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NEWPORT BEACH, CALIF. – Researchers say they have identified biomarkers that may help guide early treatment decisions in patients with acute graft-versus-host disease (GVHD).

The biomarkers, ST2 and REG3-alpha, were measured during the first month of GVHD treatment and proved more accurate than clinical response for predicting 6-month nonrelapse mortality (NRM). In fact, biomarker assessment revealed patients who responded to treatment but had a high risk of NRM and nonresponders who had a low risk of NRM.

The researchers also found that biomarkers changed over the first month of treatment but remained significant predictors of NRM. This suggests that modifying treatment according to biomarker findings at various time points could result in better outcomes for patients.

“We think this is going to transform the way we treat graft-versus-host disease,” said James L.M. Ferrara, MD, DSc, of the Icahn School of Medicine at Mount Sinai, New York.

Dr. Ferrara and Hrishikesh Srinagesh, along with their colleagues at Mount Sinai, have conducted extensive research with these biomarkers and presented some of their findings at the Acute Leukemia Forum of Hemedicus.

Comparing biomarkers and response

In one study, the researchers evaluated 355 patients who had undergone allogeneic hematopoietic stem cell transplant at 1 of 20 Mount Sinai Acute GVHD International Consortium (MAGIC) centers between January 2016 and February 2018. All patients developed acute GVHD and received systemic steroids as treatment.

Patients provided blood samples weekly for the first month of treatment, and concentrations of ST2 and REG3-alpha were measured in each sample. Both biomarker concentrations were used to calculate the biomarker probability of NRM.

“The concentration of those two biomarkers are put into a computer, and we get … a single number, and that gives us the probability of mortality,” Dr. Ferrara said. “[W]e call this the MAGIC algorithm probability, or MAP. And when a MAP is low, the patient has a very low chance of dying from graft-versus-host disease, when it’s intermediate, they have an intermediate risk, and when it’s high, they have a high risk.”

The researchers then compared the MAP and clinical response for their ability to predict 6-month NRM throughout the first month of therapy for acute GVHD.

MAP bests response

After 1 month of therapy, the MAP was more accurate than clinical response for predicting 6-month NRM. The area under the curve was 0.84 and 0.65, respectively (P less than .001).

Likewise, the MAP after 1 week of therapy was more accurate than clinical response at 1 month for predicting 6-month NRM. The area under the curve was 0.80 and 0.65, respectively (P less than .001).

“[T]he clinical responses were good, but not great, at predicting long-term outcome, where the biomarker, the MAP, was significantly better,” Dr. Ferrara said. “[A]t every time point we tested, the biomarkers were better than the clinical responses.”

The researchers also identified subgroups of clinical responders and nonresponders for whom MAP more accurately predicted 6-month NRM.

The team found that 61% of clinical nonresponders were actually low risk according to MAP. And the incidence of 6-month NRM was significantly lower in the MAP-designated low-risk patients than in MAP-designated high-risk patients – 22% and 56%, respectively (P less than .001).

On the other hand, 10% of clinical responders were high risk according to MAP. The incidence of 6-month NRM was significantly higher in the high-risk patients than in the low-risk patients – 40% and 13%, respectively (P less than .001).

Assessing changes over time

The researchers found that patients who were initially high risk by MAP but had not experienced NRM by 6 months had significant decreases in their MAP after 4 weeks of treatment (P = .003). Patients who did experience NRM had a significant increase in their MAP whether their initial MAP was low (P = .007) or high (P = .024).

“What we found was that patients who lived tended to either have low biomarkers at the start of treatment and stay low or start out with high biomarkers and have reductions over the first month of therapy,” Mr. Srinagesh said. “Conversely, patients who tended to do worse were those who had either increases in their biomarkers or stayed high at all time points.”

The researchers identified a threshold – 0.290 – for separating patients by mortality risk.

“Patients who started out above the threshold and then went below it had a 5-fold reduction in mortality, whereas patients who started out below the threshold and rose above it had a 5-fold increase in mortality,” Mr. Srinagesh said.

MAP in clinical trials and practice

Based on these findings and results from related studies, the researchers theorize that MAP would be a better endpoint for clinical trials than clinical response.

At present, there are three trials in which researchers are using MAP as an endpoint to assess the efficacy of treatment for GVHD (NCT02133924, NCT03459040, and NCT03846479). Dr. Ferrara said a fourth trial is set to begin this summer.

Additionally, MAP is being used in clinical practice. A company called Viracor Eurofins Clinical Diagnostics licensed the MAGIC algorithm and provides three related tests for consumer use.

Viracor’s aGVHD Pre-Symptomatic Algorithm assigns patients to high- and low-risk groups based on results from samples collected 7 days after transplant. The aGVHD Symptomatic Onset Algorithm assigns patients to high-, intermediate-, and low-risk groups. The aGVHD Post-Treatment Algorithm, which can be used 7 days or more after GVHD treatment initiation, stratifies steroid-resistant patients into high- or low-risk groups for both NRM and overall survival.

“We are still in early days of figuring out how to use [the biomarker tests], but … what I’ve heard is that people are finding them to be useful in their clinical practice,” Dr. Ferrara said.

Dr. Ferrara has an ownership interest in and receives royalties from Viracor. Mr. Srinagesh reported having no relevant conflicts of interest. The research was supported by grants from the National Cancer Institute and the American Cancer Society.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – Researchers say they have identified biomarkers that may help guide early treatment decisions in patients with acute graft-versus-host disease (GVHD).

The biomarkers, ST2 and REG3-alpha, were measured during the first month of GVHD treatment and proved more accurate than clinical response for predicting 6-month nonrelapse mortality (NRM). In fact, biomarker assessment revealed patients who responded to treatment but had a high risk of NRM and nonresponders who had a low risk of NRM.

The researchers also found that biomarkers changed over the first month of treatment but remained significant predictors of NRM. This suggests that modifying treatment according to biomarker findings at various time points could result in better outcomes for patients.

“We think this is going to transform the way we treat graft-versus-host disease,” said James L.M. Ferrara, MD, DSc, of the Icahn School of Medicine at Mount Sinai, New York.

Dr. Ferrara and Hrishikesh Srinagesh, along with their colleagues at Mount Sinai, have conducted extensive research with these biomarkers and presented some of their findings at the Acute Leukemia Forum of Hemedicus.

Comparing biomarkers and response

In one study, the researchers evaluated 355 patients who had undergone allogeneic hematopoietic stem cell transplant at 1 of 20 Mount Sinai Acute GVHD International Consortium (MAGIC) centers between January 2016 and February 2018. All patients developed acute GVHD and received systemic steroids as treatment.

Patients provided blood samples weekly for the first month of treatment, and concentrations of ST2 and REG3-alpha were measured in each sample. Both biomarker concentrations were used to calculate the biomarker probability of NRM.

“The concentration of those two biomarkers are put into a computer, and we get … a single number, and that gives us the probability of mortality,” Dr. Ferrara said. “[W]e call this the MAGIC algorithm probability, or MAP. And when a MAP is low, the patient has a very low chance of dying from graft-versus-host disease, when it’s intermediate, they have an intermediate risk, and when it’s high, they have a high risk.”

The researchers then compared the MAP and clinical response for their ability to predict 6-month NRM throughout the first month of therapy for acute GVHD.

MAP bests response

After 1 month of therapy, the MAP was more accurate than clinical response for predicting 6-month NRM. The area under the curve was 0.84 and 0.65, respectively (P less than .001).

Likewise, the MAP after 1 week of therapy was more accurate than clinical response at 1 month for predicting 6-month NRM. The area under the curve was 0.80 and 0.65, respectively (P less than .001).

“[T]he clinical responses were good, but not great, at predicting long-term outcome, where the biomarker, the MAP, was significantly better,” Dr. Ferrara said. “[A]t every time point we tested, the biomarkers were better than the clinical responses.”

The researchers also identified subgroups of clinical responders and nonresponders for whom MAP more accurately predicted 6-month NRM.

The team found that 61% of clinical nonresponders were actually low risk according to MAP. And the incidence of 6-month NRM was significantly lower in the MAP-designated low-risk patients than in MAP-designated high-risk patients – 22% and 56%, respectively (P less than .001).

On the other hand, 10% of clinical responders were high risk according to MAP. The incidence of 6-month NRM was significantly higher in the high-risk patients than in the low-risk patients – 40% and 13%, respectively (P less than .001).

Assessing changes over time

The researchers found that patients who were initially high risk by MAP but had not experienced NRM by 6 months had significant decreases in their MAP after 4 weeks of treatment (P = .003). Patients who did experience NRM had a significant increase in their MAP whether their initial MAP was low (P = .007) or high (P = .024).

“What we found was that patients who lived tended to either have low biomarkers at the start of treatment and stay low or start out with high biomarkers and have reductions over the first month of therapy,” Mr. Srinagesh said. “Conversely, patients who tended to do worse were those who had either increases in their biomarkers or stayed high at all time points.”

The researchers identified a threshold – 0.290 – for separating patients by mortality risk.

“Patients who started out above the threshold and then went below it had a 5-fold reduction in mortality, whereas patients who started out below the threshold and rose above it had a 5-fold increase in mortality,” Mr. Srinagesh said.

MAP in clinical trials and practice

Based on these findings and results from related studies, the researchers theorize that MAP would be a better endpoint for clinical trials than clinical response.

At present, there are three trials in which researchers are using MAP as an endpoint to assess the efficacy of treatment for GVHD (NCT02133924, NCT03459040, and NCT03846479). Dr. Ferrara said a fourth trial is set to begin this summer.

Additionally, MAP is being used in clinical practice. A company called Viracor Eurofins Clinical Diagnostics licensed the MAGIC algorithm and provides three related tests for consumer use.

Viracor’s aGVHD Pre-Symptomatic Algorithm assigns patients to high- and low-risk groups based on results from samples collected 7 days after transplant. The aGVHD Symptomatic Onset Algorithm assigns patients to high-, intermediate-, and low-risk groups. The aGVHD Post-Treatment Algorithm, which can be used 7 days or more after GVHD treatment initiation, stratifies steroid-resistant patients into high- or low-risk groups for both NRM and overall survival.

“We are still in early days of figuring out how to use [the biomarker tests], but … what I’ve heard is that people are finding them to be useful in their clinical practice,” Dr. Ferrara said.

Dr. Ferrara has an ownership interest in and receives royalties from Viracor. Mr. Srinagesh reported having no relevant conflicts of interest. The research was supported by grants from the National Cancer Institute and the American Cancer Society.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – Researchers say they have identified biomarkers that may help guide early treatment decisions in patients with acute graft-versus-host disease (GVHD).

The biomarkers, ST2 and REG3-alpha, were measured during the first month of GVHD treatment and proved more accurate than clinical response for predicting 6-month nonrelapse mortality (NRM). In fact, biomarker assessment revealed patients who responded to treatment but had a high risk of NRM and nonresponders who had a low risk of NRM.

The researchers also found that biomarkers changed over the first month of treatment but remained significant predictors of NRM. This suggests that modifying treatment according to biomarker findings at various time points could result in better outcomes for patients.

“We think this is going to transform the way we treat graft-versus-host disease,” said James L.M. Ferrara, MD, DSc, of the Icahn School of Medicine at Mount Sinai, New York.

Dr. Ferrara and Hrishikesh Srinagesh, along with their colleagues at Mount Sinai, have conducted extensive research with these biomarkers and presented some of their findings at the Acute Leukemia Forum of Hemedicus.

Comparing biomarkers and response

In one study, the researchers evaluated 355 patients who had undergone allogeneic hematopoietic stem cell transplant at 1 of 20 Mount Sinai Acute GVHD International Consortium (MAGIC) centers between January 2016 and February 2018. All patients developed acute GVHD and received systemic steroids as treatment.

Patients provided blood samples weekly for the first month of treatment, and concentrations of ST2 and REG3-alpha were measured in each sample. Both biomarker concentrations were used to calculate the biomarker probability of NRM.

“The concentration of those two biomarkers are put into a computer, and we get … a single number, and that gives us the probability of mortality,” Dr. Ferrara said. “[W]e call this the MAGIC algorithm probability, or MAP. And when a MAP is low, the patient has a very low chance of dying from graft-versus-host disease, when it’s intermediate, they have an intermediate risk, and when it’s high, they have a high risk.”

The researchers then compared the MAP and clinical response for their ability to predict 6-month NRM throughout the first month of therapy for acute GVHD.

MAP bests response

After 1 month of therapy, the MAP was more accurate than clinical response for predicting 6-month NRM. The area under the curve was 0.84 and 0.65, respectively (P less than .001).

Likewise, the MAP after 1 week of therapy was more accurate than clinical response at 1 month for predicting 6-month NRM. The area under the curve was 0.80 and 0.65, respectively (P less than .001).

“[T]he clinical responses were good, but not great, at predicting long-term outcome, where the biomarker, the MAP, was significantly better,” Dr. Ferrara said. “[A]t every time point we tested, the biomarkers were better than the clinical responses.”

The researchers also identified subgroups of clinical responders and nonresponders for whom MAP more accurately predicted 6-month NRM.

The team found that 61% of clinical nonresponders were actually low risk according to MAP. And the incidence of 6-month NRM was significantly lower in the MAP-designated low-risk patients than in MAP-designated high-risk patients – 22% and 56%, respectively (P less than .001).

On the other hand, 10% of clinical responders were high risk according to MAP. The incidence of 6-month NRM was significantly higher in the high-risk patients than in the low-risk patients – 40% and 13%, respectively (P less than .001).

Assessing changes over time

The researchers found that patients who were initially high risk by MAP but had not experienced NRM by 6 months had significant decreases in their MAP after 4 weeks of treatment (P = .003). Patients who did experience NRM had a significant increase in their MAP whether their initial MAP was low (P = .007) or high (P = .024).

“What we found was that patients who lived tended to either have low biomarkers at the start of treatment and stay low or start out with high biomarkers and have reductions over the first month of therapy,” Mr. Srinagesh said. “Conversely, patients who tended to do worse were those who had either increases in their biomarkers or stayed high at all time points.”

The researchers identified a threshold – 0.290 – for separating patients by mortality risk.

“Patients who started out above the threshold and then went below it had a 5-fold reduction in mortality, whereas patients who started out below the threshold and rose above it had a 5-fold increase in mortality,” Mr. Srinagesh said.

MAP in clinical trials and practice

Based on these findings and results from related studies, the researchers theorize that MAP would be a better endpoint for clinical trials than clinical response.

At present, there are three trials in which researchers are using MAP as an endpoint to assess the efficacy of treatment for GVHD (NCT02133924, NCT03459040, and NCT03846479). Dr. Ferrara said a fourth trial is set to begin this summer.

Additionally, MAP is being used in clinical practice. A company called Viracor Eurofins Clinical Diagnostics licensed the MAGIC algorithm and provides three related tests for consumer use.

Viracor’s aGVHD Pre-Symptomatic Algorithm assigns patients to high- and low-risk groups based on results from samples collected 7 days after transplant. The aGVHD Symptomatic Onset Algorithm assigns patients to high-, intermediate-, and low-risk groups. The aGVHD Post-Treatment Algorithm, which can be used 7 days or more after GVHD treatment initiation, stratifies steroid-resistant patients into high- or low-risk groups for both NRM and overall survival.

“We are still in early days of figuring out how to use [the biomarker tests], but … what I’ve heard is that people are finding them to be useful in their clinical practice,” Dr. Ferrara said.

Dr. Ferrara has an ownership interest in and receives royalties from Viracor. Mr. Srinagesh reported having no relevant conflicts of interest. The research was supported by grants from the National Cancer Institute and the American Cancer Society.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

SAN FRANCISCO – All five innovative startups pitched at the Shark Tank at the 2019 AGA Tech Summit, sponsored by the AGA Center for GI Innovation and Technology, are in advanced stages of development, but only one is given the opportunity to be declared the winner of the competition. The ideas ranged from a smart toilet for early disease detection to a unique strategy for obesity phenotyping, but the winner by both official decision and popular vote was a smartphone app to help patients with inflammatory bowel disease (IBD) manage the condition.

“As always, this year’s Shark Tank was a highlight of the AGA Tech Summit and represents the progress our field is making when it comes to innovation. Our panel of sharks was focused on understanding the problem each innovation solved – that’s the key when determining if an idea is novel or innovation for innovation’s sake. We were impressed with all of the technologies presented, but ultimately chose the Oshi Health IBD app as our winner because of the impact it is already having on improving the health and care of IBD patients,” said V. Raman Muthusamy, MD, AGAF, chair of the AGA Center for GI Innovation and Technology.
 

The winner: Oshi pitches “all-in-one” IBD app

By both popular vote from those attending the AGA Tech Summit as well as the six-member Shark Tank panel, Oshi Health was selected as the 2019 Shark Tank winner for its IBD app. The app was designed to help patients track symptoms, a first step in understanding flare patterns, which differ substantially between patients and emphasize the need for a personalized plan for controlling disease.

Vidyard Video

“Since we launched last June at DDW^® we have had 40,000 downloads. We are the number one IBD management app,” reported Dan Weinstein, MBA, CEO of Oshi Health.

The available app represents the first of three phases as the functionality is expanded. Currently, in addition to using the app as a tracking tool, patients can find resources to learn about their disease and to communicate with other patients about their experiences. In a second phase, information gathered by the app will be made available to physicians to provide accurate current information about disease status to better individualize therapy.

Ultimately, the app is expected to guide treatment based on information it has collected on symptom patterns and other data collected over time, although this application is further down the road and will require regulatory approval if it is designed to provide clinical advice as expected, according to Mr. Weinstein.

However, benefits have already been seen. Mr. Weinstein cited data that associated the app with a 40% improvement in medication adherence and a nearly 60-day reduction in flare duration. Calling the app “the next chapter in treat-to-target” IBD management, he believes that this is an important step forward in digital health that will improve IBD outcomes. The Shark Tank panel agreed.
 

Runners-up: Other potential innovations to improve GI health

With or without Shark Tank endorsement, the other four startups described in the competition are moving forward. Each is designed to address an important unmet need with the potential to improve patient outcomes, which is a criterion for their inclusion in the competition.

 

 

The smart toilet seat

One involves a technologically advanced toilet seat. The new seat is based on the fact that fecal matter provides insight into a broad array of disease states, but specimen collection is a hurdle for a variety of reasons, including patient resistance. A toilet seat developed by Toi Labs, called TrueLoo, is equipped with lighting and cameras that captures images of bowel movements and urination for subsequent analysis.

“The toilet seat sees what the eye cannot,” according to Vikram Kashyap, CEO of Toi Labs. He believes it has major potential for early detection of conditions ranging from dehydration to gastrointestinal cancer.

Vidyard Video

Others agree. According to Mr. Kashyap, executives of a chain of senior living facilities have already expressed interest in installing this seat to better monitor health among residents. The seat is bolted into position in place of any standard toilet seat. It collects images and data that are transmitted directly to a cellular network.

“Using our technology, the goal is to catch disease states early before they progress,” said Mr. Kashyap, who called the surveillance system a low-cost disease-screening tool. He believes the smart toilet seat could be of the most important disease detection devices developed in recent years.

AI to aid screening endoscopy

A third entrant in this year’s Shark Tank described a strategy to employ artificial intelligence (AI) to aid endoscopists in screening for dysplasia. The tool is called Ultivision and is being developed by a startup called Docbot. The CEO, Andrew Ninh, and a senior executive, Jason B. Samarasena, MD, outlined an idea that could be used in either screening colonoscopy or in surveillance of Barrett’s esophagus).

“Dysplasia is difficult to find. It is subtle and it is often missed. With better detection of dysplasia, artificial intelligence offers an opportunity to reduce risk of cancer,” Dr. Samarasena said.

The tool integrates seamlessly with existing endoscopic tools, according to Mr. Ninh. As tissue is visualized, the AI is programmed to highlight suspected dysplasia with a colored box to alert the endoscopist. The colonoscopy application is a more advanced stage of development and might be submitted for regulatory approval this year, he said. The same technology will be adapted for Barrett’s esophagus.

“It is like facial recognition for dysplasia,” said Dr. Samarasena.
 

Obesity phenotyping tool

A fourth Shark Tank entrant employs technology to phenotype obese patients to better tailor therapy. The Pheno Test, developed by Phenomix Sciences, applies “multi-omics” to a blood-based test to separate patients with obesity into four phenotypes. When therapy is tailored to the phenotype, weight loss is greater, according to Andres J. Acosta, MD, PhD, assistant professor of medicine and consultant in gastroenterology and hepatology at Mayo Clinic, Rochester, Minn.

In an initial study that compared weight loss in 55 patients treated based on phenotype with 175 patients managed with standard of care, the total body weight loss “more than doubled,” Dr. Acosta reported.

According to Dr. Acosta, obesity is driven by very different mechanisms. He described the four major phenotypes identified with his test as hungry brain (satiation signal is impaired), hungry gut (signals to eat are upregulated), emotional hunger (psychological reasons drive eating behavior), and slow metabolism (failure to burn fat at normal rates).

With the blood test, which utilizes hormones, metabolites, DNA, and other biomarkers to separate these phenotypes, treatment can be tailored appropriately, according to Dr. Acosta. His company is now seeking Food and Drug Administration clearance of the test, which he believes will have a major impact on obesity control.
 

 

 

Capsule diagnostic tool

The final entrant selected to participate in this year’s Shark Tank described an ingestible capsule that diagnoses diseases by detecting gases as it descends the gastrointestinal tract. The Atmo Gas Capsule from Atmo Biosciences measures gases at the source, accelerating the diagnosis of such diseases as irritable bowel syndrome (IBS) and IBD.

“By measuring gases at their source, the accuracy is far better than a breath test,” said Malcolm Hebblewhite, MBA, CEO of Atmo Biosciences. The capsule is an alternative to more invasive and expensive diagnostic tools and it is highly accurate.

Providing examples, Mr. Hebblewhite said that elevated levels of oxygen suggest a disorder of motility while an elevated level of carbon dioxide and hydrogen suggest IBS. The capsule transmits data to a small receiver and then on to a smartphone.

“The real-time data is displayed for the user with more complex information accessible by the practitioner remotely via the cloud,” Mr. Hebblewhite said. He cited several papers that have already been published documenting the potential of this technology.

“The capsule is a single-use disposable device that is not retrieved,” according to Mr. Hebblewhite. He reported that his company plans to pursue the diagnosis of motility as an initial clinical application. The diagnosis of IBS and other GI conditions will follow. Clinical studies are already planned.

SAN FRANCISCO – All five innovative startups pitched at the Shark Tank at the 2019 AGA Tech Summit, sponsored by the AGA Center for GI Innovation and Technology, are in advanced stages of development, but only one is given the opportunity to be declared the winner of the competition. The ideas ranged from a smart toilet for early disease detection to a unique strategy for obesity phenotyping, but the winner by both official decision and popular vote was a smartphone app to help patients with inflammatory bowel disease (IBD) manage the condition.

“As always, this year’s Shark Tank was a highlight of the AGA Tech Summit and represents the progress our field is making when it comes to innovation. Our panel of sharks was focused on understanding the problem each innovation solved – that’s the key when determining if an idea is novel or innovation for innovation’s sake. We were impressed with all of the technologies presented, but ultimately chose the Oshi Health IBD app as our winner because of the impact it is already having on improving the health and care of IBD patients,” said V. Raman Muthusamy, MD, AGAF, chair of the AGA Center for GI Innovation and Technology.
 

The winner: Oshi pitches “all-in-one” IBD app

By both popular vote from those attending the AGA Tech Summit as well as the six-member Shark Tank panel, Oshi Health was selected as the 2019 Shark Tank winner for its IBD app. The app was designed to help patients track symptoms, a first step in understanding flare patterns, which differ substantially between patients and emphasize the need for a personalized plan for controlling disease.

Vidyard Video

“Since we launched last June at DDW^® we have had 40,000 downloads. We are the number one IBD management app,” reported Dan Weinstein, MBA, CEO of Oshi Health.

The available app represents the first of three phases as the functionality is expanded. Currently, in addition to using the app as a tracking tool, patients can find resources to learn about their disease and to communicate with other patients about their experiences. In a second phase, information gathered by the app will be made available to physicians to provide accurate current information about disease status to better individualize therapy.

Ultimately, the app is expected to guide treatment based on information it has collected on symptom patterns and other data collected over time, although this application is further down the road and will require regulatory approval if it is designed to provide clinical advice as expected, according to Mr. Weinstein.

However, benefits have already been seen. Mr. Weinstein cited data that associated the app with a 40% improvement in medication adherence and a nearly 60-day reduction in flare duration. Calling the app “the next chapter in treat-to-target” IBD management, he believes that this is an important step forward in digital health that will improve IBD outcomes. The Shark Tank panel agreed.
 

Runners-up: Other potential innovations to improve GI health

With or without Shark Tank endorsement, the other four startups described in the competition are moving forward. Each is designed to address an important unmet need with the potential to improve patient outcomes, which is a criterion for their inclusion in the competition.

 

 

The smart toilet seat

One involves a technologically advanced toilet seat. The new seat is based on the fact that fecal matter provides insight into a broad array of disease states, but specimen collection is a hurdle for a variety of reasons, including patient resistance. A toilet seat developed by Toi Labs, called TrueLoo, is equipped with lighting and cameras that captures images of bowel movements and urination for subsequent analysis.

“The toilet seat sees what the eye cannot,” according to Vikram Kashyap, CEO of Toi Labs. He believes it has major potential for early detection of conditions ranging from dehydration to gastrointestinal cancer.

Vidyard Video

Others agree. According to Mr. Kashyap, executives of a chain of senior living facilities have already expressed interest in installing this seat to better monitor health among residents. The seat is bolted into position in place of any standard toilet seat. It collects images and data that are transmitted directly to a cellular network.

“Using our technology, the goal is to catch disease states early before they progress,” said Mr. Kashyap, who called the surveillance system a low-cost disease-screening tool. He believes the smart toilet seat could be of the most important disease detection devices developed in recent years.

AI to aid screening endoscopy

A third entrant in this year’s Shark Tank described a strategy to employ artificial intelligence (AI) to aid endoscopists in screening for dysplasia. The tool is called Ultivision and is being developed by a startup called Docbot. The CEO, Andrew Ninh, and a senior executive, Jason B. Samarasena, MD, outlined an idea that could be used in either screening colonoscopy or in surveillance of Barrett’s esophagus).

“Dysplasia is difficult to find. It is subtle and it is often missed. With better detection of dysplasia, artificial intelligence offers an opportunity to reduce risk of cancer,” Dr. Samarasena said.

The tool integrates seamlessly with existing endoscopic tools, according to Mr. Ninh. As tissue is visualized, the AI is programmed to highlight suspected dysplasia with a colored box to alert the endoscopist. The colonoscopy application is a more advanced stage of development and might be submitted for regulatory approval this year, he said. The same technology will be adapted for Barrett’s esophagus.

“It is like facial recognition for dysplasia,” said Dr. Samarasena.
 

Obesity phenotyping tool

A fourth Shark Tank entrant employs technology to phenotype obese patients to better tailor therapy. The Pheno Test, developed by Phenomix Sciences, applies “multi-omics” to a blood-based test to separate patients with obesity into four phenotypes. When therapy is tailored to the phenotype, weight loss is greater, according to Andres J. Acosta, MD, PhD, assistant professor of medicine and consultant in gastroenterology and hepatology at Mayo Clinic, Rochester, Minn.

In an initial study that compared weight loss in 55 patients treated based on phenotype with 175 patients managed with standard of care, the total body weight loss “more than doubled,” Dr. Acosta reported.

According to Dr. Acosta, obesity is driven by very different mechanisms. He described the four major phenotypes identified with his test as hungry brain (satiation signal is impaired), hungry gut (signals to eat are upregulated), emotional hunger (psychological reasons drive eating behavior), and slow metabolism (failure to burn fat at normal rates).

With the blood test, which utilizes hormones, metabolites, DNA, and other biomarkers to separate these phenotypes, treatment can be tailored appropriately, according to Dr. Acosta. His company is now seeking Food and Drug Administration clearance of the test, which he believes will have a major impact on obesity control.
 

 

 

Capsule diagnostic tool

The final entrant selected to participate in this year’s Shark Tank described an ingestible capsule that diagnoses diseases by detecting gases as it descends the gastrointestinal tract. The Atmo Gas Capsule from Atmo Biosciences measures gases at the source, accelerating the diagnosis of such diseases as irritable bowel syndrome (IBS) and IBD.

“By measuring gases at their source, the accuracy is far better than a breath test,” said Malcolm Hebblewhite, MBA, CEO of Atmo Biosciences. The capsule is an alternative to more invasive and expensive diagnostic tools and it is highly accurate.

Providing examples, Mr. Hebblewhite said that elevated levels of oxygen suggest a disorder of motility while an elevated level of carbon dioxide and hydrogen suggest IBS. The capsule transmits data to a small receiver and then on to a smartphone.

“The real-time data is displayed for the user with more complex information accessible by the practitioner remotely via the cloud,” Mr. Hebblewhite said. He cited several papers that have already been published documenting the potential of this technology.

“The capsule is a single-use disposable device that is not retrieved,” according to Mr. Hebblewhite. He reported that his company plans to pursue the diagnosis of motility as an initial clinical application. The diagnosis of IBS and other GI conditions will follow. Clinical studies are already planned.

SAN FRANCISCO – All five innovative startups pitched at the Shark Tank at the 2019 AGA Tech Summit, sponsored by the AGA Center for GI Innovation and Technology, are in advanced stages of development, but only one is given the opportunity to be declared the winner of the competition. The ideas ranged from a smart toilet for early disease detection to a unique strategy for obesity phenotyping, but the winner by both official decision and popular vote was a smartphone app to help patients with inflammatory bowel disease (IBD) manage the condition.

“As always, this year’s Shark Tank was a highlight of the AGA Tech Summit and represents the progress our field is making when it comes to innovation. Our panel of sharks was focused on understanding the problem each innovation solved – that’s the key when determining if an idea is novel or innovation for innovation’s sake. We were impressed with all of the technologies presented, but ultimately chose the Oshi Health IBD app as our winner because of the impact it is already having on improving the health and care of IBD patients,” said V. Raman Muthusamy, MD, AGAF, chair of the AGA Center for GI Innovation and Technology.
 

The winner: Oshi pitches “all-in-one” IBD app

By both popular vote from those attending the AGA Tech Summit as well as the six-member Shark Tank panel, Oshi Health was selected as the 2019 Shark Tank winner for its IBD app. The app was designed to help patients track symptoms, a first step in understanding flare patterns, which differ substantially between patients and emphasize the need for a personalized plan for controlling disease.

Vidyard Video

“Since we launched last June at DDW^® we have had 40,000 downloads. We are the number one IBD management app,” reported Dan Weinstein, MBA, CEO of Oshi Health.

The available app represents the first of three phases as the functionality is expanded. Currently, in addition to using the app as a tracking tool, patients can find resources to learn about their disease and to communicate with other patients about their experiences. In a second phase, information gathered by the app will be made available to physicians to provide accurate current information about disease status to better individualize therapy.

Ultimately, the app is expected to guide treatment based on information it has collected on symptom patterns and other data collected over time, although this application is further down the road and will require regulatory approval if it is designed to provide clinical advice as expected, according to Mr. Weinstein.

However, benefits have already been seen. Mr. Weinstein cited data that associated the app with a 40% improvement in medication adherence and a nearly 60-day reduction in flare duration. Calling the app “the next chapter in treat-to-target” IBD management, he believes that this is an important step forward in digital health that will improve IBD outcomes. The Shark Tank panel agreed.
 

Runners-up: Other potential innovations to improve GI health

With or without Shark Tank endorsement, the other four startups described in the competition are moving forward. Each is designed to address an important unmet need with the potential to improve patient outcomes, which is a criterion for their inclusion in the competition.

 

 

The smart toilet seat

One involves a technologically advanced toilet seat. The new seat is based on the fact that fecal matter provides insight into a broad array of disease states, but specimen collection is a hurdle for a variety of reasons, including patient resistance. A toilet seat developed by Toi Labs, called TrueLoo, is equipped with lighting and cameras that captures images of bowel movements and urination for subsequent analysis.

“The toilet seat sees what the eye cannot,” according to Vikram Kashyap, CEO of Toi Labs. He believes it has major potential for early detection of conditions ranging from dehydration to gastrointestinal cancer.

Vidyard Video

Others agree. According to Mr. Kashyap, executives of a chain of senior living facilities have already expressed interest in installing this seat to better monitor health among residents. The seat is bolted into position in place of any standard toilet seat. It collects images and data that are transmitted directly to a cellular network.

“Using our technology, the goal is to catch disease states early before they progress,” said Mr. Kashyap, who called the surveillance system a low-cost disease-screening tool. He believes the smart toilet seat could be of the most important disease detection devices developed in recent years.

AI to aid screening endoscopy

A third entrant in this year’s Shark Tank described a strategy to employ artificial intelligence (AI) to aid endoscopists in screening for dysplasia. The tool is called Ultivision and is being developed by a startup called Docbot. The CEO, Andrew Ninh, and a senior executive, Jason B. Samarasena, MD, outlined an idea that could be used in either screening colonoscopy or in surveillance of Barrett’s esophagus).

“Dysplasia is difficult to find. It is subtle and it is often missed. With better detection of dysplasia, artificial intelligence offers an opportunity to reduce risk of cancer,” Dr. Samarasena said.

The tool integrates seamlessly with existing endoscopic tools, according to Mr. Ninh. As tissue is visualized, the AI is programmed to highlight suspected dysplasia with a colored box to alert the endoscopist. The colonoscopy application is a more advanced stage of development and might be submitted for regulatory approval this year, he said. The same technology will be adapted for Barrett’s esophagus.

“It is like facial recognition for dysplasia,” said Dr. Samarasena.
 

Obesity phenotyping tool

A fourth Shark Tank entrant employs technology to phenotype obese patients to better tailor therapy. The Pheno Test, developed by Phenomix Sciences, applies “multi-omics” to a blood-based test to separate patients with obesity into four phenotypes. When therapy is tailored to the phenotype, weight loss is greater, according to Andres J. Acosta, MD, PhD, assistant professor of medicine and consultant in gastroenterology and hepatology at Mayo Clinic, Rochester, Minn.

In an initial study that compared weight loss in 55 patients treated based on phenotype with 175 patients managed with standard of care, the total body weight loss “more than doubled,” Dr. Acosta reported.

According to Dr. Acosta, obesity is driven by very different mechanisms. He described the four major phenotypes identified with his test as hungry brain (satiation signal is impaired), hungry gut (signals to eat are upregulated), emotional hunger (psychological reasons drive eating behavior), and slow metabolism (failure to burn fat at normal rates).

With the blood test, which utilizes hormones, metabolites, DNA, and other biomarkers to separate these phenotypes, treatment can be tailored appropriately, according to Dr. Acosta. His company is now seeking Food and Drug Administration clearance of the test, which he believes will have a major impact on obesity control.
 

 

 

Capsule diagnostic tool

The final entrant selected to participate in this year’s Shark Tank described an ingestible capsule that diagnoses diseases by detecting gases as it descends the gastrointestinal tract. The Atmo Gas Capsule from Atmo Biosciences measures gases at the source, accelerating the diagnosis of such diseases as irritable bowel syndrome (IBS) and IBD.

“By measuring gases at their source, the accuracy is far better than a breath test,” said Malcolm Hebblewhite, MBA, CEO of Atmo Biosciences. The capsule is an alternative to more invasive and expensive diagnostic tools and it is highly accurate.

Providing examples, Mr. Hebblewhite said that elevated levels of oxygen suggest a disorder of motility while an elevated level of carbon dioxide and hydrogen suggest IBS. The capsule transmits data to a small receiver and then on to a smartphone.

“The real-time data is displayed for the user with more complex information accessible by the practitioner remotely via the cloud,” Mr. Hebblewhite said. He cited several papers that have already been published documenting the potential of this technology.

“The capsule is a single-use disposable device that is not retrieved,” according to Mr. Hebblewhite. He reported that his company plans to pursue the diagnosis of motility as an initial clinical application. The diagnosis of IBS and other GI conditions will follow. Clinical studies are already planned.

NEWPORT BEACH, CALIF. — A model that mimics the recommender system used by Netflix and Amazon can help predict outcomes of lenalidomide treatment in patients with non–deletion 5q (non-del[5q]) myelodysplastic syndromes (MDS), according to new research.

The model was used to identify genomic biomarkers that were associated with resistance or response to lenalidomide. Researchers found these associations in 39% of patients with non-del(5q) MDS, and the model predicted response or resistance with 82% accuracy.

Yazan Madanat, MD, of the Cleveland Clinic, and his colleagues presented these findings at the Acute Leukemia Forum of Hemedicus.

Dr. Madanat explained that his group’s model is similar to the recommender system used by Netflix and Amazon, which makes suggestions for new products based on customers’ past behavior. Dr. Madanat and his colleagues used their model to show that patients with certain molecular or cytogenetic abnormalities are likely to respond or not respond to lenalidomide.

The researchers began by looking at 139 patients who had received at least two cycles of lenalidomide treatment. There were 118 patients with MDS, and 108 who had received lenalidomide monotherapy. However, the team focused on the 100 patients who had non-del(5q) MDS, 58 of whom had normal karyotype (NK) and 19 of whom had complex karyotype (CK).

The model revealed several combinations of genomic/cytogenetic abnormalities that could predict resistance to lenalidomide, including the following:

• DNMT3A and SF3B1
• EZH2 and NK
• ASXL1, TET2, and NK
• STAG2, IDH1/2, and NK
• TP53, del(5q), and CK
• BCOR/BCORL1 and NK
• JAK2, TET2, and NK
• U2AF1, +/– ETV6, and NK

However, only the following two combinations could predict response to lenalidomide:

• DDX41 and NK
• MECOM and KDM6A/B

These combinations could be applied to 39% of the patients with non-del(5q) MDS, and the model predicted response or resistance to lenalidomide with 82% accuracy.

Although the biomarkers were found in only a subset of patients, Dr. Madanat said these findings may help physicians tailor therapy for MDS patients, given the high level of accuracy the researchers observed.

“It’s really important to validate the results in a prospective manner and to ensure that we’re able to apply them clinically and potentially change the way we’re treating our patients,” he added.

Dr. Madanat and his colleagues reported having no relevant conflicts of interest.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. — A model that mimics the recommender system used by Netflix and Amazon can help predict outcomes of lenalidomide treatment in patients with non–deletion 5q (non-del[5q]) myelodysplastic syndromes (MDS), according to new research.

The model was used to identify genomic biomarkers that were associated with resistance or response to lenalidomide. Researchers found these associations in 39% of patients with non-del(5q) MDS, and the model predicted response or resistance with 82% accuracy.

Yazan Madanat, MD, of the Cleveland Clinic, and his colleagues presented these findings at the Acute Leukemia Forum of Hemedicus.

Dr. Madanat explained that his group’s model is similar to the recommender system used by Netflix and Amazon, which makes suggestions for new products based on customers’ past behavior. Dr. Madanat and his colleagues used their model to show that patients with certain molecular or cytogenetic abnormalities are likely to respond or not respond to lenalidomide.

The researchers began by looking at 139 patients who had received at least two cycles of lenalidomide treatment. There were 118 patients with MDS, and 108 who had received lenalidomide monotherapy. However, the team focused on the 100 patients who had non-del(5q) MDS, 58 of whom had normal karyotype (NK) and 19 of whom had complex karyotype (CK).

The model revealed several combinations of genomic/cytogenetic abnormalities that could predict resistance to lenalidomide, including the following:

• DNMT3A and SF3B1
• EZH2 and NK
• ASXL1, TET2, and NK
• STAG2, IDH1/2, and NK
• TP53, del(5q), and CK
• BCOR/BCORL1 and NK
• JAK2, TET2, and NK
• U2AF1, +/– ETV6, and NK

However, only the following two combinations could predict response to lenalidomide:

• DDX41 and NK
• MECOM and KDM6A/B

These combinations could be applied to 39% of the patients with non-del(5q) MDS, and the model predicted response or resistance to lenalidomide with 82% accuracy.

Although the biomarkers were found in only a subset of patients, Dr. Madanat said these findings may help physicians tailor therapy for MDS patients, given the high level of accuracy the researchers observed.

“It’s really important to validate the results in a prospective manner and to ensure that we’re able to apply them clinically and potentially change the way we’re treating our patients,” he added.

Dr. Madanat and his colleagues reported having no relevant conflicts of interest.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. — A model that mimics the recommender system used by Netflix and Amazon can help predict outcomes of lenalidomide treatment in patients with non–deletion 5q (non-del[5q]) myelodysplastic syndromes (MDS), according to new research.

The model was used to identify genomic biomarkers that were associated with resistance or response to lenalidomide. Researchers found these associations in 39% of patients with non-del(5q) MDS, and the model predicted response or resistance with 82% accuracy.

Yazan Madanat, MD, of the Cleveland Clinic, and his colleagues presented these findings at the Acute Leukemia Forum of Hemedicus.

Dr. Madanat explained that his group’s model is similar to the recommender system used by Netflix and Amazon, which makes suggestions for new products based on customers’ past behavior. Dr. Madanat and his colleagues used their model to show that patients with certain molecular or cytogenetic abnormalities are likely to respond or not respond to lenalidomide.

The researchers began by looking at 139 patients who had received at least two cycles of lenalidomide treatment. There were 118 patients with MDS, and 108 who had received lenalidomide monotherapy. However, the team focused on the 100 patients who had non-del(5q) MDS, 58 of whom had normal karyotype (NK) and 19 of whom had complex karyotype (CK).

The model revealed several combinations of genomic/cytogenetic abnormalities that could predict resistance to lenalidomide, including the following:

• DNMT3A and SF3B1
• EZH2 and NK
• ASXL1, TET2, and NK
• STAG2, IDH1/2, and NK
• TP53, del(5q), and CK
• BCOR/BCORL1 and NK
• JAK2, TET2, and NK
• U2AF1, +/– ETV6, and NK

However, only the following two combinations could predict response to lenalidomide:

• DDX41 and NK
• MECOM and KDM6A/B

These combinations could be applied to 39% of the patients with non-del(5q) MDS, and the model predicted response or resistance to lenalidomide with 82% accuracy.

Although the biomarkers were found in only a subset of patients, Dr. Madanat said these findings may help physicians tailor therapy for MDS patients, given the high level of accuracy the researchers observed.

“It’s really important to validate the results in a prospective manner and to ensure that we’re able to apply them clinically and potentially change the way we’re treating our patients,” he added.

Dr. Madanat and his colleagues reported having no relevant conflicts of interest.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEW ORLEANS – A well-attended afternoon symposium on transgender medicine gave participants at the annual meeting of the Endocrine Society a solid grounding in transgender care, from prepubescence through adulthood. Here, Joshua Safer, MD, and Michael Irwig, MD, discuss highlights of the symposium, which brought together research, best practices, and clinical practice pearls.

In his presentation, Dr. Safer focused on evidence-based strategies in medical education that can increase knowledge and comfort for trainees who are caring for transgender individuals. The basics, he said, begin with presenting well-established, scientific principles supporting current standards of transgender care.

Dr. Safer, executive director of the Mount Sinai Center for Transgender Medicine and Surgery, New York, pointed out the critical role of gonadotropin-releasing hormone antagonists in delaying puberty for transgender girls. Blockade of puberty – and elevated testosterone – can forestall otherwise irreversible male secondary sex characteristics. These include laryngeal enlargement and bony changes of facial structure, for example.

Dr. Irwig, director of andrology at George Washington University, Washington, laid out the basics of transgender hormone therapy, including clinical pearls, such as avoiding ethinyl estradiol because of the heightened risk of venous thromboembolism.

Dr. Safer is a member of the editorial advisory board of Clinical Endocrinology News. He reported that he has received consulting fees from Endo Pharmaceuticals and that his spouse is an employee of Parexel. Dr. Irwig reported no relevant conflicts of interest or financial disclosures.

[email protected]

NEW ORLEANS – A well-attended afternoon symposium on transgender medicine gave participants at the annual meeting of the Endocrine Society a solid grounding in transgender care, from prepubescence through adulthood. Here, Joshua Safer, MD, and Michael Irwig, MD, discuss highlights of the symposium, which brought together research, best practices, and clinical practice pearls.

In his presentation, Dr. Safer focused on evidence-based strategies in medical education that can increase knowledge and comfort for trainees who are caring for transgender individuals. The basics, he said, begin with presenting well-established, scientific principles supporting current standards of transgender care.

Dr. Safer, executive director of the Mount Sinai Center for Transgender Medicine and Surgery, New York, pointed out the critical role of gonadotropin-releasing hormone antagonists in delaying puberty for transgender girls. Blockade of puberty – and elevated testosterone – can forestall otherwise irreversible male secondary sex characteristics. These include laryngeal enlargement and bony changes of facial structure, for example.

Dr. Irwig, director of andrology at George Washington University, Washington, laid out the basics of transgender hormone therapy, including clinical pearls, such as avoiding ethinyl estradiol because of the heightened risk of venous thromboembolism.

Dr. Safer is a member of the editorial advisory board of Clinical Endocrinology News. He reported that he has received consulting fees from Endo Pharmaceuticals and that his spouse is an employee of Parexel. Dr. Irwig reported no relevant conflicts of interest or financial disclosures.

[email protected]

NEW ORLEANS – A well-attended afternoon symposium on transgender medicine gave participants at the annual meeting of the Endocrine Society a solid grounding in transgender care, from prepubescence through adulthood. Here, Joshua Safer, MD, and Michael Irwig, MD, discuss highlights of the symposium, which brought together research, best practices, and clinical practice pearls.

In his presentation, Dr. Safer focused on evidence-based strategies in medical education that can increase knowledge and comfort for trainees who are caring for transgender individuals. The basics, he said, begin with presenting well-established, scientific principles supporting current standards of transgender care.

Dr. Safer, executive director of the Mount Sinai Center for Transgender Medicine and Surgery, New York, pointed out the critical role of gonadotropin-releasing hormone antagonists in delaying puberty for transgender girls. Blockade of puberty – and elevated testosterone – can forestall otherwise irreversible male secondary sex characteristics. These include laryngeal enlargement and bony changes of facial structure, for example.

Dr. Irwig, director of andrology at George Washington University, Washington, laid out the basics of transgender hormone therapy, including clinical pearls, such as avoiding ethinyl estradiol because of the heightened risk of venous thromboembolism.

Dr. Safer is a member of the editorial advisory board of Clinical Endocrinology News. He reported that he has received consulting fees from Endo Pharmaceuticals and that his spouse is an employee of Parexel. Dr. Irwig reported no relevant conflicts of interest or financial disclosures.

[email protected]

SAN FRANCISCO – New York University researchers have found a ninefold increase of the bacterium Ruminococcus gnavus in the intestines of particularly ill lupus patients.

Not only that, but those patients also had highly elevated antibodies to an endotoxin-like antigen released by one particular R. gnavus strain.

That antigen is “very proinflammatory, very immunogenic. We are wondering if this is actually [what drives] the immune activation that results in immune complexes in the glomeruli” of patients with lupus nephritis, said investigator Gregg Silverman, MD, a professor of medicine and pathology and head of the laboratory of B-cell immunobiology at New York University.

R. gnavus is an obligate anaerobe found in the guts of most people, but in lupus, it might be a problem.

“We are finding a very specific relationship with lupus patients and this bacteria – and this particular antibody,” Dr. Silverman explained in an interview at an international congress on systemic lupus erythematosus. “There’s an expansion of this particular bug, but also a contraction of others” as disease activity progresses.

“It speaks to an imbalance,” he added, and it suggests a role for probiotics or even fecal transplants to restore order.

“What if instead of killing the immune system” in lupus treatment, “we should be reducing or removing a single bacterium or a single molecule?” he asked.

Dr. Silverman is one of many researchers working to unravel the role of the human microbiome in both disease and health. His findings are preliminary, and, as he cautioned, correlation is not causation. But the implications are remarkable, Dr. Silverman noted.

SAN FRANCISCO – New York University researchers have found a ninefold increase of the bacterium Ruminococcus gnavus in the intestines of particularly ill lupus patients.

Not only that, but those patients also had highly elevated antibodies to an endotoxin-like antigen released by one particular R. gnavus strain.

That antigen is “very proinflammatory, very immunogenic. We are wondering if this is actually [what drives] the immune activation that results in immune complexes in the glomeruli” of patients with lupus nephritis, said investigator Gregg Silverman, MD, a professor of medicine and pathology and head of the laboratory of B-cell immunobiology at New York University.

R. gnavus is an obligate anaerobe found in the guts of most people, but in lupus, it might be a problem.

“We are finding a very specific relationship with lupus patients and this bacteria – and this particular antibody,” Dr. Silverman explained in an interview at an international congress on systemic lupus erythematosus. “There’s an expansion of this particular bug, but also a contraction of others” as disease activity progresses.

“It speaks to an imbalance,” he added, and it suggests a role for probiotics or even fecal transplants to restore order.

“What if instead of killing the immune system” in lupus treatment, “we should be reducing or removing a single bacterium or a single molecule?” he asked.

Dr. Silverman is one of many researchers working to unravel the role of the human microbiome in both disease and health. His findings are preliminary, and, as he cautioned, correlation is not causation. But the implications are remarkable, Dr. Silverman noted.

SAN FRANCISCO – New York University researchers have found a ninefold increase of the bacterium Ruminococcus gnavus in the intestines of particularly ill lupus patients.

Not only that, but those patients also had highly elevated antibodies to an endotoxin-like antigen released by one particular R. gnavus strain.

That antigen is “very proinflammatory, very immunogenic. We are wondering if this is actually [what drives] the immune activation that results in immune complexes in the glomeruli” of patients with lupus nephritis, said investigator Gregg Silverman, MD, a professor of medicine and pathology and head of the laboratory of B-cell immunobiology at New York University.

R. gnavus is an obligate anaerobe found in the guts of most people, but in lupus, it might be a problem.

“We are finding a very specific relationship with lupus patients and this bacteria – and this particular antibody,” Dr. Silverman explained in an interview at an international congress on systemic lupus erythematosus. “There’s an expansion of this particular bug, but also a contraction of others” as disease activity progresses.

“It speaks to an imbalance,” he added, and it suggests a role for probiotics or even fecal transplants to restore order.

“What if instead of killing the immune system” in lupus treatment, “we should be reducing or removing a single bacterium or a single molecule?” he asked.

Dr. Silverman is one of many researchers working to unravel the role of the human microbiome in both disease and health. His findings are preliminary, and, as he cautioned, correlation is not causation. But the implications are remarkable, Dr. Silverman noted.

NEWPORT BEACH, CALIF. – A five-drug regimen was deemed safe in patients with newly diagnosed acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS), and it appeared to be effective regardless of patients’ FLT3 status.

Researchers tested this regimen – sorafenib plus granulocyte colony–stimulating factor (G-CSF), cladribine, high-dose cytarabine, and mitoxantrone (GCLAM) – in a phase 1 trial.

Kelsey-Leigh Garcia, a clinical research coordinator at Seattle Cancer Care Alliance, and her colleagues presented the results at the Acute Leukemia Forum of Hemedicus.

“The background for doing this study was our institutional results of GCLAM [Leukemia. 2018 Nov;32(11):2352-62] that showed a higher minimal residual disease–negative complete response rate than 7+3 [cytarabine continuously for 7 days, along with short infusions of an anthracycline on each of the first 3 days] and an international study by Röllig that showed the addition of sorafenib to 7+3 increased event-free survival versus [7+3 and] placebo [Lancet Oncol. 2015 Dec;16(16):1691-9],” Ms. Garcia said.

“GCLAM is the standard backbone at our institution, and we wanted to ask the question, ‘If we add sorafenib, can this improve upon the results of GCLAM?’ ” said Anna Halpern, MD, a hematologist-oncologist at the University of Washington, Seattle and principal investigator of the phase 1 trial.

The trial (NCT02728050) included 47 patients, 39 with AML and 8 with MDS. Patients were aged 60 years or younger and had a median age of 48. They had a median treatment-related mortality score of 1.76 (range, 0.19-12.26). A total of 11 patients (23%) had FLT3-ITD, and 4 (9%) had FLT3-TKD.

Treatment and toxicity

For induction, patients received G-CSF at 5 mcg/kg on days 0-5, cladribine at 5 mg/m² on days 1-5, and cytarabine at 2 g/m² on days 1-5. Mitoxantrone was given at 10 mg/m², 12 mg/m², 15 mg/m², or 18 mg/m² on days 1-3. Sorafenib was given at 200 mg twice daily, 400 mg in the morning and 200 mg in the afternoon, or 400 mg b.i.d. on days 10-19.

For consolidation, patients could receive up to four cycles of G-CSF, cladribine, and cytarabine plus sorafenib on days 8-27. Patients who did not proceed to transplant could receive 12 months of sorafenib as maintenance therapy.

There were four dose-limiting toxicities.

• Grade 4 intracranial hemorrhage with mitoxantrone at 12 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 4 prolonged count recovery with mitoxantrone at 15 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 4 sepsis, Sweet syndrome, and Bell’s palsy with mitoxantrone at 18 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 3 cardiomyopathy and acute pericarditis with mitoxantrone at 18 mg/m² and sorafenib at 400 mg b.i.d.

However, these toxicities did not define the maximum-tolerated dose. Therefore, the recommended phase 2 dose of mitoxantrone is 18 mg/m², and the recommended phase 2 dose of sorafenib is 400 mg b.i.d.

There were no grade 5 treatment-related adverse events. Grade 3 events included febrile neutropenia (90%), maculopapular rash (20%), infections (10%), hand-foot syndrome (2%), and diarrhea (1%). Grade 4 events included sepsis, intracranial hemorrhage, and oral mucositis (all 1%).

Response and survival

Among the 46 evaluable patients, 83% achieved a complete response, 78% had a minimal residual disease–negative complete response, and 4% had a minimal residual disease–negative complete response with incomplete count recovery. A morphological leukemia-free state was achieved by 4% of patients, and 8% had resistant disease.

Fifty-nine percent of patients went on to transplant. The median overall survival had not been reached at a median follow-up of 10 months.

The researchers compared outcomes in this trial with outcomes in a cohort of patients who had received GCLAM alone, and there were no significant differences in overall survival or event-free survival.

“The trial wasn’t powered, necessarily, for efficacy, but we compared these results to our historical cohort of medically matched and age-matched patients treated with GCLAM alone and, so far, found no differences in survival between the two groups,” Dr. Halpern said.

She noted, however, that follow-up was short in the sorafenib trial, and it included patients treated with all dose levels of sorafenib and mitoxantrone.

A phase 2 study of sorafenib plus GCLAM in newly diagnosed AML or high-risk MDS is now underway.

Dr. Halpern and Ms. Garcia reported that they had no conflicts of interest. The phase 1 trial was sponsored by the University of Washington in collaboration with the National Cancer Institute, and funding was provided by Bayer.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – A five-drug regimen was deemed safe in patients with newly diagnosed acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS), and it appeared to be effective regardless of patients’ FLT3 status.

Researchers tested this regimen – sorafenib plus granulocyte colony–stimulating factor (G-CSF), cladribine, high-dose cytarabine, and mitoxantrone (GCLAM) – in a phase 1 trial.

Kelsey-Leigh Garcia, a clinical research coordinator at Seattle Cancer Care Alliance, and her colleagues presented the results at the Acute Leukemia Forum of Hemedicus.

“The background for doing this study was our institutional results of GCLAM [Leukemia. 2018 Nov;32(11):2352-62] that showed a higher minimal residual disease–negative complete response rate than 7+3 [cytarabine continuously for 7 days, along with short infusions of an anthracycline on each of the first 3 days] and an international study by Röllig that showed the addition of sorafenib to 7+3 increased event-free survival versus [7+3 and] placebo [Lancet Oncol. 2015 Dec;16(16):1691-9],” Ms. Garcia said.

“GCLAM is the standard backbone at our institution, and we wanted to ask the question, ‘If we add sorafenib, can this improve upon the results of GCLAM?’ ” said Anna Halpern, MD, a hematologist-oncologist at the University of Washington, Seattle and principal investigator of the phase 1 trial.

The trial (NCT02728050) included 47 patients, 39 with AML and 8 with MDS. Patients were aged 60 years or younger and had a median age of 48. They had a median treatment-related mortality score of 1.76 (range, 0.19-12.26). A total of 11 patients (23%) had FLT3-ITD, and 4 (9%) had FLT3-TKD.

Treatment and toxicity

For induction, patients received G-CSF at 5 mcg/kg on days 0-5, cladribine at 5 mg/m² on days 1-5, and cytarabine at 2 g/m² on days 1-5. Mitoxantrone was given at 10 mg/m², 12 mg/m², 15 mg/m², or 18 mg/m² on days 1-3. Sorafenib was given at 200 mg twice daily, 400 mg in the morning and 200 mg in the afternoon, or 400 mg b.i.d. on days 10-19.

For consolidation, patients could receive up to four cycles of G-CSF, cladribine, and cytarabine plus sorafenib on days 8-27. Patients who did not proceed to transplant could receive 12 months of sorafenib as maintenance therapy.

There were four dose-limiting toxicities.

• Grade 4 intracranial hemorrhage with mitoxantrone at 12 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 4 prolonged count recovery with mitoxantrone at 15 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 4 sepsis, Sweet syndrome, and Bell’s palsy with mitoxantrone at 18 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 3 cardiomyopathy and acute pericarditis with mitoxantrone at 18 mg/m² and sorafenib at 400 mg b.i.d.

However, these toxicities did not define the maximum-tolerated dose. Therefore, the recommended phase 2 dose of mitoxantrone is 18 mg/m², and the recommended phase 2 dose of sorafenib is 400 mg b.i.d.

There were no grade 5 treatment-related adverse events. Grade 3 events included febrile neutropenia (90%), maculopapular rash (20%), infections (10%), hand-foot syndrome (2%), and diarrhea (1%). Grade 4 events included sepsis, intracranial hemorrhage, and oral mucositis (all 1%).

Response and survival

Among the 46 evaluable patients, 83% achieved a complete response, 78% had a minimal residual disease–negative complete response, and 4% had a minimal residual disease–negative complete response with incomplete count recovery. A morphological leukemia-free state was achieved by 4% of patients, and 8% had resistant disease.

Fifty-nine percent of patients went on to transplant. The median overall survival had not been reached at a median follow-up of 10 months.

The researchers compared outcomes in this trial with outcomes in a cohort of patients who had received GCLAM alone, and there were no significant differences in overall survival or event-free survival.

“The trial wasn’t powered, necessarily, for efficacy, but we compared these results to our historical cohort of medically matched and age-matched patients treated with GCLAM alone and, so far, found no differences in survival between the two groups,” Dr. Halpern said.

She noted, however, that follow-up was short in the sorafenib trial, and it included patients treated with all dose levels of sorafenib and mitoxantrone.

A phase 2 study of sorafenib plus GCLAM in newly diagnosed AML or high-risk MDS is now underway.

Dr. Halpern and Ms. Garcia reported that they had no conflicts of interest. The phase 1 trial was sponsored by the University of Washington in collaboration with the National Cancer Institute, and funding was provided by Bayer.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – A five-drug regimen was deemed safe in patients with newly diagnosed acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS), and it appeared to be effective regardless of patients’ FLT3 status.

Researchers tested this regimen – sorafenib plus granulocyte colony–stimulating factor (G-CSF), cladribine, high-dose cytarabine, and mitoxantrone (GCLAM) – in a phase 1 trial.

Kelsey-Leigh Garcia, a clinical research coordinator at Seattle Cancer Care Alliance, and her colleagues presented the results at the Acute Leukemia Forum of Hemedicus.

“The background for doing this study was our institutional results of GCLAM [Leukemia. 2018 Nov;32(11):2352-62] that showed a higher minimal residual disease–negative complete response rate than 7+3 [cytarabine continuously for 7 days, along with short infusions of an anthracycline on each of the first 3 days] and an international study by Röllig that showed the addition of sorafenib to 7+3 increased event-free survival versus [7+3 and] placebo [Lancet Oncol. 2015 Dec;16(16):1691-9],” Ms. Garcia said.

“GCLAM is the standard backbone at our institution, and we wanted to ask the question, ‘If we add sorafenib, can this improve upon the results of GCLAM?’ ” said Anna Halpern, MD, a hematologist-oncologist at the University of Washington, Seattle and principal investigator of the phase 1 trial.

The trial (NCT02728050) included 47 patients, 39 with AML and 8 with MDS. Patients were aged 60 years or younger and had a median age of 48. They had a median treatment-related mortality score of 1.76 (range, 0.19-12.26). A total of 11 patients (23%) had FLT3-ITD, and 4 (9%) had FLT3-TKD.

Treatment and toxicity

For induction, patients received G-CSF at 5 mcg/kg on days 0-5, cladribine at 5 mg/m² on days 1-5, and cytarabine at 2 g/m² on days 1-5. Mitoxantrone was given at 10 mg/m², 12 mg/m², 15 mg/m², or 18 mg/m² on days 1-3. Sorafenib was given at 200 mg twice daily, 400 mg in the morning and 200 mg in the afternoon, or 400 mg b.i.d. on days 10-19.

For consolidation, patients could receive up to four cycles of G-CSF, cladribine, and cytarabine plus sorafenib on days 8-27. Patients who did not proceed to transplant could receive 12 months of sorafenib as maintenance therapy.

There were four dose-limiting toxicities.

• Grade 4 intracranial hemorrhage with mitoxantrone at 12 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 4 prolonged count recovery with mitoxantrone at 15 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 4 sepsis, Sweet syndrome, and Bell’s palsy with mitoxantrone at 18 mg/m² and sorafenib at 200 mg b.i.d.
• Grade 3 cardiomyopathy and acute pericarditis with mitoxantrone at 18 mg/m² and sorafenib at 400 mg b.i.d.

However, these toxicities did not define the maximum-tolerated dose. Therefore, the recommended phase 2 dose of mitoxantrone is 18 mg/m², and the recommended phase 2 dose of sorafenib is 400 mg b.i.d.

There were no grade 5 treatment-related adverse events. Grade 3 events included febrile neutropenia (90%), maculopapular rash (20%), infections (10%), hand-foot syndrome (2%), and diarrhea (1%). Grade 4 events included sepsis, intracranial hemorrhage, and oral mucositis (all 1%).

Response and survival

Among the 46 evaluable patients, 83% achieved a complete response, 78% had a minimal residual disease–negative complete response, and 4% had a minimal residual disease–negative complete response with incomplete count recovery. A morphological leukemia-free state was achieved by 4% of patients, and 8% had resistant disease.

Fifty-nine percent of patients went on to transplant. The median overall survival had not been reached at a median follow-up of 10 months.

The researchers compared outcomes in this trial with outcomes in a cohort of patients who had received GCLAM alone, and there were no significant differences in overall survival or event-free survival.

“The trial wasn’t powered, necessarily, for efficacy, but we compared these results to our historical cohort of medically matched and age-matched patients treated with GCLAM alone and, so far, found no differences in survival between the two groups,” Dr. Halpern said.

She noted, however, that follow-up was short in the sorafenib trial, and it included patients treated with all dose levels of sorafenib and mitoxantrone.

A phase 2 study of sorafenib plus GCLAM in newly diagnosed AML or high-risk MDS is now underway.

Dr. Halpern and Ms. Garcia reported that they had no conflicts of interest. The phase 1 trial was sponsored by the University of Washington in collaboration with the National Cancer Institute, and funding was provided by Bayer.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

PHILADELPHIA – Douglas S. Paauw, MD, discussed why it’s been a bad year for antihypertensives, and provided updates on the side effects of statins and sodium-glucose cotransporter 2 inhibitors, in a video interview at the annual meeting of the American College of Physicians.

Dr. Paauw, professor of medicine at the University of Washington, Seattle, began by discussing some of the issues that occurred with antihypertensive drugs in the past year. These included the link between hydrochlorothiazide use and the increased risk of nonmelanoma skin cancers, the recalls of many drug lots of angiotensin II receptor blockers, and a study that found an increased risk of lung cancer in people who were taking ACE inhibitors.

He then described the findings of studies that examined the links between statins and muscle pain and other new research on these drugs.

He also warned physicians to be particularity cautious about prescribing sodium-glucose cotransporter 2 inhibitors to certain kinds of patients.

Dr. Paauw concluded by explaining why clarithromycin is his most hated drug.

Dr. Paauw is also the Rathmann Family Foundation Endowed Chair for Patient-Centered Clinical Education and the medicine clerkship director at the University of Washington.

[email protected]

PHILADELPHIA – Douglas S. Paauw, MD, discussed why it’s been a bad year for antihypertensives, and provided updates on the side effects of statins and sodium-glucose cotransporter 2 inhibitors, in a video interview at the annual meeting of the American College of Physicians.

Dr. Paauw, professor of medicine at the University of Washington, Seattle, began by discussing some of the issues that occurred with antihypertensive drugs in the past year. These included the link between hydrochlorothiazide use and the increased risk of nonmelanoma skin cancers, the recalls of many drug lots of angiotensin II receptor blockers, and a study that found an increased risk of lung cancer in people who were taking ACE inhibitors.

He then described the findings of studies that examined the links between statins and muscle pain and other new research on these drugs.

He also warned physicians to be particularity cautious about prescribing sodium-glucose cotransporter 2 inhibitors to certain kinds of patients.

Dr. Paauw concluded by explaining why clarithromycin is his most hated drug.

Dr. Paauw is also the Rathmann Family Foundation Endowed Chair for Patient-Centered Clinical Education and the medicine clerkship director at the University of Washington.

[email protected]

PHILADELPHIA – Douglas S. Paauw, MD, discussed why it’s been a bad year for antihypertensives, and provided updates on the side effects of statins and sodium-glucose cotransporter 2 inhibitors, in a video interview at the annual meeting of the American College of Physicians.

Dr. Paauw, professor of medicine at the University of Washington, Seattle, began by discussing some of the issues that occurred with antihypertensive drugs in the past year. These included the link between hydrochlorothiazide use and the increased risk of nonmelanoma skin cancers, the recalls of many drug lots of angiotensin II receptor blockers, and a study that found an increased risk of lung cancer in people who were taking ACE inhibitors.

He then described the findings of studies that examined the links between statins and muscle pain and other new research on these drugs.

He also warned physicians to be particularity cautious about prescribing sodium-glucose cotransporter 2 inhibitors to certain kinds of patients.

Dr. Paauw concluded by explaining why clarithromycin is his most hated drug.

Dr. Paauw is also the Rathmann Family Foundation Endowed Chair for Patient-Centered Clinical Education and the medicine clerkship director at the University of Washington.

[email protected]

NEWPORT BEACH, CALIF. – Researchers have shown they can identify transgender leukemia patients by detecting gender-karyotype mismatches, but some transgender patients may be overlooked with this method.

The researchers’ work also highlights how little we know about transgender patients with leukemia and other cancers.

Alison Alpert, MD, of the University of Rochester (N.Y.) Medical Center, and her colleagues conducted this research and presented their findings in a poster at the Acute Leukemia Forum of Hemedicus.

“There’s almost no data about transgender people with cancer ... in terms of prevalence or anything else,” Dr. Alpert noted. “And because we don’t know which patients with cancer are transgender, we can’t begin to answer any of the other big questions for patients.”

Specifically, it’s unclear what kinds of cancer transgender patients have, if there are health disparities among transgender patients, if it is safe to continue hormone therapy during cancer treatment, and if it is possible to do transition-related surgeries in the context of cancer care.

With this in mind, Dr. Alpert and her colleagues set out to identify transgender patients by detecting gender-karyotype mismatches. The team analyzed data on patients with acute myeloid leukemia (AML) or myelodysplastic syndromes enrolled in five Southwest Oncology Group (SWOG) trials.

Of the 1,748 patients analyzed, six (0.3%) had a gender-karyotype mismatch. Five patients had a 46,XY karyotype and identified as female, and one patient had a 46,XX karyotype and identified as male.

“Some transgender patients have their gender identity accurately reflected in the electronic medical record, [but] some transgender patients probably don’t,” Dr. Alpert noted. “So we identified some, but probably not all, and probably not even most, transgender patients with leukemia in this cohort.”

All six of the transgender patients identified had AML, and all were white. They ranged in age from 18 to 57 years. Four patients had achieved a complete response to therapy, and two had refractory disease.

Four patients, including one who was refractory, were still alive at last follow-up. The remaining two patients, including one who had achieved a complete response, had died.

The transgender patients identified in this analysis represent a very small percentage of the population studied, Dr. Alpert noted. Therefore, the researchers could not draw any conclusions about transgender patients with AML.

“Mostly, what we did was, we pointed out how little information we have,” Dr. Alpert said. “Oncologists don’t routinely collect gender identity information, and this information doesn’t exist in cooperative group databases either.”

“But going forward, what probably really needs to happen is that oncologists need to ask their patients whether they are transgender or not. And then, ideally, consent forms for large cooperative groups like SWOG would include gender identity data, and then we would be able to answer some of our other questions and better counsel our patients.”

Dr. Alpert and her colleagues are hoping to gain insights regarding transgender patients with lymphoma as well. The researchers are analyzing the lymphoma database at the University of Rochester Medical Center, which includes about 2,200 patients.

The team is attempting to identify transgender lymphoma patients using gender-karyotype mismatch as well as other methods, including assessing patients’ medication and surgical histories, determining whether patients have any aliases, and looking for the word “transgender” in patient charts.

“Given that the country is finally starting to talk about transgender patients, their health disparities, and their needs and experiences, it’s really time that we start collecting this data,” Dr. Alpert said.

“[I]f we are able to start to collect this data, it can help us build relationships with our patients, improve their care and outcomes, and, hopefully, be able to better counsel them about hormones and surgery.”

Dr. Alpert and her colleagues did not disclose any conflicts of interest.

The Acute Leukemia Forum is organized by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – Researchers have shown they can identify transgender leukemia patients by detecting gender-karyotype mismatches, but some transgender patients may be overlooked with this method.

The researchers’ work also highlights how little we know about transgender patients with leukemia and other cancers.

Alison Alpert, MD, of the University of Rochester (N.Y.) Medical Center, and her colleagues conducted this research and presented their findings in a poster at the Acute Leukemia Forum of Hemedicus.

“There’s almost no data about transgender people with cancer ... in terms of prevalence or anything else,” Dr. Alpert noted. “And because we don’t know which patients with cancer are transgender, we can’t begin to answer any of the other big questions for patients.”

Specifically, it’s unclear what kinds of cancer transgender patients have, if there are health disparities among transgender patients, if it is safe to continue hormone therapy during cancer treatment, and if it is possible to do transition-related surgeries in the context of cancer care.

With this in mind, Dr. Alpert and her colleagues set out to identify transgender patients by detecting gender-karyotype mismatches. The team analyzed data on patients with acute myeloid leukemia (AML) or myelodysplastic syndromes enrolled in five Southwest Oncology Group (SWOG) trials.

Of the 1,748 patients analyzed, six (0.3%) had a gender-karyotype mismatch. Five patients had a 46,XY karyotype and identified as female, and one patient had a 46,XX karyotype and identified as male.

“Some transgender patients have their gender identity accurately reflected in the electronic medical record, [but] some transgender patients probably don’t,” Dr. Alpert noted. “So we identified some, but probably not all, and probably not even most, transgender patients with leukemia in this cohort.”

All six of the transgender patients identified had AML, and all were white. They ranged in age from 18 to 57 years. Four patients had achieved a complete response to therapy, and two had refractory disease.

Four patients, including one who was refractory, were still alive at last follow-up. The remaining two patients, including one who had achieved a complete response, had died.

The transgender patients identified in this analysis represent a very small percentage of the population studied, Dr. Alpert noted. Therefore, the researchers could not draw any conclusions about transgender patients with AML.

“Mostly, what we did was, we pointed out how little information we have,” Dr. Alpert said. “Oncologists don’t routinely collect gender identity information, and this information doesn’t exist in cooperative group databases either.”

“But going forward, what probably really needs to happen is that oncologists need to ask their patients whether they are transgender or not. And then, ideally, consent forms for large cooperative groups like SWOG would include gender identity data, and then we would be able to answer some of our other questions and better counsel our patients.”

Dr. Alpert and her colleagues are hoping to gain insights regarding transgender patients with lymphoma as well. The researchers are analyzing the lymphoma database at the University of Rochester Medical Center, which includes about 2,200 patients.

The team is attempting to identify transgender lymphoma patients using gender-karyotype mismatch as well as other methods, including assessing patients’ medication and surgical histories, determining whether patients have any aliases, and looking for the word “transgender” in patient charts.

“Given that the country is finally starting to talk about transgender patients, their health disparities, and their needs and experiences, it’s really time that we start collecting this data,” Dr. Alpert said.

“[I]f we are able to start to collect this data, it can help us build relationships with our patients, improve their care and outcomes, and, hopefully, be able to better counsel them about hormones and surgery.”

Dr. Alpert and her colleagues did not disclose any conflicts of interest.

The Acute Leukemia Forum is organized by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – Researchers have shown they can identify transgender leukemia patients by detecting gender-karyotype mismatches, but some transgender patients may be overlooked with this method.

The researchers’ work also highlights how little we know about transgender patients with leukemia and other cancers.

Alison Alpert, MD, of the University of Rochester (N.Y.) Medical Center, and her colleagues conducted this research and presented their findings in a poster at the Acute Leukemia Forum of Hemedicus.

“There’s almost no data about transgender people with cancer ... in terms of prevalence or anything else,” Dr. Alpert noted. “And because we don’t know which patients with cancer are transgender, we can’t begin to answer any of the other big questions for patients.”

Specifically, it’s unclear what kinds of cancer transgender patients have, if there are health disparities among transgender patients, if it is safe to continue hormone therapy during cancer treatment, and if it is possible to do transition-related surgeries in the context of cancer care.

With this in mind, Dr. Alpert and her colleagues set out to identify transgender patients by detecting gender-karyotype mismatches. The team analyzed data on patients with acute myeloid leukemia (AML) or myelodysplastic syndromes enrolled in five Southwest Oncology Group (SWOG) trials.

Of the 1,748 patients analyzed, six (0.3%) had a gender-karyotype mismatch. Five patients had a 46,XY karyotype and identified as female, and one patient had a 46,XX karyotype and identified as male.

“Some transgender patients have their gender identity accurately reflected in the electronic medical record, [but] some transgender patients probably don’t,” Dr. Alpert noted. “So we identified some, but probably not all, and probably not even most, transgender patients with leukemia in this cohort.”

All six of the transgender patients identified had AML, and all were white. They ranged in age from 18 to 57 years. Four patients had achieved a complete response to therapy, and two had refractory disease.

Four patients, including one who was refractory, were still alive at last follow-up. The remaining two patients, including one who had achieved a complete response, had died.

The transgender patients identified in this analysis represent a very small percentage of the population studied, Dr. Alpert noted. Therefore, the researchers could not draw any conclusions about transgender patients with AML.

“Mostly, what we did was, we pointed out how little information we have,” Dr. Alpert said. “Oncologists don’t routinely collect gender identity information, and this information doesn’t exist in cooperative group databases either.”

“But going forward, what probably really needs to happen is that oncologists need to ask their patients whether they are transgender or not. And then, ideally, consent forms for large cooperative groups like SWOG would include gender identity data, and then we would be able to answer some of our other questions and better counsel our patients.”

Dr. Alpert and her colleagues are hoping to gain insights regarding transgender patients with lymphoma as well. The researchers are analyzing the lymphoma database at the University of Rochester Medical Center, which includes about 2,200 patients.

The team is attempting to identify transgender lymphoma patients using gender-karyotype mismatch as well as other methods, including assessing patients’ medication and surgical histories, determining whether patients have any aliases, and looking for the word “transgender” in patient charts.

“Given that the country is finally starting to talk about transgender patients, their health disparities, and their needs and experiences, it’s really time that we start collecting this data,” Dr. Alpert said.

“[I]f we are able to start to collect this data, it can help us build relationships with our patients, improve their care and outcomes, and, hopefully, be able to better counsel them about hormones and surgery.”

Dr. Alpert and her colleagues did not disclose any conflicts of interest.

The Acute Leukemia Forum is organized by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – The evolving treatment of acute myeloid leukemia (AML) was highlighted at the Acute Leukemia Forum of Hemedicus.

In a video interview, Martin Tallman, MD, of Memorial Sloan Kettering Cancer Center in New York, discussed several meeting presentations on the treatment of AML.

In his presentation, Craig Jordan, PhD, of the University of Colorado at Denver, Aurora, explained how the combination of venetoclax and azacitidine appears to target leukemic stem cells in AML.

Courtney DiNardo, MD, of the University of Texas MD Anderson Cancer Center, Houston, presented information on novel agents for AML, including antibody-drug conjugates; bispecific therapies; checkpoint inhibitors; and inhibitors of IDH1/2, MCL1, and MDM2.

Richard Larson, MD, of the University of Chicago, explored the possibility of an individualized approach to postremission therapy in AML.

Frederick Appelbaum, MD, of Fred Hutchinson Cancer Research Center in Seattle, showed that various maintenance therapies given after allogeneic hematopoietic stem cell transplant (HSCT) have not proven beneficial for AML patients.

Richard Jones, MD, of Johns Hopkins Medicine in Baltimore, presented data showing that post-HSCT cyclophosphamide has made haploidentical transplants safer and more effective for AML patients.

And James Ferrara, MD, of the Icahn School of Medicine at Mount Sinai, New York, detailed research showing that biomarkers of graft-versus-host disease can predict nonrelapse mortality after HSCT.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – The evolving treatment of acute myeloid leukemia (AML) was highlighted at the Acute Leukemia Forum of Hemedicus.

In a video interview, Martin Tallman, MD, of Memorial Sloan Kettering Cancer Center in New York, discussed several meeting presentations on the treatment of AML.

In his presentation, Craig Jordan, PhD, of the University of Colorado at Denver, Aurora, explained how the combination of venetoclax and azacitidine appears to target leukemic stem cells in AML.

Courtney DiNardo, MD, of the University of Texas MD Anderson Cancer Center, Houston, presented information on novel agents for AML, including antibody-drug conjugates; bispecific therapies; checkpoint inhibitors; and inhibitors of IDH1/2, MCL1, and MDM2.

Richard Larson, MD, of the University of Chicago, explored the possibility of an individualized approach to postremission therapy in AML.

Frederick Appelbaum, MD, of Fred Hutchinson Cancer Research Center in Seattle, showed that various maintenance therapies given after allogeneic hematopoietic stem cell transplant (HSCT) have not proven beneficial for AML patients.

Richard Jones, MD, of Johns Hopkins Medicine in Baltimore, presented data showing that post-HSCT cyclophosphamide has made haploidentical transplants safer and more effective for AML patients.

And James Ferrara, MD, of the Icahn School of Medicine at Mount Sinai, New York, detailed research showing that biomarkers of graft-versus-host disease can predict nonrelapse mortality after HSCT.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]

NEWPORT BEACH, CALIF. – The evolving treatment of acute myeloid leukemia (AML) was highlighted at the Acute Leukemia Forum of Hemedicus.

In a video interview, Martin Tallman, MD, of Memorial Sloan Kettering Cancer Center in New York, discussed several meeting presentations on the treatment of AML.

In his presentation, Craig Jordan, PhD, of the University of Colorado at Denver, Aurora, explained how the combination of venetoclax and azacitidine appears to target leukemic stem cells in AML.

Courtney DiNardo, MD, of the University of Texas MD Anderson Cancer Center, Houston, presented information on novel agents for AML, including antibody-drug conjugates; bispecific therapies; checkpoint inhibitors; and inhibitors of IDH1/2, MCL1, and MDM2.

Richard Larson, MD, of the University of Chicago, explored the possibility of an individualized approach to postremission therapy in AML.

Frederick Appelbaum, MD, of Fred Hutchinson Cancer Research Center in Seattle, showed that various maintenance therapies given after allogeneic hematopoietic stem cell transplant (HSCT) have not proven beneficial for AML patients.

Richard Jones, MD, of Johns Hopkins Medicine in Baltimore, presented data showing that post-HSCT cyclophosphamide has made haploidentical transplants safer and more effective for AML patients.

And James Ferrara, MD, of the Icahn School of Medicine at Mount Sinai, New York, detailed research showing that biomarkers of graft-versus-host disease can predict nonrelapse mortality after HSCT.

The Acute Leukemia Forum is held by Hemedicus, which is owned by the same company as this news organization.

[email protected]