CHICAGO -- High-dose ifosfamide has shown superior survival benefit over three other chemotherapy regimens for patients with recurring or refractory primary Ewing sarcoma (RR-ES) in the practice-changing rEECur trial.

This international trial is the first randomized head-to-head comparison of commonly used chemotherapy regimens in patients with the rare and deadly disease.

The study results are expected to change the standard of care and be practice-changing on a global scale, commented Julie Gralow, MD, chief medical officer at the annual meeting of the American Society of Clinical Oncology, where the results were presented June 5 during a plenary session.

Ewing sarcoma is a very rare cancer of the bone and soft tissue that mainly affects children and young adults, particularly in the second decade of life, explained lead author Martin McCabe, MD, clinical senior lecturer in pediatric, teenage, and young adult cancer at the University of Manchester (England). The incidence rate is 3.2 per million people under age 25 years, he said.

Dr. Gralow explained in an interview that treatment of Ewing sarcoma differs from one cancer center to another. Several different chemotherapy regimens are being used, all based on single-arm trials, with no consensus on which is best.

This international trial set out to answer that question and compared four different regimens. Participating centers were “able to solve a question by partnering, coming together, and even in a very rare population get enough patients to define the winner,” she said.

Earlier findings from this trial had shown that ifosfamide had improved survival, compared with gemcitabine and docetaxel and compared with irinotecan and temozolomide.

At the meeting, results of the comparison of ifosfamide versus a combination of topotecan and cyclophosphamide (TC) were presented.

Median overall survival was 15.4 versus 10.5 months with ifosfamide versus TC, and 1-year overall survival was 55% versus 45%, respectively, for a 94% probability that ifosfamide is better than TC for overall survival, Dr. McCabe reported.  

Median event-free survival was 16.8 months in 73 patients in the ifosfamide group versus 10.4 months for 73 patients in the TC group. Six-month event-free survival was 47% versus 37%, respectively. “Given the observed data, there is a 96% probability that ifosfamide is better than TC for event-free survival,” he said.   

High-dose ifosfamide prolonged median event-free survival by 5.7 months, compared with 3.7 months for TC.

Notably, greater event-free survival and overall survival differences were observed for patients under age 14 years, compared with those aged 14 and older, Dr. McCabe noted.

As for toxicity, similar rates of neutropenic infections were seen in the two groups, but more severe renal and brain toxicity were observed with ifosfamide, with both occurring in less than 10% of patients, he said.

Despite the practice-changing results, Dr. McCabe stressed that the “differences [between treatments] are quite small, and what we actually need is better drugs to cure more patients.”

The rEEcur trial is continuing to recruit patients to the ifosfamide group, and a fifth chemotherapy group of carboplatin and etoposide has been added.

Later this year, investigators also plan to add a new group with a molecular targeted therapeutic.
 

Important global collaboration

Dr. Gralow emphasized the global collaboration that was behind this trial, which set out to answer important questions about how best to treat a rare disease. “In this really terrific collaboration ... there was an agreement to test all these regimens that are commonly used, and so we now have data on efficacy and toxicity.”

“It’s a really important concept in rare diseases: If we all work together, we actually can study them and get answers,” she said.

“I think pediatricians and oncologists are [now] better able to talk about the risks and benefits [of the regimens],” she added.

Vicki L. Keedy, MD, an ASCO Expert in sarcoma, concurred. The findings from the rEECur trial “could help physicians talk with patients and their families about the likelihood of response, survival, and toxicity for each regimen available for relapsed Ewing sarcoma based on objective, randomized data,” she commented in an ASCO press release. 

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

CHICAGO -- High-dose ifosfamide has shown superior survival benefit over three other chemotherapy regimens for patients with recurring or refractory primary Ewing sarcoma (RR-ES) in the practice-changing rEECur trial.

This international trial is the first randomized head-to-head comparison of commonly used chemotherapy regimens in patients with the rare and deadly disease.

The study results are expected to change the standard of care and be practice-changing on a global scale, commented Julie Gralow, MD, chief medical officer at the annual meeting of the American Society of Clinical Oncology, where the results were presented June 5 during a plenary session.

Ewing sarcoma is a very rare cancer of the bone and soft tissue that mainly affects children and young adults, particularly in the second decade of life, explained lead author Martin McCabe, MD, clinical senior lecturer in pediatric, teenage, and young adult cancer at the University of Manchester (England). The incidence rate is 3.2 per million people under age 25 years, he said.

Dr. Gralow explained in an interview that treatment of Ewing sarcoma differs from one cancer center to another. Several different chemotherapy regimens are being used, all based on single-arm trials, with no consensus on which is best.

This international trial set out to answer that question and compared four different regimens. Participating centers were “able to solve a question by partnering, coming together, and even in a very rare population get enough patients to define the winner,” she said.

Earlier findings from this trial had shown that ifosfamide had improved survival, compared with gemcitabine and docetaxel and compared with irinotecan and temozolomide.

At the meeting, results of the comparison of ifosfamide versus a combination of topotecan and cyclophosphamide (TC) were presented.

Median overall survival was 15.4 versus 10.5 months with ifosfamide versus TC, and 1-year overall survival was 55% versus 45%, respectively, for a 94% probability that ifosfamide is better than TC for overall survival, Dr. McCabe reported.  

Median event-free survival was 16.8 months in 73 patients in the ifosfamide group versus 10.4 months for 73 patients in the TC group. Six-month event-free survival was 47% versus 37%, respectively. “Given the observed data, there is a 96% probability that ifosfamide is better than TC for event-free survival,” he said.   

High-dose ifosfamide prolonged median event-free survival by 5.7 months, compared with 3.7 months for TC.

Notably, greater event-free survival and overall survival differences were observed for patients under age 14 years, compared with those aged 14 and older, Dr. McCabe noted.

As for toxicity, similar rates of neutropenic infections were seen in the two groups, but more severe renal and brain toxicity were observed with ifosfamide, with both occurring in less than 10% of patients, he said.

Despite the practice-changing results, Dr. McCabe stressed that the “differences [between treatments] are quite small, and what we actually need is better drugs to cure more patients.”

The rEEcur trial is continuing to recruit patients to the ifosfamide group, and a fifth chemotherapy group of carboplatin and etoposide has been added.

Later this year, investigators also plan to add a new group with a molecular targeted therapeutic.
 

Important global collaboration

Dr. Gralow emphasized the global collaboration that was behind this trial, which set out to answer important questions about how best to treat a rare disease. “In this really terrific collaboration ... there was an agreement to test all these regimens that are commonly used, and so we now have data on efficacy and toxicity.”

“It’s a really important concept in rare diseases: If we all work together, we actually can study them and get answers,” she said.

“I think pediatricians and oncologists are [now] better able to talk about the risks and benefits [of the regimens],” she added.

Vicki L. Keedy, MD, an ASCO Expert in sarcoma, concurred. The findings from the rEECur trial “could help physicians talk with patients and their families about the likelihood of response, survival, and toxicity for each regimen available for relapsed Ewing sarcoma based on objective, randomized data,” she commented in an ASCO press release. 

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

CHICAGO -- High-dose ifosfamide has shown superior survival benefit over three other chemotherapy regimens for patients with recurring or refractory primary Ewing sarcoma (RR-ES) in the practice-changing rEECur trial.

This international trial is the first randomized head-to-head comparison of commonly used chemotherapy regimens in patients with the rare and deadly disease.

The study results are expected to change the standard of care and be practice-changing on a global scale, commented Julie Gralow, MD, chief medical officer at the annual meeting of the American Society of Clinical Oncology, where the results were presented June 5 during a plenary session.

Ewing sarcoma is a very rare cancer of the bone and soft tissue that mainly affects children and young adults, particularly in the second decade of life, explained lead author Martin McCabe, MD, clinical senior lecturer in pediatric, teenage, and young adult cancer at the University of Manchester (England). The incidence rate is 3.2 per million people under age 25 years, he said.

Dr. Gralow explained in an interview that treatment of Ewing sarcoma differs from one cancer center to another. Several different chemotherapy regimens are being used, all based on single-arm trials, with no consensus on which is best.

This international trial set out to answer that question and compared four different regimens. Participating centers were “able to solve a question by partnering, coming together, and even in a very rare population get enough patients to define the winner,” she said.

Earlier findings from this trial had shown that ifosfamide had improved survival, compared with gemcitabine and docetaxel and compared with irinotecan and temozolomide.

At the meeting, results of the comparison of ifosfamide versus a combination of topotecan and cyclophosphamide (TC) were presented.

Median overall survival was 15.4 versus 10.5 months with ifosfamide versus TC, and 1-year overall survival was 55% versus 45%, respectively, for a 94% probability that ifosfamide is better than TC for overall survival, Dr. McCabe reported.  

Median event-free survival was 16.8 months in 73 patients in the ifosfamide group versus 10.4 months for 73 patients in the TC group. Six-month event-free survival was 47% versus 37%, respectively. “Given the observed data, there is a 96% probability that ifosfamide is better than TC for event-free survival,” he said.   

High-dose ifosfamide prolonged median event-free survival by 5.7 months, compared with 3.7 months for TC.

Notably, greater event-free survival and overall survival differences were observed for patients under age 14 years, compared with those aged 14 and older, Dr. McCabe noted.

As for toxicity, similar rates of neutropenic infections were seen in the two groups, but more severe renal and brain toxicity were observed with ifosfamide, with both occurring in less than 10% of patients, he said.

Despite the practice-changing results, Dr. McCabe stressed that the “differences [between treatments] are quite small, and what we actually need is better drugs to cure more patients.”

The rEEcur trial is continuing to recruit patients to the ifosfamide group, and a fifth chemotherapy group of carboplatin and etoposide has been added.

Later this year, investigators also plan to add a new group with a molecular targeted therapeutic.
 

Important global collaboration

Dr. Gralow emphasized the global collaboration that was behind this trial, which set out to answer important questions about how best to treat a rare disease. “In this really terrific collaboration ... there was an agreement to test all these regimens that are commonly used, and so we now have data on efficacy and toxicity.”

“It’s a really important concept in rare diseases: If we all work together, we actually can study them and get answers,” she said.

“I think pediatricians and oncologists are [now] better able to talk about the risks and benefits [of the regimens],” she added.

Vicki L. Keedy, MD, an ASCO Expert in sarcoma, concurred. The findings from the rEECur trial “could help physicians talk with patients and their families about the likelihood of response, survival, and toxicity for each regimen available for relapsed Ewing sarcoma based on objective, randomized data,” she commented in an ASCO press release. 

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

It happens quickly: A child on the autism spectrum bolts from supervision and disappears – an emergency called “autism elopement.” While any child can wander off, children on the autism spectrum face particular risks. These include the lure of water and the risk of drowning.

Some youngsters on the spectrum will follow this strong attraction to water and head for a nearby pond, river, or swimming pool. Such circumstances have made drowning a leading cause of death for these missing youths.

Autism elopement can happen any time. Summer can be especially dangerous. When the weather warms, the risk of drowning death rises, says Lori McIlwain, cofounder of the National Autism Association.

“The fatality risk is higher in May, June, July for that child to exit the setting unnoticed, especially if there’s an outdoor gathering and then they go directly to water,” Ms. McIlwain says. For instance, she says children can dart away during outdoor play, barbecues, gatherings, and other activities. Or they might wander off while vacationing near a beach or hotel pool.
 

Autism elopement

Many people don’t know about this risk, including some families with youngsters on the autism spectrum. The National Center for Missing and Exploited Children is working to change that – and find solutions.

About 12 years ago, “we started noticing a very disturbing trend that children with autism were going missing and they were having grave results,” says John Bischoff, vice president of the Center’s Missing Children Division.

The Center analyzed a decade of data on accidental deaths of children on the autism spectrum. Drowning was the #1 cause, accounting for 84% of those deaths.

In 2012, researchers reported on autism and wandering in the journal Pediatrics. They analyzed answers from about 1,000 families to an online survey on the topic. Parents who had children on the spectrum and children not on the spectrum responded. Nearly half of the parents said their child with autism had tried to wander off after age 4, and 26% had gone missing long enough to cause concern.

“Of those who went missing, 24% were in danger of drowning and 65% were in danger of traffic injury,” the researchers wrote. Children on the spectrum might also be drawn to traffic signs, highways, fire trucks, and trains.

In comparison, brothers and sisters of all ages who were not on the spectrum were much less likely to have wandered off.
 

Seeking a quiet place

It’s not entirely clear why children with autism are so drawn to water, Ms. McIlwain says. But there are some clues.

“What we see is that these children exit settings that are usually bothersome,” Ms. McIlwain says. “[Those settings are] loud, with a high amount of stimuli or stress or commotion, and they go to a quiet place, usually water in a quiet area. It’s calm. It’s peaceful.”

Water isn’t the only dangerous draw. When autism elopement happens, “they also go to the woods, they go to abandoned vehicles,” she says. “So any quiet thing is usually where they will head.”
 

A family’s loss

Beth Dilg, a mother in Maryland, lost her 7-year-old daughter, Savannah Martin, who was on the autism spectrum, to drowning in 2011. Ms. Dilg had been living in Oklahoma and raising her three children alone after separating from her husband. On a chilly February day, Savannah and her 2-year-old brother left their house after Ms. Dilg had asked her 11-year-old son to keep watch while she went into the bathroom for a few minutes.

When Ms. Dilg realized the two younger kids had left, she searched the property frantically. She shouted Savannah’s name repeatedly, but the child, who had limited language, didn’t come when called. “I feel like she knew what her name was,” Ms. Dilg says, “but it wasn’t like you’d call her name and she’d come to you.”

Ms. Dilg ran to a pond near her property after her 11-year-old son said that the two siblings were in the water. Ms. Dilg entered the water and grabbed her toddler, who had survived after having been kept afloat by his bicycle helmet. But when Ms. Dilg reached Savannah, she was already unresponsive. A neighbor helped pull the children out.

It can happen in any family. Even when a parent takes precautions, a child can slip out in a moment, perhaps while the parent is asleep or taking care of personal needs or if the child is at school or elsewhere.

“It’s unrealistic to say that you’d never take your eyes off your kid,” Ms. Dilg says.

She had tried to protect Savannah by starting her on swimming lessons, installing high locks on the doors, and trying to teach her about how to stay safe.

Still, children can be skillful in finding ways to escape, Ms. Dilg says. “These kids with autism are so smart. They may not be verbal, but they have this level of intelligence,” she says. “You always have to stay a step ahead of them.”

Ms. Dilg has been a longtime volunteer with Team HOPE, a peer support group with the National Center for Missing and Exploited Children. She offers emotional support to parents whose children are missing or who have died, including the parents of youngsters with autism who have drowned.
 

Teaching first responders

If a child on the autism spectrum goes missing, searching for them can be complicated by their condition. For instance, some children cannot speak or aren’t able to respond to searchers calling their name. The National Center for Missing and Exploited Children offers training to law enforcement and provides search protocols for first responders.

The center has drawn on expertise from Laurie Reyes, an officer with Maryland’s Montgomery County Police Department. In 2005, Ms. Reyes created a special unit within the department to focus on safety for people at risk for wandering. They have conditions that include autism/intellectual and developmental disabilities, as well as Alzheimer’s and other forms of dementia.

“We have a culture of awareness here,” Ms. Reyes says. All Montgomery County recruits and officers receive training in how to interact with those on the autism spectrum, who may not respond to police commands. Police also learn how to search, including immediately checking bodies of water. “We’ve had many times where we’ve located individuals in bodies of water,” Ms. Reyes says.

Don’t wait to call 911. When a child goes missing, time matters. Ms. Reyes advises families not to search on their own. “Call 911 right away,” she says.
 

Top safety tips to help prevent autism elopement

Use these tips to help keep kids on the autism spectrum safe and prevent drownings.

Secure your home. Use window and door alarms to alert you if a door or window becomes ajar. “Those door alarms are essential,” Ms. McIlwain says.

You can buy alarms online or get them free from the National Autism Association through its Big Red Safety Box program.

You can also buy portable door alarms for travel and arrange to have door alarms at your child’s school.

Ms. McIlwain advises securing the home with adequate locks and using baby monitors. Installing visual prompts, such as a stop sign on the door, might also cue a child not to leave.

Use personal identification. Ms. McIlwain says that children with autism must wear identification, such as a wristband, that includes their name, autism diagnosis, and the name and phone number of a contact person.

If children won’t wear a wristband, IDs on shoelaces are an option, she says. But parents should be aware that kids might leave without shoes or take them off before entering water.

Parents can also weigh the pros and cons of using tracking and locater devices, Ms. McIlwain says.

Identify triggers. “What’s going to make the child want to leave the setting? Is it noise? Is it a certain thing that they fear?” Ms. McIlwain says. “There’s always a reason.”

If parents can identify particular triggers, they can use calming techniques, for example, or provide headphones to counteract bothersome noises.

Teach safety skills, such as swimming lessons. Swimming lessons are important, Ms. McIlwain says. However, children with autism are often bothered by noise and commotion. So a regular swim class might not work for them.

Instead, Ms. McIlwain encourages parents to ask their local YMCA about special-needs swimming lessons or to search for such lessons online. What usually turns out to be best is to give the child a few private swimming lessons “with somebody who understands autism.”

For the child’s final lesson, they should swim fully clothed and with shoes on, Ms. McIlwain says. “A lot of our kids go straight into water fully clothed, and they just need to be able to be familiar with how that feels and the weight of that and be able to swim like that as well.”

If a child is drawn to water, discuss a scheduled time to go so that the youngster can wait, Ms. McIlwain says. “They can see that they’re going to get that water time. They’re going to be able to go to that place. They’re going to wait instead of trying to go on their own.”

Keep a close watch and team up. “When there is a family gathering or an outdoor barbecue, a lot of times, we all think, there are more adults here, so there are going to be more eyes on all the kids. And that always ends up being opposite, right?” Ms. McIlwain says.

Be specific about who is monitoring the child’s safety.

“We encourage parents to do the ‘Tag, you’re it’ game with one another. So you basically tag an adult who is responsible for keeping an eye on that child for a period of time so that there’s always supervision.”
 

Be prepared

There are a few things you can do now to be ready in case your child slips away. These measures may help find the child quickly.

Take photos today. Keep a full-length shot and a head shot of your child and store them electronically. If your child wanders away, you can immediately send the images to law enforcement to help them search.

Write a 911 script. Have this document ready in case your child wanders. It describes, among other things, points of interest that might draw your child, as well as locations of nearby bodies of water. By having it all written down, you’ll be able to share the information quickly with first responders. The Montgomery County Police Department has a “Wandering 911 Script” that you can download and use.

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

It happens quickly: A child on the autism spectrum bolts from supervision and disappears – an emergency called “autism elopement.” While any child can wander off, children on the autism spectrum face particular risks. These include the lure of water and the risk of drowning.

Some youngsters on the spectrum will follow this strong attraction to water and head for a nearby pond, river, or swimming pool. Such circumstances have made drowning a leading cause of death for these missing youths.

Autism elopement can happen any time. Summer can be especially dangerous. When the weather warms, the risk of drowning death rises, says Lori McIlwain, cofounder of the National Autism Association.

“The fatality risk is higher in May, June, July for that child to exit the setting unnoticed, especially if there’s an outdoor gathering and then they go directly to water,” Ms. McIlwain says. For instance, she says children can dart away during outdoor play, barbecues, gatherings, and other activities. Or they might wander off while vacationing near a beach or hotel pool.
 

Autism elopement

Many people don’t know about this risk, including some families with youngsters on the autism spectrum. The National Center for Missing and Exploited Children is working to change that – and find solutions.

About 12 years ago, “we started noticing a very disturbing trend that children with autism were going missing and they were having grave results,” says John Bischoff, vice president of the Center’s Missing Children Division.

The Center analyzed a decade of data on accidental deaths of children on the autism spectrum. Drowning was the #1 cause, accounting for 84% of those deaths.

In 2012, researchers reported on autism and wandering in the journal Pediatrics. They analyzed answers from about 1,000 families to an online survey on the topic. Parents who had children on the spectrum and children not on the spectrum responded. Nearly half of the parents said their child with autism had tried to wander off after age 4, and 26% had gone missing long enough to cause concern.

“Of those who went missing, 24% were in danger of drowning and 65% were in danger of traffic injury,” the researchers wrote. Children on the spectrum might also be drawn to traffic signs, highways, fire trucks, and trains.

In comparison, brothers and sisters of all ages who were not on the spectrum were much less likely to have wandered off.
 

Seeking a quiet place

It’s not entirely clear why children with autism are so drawn to water, Ms. McIlwain says. But there are some clues.

“What we see is that these children exit settings that are usually bothersome,” Ms. McIlwain says. “[Those settings are] loud, with a high amount of stimuli or stress or commotion, and they go to a quiet place, usually water in a quiet area. It’s calm. It’s peaceful.”

Water isn’t the only dangerous draw. When autism elopement happens, “they also go to the woods, they go to abandoned vehicles,” she says. “So any quiet thing is usually where they will head.”
 

A family’s loss

Beth Dilg, a mother in Maryland, lost her 7-year-old daughter, Savannah Martin, who was on the autism spectrum, to drowning in 2011. Ms. Dilg had been living in Oklahoma and raising her three children alone after separating from her husband. On a chilly February day, Savannah and her 2-year-old brother left their house after Ms. Dilg had asked her 11-year-old son to keep watch while she went into the bathroom for a few minutes.

When Ms. Dilg realized the two younger kids had left, she searched the property frantically. She shouted Savannah’s name repeatedly, but the child, who had limited language, didn’t come when called. “I feel like she knew what her name was,” Ms. Dilg says, “but it wasn’t like you’d call her name and she’d come to you.”

Ms. Dilg ran to a pond near her property after her 11-year-old son said that the two siblings were in the water. Ms. Dilg entered the water and grabbed her toddler, who had survived after having been kept afloat by his bicycle helmet. But when Ms. Dilg reached Savannah, she was already unresponsive. A neighbor helped pull the children out.

It can happen in any family. Even when a parent takes precautions, a child can slip out in a moment, perhaps while the parent is asleep or taking care of personal needs or if the child is at school or elsewhere.

“It’s unrealistic to say that you’d never take your eyes off your kid,” Ms. Dilg says.

She had tried to protect Savannah by starting her on swimming lessons, installing high locks on the doors, and trying to teach her about how to stay safe.

Still, children can be skillful in finding ways to escape, Ms. Dilg says. “These kids with autism are so smart. They may not be verbal, but they have this level of intelligence,” she says. “You always have to stay a step ahead of them.”

Ms. Dilg has been a longtime volunteer with Team HOPE, a peer support group with the National Center for Missing and Exploited Children. She offers emotional support to parents whose children are missing or who have died, including the parents of youngsters with autism who have drowned.
 

Teaching first responders

If a child on the autism spectrum goes missing, searching for them can be complicated by their condition. For instance, some children cannot speak or aren’t able to respond to searchers calling their name. The National Center for Missing and Exploited Children offers training to law enforcement and provides search protocols for first responders.

The center has drawn on expertise from Laurie Reyes, an officer with Maryland’s Montgomery County Police Department. In 2005, Ms. Reyes created a special unit within the department to focus on safety for people at risk for wandering. They have conditions that include autism/intellectual and developmental disabilities, as well as Alzheimer’s and other forms of dementia.

“We have a culture of awareness here,” Ms. Reyes says. All Montgomery County recruits and officers receive training in how to interact with those on the autism spectrum, who may not respond to police commands. Police also learn how to search, including immediately checking bodies of water. “We’ve had many times where we’ve located individuals in bodies of water,” Ms. Reyes says.

Don’t wait to call 911. When a child goes missing, time matters. Ms. Reyes advises families not to search on their own. “Call 911 right away,” she says.
 

Top safety tips to help prevent autism elopement

Use these tips to help keep kids on the autism spectrum safe and prevent drownings.

Secure your home. Use window and door alarms to alert you if a door or window becomes ajar. “Those door alarms are essential,” Ms. McIlwain says.

You can buy alarms online or get them free from the National Autism Association through its Big Red Safety Box program.

You can also buy portable door alarms for travel and arrange to have door alarms at your child’s school.

Ms. McIlwain advises securing the home with adequate locks and using baby monitors. Installing visual prompts, such as a stop sign on the door, might also cue a child not to leave.

Use personal identification. Ms. McIlwain says that children with autism must wear identification, such as a wristband, that includes their name, autism diagnosis, and the name and phone number of a contact person.

If children won’t wear a wristband, IDs on shoelaces are an option, she says. But parents should be aware that kids might leave without shoes or take them off before entering water.

Parents can also weigh the pros and cons of using tracking and locater devices, Ms. McIlwain says.

Identify triggers. “What’s going to make the child want to leave the setting? Is it noise? Is it a certain thing that they fear?” Ms. McIlwain says. “There’s always a reason.”

If parents can identify particular triggers, they can use calming techniques, for example, or provide headphones to counteract bothersome noises.

Teach safety skills, such as swimming lessons. Swimming lessons are important, Ms. McIlwain says. However, children with autism are often bothered by noise and commotion. So a regular swim class might not work for them.

Instead, Ms. McIlwain encourages parents to ask their local YMCA about special-needs swimming lessons or to search for such lessons online. What usually turns out to be best is to give the child a few private swimming lessons “with somebody who understands autism.”

For the child’s final lesson, they should swim fully clothed and with shoes on, Ms. McIlwain says. “A lot of our kids go straight into water fully clothed, and they just need to be able to be familiar with how that feels and the weight of that and be able to swim like that as well.”

If a child is drawn to water, discuss a scheduled time to go so that the youngster can wait, Ms. McIlwain says. “They can see that they’re going to get that water time. They’re going to be able to go to that place. They’re going to wait instead of trying to go on their own.”

Keep a close watch and team up. “When there is a family gathering or an outdoor barbecue, a lot of times, we all think, there are more adults here, so there are going to be more eyes on all the kids. And that always ends up being opposite, right?” Ms. McIlwain says.

Be specific about who is monitoring the child’s safety.

“We encourage parents to do the ‘Tag, you’re it’ game with one another. So you basically tag an adult who is responsible for keeping an eye on that child for a period of time so that there’s always supervision.”
 

Be prepared

There are a few things you can do now to be ready in case your child slips away. These measures may help find the child quickly.

Take photos today. Keep a full-length shot and a head shot of your child and store them electronically. If your child wanders away, you can immediately send the images to law enforcement to help them search.

Write a 911 script. Have this document ready in case your child wanders. It describes, among other things, points of interest that might draw your child, as well as locations of nearby bodies of water. By having it all written down, you’ll be able to share the information quickly with first responders. The Montgomery County Police Department has a “Wandering 911 Script” that you can download and use.

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

It happens quickly: A child on the autism spectrum bolts from supervision and disappears – an emergency called “autism elopement.” While any child can wander off, children on the autism spectrum face particular risks. These include the lure of water and the risk of drowning.

Some youngsters on the spectrum will follow this strong attraction to water and head for a nearby pond, river, or swimming pool. Such circumstances have made drowning a leading cause of death for these missing youths.

Autism elopement can happen any time. Summer can be especially dangerous. When the weather warms, the risk of drowning death rises, says Lori McIlwain, cofounder of the National Autism Association.

“The fatality risk is higher in May, June, July for that child to exit the setting unnoticed, especially if there’s an outdoor gathering and then they go directly to water,” Ms. McIlwain says. For instance, she says children can dart away during outdoor play, barbecues, gatherings, and other activities. Or they might wander off while vacationing near a beach or hotel pool.
 

Autism elopement

Many people don’t know about this risk, including some families with youngsters on the autism spectrum. The National Center for Missing and Exploited Children is working to change that – and find solutions.

About 12 years ago, “we started noticing a very disturbing trend that children with autism were going missing and they were having grave results,” says John Bischoff, vice president of the Center’s Missing Children Division.

The Center analyzed a decade of data on accidental deaths of children on the autism spectrum. Drowning was the #1 cause, accounting for 84% of those deaths.

In 2012, researchers reported on autism and wandering in the journal Pediatrics. They analyzed answers from about 1,000 families to an online survey on the topic. Parents who had children on the spectrum and children not on the spectrum responded. Nearly half of the parents said their child with autism had tried to wander off after age 4, and 26% had gone missing long enough to cause concern.

“Of those who went missing, 24% were in danger of drowning and 65% were in danger of traffic injury,” the researchers wrote. Children on the spectrum might also be drawn to traffic signs, highways, fire trucks, and trains.

In comparison, brothers and sisters of all ages who were not on the spectrum were much less likely to have wandered off.
 

Seeking a quiet place

It’s not entirely clear why children with autism are so drawn to water, Ms. McIlwain says. But there are some clues.

“What we see is that these children exit settings that are usually bothersome,” Ms. McIlwain says. “[Those settings are] loud, with a high amount of stimuli or stress or commotion, and they go to a quiet place, usually water in a quiet area. It’s calm. It’s peaceful.”

Water isn’t the only dangerous draw. When autism elopement happens, “they also go to the woods, they go to abandoned vehicles,” she says. “So any quiet thing is usually where they will head.”
 

A family’s loss

Beth Dilg, a mother in Maryland, lost her 7-year-old daughter, Savannah Martin, who was on the autism spectrum, to drowning in 2011. Ms. Dilg had been living in Oklahoma and raising her three children alone after separating from her husband. On a chilly February day, Savannah and her 2-year-old brother left their house after Ms. Dilg had asked her 11-year-old son to keep watch while she went into the bathroom for a few minutes.

When Ms. Dilg realized the two younger kids had left, she searched the property frantically. She shouted Savannah’s name repeatedly, but the child, who had limited language, didn’t come when called. “I feel like she knew what her name was,” Ms. Dilg says, “but it wasn’t like you’d call her name and she’d come to you.”

Ms. Dilg ran to a pond near her property after her 11-year-old son said that the two siblings were in the water. Ms. Dilg entered the water and grabbed her toddler, who had survived after having been kept afloat by his bicycle helmet. But when Ms. Dilg reached Savannah, she was already unresponsive. A neighbor helped pull the children out.

It can happen in any family. Even when a parent takes precautions, a child can slip out in a moment, perhaps while the parent is asleep or taking care of personal needs or if the child is at school or elsewhere.

“It’s unrealistic to say that you’d never take your eyes off your kid,” Ms. Dilg says.

She had tried to protect Savannah by starting her on swimming lessons, installing high locks on the doors, and trying to teach her about how to stay safe.

Still, children can be skillful in finding ways to escape, Ms. Dilg says. “These kids with autism are so smart. They may not be verbal, but they have this level of intelligence,” she says. “You always have to stay a step ahead of them.”

Ms. Dilg has been a longtime volunteer with Team HOPE, a peer support group with the National Center for Missing and Exploited Children. She offers emotional support to parents whose children are missing or who have died, including the parents of youngsters with autism who have drowned.
 

Teaching first responders

If a child on the autism spectrum goes missing, searching for them can be complicated by their condition. For instance, some children cannot speak or aren’t able to respond to searchers calling their name. The National Center for Missing and Exploited Children offers training to law enforcement and provides search protocols for first responders.

The center has drawn on expertise from Laurie Reyes, an officer with Maryland’s Montgomery County Police Department. In 2005, Ms. Reyes created a special unit within the department to focus on safety for people at risk for wandering. They have conditions that include autism/intellectual and developmental disabilities, as well as Alzheimer’s and other forms of dementia.

“We have a culture of awareness here,” Ms. Reyes says. All Montgomery County recruits and officers receive training in how to interact with those on the autism spectrum, who may not respond to police commands. Police also learn how to search, including immediately checking bodies of water. “We’ve had many times where we’ve located individuals in bodies of water,” Ms. Reyes says.

Don’t wait to call 911. When a child goes missing, time matters. Ms. Reyes advises families not to search on their own. “Call 911 right away,” she says.
 

Top safety tips to help prevent autism elopement

Use these tips to help keep kids on the autism spectrum safe and prevent drownings.

Secure your home. Use window and door alarms to alert you if a door or window becomes ajar. “Those door alarms are essential,” Ms. McIlwain says.

You can buy alarms online or get them free from the National Autism Association through its Big Red Safety Box program.

You can also buy portable door alarms for travel and arrange to have door alarms at your child’s school.

Ms. McIlwain advises securing the home with adequate locks and using baby monitors. Installing visual prompts, such as a stop sign on the door, might also cue a child not to leave.

Use personal identification. Ms. McIlwain says that children with autism must wear identification, such as a wristband, that includes their name, autism diagnosis, and the name and phone number of a contact person.

If children won’t wear a wristband, IDs on shoelaces are an option, she says. But parents should be aware that kids might leave without shoes or take them off before entering water.

Parents can also weigh the pros and cons of using tracking and locater devices, Ms. McIlwain says.

Identify triggers. “What’s going to make the child want to leave the setting? Is it noise? Is it a certain thing that they fear?” Ms. McIlwain says. “There’s always a reason.”

If parents can identify particular triggers, they can use calming techniques, for example, or provide headphones to counteract bothersome noises.

Teach safety skills, such as swimming lessons. Swimming lessons are important, Ms. McIlwain says. However, children with autism are often bothered by noise and commotion. So a regular swim class might not work for them.

Instead, Ms. McIlwain encourages parents to ask their local YMCA about special-needs swimming lessons or to search for such lessons online. What usually turns out to be best is to give the child a few private swimming lessons “with somebody who understands autism.”

For the child’s final lesson, they should swim fully clothed and with shoes on, Ms. McIlwain says. “A lot of our kids go straight into water fully clothed, and they just need to be able to be familiar with how that feels and the weight of that and be able to swim like that as well.”

If a child is drawn to water, discuss a scheduled time to go so that the youngster can wait, Ms. McIlwain says. “They can see that they’re going to get that water time. They’re going to be able to go to that place. They’re going to wait instead of trying to go on their own.”

Keep a close watch and team up. “When there is a family gathering or an outdoor barbecue, a lot of times, we all think, there are more adults here, so there are going to be more eyes on all the kids. And that always ends up being opposite, right?” Ms. McIlwain says.

Be specific about who is monitoring the child’s safety.

“We encourage parents to do the ‘Tag, you’re it’ game with one another. So you basically tag an adult who is responsible for keeping an eye on that child for a period of time so that there’s always supervision.”
 

Be prepared

There are a few things you can do now to be ready in case your child slips away. These measures may help find the child quickly.

Take photos today. Keep a full-length shot and a head shot of your child and store them electronically. If your child wanders away, you can immediately send the images to law enforcement to help them search.

Write a 911 script. Have this document ready in case your child wanders. It describes, among other things, points of interest that might draw your child, as well as locations of nearby bodies of water. By having it all written down, you’ll be able to share the information quickly with first responders. The Montgomery County Police Department has a “Wandering 911 Script” that you can download and use.

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

With monkeypox now circulating in the United States, expecting mothers may worry about what might happen if they contract the infection while pregnant.

As of today, 25 cases of monkeypox have been confirmed in the United States since the outbreak began in early May, according to the U.S. Centers for Disease Control and Prevention. Although none of those cases has involved a pregnant person, the World Health Organization says monkeypox can pass from mother to fetus before delivery or to newborns by close contact during and after birth.

The case count could grow as the agency continues to investigate potential infections of the virus. In a conference call Friday, health officials stressed the importance of contact tracing, testing, and vaccine treatment.

As physicians in the United States are scrambling for information on ways to treat patients, a new study, published in Ultrasound in Obstetrics & Gynecology, could help clinicians better care for pregnant people infected with monkeypox. The authors advise consistently monitoring the fetus for infection and conducting regular ultrasounds, among other precautions. 

Asma Khalil, MBBCh, MD, a professor of obstetrics and fetal medicine at St. George’s University, London, and lead author of the new study, said the monkeypox outbreak outside Africa caught many clinicians by surprise.

“We quickly realized very few physicians caring for pregnant women knew anything at all about monkeypox and how it affects pregnancy,” Dr. Khalil told this news organization. “Clinicians caring for pregnant women are likely to be faced soon with pregnant women concerned they may have the infection – because they have a rash, for example – or indeed pregnant women who do have the infection.”

According to the CDC, monkeypox can be transmitted through direct contact with the rash, sores, or scabs caused by the virus, as well as contact with clothing, bedding, towels, or other surfaces used by an infected person. Respiratory droplets and oral fluids from a person with monkeypox have also been linked to spread of the virus, as has sexual activity.

Although the condition is rarely fatal, infants and young children are at the greatest risk of developing severe symptoms, health officials said. 

The U.S. Food and Drug Administration has approved a monkeypox vaccine, Jynneos (Bavarian Nordic A/S), for general use, but it has not been specifically approved for pregnant people. However, a study of 300 pregnant women who received the vaccine reported no adverse reactions or failed pregnancies linked to the shots.

The new review suggests that women who have a confirmed infection during pregnancy should have a doctor closely monitor the fetus until birth.

If the fetus is over 26 weeks or if the mother is unwell, the fetus should be cared for with heart monitoring, either by a doctor or remotely every 2-3 days. Ultrasounds should be performed regularly to confirm that the fetus is still growing well and that the placenta is functioning properly.

Further into the pregnancy, monitoring should include measurements of the fetus and detailed assessment of the fetal organs and the amniotic fluid. Once the infection is resolved, the risk to the fetus is small, according to Dr. Khalil. However, since data are limited, she recommended an ultrasound scan every 2-4 weeks. At birth, for the protection of the infant and the mother, the baby should be isolated until infection is no longer a risk.

The Royal College of Obstetricians & Gynaecologists is preparing guidance on the management of monkeypox in pregnant people, Dr. Khalil said. The American College of Obstetricians and Gynecologists said it is “relying on the CDC for the time being,” according to a spokesperson for ACOG. 

“There is a clear need for further research in this area,” Dr. Khalil said. “The current outbreak is an ideal opportunity to make this happen.”

Dr. Khalil has disclosed no relevant financial relationships.

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

With monkeypox now circulating in the United States, expecting mothers may worry about what might happen if they contract the infection while pregnant.

As of today, 25 cases of monkeypox have been confirmed in the United States since the outbreak began in early May, according to the U.S. Centers for Disease Control and Prevention. Although none of those cases has involved a pregnant person, the World Health Organization says monkeypox can pass from mother to fetus before delivery or to newborns by close contact during and after birth.

The case count could grow as the agency continues to investigate potential infections of the virus. In a conference call Friday, health officials stressed the importance of contact tracing, testing, and vaccine treatment.

As physicians in the United States are scrambling for information on ways to treat patients, a new study, published in Ultrasound in Obstetrics & Gynecology, could help clinicians better care for pregnant people infected with monkeypox. The authors advise consistently monitoring the fetus for infection and conducting regular ultrasounds, among other precautions. 

Asma Khalil, MBBCh, MD, a professor of obstetrics and fetal medicine at St. George’s University, London, and lead author of the new study, said the monkeypox outbreak outside Africa caught many clinicians by surprise.

“We quickly realized very few physicians caring for pregnant women knew anything at all about monkeypox and how it affects pregnancy,” Dr. Khalil told this news organization. “Clinicians caring for pregnant women are likely to be faced soon with pregnant women concerned they may have the infection – because they have a rash, for example – or indeed pregnant women who do have the infection.”

According to the CDC, monkeypox can be transmitted through direct contact with the rash, sores, or scabs caused by the virus, as well as contact with clothing, bedding, towels, or other surfaces used by an infected person. Respiratory droplets and oral fluids from a person with monkeypox have also been linked to spread of the virus, as has sexual activity.

Although the condition is rarely fatal, infants and young children are at the greatest risk of developing severe symptoms, health officials said. 

The U.S. Food and Drug Administration has approved a monkeypox vaccine, Jynneos (Bavarian Nordic A/S), for general use, but it has not been specifically approved for pregnant people. However, a study of 300 pregnant women who received the vaccine reported no adverse reactions or failed pregnancies linked to the shots.

The new review suggests that women who have a confirmed infection during pregnancy should have a doctor closely monitor the fetus until birth.

If the fetus is over 26 weeks or if the mother is unwell, the fetus should be cared for with heart monitoring, either by a doctor or remotely every 2-3 days. Ultrasounds should be performed regularly to confirm that the fetus is still growing well and that the placenta is functioning properly.

Further into the pregnancy, monitoring should include measurements of the fetus and detailed assessment of the fetal organs and the amniotic fluid. Once the infection is resolved, the risk to the fetus is small, according to Dr. Khalil. However, since data are limited, she recommended an ultrasound scan every 2-4 weeks. At birth, for the protection of the infant and the mother, the baby should be isolated until infection is no longer a risk.

The Royal College of Obstetricians & Gynaecologists is preparing guidance on the management of monkeypox in pregnant people, Dr. Khalil said. The American College of Obstetricians and Gynecologists said it is “relying on the CDC for the time being,” according to a spokesperson for ACOG. 

“There is a clear need for further research in this area,” Dr. Khalil said. “The current outbreak is an ideal opportunity to make this happen.”

Dr. Khalil has disclosed no relevant financial relationships.

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

With monkeypox now circulating in the United States, expecting mothers may worry about what might happen if they contract the infection while pregnant.

As of today, 25 cases of monkeypox have been confirmed in the United States since the outbreak began in early May, according to the U.S. Centers for Disease Control and Prevention. Although none of those cases has involved a pregnant person, the World Health Organization says monkeypox can pass from mother to fetus before delivery or to newborns by close contact during and after birth.

The case count could grow as the agency continues to investigate potential infections of the virus. In a conference call Friday, health officials stressed the importance of contact tracing, testing, and vaccine treatment.

As physicians in the United States are scrambling for information on ways to treat patients, a new study, published in Ultrasound in Obstetrics & Gynecology, could help clinicians better care for pregnant people infected with monkeypox. The authors advise consistently monitoring the fetus for infection and conducting regular ultrasounds, among other precautions. 

Asma Khalil, MBBCh, MD, a professor of obstetrics and fetal medicine at St. George’s University, London, and lead author of the new study, said the monkeypox outbreak outside Africa caught many clinicians by surprise.

“We quickly realized very few physicians caring for pregnant women knew anything at all about monkeypox and how it affects pregnancy,” Dr. Khalil told this news organization. “Clinicians caring for pregnant women are likely to be faced soon with pregnant women concerned they may have the infection – because they have a rash, for example – or indeed pregnant women who do have the infection.”

According to the CDC, monkeypox can be transmitted through direct contact with the rash, sores, or scabs caused by the virus, as well as contact with clothing, bedding, towels, or other surfaces used by an infected person. Respiratory droplets and oral fluids from a person with monkeypox have also been linked to spread of the virus, as has sexual activity.

Although the condition is rarely fatal, infants and young children are at the greatest risk of developing severe symptoms, health officials said. 

The U.S. Food and Drug Administration has approved a monkeypox vaccine, Jynneos (Bavarian Nordic A/S), for general use, but it has not been specifically approved for pregnant people. However, a study of 300 pregnant women who received the vaccine reported no adverse reactions or failed pregnancies linked to the shots.

The new review suggests that women who have a confirmed infection during pregnancy should have a doctor closely monitor the fetus until birth.

If the fetus is over 26 weeks or if the mother is unwell, the fetus should be cared for with heart monitoring, either by a doctor or remotely every 2-3 days. Ultrasounds should be performed regularly to confirm that the fetus is still growing well and that the placenta is functioning properly.

Further into the pregnancy, monitoring should include measurements of the fetus and detailed assessment of the fetal organs and the amniotic fluid. Once the infection is resolved, the risk to the fetus is small, according to Dr. Khalil. However, since data are limited, she recommended an ultrasound scan every 2-4 weeks. At birth, for the protection of the infant and the mother, the baby should be isolated until infection is no longer a risk.

The Royal College of Obstetricians & Gynaecologists is preparing guidance on the management of monkeypox in pregnant people, Dr. Khalil said. The American College of Obstetricians and Gynecologists said it is “relying on the CDC for the time being,” according to a spokesperson for ACOG. 

“There is a clear need for further research in this area,” Dr. Khalil said. “The current outbreak is an ideal opportunity to make this happen.”

Dr. Khalil has disclosed no relevant financial relationships.

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

As a physician who has had a career-long obsession with the underappreciated value of sleep, a recent study published in the journal Child Development caught my eye. The findings presented by a group of Australian-based psychologists and educators suggest a positive association between napping and learning by preschool children. While the study itself relied on a very small sample and may not prove to be repeatable, the authors included in their introduction an excellent discussion of a large collection of recent studies supporting the educational benefit of sleep in general and napping in particular.

Although sleep seems to finally be receiving some of the attention it deserves, I am still concerned that as a profession we are failing to give it the appropriate weight at our health maintenance visits. This is particularly true of napping. Understandably, napping doesn’t feel urgent to parents in those turbulent first 4 or 5 months of night wakings and erratic settling. However, as a child approaches the 6-month milestone, napping is a topic ripe for well-considered anticipatory guidance.

When the recurrent cycles of awake-eat-sleep begin to develop into a somewhat predictable pattern and solid food is introduced, it’s time to suggest to parents a strategy that will encourage a napping pattern that will hopefully habituate into toddlerhood and beyond.

It can begin simply as a matter of defining the feeding in the middle of the day as lunch and then programming the period immediately following that meal as a siesta – a segment of the day completely reserved for rest. Many warm-weather countries have been using this strategy for centuries. Try to go to the pharmacy to pick up a prescription at 2 o’clock in the afternoon in rural Spain. It just ain’t gonna happen.

Most adults and children I know seem to be sleepy during this midday postprandial period. It makes more than a little sense to harness this natural drowsiness into creating a napping habit. However, the challenge for many young families is controlling their schedule to create a period of time when nothing else is going on in the child’s environment, leaving sleep as the only option. For some parents this requires the discipline to pause their own lives long enough so that the children realize that they aren’t missing out on something fun. This means no TV, no phone conversations, no visitors. Obviously, it also means not scheduling any appointments during this siesta period. Skilled day care providers have been doing this for years. But the message hasn’t seeped into the general population and sadly I occasionally see mothers with toddlers in the grocery store at 1 in the afternoon.

Once the nap/siesta is firmly welded to lunch, this gives the parent the ability to make minor adjustments that reflect the child’s stamina. If the child seems to be tiring/getting grumpy, serve up lunch a bit early and the restorative nap follows. As the child gets older and his or her stamina improves he or she may not be sleepy but the siesta remains as a quiet time. Some days it may be a nap, some days just a rest for an hour. By counseling parents to define the period after lunch as a siesta you will be helping them avoid that dreaded transition period called “giving up the nap.”

You may already be including this strategy in your anticipatory guidance. It may help to add to your advice the accumulating evidence that napping may play an important role in the child’s development and education.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

As a physician who has had a career-long obsession with the underappreciated value of sleep, a recent study published in the journal Child Development caught my eye. The findings presented by a group of Australian-based psychologists and educators suggest a positive association between napping and learning by preschool children. While the study itself relied on a very small sample and may not prove to be repeatable, the authors included in their introduction an excellent discussion of a large collection of recent studies supporting the educational benefit of sleep in general and napping in particular.

Although sleep seems to finally be receiving some of the attention it deserves, I am still concerned that as a profession we are failing to give it the appropriate weight at our health maintenance visits. This is particularly true of napping. Understandably, napping doesn’t feel urgent to parents in those turbulent first 4 or 5 months of night wakings and erratic settling. However, as a child approaches the 6-month milestone, napping is a topic ripe for well-considered anticipatory guidance.

When the recurrent cycles of awake-eat-sleep begin to develop into a somewhat predictable pattern and solid food is introduced, it’s time to suggest to parents a strategy that will encourage a napping pattern that will hopefully habituate into toddlerhood and beyond.

It can begin simply as a matter of defining the feeding in the middle of the day as lunch and then programming the period immediately following that meal as a siesta – a segment of the day completely reserved for rest. Many warm-weather countries have been using this strategy for centuries. Try to go to the pharmacy to pick up a prescription at 2 o’clock in the afternoon in rural Spain. It just ain’t gonna happen.

Most adults and children I know seem to be sleepy during this midday postprandial period. It makes more than a little sense to harness this natural drowsiness into creating a napping habit. However, the challenge for many young families is controlling their schedule to create a period of time when nothing else is going on in the child’s environment, leaving sleep as the only option. For some parents this requires the discipline to pause their own lives long enough so that the children realize that they aren’t missing out on something fun. This means no TV, no phone conversations, no visitors. Obviously, it also means not scheduling any appointments during this siesta period. Skilled day care providers have been doing this for years. But the message hasn’t seeped into the general population and sadly I occasionally see mothers with toddlers in the grocery store at 1 in the afternoon.

Once the nap/siesta is firmly welded to lunch, this gives the parent the ability to make minor adjustments that reflect the child’s stamina. If the child seems to be tiring/getting grumpy, serve up lunch a bit early and the restorative nap follows. As the child gets older and his or her stamina improves he or she may not be sleepy but the siesta remains as a quiet time. Some days it may be a nap, some days just a rest for an hour. By counseling parents to define the period after lunch as a siesta you will be helping them avoid that dreaded transition period called “giving up the nap.”

You may already be including this strategy in your anticipatory guidance. It may help to add to your advice the accumulating evidence that napping may play an important role in the child’s development and education.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

As a physician who has had a career-long obsession with the underappreciated value of sleep, a recent study published in the journal Child Development caught my eye. The findings presented by a group of Australian-based psychologists and educators suggest a positive association between napping and learning by preschool children. While the study itself relied on a very small sample and may not prove to be repeatable, the authors included in their introduction an excellent discussion of a large collection of recent studies supporting the educational benefit of sleep in general and napping in particular.

Although sleep seems to finally be receiving some of the attention it deserves, I am still concerned that as a profession we are failing to give it the appropriate weight at our health maintenance visits. This is particularly true of napping. Understandably, napping doesn’t feel urgent to parents in those turbulent first 4 or 5 months of night wakings and erratic settling. However, as a child approaches the 6-month milestone, napping is a topic ripe for well-considered anticipatory guidance.

When the recurrent cycles of awake-eat-sleep begin to develop into a somewhat predictable pattern and solid food is introduced, it’s time to suggest to parents a strategy that will encourage a napping pattern that will hopefully habituate into toddlerhood and beyond.

It can begin simply as a matter of defining the feeding in the middle of the day as lunch and then programming the period immediately following that meal as a siesta – a segment of the day completely reserved for rest. Many warm-weather countries have been using this strategy for centuries. Try to go to the pharmacy to pick up a prescription at 2 o’clock in the afternoon in rural Spain. It just ain’t gonna happen.

Most adults and children I know seem to be sleepy during this midday postprandial period. It makes more than a little sense to harness this natural drowsiness into creating a napping habit. However, the challenge for many young families is controlling their schedule to create a period of time when nothing else is going on in the child’s environment, leaving sleep as the only option. For some parents this requires the discipline to pause their own lives long enough so that the children realize that they aren’t missing out on something fun. This means no TV, no phone conversations, no visitors. Obviously, it also means not scheduling any appointments during this siesta period. Skilled day care providers have been doing this for years. But the message hasn’t seeped into the general population and sadly I occasionally see mothers with toddlers in the grocery store at 1 in the afternoon.

Once the nap/siesta is firmly welded to lunch, this gives the parent the ability to make minor adjustments that reflect the child’s stamina. If the child seems to be tiring/getting grumpy, serve up lunch a bit early and the restorative nap follows. As the child gets older and his or her stamina improves he or she may not be sleepy but the siesta remains as a quiet time. Some days it may be a nap, some days just a rest for an hour. By counseling parents to define the period after lunch as a siesta you will be helping them avoid that dreaded transition period called “giving up the nap.”

You may already be including this strategy in your anticipatory guidance. It may help to add to your advice the accumulating evidence that napping may play an important role in the child’s development and education.

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Now hot off the press from the “always-guessed-it-was-true-but-now-you-know-it” department comes a multinational study that looked at childhood cardiovascular risk factors and longevity.

Using data collected from individuals in Finland, Australia, and the United States the International Childhood Cardiovascular Cohorts Consortium Outcomes Study investigators sought links between subjects’ body mass index, systolic blood pressure, total cholesterol, blood triglyceride level, and smoking in childhood with cardiovascular disease and outcomes as they aged into adulthood.

The children were initially enrolled in the 1970s and 1980s. The adult evaluations were done in 2015-2019 when the subjects’ average age was 46. Of the 40,000 individuals who originally entered the study, 800 were found to have cardiovascular events of which over 300 had resulted in death. I found these numbers a bit surprising given the relatively young age at which the follow-up data were collected.

What was less surprising is that people with higher than normal values for all five risk factors as children had nearly three times the risk of cardiovascular disease as adults. Researchers found that smoking at a young age was biggest risk factor with body mass index, systolic blood pressure, blood triglycerides, and cholesterol following in descending order. They also found that adults who were obese as children had triple the risk of cardiovascular disease as adults. High blood pressure in childhood doubled the risk.

It will be interesting to see if and how these trends change as the study population ages. It could be that the effect of these childhood risk factors is blunted as the those segments at the highest risk die off and/or risk- associated behaviors adopted in adulthood become more prominent. But, it feels more likely that the childhood risk factors will remain as major contributors.

Is this just another ho-hum-told-you-so study or does it have some special relevance for us as pediatricians? At a minimum these findings should inspire us to stick with our calling to commit ourselves to the health of children. A healthy adult population is clearly our legacy.

Of course the two individual risk factors in childhood that appear to be the most potent in adulthood, obesity and smoking, are also the most frustrating for pediatricians to address. However, the study suggests that we should rejoice in those few successes when we achieve them. Childhood obesity has been a tough nut to crack. On the other hand, the societal change that has made great strides in adult smoking over the last half century should encourage us that our work with the pediatric population will eventually bring rewards.

Smoking and obesity can include components of both patient and parental behavior. Monitoring cholesterol, triglycerides, and blood pressure hinges on our behavior as providers. Although there have been recent recommendations that we be more attentive, we don’t have a strong history when it comes to detecting and addressing high blood pressure in children. This study should serve as an another reminder to take blood pressure more seriously.

I was surprised and somewhat disappointed that I first learned about the results of this study in an email newsletter from the medical school I attended. I would have hoped that a paper like this from a well known peer-reviewed journal with a clear message about the relationship of childhood health and longevity should have been picked up quickly by the lay press. Again, this leaves it to us to promote the message that the health of children is important in and of itself but plays a critical role in the health of adults.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Now hot off the press from the “always-guessed-it-was-true-but-now-you-know-it” department comes a multinational study that looked at childhood cardiovascular risk factors and longevity.

Using data collected from individuals in Finland, Australia, and the United States the International Childhood Cardiovascular Cohorts Consortium Outcomes Study investigators sought links between subjects’ body mass index, systolic blood pressure, total cholesterol, blood triglyceride level, and smoking in childhood with cardiovascular disease and outcomes as they aged into adulthood.

The children were initially enrolled in the 1970s and 1980s. The adult evaluations were done in 2015-2019 when the subjects’ average age was 46. Of the 40,000 individuals who originally entered the study, 800 were found to have cardiovascular events of which over 300 had resulted in death. I found these numbers a bit surprising given the relatively young age at which the follow-up data were collected.

What was less surprising is that people with higher than normal values for all five risk factors as children had nearly three times the risk of cardiovascular disease as adults. Researchers found that smoking at a young age was biggest risk factor with body mass index, systolic blood pressure, blood triglycerides, and cholesterol following in descending order. They also found that adults who were obese as children had triple the risk of cardiovascular disease as adults. High blood pressure in childhood doubled the risk.

It will be interesting to see if and how these trends change as the study population ages. It could be that the effect of these childhood risk factors is blunted as the those segments at the highest risk die off and/or risk- associated behaviors adopted in adulthood become more prominent. But, it feels more likely that the childhood risk factors will remain as major contributors.

Is this just another ho-hum-told-you-so study or does it have some special relevance for us as pediatricians? At a minimum these findings should inspire us to stick with our calling to commit ourselves to the health of children. A healthy adult population is clearly our legacy.

Of course the two individual risk factors in childhood that appear to be the most potent in adulthood, obesity and smoking, are also the most frustrating for pediatricians to address. However, the study suggests that we should rejoice in those few successes when we achieve them. Childhood obesity has been a tough nut to crack. On the other hand, the societal change that has made great strides in adult smoking over the last half century should encourage us that our work with the pediatric population will eventually bring rewards.

Smoking and obesity can include components of both patient and parental behavior. Monitoring cholesterol, triglycerides, and blood pressure hinges on our behavior as providers. Although there have been recent recommendations that we be more attentive, we don’t have a strong history when it comes to detecting and addressing high blood pressure in children. This study should serve as an another reminder to take blood pressure more seriously.

I was surprised and somewhat disappointed that I first learned about the results of this study in an email newsletter from the medical school I attended. I would have hoped that a paper like this from a well known peer-reviewed journal with a clear message about the relationship of childhood health and longevity should have been picked up quickly by the lay press. Again, this leaves it to us to promote the message that the health of children is important in and of itself but plays a critical role in the health of adults.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

Now hot off the press from the “always-guessed-it-was-true-but-now-you-know-it” department comes a multinational study that looked at childhood cardiovascular risk factors and longevity.

Using data collected from individuals in Finland, Australia, and the United States the International Childhood Cardiovascular Cohorts Consortium Outcomes Study investigators sought links between subjects’ body mass index, systolic blood pressure, total cholesterol, blood triglyceride level, and smoking in childhood with cardiovascular disease and outcomes as they aged into adulthood.

The children were initially enrolled in the 1970s and 1980s. The adult evaluations were done in 2015-2019 when the subjects’ average age was 46. Of the 40,000 individuals who originally entered the study, 800 were found to have cardiovascular events of which over 300 had resulted in death. I found these numbers a bit surprising given the relatively young age at which the follow-up data were collected.

What was less surprising is that people with higher than normal values for all five risk factors as children had nearly three times the risk of cardiovascular disease as adults. Researchers found that smoking at a young age was biggest risk factor with body mass index, systolic blood pressure, blood triglycerides, and cholesterol following in descending order. They also found that adults who were obese as children had triple the risk of cardiovascular disease as adults. High blood pressure in childhood doubled the risk.

It will be interesting to see if and how these trends change as the study population ages. It could be that the effect of these childhood risk factors is blunted as the those segments at the highest risk die off and/or risk- associated behaviors adopted in adulthood become more prominent. But, it feels more likely that the childhood risk factors will remain as major contributors.

Is this just another ho-hum-told-you-so study or does it have some special relevance for us as pediatricians? At a minimum these findings should inspire us to stick with our calling to commit ourselves to the health of children. A healthy adult population is clearly our legacy.

Of course the two individual risk factors in childhood that appear to be the most potent in adulthood, obesity and smoking, are also the most frustrating for pediatricians to address. However, the study suggests that we should rejoice in those few successes when we achieve them. Childhood obesity has been a tough nut to crack. On the other hand, the societal change that has made great strides in adult smoking over the last half century should encourage us that our work with the pediatric population will eventually bring rewards.

Smoking and obesity can include components of both patient and parental behavior. Monitoring cholesterol, triglycerides, and blood pressure hinges on our behavior as providers. Although there have been recent recommendations that we be more attentive, we don’t have a strong history when it comes to detecting and addressing high blood pressure in children. This study should serve as an another reminder to take blood pressure more seriously.

I was surprised and somewhat disappointed that I first learned about the results of this study in an email newsletter from the medical school I attended. I would have hoped that a paper like this from a well known peer-reviewed journal with a clear message about the relationship of childhood health and longevity should have been picked up quickly by the lay press. Again, this leaves it to us to promote the message that the health of children is important in and of itself but plays a critical role in the health of adults.
 

Dr. Wilkoff practiced primary care pediatrics in Brunswick, Maine, for nearly 40 years. He has authored several books on behavioral pediatrics, including “How to Say No to Your Toddler.” Other than a Littman stethoscope he accepted as a first-year medical student in 1966, Dr. Wilkoff reports having nothing to disclose. Email him at [email protected].

CHICAGO – New results from a trial in patients with newly diagnosed multiple myeloma (MM) offer some answers to questions about which treatment route to choose.

The trial, known as DETERMINATION, found that newly diagnosed patients treated with a triplet of drugs had longer progression-free survival (PFS) if they received an autologous stem cell transplant (ASCT) soon after the drug therapy than if they simply had their stem cells collected for a possible future transplant.

Patients who received the triplet of lenalidomide, bortezomib, and dexamethasone (RVD) plus ASCT had a median PFS of 67.5 months, compared with 46.2 months for those who received RVD but did not have a transplant soon after.

However, patients were just as likely to be alive more than 6 years after treatment regardless of whether or not they underwent an immediate stem cell transplant.

In addition, treatment-related adverse events of grade 3 or above were higher in the group that received the transplant immediately after the triplet therapy.  

The results were presented during a plenary session at the American Society of Clinical Oncology annual meeting and simultaneously published in the New England Journal of Medicine.

“Our findings confirm the PFS benefit of transplantation as first-line treatment for patients with myeloma and confirms stem cell transplant as a standard of care with certain triplet therapy,” said lead author Paul G. Richardson, MD, professor of medicine, Harvard Medical School, and clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center at Dana Farber Cancer Institute, Boston.

Another finding from the trial was that the use of maintenance lenalidomide in both groups continuously until progression conferred substantial clinical benefit.

“We can also say that the use of lenalidomide maintenance therapy is also a standard of care,” he added.
 

Study details

In this trial, Dr. Richardson and colleagues randomly assigned 873 patients newly diagnosed with multiple myeloma to the RVD-alone group (n = 357) or the transplantation group (n = 365). All patients had received one cycle of RVD prior to randomization and then received two additional RVD cycles plus stem-cell mobilization followed by either five additional RVD cycles (the RVD-alone group) or high-dose melphalan plus ASCT followed by two additional RVD cycles (the transplantation group). Lenalidomide was administered to all patients until disease progression, unacceptable side effects, or both.

At a median follow-up of 76.0 months, the risk of disease progression or death was 53% higher among patients who received RVD alone versus the transplantation group (hazard ratio [HR], 1.53; P < .001). The median duration of PFS among patients with a high-risk cytogenetic profile was 55.5 vs. 17.1 months, favoring the transplantation group.

The percentage of patients who were alive without progression at 5 years was 58.4% vs 41.6%, respectively (HR, 1.66) and median duration of response was 56.4 vs 38.9 months, also favoring transplantation (HR, 1.45).

The estimated 5-year overall survival was similar between groups: 80.7% for transplantation and 79.2% for RVD alone (HR for death, 1.10; P > .99). For patients with a high-risk cytogenetic profile, 5-year survival was 63.4% versus 54.3%, respectively.

“This tells us that for patients who had kept transplant in reserve, they had the same overall survival as those who had had a transplant right away, despite there being such impressive initial disease control for the patients in whom transplant was used early,” Dr. Richardson said in a press release from his institution.

Patients who did not undergo immediate transplant received treatment when their disease progressed with newer and active therapies, such as monoclonal antibodies and/or next-generation novel agents, he noted. Only 28% of patients used the reserve option of a transplant.

“It demonstrates the extent to which patients now have options and that we have new data to guide them in balancing the pluses and minuses of each approach,” he added.

When looking at safety, the authors noted that the most common treatment-related adverse events of grade 3 or higher occurred in 279 patients (78.2%) in the RVD-alone group and 344 patients (94.2%) in the transplantation group. Of those patients, 60.5% and 89.9%, respectively, reported hematologic events of grade 3 or higher (P < .001). The 5-year cumulative incidence of invasive second primary cancers was similar in both cohorts (RVD-alone group, 4.9%; transplantation group, 6.5%).

However, while the risk of secondary cancers was similar between groups, Dr. Richardson noted that there was a higher incidence of acute myeloid leukemia and myelodysplastic syndromes in the transplant cohort.

“There was also a significant drop in quality of life across transplant procedures, but the good news is that it was recoverable rapidly,” he said. “What is also really important is that we have prospective, multicenter, national comparative data on toxicity. That’s very important for providing patients with a choice as they move forward with their treatment plan.”

He noted that treatment continues to evolve. “This study was designed in 2009, begun in 2010, and now there is mature data in 2022,” Dr. Richardson said. “This is particularly relevant as we have now further improved the induction treatment for younger patients with newly diagnosed myeloma using quadruplet regimens incorporating monoclonal antibodies and novel next-generation therapies. The results from these studies are extremely exciting.

“Now more than ever, treatment for multiple myeloma can be adapted for each patient,” Dr. Richardson said. “Our study provides important information about the benefits of transplant in the era of highly effective novel therapies and continuous maintenance, as well as the potential risks, to help patients and their physicians decide what approach may be best for them. This is particularly relevant as we have now further improved the induction treatment for younger patients with newly diagnosed myeloma using quadruplet regimens incorporating monoclonal antibodies, such as RVD combined with daratumumab.”
 

Lack of difference in overall survival

These new results further support an already established role of autologous hematopoietic stem cell transplantation in the management of patients with multiple myeloma, said Samer Al-Homsi, MD, clinical professor of medicine and director of the blood and marrow transplant program at Perlmutter Cancer Center, NYU Langone, New York, who was approached for comment.

“The treatment regimen is applicable to patients who are determined by an expert in transplantation to be fit to receive autologous hematopoietic transplantation,” he added. “Although this study, like many others, establishes hematopoietic stem cell transplantation as part of the standard of care in multiple myeloma, only a fraction of patients are actually offered this important modality of treatment for a variety of reasons, including provider bias,” he noted. “In fact, although improvement in supportive care has enhanced the safety of the procedure, many patients are denied this therapy.” 

Dr. Al-Homsi noted that the lack of difference in overall survival might be due to the fact that some patients (28%) in the RVD-alone group did end up undergoing transplantation at the time of progression. “Also, longer follow-up might reveal a difference in overall survival,” he said.

The toxicities are manageable, and the incidence of secondary malignancies was not significantly different between cohorts. “However,” he emphasized, “lenalidomide has been associated in other studies with increased incidence of secondary malignancies and it must be noted that this study used extended administration of lenalidomide until progression.” 

Support for this study was provided by grants to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute, the National Cancer Institute, R. J. Corman Multiple Myeloma Foundation, Celgene/Bristol Myers Squibb, and Millennium/Takeda Pharmaceutical. Dr. Richardson has reported relationships with Celgene, Janssen, Jazz Pharmaceuticals, Karyopharm Therapeutics, Oncopeptides, Sanofi, Secura Bio, Takeda, and Bristol Myers Squibb. Dr. Al-Homsi has reported no relevant financial relationships.

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

CHICAGO – New results from a trial in patients with newly diagnosed multiple myeloma (MM) offer some answers to questions about which treatment route to choose.

The trial, known as DETERMINATION, found that newly diagnosed patients treated with a triplet of drugs had longer progression-free survival (PFS) if they received an autologous stem cell transplant (ASCT) soon after the drug therapy than if they simply had their stem cells collected for a possible future transplant.

Patients who received the triplet of lenalidomide, bortezomib, and dexamethasone (RVD) plus ASCT had a median PFS of 67.5 months, compared with 46.2 months for those who received RVD but did not have a transplant soon after.

However, patients were just as likely to be alive more than 6 years after treatment regardless of whether or not they underwent an immediate stem cell transplant.

In addition, treatment-related adverse events of grade 3 or above were higher in the group that received the transplant immediately after the triplet therapy.  

The results were presented during a plenary session at the American Society of Clinical Oncology annual meeting and simultaneously published in the New England Journal of Medicine.

“Our findings confirm the PFS benefit of transplantation as first-line treatment for patients with myeloma and confirms stem cell transplant as a standard of care with certain triplet therapy,” said lead author Paul G. Richardson, MD, professor of medicine, Harvard Medical School, and clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center at Dana Farber Cancer Institute, Boston.

Another finding from the trial was that the use of maintenance lenalidomide in both groups continuously until progression conferred substantial clinical benefit.

“We can also say that the use of lenalidomide maintenance therapy is also a standard of care,” he added.
 

Study details

In this trial, Dr. Richardson and colleagues randomly assigned 873 patients newly diagnosed with multiple myeloma to the RVD-alone group (n = 357) or the transplantation group (n = 365). All patients had received one cycle of RVD prior to randomization and then received two additional RVD cycles plus stem-cell mobilization followed by either five additional RVD cycles (the RVD-alone group) or high-dose melphalan plus ASCT followed by two additional RVD cycles (the transplantation group). Lenalidomide was administered to all patients until disease progression, unacceptable side effects, or both.

At a median follow-up of 76.0 months, the risk of disease progression or death was 53% higher among patients who received RVD alone versus the transplantation group (hazard ratio [HR], 1.53; P < .001). The median duration of PFS among patients with a high-risk cytogenetic profile was 55.5 vs. 17.1 months, favoring the transplantation group.

The percentage of patients who were alive without progression at 5 years was 58.4% vs 41.6%, respectively (HR, 1.66) and median duration of response was 56.4 vs 38.9 months, also favoring transplantation (HR, 1.45).

The estimated 5-year overall survival was similar between groups: 80.7% for transplantation and 79.2% for RVD alone (HR for death, 1.10; P > .99). For patients with a high-risk cytogenetic profile, 5-year survival was 63.4% versus 54.3%, respectively.

“This tells us that for patients who had kept transplant in reserve, they had the same overall survival as those who had had a transplant right away, despite there being such impressive initial disease control for the patients in whom transplant was used early,” Dr. Richardson said in a press release from his institution.

Patients who did not undergo immediate transplant received treatment when their disease progressed with newer and active therapies, such as monoclonal antibodies and/or next-generation novel agents, he noted. Only 28% of patients used the reserve option of a transplant.

“It demonstrates the extent to which patients now have options and that we have new data to guide them in balancing the pluses and minuses of each approach,” he added.

When looking at safety, the authors noted that the most common treatment-related adverse events of grade 3 or higher occurred in 279 patients (78.2%) in the RVD-alone group and 344 patients (94.2%) in the transplantation group. Of those patients, 60.5% and 89.9%, respectively, reported hematologic events of grade 3 or higher (P < .001). The 5-year cumulative incidence of invasive second primary cancers was similar in both cohorts (RVD-alone group, 4.9%; transplantation group, 6.5%).

However, while the risk of secondary cancers was similar between groups, Dr. Richardson noted that there was a higher incidence of acute myeloid leukemia and myelodysplastic syndromes in the transplant cohort.

“There was also a significant drop in quality of life across transplant procedures, but the good news is that it was recoverable rapidly,” he said. “What is also really important is that we have prospective, multicenter, national comparative data on toxicity. That’s very important for providing patients with a choice as they move forward with their treatment plan.”

He noted that treatment continues to evolve. “This study was designed in 2009, begun in 2010, and now there is mature data in 2022,” Dr. Richardson said. “This is particularly relevant as we have now further improved the induction treatment for younger patients with newly diagnosed myeloma using quadruplet regimens incorporating monoclonal antibodies and novel next-generation therapies. The results from these studies are extremely exciting.

“Now more than ever, treatment for multiple myeloma can be adapted for each patient,” Dr. Richardson said. “Our study provides important information about the benefits of transplant in the era of highly effective novel therapies and continuous maintenance, as well as the potential risks, to help patients and their physicians decide what approach may be best for them. This is particularly relevant as we have now further improved the induction treatment for younger patients with newly diagnosed myeloma using quadruplet regimens incorporating monoclonal antibodies, such as RVD combined with daratumumab.”
 

Lack of difference in overall survival

These new results further support an already established role of autologous hematopoietic stem cell transplantation in the management of patients with multiple myeloma, said Samer Al-Homsi, MD, clinical professor of medicine and director of the blood and marrow transplant program at Perlmutter Cancer Center, NYU Langone, New York, who was approached for comment.

“The treatment regimen is applicable to patients who are determined by an expert in transplantation to be fit to receive autologous hematopoietic transplantation,” he added. “Although this study, like many others, establishes hematopoietic stem cell transplantation as part of the standard of care in multiple myeloma, only a fraction of patients are actually offered this important modality of treatment for a variety of reasons, including provider bias,” he noted. “In fact, although improvement in supportive care has enhanced the safety of the procedure, many patients are denied this therapy.” 

Dr. Al-Homsi noted that the lack of difference in overall survival might be due to the fact that some patients (28%) in the RVD-alone group did end up undergoing transplantation at the time of progression. “Also, longer follow-up might reveal a difference in overall survival,” he said.

The toxicities are manageable, and the incidence of secondary malignancies was not significantly different between cohorts. “However,” he emphasized, “lenalidomide has been associated in other studies with increased incidence of secondary malignancies and it must be noted that this study used extended administration of lenalidomide until progression.” 

Support for this study was provided by grants to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute, the National Cancer Institute, R. J. Corman Multiple Myeloma Foundation, Celgene/Bristol Myers Squibb, and Millennium/Takeda Pharmaceutical. Dr. Richardson has reported relationships with Celgene, Janssen, Jazz Pharmaceuticals, Karyopharm Therapeutics, Oncopeptides, Sanofi, Secura Bio, Takeda, and Bristol Myers Squibb. Dr. Al-Homsi has reported no relevant financial relationships.

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

CHICAGO – New results from a trial in patients with newly diagnosed multiple myeloma (MM) offer some answers to questions about which treatment route to choose.

The trial, known as DETERMINATION, found that newly diagnosed patients treated with a triplet of drugs had longer progression-free survival (PFS) if they received an autologous stem cell transplant (ASCT) soon after the drug therapy than if they simply had their stem cells collected for a possible future transplant.

Patients who received the triplet of lenalidomide, bortezomib, and dexamethasone (RVD) plus ASCT had a median PFS of 67.5 months, compared with 46.2 months for those who received RVD but did not have a transplant soon after.

However, patients were just as likely to be alive more than 6 years after treatment regardless of whether or not they underwent an immediate stem cell transplant.

In addition, treatment-related adverse events of grade 3 or above were higher in the group that received the transplant immediately after the triplet therapy.  

The results were presented during a plenary session at the American Society of Clinical Oncology annual meeting and simultaneously published in the New England Journal of Medicine.

“Our findings confirm the PFS benefit of transplantation as first-line treatment for patients with myeloma and confirms stem cell transplant as a standard of care with certain triplet therapy,” said lead author Paul G. Richardson, MD, professor of medicine, Harvard Medical School, and clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center at Dana Farber Cancer Institute, Boston.

Another finding from the trial was that the use of maintenance lenalidomide in both groups continuously until progression conferred substantial clinical benefit.

“We can also say that the use of lenalidomide maintenance therapy is also a standard of care,” he added.
 

Study details

In this trial, Dr. Richardson and colleagues randomly assigned 873 patients newly diagnosed with multiple myeloma to the RVD-alone group (n = 357) or the transplantation group (n = 365). All patients had received one cycle of RVD prior to randomization and then received two additional RVD cycles plus stem-cell mobilization followed by either five additional RVD cycles (the RVD-alone group) or high-dose melphalan plus ASCT followed by two additional RVD cycles (the transplantation group). Lenalidomide was administered to all patients until disease progression, unacceptable side effects, or both.

At a median follow-up of 76.0 months, the risk of disease progression or death was 53% higher among patients who received RVD alone versus the transplantation group (hazard ratio [HR], 1.53; P < .001). The median duration of PFS among patients with a high-risk cytogenetic profile was 55.5 vs. 17.1 months, favoring the transplantation group.

The percentage of patients who were alive without progression at 5 years was 58.4% vs 41.6%, respectively (HR, 1.66) and median duration of response was 56.4 vs 38.9 months, also favoring transplantation (HR, 1.45).

The estimated 5-year overall survival was similar between groups: 80.7% for transplantation and 79.2% for RVD alone (HR for death, 1.10; P > .99). For patients with a high-risk cytogenetic profile, 5-year survival was 63.4% versus 54.3%, respectively.

“This tells us that for patients who had kept transplant in reserve, they had the same overall survival as those who had had a transplant right away, despite there being such impressive initial disease control for the patients in whom transplant was used early,” Dr. Richardson said in a press release from his institution.

Patients who did not undergo immediate transplant received treatment when their disease progressed with newer and active therapies, such as monoclonal antibodies and/or next-generation novel agents, he noted. Only 28% of patients used the reserve option of a transplant.

“It demonstrates the extent to which patients now have options and that we have new data to guide them in balancing the pluses and minuses of each approach,” he added.

When looking at safety, the authors noted that the most common treatment-related adverse events of grade 3 or higher occurred in 279 patients (78.2%) in the RVD-alone group and 344 patients (94.2%) in the transplantation group. Of those patients, 60.5% and 89.9%, respectively, reported hematologic events of grade 3 or higher (P < .001). The 5-year cumulative incidence of invasive second primary cancers was similar in both cohorts (RVD-alone group, 4.9%; transplantation group, 6.5%).

However, while the risk of secondary cancers was similar between groups, Dr. Richardson noted that there was a higher incidence of acute myeloid leukemia and myelodysplastic syndromes in the transplant cohort.

“There was also a significant drop in quality of life across transplant procedures, but the good news is that it was recoverable rapidly,” he said. “What is also really important is that we have prospective, multicenter, national comparative data on toxicity. That’s very important for providing patients with a choice as they move forward with their treatment plan.”

He noted that treatment continues to evolve. “This study was designed in 2009, begun in 2010, and now there is mature data in 2022,” Dr. Richardson said. “This is particularly relevant as we have now further improved the induction treatment for younger patients with newly diagnosed myeloma using quadruplet regimens incorporating monoclonal antibodies and novel next-generation therapies. The results from these studies are extremely exciting.

“Now more than ever, treatment for multiple myeloma can be adapted for each patient,” Dr. Richardson said. “Our study provides important information about the benefits of transplant in the era of highly effective novel therapies and continuous maintenance, as well as the potential risks, to help patients and their physicians decide what approach may be best for them. This is particularly relevant as we have now further improved the induction treatment for younger patients with newly diagnosed myeloma using quadruplet regimens incorporating monoclonal antibodies, such as RVD combined with daratumumab.”
 

Lack of difference in overall survival

These new results further support an already established role of autologous hematopoietic stem cell transplantation in the management of patients with multiple myeloma, said Samer Al-Homsi, MD, clinical professor of medicine and director of the blood and marrow transplant program at Perlmutter Cancer Center, NYU Langone, New York, who was approached for comment.

“The treatment regimen is applicable to patients who are determined by an expert in transplantation to be fit to receive autologous hematopoietic transplantation,” he added. “Although this study, like many others, establishes hematopoietic stem cell transplantation as part of the standard of care in multiple myeloma, only a fraction of patients are actually offered this important modality of treatment for a variety of reasons, including provider bias,” he noted. “In fact, although improvement in supportive care has enhanced the safety of the procedure, many patients are denied this therapy.” 

Dr. Al-Homsi noted that the lack of difference in overall survival might be due to the fact that some patients (28%) in the RVD-alone group did end up undergoing transplantation at the time of progression. “Also, longer follow-up might reveal a difference in overall survival,” he said.

The toxicities are manageable, and the incidence of secondary malignancies was not significantly different between cohorts. “However,” he emphasized, “lenalidomide has been associated in other studies with increased incidence of secondary malignancies and it must be noted that this study used extended administration of lenalidomide until progression.” 

Support for this study was provided by grants to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute, the National Cancer Institute, R. J. Corman Multiple Myeloma Foundation, Celgene/Bristol Myers Squibb, and Millennium/Takeda Pharmaceutical. Dr. Richardson has reported relationships with Celgene, Janssen, Jazz Pharmaceuticals, Karyopharm Therapeutics, Oncopeptides, Sanofi, Secura Bio, Takeda, and Bristol Myers Squibb. Dr. Al-Homsi has reported no relevant financial relationships.

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

SAN DIEGO – The risk of laser or intense-pulsed light–induced vitiligo from treating vitiligo with either modality appears to be low, but caution is recommended until further studies are carried out.

Those are the preliminary conclusions from a systematic review and survey of experts that Albert Wolkerstorfer, MD, presented during a clinical abstract session at the annual conference of the American Society for Laser Medicine and Surgery.

According to Dr. Wolkerstorfer, a dermatologist at Amsterdam University Medical Center, clinicians are reluctant to perform laser/intense pulsed light (IPL) treatments in patients with vitiligo because of the absence of clear guidelines, so he and his colleagues set out to investigate the risks of laser/IPL-induced vitiligo in patients with vitiligo and to seek out international consensus on recommendations from experts. “There is hardly any literature about it and certainly no guidelines,” he pointed out.

Dr. Wolkerstorfer and his colleagues designed three consecutive studies: A systematic review of laser/IPL-induced vitiligo; an international survey among 14 vitiligo experts from 10 countries about the occurrence of laser‐induced vitiligo, and a Delphi technique aimed at establishing a broad consensus about recommendations for safe use of lasers in vitiligo patients. At the time of the meeting, the Delphi process was still being carried out, so he did not discuss that study.

For the systematic review, the researchers found 11,073 unique hits on PubMed, Embase, and CINAHL using the terms “vitiligo,” “depigmentation,” “hypopigmentation,” and “leukoderma.” Only six case reports of laser/IPL-induced vitiligo were included in the final analysis. Of these, three had de novo vitiligo and three had vitiligo/halo nevi. These cases included two that occurred following treatment of port wine stains with the 585-nm laser; one that occurred following treatment of dyspigmentation with IPL; one that occurred following treatment of hypertrichosis with the 1,064-nm laser, one that occurred following treatment of hypertrichosis with the 755-nm laser, and one case that occurred following treatment of melasma with the ablative laser.

For the international survey of 14 experts from 10 countries, respondents said they had 10,670 new face-to-face vitiligo consultations in the past year. They reported that 30 of the vitiligo cases (0.3%) were likely caused by laser/IPL. Of these 30 cases, 18 (60%) had de novo vitiligo.

Of these cases, vitiligo occurred most frequently after laser hair reduction (47%), followed by use of the fractional laser (17%), and the ablative laser (13%). The interval between laser/IPL treatment and onset of vitiligo was 0-4 weeks in 27% of cases and 4-12 weeks in 57% of cases. Direct complications such as blistering, crusting, and erosions occurred in 57% of cases.

“Our conclusion is that laser and IPL-induced vitiligo is a rare phenomenon, and it often affects patients without prior vitiligo, which was really a surprise to us,” Dr. Wolkerstorfer said. “Complications seem to increase the risk,” he added.

“Despite the apparently low risk, we recommend caution [in patients with vitiligo], especially with aggressive laser procedures,” he said. “We recommend using conservative settings, not to treat active vitiligo patients ... and to perform test spots prior to treating large areas.” But he characterized this recommendation as “totally preliminary” pending results of the Delphi technique aimed at building consensus about laser/IPL treatments in vitiligo.

In an interview at the meeting, one of the session moderators, Oge Onwudiwe, MD, a dermatologist who practices in Alexandria, Va., said that as clinicians await results of the study’s Delphi consensus, current use of lasers and IPL in patients with vitiligo “is based on your clinical judgment and whether the vitiligo is active or inactive. If the patient has vitiligo and you’re doing laser hair removal in the armpit, they may get active lesions in that area, but they can cover it. So, they may take that as a ‘win’ with the risk. But if it can erupt in other areas, that’s a risk they must be willing to take.”

Dr. Wolkerstorfer disclosed that he has received grant or research funding from Lumenis, Novartis, and Avita Medical. He is an advisory board member for Incyte. Dr. Onwudiwe reported having no disclosures.

SAN DIEGO – The risk of laser or intense-pulsed light–induced vitiligo from treating vitiligo with either modality appears to be low, but caution is recommended until further studies are carried out.

Those are the preliminary conclusions from a systematic review and survey of experts that Albert Wolkerstorfer, MD, presented during a clinical abstract session at the annual conference of the American Society for Laser Medicine and Surgery.

According to Dr. Wolkerstorfer, a dermatologist at Amsterdam University Medical Center, clinicians are reluctant to perform laser/intense pulsed light (IPL) treatments in patients with vitiligo because of the absence of clear guidelines, so he and his colleagues set out to investigate the risks of laser/IPL-induced vitiligo in patients with vitiligo and to seek out international consensus on recommendations from experts. “There is hardly any literature about it and certainly no guidelines,” he pointed out.

Dr. Wolkerstorfer and his colleagues designed three consecutive studies: A systematic review of laser/IPL-induced vitiligo; an international survey among 14 vitiligo experts from 10 countries about the occurrence of laser‐induced vitiligo, and a Delphi technique aimed at establishing a broad consensus about recommendations for safe use of lasers in vitiligo patients. At the time of the meeting, the Delphi process was still being carried out, so he did not discuss that study.

For the systematic review, the researchers found 11,073 unique hits on PubMed, Embase, and CINAHL using the terms “vitiligo,” “depigmentation,” “hypopigmentation,” and “leukoderma.” Only six case reports of laser/IPL-induced vitiligo were included in the final analysis. Of these, three had de novo vitiligo and three had vitiligo/halo nevi. These cases included two that occurred following treatment of port wine stains with the 585-nm laser; one that occurred following treatment of dyspigmentation with IPL; one that occurred following treatment of hypertrichosis with the 1,064-nm laser, one that occurred following treatment of hypertrichosis with the 755-nm laser, and one case that occurred following treatment of melasma with the ablative laser.

For the international survey of 14 experts from 10 countries, respondents said they had 10,670 new face-to-face vitiligo consultations in the past year. They reported that 30 of the vitiligo cases (0.3%) were likely caused by laser/IPL. Of these 30 cases, 18 (60%) had de novo vitiligo.

Of these cases, vitiligo occurred most frequently after laser hair reduction (47%), followed by use of the fractional laser (17%), and the ablative laser (13%). The interval between laser/IPL treatment and onset of vitiligo was 0-4 weeks in 27% of cases and 4-12 weeks in 57% of cases. Direct complications such as blistering, crusting, and erosions occurred in 57% of cases.

“Our conclusion is that laser and IPL-induced vitiligo is a rare phenomenon, and it often affects patients without prior vitiligo, which was really a surprise to us,” Dr. Wolkerstorfer said. “Complications seem to increase the risk,” he added.

“Despite the apparently low risk, we recommend caution [in patients with vitiligo], especially with aggressive laser procedures,” he said. “We recommend using conservative settings, not to treat active vitiligo patients ... and to perform test spots prior to treating large areas.” But he characterized this recommendation as “totally preliminary” pending results of the Delphi technique aimed at building consensus about laser/IPL treatments in vitiligo.

In an interview at the meeting, one of the session moderators, Oge Onwudiwe, MD, a dermatologist who practices in Alexandria, Va., said that as clinicians await results of the study’s Delphi consensus, current use of lasers and IPL in patients with vitiligo “is based on your clinical judgment and whether the vitiligo is active or inactive. If the patient has vitiligo and you’re doing laser hair removal in the armpit, they may get active lesions in that area, but they can cover it. So, they may take that as a ‘win’ with the risk. But if it can erupt in other areas, that’s a risk they must be willing to take.”

Dr. Wolkerstorfer disclosed that he has received grant or research funding from Lumenis, Novartis, and Avita Medical. He is an advisory board member for Incyte. Dr. Onwudiwe reported having no disclosures.

SAN DIEGO – The risk of laser or intense-pulsed light–induced vitiligo from treating vitiligo with either modality appears to be low, but caution is recommended until further studies are carried out.

Those are the preliminary conclusions from a systematic review and survey of experts that Albert Wolkerstorfer, MD, presented during a clinical abstract session at the annual conference of the American Society for Laser Medicine and Surgery.

According to Dr. Wolkerstorfer, a dermatologist at Amsterdam University Medical Center, clinicians are reluctant to perform laser/intense pulsed light (IPL) treatments in patients with vitiligo because of the absence of clear guidelines, so he and his colleagues set out to investigate the risks of laser/IPL-induced vitiligo in patients with vitiligo and to seek out international consensus on recommendations from experts. “There is hardly any literature about it and certainly no guidelines,” he pointed out.

Dr. Wolkerstorfer and his colleagues designed three consecutive studies: A systematic review of laser/IPL-induced vitiligo; an international survey among 14 vitiligo experts from 10 countries about the occurrence of laser‐induced vitiligo, and a Delphi technique aimed at establishing a broad consensus about recommendations for safe use of lasers in vitiligo patients. At the time of the meeting, the Delphi process was still being carried out, so he did not discuss that study.

For the systematic review, the researchers found 11,073 unique hits on PubMed, Embase, and CINAHL using the terms “vitiligo,” “depigmentation,” “hypopigmentation,” and “leukoderma.” Only six case reports of laser/IPL-induced vitiligo were included in the final analysis. Of these, three had de novo vitiligo and three had vitiligo/halo nevi. These cases included two that occurred following treatment of port wine stains with the 585-nm laser; one that occurred following treatment of dyspigmentation with IPL; one that occurred following treatment of hypertrichosis with the 1,064-nm laser, one that occurred following treatment of hypertrichosis with the 755-nm laser, and one case that occurred following treatment of melasma with the ablative laser.

For the international survey of 14 experts from 10 countries, respondents said they had 10,670 new face-to-face vitiligo consultations in the past year. They reported that 30 of the vitiligo cases (0.3%) were likely caused by laser/IPL. Of these 30 cases, 18 (60%) had de novo vitiligo.

Of these cases, vitiligo occurred most frequently after laser hair reduction (47%), followed by use of the fractional laser (17%), and the ablative laser (13%). The interval between laser/IPL treatment and onset of vitiligo was 0-4 weeks in 27% of cases and 4-12 weeks in 57% of cases. Direct complications such as blistering, crusting, and erosions occurred in 57% of cases.

“Our conclusion is that laser and IPL-induced vitiligo is a rare phenomenon, and it often affects patients without prior vitiligo, which was really a surprise to us,” Dr. Wolkerstorfer said. “Complications seem to increase the risk,” he added.

“Despite the apparently low risk, we recommend caution [in patients with vitiligo], especially with aggressive laser procedures,” he said. “We recommend using conservative settings, not to treat active vitiligo patients ... and to perform test spots prior to treating large areas.” But he characterized this recommendation as “totally preliminary” pending results of the Delphi technique aimed at building consensus about laser/IPL treatments in vitiligo.

In an interview at the meeting, one of the session moderators, Oge Onwudiwe, MD, a dermatologist who practices in Alexandria, Va., said that as clinicians await results of the study’s Delphi consensus, current use of lasers and IPL in patients with vitiligo “is based on your clinical judgment and whether the vitiligo is active or inactive. If the patient has vitiligo and you’re doing laser hair removal in the armpit, they may get active lesions in that area, but they can cover it. So, they may take that as a ‘win’ with the risk. But if it can erupt in other areas, that’s a risk they must be willing to take.”

Dr. Wolkerstorfer disclosed that he has received grant or research funding from Lumenis, Novartis, and Avita Medical. He is an advisory board member for Incyte. Dr. Onwudiwe reported having no disclosures.

Mental illness has been waiting in the wings for years; ignored, ridiculed, minimized, and stigmatized. Those who succumbed to it tried to lend testimonials, but to no avail. Those who were spared its effects remained in disbelief. So, it stayed on the sidelines, growing in intensity and breadth, yet stifled by the masses, until 2 years ago.

In March 2020, when COVID-19 became a pandemic, the importance of mental health finally became undeniable. As the pandemic’s effects progressed and wreaked havoc on our nation, our mental illness rates simultaneously surged. This surge paralleled that of the COVID-19 pandemic’s and in fact, contributed to a secondary crisis, allowing mental health to finally be addressed and gain center stage status.

But “mental health” is not easily defined, as it takes on many forms and is expressed in a variety of ways and via a myriad of symptoms. It does not discriminate by gender, race, age, socioeconomic status, educational level, profession, religion, or geography. At times, mental health status is consistent but at other times it can fluctuate in intensity, duration, and expression. It can be difficult to manage, yet there are various treatment modalities that can be implemented to lessen the impact of mental illness. Stressful events seem to potentiate its manifestation and yet, there are times it seems to appear spontaneously, much as an uninvited guest.

Mental health has a strong synergistic relationship with physical health, as they are very interdependent and allow us to function at our best only when they are both operating optimally. It should come as no surprise then, that the COVID-19 pandemic contributed to the exponential surge of mental illnesses. Capitalizing on its nondiscriminatory nature, mental illness impacted a large segment of the population – both those suffering from COVID-19 as well as those treating them.

As the nation starts to heal from the immediate and lingering physical and emotional consequences of the COVID-19 pandemic, President Biden has chosen to address and try to meet the needs of the health care heroes, the healers. The signing of H.R. 1667, the Dr. Lorna Breen Health Care Provider Protection Act into law on March 18, 2022, showed dedication to the health care community that has given tirelessly to our nation during the COVID-19 pandemic, and is itself recuperating from that effort.

Taking a top-down approach is essential to assuring the health of the nation. If our healers are not healthy, physically and mentally, they will not be able treat those whom they are dedicated to helping. Openly discussing and acknowledging the mental health problems of health care workers as a community makes it okay to not be okay. It normalizes the need for health care workers to prioritize their own mental health. It can also start to ease the fear of professional backlash or repercussions for practicing self-care.

I, for one, am very grateful for the prioritizing and promoting of the importance of mental health and wellness amongst health care workers. This helps to reduce the stigma of mental illness, helps us understand its impact, and allows us to formulate strategies and solutions to address its effects. The time has come.

Dr. Jarkon is a psychiatrist and director of the Center for Behavioral Health at the New York Institute of Technology College of Osteopathic Medicine in Old Westbury, N.Y.

Mental illness has been waiting in the wings for years; ignored, ridiculed, minimized, and stigmatized. Those who succumbed to it tried to lend testimonials, but to no avail. Those who were spared its effects remained in disbelief. So, it stayed on the sidelines, growing in intensity and breadth, yet stifled by the masses, until 2 years ago.

In March 2020, when COVID-19 became a pandemic, the importance of mental health finally became undeniable. As the pandemic’s effects progressed and wreaked havoc on our nation, our mental illness rates simultaneously surged. This surge paralleled that of the COVID-19 pandemic’s and in fact, contributed to a secondary crisis, allowing mental health to finally be addressed and gain center stage status.

But “mental health” is not easily defined, as it takes on many forms and is expressed in a variety of ways and via a myriad of symptoms. It does not discriminate by gender, race, age, socioeconomic status, educational level, profession, religion, or geography. At times, mental health status is consistent but at other times it can fluctuate in intensity, duration, and expression. It can be difficult to manage, yet there are various treatment modalities that can be implemented to lessen the impact of mental illness. Stressful events seem to potentiate its manifestation and yet, there are times it seems to appear spontaneously, much as an uninvited guest.

Mental health has a strong synergistic relationship with physical health, as they are very interdependent and allow us to function at our best only when they are both operating optimally. It should come as no surprise then, that the COVID-19 pandemic contributed to the exponential surge of mental illnesses. Capitalizing on its nondiscriminatory nature, mental illness impacted a large segment of the population – both those suffering from COVID-19 as well as those treating them.

As the nation starts to heal from the immediate and lingering physical and emotional consequences of the COVID-19 pandemic, President Biden has chosen to address and try to meet the needs of the health care heroes, the healers. The signing of H.R. 1667, the Dr. Lorna Breen Health Care Provider Protection Act into law on March 18, 2022, showed dedication to the health care community that has given tirelessly to our nation during the COVID-19 pandemic, and is itself recuperating from that effort.

Taking a top-down approach is essential to assuring the health of the nation. If our healers are not healthy, physically and mentally, they will not be able treat those whom they are dedicated to helping. Openly discussing and acknowledging the mental health problems of health care workers as a community makes it okay to not be okay. It normalizes the need for health care workers to prioritize their own mental health. It can also start to ease the fear of professional backlash or repercussions for practicing self-care.

I, for one, am very grateful for the prioritizing and promoting of the importance of mental health and wellness amongst health care workers. This helps to reduce the stigma of mental illness, helps us understand its impact, and allows us to formulate strategies and solutions to address its effects. The time has come.

Dr. Jarkon is a psychiatrist and director of the Center for Behavioral Health at the New York Institute of Technology College of Osteopathic Medicine in Old Westbury, N.Y.

Mental illness has been waiting in the wings for years; ignored, ridiculed, minimized, and stigmatized. Those who succumbed to it tried to lend testimonials, but to no avail. Those who were spared its effects remained in disbelief. So, it stayed on the sidelines, growing in intensity and breadth, yet stifled by the masses, until 2 years ago.

In March 2020, when COVID-19 became a pandemic, the importance of mental health finally became undeniable. As the pandemic’s effects progressed and wreaked havoc on our nation, our mental illness rates simultaneously surged. This surge paralleled that of the COVID-19 pandemic’s and in fact, contributed to a secondary crisis, allowing mental health to finally be addressed and gain center stage status.

But “mental health” is not easily defined, as it takes on many forms and is expressed in a variety of ways and via a myriad of symptoms. It does not discriminate by gender, race, age, socioeconomic status, educational level, profession, religion, or geography. At times, mental health status is consistent but at other times it can fluctuate in intensity, duration, and expression. It can be difficult to manage, yet there are various treatment modalities that can be implemented to lessen the impact of mental illness. Stressful events seem to potentiate its manifestation and yet, there are times it seems to appear spontaneously, much as an uninvited guest.

Mental health has a strong synergistic relationship with physical health, as they are very interdependent and allow us to function at our best only when they are both operating optimally. It should come as no surprise then, that the COVID-19 pandemic contributed to the exponential surge of mental illnesses. Capitalizing on its nondiscriminatory nature, mental illness impacted a large segment of the population – both those suffering from COVID-19 as well as those treating them.

As the nation starts to heal from the immediate and lingering physical and emotional consequences of the COVID-19 pandemic, President Biden has chosen to address and try to meet the needs of the health care heroes, the healers. The signing of H.R. 1667, the Dr. Lorna Breen Health Care Provider Protection Act into law on March 18, 2022, showed dedication to the health care community that has given tirelessly to our nation during the COVID-19 pandemic, and is itself recuperating from that effort.

Taking a top-down approach is essential to assuring the health of the nation. If our healers are not healthy, physically and mentally, they will not be able treat those whom they are dedicated to helping. Openly discussing and acknowledging the mental health problems of health care workers as a community makes it okay to not be okay. It normalizes the need for health care workers to prioritize their own mental health. It can also start to ease the fear of professional backlash or repercussions for practicing self-care.

I, for one, am very grateful for the prioritizing and promoting of the importance of mental health and wellness amongst health care workers. This helps to reduce the stigma of mental illness, helps us understand its impact, and allows us to formulate strategies and solutions to address its effects. The time has come.

Dr. Jarkon is a psychiatrist and director of the Center for Behavioral Health at the New York Institute of Technology College of Osteopathic Medicine in Old Westbury, N.Y.

A novel blood-based assay could one day be used to diagnose Parkinson’s disease and possibly other chronic inflammatory conditions, according to investigators. In addition to being highly accurate, the assay, which detects changes in expression of cytochrome P450s, is faster and easier to perform than other Parkinson’s disease assays under investigation, reported lead author Kohei Ihara, PhD, of Kobe University, Japan, and colleagues.

“Effective diagnostic systems and biomarkers for patients without subjective motor symptoms have not yet been established,” the investigators wrote in Nature Scientific Reports. “Consequently, the poor diagnostic options for Parkinson’s disease delay the development of therapeutic approaches and medication. Therefore, the development of efficient diagnostic systems and biomarkers is crucial for overcoming Parkinson’s disease.”

According to Dr. Ihara and colleagues, various cytochrome P450 expression patterns and associated serum metabolites correlate with chronic conditions, making them possible markers of disease. To detect these changes, they developed the present assay. It relies upon recombinant P450s expressed on the surface of Escherichia coli. By mixing the E. coli with serum and Vivid, a fluorescent substrate, the investigators can measure “the inhibition rate of the Vivid decomposition reaction” that was driven by “serum metabolites associated with P450s,” revealing underlying expression and, if present, disease.

After some promising initial experiments with mouse models of ulcerative colitis and diabetes, Dr. Ihara and colleagues focused on a rat model of Parkinson’s disease. Evaluating inhibition rates associated with four P450s revealed area-under-the-curve (AUC) values of 0.814-0.914. Two of those P450s were also associated with progression of disease symptoms.

“Therefore, we concluded that the P450 inhibition assay could discriminate between Parkinson’s disease model rats and control rats,” the investigators wrote.

Next, the investigators tested the approach with a case-control study involving 20 patients with Parkinson’s disease and 20 healthy volunteers. Twelve P450s were analyzed, three of which revealed significant differences between patients with Parkinson’s disease and controls, with AUCs ranging from 0.740-0.775. Each of the three P450 enzymes also correlated significantly with stage of disease on the Hoehn & Yahr scale, although severity and frequency of symptoms were not reported.

To increase accuracy of the technique, the investigators developed a logistic regression model using two of the three P450s, generating an AUC of 0.910. Further testing showed that the P450 inhibition assay could distinguish between patients with Parkinson’s disease and Alzheimer’s disease, as well as other chronic inflammatory diseases.

“The P450 inhibition assay is easier to perform and is faster than other assays because this assay does not require pretreatment, such as purification of exosomes, and it involves a single enzymatic reaction,” the investigators wrote, suggesting that the assay may be suitable for real-world diagnosis.
 

‘Promising’ findings need replication

According to Douglas Galasko, MD, a neurologist and professor of neurosciences at UC San Diego Health, the reported accuracy of the assay “seems spectacular,” and the findings are “promising,” but they need to be replicated, “particularly in early-stage patients where the diagnosis [of Parkinson’s disease] is more difficult and important to make.” In practice, the assay would likely see greatest usage for “early diagnosis or diagnosis of unusual or challenging cases,” so accuracy testing needs to be conducted in this setting, he said.

Dr. Galasko, who was not involved in the study, predicted that liquid biopsy for detecting Parkinson’s disease is unlikely to hit the clinic floor anytime soon. “We’re not really close with blood-based biomarkers for Parkinson’s disease,” he said, “unlike the situation for Alzheimer’s disease, where there are several promising blood-based biomarkers.”

For diagnosing Parkinson’s disease, Dr. Galasko suggested that assays using skin biopsies to measure alpha-synuclein accumulation may be closer to approval.

The study was supported by JSPS KAKENHI Grant Number 20K20223 and the Sumitomo Electric Industries Group Corporate Social Responsibility Foundation. The investigators disclosed no conflicts of interest.

A novel blood-based assay could one day be used to diagnose Parkinson’s disease and possibly other chronic inflammatory conditions, according to investigators. In addition to being highly accurate, the assay, which detects changes in expression of cytochrome P450s, is faster and easier to perform than other Parkinson’s disease assays under investigation, reported lead author Kohei Ihara, PhD, of Kobe University, Japan, and colleagues.

“Effective diagnostic systems and biomarkers for patients without subjective motor symptoms have not yet been established,” the investigators wrote in Nature Scientific Reports. “Consequently, the poor diagnostic options for Parkinson’s disease delay the development of therapeutic approaches and medication. Therefore, the development of efficient diagnostic systems and biomarkers is crucial for overcoming Parkinson’s disease.”

According to Dr. Ihara and colleagues, various cytochrome P450 expression patterns and associated serum metabolites correlate with chronic conditions, making them possible markers of disease. To detect these changes, they developed the present assay. It relies upon recombinant P450s expressed on the surface of Escherichia coli. By mixing the E. coli with serum and Vivid, a fluorescent substrate, the investigators can measure “the inhibition rate of the Vivid decomposition reaction” that was driven by “serum metabolites associated with P450s,” revealing underlying expression and, if present, disease.

After some promising initial experiments with mouse models of ulcerative colitis and diabetes, Dr. Ihara and colleagues focused on a rat model of Parkinson’s disease. Evaluating inhibition rates associated with four P450s revealed area-under-the-curve (AUC) values of 0.814-0.914. Two of those P450s were also associated with progression of disease symptoms.

“Therefore, we concluded that the P450 inhibition assay could discriminate between Parkinson’s disease model rats and control rats,” the investigators wrote.

Next, the investigators tested the approach with a case-control study involving 20 patients with Parkinson’s disease and 20 healthy volunteers. Twelve P450s were analyzed, three of which revealed significant differences between patients with Parkinson’s disease and controls, with AUCs ranging from 0.740-0.775. Each of the three P450 enzymes also correlated significantly with stage of disease on the Hoehn & Yahr scale, although severity and frequency of symptoms were not reported.

To increase accuracy of the technique, the investigators developed a logistic regression model using two of the three P450s, generating an AUC of 0.910. Further testing showed that the P450 inhibition assay could distinguish between patients with Parkinson’s disease and Alzheimer’s disease, as well as other chronic inflammatory diseases.

“The P450 inhibition assay is easier to perform and is faster than other assays because this assay does not require pretreatment, such as purification of exosomes, and it involves a single enzymatic reaction,” the investigators wrote, suggesting that the assay may be suitable for real-world diagnosis.
 

‘Promising’ findings need replication

According to Douglas Galasko, MD, a neurologist and professor of neurosciences at UC San Diego Health, the reported accuracy of the assay “seems spectacular,” and the findings are “promising,” but they need to be replicated, “particularly in early-stage patients where the diagnosis [of Parkinson’s disease] is more difficult and important to make.” In practice, the assay would likely see greatest usage for “early diagnosis or diagnosis of unusual or challenging cases,” so accuracy testing needs to be conducted in this setting, he said.

Dr. Galasko, who was not involved in the study, predicted that liquid biopsy for detecting Parkinson’s disease is unlikely to hit the clinic floor anytime soon. “We’re not really close with blood-based biomarkers for Parkinson’s disease,” he said, “unlike the situation for Alzheimer’s disease, where there are several promising blood-based biomarkers.”

For diagnosing Parkinson’s disease, Dr. Galasko suggested that assays using skin biopsies to measure alpha-synuclein accumulation may be closer to approval.

The study was supported by JSPS KAKENHI Grant Number 20K20223 and the Sumitomo Electric Industries Group Corporate Social Responsibility Foundation. The investigators disclosed no conflicts of interest.

A novel blood-based assay could one day be used to diagnose Parkinson’s disease and possibly other chronic inflammatory conditions, according to investigators. In addition to being highly accurate, the assay, which detects changes in expression of cytochrome P450s, is faster and easier to perform than other Parkinson’s disease assays under investigation, reported lead author Kohei Ihara, PhD, of Kobe University, Japan, and colleagues.

“Effective diagnostic systems and biomarkers for patients without subjective motor symptoms have not yet been established,” the investigators wrote in Nature Scientific Reports. “Consequently, the poor diagnostic options for Parkinson’s disease delay the development of therapeutic approaches and medication. Therefore, the development of efficient diagnostic systems and biomarkers is crucial for overcoming Parkinson’s disease.”

According to Dr. Ihara and colleagues, various cytochrome P450 expression patterns and associated serum metabolites correlate with chronic conditions, making them possible markers of disease. To detect these changes, they developed the present assay. It relies upon recombinant P450s expressed on the surface of Escherichia coli. By mixing the E. coli with serum and Vivid, a fluorescent substrate, the investigators can measure “the inhibition rate of the Vivid decomposition reaction” that was driven by “serum metabolites associated with P450s,” revealing underlying expression and, if present, disease.

After some promising initial experiments with mouse models of ulcerative colitis and diabetes, Dr. Ihara and colleagues focused on a rat model of Parkinson’s disease. Evaluating inhibition rates associated with four P450s revealed area-under-the-curve (AUC) values of 0.814-0.914. Two of those P450s were also associated with progression of disease symptoms.

“Therefore, we concluded that the P450 inhibition assay could discriminate between Parkinson’s disease model rats and control rats,” the investigators wrote.

Next, the investigators tested the approach with a case-control study involving 20 patients with Parkinson’s disease and 20 healthy volunteers. Twelve P450s were analyzed, three of which revealed significant differences between patients with Parkinson’s disease and controls, with AUCs ranging from 0.740-0.775. Each of the three P450 enzymes also correlated significantly with stage of disease on the Hoehn & Yahr scale, although severity and frequency of symptoms were not reported.

To increase accuracy of the technique, the investigators developed a logistic regression model using two of the three P450s, generating an AUC of 0.910. Further testing showed that the P450 inhibition assay could distinguish between patients with Parkinson’s disease and Alzheimer’s disease, as well as other chronic inflammatory diseases.

“The P450 inhibition assay is easier to perform and is faster than other assays because this assay does not require pretreatment, such as purification of exosomes, and it involves a single enzymatic reaction,” the investigators wrote, suggesting that the assay may be suitable for real-world diagnosis.
 

‘Promising’ findings need replication

According to Douglas Galasko, MD, a neurologist and professor of neurosciences at UC San Diego Health, the reported accuracy of the assay “seems spectacular,” and the findings are “promising,” but they need to be replicated, “particularly in early-stage patients where the diagnosis [of Parkinson’s disease] is more difficult and important to make.” In practice, the assay would likely see greatest usage for “early diagnosis or diagnosis of unusual or challenging cases,” so accuracy testing needs to be conducted in this setting, he said.

Dr. Galasko, who was not involved in the study, predicted that liquid biopsy for detecting Parkinson’s disease is unlikely to hit the clinic floor anytime soon. “We’re not really close with blood-based biomarkers for Parkinson’s disease,” he said, “unlike the situation for Alzheimer’s disease, where there are several promising blood-based biomarkers.”

For diagnosing Parkinson’s disease, Dr. Galasko suggested that assays using skin biopsies to measure alpha-synuclein accumulation may be closer to approval.

The study was supported by JSPS KAKENHI Grant Number 20K20223 and the Sumitomo Electric Industries Group Corporate Social Responsibility Foundation. The investigators disclosed no conflicts of interest.

NEW ORLEANS – High out-of-pocket costs for medications used by patients with diabetes are tied to reduced use of these drugs and ultimately worse clinical outcomes, according to findings from two separate studies.

One study looked at the insurance records of more than 70,000 U.S. patients with type 2 diabetes and established cardiovascular disease who were already on metformin. The findings showed that, after adjustment for confounders, the quartile of patients with the highest out-of-pocket cost for an agent from the sodium-glucose cotransporter 2 (SGLT2)–inhibitor class filled a prescription for one of these drugs a significant 21% less often than did patients from the quartile with the lowest personal expense, after adjustment for a variety of potential confounding factors, reported Jing Luo, MD, at the annual scientific sessions of the American Diabetes Association.

A similar analysis run by Dr. Luo and his associates looking at glucagonlike peptide-1 (GLP-1) receptor agonists showed that the quartile of patients who had to pay the most for one of those drugs had an adjusted 12% lower rate of filling a prescription, compared with those with the lowest out-of-pocket expense, a difference that fell just short of significance.

“If we consistently see that high drug costs affect use of highly effective medications in patients with type 2 diabetes and risk factors, it’s quite problematic because it’s not just a matter of money, but it also makes a difference in the patient’s quality of care,” Dr. Luo said in an interview.

Prevention drug lists can help

Consistency turned up in a second report at the same ADA session that retrospectively reviewed data collected during 2004-2017 by a single large U.S. health insurer to identify 3,315 matched pairs of children and adults with diabetes who all had high-deductible health plans for their medical insurance, along with an associated health savings account.

One set of patients in each matched pair began to receive, at some point during follow-up, coverage with a prevention drug list (PDL; also called a formulary) that provided them with a variety of specified agents at no charge. They included oral antidiabetes agents, insulin, antihypertensives, and lipid-lowering drugs. The other half of the matched pairs of patients received no PDL coverage and had copays for their antidiabetes medications.

The findings showed that the rates of out-of-pocket costs for antidiabetes drugs, antidiabetic medications used, and acute diabetes complications all tracked extremely closely between the matched pairs before half of them started to receive their PDL coverage. However, after PDL coverage kicked in, out of pocket costs dropped by 32% for the people with PDL coverage, compared with those who did not receive this coverage. Oral antidiabetes medication use rose modestly, but acute diabetes complications “declined substantially,” with a 14% relative reduction overall in those with PDL coverage, compared with those without, reported J. Franklin Wharam, MBBCh, a professor and health policy researcher at Duke University in Durham, N.C. In the roughly half of the study cohort who fell into a low-income category based on where they lived, the rate of excess acute diabetes complications was 23% higher for those without a PDL, compared with those who had that coverage.

PDL coverage linked with “large reductions in acute, preventable diabetes complications,” concluded Dr. Wharam. “Policy makers and employers should incentivize PDL uptake among low-income patients with diabetes.”

Newer, more effective drugs cost a lot

“The more comorbidities that patients have, the greater is the strength of the evidence for using newer antidiabetes drugs that are more expensive,” but that would mean spending much more on this part of patient care, noted Dr. Luo, an internal medicine physician and researcher at the University of Pittsburgh. “It will cost a lot of money, and I’m not sure what the solution is. It’s a huge conundrum.”

About 30 million Americans have type 2 diabetes. If every one of them went on an SGLT2 inhibitor, or went on an SGLT2 inhibitor plus a GLP-1 receptor agonist, “it would bankrupt the U.S. health care system, so we can’t do that,” commented Sylvio E. Inzucchi, MD, in an interview. “The only thing holding this back is cost. We target these drugs to the patients most apt to benefit from them. If they were generic they would be used much more widely,” noted Dr. Inzucchi, professor and clinical chief of endocrinology at Yale University in New Haven, Conn.

The study run by Dr. Luo and his associates retrospectively reviewed data from 72,743 U.S. adults included in the Optum Clinformatics database during December 2017–December 2019. All included patients had type 2 diabetes, received metformin monotherapy, and had established atherosclerotic cardiovascular disease. They averaged 72 years of age, 56% were men, and 88% were on a Medicare Advantage plan, while the remainder had commercial insurance. Their average hemoglobin A1c level was 6.8%.

People in the quartile with the lowest copays spent an average of about $20/month for either an SGLT2 inhibitor or a GLP-1 receptor agonist. Those in the quartile with the highest copays spent roughly $100/month for agents from each of these two classes. The analysis followed patients for a median of 914 days.

In addition to finding disparate rates of drug use between these two quartiles, the analysis also showed that higher copays linked with longer times to initially fill prescriptions for these drugs. But while those with higher copays took longer to start both classes than did those with the smallest copays, even those with the lowest out-of-pocket costs averaged about a year to initiate treatment.

Dr. Luo attributed this delay to other factors besides costs to patients, such as clinicians prescribing other classes of second-line oral antidiabetes agents, clinical inertia, and lack of awareness by clinicians of the special benefits of SGLT2 inhibitors and GLP-1 receptor antagonists for patients with type 2 diabetes and cardiovascular disease.

“A lot of clinical and social factors drive medication use,” not just out-of-pocket cost, he explained.

Dr. Luo is a consultant to Alosa Health. Dr. Wharam had no disclosures. Dr. Inzucchi is an adviser to Abbott Diagnostics, Esperion Therapeutics, and vTv Therapeutics, a consultant to Merck and Pfizer, and has other relationships with AstraZeneca, Boehringer Ingelheim, Lexicon, and Novo Nordisk.

NEW ORLEANS – High out-of-pocket costs for medications used by patients with diabetes are tied to reduced use of these drugs and ultimately worse clinical outcomes, according to findings from two separate studies.

One study looked at the insurance records of more than 70,000 U.S. patients with type 2 diabetes and established cardiovascular disease who were already on metformin. The findings showed that, after adjustment for confounders, the quartile of patients with the highest out-of-pocket cost for an agent from the sodium-glucose cotransporter 2 (SGLT2)–inhibitor class filled a prescription for one of these drugs a significant 21% less often than did patients from the quartile with the lowest personal expense, after adjustment for a variety of potential confounding factors, reported Jing Luo, MD, at the annual scientific sessions of the American Diabetes Association.

A similar analysis run by Dr. Luo and his associates looking at glucagonlike peptide-1 (GLP-1) receptor agonists showed that the quartile of patients who had to pay the most for one of those drugs had an adjusted 12% lower rate of filling a prescription, compared with those with the lowest out-of-pocket expense, a difference that fell just short of significance.

“If we consistently see that high drug costs affect use of highly effective medications in patients with type 2 diabetes and risk factors, it’s quite problematic because it’s not just a matter of money, but it also makes a difference in the patient’s quality of care,” Dr. Luo said in an interview.

Prevention drug lists can help

Consistency turned up in a second report at the same ADA session that retrospectively reviewed data collected during 2004-2017 by a single large U.S. health insurer to identify 3,315 matched pairs of children and adults with diabetes who all had high-deductible health plans for their medical insurance, along with an associated health savings account.

One set of patients in each matched pair began to receive, at some point during follow-up, coverage with a prevention drug list (PDL; also called a formulary) that provided them with a variety of specified agents at no charge. They included oral antidiabetes agents, insulin, antihypertensives, and lipid-lowering drugs. The other half of the matched pairs of patients received no PDL coverage and had copays for their antidiabetes medications.

The findings showed that the rates of out-of-pocket costs for antidiabetes drugs, antidiabetic medications used, and acute diabetes complications all tracked extremely closely between the matched pairs before half of them started to receive their PDL coverage. However, after PDL coverage kicked in, out of pocket costs dropped by 32% for the people with PDL coverage, compared with those who did not receive this coverage. Oral antidiabetes medication use rose modestly, but acute diabetes complications “declined substantially,” with a 14% relative reduction overall in those with PDL coverage, compared with those without, reported J. Franklin Wharam, MBBCh, a professor and health policy researcher at Duke University in Durham, N.C. In the roughly half of the study cohort who fell into a low-income category based on where they lived, the rate of excess acute diabetes complications was 23% higher for those without a PDL, compared with those who had that coverage.

PDL coverage linked with “large reductions in acute, preventable diabetes complications,” concluded Dr. Wharam. “Policy makers and employers should incentivize PDL uptake among low-income patients with diabetes.”

Newer, more effective drugs cost a lot

“The more comorbidities that patients have, the greater is the strength of the evidence for using newer antidiabetes drugs that are more expensive,” but that would mean spending much more on this part of patient care, noted Dr. Luo, an internal medicine physician and researcher at the University of Pittsburgh. “It will cost a lot of money, and I’m not sure what the solution is. It’s a huge conundrum.”

About 30 million Americans have type 2 diabetes. If every one of them went on an SGLT2 inhibitor, or went on an SGLT2 inhibitor plus a GLP-1 receptor agonist, “it would bankrupt the U.S. health care system, so we can’t do that,” commented Sylvio E. Inzucchi, MD, in an interview. “The only thing holding this back is cost. We target these drugs to the patients most apt to benefit from them. If they were generic they would be used much more widely,” noted Dr. Inzucchi, professor and clinical chief of endocrinology at Yale University in New Haven, Conn.

The study run by Dr. Luo and his associates retrospectively reviewed data from 72,743 U.S. adults included in the Optum Clinformatics database during December 2017–December 2019. All included patients had type 2 diabetes, received metformin monotherapy, and had established atherosclerotic cardiovascular disease. They averaged 72 years of age, 56% were men, and 88% were on a Medicare Advantage plan, while the remainder had commercial insurance. Their average hemoglobin A1c level was 6.8%.

People in the quartile with the lowest copays spent an average of about $20/month for either an SGLT2 inhibitor or a GLP-1 receptor agonist. Those in the quartile with the highest copays spent roughly $100/month for agents from each of these two classes. The analysis followed patients for a median of 914 days.

In addition to finding disparate rates of drug use between these two quartiles, the analysis also showed that higher copays linked with longer times to initially fill prescriptions for these drugs. But while those with higher copays took longer to start both classes than did those with the smallest copays, even those with the lowest out-of-pocket costs averaged about a year to initiate treatment.

Dr. Luo attributed this delay to other factors besides costs to patients, such as clinicians prescribing other classes of second-line oral antidiabetes agents, clinical inertia, and lack of awareness by clinicians of the special benefits of SGLT2 inhibitors and GLP-1 receptor antagonists for patients with type 2 diabetes and cardiovascular disease.

“A lot of clinical and social factors drive medication use,” not just out-of-pocket cost, he explained.

Dr. Luo is a consultant to Alosa Health. Dr. Wharam had no disclosures. Dr. Inzucchi is an adviser to Abbott Diagnostics, Esperion Therapeutics, and vTv Therapeutics, a consultant to Merck and Pfizer, and has other relationships with AstraZeneca, Boehringer Ingelheim, Lexicon, and Novo Nordisk.

NEW ORLEANS – High out-of-pocket costs for medications used by patients with diabetes are tied to reduced use of these drugs and ultimately worse clinical outcomes, according to findings from two separate studies.

One study looked at the insurance records of more than 70,000 U.S. patients with type 2 diabetes and established cardiovascular disease who were already on metformin. The findings showed that, after adjustment for confounders, the quartile of patients with the highest out-of-pocket cost for an agent from the sodium-glucose cotransporter 2 (SGLT2)–inhibitor class filled a prescription for one of these drugs a significant 21% less often than did patients from the quartile with the lowest personal expense, after adjustment for a variety of potential confounding factors, reported Jing Luo, MD, at the annual scientific sessions of the American Diabetes Association.

A similar analysis run by Dr. Luo and his associates looking at glucagonlike peptide-1 (GLP-1) receptor agonists showed that the quartile of patients who had to pay the most for one of those drugs had an adjusted 12% lower rate of filling a prescription, compared with those with the lowest out-of-pocket expense, a difference that fell just short of significance.

“If we consistently see that high drug costs affect use of highly effective medications in patients with type 2 diabetes and risk factors, it’s quite problematic because it’s not just a matter of money, but it also makes a difference in the patient’s quality of care,” Dr. Luo said in an interview.

Prevention drug lists can help

Consistency turned up in a second report at the same ADA session that retrospectively reviewed data collected during 2004-2017 by a single large U.S. health insurer to identify 3,315 matched pairs of children and adults with diabetes who all had high-deductible health plans for their medical insurance, along with an associated health savings account.

One set of patients in each matched pair began to receive, at some point during follow-up, coverage with a prevention drug list (PDL; also called a formulary) that provided them with a variety of specified agents at no charge. They included oral antidiabetes agents, insulin, antihypertensives, and lipid-lowering drugs. The other half of the matched pairs of patients received no PDL coverage and had copays for their antidiabetes medications.

The findings showed that the rates of out-of-pocket costs for antidiabetes drugs, antidiabetic medications used, and acute diabetes complications all tracked extremely closely between the matched pairs before half of them started to receive their PDL coverage. However, after PDL coverage kicked in, out of pocket costs dropped by 32% for the people with PDL coverage, compared with those who did not receive this coverage. Oral antidiabetes medication use rose modestly, but acute diabetes complications “declined substantially,” with a 14% relative reduction overall in those with PDL coverage, compared with those without, reported J. Franklin Wharam, MBBCh, a professor and health policy researcher at Duke University in Durham, N.C. In the roughly half of the study cohort who fell into a low-income category based on where they lived, the rate of excess acute diabetes complications was 23% higher for those without a PDL, compared with those who had that coverage.

PDL coverage linked with “large reductions in acute, preventable diabetes complications,” concluded Dr. Wharam. “Policy makers and employers should incentivize PDL uptake among low-income patients with diabetes.”

Newer, more effective drugs cost a lot

“The more comorbidities that patients have, the greater is the strength of the evidence for using newer antidiabetes drugs that are more expensive,” but that would mean spending much more on this part of patient care, noted Dr. Luo, an internal medicine physician and researcher at the University of Pittsburgh. “It will cost a lot of money, and I’m not sure what the solution is. It’s a huge conundrum.”

About 30 million Americans have type 2 diabetes. If every one of them went on an SGLT2 inhibitor, or went on an SGLT2 inhibitor plus a GLP-1 receptor agonist, “it would bankrupt the U.S. health care system, so we can’t do that,” commented Sylvio E. Inzucchi, MD, in an interview. “The only thing holding this back is cost. We target these drugs to the patients most apt to benefit from them. If they were generic they would be used much more widely,” noted Dr. Inzucchi, professor and clinical chief of endocrinology at Yale University in New Haven, Conn.

The study run by Dr. Luo and his associates retrospectively reviewed data from 72,743 U.S. adults included in the Optum Clinformatics database during December 2017–December 2019. All included patients had type 2 diabetes, received metformin monotherapy, and had established atherosclerotic cardiovascular disease. They averaged 72 years of age, 56% were men, and 88% were on a Medicare Advantage plan, while the remainder had commercial insurance. Their average hemoglobin A1c level was 6.8%.

People in the quartile with the lowest copays spent an average of about $20/month for either an SGLT2 inhibitor or a GLP-1 receptor agonist. Those in the quartile with the highest copays spent roughly $100/month for agents from each of these two classes. The analysis followed patients for a median of 914 days.

In addition to finding disparate rates of drug use between these two quartiles, the analysis also showed that higher copays linked with longer times to initially fill prescriptions for these drugs. But while those with higher copays took longer to start both classes than did those with the smallest copays, even those with the lowest out-of-pocket costs averaged about a year to initiate treatment.

Dr. Luo attributed this delay to other factors besides costs to patients, such as clinicians prescribing other classes of second-line oral antidiabetes agents, clinical inertia, and lack of awareness by clinicians of the special benefits of SGLT2 inhibitors and GLP-1 receptor antagonists for patients with type 2 diabetes and cardiovascular disease.

“A lot of clinical and social factors drive medication use,” not just out-of-pocket cost, he explained.

Dr. Luo is a consultant to Alosa Health. Dr. Wharam had no disclosures. Dr. Inzucchi is an adviser to Abbott Diagnostics, Esperion Therapeutics, and vTv Therapeutics, a consultant to Merck and Pfizer, and has other relationships with AstraZeneca, Boehringer Ingelheim, Lexicon, and Novo Nordisk.