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Docs should engage employers directly if they want better payment models
WASHINGTON – If doctors want to improve their reimbursement and at the same time be a catalyst for reducing costs to the overall health care delivery system, they need to be stronger advocates for themselves.
This was the message of Harold D. Miller, president and CEO of the Center for Healthcare Quality and Payment Reform, in his presentation to the American Gastroenterological Association Partners in Value meeting on Oct. 6.
He called specifically on doctors to exercise initiative and accept the accountability that comes with leading the charge for payment reform.
“I think there is a much better way, which is bottom up, which is to say ask the physicians and the hospitals to say what are the ways you can improve care and reduce costs,” he continued. “Then get payers to provide adequate support for that but have physicians take accountability for actually achieving those results they think are possible. And then you have patients who get good care and you keep physicians and hospitals financially sustainable, which nobody in Washington is talking about how to actually do.”
Getting to that point is going to require physicians to be much more proactive in who they communicate with to get the information that is necessary to build payment models from the bottom up.
“I think there are lots of potential solutions, but I think it needs to be talked about,” Mr. Miller said. “If I were to leave you with one message, the problem is that employers, Congress, etc., are not hearing from physicians that you want to do something different.”
He noted that part of the issue is the adversarial relationship doctors have with the payer community, noting that most “health plans demonize you all.”
Mr. Miller added: “They go in to employers and they tell employers that the only thing standing between the employers and certain health insurance bankruptcy is the health plan because all the doctors want to do is spend more money.”
To change that, it is going to require doctors to be much more proactive in reaching out past the payer middleman to start engaging directly with employers.
“Employers do not see doctors as their partners,” Mr. Miller noted. “The people who pay have to start seeing you as wanting to solve the same problem they are trying to solve.”
And working with employers could help physicians insofar as getting access to data that would be crucial in developing the kinds of payment models that would benefit all of the financial aspects of health care delivery while at the same time improving care.
Mr. Miller recounted how various state and local governments in Maine were trying to extract clinical information that might not be ascertained from claims data from Anthem, the largest or second largest health insurance company in America. Initially, Anthem balked, prompting the state and local entities to issue requests for information and seek to replace Anthem as the main provider for its health insurance coverage.
“Anthem completely changed its attitude,” he said. “All of a sudden, Anthem was back in. ... Anthem felt that impact all the way in Indianapolis. Sam Nussbaum, MD [former chief medical officer at Anthem], said to me, ‘We were punished in Maine.’ ”
He noted that some big employers are seeking out direct contracts with health systems because they are not getting support from the health plans.
“But they need to hear from you and what it is you need and what you are going to do with it,” he said. “It is not just give us data, see ya. It is here’s what we are trying to do. Here is the information we need and why we need it. If you say to an employer ‘I want to know how many patients are being hospitalized so that I can help you reduce hospitalizations,’ do you think they are going to say nah, we are too busy for that?”
WASHINGTON – If doctors want to improve their reimbursement and at the same time be a catalyst for reducing costs to the overall health care delivery system, they need to be stronger advocates for themselves.
This was the message of Harold D. Miller, president and CEO of the Center for Healthcare Quality and Payment Reform, in his presentation to the American Gastroenterological Association Partners in Value meeting on Oct. 6.
He called specifically on doctors to exercise initiative and accept the accountability that comes with leading the charge for payment reform.
“I think there is a much better way, which is bottom up, which is to say ask the physicians and the hospitals to say what are the ways you can improve care and reduce costs,” he continued. “Then get payers to provide adequate support for that but have physicians take accountability for actually achieving those results they think are possible. And then you have patients who get good care and you keep physicians and hospitals financially sustainable, which nobody in Washington is talking about how to actually do.”
Getting to that point is going to require physicians to be much more proactive in who they communicate with to get the information that is necessary to build payment models from the bottom up.
“I think there are lots of potential solutions, but I think it needs to be talked about,” Mr. Miller said. “If I were to leave you with one message, the problem is that employers, Congress, etc., are not hearing from physicians that you want to do something different.”
He noted that part of the issue is the adversarial relationship doctors have with the payer community, noting that most “health plans demonize you all.”
Mr. Miller added: “They go in to employers and they tell employers that the only thing standing between the employers and certain health insurance bankruptcy is the health plan because all the doctors want to do is spend more money.”
To change that, it is going to require doctors to be much more proactive in reaching out past the payer middleman to start engaging directly with employers.
“Employers do not see doctors as their partners,” Mr. Miller noted. “The people who pay have to start seeing you as wanting to solve the same problem they are trying to solve.”
And working with employers could help physicians insofar as getting access to data that would be crucial in developing the kinds of payment models that would benefit all of the financial aspects of health care delivery while at the same time improving care.
Mr. Miller recounted how various state and local governments in Maine were trying to extract clinical information that might not be ascertained from claims data from Anthem, the largest or second largest health insurance company in America. Initially, Anthem balked, prompting the state and local entities to issue requests for information and seek to replace Anthem as the main provider for its health insurance coverage.
“Anthem completely changed its attitude,” he said. “All of a sudden, Anthem was back in. ... Anthem felt that impact all the way in Indianapolis. Sam Nussbaum, MD [former chief medical officer at Anthem], said to me, ‘We were punished in Maine.’ ”
He noted that some big employers are seeking out direct contracts with health systems because they are not getting support from the health plans.
“But they need to hear from you and what it is you need and what you are going to do with it,” he said. “It is not just give us data, see ya. It is here’s what we are trying to do. Here is the information we need and why we need it. If you say to an employer ‘I want to know how many patients are being hospitalized so that I can help you reduce hospitalizations,’ do you think they are going to say nah, we are too busy for that?”
WASHINGTON – If doctors want to improve their reimbursement and at the same time be a catalyst for reducing costs to the overall health care delivery system, they need to be stronger advocates for themselves.
This was the message of Harold D. Miller, president and CEO of the Center for Healthcare Quality and Payment Reform, in his presentation to the American Gastroenterological Association Partners in Value meeting on Oct. 6.
He called specifically on doctors to exercise initiative and accept the accountability that comes with leading the charge for payment reform.
“I think there is a much better way, which is bottom up, which is to say ask the physicians and the hospitals to say what are the ways you can improve care and reduce costs,” he continued. “Then get payers to provide adequate support for that but have physicians take accountability for actually achieving those results they think are possible. And then you have patients who get good care and you keep physicians and hospitals financially sustainable, which nobody in Washington is talking about how to actually do.”
Getting to that point is going to require physicians to be much more proactive in who they communicate with to get the information that is necessary to build payment models from the bottom up.
“I think there are lots of potential solutions, but I think it needs to be talked about,” Mr. Miller said. “If I were to leave you with one message, the problem is that employers, Congress, etc., are not hearing from physicians that you want to do something different.”
He noted that part of the issue is the adversarial relationship doctors have with the payer community, noting that most “health plans demonize you all.”
Mr. Miller added: “They go in to employers and they tell employers that the only thing standing between the employers and certain health insurance bankruptcy is the health plan because all the doctors want to do is spend more money.”
To change that, it is going to require doctors to be much more proactive in reaching out past the payer middleman to start engaging directly with employers.
“Employers do not see doctors as their partners,” Mr. Miller noted. “The people who pay have to start seeing you as wanting to solve the same problem they are trying to solve.”
And working with employers could help physicians insofar as getting access to data that would be crucial in developing the kinds of payment models that would benefit all of the financial aspects of health care delivery while at the same time improving care.
Mr. Miller recounted how various state and local governments in Maine were trying to extract clinical information that might not be ascertained from claims data from Anthem, the largest or second largest health insurance company in America. Initially, Anthem balked, prompting the state and local entities to issue requests for information and seek to replace Anthem as the main provider for its health insurance coverage.
“Anthem completely changed its attitude,” he said. “All of a sudden, Anthem was back in. ... Anthem felt that impact all the way in Indianapolis. Sam Nussbaum, MD [former chief medical officer at Anthem], said to me, ‘We were punished in Maine.’ ”
He noted that some big employers are seeking out direct contracts with health systems because they are not getting support from the health plans.
“But they need to hear from you and what it is you need and what you are going to do with it,” he said. “It is not just give us data, see ya. It is here’s what we are trying to do. Here is the information we need and why we need it. If you say to an employer ‘I want to know how many patients are being hospitalized so that I can help you reduce hospitalizations,’ do you think they are going to say nah, we are too busy for that?”
Role grows for heart failure patient-reported outcomes
DALLAS – The Food and Drug Administration is keenly seeking patient-reported outcomes as endpoints in cardiovascular drug or device trials, particularly for heart failure patients, but the bar remains high for getting such an outcome into labeling, said agency officials who regulate cardiovascular disease therapies.
The FDA issued guidance nearly 8 years ago on how to integrate patient-reported outcome (PRO) measures into medical product development, but so far no heart failure drug nor device has met the agency’s standards for documented success in improving a PRO, despite the clear need for these patients to receive patient-centered care, clinicians said.
“We don’t yet have a patient-reported outcome in a label for heart failure,” Paul A. Heidenreich, MD, said during a session on PROs at the annual scientific meeting of the Heart Failure Society of America. He voiced hope that a PRO might end up on the label of a heart failure drug or device sometime in 2018. “Almost half of FDA submissions now include a PRO” as part of the data package, added Dr. Heidenreich, a cardiologist and professor of medicine at Stanford (Calif.) University.
For years, PROs for heart failure weren’t often used in trials, and they remain largely absent from routine practice – an absence Dr. Heidenreich lamented. “Just focusing on mortality in heart failure is really not patient centered,” he said.
Heart failure physicians “are very good at disease-centered care” that focuses on survival and reducing hospitalizations, but “survival is often not as important to patients,” noted Mary Norine Walsh, MD, medical director of the heart failure and cardiac transplantation program at St. Vincent Medical Group in Indianapolis. She suggested “tailoring treatment to improve patient symptoms, physical function, and quality of life” without necessarily reducing hospital readmissions or increasing survival rates. “Self-reported measures have more meaning for patients,” she said, and called for using PROs to better target interventions to the patients who can most benefit from them.
Two FDA representatives who spoke during the session agreed on the importance of PROs and attested to the agency’s interest in greater reliance on them.
“PROs are a critical complement to the other measures made in device trials,” said Bram Zuckerman, MD, director of the FDA’s division of cardiovascular devices. “We need PRO information because it reflects important aspects of patients’ health-related quality of life.”
The most commonly used PRO measures in device trials today are the Kansas City Cardiomyopathy Questionnaire (J Am Coll Cardiol. 2000 Apr;35[5]:1245-55) and the Minnesota Living With Heart Failure questionnaire, he noted.
“Neither is perfect, but there is a track record in heart failure device development that these two PROs can be helpful.” The FDA’s cardiovascular device division “wants to use PRO information,” Dr. Zuckerman said.
“All-cause mortality is the most unbiased endpoint, but there is interest in PROs,” agreed Ebony Dashiell-Aje, PhD, from the FDA’s office of new drugs in the Center for Drug Evaluation and Research. She highlighted the encouragement that the FDA gave to drug and device developers to include PROs in trials, both in its 2009 guidance document as well as in a “roadmap” from the agency on how to measure PROs in clinical trials. “Unfortunately, in heart failure we struggle to find tools that can adequately measure the patient’s perspective and be sensitive enough to detect a treatment benefit,” she said.
Norman Stockbridge, MD, director of the division of cardiovascular and renal products in the agency’s Office of Drug Evaluation, cited even bigger barriers to FDA approval of PROs as labeled effects from drugs or devices.
Getting a PRO endpoint supported by clinical-trial results that qualify it for an FDA label faces two big challenges. One challenge, he said, is “how much of an effect we need to see in a complex scoring algorithm to know that patients actually received some benefit in a disease that often varies from day to day and from week to week.” The second challenge is that, “in a disease with a high background rate of bad outcomes, you need some evidence that the benefit [from the treatment] is worth any risk,” which is something that can be hard to prove in heart failure when many patients don’t live more than 2 years with the disease, Dr. Stockbridge said in an interview.
“You need to be able to make the argument that the [PRO] benefit is likely perceptible to patients, but that is only half the problem. The other half is whether the developer can rule out that survival is not less than it would have been with no treatment. If patients take this, will they feel better but have a greater risk of being hurt?”
So far, no drug or device developer has succeeded in proving this to the FDA, despite the agency’s 2009 guidance on how it could be done.
That guidance “is one of the two worst and most destructive guidance documents we ever published,” Dr. Stockbridge declared.
Dr. Walsh, Dr. Heidenreich, Dr. Zuckerman, Dr. Dashiell-Aje, and Dr. Stockbridge had no relevant disclosures.
[email protected]
On Twitter @mitchelzoler
DALLAS – The Food and Drug Administration is keenly seeking patient-reported outcomes as endpoints in cardiovascular drug or device trials, particularly for heart failure patients, but the bar remains high for getting such an outcome into labeling, said agency officials who regulate cardiovascular disease therapies.
The FDA issued guidance nearly 8 years ago on how to integrate patient-reported outcome (PRO) measures into medical product development, but so far no heart failure drug nor device has met the agency’s standards for documented success in improving a PRO, despite the clear need for these patients to receive patient-centered care, clinicians said.
“We don’t yet have a patient-reported outcome in a label for heart failure,” Paul A. Heidenreich, MD, said during a session on PROs at the annual scientific meeting of the Heart Failure Society of America. He voiced hope that a PRO might end up on the label of a heart failure drug or device sometime in 2018. “Almost half of FDA submissions now include a PRO” as part of the data package, added Dr. Heidenreich, a cardiologist and professor of medicine at Stanford (Calif.) University.
For years, PROs for heart failure weren’t often used in trials, and they remain largely absent from routine practice – an absence Dr. Heidenreich lamented. “Just focusing on mortality in heart failure is really not patient centered,” he said.
Heart failure physicians “are very good at disease-centered care” that focuses on survival and reducing hospitalizations, but “survival is often not as important to patients,” noted Mary Norine Walsh, MD, medical director of the heart failure and cardiac transplantation program at St. Vincent Medical Group in Indianapolis. She suggested “tailoring treatment to improve patient symptoms, physical function, and quality of life” without necessarily reducing hospital readmissions or increasing survival rates. “Self-reported measures have more meaning for patients,” she said, and called for using PROs to better target interventions to the patients who can most benefit from them.
Two FDA representatives who spoke during the session agreed on the importance of PROs and attested to the agency’s interest in greater reliance on them.
“PROs are a critical complement to the other measures made in device trials,” said Bram Zuckerman, MD, director of the FDA’s division of cardiovascular devices. “We need PRO information because it reflects important aspects of patients’ health-related quality of life.”
The most commonly used PRO measures in device trials today are the Kansas City Cardiomyopathy Questionnaire (J Am Coll Cardiol. 2000 Apr;35[5]:1245-55) and the Minnesota Living With Heart Failure questionnaire, he noted.
“Neither is perfect, but there is a track record in heart failure device development that these two PROs can be helpful.” The FDA’s cardiovascular device division “wants to use PRO information,” Dr. Zuckerman said.
“All-cause mortality is the most unbiased endpoint, but there is interest in PROs,” agreed Ebony Dashiell-Aje, PhD, from the FDA’s office of new drugs in the Center for Drug Evaluation and Research. She highlighted the encouragement that the FDA gave to drug and device developers to include PROs in trials, both in its 2009 guidance document as well as in a “roadmap” from the agency on how to measure PROs in clinical trials. “Unfortunately, in heart failure we struggle to find tools that can adequately measure the patient’s perspective and be sensitive enough to detect a treatment benefit,” she said.
Norman Stockbridge, MD, director of the division of cardiovascular and renal products in the agency’s Office of Drug Evaluation, cited even bigger barriers to FDA approval of PROs as labeled effects from drugs or devices.
Getting a PRO endpoint supported by clinical-trial results that qualify it for an FDA label faces two big challenges. One challenge, he said, is “how much of an effect we need to see in a complex scoring algorithm to know that patients actually received some benefit in a disease that often varies from day to day and from week to week.” The second challenge is that, “in a disease with a high background rate of bad outcomes, you need some evidence that the benefit [from the treatment] is worth any risk,” which is something that can be hard to prove in heart failure when many patients don’t live more than 2 years with the disease, Dr. Stockbridge said in an interview.
“You need to be able to make the argument that the [PRO] benefit is likely perceptible to patients, but that is only half the problem. The other half is whether the developer can rule out that survival is not less than it would have been with no treatment. If patients take this, will they feel better but have a greater risk of being hurt?”
So far, no drug or device developer has succeeded in proving this to the FDA, despite the agency’s 2009 guidance on how it could be done.
That guidance “is one of the two worst and most destructive guidance documents we ever published,” Dr. Stockbridge declared.
Dr. Walsh, Dr. Heidenreich, Dr. Zuckerman, Dr. Dashiell-Aje, and Dr. Stockbridge had no relevant disclosures.
[email protected]
On Twitter @mitchelzoler
DALLAS – The Food and Drug Administration is keenly seeking patient-reported outcomes as endpoints in cardiovascular drug or device trials, particularly for heart failure patients, but the bar remains high for getting such an outcome into labeling, said agency officials who regulate cardiovascular disease therapies.
The FDA issued guidance nearly 8 years ago on how to integrate patient-reported outcome (PRO) measures into medical product development, but so far no heart failure drug nor device has met the agency’s standards for documented success in improving a PRO, despite the clear need for these patients to receive patient-centered care, clinicians said.
“We don’t yet have a patient-reported outcome in a label for heart failure,” Paul A. Heidenreich, MD, said during a session on PROs at the annual scientific meeting of the Heart Failure Society of America. He voiced hope that a PRO might end up on the label of a heart failure drug or device sometime in 2018. “Almost half of FDA submissions now include a PRO” as part of the data package, added Dr. Heidenreich, a cardiologist and professor of medicine at Stanford (Calif.) University.
For years, PROs for heart failure weren’t often used in trials, and they remain largely absent from routine practice – an absence Dr. Heidenreich lamented. “Just focusing on mortality in heart failure is really not patient centered,” he said.
Heart failure physicians “are very good at disease-centered care” that focuses on survival and reducing hospitalizations, but “survival is often not as important to patients,” noted Mary Norine Walsh, MD, medical director of the heart failure and cardiac transplantation program at St. Vincent Medical Group in Indianapolis. She suggested “tailoring treatment to improve patient symptoms, physical function, and quality of life” without necessarily reducing hospital readmissions or increasing survival rates. “Self-reported measures have more meaning for patients,” she said, and called for using PROs to better target interventions to the patients who can most benefit from them.
Two FDA representatives who spoke during the session agreed on the importance of PROs and attested to the agency’s interest in greater reliance on them.
“PROs are a critical complement to the other measures made in device trials,” said Bram Zuckerman, MD, director of the FDA’s division of cardiovascular devices. “We need PRO information because it reflects important aspects of patients’ health-related quality of life.”
The most commonly used PRO measures in device trials today are the Kansas City Cardiomyopathy Questionnaire (J Am Coll Cardiol. 2000 Apr;35[5]:1245-55) and the Minnesota Living With Heart Failure questionnaire, he noted.
“Neither is perfect, but there is a track record in heart failure device development that these two PROs can be helpful.” The FDA’s cardiovascular device division “wants to use PRO information,” Dr. Zuckerman said.
“All-cause mortality is the most unbiased endpoint, but there is interest in PROs,” agreed Ebony Dashiell-Aje, PhD, from the FDA’s office of new drugs in the Center for Drug Evaluation and Research. She highlighted the encouragement that the FDA gave to drug and device developers to include PROs in trials, both in its 2009 guidance document as well as in a “roadmap” from the agency on how to measure PROs in clinical trials. “Unfortunately, in heart failure we struggle to find tools that can adequately measure the patient’s perspective and be sensitive enough to detect a treatment benefit,” she said.
Norman Stockbridge, MD, director of the division of cardiovascular and renal products in the agency’s Office of Drug Evaluation, cited even bigger barriers to FDA approval of PROs as labeled effects from drugs or devices.
Getting a PRO endpoint supported by clinical-trial results that qualify it for an FDA label faces two big challenges. One challenge, he said, is “how much of an effect we need to see in a complex scoring algorithm to know that patients actually received some benefit in a disease that often varies from day to day and from week to week.” The second challenge is that, “in a disease with a high background rate of bad outcomes, you need some evidence that the benefit [from the treatment] is worth any risk,” which is something that can be hard to prove in heart failure when many patients don’t live more than 2 years with the disease, Dr. Stockbridge said in an interview.
“You need to be able to make the argument that the [PRO] benefit is likely perceptible to patients, but that is only half the problem. The other half is whether the developer can rule out that survival is not less than it would have been with no treatment. If patients take this, will they feel better but have a greater risk of being hurt?”
So far, no drug or device developer has succeeded in proving this to the FDA, despite the agency’s 2009 guidance on how it could be done.
That guidance “is one of the two worst and most destructive guidance documents we ever published,” Dr. Stockbridge declared.
Dr. Walsh, Dr. Heidenreich, Dr. Zuckerman, Dr. Dashiell-Aje, and Dr. Stockbridge had no relevant disclosures.
[email protected]
On Twitter @mitchelzoler
EXPERT ANALYSIS FROM THE HFSA ANNUAL SCIENTIFIC MEETING
VIDEO: Measuring, treating brain hypoxia looks promising for TBI
SAN DIEGO – It’s been possible for over 15 years for neurointensivists to measure the partial pressure of oxygen in the brain of patients following traumatic brain injury.
But the technology has not been widely adopted because there have been no high-quality data showing that it’s useful. As a result, in most hospitals, TBI treatment is guided mostly by intracranial pressure.
The evidence gap is being filled. In a recent phase 2 trial, there was a trend towards benefit when treatment was guided by both intracranial pressure and the brain oxygenation (Crit Care Med. 2017 Nov;45[11]:1907-14). The study was powered for nonfutility, not clinically meaningful change, but the National Institute of Neurological Disorders and Stroke has recently funded a 45-site, phase 3 trial that will definitively answer whether treatment protocols informed by both pressure and oxygen improve neurologic outcomes, said principal investigator Ramon Diaz-Arrastia, MD, PhD, a professor of neurology at the University of Pennsylvania, Philadelphia.
In an interview at the annual meeting of the American Neurological Association, he explained the work, and exactly how paying attention to brain oxygen levels changed treatment in the phase 2 study. It didn’t take anything unusual to maintain oxygen partial pressure above 20 mm Hg.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
SAN DIEGO – It’s been possible for over 15 years for neurointensivists to measure the partial pressure of oxygen in the brain of patients following traumatic brain injury.
But the technology has not been widely adopted because there have been no high-quality data showing that it’s useful. As a result, in most hospitals, TBI treatment is guided mostly by intracranial pressure.
The evidence gap is being filled. In a recent phase 2 trial, there was a trend towards benefit when treatment was guided by both intracranial pressure and the brain oxygenation (Crit Care Med. 2017 Nov;45[11]:1907-14). The study was powered for nonfutility, not clinically meaningful change, but the National Institute of Neurological Disorders and Stroke has recently funded a 45-site, phase 3 trial that will definitively answer whether treatment protocols informed by both pressure and oxygen improve neurologic outcomes, said principal investigator Ramon Diaz-Arrastia, MD, PhD, a professor of neurology at the University of Pennsylvania, Philadelphia.
In an interview at the annual meeting of the American Neurological Association, he explained the work, and exactly how paying attention to brain oxygen levels changed treatment in the phase 2 study. It didn’t take anything unusual to maintain oxygen partial pressure above 20 mm Hg.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
SAN DIEGO – It’s been possible for over 15 years for neurointensivists to measure the partial pressure of oxygen in the brain of patients following traumatic brain injury.
But the technology has not been widely adopted because there have been no high-quality data showing that it’s useful. As a result, in most hospitals, TBI treatment is guided mostly by intracranial pressure.
The evidence gap is being filled. In a recent phase 2 trial, there was a trend towards benefit when treatment was guided by both intracranial pressure and the brain oxygenation (Crit Care Med. 2017 Nov;45[11]:1907-14). The study was powered for nonfutility, not clinically meaningful change, but the National Institute of Neurological Disorders and Stroke has recently funded a 45-site, phase 3 trial that will definitively answer whether treatment protocols informed by both pressure and oxygen improve neurologic outcomes, said principal investigator Ramon Diaz-Arrastia, MD, PhD, a professor of neurology at the University of Pennsylvania, Philadelphia.
In an interview at the annual meeting of the American Neurological Association, he explained the work, and exactly how paying attention to brain oxygen levels changed treatment in the phase 2 study. It didn’t take anything unusual to maintain oxygen partial pressure above 20 mm Hg.
The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
AT ANA 2017
VIDEO: Alzheimer’s blood test expected soon
SAN DIEGO – A blood test that could be on the market in as little as 2 years has an accuracy of 89% for detecting amyloid plaques in the brain, according to a report at the annual meeting of the American Neurological Association.
It measures the ratio of amyloid-beta 42 to amyloid-beta 40; the numbers refer to how many amino acids are in the proteins. In healthy individuals, the ratio is “remarkably consistent, but when beta-42 starts to stick to plaques in the brain, it doesn’t get out into the blood, and the ratio drops; that’s what we are detecting.” It’s highly accurate in both “Alzheimer’s patients and people who are completely normal who have amyloid plaques in their brains,” said Randall Bateman, MD, a professor of neurology at Washington University, St. Louis.
The test is being developed by C2N Diagnostics; Dr. Bateman is a cofounder and scientific adviser. The company is working with the Food and Drug Administration and the Centers for Medicare and Medicaid Services to commercialize the test.
If it makes it to market – which seems likely – it could be a game changer, not only for Alzheimer’s research, but also for screening, preclinical detection, and early treatment. At present, CNS amyloidosis is detected largely by positron emission tomography and radioactive tracers.
In an interview at the meeting, Dr. Bateman explained the test, the research behind it, and what it could mean for neurologists and patients. In short, “when effective drugs are found, the blood beta-amyloid test [could] be used to screen millions of people in the general public to identify who is at risk for Alzheimer’s disease” so they can “start treatments even before memory loss and brain damage begin,” he said.
SAN DIEGO – A blood test that could be on the market in as little as 2 years has an accuracy of 89% for detecting amyloid plaques in the brain, according to a report at the annual meeting of the American Neurological Association.
It measures the ratio of amyloid-beta 42 to amyloid-beta 40; the numbers refer to how many amino acids are in the proteins. In healthy individuals, the ratio is “remarkably consistent, but when beta-42 starts to stick to plaques in the brain, it doesn’t get out into the blood, and the ratio drops; that’s what we are detecting.” It’s highly accurate in both “Alzheimer’s patients and people who are completely normal who have amyloid plaques in their brains,” said Randall Bateman, MD, a professor of neurology at Washington University, St. Louis.
The test is being developed by C2N Diagnostics; Dr. Bateman is a cofounder and scientific adviser. The company is working with the Food and Drug Administration and the Centers for Medicare and Medicaid Services to commercialize the test.
If it makes it to market – which seems likely – it could be a game changer, not only for Alzheimer’s research, but also for screening, preclinical detection, and early treatment. At present, CNS amyloidosis is detected largely by positron emission tomography and radioactive tracers.
In an interview at the meeting, Dr. Bateman explained the test, the research behind it, and what it could mean for neurologists and patients. In short, “when effective drugs are found, the blood beta-amyloid test [could] be used to screen millions of people in the general public to identify who is at risk for Alzheimer’s disease” so they can “start treatments even before memory loss and brain damage begin,” he said.
SAN DIEGO – A blood test that could be on the market in as little as 2 years has an accuracy of 89% for detecting amyloid plaques in the brain, according to a report at the annual meeting of the American Neurological Association.
It measures the ratio of amyloid-beta 42 to amyloid-beta 40; the numbers refer to how many amino acids are in the proteins. In healthy individuals, the ratio is “remarkably consistent, but when beta-42 starts to stick to plaques in the brain, it doesn’t get out into the blood, and the ratio drops; that’s what we are detecting.” It’s highly accurate in both “Alzheimer’s patients and people who are completely normal who have amyloid plaques in their brains,” said Randall Bateman, MD, a professor of neurology at Washington University, St. Louis.
The test is being developed by C2N Diagnostics; Dr. Bateman is a cofounder and scientific adviser. The company is working with the Food and Drug Administration and the Centers for Medicare and Medicaid Services to commercialize the test.
If it makes it to market – which seems likely – it could be a game changer, not only for Alzheimer’s research, but also for screening, preclinical detection, and early treatment. At present, CNS amyloidosis is detected largely by positron emission tomography and radioactive tracers.
In an interview at the meeting, Dr. Bateman explained the test, the research behind it, and what it could mean for neurologists and patients. In short, “when effective drugs are found, the blood beta-amyloid test [could] be used to screen millions of people in the general public to identify who is at risk for Alzheimer’s disease” so they can “start treatments even before memory loss and brain damage begin,” he said.
AT ANA 2017
Pediatric hospitalists take on the challenge of antibiotic stewardship
When Carol Glaser, MD, was in training, the philosophy around antibiotic prescribing often went something like this: “Ten days of antibiotics is good, but let’s do a few more days just to be sure,” she said.
Today, however, the new mantra is “less is more.” Dr. Glaser is an experienced pediatric infectious disease physician and the lead physician for pediatric antimicrobial stewardship at The Permanente Medical Group, Kaiser Permanente, at the Oakland (Calif.) Medical Center. While antibiotic stewardship is an issue relevant to nearly all hospitalists, for pediatric patients, the considerations can be unique and particularly serious.
Dr. Shah, a pediatric infectious disease physician at Cincinnati Children’s Hospital, spoke last spring at HM17, the Society of Hospital Medicine’s annual meeting. His talk drew from issues raised on pediatric hospital medicine electronic mailing lists and from audience questions. These centered on decisions regarding the use of intravenous versus oral antibiotics for pediatric patients – or what he refers to as intravenous-to-oral conversion – as well as antibiotic treatment duration.
“For many conditions in pediatrics, we used to treat with intravenous antibiotics initially – and sometimes for the entire course – and now we’re using oral antibiotics for the entire course,” Dr. Shah said. He noted that urinary tract infections were once treated with IV antibiotics in the hospital but are now routinely treated orally in an outpatient setting.
Dr. Shah cited two studies, both of which he coauthored as part of the Pediatric Research in Inpatient Settings Network, which compared intravenous versus oral antibiotics treatments given after discharge: The first, published in JAMA Pediatrics in 2014, examined treatment for osteomyelitis, while the second, which focused on complicated pneumonia, was published in Pediatrics in 2016.1,2
Both were observational, retrospective studies involving more than 2,000 children across more than 30 hospitals. The JAMA Pediatrics study found that roughly half of the patients were discharged with a peripherally inserted central catheter (PICC) line, and half were prescribed oral antibiotics. In some hospitals, 100% of patients were sent home with a PICC line, and in others, all children were sent home on oral antibiotics. Although treatment failure rates were the same for both groups, 15% of the patients sent home with a PICC line had to return to the emergency department because of PICC-related complications. Some were hospitalized.1
The Pediatrics study found less variation in PICC versus oral antibiotic use across hospitals for patients with complicated pneumonia, but the treatment failure rate was slightly higher for PICC patients at 3.2%, compared with 2.6% for those on oral antibiotics. This difference, however, was not statistically significant. PICC-related complications were observed in 7.1% of patients with PICC lines also were more likely to experience adverse drug reactions, compared with patients on oral antibiotics.2
“PICC lines have some advantages, particularly when children are unable or unwilling to take oral antibiotics, but they also have risks” said Dr. Shah. “If outcomes are equivalent, why would you subject patients to the risks of a catheter? And, every time they get a fever at home with a PICC line, they need urgent evaluation for the possibility of a catheter-associated bacterial infection. There is an emotional cost, as well, to taking care of catheters in the home setting.”
Additionally, economic pressures are compelling hospitals to reduce costs and resource utilization while maintaining or improving the quality of care, Dr. Shah pointed out. “Hospitalists do many things well, and quality improvement is one of those areas. That approach really aligns with antimicrobial stewardship, and there is greater incentive with episode-based payment models and financial penalties for excess readmissions. Reducing post-discharge IV antibiotic use aligns with stewardship goals and reduces the likelihood of hospital readmissions.”
The hospital medicine division at Dr. Shah’s hospital helped assemble a multidisciplinary team involving emergency physicians, pharmacists, nursing staff, hospitalists, and infectious disease physicians to encourage the use of appropriate, narrow-spectrum antibiotics and reduce the duration of antibiotic therapies. For example, skin and soft-tissue infections that were once treated for 10-14 days are now sufficiently treated in 5-7days. These efforts to improve outcomes through better adherence to evidence-based practices, including better stewardship, earned the team the SHM Teamwork in Quality Improvement Award in 2014.
“Quality improvement is really about changing the system, and hospitalists, who excel in QI, are poised to help drive antimicrobial stewardship efforts,” Dr. Shah said.
At Oakland Medical Center, Dr. Glaser helped implement handshake rounds, an idea they adopted from a group in Colorado. Every day, with every patient, the antimicrobial stewardship team meets with representatives of the teams – pediatric intensive care, the wards, the NICU, and others – to review antibiotic treatment plans for the choice of antimicrobial drug, for the duration of treatment, and for specific conditions. “We work really closely with hospitalists and our strong pediatric pharmacy team every day to ask: ‘Do we have the right dose? Do we really need to use this antibiotic?’ ” Dr. Glaser said.
Last year, she also worked to incorporate antimicrobial stewardship principles into the hospital’s residency program. “I think the most important thing we’re doing is changing the culture,” she said. “For these young physicians, we’re giving them the knowledge to empower them rather than telling them what to do and giving them a better, fundamental understanding of infectious disease.”
For instance, most pediatric respiratory illnesses are caused by a virus, yet physicians will still prescribe antibiotics for a host of reasons – including the expectations of parents, the guesswork that can go into diagnosing a young patient who cannot describe what is wrong, and the fear that children will get sicker if an antibiotic is not started early.
“A lot of it is figuring out the best approach with the least amount of side effects but covering what we need to cover for a given patient,” she said.
A number of physicians from Dr. Glaser’s team presented stewardship data from their hospital at the July 2017 Pediatric Hospital Medicine meeting in Nashville, demonstrating that, overall, they are using fewer antibiotics and that fewer of those used are broad spectrum. This satisfies the “pillars of stewardship,” Dr. Glaser said. Use antibiotics only when you need them, use them only as long as you need, and then make sure you use the most narrow-spectrum antibiotic you possibly can, she said.
Oakland Medical Center has benefited from a strong commitment to antimicrobial stewardship efforts, Dr. Glaser said, noting that many programs may lack such support, a problem that can be one of the biggest hurdles antimicrobial stewardship efforts face. The support at her hospital “has been an immense help in getting our program to where it is today.”
References
1. Keren R, Shah SS, Srivastava R, et al. Comparative effectiveness of intravenous vs oral antibiotics for postdischarge treatment of acute osteomyelitis in children. JAMA Pediatr. 2015 Feb:169(2):120-8.
2. Shah SS, Srivastava R, Wu S, et al. Intravenous versus oral antibiotics for postdischarge treatment of complicated pneumonia. Pediatrics. 2016 Dec;138(6). pii: e20161692.
When Carol Glaser, MD, was in training, the philosophy around antibiotic prescribing often went something like this: “Ten days of antibiotics is good, but let’s do a few more days just to be sure,” she said.
Today, however, the new mantra is “less is more.” Dr. Glaser is an experienced pediatric infectious disease physician and the lead physician for pediatric antimicrobial stewardship at The Permanente Medical Group, Kaiser Permanente, at the Oakland (Calif.) Medical Center. While antibiotic stewardship is an issue relevant to nearly all hospitalists, for pediatric patients, the considerations can be unique and particularly serious.
Dr. Shah, a pediatric infectious disease physician at Cincinnati Children’s Hospital, spoke last spring at HM17, the Society of Hospital Medicine’s annual meeting. His talk drew from issues raised on pediatric hospital medicine electronic mailing lists and from audience questions. These centered on decisions regarding the use of intravenous versus oral antibiotics for pediatric patients – or what he refers to as intravenous-to-oral conversion – as well as antibiotic treatment duration.
“For many conditions in pediatrics, we used to treat with intravenous antibiotics initially – and sometimes for the entire course – and now we’re using oral antibiotics for the entire course,” Dr. Shah said. He noted that urinary tract infections were once treated with IV antibiotics in the hospital but are now routinely treated orally in an outpatient setting.
Dr. Shah cited two studies, both of which he coauthored as part of the Pediatric Research in Inpatient Settings Network, which compared intravenous versus oral antibiotics treatments given after discharge: The first, published in JAMA Pediatrics in 2014, examined treatment for osteomyelitis, while the second, which focused on complicated pneumonia, was published in Pediatrics in 2016.1,2
Both were observational, retrospective studies involving more than 2,000 children across more than 30 hospitals. The JAMA Pediatrics study found that roughly half of the patients were discharged with a peripherally inserted central catheter (PICC) line, and half were prescribed oral antibiotics. In some hospitals, 100% of patients were sent home with a PICC line, and in others, all children were sent home on oral antibiotics. Although treatment failure rates were the same for both groups, 15% of the patients sent home with a PICC line had to return to the emergency department because of PICC-related complications. Some were hospitalized.1
The Pediatrics study found less variation in PICC versus oral antibiotic use across hospitals for patients with complicated pneumonia, but the treatment failure rate was slightly higher for PICC patients at 3.2%, compared with 2.6% for those on oral antibiotics. This difference, however, was not statistically significant. PICC-related complications were observed in 7.1% of patients with PICC lines also were more likely to experience adverse drug reactions, compared with patients on oral antibiotics.2
“PICC lines have some advantages, particularly when children are unable or unwilling to take oral antibiotics, but they also have risks” said Dr. Shah. “If outcomes are equivalent, why would you subject patients to the risks of a catheter? And, every time they get a fever at home with a PICC line, they need urgent evaluation for the possibility of a catheter-associated bacterial infection. There is an emotional cost, as well, to taking care of catheters in the home setting.”
Additionally, economic pressures are compelling hospitals to reduce costs and resource utilization while maintaining or improving the quality of care, Dr. Shah pointed out. “Hospitalists do many things well, and quality improvement is one of those areas. That approach really aligns with antimicrobial stewardship, and there is greater incentive with episode-based payment models and financial penalties for excess readmissions. Reducing post-discharge IV antibiotic use aligns with stewardship goals and reduces the likelihood of hospital readmissions.”
The hospital medicine division at Dr. Shah’s hospital helped assemble a multidisciplinary team involving emergency physicians, pharmacists, nursing staff, hospitalists, and infectious disease physicians to encourage the use of appropriate, narrow-spectrum antibiotics and reduce the duration of antibiotic therapies. For example, skin and soft-tissue infections that were once treated for 10-14 days are now sufficiently treated in 5-7days. These efforts to improve outcomes through better adherence to evidence-based practices, including better stewardship, earned the team the SHM Teamwork in Quality Improvement Award in 2014.
“Quality improvement is really about changing the system, and hospitalists, who excel in QI, are poised to help drive antimicrobial stewardship efforts,” Dr. Shah said.
At Oakland Medical Center, Dr. Glaser helped implement handshake rounds, an idea they adopted from a group in Colorado. Every day, with every patient, the antimicrobial stewardship team meets with representatives of the teams – pediatric intensive care, the wards, the NICU, and others – to review antibiotic treatment plans for the choice of antimicrobial drug, for the duration of treatment, and for specific conditions. “We work really closely with hospitalists and our strong pediatric pharmacy team every day to ask: ‘Do we have the right dose? Do we really need to use this antibiotic?’ ” Dr. Glaser said.
Last year, she also worked to incorporate antimicrobial stewardship principles into the hospital’s residency program. “I think the most important thing we’re doing is changing the culture,” she said. “For these young physicians, we’re giving them the knowledge to empower them rather than telling them what to do and giving them a better, fundamental understanding of infectious disease.”
For instance, most pediatric respiratory illnesses are caused by a virus, yet physicians will still prescribe antibiotics for a host of reasons – including the expectations of parents, the guesswork that can go into diagnosing a young patient who cannot describe what is wrong, and the fear that children will get sicker if an antibiotic is not started early.
“A lot of it is figuring out the best approach with the least amount of side effects but covering what we need to cover for a given patient,” she said.
A number of physicians from Dr. Glaser’s team presented stewardship data from their hospital at the July 2017 Pediatric Hospital Medicine meeting in Nashville, demonstrating that, overall, they are using fewer antibiotics and that fewer of those used are broad spectrum. This satisfies the “pillars of stewardship,” Dr. Glaser said. Use antibiotics only when you need them, use them only as long as you need, and then make sure you use the most narrow-spectrum antibiotic you possibly can, she said.
Oakland Medical Center has benefited from a strong commitment to antimicrobial stewardship efforts, Dr. Glaser said, noting that many programs may lack such support, a problem that can be one of the biggest hurdles antimicrobial stewardship efforts face. The support at her hospital “has been an immense help in getting our program to where it is today.”
References
1. Keren R, Shah SS, Srivastava R, et al. Comparative effectiveness of intravenous vs oral antibiotics for postdischarge treatment of acute osteomyelitis in children. JAMA Pediatr. 2015 Feb:169(2):120-8.
2. Shah SS, Srivastava R, Wu S, et al. Intravenous versus oral antibiotics for postdischarge treatment of complicated pneumonia. Pediatrics. 2016 Dec;138(6). pii: e20161692.
When Carol Glaser, MD, was in training, the philosophy around antibiotic prescribing often went something like this: “Ten days of antibiotics is good, but let’s do a few more days just to be sure,” she said.
Today, however, the new mantra is “less is more.” Dr. Glaser is an experienced pediatric infectious disease physician and the lead physician for pediatric antimicrobial stewardship at The Permanente Medical Group, Kaiser Permanente, at the Oakland (Calif.) Medical Center. While antibiotic stewardship is an issue relevant to nearly all hospitalists, for pediatric patients, the considerations can be unique and particularly serious.
Dr. Shah, a pediatric infectious disease physician at Cincinnati Children’s Hospital, spoke last spring at HM17, the Society of Hospital Medicine’s annual meeting. His talk drew from issues raised on pediatric hospital medicine electronic mailing lists and from audience questions. These centered on decisions regarding the use of intravenous versus oral antibiotics for pediatric patients – or what he refers to as intravenous-to-oral conversion – as well as antibiotic treatment duration.
“For many conditions in pediatrics, we used to treat with intravenous antibiotics initially – and sometimes for the entire course – and now we’re using oral antibiotics for the entire course,” Dr. Shah said. He noted that urinary tract infections were once treated with IV antibiotics in the hospital but are now routinely treated orally in an outpatient setting.
Dr. Shah cited two studies, both of which he coauthored as part of the Pediatric Research in Inpatient Settings Network, which compared intravenous versus oral antibiotics treatments given after discharge: The first, published in JAMA Pediatrics in 2014, examined treatment for osteomyelitis, while the second, which focused on complicated pneumonia, was published in Pediatrics in 2016.1,2
Both were observational, retrospective studies involving more than 2,000 children across more than 30 hospitals. The JAMA Pediatrics study found that roughly half of the patients were discharged with a peripherally inserted central catheter (PICC) line, and half were prescribed oral antibiotics. In some hospitals, 100% of patients were sent home with a PICC line, and in others, all children were sent home on oral antibiotics. Although treatment failure rates were the same for both groups, 15% of the patients sent home with a PICC line had to return to the emergency department because of PICC-related complications. Some were hospitalized.1
The Pediatrics study found less variation in PICC versus oral antibiotic use across hospitals for patients with complicated pneumonia, but the treatment failure rate was slightly higher for PICC patients at 3.2%, compared with 2.6% for those on oral antibiotics. This difference, however, was not statistically significant. PICC-related complications were observed in 7.1% of patients with PICC lines also were more likely to experience adverse drug reactions, compared with patients on oral antibiotics.2
“PICC lines have some advantages, particularly when children are unable or unwilling to take oral antibiotics, but they also have risks” said Dr. Shah. “If outcomes are equivalent, why would you subject patients to the risks of a catheter? And, every time they get a fever at home with a PICC line, they need urgent evaluation for the possibility of a catheter-associated bacterial infection. There is an emotional cost, as well, to taking care of catheters in the home setting.”
Additionally, economic pressures are compelling hospitals to reduce costs and resource utilization while maintaining or improving the quality of care, Dr. Shah pointed out. “Hospitalists do many things well, and quality improvement is one of those areas. That approach really aligns with antimicrobial stewardship, and there is greater incentive with episode-based payment models and financial penalties for excess readmissions. Reducing post-discharge IV antibiotic use aligns with stewardship goals and reduces the likelihood of hospital readmissions.”
The hospital medicine division at Dr. Shah’s hospital helped assemble a multidisciplinary team involving emergency physicians, pharmacists, nursing staff, hospitalists, and infectious disease physicians to encourage the use of appropriate, narrow-spectrum antibiotics and reduce the duration of antibiotic therapies. For example, skin and soft-tissue infections that were once treated for 10-14 days are now sufficiently treated in 5-7days. These efforts to improve outcomes through better adherence to evidence-based practices, including better stewardship, earned the team the SHM Teamwork in Quality Improvement Award in 2014.
“Quality improvement is really about changing the system, and hospitalists, who excel in QI, are poised to help drive antimicrobial stewardship efforts,” Dr. Shah said.
At Oakland Medical Center, Dr. Glaser helped implement handshake rounds, an idea they adopted from a group in Colorado. Every day, with every patient, the antimicrobial stewardship team meets with representatives of the teams – pediatric intensive care, the wards, the NICU, and others – to review antibiotic treatment plans for the choice of antimicrobial drug, for the duration of treatment, and for specific conditions. “We work really closely with hospitalists and our strong pediatric pharmacy team every day to ask: ‘Do we have the right dose? Do we really need to use this antibiotic?’ ” Dr. Glaser said.
Last year, she also worked to incorporate antimicrobial stewardship principles into the hospital’s residency program. “I think the most important thing we’re doing is changing the culture,” she said. “For these young physicians, we’re giving them the knowledge to empower them rather than telling them what to do and giving them a better, fundamental understanding of infectious disease.”
For instance, most pediatric respiratory illnesses are caused by a virus, yet physicians will still prescribe antibiotics for a host of reasons – including the expectations of parents, the guesswork that can go into diagnosing a young patient who cannot describe what is wrong, and the fear that children will get sicker if an antibiotic is not started early.
“A lot of it is figuring out the best approach with the least amount of side effects but covering what we need to cover for a given patient,” she said.
A number of physicians from Dr. Glaser’s team presented stewardship data from their hospital at the July 2017 Pediatric Hospital Medicine meeting in Nashville, demonstrating that, overall, they are using fewer antibiotics and that fewer of those used are broad spectrum. This satisfies the “pillars of stewardship,” Dr. Glaser said. Use antibiotics only when you need them, use them only as long as you need, and then make sure you use the most narrow-spectrum antibiotic you possibly can, she said.
Oakland Medical Center has benefited from a strong commitment to antimicrobial stewardship efforts, Dr. Glaser said, noting that many programs may lack such support, a problem that can be one of the biggest hurdles antimicrobial stewardship efforts face. The support at her hospital “has been an immense help in getting our program to where it is today.”
References
1. Keren R, Shah SS, Srivastava R, et al. Comparative effectiveness of intravenous vs oral antibiotics for postdischarge treatment of acute osteomyelitis in children. JAMA Pediatr. 2015 Feb:169(2):120-8.
2. Shah SS, Srivastava R, Wu S, et al. Intravenous versus oral antibiotics for postdischarge treatment of complicated pneumonia. Pediatrics. 2016 Dec;138(6). pii: e20161692.
FDA approves second CAR-T therapy
A second chimeric antigen receptor (CAR) T-cell therapy has gained FDA approval, this time for the treatment of large B-cell lymphoma in adults.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “This approval demonstrates the continued momentum of this promising new area of medicine, and we’re committed to supporting and helping expedite the development of these products.”
Approval was based on ZUMA-1, a multicenter clinical trial of 101 adults with refractory or relapsed large B-cell lymphoma. Almost three-quarters (72%) of patients responded, including 51% who achieved complete remission.
CAR-T therapy can cause severe, life-threatening side effects, most notably cytokine release syndrome (CRS) and neurologic toxicities, for which axicabtagene ciloleucel will carry a boxed warning and will come with a risk evaluation and mitigation strategy (REMS), according to the FDA.
The list price for a single treatment of axicabtagene ciloleucel is $373,000, according to the manufacturer.
“We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine,” Dr. Gottlieb said in a statement. “That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”
Axicabtagene ciloleucel was developed by Kite Pharma, which was acquired recently by Gilead Sciences.
[email protected]
On Twitter @denisefulton
A second chimeric antigen receptor (CAR) T-cell therapy has gained FDA approval, this time for the treatment of large B-cell lymphoma in adults.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “This approval demonstrates the continued momentum of this promising new area of medicine, and we’re committed to supporting and helping expedite the development of these products.”
Approval was based on ZUMA-1, a multicenter clinical trial of 101 adults with refractory or relapsed large B-cell lymphoma. Almost three-quarters (72%) of patients responded, including 51% who achieved complete remission.
CAR-T therapy can cause severe, life-threatening side effects, most notably cytokine release syndrome (CRS) and neurologic toxicities, for which axicabtagene ciloleucel will carry a boxed warning and will come with a risk evaluation and mitigation strategy (REMS), according to the FDA.
The list price for a single treatment of axicabtagene ciloleucel is $373,000, according to the manufacturer.
“We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine,” Dr. Gottlieb said in a statement. “That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”
Axicabtagene ciloleucel was developed by Kite Pharma, which was acquired recently by Gilead Sciences.
[email protected]
On Twitter @denisefulton
A second chimeric antigen receptor (CAR) T-cell therapy has gained FDA approval, this time for the treatment of large B-cell lymphoma in adults.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases,” FDA Commissioner Scott Gottlieb, MD, said in a statement. “This approval demonstrates the continued momentum of this promising new area of medicine, and we’re committed to supporting and helping expedite the development of these products.”
Approval was based on ZUMA-1, a multicenter clinical trial of 101 adults with refractory or relapsed large B-cell lymphoma. Almost three-quarters (72%) of patients responded, including 51% who achieved complete remission.
CAR-T therapy can cause severe, life-threatening side effects, most notably cytokine release syndrome (CRS) and neurologic toxicities, for which axicabtagene ciloleucel will carry a boxed warning and will come with a risk evaluation and mitigation strategy (REMS), according to the FDA.
The list price for a single treatment of axicabtagene ciloleucel is $373,000, according to the manufacturer.
“We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine,” Dr. Gottlieb said in a statement. “That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”
Axicabtagene ciloleucel was developed by Kite Pharma, which was acquired recently by Gilead Sciences.
[email protected]
On Twitter @denisefulton
VA Partnership Expands Access to Lung Screening Programs
Lung cancer has an 80% cure rate when caught early, and screening programs are key to providing this chance. The VA and the Bristol-Myers Squibb Foundation have established the VA-Partnership to increase Access to Lung Screening (VA-PALS) Implementation Network.
The initiative builds upon experience gained from other screening programs, the VA says, including those of the VA’s Office of Rural Health, which is supporting the project’s goal to reach veterans living in rural areas. It also adds to a portfolio of other major VA lung cancer initiatives, including the VALOR Trial (Veterans Affairs Lung Cancer Or Stereotactic Radiotherapy) and the APOLLO Network (Applied Proteogenomics OrganizationaL Learning and Outcomes).
“Research shows that with comprehensive lung screening programs, early identification of lung cancer leads to more effective treatments and, ultimately, saves lives,” said John Damonti, president of Bristol-Myers Squibb Foundation, the project’s sponsor.
The project will launch with lung-screening services at the Phoenix VA Health Care System in Arizona by December 2017, and then extend these services to 9 additional VA medical facilities starting in 2018.
Lung cancer has an 80% cure rate when caught early, and screening programs are key to providing this chance. The VA and the Bristol-Myers Squibb Foundation have established the VA-Partnership to increase Access to Lung Screening (VA-PALS) Implementation Network.
The initiative builds upon experience gained from other screening programs, the VA says, including those of the VA’s Office of Rural Health, which is supporting the project’s goal to reach veterans living in rural areas. It also adds to a portfolio of other major VA lung cancer initiatives, including the VALOR Trial (Veterans Affairs Lung Cancer Or Stereotactic Radiotherapy) and the APOLLO Network (Applied Proteogenomics OrganizationaL Learning and Outcomes).
“Research shows that with comprehensive lung screening programs, early identification of lung cancer leads to more effective treatments and, ultimately, saves lives,” said John Damonti, president of Bristol-Myers Squibb Foundation, the project’s sponsor.
The project will launch with lung-screening services at the Phoenix VA Health Care System in Arizona by December 2017, and then extend these services to 9 additional VA medical facilities starting in 2018.
Lung cancer has an 80% cure rate when caught early, and screening programs are key to providing this chance. The VA and the Bristol-Myers Squibb Foundation have established the VA-Partnership to increase Access to Lung Screening (VA-PALS) Implementation Network.
The initiative builds upon experience gained from other screening programs, the VA says, including those of the VA’s Office of Rural Health, which is supporting the project’s goal to reach veterans living in rural areas. It also adds to a portfolio of other major VA lung cancer initiatives, including the VALOR Trial (Veterans Affairs Lung Cancer Or Stereotactic Radiotherapy) and the APOLLO Network (Applied Proteogenomics OrganizationaL Learning and Outcomes).
“Research shows that with comprehensive lung screening programs, early identification of lung cancer leads to more effective treatments and, ultimately, saves lives,” said John Damonti, president of Bristol-Myers Squibb Foundation, the project’s sponsor.
The project will launch with lung-screening services at the Phoenix VA Health Care System in Arizona by December 2017, and then extend these services to 9 additional VA medical facilities starting in 2018.
This Is No Measly Rash
A 7-year-old girl is urgently referred to dermatology for a rash of several weeks’ duration. It is the rash itself, rather than any related symptoms, that frightens the family; the possibility of measles (raised by their primary care provider) compounded their concern. Antifungal cream (clotrimazole) has been of no help.
Physically, the child feels fine, without fever or malaise. But further questioning reveals that she was diagnosed with and treated for strep throat “about a month before” the rash developed.
The child was recently adopted by her aunt and uncle after her parents were killed in an automobile accident. This, understandably, has caused her to fall behind in school. She has no pets, no siblings, and no family history of skin disease.
EXAMINATION
Numerous discrete, round papules and plaques are distributed very evenly on the trunk. They are uniformly scaly and pink, measuring 2 to 3 cm each. In addition, several areas of thick, white, tenacious scaling are seen in the scalp.
The patient’s elbows, knees, and nails are free of any notable change.
What is the diagnosis?
This is a typical case of guttate psoriasis, which is genetic in predisposition but triggered by strep in the susceptible patient. It’s tempting to partially attribute this case to the child’s high stress level, but this is purely speculative.
Psoriasis affects about 7 million people in the US—more than 150 million worldwide—in a variety of forms. The guttate morphology is seen primarily in children, with plaques that favor extensor surfaces of the arms, trunk, and legs. Biopsy was not needed in this case; if performed, it would have shown characteristic changes (eg, parakeratosis, fusing of rete ridges).
These changes occur because psoriasis, an autoimmune disease, targets keratinocytes—cells that regenerate over a 28-day period to replace the outer layer of skin as it flakes off. With psoriasis, this process is accelerated; keratinocytes move and slough off within a week, creating scaly, inflamed lesions.
Long-term follow-up is needed to monitor the patient’s overall health, because psoriasis increases comorbidity of conditions such as coronary vessel disease, diabetes, stroke, and cancer. In patients with any form of psoriasis, there is the potential for psoriatic arthropathy, a destructive, crippling form of arthropathy that affects up to 25% of all patients. And in a third of guttate psoriasis cases, the condition evolves into permanent plaque psoriasis. For this reason, the guttate variety must be treated aggressively with a combination of phototherapy and topical steroids, adequate treatment of any residual strep, and, in adults, the occasional addition of methotrexate.
Regarding the family’s concern, measles does not involve scaly papules and plaques and would likely cause other constitutional symptoms (eg, fever, malaise, myalgia). The other item in the differential, tinea corporis, involves lesions that are not as uniformly scaly, numerous, or evenly spaced. And with tinea, scaling typically occurs on the peripheral leading edge of the lesion.
TAKE-HOME LEARNING POINTS
- Guttate psoriasis is more common in children than in adults and is often triggered by strep infection, though stress has also been implicated as a trigger.
- In about a third of all cases, this type evolves into permanent plaque psoriasis. All patients with psoriasis are at risk for psoriatic arthropathy (25% of patients).
- Measles does not involve scaly papules and plaques and would likely cause constitutional symptoms (eg, fever, malaise, myalgia).
- Tinea corporis lesions are not uniformly scaly, nor would they be as numerous or evenly spaced; most of the scale in tinea is seen on the peripheral leading edge of the lesion.
A 7-year-old girl is urgently referred to dermatology for a rash of several weeks’ duration. It is the rash itself, rather than any related symptoms, that frightens the family; the possibility of measles (raised by their primary care provider) compounded their concern. Antifungal cream (clotrimazole) has been of no help.
Physically, the child feels fine, without fever or malaise. But further questioning reveals that she was diagnosed with and treated for strep throat “about a month before” the rash developed.
The child was recently adopted by her aunt and uncle after her parents were killed in an automobile accident. This, understandably, has caused her to fall behind in school. She has no pets, no siblings, and no family history of skin disease.
EXAMINATION
Numerous discrete, round papules and plaques are distributed very evenly on the trunk. They are uniformly scaly and pink, measuring 2 to 3 cm each. In addition, several areas of thick, white, tenacious scaling are seen in the scalp.
The patient’s elbows, knees, and nails are free of any notable change.
What is the diagnosis?
This is a typical case of guttate psoriasis, which is genetic in predisposition but triggered by strep in the susceptible patient. It’s tempting to partially attribute this case to the child’s high stress level, but this is purely speculative.
Psoriasis affects about 7 million people in the US—more than 150 million worldwide—in a variety of forms. The guttate morphology is seen primarily in children, with plaques that favor extensor surfaces of the arms, trunk, and legs. Biopsy was not needed in this case; if performed, it would have shown characteristic changes (eg, parakeratosis, fusing of rete ridges).
These changes occur because psoriasis, an autoimmune disease, targets keratinocytes—cells that regenerate over a 28-day period to replace the outer layer of skin as it flakes off. With psoriasis, this process is accelerated; keratinocytes move and slough off within a week, creating scaly, inflamed lesions.
Long-term follow-up is needed to monitor the patient’s overall health, because psoriasis increases comorbidity of conditions such as coronary vessel disease, diabetes, stroke, and cancer. In patients with any form of psoriasis, there is the potential for psoriatic arthropathy, a destructive, crippling form of arthropathy that affects up to 25% of all patients. And in a third of guttate psoriasis cases, the condition evolves into permanent plaque psoriasis. For this reason, the guttate variety must be treated aggressively with a combination of phototherapy and topical steroids, adequate treatment of any residual strep, and, in adults, the occasional addition of methotrexate.
Regarding the family’s concern, measles does not involve scaly papules and plaques and would likely cause other constitutional symptoms (eg, fever, malaise, myalgia). The other item in the differential, tinea corporis, involves lesions that are not as uniformly scaly, numerous, or evenly spaced. And with tinea, scaling typically occurs on the peripheral leading edge of the lesion.
TAKE-HOME LEARNING POINTS
- Guttate psoriasis is more common in children than in adults and is often triggered by strep infection, though stress has also been implicated as a trigger.
- In about a third of all cases, this type evolves into permanent plaque psoriasis. All patients with psoriasis are at risk for psoriatic arthropathy (25% of patients).
- Measles does not involve scaly papules and plaques and would likely cause constitutional symptoms (eg, fever, malaise, myalgia).
- Tinea corporis lesions are not uniformly scaly, nor would they be as numerous or evenly spaced; most of the scale in tinea is seen on the peripheral leading edge of the lesion.
A 7-year-old girl is urgently referred to dermatology for a rash of several weeks’ duration. It is the rash itself, rather than any related symptoms, that frightens the family; the possibility of measles (raised by their primary care provider) compounded their concern. Antifungal cream (clotrimazole) has been of no help.
Physically, the child feels fine, without fever or malaise. But further questioning reveals that she was diagnosed with and treated for strep throat “about a month before” the rash developed.
The child was recently adopted by her aunt and uncle after her parents were killed in an automobile accident. This, understandably, has caused her to fall behind in school. She has no pets, no siblings, and no family history of skin disease.
EXAMINATION
Numerous discrete, round papules and plaques are distributed very evenly on the trunk. They are uniformly scaly and pink, measuring 2 to 3 cm each. In addition, several areas of thick, white, tenacious scaling are seen in the scalp.
The patient’s elbows, knees, and nails are free of any notable change.
What is the diagnosis?
This is a typical case of guttate psoriasis, which is genetic in predisposition but triggered by strep in the susceptible patient. It’s tempting to partially attribute this case to the child’s high stress level, but this is purely speculative.
Psoriasis affects about 7 million people in the US—more than 150 million worldwide—in a variety of forms. The guttate morphology is seen primarily in children, with plaques that favor extensor surfaces of the arms, trunk, and legs. Biopsy was not needed in this case; if performed, it would have shown characteristic changes (eg, parakeratosis, fusing of rete ridges).
These changes occur because psoriasis, an autoimmune disease, targets keratinocytes—cells that regenerate over a 28-day period to replace the outer layer of skin as it flakes off. With psoriasis, this process is accelerated; keratinocytes move and slough off within a week, creating scaly, inflamed lesions.
Long-term follow-up is needed to monitor the patient’s overall health, because psoriasis increases comorbidity of conditions such as coronary vessel disease, diabetes, stroke, and cancer. In patients with any form of psoriasis, there is the potential for psoriatic arthropathy, a destructive, crippling form of arthropathy that affects up to 25% of all patients. And in a third of guttate psoriasis cases, the condition evolves into permanent plaque psoriasis. For this reason, the guttate variety must be treated aggressively with a combination of phototherapy and topical steroids, adequate treatment of any residual strep, and, in adults, the occasional addition of methotrexate.
Regarding the family’s concern, measles does not involve scaly papules and plaques and would likely cause other constitutional symptoms (eg, fever, malaise, myalgia). The other item in the differential, tinea corporis, involves lesions that are not as uniformly scaly, numerous, or evenly spaced. And with tinea, scaling typically occurs on the peripheral leading edge of the lesion.
TAKE-HOME LEARNING POINTS
- Guttate psoriasis is more common in children than in adults and is often triggered by strep infection, though stress has also been implicated as a trigger.
- In about a third of all cases, this type evolves into permanent plaque psoriasis. All patients with psoriasis are at risk for psoriatic arthropathy (25% of patients).
- Measles does not involve scaly papules and plaques and would likely cause constitutional symptoms (eg, fever, malaise, myalgia).
- Tinea corporis lesions are not uniformly scaly, nor would they be as numerous or evenly spaced; most of the scale in tinea is seen on the peripheral leading edge of the lesion.
CAR T-cell therapy approved to treat lymphomas
The US Food and Drug Administration (FDA) has approved axicabtagene ciloleucel (Yescarta™, formerly KTE-C19) for use in adults with relapsed or refractory large B-cell lymphoma who have received 2 or more lines of systemic therapy.
Axicabtagene ciloleucel is the first chimeric antigen receptor (CAR) T-cell therapy approved to treat lymphomas.
The approval encompasses diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and transformed follicular lymphoma.
Axicabtagene ciloleucel is not approved to treat primary central nervous system lymphoma.
The FDA’s approval of axicabtagene ciloleucel was based on results from the phase 2 ZUMA-1 trial. Updated results from this trial were presented at the AACR Annual Meeting 2017.
Risks
Axicabtagene ciloleucel has a Boxed Warning in its product label noting that the therapy can cause cytokine release syndrome (CRS) and neurologic toxicities. Full prescribing information for axicabtagene ciloleucel is available at https://www.yescarta.com/.
Because of the risk of CRS and neurologic toxicities, axicabtagene ciloleucel was approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use. The FDA is requiring that hospitals and clinics that dispense axicabtagene ciloleucel be specially certified.
As part of that certification, staff who prescribe, dispense, or administer axicabtagene ciloleucel are required to be trained to recognize and manage CRS and nervous system toxicities. In addition, patients must be informed of the potential serious side effects associated with axicabtagene ciloleucel and of the importance of promptly returning to the treatment site if side effects develop.
Additional information about the REMS program can be found at https://www.yescartarems.com/.
To further evaluate the long-term safety of axicabtagene ciloleucel, the FDA is requiring the manufacturer—Kite, a Gilead company—to conduct a post-marketing observational study of patients treated with axicabtagene ciloleucel.
Access and cost
The list price of axicabtagene ciloleucel is $373,000.
The product will be manufactured in Kite’s commercial manufacturing facility in El Segundo, California.
In 2017, Kite established a multi-disciplinary field team focused on providing education and logistics training for medical centers. Now, this team has provided final site certification to 16 centers, enabling them to make axicabtagene ciloleucel available to appropriate patients.
Kite is working to train staff at more than 30 additional centers, with an eventual target of 70 to 90 centers across the US. The latest information on authorized centers is available at https://www.yescarta.com/authorized-treatment-centers/. 
The US Food and Drug Administration (FDA) has approved axicabtagene ciloleucel (Yescarta™, formerly KTE-C19) for use in adults with relapsed or refractory large B-cell lymphoma who have received 2 or more lines of systemic therapy.
Axicabtagene ciloleucel is the first chimeric antigen receptor (CAR) T-cell therapy approved to treat lymphomas.
The approval encompasses diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and transformed follicular lymphoma.
Axicabtagene ciloleucel is not approved to treat primary central nervous system lymphoma.
The FDA’s approval of axicabtagene ciloleucel was based on results from the phase 2 ZUMA-1 trial. Updated results from this trial were presented at the AACR Annual Meeting 2017.
Risks
Axicabtagene ciloleucel has a Boxed Warning in its product label noting that the therapy can cause cytokine release syndrome (CRS) and neurologic toxicities. Full prescribing information for axicabtagene ciloleucel is available at https://www.yescarta.com/.
Because of the risk of CRS and neurologic toxicities, axicabtagene ciloleucel was approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use. The FDA is requiring that hospitals and clinics that dispense axicabtagene ciloleucel be specially certified.
As part of that certification, staff who prescribe, dispense, or administer axicabtagene ciloleucel are required to be trained to recognize and manage CRS and nervous system toxicities. In addition, patients must be informed of the potential serious side effects associated with axicabtagene ciloleucel and of the importance of promptly returning to the treatment site if side effects develop.
Additional information about the REMS program can be found at https://www.yescartarems.com/.
To further evaluate the long-term safety of axicabtagene ciloleucel, the FDA is requiring the manufacturer—Kite, a Gilead company—to conduct a post-marketing observational study of patients treated with axicabtagene ciloleucel.
Access and cost
The list price of axicabtagene ciloleucel is $373,000.
The product will be manufactured in Kite’s commercial manufacturing facility in El Segundo, California.
In 2017, Kite established a multi-disciplinary field team focused on providing education and logistics training for medical centers. Now, this team has provided final site certification to 16 centers, enabling them to make axicabtagene ciloleucel available to appropriate patients.
Kite is working to train staff at more than 30 additional centers, with an eventual target of 70 to 90 centers across the US. The latest information on authorized centers is available at https://www.yescarta.com/authorized-treatment-centers/.
The US Food and Drug Administration (FDA) has approved axicabtagene ciloleucel (Yescarta™, formerly KTE-C19) for use in adults with relapsed or refractory large B-cell lymphoma who have received 2 or more lines of systemic therapy.
Axicabtagene ciloleucel is the first chimeric antigen receptor (CAR) T-cell therapy approved to treat lymphomas.
The approval encompasses diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and transformed follicular lymphoma.
Axicabtagene ciloleucel is not approved to treat primary central nervous system lymphoma.
The FDA’s approval of axicabtagene ciloleucel was based on results from the phase 2 ZUMA-1 trial. Updated results from this trial were presented at the AACR Annual Meeting 2017.
Risks
Axicabtagene ciloleucel has a Boxed Warning in its product label noting that the therapy can cause cytokine release syndrome (CRS) and neurologic toxicities. Full prescribing information for axicabtagene ciloleucel is available at https://www.yescarta.com/.
Because of the risk of CRS and neurologic toxicities, axicabtagene ciloleucel was approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use. The FDA is requiring that hospitals and clinics that dispense axicabtagene ciloleucel be specially certified.
As part of that certification, staff who prescribe, dispense, or administer axicabtagene ciloleucel are required to be trained to recognize and manage CRS and nervous system toxicities. In addition, patients must be informed of the potential serious side effects associated with axicabtagene ciloleucel and of the importance of promptly returning to the treatment site if side effects develop.
Additional information about the REMS program can be found at https://www.yescartarems.com/.
To further evaluate the long-term safety of axicabtagene ciloleucel, the FDA is requiring the manufacturer—Kite, a Gilead company—to conduct a post-marketing observational study of patients treated with axicabtagene ciloleucel.
Access and cost
The list price of axicabtagene ciloleucel is $373,000.
The product will be manufactured in Kite’s commercial manufacturing facility in El Segundo, California.
In 2017, Kite established a multi-disciplinary field team focused on providing education and logistics training for medical centers. Now, this team has provided final site certification to 16 centers, enabling them to make axicabtagene ciloleucel available to appropriate patients.
Kite is working to train staff at more than 30 additional centers, with an eventual target of 70 to 90 centers across the US. The latest information on authorized centers is available at https://www.yescarta.com/authorized-treatment-centers/.
Cheek pain
Based on the presence of bilateral Wickham striae, the FP diagnosed oral lichen planus in this patient. If the pattern were unilateral or the patient had a history of tobacco or alcohol use, the FP’s suspicion would have turned to a diagnosis of oral leukoplakia—a precursor to squamous cell carcinoma.
A mid-potency topical steroid is a good initial treatment for oral lichen planus. Triamcinolone can be prescribed in an oral base, but because it is very thick and sticky, it is better for local application to small areas around the teeth. Other treatment options include a gel or ointment, but these are not necessarily better inside the mouth. Most patients will find the cream easier to apply, even if it doesn’t taste good. If a mid-potency steroid doesn’t work, it is possible to use a high-potency steroid and change the vehicle according to the patient’s preference.
The FP in this case prescribed topical triamcinolone 0.1% cream to be applied twice daily to the buccal mucosa. At follow-up one month later, the patient’s cheeks no longer hurt, and the white Wickham striae were less visible. The FP instructed the patient to continue using the topical steroid twice daily as needed, and to return if her condition worsened.
Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Kraft RL, Usatine R. Lichen planus. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013: 901-909.
To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/
You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com
Based on the presence of bilateral Wickham striae, the FP diagnosed oral lichen planus in this patient. If the pattern were unilateral or the patient had a history of tobacco or alcohol use, the FP’s suspicion would have turned to a diagnosis of oral leukoplakia—a precursor to squamous cell carcinoma.
A mid-potency topical steroid is a good initial treatment for oral lichen planus. Triamcinolone can be prescribed in an oral base, but because it is very thick and sticky, it is better for local application to small areas around the teeth. Other treatment options include a gel or ointment, but these are not necessarily better inside the mouth. Most patients will find the cream easier to apply, even if it doesn’t taste good. If a mid-potency steroid doesn’t work, it is possible to use a high-potency steroid and change the vehicle according to the patient’s preference.
The FP in this case prescribed topical triamcinolone 0.1% cream to be applied twice daily to the buccal mucosa. At follow-up one month later, the patient’s cheeks no longer hurt, and the white Wickham striae were less visible. The FP instructed the patient to continue using the topical steroid twice daily as needed, and to return if her condition worsened.
Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Kraft RL, Usatine R. Lichen planus. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013: 901-909.
To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/
You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com
Based on the presence of bilateral Wickham striae, the FP diagnosed oral lichen planus in this patient. If the pattern were unilateral or the patient had a history of tobacco or alcohol use, the FP’s suspicion would have turned to a diagnosis of oral leukoplakia—a precursor to squamous cell carcinoma.
A mid-potency topical steroid is a good initial treatment for oral lichen planus. Triamcinolone can be prescribed in an oral base, but because it is very thick and sticky, it is better for local application to small areas around the teeth. Other treatment options include a gel or ointment, but these are not necessarily better inside the mouth. Most patients will find the cream easier to apply, even if it doesn’t taste good. If a mid-potency steroid doesn’t work, it is possible to use a high-potency steroid and change the vehicle according to the patient’s preference.
The FP in this case prescribed topical triamcinolone 0.1% cream to be applied twice daily to the buccal mucosa. At follow-up one month later, the patient’s cheeks no longer hurt, and the white Wickham striae were less visible. The FP instructed the patient to continue using the topical steroid twice daily as needed, and to return if her condition worsened.
Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Kraft RL, Usatine R. Lichen planus. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013: 901-909.
To learn more about the Color Atlas of Family Medicine, see: www.amazon.com/Color-Family-Medicine-Richard-Usatine/dp/0071769641/
You can now get the second edition of the Color Atlas of Family Medicine as an app by clicking on this link: usatinemedia.com
