saying whatever came to their minds.

These thoughts often distorted reality, he observed; they were “maladaptive beliefs,” and when they changed, patients’ emotional states improved.

Dr. Beck wasn’t alone. The psychologist Albert Ellis, PhD, in New York, had come to similar conclusions a decade earlier, though with a more coldly logical and challenging style. The prominent British psychologist Hans Eysenck, PhD, had argued strongly that Freudian psychoanalysis was ineffective and that behavioral approaches were better.

Dr. Beck turned the Freudian equation around: Instead of emotion as cause and thought as effect, it was thought which affected emotion, for better or worse. Once you connected behavior as the outcome, you had the essence of CBT: thought, emotion, and behavior – each affecting the other, with thought being the strongest axis of change.

The process wasn’t bloodless. Behaviorists defended their turf against cognitivists, just as much as Freudians rejected both. At one point the behaviorists in the Association for the Advancement of Behavior Therapy tried to expel the advocates of a cognitive approach. Dr. Beck responded by leading the cognitivists in creating a new journal; he emphasized the importance of research being the main mechanism to decide what treatments worked the best.

Putting these ideas out in the 1960s and 1970s, Dr. Beck garnered support from researchers when he manualized the approach. Freudian psychoanalysis was idiosyncratic; it was almost impossible to study empirically, because the therapist would be responding to the unpredictable dreams and memories of patients engaged in free association. Each case was unique.

But CBT was systematic: The same general approach was taken to all patients; the same negative cognitions were found in depression, for instance, like all-or-nothing thinking or overgeneralization. Once manualized, CBT became the standard method of psychotherapy studied with the newly developed method of randomized controlled trials (RCTs).

By the 1980s, RCTs had proven the efficacy of CBT in depression, and the approach took off.

Dr. Beck already had developed a series of rating scales: the Beck Depression Inventory, the Beck Scale for Suicidal Ideation, the Beck Anxiety Inventory, the Beck Hopelessness Scale. Widely used, these scales extended his influence enormously. Copyrighted, they created a new industry of psychological research.

Dr. Beck’s own work was mainly in depression, but his followers extended it everywhere else: anxiety disorders and phobias, eating disorders, substance abuse, bipolar illness, even schizophrenia. Meanwhile, Freudian psychoanalysis fell into a steep decline from which it never recovered.

Dr. Beck’s CBT became king of the hill in psychotherapy, but it wasn’t without criticism.

Some argued that it was abetted by insurance restrictions on psychotherapy, which favored shorter-term CBT; others that its research was biased in its favor because psychotherapy treatments, unlike medications, cannot be blinded; others that its efficacy could not be shown to be specific to its theory, as opposed to the interpersonal relationship between therapist and client.

Still, CBT has transformed psychotherapy and continues to expand its influence. Computer-based CBT has been proven effective, and digital CBT has become a standard approach in many smartphone applications and is central to the claims of multiple new biotechnology companies advocating for digital psychotherapy.

Aaron Beck continued publishing scientific articles to age 98. His last papers reviewed his life’s work. He characteristically gave credit to others, calmly recollected how he traveled away from psychoanalysis, described how his work started and ended in schizophrenia, and noted that the “working relationship with the therapist” remained a key factor for the success of CBT.

That parting comment reminds us that behind all the technology and research stands the kindly man in the dark suit, white shirt, and bright red bow tie, looking at you warmly, asking about your thoughts, and curiously wondering what might be another explanation or viewpoint you hadn’t considered.
 

Nassir Ghaemi, MD, MPH, is a professor of psychiatry at Tufts Medical Center and a lecturer in psychiatry at Harvard Medical School. He is the author of several general-interest books on psychiatry. A version of this article first appeared on Medscape.com.

He always dressed the same at conferences: dark suit, white shirt, bright red bow tie.

Courtesy of Beck Institute for Cognitive Behavior Therapy

For all his fame, he was very kind, warmly greeting those who wanted to see him and immediately turning attention toward their research rather than his own. Aaron Beck actually didn’t lecture much; he preferred to roleplay cognitive therapy with an audience member acting as the patient. He would engage in what he called Socratic questioning, or more formally, cognitive restructuring, with warmth and true curiosity:

• What might be another explanation or viewpoint?
• What are the effects of thinking this way?
• Can you think of any evidence that supports the opposite view?

The audience member/patient would benefit not only from thinking about things differently, but also from the captivating interaction with the man, Aaron Temkin Beck, MD, (who went by Tim), youngest child of Jewish immigrants from the Ukraine.

When written up in treatment manuals, cognitive restructuring can seem cold and overly logical, but in person, Dr. Beck made it come to life. This ability to nurture curiosity was a special talent; his friend and fellow cognitive psychologist Donald Meichenbaum, PhD, recalls that even over lunch, he never stopped asking questions, personal and professional, on a wide range of topics.

It is widely accepted that Dr. Beck, who died Nov. 1 at the age of 100 in suburban Philadelphia, was the most important figure in the field of cognitive-behavioral therapy (CBT).

He didn’t invent the field. Behaviorism predated him by generations, founded by figures such as John Watson and B.F. Skinner. Those psychologists set up behaviorism as an alternative to the reigning power of Freudian psychoanalysis, but they ran a distant second.

It wasn’t until Dr. Beck added a new approach, cognitive therapy, to the behavioristic movement that the new mélange, CBT, began to gain traction with clinicians and researchers. Dr. Beck, who had trained in psychiatry, developed his ideas in the 1960s while observing what he believed were limitations in the classic Freudian methods. He recognized that patients had “automatic thoughts,” not just unconscious emotions, when they engaged in Freudian free association, saying whatever came to their minds.

These thoughts often distorted reality, he observed; they were “maladaptive beliefs,” and when they changed, patients’ emotional states improved.

Dr. Beck wasn’t alone. The psychologist Albert Ellis, PhD, in New York, had come to similar conclusions a decade earlier, though with a more coldly logical and challenging style. The prominent British psychologist Hans Eysenck, PhD, had argued strongly that Freudian psychoanalysis was ineffective and that behavioral approaches were better.

Dr. Beck turned the Freudian equation around: Instead of emotion as cause and thought as effect, it was thought which affected emotion, for better or worse. Once you connected behavior as the outcome, you had the essence of CBT: thought, emotion, and behavior – each affecting the other, with thought being the strongest axis of change.

The process wasn’t bloodless. Behaviorists defended their turf against cognitivists, just as much as Freudians rejected both. At one point the behaviorists in the Association for the Advancement of Behavior Therapy tried to expel the advocates of a cognitive approach. Dr. Beck responded by leading the cognitivists in creating a new journal; he emphasized the importance of research being the main mechanism to decide what treatments worked the best.

Putting these ideas out in the 1960s and 1970s, Dr. Beck garnered support from researchers when he manualized the approach. Freudian psychoanalysis was idiosyncratic; it was almost impossible to study empirically, because the therapist would be responding to the unpredictable dreams and memories of patients engaged in free association. Each case was unique.

But CBT was systematic: The same general approach was taken to all patients; the same negative cognitions were found in depression, for instance, like all-or-nothing thinking or overgeneralization. Once manualized, CBT became the standard method of psychotherapy studied with the newly developed method of randomized controlled trials (RCTs).

By the 1980s, RCTs had proven the efficacy of CBT in depression, and the approach took off.

Dr. Beck already had developed a series of rating scales: the Beck Depression Inventory, the Beck Scale for Suicidal Ideation, the Beck Anxiety Inventory, the Beck Hopelessness Scale. Widely used, these scales extended his influence enormously. Copyrighted, they created a new industry of psychological research.

Dr. Beck’s own work was mainly in depression, but his followers extended it everywhere else: anxiety disorders and phobias, eating disorders, substance abuse, bipolar illness, even schizophrenia. Meanwhile, Freudian psychoanalysis fell into a steep decline from which it never recovered.

Dr. Beck’s CBT became king of the hill in psychotherapy, but it wasn’t without criticism.

Some argued that it was abetted by insurance restrictions on psychotherapy, which favored shorter-term CBT; others that its research was biased in its favor because psychotherapy treatments, unlike medications, cannot be blinded; others that its efficacy could not be shown to be specific to its theory, as opposed to the interpersonal relationship between therapist and client.

Still, CBT has transformed psychotherapy and continues to expand its influence. Computer-based CBT has been proven effective, and digital CBT has become a standard approach in many smartphone applications and is central to the claims of multiple new biotechnology companies advocating for digital psychotherapy.

Aaron Beck continued publishing scientific articles to age 98. His last papers reviewed his life’s work. He characteristically gave credit to others, calmly recollected how he traveled away from psychoanalysis, described how his work started and ended in schizophrenia, and noted that the “working relationship with the therapist” remained a key factor for the success of CBT.

That parting comment reminds us that behind all the technology and research stands the kindly man in the dark suit, white shirt, and bright red bow tie, looking at you warmly, asking about your thoughts, and curiously wondering what might be another explanation or viewpoint you hadn’t considered.
 

Nassir Ghaemi, MD, MPH, is a professor of psychiatry at Tufts Medical Center and a lecturer in psychiatry at Harvard Medical School. He is the author of several general-interest books on psychiatry. A version of this article first appeared on Medscape.com.

He always dressed the same at conferences: dark suit, white shirt, bright red bow tie.

Courtesy of Beck Institute for Cognitive Behavior Therapy

For all his fame, he was very kind, warmly greeting those who wanted to see him and immediately turning attention toward their research rather than his own. Aaron Beck actually didn’t lecture much; he preferred to roleplay cognitive therapy with an audience member acting as the patient. He would engage in what he called Socratic questioning, or more formally, cognitive restructuring, with warmth and true curiosity:

• What might be another explanation or viewpoint?
• What are the effects of thinking this way?
• Can you think of any evidence that supports the opposite view?

The audience member/patient would benefit not only from thinking about things differently, but also from the captivating interaction with the man, Aaron Temkin Beck, MD, (who went by Tim), youngest child of Jewish immigrants from the Ukraine.

When written up in treatment manuals, cognitive restructuring can seem cold and overly logical, but in person, Dr. Beck made it come to life. This ability to nurture curiosity was a special talent; his friend and fellow cognitive psychologist Donald Meichenbaum, PhD, recalls that even over lunch, he never stopped asking questions, personal and professional, on a wide range of topics.

It is widely accepted that Dr. Beck, who died Nov. 1 at the age of 100 in suburban Philadelphia, was the most important figure in the field of cognitive-behavioral therapy (CBT).

He didn’t invent the field. Behaviorism predated him by generations, founded by figures such as John Watson and B.F. Skinner. Those psychologists set up behaviorism as an alternative to the reigning power of Freudian psychoanalysis, but they ran a distant second.

It wasn’t until Dr. Beck added a new approach, cognitive therapy, to the behavioristic movement that the new mélange, CBT, began to gain traction with clinicians and researchers. Dr. Beck, who had trained in psychiatry, developed his ideas in the 1960s while observing what he believed were limitations in the classic Freudian methods. He recognized that patients had “automatic thoughts,” not just unconscious emotions, when they engaged in Freudian free association, saying whatever came to their minds.

These thoughts often distorted reality, he observed; they were “maladaptive beliefs,” and when they changed, patients’ emotional states improved.

Dr. Beck wasn’t alone. The psychologist Albert Ellis, PhD, in New York, had come to similar conclusions a decade earlier, though with a more coldly logical and challenging style. The prominent British psychologist Hans Eysenck, PhD, had argued strongly that Freudian psychoanalysis was ineffective and that behavioral approaches were better.

Dr. Beck turned the Freudian equation around: Instead of emotion as cause and thought as effect, it was thought which affected emotion, for better or worse. Once you connected behavior as the outcome, you had the essence of CBT: thought, emotion, and behavior – each affecting the other, with thought being the strongest axis of change.

The process wasn’t bloodless. Behaviorists defended their turf against cognitivists, just as much as Freudians rejected both. At one point the behaviorists in the Association for the Advancement of Behavior Therapy tried to expel the advocates of a cognitive approach. Dr. Beck responded by leading the cognitivists in creating a new journal; he emphasized the importance of research being the main mechanism to decide what treatments worked the best.

Putting these ideas out in the 1960s and 1970s, Dr. Beck garnered support from researchers when he manualized the approach. Freudian psychoanalysis was idiosyncratic; it was almost impossible to study empirically, because the therapist would be responding to the unpredictable dreams and memories of patients engaged in free association. Each case was unique.

But CBT was systematic: The same general approach was taken to all patients; the same negative cognitions were found in depression, for instance, like all-or-nothing thinking or overgeneralization. Once manualized, CBT became the standard method of psychotherapy studied with the newly developed method of randomized controlled trials (RCTs).

By the 1980s, RCTs had proven the efficacy of CBT in depression, and the approach took off.

Dr. Beck already had developed a series of rating scales: the Beck Depression Inventory, the Beck Scale for Suicidal Ideation, the Beck Anxiety Inventory, the Beck Hopelessness Scale. Widely used, these scales extended his influence enormously. Copyrighted, they created a new industry of psychological research.

Dr. Beck’s own work was mainly in depression, but his followers extended it everywhere else: anxiety disorders and phobias, eating disorders, substance abuse, bipolar illness, even schizophrenia. Meanwhile, Freudian psychoanalysis fell into a steep decline from which it never recovered.

Dr. Beck’s CBT became king of the hill in psychotherapy, but it wasn’t without criticism.

Some argued that it was abetted by insurance restrictions on psychotherapy, which favored shorter-term CBT; others that its research was biased in its favor because psychotherapy treatments, unlike medications, cannot be blinded; others that its efficacy could not be shown to be specific to its theory, as opposed to the interpersonal relationship between therapist and client.

Still, CBT has transformed psychotherapy and continues to expand its influence. Computer-based CBT has been proven effective, and digital CBT has become a standard approach in many smartphone applications and is central to the claims of multiple new biotechnology companies advocating for digital psychotherapy.

Aaron Beck continued publishing scientific articles to age 98. His last papers reviewed his life’s work. He characteristically gave credit to others, calmly recollected how he traveled away from psychoanalysis, described how his work started and ended in schizophrenia, and noted that the “working relationship with the therapist” remained a key factor for the success of CBT.

That parting comment reminds us that behind all the technology and research stands the kindly man in the dark suit, white shirt, and bright red bow tie, looking at you warmly, asking about your thoughts, and curiously wondering what might be another explanation or viewpoint you hadn’t considered.
 

Nassir Ghaemi, MD, MPH, is a professor of psychiatry at Tufts Medical Center and a lecturer in psychiatry at Harvard Medical School. He is the author of several general-interest books on psychiatry. A version of this article first appeared on Medscape.com.

LAS VEGAS – Risankizumab (Skyrizi, AbbVie) provides early and lasting benefits for patients with Crohn’s disease, phase 3 trials indicate.

Based on these and other recent findings, the drug could be used as a first-line treatment and even displace ustekinumab (Stelara, Janssen), which itself was approved by the Food and Drug Administration for Crohn’s disease in 2016, according to David Rubin, MD, the Joseph B. Kirsner Professor of Medicine at the University of Chicago.

“The drug works fast,” Dr. Rubin said in an interview. “If you start this therapy in patients with moderate to severe Crohn’s disease, they’re likely to feel better within the first few weeks.”

Dr. Rubin presented the findings on the drug’s early onset at the annual meeting of the American College of Gastroenterology. A related trial presented at the meeting showed the drug continuing to perform well up to 52 weeks.

Advances in immunomodulation have allowed drug companies to feed multiple new therapies into the pipeline for Crohn’s disease and related conditions in recent years, giving hope to the many patients who have not been able to benefit from older classes of drugs, such as biologics.

A humanized immunoglobulin G1 (IgG1) monoclonal antibody, risankizumab blocks interleukin (IL) 23 by binding to its p19 subunit. IL-23 is a cytokine implicated in several chronic immune disorders, including Crohn’s disease and psoriasis. Researchers hope that risankizumab will prove more selective, with a better safety profile, than previous drugs in its class. The FDA approved risankizumab in April 2019 for the treatment of moderate to severe plaque psoriasis.
 

MOTIVATE and ADVANCE studies

The two induction trials for Crohn’s disease enrolled slightly different populations.

The MOTIVATE study enrolled patients who had responded inadequately or were intolerant to biologic therapy. In this trial, the investigators assigned 205 patients to 1,200 mg of risankizumab, 206 patients to 600 mg of risankizumab, and 207 patients to placebo.

The ADVANCE study enrolled patients who had responded inadequately or could not tolerate either biologic or conventional therapy. In this trial, investigators randomly assigned 372 patients to 1,200 mg of risankizumab, 373 patients to 600 mg of risankizumab, and 186 patients to placebo.

In both trials, intravenous injections were given at weeks 0, 4, and 8.

The researchers defined a Crohn’s Disease Activity Index (CDAI) clinical remission as a score less than 150. They defined a Stool Frequency and Abdominal Pain Score (SF/APS) clinical remission as a soft stool frequency of no more than 2.8, and an abdominal pain score of no more than 1 and not worse than baseline.

A CDAI clinical response was at least a 100-point decrease from baseline. The SF/APS enhanced clinical response was at least a 60% decrease in average daily stool frequency or at least a 35% decrease in average daily abdominal pain, with both not worse than baseline.

At 4 weeks, the researchers found that the percentage of patients who achieved CDAI clinical remission in both risankizumab groups of both studies was greater than in the placebo group. The difference was statistically significant (P ≤ .01 in ADVANCE and P ≤ .05 in MOTIVATE), and it continued to grow at 8 weeks and 12 weeks.

By 12 weeks in the ADVANCE trial, according to a press release from AbbVie, 45% of patients on the 600-mg dose of risankizumab and 42% on the 1,200-mg dose of risankizumab had achieved CDAI clinical remission, compared with 25% of those on placebo, which was statistically significant (P < .001). For the MOTIVATE trial, the results were significantly better for patients in the risankizumab groups than for those in the placebo group.

In both trials, the treated groups continued to improve faster than the placebo groups through 12 weeks. Improvements in SF/APS enhanced clinical response largely paralleled those for CDAI clinical remission.

“It did show very good results,” session moderator Jonathan Leighton, MD, professor of medicine and chair of the division of gastroenterology at Mayo Clinic in Phoenix, Ariz., said in an interview with Medscape Medical News. “But basically, it’s so early that we don’t have all the data.” In particular, he would have liked to see whether patients responded to the drug before week 4.
 

FORTIFY study

In FORTIFY, the maintenance trial that followed, the researchers rerandomized those patients who had responded to risankizumab into three groups. Two groups received subcutaneous injections of risankizumab, with 179 patients getting 360 mg and another 179 patients getting 180 mg. The placebo group included the remaining 184 patients.

At week 52, 40.9% of patients in the placebo group were in clinical remission, compared with 52.2% in the 360-mg group and 55.4% in the 180-mg group, which was statistically significant (P = .005 for 360 mg, and P = .003 for 180 mg.)

“It showed us that [risankizumab] could achieve deep remission, which means patients achieving remission endoscopically in combination with clinical remission,” the presenter, Marla Dubinsky, MD, professor of pediatrics and medicine in the division of pediatric gastroenterology at Icahn School of Medicine at Mount Sinai in New York, said in an interview.

Over the 52 weeks, deep remission and endoscopic remission rates increased in the 360-mg group, held steady in the 180-mg group, and decreased in the placebo group. Mean fecal calprotectin and C-reactive protein levels decreased in the risankizumab groups and increased in the placebo group.

There were more total treatment-emergent adverse events per 100 patient-years in the placebo group (339.7) than in the 360-mg group (269.3) or the 180-mg group (283.5). The same difference between groups was true of severe treatment-emergent adverse events. Serious events and events leading to discontinuation were similar in the three groups.

Dr. Leighton reports financial relationships to Olympus and Pfizer. Dr. Rubin reports financial relationships to AbbVie, AltruBio, Allergan, Arena Pharmaceuticals, Athos Therapeutics, Bellatrix, Boehringer Ingelheim, Bristol Myers Squibb, Celgene/Syneos, Connect Biopharma, GalenPharma/Atlantica, Genentech/Roche, Gilead, InDex Pharmaceuticals, Ironwood, Iterative Scopes, Janssen, Lilly, Materia Prima Farmaceutica, Pfizer, Prometheus Biosciences, Reistone, Takeda, and TECHLAB. Dr. Dubinsky reports financial relationships to all or most of the companies making drugs for inflammatory bowel disease. The studies were funded by AbbVie.

A version of this article first appeared on Medscape.com.

LAS VEGAS – Risankizumab (Skyrizi, AbbVie) provides early and lasting benefits for patients with Crohn’s disease, phase 3 trials indicate.

Based on these and other recent findings, the drug could be used as a first-line treatment and even displace ustekinumab (Stelara, Janssen), which itself was approved by the Food and Drug Administration for Crohn’s disease in 2016, according to David Rubin, MD, the Joseph B. Kirsner Professor of Medicine at the University of Chicago.

“The drug works fast,” Dr. Rubin said in an interview. “If you start this therapy in patients with moderate to severe Crohn’s disease, they’re likely to feel better within the first few weeks.”

Dr. Rubin presented the findings on the drug’s early onset at the annual meeting of the American College of Gastroenterology. A related trial presented at the meeting showed the drug continuing to perform well up to 52 weeks.

Advances in immunomodulation have allowed drug companies to feed multiple new therapies into the pipeline for Crohn’s disease and related conditions in recent years, giving hope to the many patients who have not been able to benefit from older classes of drugs, such as biologics.

A humanized immunoglobulin G1 (IgG1) monoclonal antibody, risankizumab blocks interleukin (IL) 23 by binding to its p19 subunit. IL-23 is a cytokine implicated in several chronic immune disorders, including Crohn’s disease and psoriasis. Researchers hope that risankizumab will prove more selective, with a better safety profile, than previous drugs in its class. The FDA approved risankizumab in April 2019 for the treatment of moderate to severe plaque psoriasis.
 

MOTIVATE and ADVANCE studies

The two induction trials for Crohn’s disease enrolled slightly different populations.

The MOTIVATE study enrolled patients who had responded inadequately or were intolerant to biologic therapy. In this trial, the investigators assigned 205 patients to 1,200 mg of risankizumab, 206 patients to 600 mg of risankizumab, and 207 patients to placebo.

The ADVANCE study enrolled patients who had responded inadequately or could not tolerate either biologic or conventional therapy. In this trial, investigators randomly assigned 372 patients to 1,200 mg of risankizumab, 373 patients to 600 mg of risankizumab, and 186 patients to placebo.

In both trials, intravenous injections were given at weeks 0, 4, and 8.

The researchers defined a Crohn’s Disease Activity Index (CDAI) clinical remission as a score less than 150. They defined a Stool Frequency and Abdominal Pain Score (SF/APS) clinical remission as a soft stool frequency of no more than 2.8, and an abdominal pain score of no more than 1 and not worse than baseline.

A CDAI clinical response was at least a 100-point decrease from baseline. The SF/APS enhanced clinical response was at least a 60% decrease in average daily stool frequency or at least a 35% decrease in average daily abdominal pain, with both not worse than baseline.

At 4 weeks, the researchers found that the percentage of patients who achieved CDAI clinical remission in both risankizumab groups of both studies was greater than in the placebo group. The difference was statistically significant (P ≤ .01 in ADVANCE and P ≤ .05 in MOTIVATE), and it continued to grow at 8 weeks and 12 weeks.

By 12 weeks in the ADVANCE trial, according to a press release from AbbVie, 45% of patients on the 600-mg dose of risankizumab and 42% on the 1,200-mg dose of risankizumab had achieved CDAI clinical remission, compared with 25% of those on placebo, which was statistically significant (P < .001). For the MOTIVATE trial, the results were significantly better for patients in the risankizumab groups than for those in the placebo group.

In both trials, the treated groups continued to improve faster than the placebo groups through 12 weeks. Improvements in SF/APS enhanced clinical response largely paralleled those for CDAI clinical remission.

“It did show very good results,” session moderator Jonathan Leighton, MD, professor of medicine and chair of the division of gastroenterology at Mayo Clinic in Phoenix, Ariz., said in an interview with Medscape Medical News. “But basically, it’s so early that we don’t have all the data.” In particular, he would have liked to see whether patients responded to the drug before week 4.
 

FORTIFY study

In FORTIFY, the maintenance trial that followed, the researchers rerandomized those patients who had responded to risankizumab into three groups. Two groups received subcutaneous injections of risankizumab, with 179 patients getting 360 mg and another 179 patients getting 180 mg. The placebo group included the remaining 184 patients.

At week 52, 40.9% of patients in the placebo group were in clinical remission, compared with 52.2% in the 360-mg group and 55.4% in the 180-mg group, which was statistically significant (P = .005 for 360 mg, and P = .003 for 180 mg.)

“It showed us that [risankizumab] could achieve deep remission, which means patients achieving remission endoscopically in combination with clinical remission,” the presenter, Marla Dubinsky, MD, professor of pediatrics and medicine in the division of pediatric gastroenterology at Icahn School of Medicine at Mount Sinai in New York, said in an interview.

Over the 52 weeks, deep remission and endoscopic remission rates increased in the 360-mg group, held steady in the 180-mg group, and decreased in the placebo group. Mean fecal calprotectin and C-reactive protein levels decreased in the risankizumab groups and increased in the placebo group.

There were more total treatment-emergent adverse events per 100 patient-years in the placebo group (339.7) than in the 360-mg group (269.3) or the 180-mg group (283.5). The same difference between groups was true of severe treatment-emergent adverse events. Serious events and events leading to discontinuation were similar in the three groups.

Dr. Leighton reports financial relationships to Olympus and Pfizer. Dr. Rubin reports financial relationships to AbbVie, AltruBio, Allergan, Arena Pharmaceuticals, Athos Therapeutics, Bellatrix, Boehringer Ingelheim, Bristol Myers Squibb, Celgene/Syneos, Connect Biopharma, GalenPharma/Atlantica, Genentech/Roche, Gilead, InDex Pharmaceuticals, Ironwood, Iterative Scopes, Janssen, Lilly, Materia Prima Farmaceutica, Pfizer, Prometheus Biosciences, Reistone, Takeda, and TECHLAB. Dr. Dubinsky reports financial relationships to all or most of the companies making drugs for inflammatory bowel disease. The studies were funded by AbbVie.

A version of this article first appeared on Medscape.com.

LAS VEGAS – Risankizumab (Skyrizi, AbbVie) provides early and lasting benefits for patients with Crohn’s disease, phase 3 trials indicate.

Based on these and other recent findings, the drug could be used as a first-line treatment and even displace ustekinumab (Stelara, Janssen), which itself was approved by the Food and Drug Administration for Crohn’s disease in 2016, according to David Rubin, MD, the Joseph B. Kirsner Professor of Medicine at the University of Chicago.

“The drug works fast,” Dr. Rubin said in an interview. “If you start this therapy in patients with moderate to severe Crohn’s disease, they’re likely to feel better within the first few weeks.”

Dr. Rubin presented the findings on the drug’s early onset at the annual meeting of the American College of Gastroenterology. A related trial presented at the meeting showed the drug continuing to perform well up to 52 weeks.

Advances in immunomodulation have allowed drug companies to feed multiple new therapies into the pipeline for Crohn’s disease and related conditions in recent years, giving hope to the many patients who have not been able to benefit from older classes of drugs, such as biologics.

A humanized immunoglobulin G1 (IgG1) monoclonal antibody, risankizumab blocks interleukin (IL) 23 by binding to its p19 subunit. IL-23 is a cytokine implicated in several chronic immune disorders, including Crohn’s disease and psoriasis. Researchers hope that risankizumab will prove more selective, with a better safety profile, than previous drugs in its class. The FDA approved risankizumab in April 2019 for the treatment of moderate to severe plaque psoriasis.
 

MOTIVATE and ADVANCE studies

The two induction trials for Crohn’s disease enrolled slightly different populations.

The MOTIVATE study enrolled patients who had responded inadequately or were intolerant to biologic therapy. In this trial, the investigators assigned 205 patients to 1,200 mg of risankizumab, 206 patients to 600 mg of risankizumab, and 207 patients to placebo.

The ADVANCE study enrolled patients who had responded inadequately or could not tolerate either biologic or conventional therapy. In this trial, investigators randomly assigned 372 patients to 1,200 mg of risankizumab, 373 patients to 600 mg of risankizumab, and 186 patients to placebo.

In both trials, intravenous injections were given at weeks 0, 4, and 8.

The researchers defined a Crohn’s Disease Activity Index (CDAI) clinical remission as a score less than 150. They defined a Stool Frequency and Abdominal Pain Score (SF/APS) clinical remission as a soft stool frequency of no more than 2.8, and an abdominal pain score of no more than 1 and not worse than baseline.

A CDAI clinical response was at least a 100-point decrease from baseline. The SF/APS enhanced clinical response was at least a 60% decrease in average daily stool frequency or at least a 35% decrease in average daily abdominal pain, with both not worse than baseline.

At 4 weeks, the researchers found that the percentage of patients who achieved CDAI clinical remission in both risankizumab groups of both studies was greater than in the placebo group. The difference was statistically significant (P ≤ .01 in ADVANCE and P ≤ .05 in MOTIVATE), and it continued to grow at 8 weeks and 12 weeks.

By 12 weeks in the ADVANCE trial, according to a press release from AbbVie, 45% of patients on the 600-mg dose of risankizumab and 42% on the 1,200-mg dose of risankizumab had achieved CDAI clinical remission, compared with 25% of those on placebo, which was statistically significant (P < .001). For the MOTIVATE trial, the results were significantly better for patients in the risankizumab groups than for those in the placebo group.

In both trials, the treated groups continued to improve faster than the placebo groups through 12 weeks. Improvements in SF/APS enhanced clinical response largely paralleled those for CDAI clinical remission.

“It did show very good results,” session moderator Jonathan Leighton, MD, professor of medicine and chair of the division of gastroenterology at Mayo Clinic in Phoenix, Ariz., said in an interview with Medscape Medical News. “But basically, it’s so early that we don’t have all the data.” In particular, he would have liked to see whether patients responded to the drug before week 4.
 

FORTIFY study

In FORTIFY, the maintenance trial that followed, the researchers rerandomized those patients who had responded to risankizumab into three groups. Two groups received subcutaneous injections of risankizumab, with 179 patients getting 360 mg and another 179 patients getting 180 mg. The placebo group included the remaining 184 patients.

At week 52, 40.9% of patients in the placebo group were in clinical remission, compared with 52.2% in the 360-mg group and 55.4% in the 180-mg group, which was statistically significant (P = .005 for 360 mg, and P = .003 for 180 mg.)

“It showed us that [risankizumab] could achieve deep remission, which means patients achieving remission endoscopically in combination with clinical remission,” the presenter, Marla Dubinsky, MD, professor of pediatrics and medicine in the division of pediatric gastroenterology at Icahn School of Medicine at Mount Sinai in New York, said in an interview.

Over the 52 weeks, deep remission and endoscopic remission rates increased in the 360-mg group, held steady in the 180-mg group, and decreased in the placebo group. Mean fecal calprotectin and C-reactive protein levels decreased in the risankizumab groups and increased in the placebo group.

There were more total treatment-emergent adverse events per 100 patient-years in the placebo group (339.7) than in the 360-mg group (269.3) or the 180-mg group (283.5). The same difference between groups was true of severe treatment-emergent adverse events. Serious events and events leading to discontinuation were similar in the three groups.

Dr. Leighton reports financial relationships to Olympus and Pfizer. Dr. Rubin reports financial relationships to AbbVie, AltruBio, Allergan, Arena Pharmaceuticals, Athos Therapeutics, Bellatrix, Boehringer Ingelheim, Bristol Myers Squibb, Celgene/Syneos, Connect Biopharma, GalenPharma/Atlantica, Genentech/Roche, Gilead, InDex Pharmaceuticals, Ironwood, Iterative Scopes, Janssen, Lilly, Materia Prima Farmaceutica, Pfizer, Prometheus Biosciences, Reistone, Takeda, and TECHLAB. Dr. Dubinsky reports financial relationships to all or most of the companies making drugs for inflammatory bowel disease. The studies were funded by AbbVie.

A version of this article first appeared on Medscape.com.