A breakthrough in medical testing now allows for colorectal cancer screening with just a simple blood test, promising a more accessible and less invasive way to catch the disease early. 

The FDA on July 29 approved the test, called Shield, which can accurately detect tumors in the colon or rectum about 87% of the time when the cancer is in treatable early stages. The approval was announced July 29 by the test’s maker, Guardant Health, and comes just months after promising clinical trial results were published in The New England Journal of Medicine.

Colorectal cancer is among the most common types of cancer diagnosed in the United States each year, along with being one of the leading causes of cancer deaths. The condition is treatable in early stages, but about 1 in 3 people don’t stay up to date on regular screenings, which should begin at age 45.

The simplicity of a blood test could make it more likely for people to be screened for and, ultimately, survive the disease. Other primary screening options include feces-based tests or colonoscopy. The 5-year survival rate for colorectal cancer is 64%.

While highly accurate at detecting DNA shed by tumors during treatable stages of colorectal cancer, the Shield test was not as effective at detecting precancerous areas of tissue, which are typically removed after being detected.

In its news release, Guardant Health officials said they anticipate the test to be covered under Medicare. The out-of-pocket cost for people whose insurance does not cover the test has not yet been announced. The test is expected to be available by next week, The New York Times reported.

If someone’s Shield test comes back positive, the person would then get more tests to confirm the result. Shield was shown in trials to have a 10% false positive rate.

“I was in for a routine physical, and my doctor asked when I had my last colonoscopy,” said John Gormly, a 77-year-old business executive in Newport Beach, California, according to a Guardant Health news release. “I said it’s been a long time, so he offered to give me the Shield blood test. A few days later, the result came back positive, so he referred me for a colonoscopy. It turned out I had stage II colon cancer. The tumor was removed, and I recovered very quickly. Thank God I had taken that blood test.”
 

A version of this article appeared on WebMD.com.

A breakthrough in medical testing now allows for colorectal cancer screening with just a simple blood test, promising a more accessible and less invasive way to catch the disease early. 

The FDA on July 29 approved the test, called Shield, which can accurately detect tumors in the colon or rectum about 87% of the time when the cancer is in treatable early stages. The approval was announced July 29 by the test’s maker, Guardant Health, and comes just months after promising clinical trial results were published in The New England Journal of Medicine.

Colorectal cancer is among the most common types of cancer diagnosed in the United States each year, along with being one of the leading causes of cancer deaths. The condition is treatable in early stages, but about 1 in 3 people don’t stay up to date on regular screenings, which should begin at age 45.

The simplicity of a blood test could make it more likely for people to be screened for and, ultimately, survive the disease. Other primary screening options include feces-based tests or colonoscopy. The 5-year survival rate for colorectal cancer is 64%.

While highly accurate at detecting DNA shed by tumors during treatable stages of colorectal cancer, the Shield test was not as effective at detecting precancerous areas of tissue, which are typically removed after being detected.

In its news release, Guardant Health officials said they anticipate the test to be covered under Medicare. The out-of-pocket cost for people whose insurance does not cover the test has not yet been announced. The test is expected to be available by next week, The New York Times reported.

If someone’s Shield test comes back positive, the person would then get more tests to confirm the result. Shield was shown in trials to have a 10% false positive rate.

“I was in for a routine physical, and my doctor asked when I had my last colonoscopy,” said John Gormly, a 77-year-old business executive in Newport Beach, California, according to a Guardant Health news release. “I said it’s been a long time, so he offered to give me the Shield blood test. A few days later, the result came back positive, so he referred me for a colonoscopy. It turned out I had stage II colon cancer. The tumor was removed, and I recovered very quickly. Thank God I had taken that blood test.”
 

A version of this article appeared on WebMD.com.

A breakthrough in medical testing now allows for colorectal cancer screening with just a simple blood test, promising a more accessible and less invasive way to catch the disease early. 

The FDA on July 29 approved the test, called Shield, which can accurately detect tumors in the colon or rectum about 87% of the time when the cancer is in treatable early stages. The approval was announced July 29 by the test’s maker, Guardant Health, and comes just months after promising clinical trial results were published in The New England Journal of Medicine.

Colorectal cancer is among the most common types of cancer diagnosed in the United States each year, along with being one of the leading causes of cancer deaths. The condition is treatable in early stages, but about 1 in 3 people don’t stay up to date on regular screenings, which should begin at age 45.

The simplicity of a blood test could make it more likely for people to be screened for and, ultimately, survive the disease. Other primary screening options include feces-based tests or colonoscopy. The 5-year survival rate for colorectal cancer is 64%.

While highly accurate at detecting DNA shed by tumors during treatable stages of colorectal cancer, the Shield test was not as effective at detecting precancerous areas of tissue, which are typically removed after being detected.

In its news release, Guardant Health officials said they anticipate the test to be covered under Medicare. The out-of-pocket cost for people whose insurance does not cover the test has not yet been announced. The test is expected to be available by next week, The New York Times reported.

If someone’s Shield test comes back positive, the person would then get more tests to confirm the result. Shield was shown in trials to have a 10% false positive rate.

“I was in for a routine physical, and my doctor asked when I had my last colonoscopy,” said John Gormly, a 77-year-old business executive in Newport Beach, California, according to a Guardant Health news release. “I said it’s been a long time, so he offered to give me the Shield blood test. A few days later, the result came back positive, so he referred me for a colonoscopy. It turned out I had stage II colon cancer. The tumor was removed, and I recovered very quickly. Thank God I had taken that blood test.”
 

A version of this article appeared on WebMD.com.

As disease-modifying treatments for Alzheimer’s disease (AD) become available, equipping primary care physicians with a highly accurate blood test could significantly reduce diagnostic wait times. Currently, the patient diagnostic journey is often prolonged owing to the limited number of AD specialists, causing concern among healthcare providers and patients alike. Now, a new study suggests that use of high-performing blood tests in primary care could identify potential patients with AD much earlier, possibly reducing wait times for specialist care and receipt of treatment.

“We need to triage in primary care and send preferentially the ones that actually could be eligible for treatment, and not those who are just worried because their grandmother reported that she has Alzheimer’s,” lead researcher Soeren Mattke, MD, DSc, told this news organization.

“By combining a brief cognitive test with an accurate blood test of Alzheimer’s pathology in primary care, we can reduce unnecessary referrals, and shorten appointment wait times,” said Dr. Mattke, director of the Brain Health Observatory at the University of Southern California in Los Angeles.

The findings were presented at the Alzheimer’s Association International Conference (AAIC) 2024.
 

Projected Wait Times 100 Months by 2033

The investigators used a Markov model to estimate wait times for patients eligible for AD treatment, taking into account constrained capacity for specialist visits.

The model included the projected US population of people aged 55 years or older from 2023 to 2032. It assumed that individuals would undergo a brief cognitive assessment in primary care and, if suggestive of early-stage cognitive impairment, be referred to a AD specialist under three scenarios: no blood test, blood test to rule out AD pathology, and blood test to confirm AD pathology.

According to the model, without an accurate blood test for AD pathology, projected wait times to see a specialist are about 12 months in 2024 and will increase to more than 100 months in 2033, largely owing to a lack of specialist appointments.

In contrast, with the availability of an accurate blood test to rule out AD, average wait times would be just 3 months in 2024 and increase to only about 13 months in 2033, because far fewer patients would need to see a specialist.

Availability of a blood test to rule in AD pathology in primary care would have a limited effect on wait times because 50% of patients would still undergo confirmatory testing based on expert assumptions, the model suggests.
 

Prioritizing Resources 

“Millions of people have mild memory complaints, and if they all start coming to neurologists, it could completely flood the system and create long wait times for everybody,” Dr. Mattke told this news organization.

The problem, he said, is that brief cognitive tests performed in primary care are not particularly specific for mild cognitive impairment.

“They work pretty well for manifest advanced dementia but for mild cognitive impairment, which is a very subtle, symptomatic disease, they are only about 75% accurate. One quarter are false-positives. That’s a lot of people,” Dr. Mattke said.

He also noted that although earlier blood tests were about 75% accurate, they are now about 90% accurate, “so we are getting to a level where we can pretty much say with confidence that this is likely Alzheimer’s,” Dr. Mattke said.

Commenting on this research for this news organization, Heather Snyder, PhD, vice president of medical and scientific relations at the Alzheimer’s Association, said it is clear that blood tests, “once confirmed, could have a significant impact on the wait times” for dementia assessment. 

“After an initial blood test, we might be able to rule out or rule in individuals who should go to a specialist for further follow-up and testing. This allows us to really ensure that we’re prioritizing resources accordingly,” said Dr. Snyder, who was not involved in the study. 

This project was supported by a research contract from C2N Diagnostics LLC to USC. Dr. Mattke serves on the board of directors of Senscio Systems Inc. and the scientific advisory board of ALZPath and Boston Millennia Partners and has received consulting fees from Biogen, C2N, Eisai, Eli Lilly, Novartis, and Roche/Genentech. Dr. Snyder has no relevant disclosures.

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

As disease-modifying treatments for Alzheimer’s disease (AD) become available, equipping primary care physicians with a highly accurate blood test could significantly reduce diagnostic wait times. Currently, the patient diagnostic journey is often prolonged owing to the limited number of AD specialists, causing concern among healthcare providers and patients alike. Now, a new study suggests that use of high-performing blood tests in primary care could identify potential patients with AD much earlier, possibly reducing wait times for specialist care and receipt of treatment.

“We need to triage in primary care and send preferentially the ones that actually could be eligible for treatment, and not those who are just worried because their grandmother reported that she has Alzheimer’s,” lead researcher Soeren Mattke, MD, DSc, told this news organization.

“By combining a brief cognitive test with an accurate blood test of Alzheimer’s pathology in primary care, we can reduce unnecessary referrals, and shorten appointment wait times,” said Dr. Mattke, director of the Brain Health Observatory at the University of Southern California in Los Angeles.

The findings were presented at the Alzheimer’s Association International Conference (AAIC) 2024.
 

Projected Wait Times 100 Months by 2033

The investigators used a Markov model to estimate wait times for patients eligible for AD treatment, taking into account constrained capacity for specialist visits.

The model included the projected US population of people aged 55 years or older from 2023 to 2032. It assumed that individuals would undergo a brief cognitive assessment in primary care and, if suggestive of early-stage cognitive impairment, be referred to a AD specialist under three scenarios: no blood test, blood test to rule out AD pathology, and blood test to confirm AD pathology.

According to the model, without an accurate blood test for AD pathology, projected wait times to see a specialist are about 12 months in 2024 and will increase to more than 100 months in 2033, largely owing to a lack of specialist appointments.

In contrast, with the availability of an accurate blood test to rule out AD, average wait times would be just 3 months in 2024 and increase to only about 13 months in 2033, because far fewer patients would need to see a specialist.

Availability of a blood test to rule in AD pathology in primary care would have a limited effect on wait times because 50% of patients would still undergo confirmatory testing based on expert assumptions, the model suggests.
 

Prioritizing Resources 

“Millions of people have mild memory complaints, and if they all start coming to neurologists, it could completely flood the system and create long wait times for everybody,” Dr. Mattke told this news organization.

The problem, he said, is that brief cognitive tests performed in primary care are not particularly specific for mild cognitive impairment.

“They work pretty well for manifest advanced dementia but for mild cognitive impairment, which is a very subtle, symptomatic disease, they are only about 75% accurate. One quarter are false-positives. That’s a lot of people,” Dr. Mattke said.

He also noted that although earlier blood tests were about 75% accurate, they are now about 90% accurate, “so we are getting to a level where we can pretty much say with confidence that this is likely Alzheimer’s,” Dr. Mattke said.

Commenting on this research for this news organization, Heather Snyder, PhD, vice president of medical and scientific relations at the Alzheimer’s Association, said it is clear that blood tests, “once confirmed, could have a significant impact on the wait times” for dementia assessment. 

“After an initial blood test, we might be able to rule out or rule in individuals who should go to a specialist for further follow-up and testing. This allows us to really ensure that we’re prioritizing resources accordingly,” said Dr. Snyder, who was not involved in the study. 

This project was supported by a research contract from C2N Diagnostics LLC to USC. Dr. Mattke serves on the board of directors of Senscio Systems Inc. and the scientific advisory board of ALZPath and Boston Millennia Partners and has received consulting fees from Biogen, C2N, Eisai, Eli Lilly, Novartis, and Roche/Genentech. Dr. Snyder has no relevant disclosures.

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

As disease-modifying treatments for Alzheimer’s disease (AD) become available, equipping primary care physicians with a highly accurate blood test could significantly reduce diagnostic wait times. Currently, the patient diagnostic journey is often prolonged owing to the limited number of AD specialists, causing concern among healthcare providers and patients alike. Now, a new study suggests that use of high-performing blood tests in primary care could identify potential patients with AD much earlier, possibly reducing wait times for specialist care and receipt of treatment.

“We need to triage in primary care and send preferentially the ones that actually could be eligible for treatment, and not those who are just worried because their grandmother reported that she has Alzheimer’s,” lead researcher Soeren Mattke, MD, DSc, told this news organization.

“By combining a brief cognitive test with an accurate blood test of Alzheimer’s pathology in primary care, we can reduce unnecessary referrals, and shorten appointment wait times,” said Dr. Mattke, director of the Brain Health Observatory at the University of Southern California in Los Angeles.

The findings were presented at the Alzheimer’s Association International Conference (AAIC) 2024.
 

Projected Wait Times 100 Months by 2033

The investigators used a Markov model to estimate wait times for patients eligible for AD treatment, taking into account constrained capacity for specialist visits.

The model included the projected US population of people aged 55 years or older from 2023 to 2032. It assumed that individuals would undergo a brief cognitive assessment in primary care and, if suggestive of early-stage cognitive impairment, be referred to a AD specialist under three scenarios: no blood test, blood test to rule out AD pathology, and blood test to confirm AD pathology.

According to the model, without an accurate blood test for AD pathology, projected wait times to see a specialist are about 12 months in 2024 and will increase to more than 100 months in 2033, largely owing to a lack of specialist appointments.

In contrast, with the availability of an accurate blood test to rule out AD, average wait times would be just 3 months in 2024 and increase to only about 13 months in 2033, because far fewer patients would need to see a specialist.

Availability of a blood test to rule in AD pathology in primary care would have a limited effect on wait times because 50% of patients would still undergo confirmatory testing based on expert assumptions, the model suggests.
 

Prioritizing Resources 

“Millions of people have mild memory complaints, and if they all start coming to neurologists, it could completely flood the system and create long wait times for everybody,” Dr. Mattke told this news organization.

The problem, he said, is that brief cognitive tests performed in primary care are not particularly specific for mild cognitive impairment.

“They work pretty well for manifest advanced dementia but for mild cognitive impairment, which is a very subtle, symptomatic disease, they are only about 75% accurate. One quarter are false-positives. That’s a lot of people,” Dr. Mattke said.

He also noted that although earlier blood tests were about 75% accurate, they are now about 90% accurate, “so we are getting to a level where we can pretty much say with confidence that this is likely Alzheimer’s,” Dr. Mattke said.

Commenting on this research for this news organization, Heather Snyder, PhD, vice president of medical and scientific relations at the Alzheimer’s Association, said it is clear that blood tests, “once confirmed, could have a significant impact on the wait times” for dementia assessment. 

“After an initial blood test, we might be able to rule out or rule in individuals who should go to a specialist for further follow-up and testing. This allows us to really ensure that we’re prioritizing resources accordingly,” said Dr. Snyder, who was not involved in the study. 

This project was supported by a research contract from C2N Diagnostics LLC to USC. Dr. Mattke serves on the board of directors of Senscio Systems Inc. and the scientific advisory board of ALZPath and Boston Millennia Partners and has received consulting fees from Biogen, C2N, Eisai, Eli Lilly, Novartis, and Roche/Genentech. Dr. Snyder has no relevant disclosures.

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

This transcript has been edited for clarity.

Tardive dyskinesia is a chronic, potentially irreversible, hyperkinetic movement disorder. And the challenge with tardive dyskinesia is that it’s underdiagnosed and undertreated. With the expanded use of dopamine receptor–blocking agents, there are about 7.5 million Americans who are now exposed and at risk for tardive dyskinesia.

It’s thought that about 500,000-750,000 of these patients may in fact have tardive dyskinesia, but only 15% are treated. So why are people not being treated for tardive dyskinesia? Well, there are a number of possible answers.

Until a few years ago, there were no Food and Drug Administration (FDA)–approved treatments for tardive dyskinesia, and these antipsychotic medications that the patients were taking, in many cases, were potentially lifesaving drugs, so they couldn’t simply be stopped. As a result of that, I think physicians developed a certain psychic blindness to identifying tardive dyskinesia, because it was their drugs that were causing the disease and yet they couldn’t be stopped. So, there really wasn’t much they could do in terms of making the diagnosis.

In addition, they were trained that tardive dyskinesia doesn’t have much impact on patients. But we now know, through surveys and other studies, that tardive dyskinesia can have a tremendous impact on patients and on your ability to treat the patient’s underlying mental health issues. It’s estimated that 50% of patients with tardive dyskinesia actually reduce the amount of antipsychotic medication they’re taking on their own, and about 40% may in fact stop their antipsychotic medication altogether.

Thirty-five percent of patients stopped seeing their doctor after they developed tardive dyskinesia, and about 20% of patients actually told other patients not to take their antipsychotic medication. So, tardive dyskinesia is impacting your ability to treat patients. In addition, it impacts the patients themselves. Nearly three out of four patients with tardive dyskinesia said, in surveys, that it caused severe impact on their psychosocial functioning.

It also impacted caregivers, with 70% of caregivers saying that the patients with tardive dyskinesia made them more anxious and limited them socially. So, we have this tremendous impact from tardive dyskinesia.

In addition, physicians sometimes don’t identify tardive dyskinesia correctly. They mistake it for another movement disorder: drug-induced parkinsonism. Or it falls under the rubric of extrapyramidal symptoms (EPS), and they were trained that you treat EPS with benztropine. The challenge with that is that benztropine is only indicated for acute dystonia or for drug-induced parkinsonism. It actually makes tardive dyskinesia worse. And, in the product insert for benztropine, it’s recommended that it should not be used in tardive dyskinesia. So if you have a patient whom you suspect has tardive dyskinesia, you have to discontinue the benztropine. That’s a really important first step.

And then, what else should you do? There are now two FDA-approved treatments for tardive dyskinesia. These are valbenazine and deutetrabenazine. Both of these drugs have been demonstrated in large double-blind, placebo-controlled studies to reduce tardive dyskinesia, as measured by the Abnormal Involuntary Movement Scale, by about 30%. These drugs have been demonstrated to be safe and well tolerated, with the main side effect being somnolence.

Some people can also develop parkinsonism. Why could there be Parkinsonism? This is because vesicular monoamine transporter 2 (VMAT2) inhibitors work by reducing the amount of dopamine that can be packaged in the presynaptic neuron. That means that less dopamine is available to the synapse, and this reduces movement. The American Psychiatric Association has issued guidelines for the treatment of tardive dyskinesia and has said that moderate to severe tardive dyskinesia should be treated first-line with VMAT2 inhibitors and that mild tardive dyskinesia should also be treated with VMAT2 inhibitors if the tardive dyskinesia is impacting the patient.

Given the impact that tardive dyskinesia has on patients and caregivers, and the physician’s ability to treat these patients’ mental health issues, we need to become aggressive and treat the tardive dyskinesia so that patients can improve and be able to have their movements treated without impacting their underlying mental health issues.

Daniel Kremens, professor, Department of Neurology, Sidney Kimmel Medical College, Thomas Jefferson University, codirector, Parkinson’s Disease and Movement Disorders Division, Jack and Vickie Farber Center for Neuroscience, Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, has disclosed relevant financial relationships with Teva Pharmaceuticals, AbbVie, Merz, Allergan, Bial, Cerevel, Amneal, Acadia, Supernus, Adamas, Acorda, Kyowa Kirin, and Neurocrine.

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

This transcript has been edited for clarity.

Tardive dyskinesia is a chronic, potentially irreversible, hyperkinetic movement disorder. And the challenge with tardive dyskinesia is that it’s underdiagnosed and undertreated. With the expanded use of dopamine receptor–blocking agents, there are about 7.5 million Americans who are now exposed and at risk for tardive dyskinesia.

It’s thought that about 500,000-750,000 of these patients may in fact have tardive dyskinesia, but only 15% are treated. So why are people not being treated for tardive dyskinesia? Well, there are a number of possible answers.

Until a few years ago, there were no Food and Drug Administration (FDA)–approved treatments for tardive dyskinesia, and these antipsychotic medications that the patients were taking, in many cases, were potentially lifesaving drugs, so they couldn’t simply be stopped. As a result of that, I think physicians developed a certain psychic blindness to identifying tardive dyskinesia, because it was their drugs that were causing the disease and yet they couldn’t be stopped. So, there really wasn’t much they could do in terms of making the diagnosis.

In addition, they were trained that tardive dyskinesia doesn’t have much impact on patients. But we now know, through surveys and other studies, that tardive dyskinesia can have a tremendous impact on patients and on your ability to treat the patient’s underlying mental health issues. It’s estimated that 50% of patients with tardive dyskinesia actually reduce the amount of antipsychotic medication they’re taking on their own, and about 40% may in fact stop their antipsychotic medication altogether.

Thirty-five percent of patients stopped seeing their doctor after they developed tardive dyskinesia, and about 20% of patients actually told other patients not to take their antipsychotic medication. So, tardive dyskinesia is impacting your ability to treat patients. In addition, it impacts the patients themselves. Nearly three out of four patients with tardive dyskinesia said, in surveys, that it caused severe impact on their psychosocial functioning.

It also impacted caregivers, with 70% of caregivers saying that the patients with tardive dyskinesia made them more anxious and limited them socially. So, we have this tremendous impact from tardive dyskinesia.

In addition, physicians sometimes don’t identify tardive dyskinesia correctly. They mistake it for another movement disorder: drug-induced parkinsonism. Or it falls under the rubric of extrapyramidal symptoms (EPS), and they were trained that you treat EPS with benztropine. The challenge with that is that benztropine is only indicated for acute dystonia or for drug-induced parkinsonism. It actually makes tardive dyskinesia worse. And, in the product insert for benztropine, it’s recommended that it should not be used in tardive dyskinesia. So if you have a patient whom you suspect has tardive dyskinesia, you have to discontinue the benztropine. That’s a really important first step.

And then, what else should you do? There are now two FDA-approved treatments for tardive dyskinesia. These are valbenazine and deutetrabenazine. Both of these drugs have been demonstrated in large double-blind, placebo-controlled studies to reduce tardive dyskinesia, as measured by the Abnormal Involuntary Movement Scale, by about 30%. These drugs have been demonstrated to be safe and well tolerated, with the main side effect being somnolence.

Some people can also develop parkinsonism. Why could there be Parkinsonism? This is because vesicular monoamine transporter 2 (VMAT2) inhibitors work by reducing the amount of dopamine that can be packaged in the presynaptic neuron. That means that less dopamine is available to the synapse, and this reduces movement. The American Psychiatric Association has issued guidelines for the treatment of tardive dyskinesia and has said that moderate to severe tardive dyskinesia should be treated first-line with VMAT2 inhibitors and that mild tardive dyskinesia should also be treated with VMAT2 inhibitors if the tardive dyskinesia is impacting the patient.

Given the impact that tardive dyskinesia has on patients and caregivers, and the physician’s ability to treat these patients’ mental health issues, we need to become aggressive and treat the tardive dyskinesia so that patients can improve and be able to have their movements treated without impacting their underlying mental health issues.

Daniel Kremens, professor, Department of Neurology, Sidney Kimmel Medical College, Thomas Jefferson University, codirector, Parkinson’s Disease and Movement Disorders Division, Jack and Vickie Farber Center for Neuroscience, Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, has disclosed relevant financial relationships with Teva Pharmaceuticals, AbbVie, Merz, Allergan, Bial, Cerevel, Amneal, Acadia, Supernus, Adamas, Acorda, Kyowa Kirin, and Neurocrine.

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

This transcript has been edited for clarity.

Tardive dyskinesia is a chronic, potentially irreversible, hyperkinetic movement disorder. And the challenge with tardive dyskinesia is that it’s underdiagnosed and undertreated. With the expanded use of dopamine receptor–blocking agents, there are about 7.5 million Americans who are now exposed and at risk for tardive dyskinesia.

It’s thought that about 500,000-750,000 of these patients may in fact have tardive dyskinesia, but only 15% are treated. So why are people not being treated for tardive dyskinesia? Well, there are a number of possible answers.

Until a few years ago, there were no Food and Drug Administration (FDA)–approved treatments for tardive dyskinesia, and these antipsychotic medications that the patients were taking, in many cases, were potentially lifesaving drugs, so they couldn’t simply be stopped. As a result of that, I think physicians developed a certain psychic blindness to identifying tardive dyskinesia, because it was their drugs that were causing the disease and yet they couldn’t be stopped. So, there really wasn’t much they could do in terms of making the diagnosis.

In addition, they were trained that tardive dyskinesia doesn’t have much impact on patients. But we now know, through surveys and other studies, that tardive dyskinesia can have a tremendous impact on patients and on your ability to treat the patient’s underlying mental health issues. It’s estimated that 50% of patients with tardive dyskinesia actually reduce the amount of antipsychotic medication they’re taking on their own, and about 40% may in fact stop their antipsychotic medication altogether.

Thirty-five percent of patients stopped seeing their doctor after they developed tardive dyskinesia, and about 20% of patients actually told other patients not to take their antipsychotic medication. So, tardive dyskinesia is impacting your ability to treat patients. In addition, it impacts the patients themselves. Nearly three out of four patients with tardive dyskinesia said, in surveys, that it caused severe impact on their psychosocial functioning.

It also impacted caregivers, with 70% of caregivers saying that the patients with tardive dyskinesia made them more anxious and limited them socially. So, we have this tremendous impact from tardive dyskinesia.

In addition, physicians sometimes don’t identify tardive dyskinesia correctly. They mistake it for another movement disorder: drug-induced parkinsonism. Or it falls under the rubric of extrapyramidal symptoms (EPS), and they were trained that you treat EPS with benztropine. The challenge with that is that benztropine is only indicated for acute dystonia or for drug-induced parkinsonism. It actually makes tardive dyskinesia worse. And, in the product insert for benztropine, it’s recommended that it should not be used in tardive dyskinesia. So if you have a patient whom you suspect has tardive dyskinesia, you have to discontinue the benztropine. That’s a really important first step.

And then, what else should you do? There are now two FDA-approved treatments for tardive dyskinesia. These are valbenazine and deutetrabenazine. Both of these drugs have been demonstrated in large double-blind, placebo-controlled studies to reduce tardive dyskinesia, as measured by the Abnormal Involuntary Movement Scale, by about 30%. These drugs have been demonstrated to be safe and well tolerated, with the main side effect being somnolence.

Some people can also develop parkinsonism. Why could there be Parkinsonism? This is because vesicular monoamine transporter 2 (VMAT2) inhibitors work by reducing the amount of dopamine that can be packaged in the presynaptic neuron. That means that less dopamine is available to the synapse, and this reduces movement. The American Psychiatric Association has issued guidelines for the treatment of tardive dyskinesia and has said that moderate to severe tardive dyskinesia should be treated first-line with VMAT2 inhibitors and that mild tardive dyskinesia should also be treated with VMAT2 inhibitors if the tardive dyskinesia is impacting the patient.

Given the impact that tardive dyskinesia has on patients and caregivers, and the physician’s ability to treat these patients’ mental health issues, we need to become aggressive and treat the tardive dyskinesia so that patients can improve and be able to have their movements treated without impacting their underlying mental health issues.

Daniel Kremens, professor, Department of Neurology, Sidney Kimmel Medical College, Thomas Jefferson University, codirector, Parkinson’s Disease and Movement Disorders Division, Jack and Vickie Farber Center for Neuroscience, Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, has disclosed relevant financial relationships with Teva Pharmaceuticals, AbbVie, Merz, Allergan, Bial, Cerevel, Amneal, Acadia, Supernus, Adamas, Acorda, Kyowa Kirin, and Neurocrine.

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

Small Talk, Big Topics, AGA’s podcast for trainees and early career GIs, is back for season three!

In a special episode live from Digestive Disease Week® (DDW) 2024, host Dr. Matthew Whitson talks with returning guest Dr. Janice Jou. Dr. Jou is a transplant hematologist at the Portland VA and currently serves as professor of medicine and fellowship program director at Oregon Health & Science University. Don’t miss her insight as she shares advice all about what she wishes she knew when going into her first job in gastroenterology. Dr. Jou also answers questions from the audience on topics including “when to say no” and the importance of encouraging emotional transparency with fellows and faculty.

Catch up with past episodes and subscribe wherever you listen to podcasts. You can also listen by clicking on the episode name below.

• Episode 5: Janice Jou: Live from #DDW2024 with tips for your first job
• Episode 4: Loren Rabinowitz and Rachel Issaka: Building research collaborations
• Episode 3: Andy Tau: How to treat GI emergencies
• Episode 2: Laurel Fisher and Asma Khapra: Advancing and advocating for women in GI
• Episode 1: Barbara Jung: Unpacking mentorship with AGA’s president

Small Talk, Big Topics, AGA’s podcast for trainees and early career GIs, is back for season three!

In a special episode live from Digestive Disease Week® (DDW) 2024, host Dr. Matthew Whitson talks with returning guest Dr. Janice Jou. Dr. Jou is a transplant hematologist at the Portland VA and currently serves as professor of medicine and fellowship program director at Oregon Health & Science University. Don’t miss her insight as she shares advice all about what she wishes she knew when going into her first job in gastroenterology. Dr. Jou also answers questions from the audience on topics including “when to say no” and the importance of encouraging emotional transparency with fellows and faculty.

Catch up with past episodes and subscribe wherever you listen to podcasts. You can also listen by clicking on the episode name below.

• Episode 5: Janice Jou: Live from #DDW2024 with tips for your first job
• Episode 4: Loren Rabinowitz and Rachel Issaka: Building research collaborations
• Episode 3: Andy Tau: How to treat GI emergencies
• Episode 2: Laurel Fisher and Asma Khapra: Advancing and advocating for women in GI
• Episode 1: Barbara Jung: Unpacking mentorship with AGA’s president

Small Talk, Big Topics, AGA’s podcast for trainees and early career GIs, is back for season three!

In a special episode live from Digestive Disease Week® (DDW) 2024, host Dr. Matthew Whitson talks with returning guest Dr. Janice Jou. Dr. Jou is a transplant hematologist at the Portland VA and currently serves as professor of medicine and fellowship program director at Oregon Health & Science University. Don’t miss her insight as she shares advice all about what she wishes she knew when going into her first job in gastroenterology. Dr. Jou also answers questions from the audience on topics including “when to say no” and the importance of encouraging emotional transparency with fellows and faculty.

Catch up with past episodes and subscribe wherever you listen to podcasts. You can also listen by clicking on the episode name below.

• Episode 5: Janice Jou: Live from #DDW2024 with tips for your first job
• Episode 4: Loren Rabinowitz and Rachel Issaka: Building research collaborations
• Episode 3: Andy Tau: How to treat GI emergencies
• Episode 2: Laurel Fisher and Asma Khapra: Advancing and advocating for women in GI
• Episode 1: Barbara Jung: Unpacking mentorship with AGA’s president

The Diagnosis: Prurigo Pigmentosa

A comprehensive metabolic panel collected from our patient 1 month earlier did not reveal any abnormalities. Serum methylmalonic acid and homocysteine were both elevated at 417 nmol/L (reference range [for those aged 2–59 years], 55–335 nmol/L) and 23 μmol/L (reference range, 5–15 μmol/L), respectively. Serum folate and 25-hydroxyvitamin D were low at 3.1 ng/mL (reference range, >4.8 ng/mL) and 5 ng/mL (reference range, 30–80 ng/mL), respectively. Vitamin B₁₂ was within reference range. Two 4-mm punch biopsies collected from the upper back showed spongiotic dermatitis.

Our patient’s histopathology results along with the rash distribution and medical history of anorexia increased suspicion for prurigo pigmentosa. A trial of oral doxycycline 100 mg twice daily for 2 weeks was prescribed. At 2-week follow-up, the patient’s mother revealed a history of ketosis in her daughter, solidifying the diagnosis. The patient was counseled on maintaining a healthy diet to prevent future breakouts. The patient’s rash resolved with diet modification and doxycycline; however, it recurred upon relapse of anorexia 4 months later.

Prurigo pigmentosa, originally identified in Japan by Nagashima et al,¹ is an uncommon recurrent inflammatory disorder predominantly observed in young adults of Asian descent. Subsequently, it was reported to occur among individuals from different ethnic backgrounds, indicating potential underdiagnosis or misdiagnosis in Western countries.² Although a direct pathogenic cause for prurigo pigmentosa has not been identified, a strong association has been linked to diet, specifically when ketosis is induced, such as in ketogenic diets and anorexia nervosa.^3-5 Other possible causes include sunlight exposure, clothing friction, and sweating.^1,5 The disease course is characterized by intermittent flares and spontaneous resolution, with recurrence in most cases. During the active phase, intensely pruritic, papulovesicular or urticarial papules are predominant and most often are localized to the upper body and torso, including the back, shoulders, neck, and chest.⁵ These flares can persist for several days but eventually subside, leaving behind a characteristic reticular pigmentation that can persist for months.⁵ First-line treatment often involves the use of tetracycline antibiotics, such as minocycline or doxycycline. ^2,4,5 Dapsone often is used with successful resolution. ⁶ Dietary modifications also have been found to be effective in treating prurigo pigmentosa, particularly in patients presenting with dietary insufficiency.^6,7 Increased carbohydrate intake has been shown to promote resolution. ⁶ Topical corticosteroids demonstrate limited efficacy in controlling flares.^6,8

Histopathology has been variably described, with initial findings reported as nonspecific.¹ However, it was later described as a distinct inflammatory disease of the skin with histologically distinct stages.^2,9 Early stages reveal scattered dermal, dermal papillary, and perivascular neutrophilic infiltration.⁹ The lesions then progress and become fully developed, at which point neutrophilic infiltration becomes more prominent, accompanied by the presence of intraepidermal neutrophils and spongiosis. As the lesions resolve, the infiltration transitions to lymphocytic, and lichenoid changes can sometimes be appreciated along with epidermal hyperplasia, hyperpigmentation, and dermal melanophages.⁹ Although these findings aid in the diagnosis of prurigo pigmentosa, a clinicopathologic correlation is necessary to establish a definitive diagnosis.

Because prurigo pigmentosa is rare, it often is misdiagnosed as another condition with a similar presentation and nonspecific biopsy findings.⁶ Allergic contact dermatitis is a common type IV delayed hypersensitivity reaction that manifests similar to prurigo pigmentosa with pruritus and a well-demarcated distribution¹⁰ that is related to the pattern of allergen exposure; in the case of allergic contact dermatitis related to textiles, a well-demarcated rash will appear in the distribution area of the associated clothing (eg, shirt, pants, shorts).¹¹ Development of allergy involves exposure and sensitization to an allergen, followed by subsequent re-exposure that results in cutaneous T-cell activation and inflammation. ¹⁰ Histopathology shows nonspecific spongiotic inflammation, and the gold standard for diagnosis is patch testing to identify the causative substance(s). Definitive treatment includes avoidance of identified allergies; however, if patients are unable to avoid the allergen or the cause is unknown, then corticosteroids, antihistamines, and/or calcineurin inhibitors are beneficial in controlling symptoms and flares.¹⁰

Pityrosporum folliculitis (also known as Malassezia folliculitis) is a fungal acneform condition that arises from overgrowth of normal skin flora Malassezia yeast,¹² which may be due to occlusion of follicles or disruption of the normal flora composition. Clinically, the manifestation may resemble prurigo pigmentosa in distribution and presence of intense pruritus. However, pustular lesions and involvement of the face can aid in differentiating Pityrosporum from prurigo pigmentosa, which can be confirmed via periodic acid–Schiff staining with numerous round yeasts within affected follicles. Oral antifungal therapy typically yields rapid improvement and resolution of symptoms.¹²

Urticaria and prurigo pigmentosa share similar clinical characteristics, with symptoms of intense pruritus and urticarial lesions on the trunk.^2,13 Urticaria is an IgEmediated type I hypersensitivity reaction characterized by wheals (ie, edematous red or pink lesions of variable size and shape that typically resolve spontaneously within 24–48 hours).¹³ Notably, urticaria will improve and in some cases completely resolve with antihistamines or anti-IgE antibody treatment, which may aid in distinguishing it from prurigo pigmentosa, as the latter typically exhibits limited response to such treatment.² Histopathology also can assist in the diagnosis by ruling out other causes of similar rash; however, biopsies are not routinely done unless other inflammatory conditions are of high suspicion.¹³

Bullous pemphigoid is an autoimmune, subepidermal, blistering dermatosis that is most common among the elderly.¹⁴ It is characterized by the presence of IgG antibodies that target BP180 and BP230, which initiate inflammatory cascades that lead to tissue damage and blister formation. It typically manifests as pruritic blistering eruptions, primarily on the limbs and trunk, but may involve the head, neck, or palmoplantar regions.¹⁴ Although blistering eruptions are the prodrome of the disease, some cases may present with nonspecific urticarial or eczematous lesions^14,15 that may resemble prurigo pigmentosa. The diagnosis is confirmed through direct immunofluorescence microscopy of biopsied lesions, which reveals IgG and/or C3 deposits along the dermoepidermal junction.14 Management of bullous pemphigoid involves timely initiation of dapsone or systemic corticosteroids, which have demonstrated high efficacy in controlling the disease and its associated symptoms.¹⁵

Our patient achieved a favorable response to diet modification and doxycycline therapy consistent with the diagnosis of prurigo pigmentosa. Unfortunately, the condition recurred following a relapse of anorexia. Management of prurigo pigmentosa necessitates not only accurate diagnosis but also addressing any underlying factors that may contribute to disease exacerbation. We anticipate the eating disorder will pose a major challenge in achieving long-term control of prurigo pigmentosa.

The Diagnosis: Prurigo Pigmentosa

A comprehensive metabolic panel collected from our patient 1 month earlier did not reveal any abnormalities. Serum methylmalonic acid and homocysteine were both elevated at 417 nmol/L (reference range [for those aged 2–59 years], 55–335 nmol/L) and 23 μmol/L (reference range, 5–15 μmol/L), respectively. Serum folate and 25-hydroxyvitamin D were low at 3.1 ng/mL (reference range, >4.8 ng/mL) and 5 ng/mL (reference range, 30–80 ng/mL), respectively. Vitamin B₁₂ was within reference range. Two 4-mm punch biopsies collected from the upper back showed spongiotic dermatitis.

Our patient’s histopathology results along with the rash distribution and medical history of anorexia increased suspicion for prurigo pigmentosa. A trial of oral doxycycline 100 mg twice daily for 2 weeks was prescribed. At 2-week follow-up, the patient’s mother revealed a history of ketosis in her daughter, solidifying the diagnosis. The patient was counseled on maintaining a healthy diet to prevent future breakouts. The patient’s rash resolved with diet modification and doxycycline; however, it recurred upon relapse of anorexia 4 months later.

Prurigo pigmentosa, originally identified in Japan by Nagashima et al,¹ is an uncommon recurrent inflammatory disorder predominantly observed in young adults of Asian descent. Subsequently, it was reported to occur among individuals from different ethnic backgrounds, indicating potential underdiagnosis or misdiagnosis in Western countries.² Although a direct pathogenic cause for prurigo pigmentosa has not been identified, a strong association has been linked to diet, specifically when ketosis is induced, such as in ketogenic diets and anorexia nervosa.^3-5 Other possible causes include sunlight exposure, clothing friction, and sweating.^1,5 The disease course is characterized by intermittent flares and spontaneous resolution, with recurrence in most cases. During the active phase, intensely pruritic, papulovesicular or urticarial papules are predominant and most often are localized to the upper body and torso, including the back, shoulders, neck, and chest.⁵ These flares can persist for several days but eventually subside, leaving behind a characteristic reticular pigmentation that can persist for months.⁵ First-line treatment often involves the use of tetracycline antibiotics, such as minocycline or doxycycline. ^2,4,5 Dapsone often is used with successful resolution. ⁶ Dietary modifications also have been found to be effective in treating prurigo pigmentosa, particularly in patients presenting with dietary insufficiency.^6,7 Increased carbohydrate intake has been shown to promote resolution. ⁶ Topical corticosteroids demonstrate limited efficacy in controlling flares.^6,8

Histopathology has been variably described, with initial findings reported as nonspecific.¹ However, it was later described as a distinct inflammatory disease of the skin with histologically distinct stages.^2,9 Early stages reveal scattered dermal, dermal papillary, and perivascular neutrophilic infiltration.⁹ The lesions then progress and become fully developed, at which point neutrophilic infiltration becomes more prominent, accompanied by the presence of intraepidermal neutrophils and spongiosis. As the lesions resolve, the infiltration transitions to lymphocytic, and lichenoid changes can sometimes be appreciated along with epidermal hyperplasia, hyperpigmentation, and dermal melanophages.⁹ Although these findings aid in the diagnosis of prurigo pigmentosa, a clinicopathologic correlation is necessary to establish a definitive diagnosis.

Because prurigo pigmentosa is rare, it often is misdiagnosed as another condition with a similar presentation and nonspecific biopsy findings.⁶ Allergic contact dermatitis is a common type IV delayed hypersensitivity reaction that manifests similar to prurigo pigmentosa with pruritus and a well-demarcated distribution¹⁰ that is related to the pattern of allergen exposure; in the case of allergic contact dermatitis related to textiles, a well-demarcated rash will appear in the distribution area of the associated clothing (eg, shirt, pants, shorts).¹¹ Development of allergy involves exposure and sensitization to an allergen, followed by subsequent re-exposure that results in cutaneous T-cell activation and inflammation. ¹⁰ Histopathology shows nonspecific spongiotic inflammation, and the gold standard for diagnosis is patch testing to identify the causative substance(s). Definitive treatment includes avoidance of identified allergies; however, if patients are unable to avoid the allergen or the cause is unknown, then corticosteroids, antihistamines, and/or calcineurin inhibitors are beneficial in controlling symptoms and flares.¹⁰

Pityrosporum folliculitis (also known as Malassezia folliculitis) is a fungal acneform condition that arises from overgrowth of normal skin flora Malassezia yeast,¹² which may be due to occlusion of follicles or disruption of the normal flora composition. Clinically, the manifestation may resemble prurigo pigmentosa in distribution and presence of intense pruritus. However, pustular lesions and involvement of the face can aid in differentiating Pityrosporum from prurigo pigmentosa, which can be confirmed via periodic acid–Schiff staining with numerous round yeasts within affected follicles. Oral antifungal therapy typically yields rapid improvement and resolution of symptoms.¹²

Urticaria and prurigo pigmentosa share similar clinical characteristics, with symptoms of intense pruritus and urticarial lesions on the trunk.^2,13 Urticaria is an IgEmediated type I hypersensitivity reaction characterized by wheals (ie, edematous red or pink lesions of variable size and shape that typically resolve spontaneously within 24–48 hours).¹³ Notably, urticaria will improve and in some cases completely resolve with antihistamines or anti-IgE antibody treatment, which may aid in distinguishing it from prurigo pigmentosa, as the latter typically exhibits limited response to such treatment.² Histopathology also can assist in the diagnosis by ruling out other causes of similar rash; however, biopsies are not routinely done unless other inflammatory conditions are of high suspicion.¹³

Bullous pemphigoid is an autoimmune, subepidermal, blistering dermatosis that is most common among the elderly.¹⁴ It is characterized by the presence of IgG antibodies that target BP180 and BP230, which initiate inflammatory cascades that lead to tissue damage and blister formation. It typically manifests as pruritic blistering eruptions, primarily on the limbs and trunk, but may involve the head, neck, or palmoplantar regions.¹⁴ Although blistering eruptions are the prodrome of the disease, some cases may present with nonspecific urticarial or eczematous lesions^14,15 that may resemble prurigo pigmentosa. The diagnosis is confirmed through direct immunofluorescence microscopy of biopsied lesions, which reveals IgG and/or C3 deposits along the dermoepidermal junction.14 Management of bullous pemphigoid involves timely initiation of dapsone or systemic corticosteroids, which have demonstrated high efficacy in controlling the disease and its associated symptoms.¹⁵

Our patient achieved a favorable response to diet modification and doxycycline therapy consistent with the diagnosis of prurigo pigmentosa. Unfortunately, the condition recurred following a relapse of anorexia. Management of prurigo pigmentosa necessitates not only accurate diagnosis but also addressing any underlying factors that may contribute to disease exacerbation. We anticipate the eating disorder will pose a major challenge in achieving long-term control of prurigo pigmentosa.

The Diagnosis: Prurigo Pigmentosa

A comprehensive metabolic panel collected from our patient 1 month earlier did not reveal any abnormalities. Serum methylmalonic acid and homocysteine were both elevated at 417 nmol/L (reference range [for those aged 2–59 years], 55–335 nmol/L) and 23 μmol/L (reference range, 5–15 μmol/L), respectively. Serum folate and 25-hydroxyvitamin D were low at 3.1 ng/mL (reference range, >4.8 ng/mL) and 5 ng/mL (reference range, 30–80 ng/mL), respectively. Vitamin B₁₂ was within reference range. Two 4-mm punch biopsies collected from the upper back showed spongiotic dermatitis.

Our patient’s histopathology results along with the rash distribution and medical history of anorexia increased suspicion for prurigo pigmentosa. A trial of oral doxycycline 100 mg twice daily for 2 weeks was prescribed. At 2-week follow-up, the patient’s mother revealed a history of ketosis in her daughter, solidifying the diagnosis. The patient was counseled on maintaining a healthy diet to prevent future breakouts. The patient’s rash resolved with diet modification and doxycycline; however, it recurred upon relapse of anorexia 4 months later.

Prurigo pigmentosa, originally identified in Japan by Nagashima et al,¹ is an uncommon recurrent inflammatory disorder predominantly observed in young adults of Asian descent. Subsequently, it was reported to occur among individuals from different ethnic backgrounds, indicating potential underdiagnosis or misdiagnosis in Western countries.² Although a direct pathogenic cause for prurigo pigmentosa has not been identified, a strong association has been linked to diet, specifically when ketosis is induced, such as in ketogenic diets and anorexia nervosa.^3-5 Other possible causes include sunlight exposure, clothing friction, and sweating.^1,5 The disease course is characterized by intermittent flares and spontaneous resolution, with recurrence in most cases. During the active phase, intensely pruritic, papulovesicular or urticarial papules are predominant and most often are localized to the upper body and torso, including the back, shoulders, neck, and chest.⁵ These flares can persist for several days but eventually subside, leaving behind a characteristic reticular pigmentation that can persist for months.⁵ First-line treatment often involves the use of tetracycline antibiotics, such as minocycline or doxycycline. ^2,4,5 Dapsone often is used with successful resolution. ⁶ Dietary modifications also have been found to be effective in treating prurigo pigmentosa, particularly in patients presenting with dietary insufficiency.^6,7 Increased carbohydrate intake has been shown to promote resolution. ⁶ Topical corticosteroids demonstrate limited efficacy in controlling flares.^6,8

Histopathology has been variably described, with initial findings reported as nonspecific.¹ However, it was later described as a distinct inflammatory disease of the skin with histologically distinct stages.^2,9 Early stages reveal scattered dermal, dermal papillary, and perivascular neutrophilic infiltration.⁹ The lesions then progress and become fully developed, at which point neutrophilic infiltration becomes more prominent, accompanied by the presence of intraepidermal neutrophils and spongiosis. As the lesions resolve, the infiltration transitions to lymphocytic, and lichenoid changes can sometimes be appreciated along with epidermal hyperplasia, hyperpigmentation, and dermal melanophages.⁹ Although these findings aid in the diagnosis of prurigo pigmentosa, a clinicopathologic correlation is necessary to establish a definitive diagnosis.

Because prurigo pigmentosa is rare, it often is misdiagnosed as another condition with a similar presentation and nonspecific biopsy findings.⁶ Allergic contact dermatitis is a common type IV delayed hypersensitivity reaction that manifests similar to prurigo pigmentosa with pruritus and a well-demarcated distribution¹⁰ that is related to the pattern of allergen exposure; in the case of allergic contact dermatitis related to textiles, a well-demarcated rash will appear in the distribution area of the associated clothing (eg, shirt, pants, shorts).¹¹ Development of allergy involves exposure and sensitization to an allergen, followed by subsequent re-exposure that results in cutaneous T-cell activation and inflammation. ¹⁰ Histopathology shows nonspecific spongiotic inflammation, and the gold standard for diagnosis is patch testing to identify the causative substance(s). Definitive treatment includes avoidance of identified allergies; however, if patients are unable to avoid the allergen or the cause is unknown, then corticosteroids, antihistamines, and/or calcineurin inhibitors are beneficial in controlling symptoms and flares.¹⁰

Pityrosporum folliculitis (also known as Malassezia folliculitis) is a fungal acneform condition that arises from overgrowth of normal skin flora Malassezia yeast,¹² which may be due to occlusion of follicles or disruption of the normal flora composition. Clinically, the manifestation may resemble prurigo pigmentosa in distribution and presence of intense pruritus. However, pustular lesions and involvement of the face can aid in differentiating Pityrosporum from prurigo pigmentosa, which can be confirmed via periodic acid–Schiff staining with numerous round yeasts within affected follicles. Oral antifungal therapy typically yields rapid improvement and resolution of symptoms.¹²

Urticaria and prurigo pigmentosa share similar clinical characteristics, with symptoms of intense pruritus and urticarial lesions on the trunk.^2,13 Urticaria is an IgEmediated type I hypersensitivity reaction characterized by wheals (ie, edematous red or pink lesions of variable size and shape that typically resolve spontaneously within 24–48 hours).¹³ Notably, urticaria will improve and in some cases completely resolve with antihistamines or anti-IgE antibody treatment, which may aid in distinguishing it from prurigo pigmentosa, as the latter typically exhibits limited response to such treatment.² Histopathology also can assist in the diagnosis by ruling out other causes of similar rash; however, biopsies are not routinely done unless other inflammatory conditions are of high suspicion.¹³

Bullous pemphigoid is an autoimmune, subepidermal, blistering dermatosis that is most common among the elderly.¹⁴ It is characterized by the presence of IgG antibodies that target BP180 and BP230, which initiate inflammatory cascades that lead to tissue damage and blister formation. It typically manifests as pruritic blistering eruptions, primarily on the limbs and trunk, but may involve the head, neck, or palmoplantar regions.¹⁴ Although blistering eruptions are the prodrome of the disease, some cases may present with nonspecific urticarial or eczematous lesions^14,15 that may resemble prurigo pigmentosa. The diagnosis is confirmed through direct immunofluorescence microscopy of biopsied lesions, which reveals IgG and/or C3 deposits along the dermoepidermal junction.14 Management of bullous pemphigoid involves timely initiation of dapsone or systemic corticosteroids, which have demonstrated high efficacy in controlling the disease and its associated symptoms.¹⁵

Our patient achieved a favorable response to diet modification and doxycycline therapy consistent with the diagnosis of prurigo pigmentosa. Unfortunately, the condition recurred following a relapse of anorexia. Management of prurigo pigmentosa necessitates not only accurate diagnosis but also addressing any underlying factors that may contribute to disease exacerbation. We anticipate the eating disorder will pose a major challenge in achieving long-term control of prurigo pigmentosa.

Using a large, real-world population, researchers have developed models that predict cognitive decline in amyloid-positive patients with either mild cognitive impairment (MCI) or mild dementia.

The models may help clinicians better answer common questions from their patients about their rate of cognitive decline, noted the investigators, led by Pieter J. van der Veere, MD, Alzheimer Center and Department of Neurology, Amsterdam Neuroscience, VU University Medical Center, Amsterdam, the Netherlands.

The findings were published online in Neurology.
 

Easy-to-Use Prototype

On average, it takes 4 years for MCI to progress to dementia. While new disease-modifying drugs targeting amyloid may slow progression, whether this effect is clinically meaningful is debatable, the investigators noted.

Earlier published models predicting cognitive decline either are limited to patients with MCI or haven’t been developed for easy clinical use, they added.

For the single-center study, researchers selected 961 amyloid-positive patients, mean age 65 years, who had at least two longitudinal Mini-Mental State Examinations (MMSEs). Of these, 310 had MCI, and 651 had mild dementia; 48% were women, and over 90% were White.

Researchers used linear mixed modeling to predict MMSE over time. They included age, sex, baseline MMSE, apolipoprotein E epsilon 4 status, cerebrospinal fluid (CSF) beta-amyloid (Aß) 1-42 and plasma phosphorylated-tau markers, and MRI total brain and hippocampal volume measures in the various models, including the final biomarker prediction models.

At follow-up, investigators found that the yearly decline in MMSEs increased in patients with both MCI and mild dementia. In MCI, the average MMSE declined from 26.4 (95% confidence interval [CI], 26.2-26.7) at baseline to 21.0 (95% CI, 20.2-21.7) after 5 years.

In mild dementia, the average MMSE declined from 22.4 (95% CI, 22.0-22.7) to 7.8 (95% CI, 6.8-8.9) at 5 years.

The predicted mean time to reach an MMSE of 20 (indicating mild dementia) for a hypothetical patient with MCI and a baseline MMSE of 28 and CSF Aß 1-42 of 925 pg/mL was 6 years (95% CI, 5.4-6.7 years).

However, with a hypothetical drug treatment that reduces the rate of decline by 30%, the patient would not reach the stage of moderate dementia for 8.6 years.

For a hypothetical patient with mild dementia with a baseline MMSE of 20 and CSF Aß 1-42 of 625 pg/mL, the predicted mean time to reach an MMSE of 15 was 2.3 years (95% CI, 2.1-2.5), or 3.3 years if decline is reduced by 30% with drug treatment.

External validation of the prediction models using data from the Alzheimer’s Disease Neuroimaging Initiative, a longitudinal cohort of patients not cognitively impaired or with MCI or dementia, showed comparable performance between the model-building approaches.

Researchers have incorporated the models in an easy-to-use calculator as a prototype tool that physicians can use to discuss prognosis, the uncertainty surrounding the predictions, and the impact of intervention strategies with patients.

Future prediction models may be able to predict patient-reported outcomes such as quality of life and daily functioning, the researchers noted.

“Until then, there is an important role for clinicians in translating the observed and predicted cognitive functions,” they wrote.

Compared with other studies predicting the MMSE decline using different statistical techniques, these new models showed similar or even better predictive performance while requiring less or similar information, the investigators noted.

The study used MMSE as a measure of cognition, but there may be intraindividual variation in these measures among cognitively normal patients, and those with cognitive decline may score lower if measurements are taken later in the day. Another study limitation was that the models were built for use in memory clinics, so generalizability to the general population could be limited.

The study was supported by Eisai, ZonMW, and Health~Holland Top Sector Life Sciences & Health. See paper for financial disclosures.

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

Using a large, real-world population, researchers have developed models that predict cognitive decline in amyloid-positive patients with either mild cognitive impairment (MCI) or mild dementia.

The models may help clinicians better answer common questions from their patients about their rate of cognitive decline, noted the investigators, led by Pieter J. van der Veere, MD, Alzheimer Center and Department of Neurology, Amsterdam Neuroscience, VU University Medical Center, Amsterdam, the Netherlands.

The findings were published online in Neurology.
 

Easy-to-Use Prototype

On average, it takes 4 years for MCI to progress to dementia. While new disease-modifying drugs targeting amyloid may slow progression, whether this effect is clinically meaningful is debatable, the investigators noted.

Earlier published models predicting cognitive decline either are limited to patients with MCI or haven’t been developed for easy clinical use, they added.

For the single-center study, researchers selected 961 amyloid-positive patients, mean age 65 years, who had at least two longitudinal Mini-Mental State Examinations (MMSEs). Of these, 310 had MCI, and 651 had mild dementia; 48% were women, and over 90% were White.

Researchers used linear mixed modeling to predict MMSE over time. They included age, sex, baseline MMSE, apolipoprotein E epsilon 4 status, cerebrospinal fluid (CSF) beta-amyloid (Aß) 1-42 and plasma phosphorylated-tau markers, and MRI total brain and hippocampal volume measures in the various models, including the final biomarker prediction models.

At follow-up, investigators found that the yearly decline in MMSEs increased in patients with both MCI and mild dementia. In MCI, the average MMSE declined from 26.4 (95% confidence interval [CI], 26.2-26.7) at baseline to 21.0 (95% CI, 20.2-21.7) after 5 years.

In mild dementia, the average MMSE declined from 22.4 (95% CI, 22.0-22.7) to 7.8 (95% CI, 6.8-8.9) at 5 years.

The predicted mean time to reach an MMSE of 20 (indicating mild dementia) for a hypothetical patient with MCI and a baseline MMSE of 28 and CSF Aß 1-42 of 925 pg/mL was 6 years (95% CI, 5.4-6.7 years).

However, with a hypothetical drug treatment that reduces the rate of decline by 30%, the patient would not reach the stage of moderate dementia for 8.6 years.

For a hypothetical patient with mild dementia with a baseline MMSE of 20 and CSF Aß 1-42 of 625 pg/mL, the predicted mean time to reach an MMSE of 15 was 2.3 years (95% CI, 2.1-2.5), or 3.3 years if decline is reduced by 30% with drug treatment.

External validation of the prediction models using data from the Alzheimer’s Disease Neuroimaging Initiative, a longitudinal cohort of patients not cognitively impaired or with MCI or dementia, showed comparable performance between the model-building approaches.

Researchers have incorporated the models in an easy-to-use calculator as a prototype tool that physicians can use to discuss prognosis, the uncertainty surrounding the predictions, and the impact of intervention strategies with patients.

Future prediction models may be able to predict patient-reported outcomes such as quality of life and daily functioning, the researchers noted.

“Until then, there is an important role for clinicians in translating the observed and predicted cognitive functions,” they wrote.

Compared with other studies predicting the MMSE decline using different statistical techniques, these new models showed similar or even better predictive performance while requiring less or similar information, the investigators noted.

The study used MMSE as a measure of cognition, but there may be intraindividual variation in these measures among cognitively normal patients, and those with cognitive decline may score lower if measurements are taken later in the day. Another study limitation was that the models were built for use in memory clinics, so generalizability to the general population could be limited.

The study was supported by Eisai, ZonMW, and Health~Holland Top Sector Life Sciences & Health. See paper for financial disclosures.

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

Using a large, real-world population, researchers have developed models that predict cognitive decline in amyloid-positive patients with either mild cognitive impairment (MCI) or mild dementia.

The models may help clinicians better answer common questions from their patients about their rate of cognitive decline, noted the investigators, led by Pieter J. van der Veere, MD, Alzheimer Center and Department of Neurology, Amsterdam Neuroscience, VU University Medical Center, Amsterdam, the Netherlands.

The findings were published online in Neurology.
 

Easy-to-Use Prototype

On average, it takes 4 years for MCI to progress to dementia. While new disease-modifying drugs targeting amyloid may slow progression, whether this effect is clinically meaningful is debatable, the investigators noted.

Earlier published models predicting cognitive decline either are limited to patients with MCI or haven’t been developed for easy clinical use, they added.

For the single-center study, researchers selected 961 amyloid-positive patients, mean age 65 years, who had at least two longitudinal Mini-Mental State Examinations (MMSEs). Of these, 310 had MCI, and 651 had mild dementia; 48% were women, and over 90% were White.

Researchers used linear mixed modeling to predict MMSE over time. They included age, sex, baseline MMSE, apolipoprotein E epsilon 4 status, cerebrospinal fluid (CSF) beta-amyloid (Aß) 1-42 and plasma phosphorylated-tau markers, and MRI total brain and hippocampal volume measures in the various models, including the final biomarker prediction models.

At follow-up, investigators found that the yearly decline in MMSEs increased in patients with both MCI and mild dementia. In MCI, the average MMSE declined from 26.4 (95% confidence interval [CI], 26.2-26.7) at baseline to 21.0 (95% CI, 20.2-21.7) after 5 years.

In mild dementia, the average MMSE declined from 22.4 (95% CI, 22.0-22.7) to 7.8 (95% CI, 6.8-8.9) at 5 years.

The predicted mean time to reach an MMSE of 20 (indicating mild dementia) for a hypothetical patient with MCI and a baseline MMSE of 28 and CSF Aß 1-42 of 925 pg/mL was 6 years (95% CI, 5.4-6.7 years).

However, with a hypothetical drug treatment that reduces the rate of decline by 30%, the patient would not reach the stage of moderate dementia for 8.6 years.

For a hypothetical patient with mild dementia with a baseline MMSE of 20 and CSF Aß 1-42 of 625 pg/mL, the predicted mean time to reach an MMSE of 15 was 2.3 years (95% CI, 2.1-2.5), or 3.3 years if decline is reduced by 30% with drug treatment.

External validation of the prediction models using data from the Alzheimer’s Disease Neuroimaging Initiative, a longitudinal cohort of patients not cognitively impaired or with MCI or dementia, showed comparable performance between the model-building approaches.

Researchers have incorporated the models in an easy-to-use calculator as a prototype tool that physicians can use to discuss prognosis, the uncertainty surrounding the predictions, and the impact of intervention strategies with patients.

Future prediction models may be able to predict patient-reported outcomes such as quality of life and daily functioning, the researchers noted.

“Until then, there is an important role for clinicians in translating the observed and predicted cognitive functions,” they wrote.

Compared with other studies predicting the MMSE decline using different statistical techniques, these new models showed similar or even better predictive performance while requiring less or similar information, the investigators noted.

The study used MMSE as a measure of cognition, but there may be intraindividual variation in these measures among cognitively normal patients, and those with cognitive decline may score lower if measurements are taken later in the day. Another study limitation was that the models were built for use in memory clinics, so generalizability to the general population could be limited.

The study was supported by Eisai, ZonMW, and Health~Holland Top Sector Life Sciences & Health. See paper for financial disclosures.

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

AstraZeneca was taken to task July 25 by the US Food and Drug Administration’s (FDA’s) Oncology Drug Advisory Committee (ODAC) for failing to heed an agency request about the design of a durvalumab (Imfinzi) trial for non–small cell lung cancer (NSCLC).

The trial in question, AEGEAN, investigated perioperative durvalumab for resectable NSCLC tumors across 802 patients. Patients without EGFR or ALK mutations were randomly assigned to receive durvalumab before surgery alongside platinum-containing chemotherapy and after surgery for a year as monotherapy or to receive chemotherapy and surgery alone. 

Patients receiving durvalumab demonstrated better event-free survival at 1 year (73.4% vs 64.5% without durvalumab) and a better pathologic complete response rate (17.2% vs 4.3% without). Currently, AstraZeneca is seeking to add the indication for durvalumab to those the agent already has. 

However, at the July 25 ODAC meeting, the committee explained that the AEGEAN trial design makes it impossible to tell whether patients benefited from durvalumab before surgery, after it, or at both points. 

Mounting evidence, including from AstraZeneca’s own studies, suggests that the benefit of immune checkpoint inhibitors, such as durvalumab, comes before surgery. That means prescribing durvalumab after surgery could be exposing patients to serious side effects and financial toxicity, with potentially no clinical benefit, “magnifying the risk of potential overtreatment,” the committee cautioned. 

When AEGEAN was being designed in 2018, FDA requested that AstraZeneca address the uncertainty surrounding when to use durvalumab by including separate neoadjuvant and adjuvant arms, or at least an arm where patients were treated with neoadjuvant durvalumab alone to compare with treatment both before and after surgery. 

The company didn’t follow through and, during the July 25 meeting, the committee wanted answers. “Why did you not comply with this?” asked ODAC committee acting chair Daniel Spratt, MD, a radiation oncologist at Case Western Reserve University in Cleveland, Ohio. 

AstraZeneca personnel explained that doing so would have required many more subjects, made the trial more expensive, and added about 2 years to AEGEAN.

One speaker noted that the company, which makes more than $4 billion a year on durvalumab, would have taken about 2 days to recoup that added cost. Others wondered whether the motive was to sell durvalumab for as long as possible across a patient’s course of treatment. 

Perhaps the biggest reason the company ignored the request is that “it wasn’t our understanding at that time that this was a barrier to approval,” an AstraZeneca regulatory affairs specialist said. 

To this end, the agency asked its advisory panel to vote on whether it should require — instead of simply request, as it did with AstraZeneca — companies to prove that patients need immunotherapy both before and after surgery in resectable NSCLC.

The 11-member panel voted unanimously that it should make this a requirement, and several members said it should do so in other cancers as well.

However, when the agency asked whether durvalumab’s resectable NSCLC approval should be delayed until AstraZeneca conducts a trial to answer the neoadjuvant vs adjuvant question, the panel members didn’t think so. 

The consensus was that because AEGEAN showed a decent benefit, patients and physicians should have it as an option, and approval shouldn’t be delayed. The panel said that the bigger question about the benefit of maintenance therapy should be left to future studies. 

FDA usually follows the advice of its advisory panels.
 

A version of this article appeared on Medscape.com.

AstraZeneca was taken to task July 25 by the US Food and Drug Administration’s (FDA’s) Oncology Drug Advisory Committee (ODAC) for failing to heed an agency request about the design of a durvalumab (Imfinzi) trial for non–small cell lung cancer (NSCLC).

The trial in question, AEGEAN, investigated perioperative durvalumab for resectable NSCLC tumors across 802 patients. Patients without EGFR or ALK mutations were randomly assigned to receive durvalumab before surgery alongside platinum-containing chemotherapy and after surgery for a year as monotherapy or to receive chemotherapy and surgery alone. 

Patients receiving durvalumab demonstrated better event-free survival at 1 year (73.4% vs 64.5% without durvalumab) and a better pathologic complete response rate (17.2% vs 4.3% without). Currently, AstraZeneca is seeking to add the indication for durvalumab to those the agent already has. 

However, at the July 25 ODAC meeting, the committee explained that the AEGEAN trial design makes it impossible to tell whether patients benefited from durvalumab before surgery, after it, or at both points. 

Mounting evidence, including from AstraZeneca’s own studies, suggests that the benefit of immune checkpoint inhibitors, such as durvalumab, comes before surgery. That means prescribing durvalumab after surgery could be exposing patients to serious side effects and financial toxicity, with potentially no clinical benefit, “magnifying the risk of potential overtreatment,” the committee cautioned. 

When AEGEAN was being designed in 2018, FDA requested that AstraZeneca address the uncertainty surrounding when to use durvalumab by including separate neoadjuvant and adjuvant arms, or at least an arm where patients were treated with neoadjuvant durvalumab alone to compare with treatment both before and after surgery. 

The company didn’t follow through and, during the July 25 meeting, the committee wanted answers. “Why did you not comply with this?” asked ODAC committee acting chair Daniel Spratt, MD, a radiation oncologist at Case Western Reserve University in Cleveland, Ohio. 

AstraZeneca personnel explained that doing so would have required many more subjects, made the trial more expensive, and added about 2 years to AEGEAN.

One speaker noted that the company, which makes more than $4 billion a year on durvalumab, would have taken about 2 days to recoup that added cost. Others wondered whether the motive was to sell durvalumab for as long as possible across a patient’s course of treatment. 

Perhaps the biggest reason the company ignored the request is that “it wasn’t our understanding at that time that this was a barrier to approval,” an AstraZeneca regulatory affairs specialist said. 

To this end, the agency asked its advisory panel to vote on whether it should require — instead of simply request, as it did with AstraZeneca — companies to prove that patients need immunotherapy both before and after surgery in resectable NSCLC.

The 11-member panel voted unanimously that it should make this a requirement, and several members said it should do so in other cancers as well.

However, when the agency asked whether durvalumab’s resectable NSCLC approval should be delayed until AstraZeneca conducts a trial to answer the neoadjuvant vs adjuvant question, the panel members didn’t think so. 

The consensus was that because AEGEAN showed a decent benefit, patients and physicians should have it as an option, and approval shouldn’t be delayed. The panel said that the bigger question about the benefit of maintenance therapy should be left to future studies. 

FDA usually follows the advice of its advisory panels.
 

A version of this article appeared on Medscape.com.

AstraZeneca was taken to task July 25 by the US Food and Drug Administration’s (FDA’s) Oncology Drug Advisory Committee (ODAC) for failing to heed an agency request about the design of a durvalumab (Imfinzi) trial for non–small cell lung cancer (NSCLC).

The trial in question, AEGEAN, investigated perioperative durvalumab for resectable NSCLC tumors across 802 patients. Patients without EGFR or ALK mutations were randomly assigned to receive durvalumab before surgery alongside platinum-containing chemotherapy and after surgery for a year as monotherapy or to receive chemotherapy and surgery alone. 

Patients receiving durvalumab demonstrated better event-free survival at 1 year (73.4% vs 64.5% without durvalumab) and a better pathologic complete response rate (17.2% vs 4.3% without). Currently, AstraZeneca is seeking to add the indication for durvalumab to those the agent already has. 

However, at the July 25 ODAC meeting, the committee explained that the AEGEAN trial design makes it impossible to tell whether patients benefited from durvalumab before surgery, after it, or at both points. 

Mounting evidence, including from AstraZeneca’s own studies, suggests that the benefit of immune checkpoint inhibitors, such as durvalumab, comes before surgery. That means prescribing durvalumab after surgery could be exposing patients to serious side effects and financial toxicity, with potentially no clinical benefit, “magnifying the risk of potential overtreatment,” the committee cautioned. 

When AEGEAN was being designed in 2018, FDA requested that AstraZeneca address the uncertainty surrounding when to use durvalumab by including separate neoadjuvant and adjuvant arms, or at least an arm where patients were treated with neoadjuvant durvalumab alone to compare with treatment both before and after surgery. 

The company didn’t follow through and, during the July 25 meeting, the committee wanted answers. “Why did you not comply with this?” asked ODAC committee acting chair Daniel Spratt, MD, a radiation oncologist at Case Western Reserve University in Cleveland, Ohio. 

AstraZeneca personnel explained that doing so would have required many more subjects, made the trial more expensive, and added about 2 years to AEGEAN.

One speaker noted that the company, which makes more than $4 billion a year on durvalumab, would have taken about 2 days to recoup that added cost. Others wondered whether the motive was to sell durvalumab for as long as possible across a patient’s course of treatment. 

Perhaps the biggest reason the company ignored the request is that “it wasn’t our understanding at that time that this was a barrier to approval,” an AstraZeneca regulatory affairs specialist said. 

To this end, the agency asked its advisory panel to vote on whether it should require — instead of simply request, as it did with AstraZeneca — companies to prove that patients need immunotherapy both before and after surgery in resectable NSCLC.

The 11-member panel voted unanimously that it should make this a requirement, and several members said it should do so in other cancers as well.

However, when the agency asked whether durvalumab’s resectable NSCLC approval should be delayed until AstraZeneca conducts a trial to answer the neoadjuvant vs adjuvant question, the panel members didn’t think so. 

The consensus was that because AEGEAN showed a decent benefit, patients and physicians should have it as an option, and approval shouldn’t be delayed. The panel said that the bigger question about the benefit of maintenance therapy should be left to future studies. 

FDA usually follows the advice of its advisory panels.
 

A version of this article appeared on Medscape.com.

Among patients who presented to a combined pediatric rheumatology/dermatology clinic (RDC) at the University of California, San Francisco (UCSF), 24% presented without a confirmed diagnosis, and only 41% received a diagnosis during their first clinic visit, results from a retrospective cohort study showed.

“This finding highlights the complexity of patients referred to this clinic,” the study’s first author, Jessica Crockett, a fourth-year medical student at UCSF, told this news organization following the annual meeting of the Society for Pediatric Dermatology, where the study was presented during a poster session. “Integrated care models such as rheumatology/dermatology clinics (RDCs) have been shown to facilitate complete clinical evaluations, establish new or revised diagnoses, and streamline care for adult patients with complex autoimmune skin diseases. However, few pediatric RDCs exist nationwide, and data therefore is quite limited.”

To advance the understanding of pediatric RDC practice patterns, the influence of the care model on patient care, and professional development for trainees and clinicians, Ms. Crockett collaborated with senior author Kelly Cordoro, MD, professor of dermatology and pediatrics at UCSF, and colleagues to evaluate a cohort of 71 patients who received care at the UCSF pediatric RDC. The clinic, which was launched in 2017, includes two dermatologists, two rheumatologists, trainees, a social worker, and a nurse. Team members participate in a preclinic conference to review patient data and images, discuss relevant literature, and develop an approach to each patient.

In a separate part of the study, the researchers distributed a survey to 17 pediatric dermatologists who participate in unique RDCs in North America. Respondents were asked to describe the variability of clinical operations, participants, administrative/clinical support, and educational value for participating physicians and trainees.

Of the 71 patients cared for at the UCSF pediatric RDC, 69% were female, 44% were White, 51% were aged 13-21 years, 42% were aged 3-12 years, and 7% were aged 0-11 years at their first clinic visit. The top four primary RDC diagnoses were linear morphea (33%), lupus (23%), psoriasis (13%), and juvenile dermatomyositis (10%).

Nearly one in four patients (17, or 24%) presented to the RDC without a confirmed diagnosis. A diagnosis was established at the first RDC visit for 7 of these 17 patients (41%). Among 54 patients who presented with an established diagnosis, the first RDC visit confirmed the diagnosis for 52 (96%) and revised it for 2 (4%). “Initial pediatric RDC evaluation significantly influenced patient care by confirming or revising preexisting diagnoses, rendering new diagnoses, and streamlining additional laboratory and imaging recommendations,” the researchers wrote in their poster.

The evaluation also resulted in modified disease management in the form of systemic medication changes or dosage adjustments as well as the initiation of novel therapies. For example, systemic medication changes were made during the first RDC visit in 34 of the 46 patients (74%) who were on systemic medication at presentation.

“Seeing complex patients together in real time allows specialists and other team members (social work, nursing, PT/OT, for example) to share ideas, communicate clearly to families, and efficiently develop recommendations,” Ms. Crockett said of the UCSF pediatric RDC. “Exposure to other specialists while caring for patients enhances medical knowledge, communication skills, and professional competency of faculty and trainees alike.”

In the survey portion of the study, each of the 17 dermatologists reported that the pediatric RDC is valuable for patient care, and 88% believed the RDC was a valuable use of their time. However, only 59% of respondents reported having administrative support, and only 29% had a dedicated clinic coordinator or navigator.

“We were surprised to find that only a quarter of pediatric RDCs incorporate an educational conference,” Dr. Cordoro told this news organization. “We have found that assembling the care team prior to seeing patients to review clinical data, discuss relevant literature, and define the clinical questions for each patient is an integral part of the clinical operation. The trainees are involved in these conference presentations, and it really enhances their understanding of the complex diagnoses we manage in this clinic and the issues faced by affected children and families. The preclinical conference increases efficiency, positively influences patient care, and supports professional development for all participants.”

The study was indirectly supported by a fellowship grant awarded to Ms. Crockett from the Pediatric Dermatology Research Alliance. The researchers reported having no relevant disclosures.

A version of this article appeared on Medscape.com.

Among patients who presented to a combined pediatric rheumatology/dermatology clinic (RDC) at the University of California, San Francisco (UCSF), 24% presented without a confirmed diagnosis, and only 41% received a diagnosis during their first clinic visit, results from a retrospective cohort study showed.

“This finding highlights the complexity of patients referred to this clinic,” the study’s first author, Jessica Crockett, a fourth-year medical student at UCSF, told this news organization following the annual meeting of the Society for Pediatric Dermatology, where the study was presented during a poster session. “Integrated care models such as rheumatology/dermatology clinics (RDCs) have been shown to facilitate complete clinical evaluations, establish new or revised diagnoses, and streamline care for adult patients with complex autoimmune skin diseases. However, few pediatric RDCs exist nationwide, and data therefore is quite limited.”

To advance the understanding of pediatric RDC practice patterns, the influence of the care model on patient care, and professional development for trainees and clinicians, Ms. Crockett collaborated with senior author Kelly Cordoro, MD, professor of dermatology and pediatrics at UCSF, and colleagues to evaluate a cohort of 71 patients who received care at the UCSF pediatric RDC. The clinic, which was launched in 2017, includes two dermatologists, two rheumatologists, trainees, a social worker, and a nurse. Team members participate in a preclinic conference to review patient data and images, discuss relevant literature, and develop an approach to each patient.

In a separate part of the study, the researchers distributed a survey to 17 pediatric dermatologists who participate in unique RDCs in North America. Respondents were asked to describe the variability of clinical operations, participants, administrative/clinical support, and educational value for participating physicians and trainees.

Of the 71 patients cared for at the UCSF pediatric RDC, 69% were female, 44% were White, 51% were aged 13-21 years, 42% were aged 3-12 years, and 7% were aged 0-11 years at their first clinic visit. The top four primary RDC diagnoses were linear morphea (33%), lupus (23%), psoriasis (13%), and juvenile dermatomyositis (10%).

Nearly one in four patients (17, or 24%) presented to the RDC without a confirmed diagnosis. A diagnosis was established at the first RDC visit for 7 of these 17 patients (41%). Among 54 patients who presented with an established diagnosis, the first RDC visit confirmed the diagnosis for 52 (96%) and revised it for 2 (4%). “Initial pediatric RDC evaluation significantly influenced patient care by confirming or revising preexisting diagnoses, rendering new diagnoses, and streamlining additional laboratory and imaging recommendations,” the researchers wrote in their poster.

The evaluation also resulted in modified disease management in the form of systemic medication changes or dosage adjustments as well as the initiation of novel therapies. For example, systemic medication changes were made during the first RDC visit in 34 of the 46 patients (74%) who were on systemic medication at presentation.

“Seeing complex patients together in real time allows specialists and other team members (social work, nursing, PT/OT, for example) to share ideas, communicate clearly to families, and efficiently develop recommendations,” Ms. Crockett said of the UCSF pediatric RDC. “Exposure to other specialists while caring for patients enhances medical knowledge, communication skills, and professional competency of faculty and trainees alike.”

In the survey portion of the study, each of the 17 dermatologists reported that the pediatric RDC is valuable for patient care, and 88% believed the RDC was a valuable use of their time. However, only 59% of respondents reported having administrative support, and only 29% had a dedicated clinic coordinator or navigator.

“We were surprised to find that only a quarter of pediatric RDCs incorporate an educational conference,” Dr. Cordoro told this news organization. “We have found that assembling the care team prior to seeing patients to review clinical data, discuss relevant literature, and define the clinical questions for each patient is an integral part of the clinical operation. The trainees are involved in these conference presentations, and it really enhances their understanding of the complex diagnoses we manage in this clinic and the issues faced by affected children and families. The preclinical conference increases efficiency, positively influences patient care, and supports professional development for all participants.”

The study was indirectly supported by a fellowship grant awarded to Ms. Crockett from the Pediatric Dermatology Research Alliance. The researchers reported having no relevant disclosures.

A version of this article appeared on Medscape.com.

Among patients who presented to a combined pediatric rheumatology/dermatology clinic (RDC) at the University of California, San Francisco (UCSF), 24% presented without a confirmed diagnosis, and only 41% received a diagnosis during their first clinic visit, results from a retrospective cohort study showed.

“This finding highlights the complexity of patients referred to this clinic,” the study’s first author, Jessica Crockett, a fourth-year medical student at UCSF, told this news organization following the annual meeting of the Society for Pediatric Dermatology, where the study was presented during a poster session. “Integrated care models such as rheumatology/dermatology clinics (RDCs) have been shown to facilitate complete clinical evaluations, establish new or revised diagnoses, and streamline care for adult patients with complex autoimmune skin diseases. However, few pediatric RDCs exist nationwide, and data therefore is quite limited.”

To advance the understanding of pediatric RDC practice patterns, the influence of the care model on patient care, and professional development for trainees and clinicians, Ms. Crockett collaborated with senior author Kelly Cordoro, MD, professor of dermatology and pediatrics at UCSF, and colleagues to evaluate a cohort of 71 patients who received care at the UCSF pediatric RDC. The clinic, which was launched in 2017, includes two dermatologists, two rheumatologists, trainees, a social worker, and a nurse. Team members participate in a preclinic conference to review patient data and images, discuss relevant literature, and develop an approach to each patient.

In a separate part of the study, the researchers distributed a survey to 17 pediatric dermatologists who participate in unique RDCs in North America. Respondents were asked to describe the variability of clinical operations, participants, administrative/clinical support, and educational value for participating physicians and trainees.

Of the 71 patients cared for at the UCSF pediatric RDC, 69% were female, 44% were White, 51% were aged 13-21 years, 42% were aged 3-12 years, and 7% were aged 0-11 years at their first clinic visit. The top four primary RDC diagnoses were linear morphea (33%), lupus (23%), psoriasis (13%), and juvenile dermatomyositis (10%).

Nearly one in four patients (17, or 24%) presented to the RDC without a confirmed diagnosis. A diagnosis was established at the first RDC visit for 7 of these 17 patients (41%). Among 54 patients who presented with an established diagnosis, the first RDC visit confirmed the diagnosis for 52 (96%) and revised it for 2 (4%). “Initial pediatric RDC evaluation significantly influenced patient care by confirming or revising preexisting diagnoses, rendering new diagnoses, and streamlining additional laboratory and imaging recommendations,” the researchers wrote in their poster.

The evaluation also resulted in modified disease management in the form of systemic medication changes or dosage adjustments as well as the initiation of novel therapies. For example, systemic medication changes were made during the first RDC visit in 34 of the 46 patients (74%) who were on systemic medication at presentation.

“Seeing complex patients together in real time allows specialists and other team members (social work, nursing, PT/OT, for example) to share ideas, communicate clearly to families, and efficiently develop recommendations,” Ms. Crockett said of the UCSF pediatric RDC. “Exposure to other specialists while caring for patients enhances medical knowledge, communication skills, and professional competency of faculty and trainees alike.”

In the survey portion of the study, each of the 17 dermatologists reported that the pediatric RDC is valuable for patient care, and 88% believed the RDC was a valuable use of their time. However, only 59% of respondents reported having administrative support, and only 29% had a dedicated clinic coordinator or navigator.

“We were surprised to find that only a quarter of pediatric RDCs incorporate an educational conference,” Dr. Cordoro told this news organization. “We have found that assembling the care team prior to seeing patients to review clinical data, discuss relevant literature, and define the clinical questions for each patient is an integral part of the clinical operation. The trainees are involved in these conference presentations, and it really enhances their understanding of the complex diagnoses we manage in this clinic and the issues faced by affected children and families. The preclinical conference increases efficiency, positively influences patient care, and supports professional development for all participants.”

The study was indirectly supported by a fellowship grant awarded to Ms. Crockett from the Pediatric Dermatology Research Alliance. The researchers reported having no relevant disclosures.

A version of this article appeared on Medscape.com.

When Black, Hispanic, and American Indian/Alaska Native parents or guardians were asked about barriers and facilitators to accessing pediatric dermatology care for their children, a theme emerged that surprised lead study author Lucinda L. Kohn, MD, MHS.

“Most families said that racial concordance didn’t matter that much, but they did place high value on being heard,” Dr. Kohn, of the Department of Dermatology at the University of Colorado, Aurora, told this news organization following the Society for Pediatric Dermatology annual meeting, where the study was presented during a poster session. “Being heard means that their experience was respected; that their questions and worries were anticipated, addressed, and answered; and that their feelings were acknowledged.”

Dr. Kohn

As a way to understand these families’ knowledge, attitudes, and beliefs about access to pediatric dermatology care and how the hospital system and medical team could better support them, Dr. Kohn and colleagues conducted in-depth, semi-structured interviews with 32 English-speaking parents and/or guardians of children who received care at the Children’s Hospital Colorado Anschutz Medical Campus pediatric dermatology clinic. The researchers conducted and recorded the 30- to 60-minute interviews via Zoom or phone call from October 17, 2023, to January 23, 2024. Domains of interest included participant background and experiences, communication preferences, and experience accessing pediatric dermatology care. Next, Dr. Kohn and colleagues used a reflexive, team-based inductive approach to carry out a thematic analysis from the interviews.

The mean age of the 32 study participants was 38.9 years; 14 (43.75%) identified as Hispanic, 11 (34.38%) as Black, and 12 (37.50%) as American Indian/Alaska Native (response categories were not mutually exclusive). Several themes emerged from analysis of the interviews. Barriers to receiving pediatric dermatology care included distrust of the healthcare system, generational and community lack of awareness about dermatology, distance to the hospital, and household income.

“One family mentioned that they needed to save up for 3 months to be able to afford the drive, hotel, and food needed for their child to attend their pediatric dermatology visit,” Dr. Kohn said. “As we know, most pediatric dermatology visits are 10-15 minutes long, so that they needed to cut groceries for 3 months to be able to see a pediatric dermatologist for 10-15 minutes is just heart wrenching. Families also didn’t understand the large teams that we have in medicine: The medical students, residents, nurses, medical assistants, attendings, and physician extenders.”

One key facilitator to receiving pediatric dermatology care was the family’s perception that the provider shares their minoritized experience because of similarities in skin tone. “When it’s your own race, whether it’s Black, Hispanic, or you know, we feel like when it’s someone like me, they will look out for me more,” one study participant said. Other facilitators expressed by the study participants included increased representation from the family’s community at all levels of healthcare (“the more you see providers and people in a space that look like you, I think the more welcoming it will feel,” one said) and normalizing dermatology care (“letting it be known that going to the dermatologist is just like going to a regular doctor,” another said).

Dr. Kohn acknowledged certain limitations of the study, including its single-center qualitative design. “Qualitative studies are not generalizable, but they do dive into the lived experiences of a few,” she said. “There aren’t a lot of qualitative studies in derm, so even though this was a very simple study, we hope the findings will help us to support our most diverse and underserved families access the pediatric dermatology care that they need.”

The researchers reported having no relevant financial disclosures. The study was recognized as an award-winning poster at the meeting.

A version of this article appeared on Medscape.com.

When Black, Hispanic, and American Indian/Alaska Native parents or guardians were asked about barriers and facilitators to accessing pediatric dermatology care for their children, a theme emerged that surprised lead study author Lucinda L. Kohn, MD, MHS.

“Most families said that racial concordance didn’t matter that much, but they did place high value on being heard,” Dr. Kohn, of the Department of Dermatology at the University of Colorado, Aurora, told this news organization following the Society for Pediatric Dermatology annual meeting, where the study was presented during a poster session. “Being heard means that their experience was respected; that their questions and worries were anticipated, addressed, and answered; and that their feelings were acknowledged.”

Dr. Kohn

As a way to understand these families’ knowledge, attitudes, and beliefs about access to pediatric dermatology care and how the hospital system and medical team could better support them, Dr. Kohn and colleagues conducted in-depth, semi-structured interviews with 32 English-speaking parents and/or guardians of children who received care at the Children’s Hospital Colorado Anschutz Medical Campus pediatric dermatology clinic. The researchers conducted and recorded the 30- to 60-minute interviews via Zoom or phone call from October 17, 2023, to January 23, 2024. Domains of interest included participant background and experiences, communication preferences, and experience accessing pediatric dermatology care. Next, Dr. Kohn and colleagues used a reflexive, team-based inductive approach to carry out a thematic analysis from the interviews.

The mean age of the 32 study participants was 38.9 years; 14 (43.75%) identified as Hispanic, 11 (34.38%) as Black, and 12 (37.50%) as American Indian/Alaska Native (response categories were not mutually exclusive). Several themes emerged from analysis of the interviews. Barriers to receiving pediatric dermatology care included distrust of the healthcare system, generational and community lack of awareness about dermatology, distance to the hospital, and household income.

“One family mentioned that they needed to save up for 3 months to be able to afford the drive, hotel, and food needed for their child to attend their pediatric dermatology visit,” Dr. Kohn said. “As we know, most pediatric dermatology visits are 10-15 minutes long, so that they needed to cut groceries for 3 months to be able to see a pediatric dermatologist for 10-15 minutes is just heart wrenching. Families also didn’t understand the large teams that we have in medicine: The medical students, residents, nurses, medical assistants, attendings, and physician extenders.”

One key facilitator to receiving pediatric dermatology care was the family’s perception that the provider shares their minoritized experience because of similarities in skin tone. “When it’s your own race, whether it’s Black, Hispanic, or you know, we feel like when it’s someone like me, they will look out for me more,” one study participant said. Other facilitators expressed by the study participants included increased representation from the family’s community at all levels of healthcare (“the more you see providers and people in a space that look like you, I think the more welcoming it will feel,” one said) and normalizing dermatology care (“letting it be known that going to the dermatologist is just like going to a regular doctor,” another said).

Dr. Kohn acknowledged certain limitations of the study, including its single-center qualitative design. “Qualitative studies are not generalizable, but they do dive into the lived experiences of a few,” she said. “There aren’t a lot of qualitative studies in derm, so even though this was a very simple study, we hope the findings will help us to support our most diverse and underserved families access the pediatric dermatology care that they need.”

The researchers reported having no relevant financial disclosures. The study was recognized as an award-winning poster at the meeting.

A version of this article appeared on Medscape.com.

When Black, Hispanic, and American Indian/Alaska Native parents or guardians were asked about barriers and facilitators to accessing pediatric dermatology care for their children, a theme emerged that surprised lead study author Lucinda L. Kohn, MD, MHS.

“Most families said that racial concordance didn’t matter that much, but they did place high value on being heard,” Dr. Kohn, of the Department of Dermatology at the University of Colorado, Aurora, told this news organization following the Society for Pediatric Dermatology annual meeting, where the study was presented during a poster session. “Being heard means that their experience was respected; that their questions and worries were anticipated, addressed, and answered; and that their feelings were acknowledged.”

Dr. Kohn

As a way to understand these families’ knowledge, attitudes, and beliefs about access to pediatric dermatology care and how the hospital system and medical team could better support them, Dr. Kohn and colleagues conducted in-depth, semi-structured interviews with 32 English-speaking parents and/or guardians of children who received care at the Children’s Hospital Colorado Anschutz Medical Campus pediatric dermatology clinic. The researchers conducted and recorded the 30- to 60-minute interviews via Zoom or phone call from October 17, 2023, to January 23, 2024. Domains of interest included participant background and experiences, communication preferences, and experience accessing pediatric dermatology care. Next, Dr. Kohn and colleagues used a reflexive, team-based inductive approach to carry out a thematic analysis from the interviews.

The mean age of the 32 study participants was 38.9 years; 14 (43.75%) identified as Hispanic, 11 (34.38%) as Black, and 12 (37.50%) as American Indian/Alaska Native (response categories were not mutually exclusive). Several themes emerged from analysis of the interviews. Barriers to receiving pediatric dermatology care included distrust of the healthcare system, generational and community lack of awareness about dermatology, distance to the hospital, and household income.

“One family mentioned that they needed to save up for 3 months to be able to afford the drive, hotel, and food needed for their child to attend their pediatric dermatology visit,” Dr. Kohn said. “As we know, most pediatric dermatology visits are 10-15 minutes long, so that they needed to cut groceries for 3 months to be able to see a pediatric dermatologist for 10-15 minutes is just heart wrenching. Families also didn’t understand the large teams that we have in medicine: The medical students, residents, nurses, medical assistants, attendings, and physician extenders.”

One key facilitator to receiving pediatric dermatology care was the family’s perception that the provider shares their minoritized experience because of similarities in skin tone. “When it’s your own race, whether it’s Black, Hispanic, or you know, we feel like when it’s someone like me, they will look out for me more,” one study participant said. Other facilitators expressed by the study participants included increased representation from the family’s community at all levels of healthcare (“the more you see providers and people in a space that look like you, I think the more welcoming it will feel,” one said) and normalizing dermatology care (“letting it be known that going to the dermatologist is just like going to a regular doctor,” another said).

Dr. Kohn acknowledged certain limitations of the study, including its single-center qualitative design. “Qualitative studies are not generalizable, but they do dive into the lived experiences of a few,” she said. “There aren’t a lot of qualitative studies in derm, so even though this was a very simple study, we hope the findings will help us to support our most diverse and underserved families access the pediatric dermatology care that they need.”

The researchers reported having no relevant financial disclosures. The study was recognized as an award-winning poster at the meeting.

A version of this article appeared on Medscape.com.

Colchicine is an effective treatment for most pediatric patients with complex aphthous stomatitis (CAS), results from a small retrospective study showed.

“Complex aphthous stomatitis in children is typically treated with topical supportive care, which is often not effective,” one of the study investigators, Ananya Shah, a third-year medical student at the University of Rochester School of Medicine & Dentistry, Rochester, New York, told this news organization following the Society for Pediatric Dermatology annual meeting, where the study was presented during a poster session. “There is limited research on CAS and its treatment in children. Colchicine is often used for treatment of CAS in adults, but its use in children has not been studied.”

Ms. Shah, in collaboration with Hilary Kunkel, MD, Nessa Aghazadeh, MD, and Megha Tollefson, MD, of the Department of Dermatology, Mayo Clinic, Rochester, Minnesota, retrospectively reviewed the charts of 20 children diagnosed with CAS who were treated with colchicine, an anti-inflammatory drug often used to treat gout, at the clinic between 2000 and 2023. Treatment responses were defined as no response, partial response, and complete response. Half of the patients were girls, and their median age at presentation was 5 years.

Ulcers were most commonly located in the buccal mucosa (80%), followed by the gingiva (50%), the mucosal lip (50%), and the palate (40%). Nearly all patients (95%) reported that the CAS caused difficulties with eating or drinking. Other effects on their quality of life included weight loss (35%), bleeding (30%), and difficulty brushing teeth (25%). “I was surprised by how much CAS impacts pediatric patients’ quality of life,” Ms. Shah said. “Almost all of the patients experienced trouble with basic activities of daily living, including eating and drinking. In addition, CAS negatively impacted mental health and led to missed school for patients.”

The researchers had follow-up data on responses to colchicine for 14 of the 20 patients. Of these, 12 (86%) had symptom improvement, 5 (36%) had a complete response, 8 (57%) had a partial response, and 1 (7%) did not respond. Nine patients (64%) experienced side effects. Of these, six had diarrhea, two had nausea, and one had constipation.

“Colchicine should be considered as a treatment in pediatric patients who have refractory complex aphthous stomatitis as it is generally well tolerated with minimal side effects,” Ms. Shah said. She acknowledged certain limitations of the study, including its single-center, retrospective design.

The researchers reported having no relevant disclosures.

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

Colchicine is an effective treatment for most pediatric patients with complex aphthous stomatitis (CAS), results from a small retrospective study showed.

“Complex aphthous stomatitis in children is typically treated with topical supportive care, which is often not effective,” one of the study investigators, Ananya Shah, a third-year medical student at the University of Rochester School of Medicine & Dentistry, Rochester, New York, told this news organization following the Society for Pediatric Dermatology annual meeting, where the study was presented during a poster session. “There is limited research on CAS and its treatment in children. Colchicine is often used for treatment of CAS in adults, but its use in children has not been studied.”

Ms. Shah, in collaboration with Hilary Kunkel, MD, Nessa Aghazadeh, MD, and Megha Tollefson, MD, of the Department of Dermatology, Mayo Clinic, Rochester, Minnesota, retrospectively reviewed the charts of 20 children diagnosed with CAS who were treated with colchicine, an anti-inflammatory drug often used to treat gout, at the clinic between 2000 and 2023. Treatment responses were defined as no response, partial response, and complete response. Half of the patients were girls, and their median age at presentation was 5 years.

Ulcers were most commonly located in the buccal mucosa (80%), followed by the gingiva (50%), the mucosal lip (50%), and the palate (40%). Nearly all patients (95%) reported that the CAS caused difficulties with eating or drinking. Other effects on their quality of life included weight loss (35%), bleeding (30%), and difficulty brushing teeth (25%). “I was surprised by how much CAS impacts pediatric patients’ quality of life,” Ms. Shah said. “Almost all of the patients experienced trouble with basic activities of daily living, including eating and drinking. In addition, CAS negatively impacted mental health and led to missed school for patients.”

The researchers had follow-up data on responses to colchicine for 14 of the 20 patients. Of these, 12 (86%) had symptom improvement, 5 (36%) had a complete response, 8 (57%) had a partial response, and 1 (7%) did not respond. Nine patients (64%) experienced side effects. Of these, six had diarrhea, two had nausea, and one had constipation.

“Colchicine should be considered as a treatment in pediatric patients who have refractory complex aphthous stomatitis as it is generally well tolerated with minimal side effects,” Ms. Shah said. She acknowledged certain limitations of the study, including its single-center, retrospective design.

The researchers reported having no relevant disclosures.

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

Colchicine is an effective treatment for most pediatric patients with complex aphthous stomatitis (CAS), results from a small retrospective study showed.

“Complex aphthous stomatitis in children is typically treated with topical supportive care, which is often not effective,” one of the study investigators, Ananya Shah, a third-year medical student at the University of Rochester School of Medicine & Dentistry, Rochester, New York, told this news organization following the Society for Pediatric Dermatology annual meeting, where the study was presented during a poster session. “There is limited research on CAS and its treatment in children. Colchicine is often used for treatment of CAS in adults, but its use in children has not been studied.”

Ms. Shah, in collaboration with Hilary Kunkel, MD, Nessa Aghazadeh, MD, and Megha Tollefson, MD, of the Department of Dermatology, Mayo Clinic, Rochester, Minnesota, retrospectively reviewed the charts of 20 children diagnosed with CAS who were treated with colchicine, an anti-inflammatory drug often used to treat gout, at the clinic between 2000 and 2023. Treatment responses were defined as no response, partial response, and complete response. Half of the patients were girls, and their median age at presentation was 5 years.

Ulcers were most commonly located in the buccal mucosa (80%), followed by the gingiva (50%), the mucosal lip (50%), and the palate (40%). Nearly all patients (95%) reported that the CAS caused difficulties with eating or drinking. Other effects on their quality of life included weight loss (35%), bleeding (30%), and difficulty brushing teeth (25%). “I was surprised by how much CAS impacts pediatric patients’ quality of life,” Ms. Shah said. “Almost all of the patients experienced trouble with basic activities of daily living, including eating and drinking. In addition, CAS negatively impacted mental health and led to missed school for patients.”

The researchers had follow-up data on responses to colchicine for 14 of the 20 patients. Of these, 12 (86%) had symptom improvement, 5 (36%) had a complete response, 8 (57%) had a partial response, and 1 (7%) did not respond. Nine patients (64%) experienced side effects. Of these, six had diarrhea, two had nausea, and one had constipation.

“Colchicine should be considered as a treatment in pediatric patients who have refractory complex aphthous stomatitis as it is generally well tolerated with minimal side effects,” Ms. Shah said. She acknowledged certain limitations of the study, including its single-center, retrospective design.

The researchers reported having no relevant disclosures.

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