Hard road disproving that statins make you dumb

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The impact of lipid-lowering drugs on patients’ mental states was on the minds of many attendees at the American College of Cardiology’s annual meeting in March.

The highest-profile report came from EBBINGHAUS (Evaluating PCSK9 Binding Antibody Influence on Cognitive Health in High Cardiovascular Risk Subjects), a substudy of the FOURIER (Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk) trial, the meeting’s blockbuster. For the first time, it proved that profoundly lowering low density lipoprotein cholesterol with the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor evolocumab led to a significant reduction in adverse clinical events. EBBINGHAUS focused on about 2,000 of the 27,000 FOURIER patients and subjected equal numbers of placebo and evolocumab patients to a battery of cognitive and memory tests over a median of 20 months. The results showed no hint of a decrement in brain function in the patients taking evolocumab, compared with either their baseline state or the controls who received placebo.

Mitchel L. Zoler/Frontline Medical News
Dr. Sandra J. Lewis
The reason why the researchers who designed FOURIER also ran EBBINGHAUS was that statins, the established lipid-lowering drugs, have received a bad rap, with alleged memory and cognitive side effects. As I reported in my news story on EBBINGHAUS, such clinicians as Sandra J. Lewis, MD, were concerned that patients were claiming that statins “make them dumb” on an almost daily basis. That’s a perception that she and others at the meeting attributed to Internet posts about statins that are filled with pseudoscience and horror tales. “We need your help to combat Dr. Google, who has a lot of statin misinformation,” pleaded Robert P. Giugliano, MD, lead investigator of EBBINGHAUS, during a press conference for his study.

That perception wasn’t helped when, in 2012, the Food and Drug Administration required the labels of all statins to include a reference to postmarketing reports of cognitive side effects such as memory impairment and confusion. The current label for one statin says: “There have been rare postmarketing reports of cognitive impairment (e.g., memory loss, forgetfulness, amnesia, memory impairment, confusion) associated with statin use. These cognitive issues have been reported for all statins.”

Mitchel L. Zoler/Frontline Medical News
Dr. Robert P. Giugliano
When the FDA issued its requirement in 2012, the rationale it presented could be objectively judged as modest at best. The agency said that “postmarketing adverse event reports generally described individuals over the age of 50 years who experienced notable, but ill-defined, memory loss or impairment that was reversible upon discontinuation of statin therapy. Time to onset of the event was highly variable, ranging from 1 day to years after statin exposure. The cases did not appear to be associated with fixed or progressive dementia, such as Alzheimer’s disease. The review did not reveal an association between the adverse event and the specific statin, the age of the individual, the statin dose, or concomitant medication use.” The FDA statement on the evidence behind its move added that “data from the observational studies and clinical trials did not suggest that cognitive changes associated with statin use are common or lead to clinically significant cognitive decline.”

Following the FDA’s action, a series of analyses appeared that reviewed the evidence and found nothing to substantiate the concern. For example, a 2012 review done in direct response to the FDA labeling change looked at case reports, observational studies, and randomized trials and found “no convincing evidence for change in cognitive function” with statin use (J Am Coll Cardiol. 2012 Sept 4;60[10]:875-81). A 2015 meta-analysis that reviewed 14 studies with cognitive testing on more than 27,000 people randomized to either a statin or placebo also found no evidence for a statin effect on mental function (J Gen Intern Med. 2015 March;30[3]:348-58). “Given these results, it is questionable whether the FDA class warning about potential cognitive adverse effects of statins is still warranted,” the meta-analysis authors concluded.

Despite this, concerns about the impact of statins on cognition and memory linger for many patients, witness the anecdotal experiences of clinicians at the meeting. This led a team of researchers at the University of Connecticut and Hartford Hospital to try to directly address the controversy. They also reported their findings at the ACC meeting.

They ran their study as part of a larger trial, STOMP (Effect of Statins on Skeletal Muscle Function and Performance), which randomized 420 healthy and statin-naive individuals to 6 months of treatment with 80 mg atorvastatin or placebo (Circulation. 2013 Jan 2;127[1]:96-103). Their memory substudy included 66 people from the atorvastatin group and 84 placebo-treated controls who averaged 49 years old. Participants underwent a battery of eight memory, cognitive, attention, and executive function tests after 6 months on treatment and again 2 months after statin treatment stopped.

Mitchel L. Zoler/Frontline Medical News
Dr. Beth A. Taylor
“We saw what many other [statin] studies saw: minimal effects in both groups,” reported Beth A. Taylor, PhD, director of exercise physiology research at Hartford (Conn.) Hospital at the meeting. The linchpin of the test battery was the Cognitive Failures Questionnaire. “No matter how we looked at the results [from this test] we saw no differences between the atorvastatin and placebo groups for cognitive failures,” Dr. Taylor said.

She and her associates took testing a step further and used an assessment never before applied to people taking statins. They ran functional MRIs on a subgroup of the participants while they took two additional memory tests at the end of 6 months on atorvastatin and again 2 months after atorvastatin stopped. They ran MRI scans during a figural memory task test on 42 placebo participants and 35 atorvastatin patients and during a Sternberg Task to test short-term memory on 68 people from the placebo group and 52 who received atorvastatin.

The functional MRI results showed some small but statistically significant changes during both tests in patterns of regional neural activation among those in the statin groups while on and off statins and also when compared with those who received placebo, but Dr. Taylor stressed that her group saw MRI differences between the statin and placebo subjects not only when people were on atorvastatin but also when they had been off the drug for 2 months. She also cautioned that “the clinical implications of the findings are unclear.”

Overall, the entire study’s results showed “no convincing evidence of measurable verbal or nonverbal memory dysfunction” linked with statin use, but Dr. Taylor also noted that the study was relatively small.

Mitchel L. Zoler/Frontline Medical News
Dr. Neil J. Stone
Speaking as a discussant, Neil J. Stone, MD, who thoroughly reviewed the statin literature as chair of the American College of Cardiology/American Heart Association panel that issued the most recent U.S. guidelines for cholesterol treatment to reduce cardiovascular disease risk, noted that Dr. Taylor’s findings agreed with what his panel found: No signal exists for an effect of statin on cognition and memory. He also highlighted the challenge of looking for cognitive and memory effects that might be caused by statins in older people who already might have age-related memory problems or may have memory or cognitive impairments triggered by other drugs. “There are a lot of variables, with possible neurogenic causes, systemic causes, and exogenous causes of memory and cognitive changes,” Dr. Stone said.

Proving the absence of a problem is always difficult. Adding Dr. Taylor’s new evidence to the case that statins really are safe when it comes to cognition and memory will undoubtedly fail to convince committed skeptics.

 

 

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The impact of lipid-lowering drugs on patients’ mental states was on the minds of many attendees at the American College of Cardiology’s annual meeting in March.

The highest-profile report came from EBBINGHAUS (Evaluating PCSK9 Binding Antibody Influence on Cognitive Health in High Cardiovascular Risk Subjects), a substudy of the FOURIER (Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk) trial, the meeting’s blockbuster. For the first time, it proved that profoundly lowering low density lipoprotein cholesterol with the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor evolocumab led to a significant reduction in adverse clinical events. EBBINGHAUS focused on about 2,000 of the 27,000 FOURIER patients and subjected equal numbers of placebo and evolocumab patients to a battery of cognitive and memory tests over a median of 20 months. The results showed no hint of a decrement in brain function in the patients taking evolocumab, compared with either their baseline state or the controls who received placebo.

Mitchel L. Zoler/Frontline Medical News
Dr. Sandra J. Lewis
The reason why the researchers who designed FOURIER also ran EBBINGHAUS was that statins, the established lipid-lowering drugs, have received a bad rap, with alleged memory and cognitive side effects. As I reported in my news story on EBBINGHAUS, such clinicians as Sandra J. Lewis, MD, were concerned that patients were claiming that statins “make them dumb” on an almost daily basis. That’s a perception that she and others at the meeting attributed to Internet posts about statins that are filled with pseudoscience and horror tales. “We need your help to combat Dr. Google, who has a lot of statin misinformation,” pleaded Robert P. Giugliano, MD, lead investigator of EBBINGHAUS, during a press conference for his study.

That perception wasn’t helped when, in 2012, the Food and Drug Administration required the labels of all statins to include a reference to postmarketing reports of cognitive side effects such as memory impairment and confusion. The current label for one statin says: “There have been rare postmarketing reports of cognitive impairment (e.g., memory loss, forgetfulness, amnesia, memory impairment, confusion) associated with statin use. These cognitive issues have been reported for all statins.”

Mitchel L. Zoler/Frontline Medical News
Dr. Robert P. Giugliano
When the FDA issued its requirement in 2012, the rationale it presented could be objectively judged as modest at best. The agency said that “postmarketing adverse event reports generally described individuals over the age of 50 years who experienced notable, but ill-defined, memory loss or impairment that was reversible upon discontinuation of statin therapy. Time to onset of the event was highly variable, ranging from 1 day to years after statin exposure. The cases did not appear to be associated with fixed or progressive dementia, such as Alzheimer’s disease. The review did not reveal an association between the adverse event and the specific statin, the age of the individual, the statin dose, or concomitant medication use.” The FDA statement on the evidence behind its move added that “data from the observational studies and clinical trials did not suggest that cognitive changes associated with statin use are common or lead to clinically significant cognitive decline.”

Following the FDA’s action, a series of analyses appeared that reviewed the evidence and found nothing to substantiate the concern. For example, a 2012 review done in direct response to the FDA labeling change looked at case reports, observational studies, and randomized trials and found “no convincing evidence for change in cognitive function” with statin use (J Am Coll Cardiol. 2012 Sept 4;60[10]:875-81). A 2015 meta-analysis that reviewed 14 studies with cognitive testing on more than 27,000 people randomized to either a statin or placebo also found no evidence for a statin effect on mental function (J Gen Intern Med. 2015 March;30[3]:348-58). “Given these results, it is questionable whether the FDA class warning about potential cognitive adverse effects of statins is still warranted,” the meta-analysis authors concluded.

Despite this, concerns about the impact of statins on cognition and memory linger for many patients, witness the anecdotal experiences of clinicians at the meeting. This led a team of researchers at the University of Connecticut and Hartford Hospital to try to directly address the controversy. They also reported their findings at the ACC meeting.

They ran their study as part of a larger trial, STOMP (Effect of Statins on Skeletal Muscle Function and Performance), which randomized 420 healthy and statin-naive individuals to 6 months of treatment with 80 mg atorvastatin or placebo (Circulation. 2013 Jan 2;127[1]:96-103). Their memory substudy included 66 people from the atorvastatin group and 84 placebo-treated controls who averaged 49 years old. Participants underwent a battery of eight memory, cognitive, attention, and executive function tests after 6 months on treatment and again 2 months after statin treatment stopped.

Mitchel L. Zoler/Frontline Medical News
Dr. Beth A. Taylor
“We saw what many other [statin] studies saw: minimal effects in both groups,” reported Beth A. Taylor, PhD, director of exercise physiology research at Hartford (Conn.) Hospital at the meeting. The linchpin of the test battery was the Cognitive Failures Questionnaire. “No matter how we looked at the results [from this test] we saw no differences between the atorvastatin and placebo groups for cognitive failures,” Dr. Taylor said.

She and her associates took testing a step further and used an assessment never before applied to people taking statins. They ran functional MRIs on a subgroup of the participants while they took two additional memory tests at the end of 6 months on atorvastatin and again 2 months after atorvastatin stopped. They ran MRI scans during a figural memory task test on 42 placebo participants and 35 atorvastatin patients and during a Sternberg Task to test short-term memory on 68 people from the placebo group and 52 who received atorvastatin.

The functional MRI results showed some small but statistically significant changes during both tests in patterns of regional neural activation among those in the statin groups while on and off statins and also when compared with those who received placebo, but Dr. Taylor stressed that her group saw MRI differences between the statin and placebo subjects not only when people were on atorvastatin but also when they had been off the drug for 2 months. She also cautioned that “the clinical implications of the findings are unclear.”

Overall, the entire study’s results showed “no convincing evidence of measurable verbal or nonverbal memory dysfunction” linked with statin use, but Dr. Taylor also noted that the study was relatively small.

Mitchel L. Zoler/Frontline Medical News
Dr. Neil J. Stone
Speaking as a discussant, Neil J. Stone, MD, who thoroughly reviewed the statin literature as chair of the American College of Cardiology/American Heart Association panel that issued the most recent U.S. guidelines for cholesterol treatment to reduce cardiovascular disease risk, noted that Dr. Taylor’s findings agreed with what his panel found: No signal exists for an effect of statin on cognition and memory. He also highlighted the challenge of looking for cognitive and memory effects that might be caused by statins in older people who already might have age-related memory problems or may have memory or cognitive impairments triggered by other drugs. “There are a lot of variables, with possible neurogenic causes, systemic causes, and exogenous causes of memory and cognitive changes,” Dr. Stone said.

Proving the absence of a problem is always difficult. Adding Dr. Taylor’s new evidence to the case that statins really are safe when it comes to cognition and memory will undoubtedly fail to convince committed skeptics.

 

 

 

The impact of lipid-lowering drugs on patients’ mental states was on the minds of many attendees at the American College of Cardiology’s annual meeting in March.

The highest-profile report came from EBBINGHAUS (Evaluating PCSK9 Binding Antibody Influence on Cognitive Health in High Cardiovascular Risk Subjects), a substudy of the FOURIER (Further Cardiovascular Outcomes Research With PCSK9 Inhibition in Subjects With Elevated Risk) trial, the meeting’s blockbuster. For the first time, it proved that profoundly lowering low density lipoprotein cholesterol with the proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor evolocumab led to a significant reduction in adverse clinical events. EBBINGHAUS focused on about 2,000 of the 27,000 FOURIER patients and subjected equal numbers of placebo and evolocumab patients to a battery of cognitive and memory tests over a median of 20 months. The results showed no hint of a decrement in brain function in the patients taking evolocumab, compared with either their baseline state or the controls who received placebo.

Mitchel L. Zoler/Frontline Medical News
Dr. Sandra J. Lewis
The reason why the researchers who designed FOURIER also ran EBBINGHAUS was that statins, the established lipid-lowering drugs, have received a bad rap, with alleged memory and cognitive side effects. As I reported in my news story on EBBINGHAUS, such clinicians as Sandra J. Lewis, MD, were concerned that patients were claiming that statins “make them dumb” on an almost daily basis. That’s a perception that she and others at the meeting attributed to Internet posts about statins that are filled with pseudoscience and horror tales. “We need your help to combat Dr. Google, who has a lot of statin misinformation,” pleaded Robert P. Giugliano, MD, lead investigator of EBBINGHAUS, during a press conference for his study.

That perception wasn’t helped when, in 2012, the Food and Drug Administration required the labels of all statins to include a reference to postmarketing reports of cognitive side effects such as memory impairment and confusion. The current label for one statin says: “There have been rare postmarketing reports of cognitive impairment (e.g., memory loss, forgetfulness, amnesia, memory impairment, confusion) associated with statin use. These cognitive issues have been reported for all statins.”

Mitchel L. Zoler/Frontline Medical News
Dr. Robert P. Giugliano
When the FDA issued its requirement in 2012, the rationale it presented could be objectively judged as modest at best. The agency said that “postmarketing adverse event reports generally described individuals over the age of 50 years who experienced notable, but ill-defined, memory loss or impairment that was reversible upon discontinuation of statin therapy. Time to onset of the event was highly variable, ranging from 1 day to years after statin exposure. The cases did not appear to be associated with fixed or progressive dementia, such as Alzheimer’s disease. The review did not reveal an association between the adverse event and the specific statin, the age of the individual, the statin dose, or concomitant medication use.” The FDA statement on the evidence behind its move added that “data from the observational studies and clinical trials did not suggest that cognitive changes associated with statin use are common or lead to clinically significant cognitive decline.”

Following the FDA’s action, a series of analyses appeared that reviewed the evidence and found nothing to substantiate the concern. For example, a 2012 review done in direct response to the FDA labeling change looked at case reports, observational studies, and randomized trials and found “no convincing evidence for change in cognitive function” with statin use (J Am Coll Cardiol. 2012 Sept 4;60[10]:875-81). A 2015 meta-analysis that reviewed 14 studies with cognitive testing on more than 27,000 people randomized to either a statin or placebo also found no evidence for a statin effect on mental function (J Gen Intern Med. 2015 March;30[3]:348-58). “Given these results, it is questionable whether the FDA class warning about potential cognitive adverse effects of statins is still warranted,” the meta-analysis authors concluded.

Despite this, concerns about the impact of statins on cognition and memory linger for many patients, witness the anecdotal experiences of clinicians at the meeting. This led a team of researchers at the University of Connecticut and Hartford Hospital to try to directly address the controversy. They also reported their findings at the ACC meeting.

They ran their study as part of a larger trial, STOMP (Effect of Statins on Skeletal Muscle Function and Performance), which randomized 420 healthy and statin-naive individuals to 6 months of treatment with 80 mg atorvastatin or placebo (Circulation. 2013 Jan 2;127[1]:96-103). Their memory substudy included 66 people from the atorvastatin group and 84 placebo-treated controls who averaged 49 years old. Participants underwent a battery of eight memory, cognitive, attention, and executive function tests after 6 months on treatment and again 2 months after statin treatment stopped.

Mitchel L. Zoler/Frontline Medical News
Dr. Beth A. Taylor
“We saw what many other [statin] studies saw: minimal effects in both groups,” reported Beth A. Taylor, PhD, director of exercise physiology research at Hartford (Conn.) Hospital at the meeting. The linchpin of the test battery was the Cognitive Failures Questionnaire. “No matter how we looked at the results [from this test] we saw no differences between the atorvastatin and placebo groups for cognitive failures,” Dr. Taylor said.

She and her associates took testing a step further and used an assessment never before applied to people taking statins. They ran functional MRIs on a subgroup of the participants while they took two additional memory tests at the end of 6 months on atorvastatin and again 2 months after atorvastatin stopped. They ran MRI scans during a figural memory task test on 42 placebo participants and 35 atorvastatin patients and during a Sternberg Task to test short-term memory on 68 people from the placebo group and 52 who received atorvastatin.

The functional MRI results showed some small but statistically significant changes during both tests in patterns of regional neural activation among those in the statin groups while on and off statins and also when compared with those who received placebo, but Dr. Taylor stressed that her group saw MRI differences between the statin and placebo subjects not only when people were on atorvastatin but also when they had been off the drug for 2 months. She also cautioned that “the clinical implications of the findings are unclear.”

Overall, the entire study’s results showed “no convincing evidence of measurable verbal or nonverbal memory dysfunction” linked with statin use, but Dr. Taylor also noted that the study was relatively small.

Mitchel L. Zoler/Frontline Medical News
Dr. Neil J. Stone
Speaking as a discussant, Neil J. Stone, MD, who thoroughly reviewed the statin literature as chair of the American College of Cardiology/American Heart Association panel that issued the most recent U.S. guidelines for cholesterol treatment to reduce cardiovascular disease risk, noted that Dr. Taylor’s findings agreed with what his panel found: No signal exists for an effect of statin on cognition and memory. He also highlighted the challenge of looking for cognitive and memory effects that might be caused by statins in older people who already might have age-related memory problems or may have memory or cognitive impairments triggered by other drugs. “There are a lot of variables, with possible neurogenic causes, systemic causes, and exogenous causes of memory and cognitive changes,” Dr. Stone said.

Proving the absence of a problem is always difficult. Adding Dr. Taylor’s new evidence to the case that statins really are safe when it comes to cognition and memory will undoubtedly fail to convince committed skeptics.

 

 

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VA Secretary Shulkin Calls for New Powers to Fire VA Employees

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Thu, 01/25/2018 - 11:45
In the wake of a VA employee caught watching pornography while with a patient, VA asks for faster removal of VA problem employees.

Citing his inability to immediately remove a VA employee who was caught watching pornography while with a patient, VA Secretary David J. Shulkin, MD, has called on Congress to enact legislation that makes it easier and faster to remove employees at the VA. “This is an example of why we need accountability legislation as soon as possible,” Dr. Shulkin said in a statement. “It’s unacceptable that VA has to wait 30 days to act on a proposed removal.”

Currently, VA employees receive at least 30 days notice of firing, have a right to a grievance hearing, and must be paid throughout the final adjudication as long as there is no evidence of a crime. However, employees can be removed from patient interaction or placed on administrative leave.

In March, the House of Representatives passed the VA Accountability First Act of 2017 (HR 1259), which would reduce the advanced warning time to 10 days and speed up the appeals process, but the bill has yet to be considered by Senate. “This situation underscores the need for Congress to get VA accountability legislation to President Trump's desk, and I thank Secretary Shulkin for making this a top priority,” Rep. David P. “Phil” Roe, MD (R-Tenn.), chairman of the House Veterans’ Affairs Committee concurred. “I was proud my bill, the VA Accountability First Act of 2017, passed the House with bipartisan support earlier this month. Veterans deserve better. I encourage my Senate colleagues to consider my legislation, and I look forward to working with Secretary Shulkin to change the way VA does business.”

Despite the bipartisan support cited by Rep. Roe, many Democrats have indicated that they are wary of the bill because it strips VA civil servant employees of many employment protections. Ranking Democratic House committee on Veterans’ Affairs member Rep. Tim Walz (D-Minn) warned that the changes in employee protections in the bill make it less likely it will pass through the Senate and that it could face court challenges. “By refusing to compromise on the 1 percent of this legislation we disagree on, Republicans have made it harder to pass the 99 percent of the legislation that is vital to making improvements,” according to a report in Stars and Stripes.

Organizations that represent VA workers also have voiced opposition. The American Federation of Government Employees (AFGE), which represents 270,000 VA employees, also argued that the bill “would render useless” the process that providers use “to protect their voice at work and defend themselves against managers’ retaliation and discrimination.” In a letter to the House committee, AFGE argued that the bill, “weakens the critical protections that VA employees need to speak up against mismanagement and patient harm.”

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In the wake of a VA employee caught watching pornography while with a patient, VA asks for faster removal of VA problem employees.
In the wake of a VA employee caught watching pornography while with a patient, VA asks for faster removal of VA problem employees.

Citing his inability to immediately remove a VA employee who was caught watching pornography while with a patient, VA Secretary David J. Shulkin, MD, has called on Congress to enact legislation that makes it easier and faster to remove employees at the VA. “This is an example of why we need accountability legislation as soon as possible,” Dr. Shulkin said in a statement. “It’s unacceptable that VA has to wait 30 days to act on a proposed removal.”

Currently, VA employees receive at least 30 days notice of firing, have a right to a grievance hearing, and must be paid throughout the final adjudication as long as there is no evidence of a crime. However, employees can be removed from patient interaction or placed on administrative leave.

In March, the House of Representatives passed the VA Accountability First Act of 2017 (HR 1259), which would reduce the advanced warning time to 10 days and speed up the appeals process, but the bill has yet to be considered by Senate. “This situation underscores the need for Congress to get VA accountability legislation to President Trump's desk, and I thank Secretary Shulkin for making this a top priority,” Rep. David P. “Phil” Roe, MD (R-Tenn.), chairman of the House Veterans’ Affairs Committee concurred. “I was proud my bill, the VA Accountability First Act of 2017, passed the House with bipartisan support earlier this month. Veterans deserve better. I encourage my Senate colleagues to consider my legislation, and I look forward to working with Secretary Shulkin to change the way VA does business.”

Despite the bipartisan support cited by Rep. Roe, many Democrats have indicated that they are wary of the bill because it strips VA civil servant employees of many employment protections. Ranking Democratic House committee on Veterans’ Affairs member Rep. Tim Walz (D-Minn) warned that the changes in employee protections in the bill make it less likely it will pass through the Senate and that it could face court challenges. “By refusing to compromise on the 1 percent of this legislation we disagree on, Republicans have made it harder to pass the 99 percent of the legislation that is vital to making improvements,” according to a report in Stars and Stripes.

Organizations that represent VA workers also have voiced opposition. The American Federation of Government Employees (AFGE), which represents 270,000 VA employees, also argued that the bill “would render useless” the process that providers use “to protect their voice at work and defend themselves against managers’ retaliation and discrimination.” In a letter to the House committee, AFGE argued that the bill, “weakens the critical protections that VA employees need to speak up against mismanagement and patient harm.”

Citing his inability to immediately remove a VA employee who was caught watching pornography while with a patient, VA Secretary David J. Shulkin, MD, has called on Congress to enact legislation that makes it easier and faster to remove employees at the VA. “This is an example of why we need accountability legislation as soon as possible,” Dr. Shulkin said in a statement. “It’s unacceptable that VA has to wait 30 days to act on a proposed removal.”

Currently, VA employees receive at least 30 days notice of firing, have a right to a grievance hearing, and must be paid throughout the final adjudication as long as there is no evidence of a crime. However, employees can be removed from patient interaction or placed on administrative leave.

In March, the House of Representatives passed the VA Accountability First Act of 2017 (HR 1259), which would reduce the advanced warning time to 10 days and speed up the appeals process, but the bill has yet to be considered by Senate. “This situation underscores the need for Congress to get VA accountability legislation to President Trump's desk, and I thank Secretary Shulkin for making this a top priority,” Rep. David P. “Phil” Roe, MD (R-Tenn.), chairman of the House Veterans’ Affairs Committee concurred. “I was proud my bill, the VA Accountability First Act of 2017, passed the House with bipartisan support earlier this month. Veterans deserve better. I encourage my Senate colleagues to consider my legislation, and I look forward to working with Secretary Shulkin to change the way VA does business.”

Despite the bipartisan support cited by Rep. Roe, many Democrats have indicated that they are wary of the bill because it strips VA civil servant employees of many employment protections. Ranking Democratic House committee on Veterans’ Affairs member Rep. Tim Walz (D-Minn) warned that the changes in employee protections in the bill make it less likely it will pass through the Senate and that it could face court challenges. “By refusing to compromise on the 1 percent of this legislation we disagree on, Republicans have made it harder to pass the 99 percent of the legislation that is vital to making improvements,” according to a report in Stars and Stripes.

Organizations that represent VA workers also have voiced opposition. The American Federation of Government Employees (AFGE), which represents 270,000 VA employees, also argued that the bill “would render useless” the process that providers use “to protect their voice at work and defend themselves against managers’ retaliation and discrimination.” In a letter to the House committee, AFGE argued that the bill, “weakens the critical protections that VA employees need to speak up against mismanagement and patient harm.”

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Strontium, ketamine target troublesome itch

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Fri, 01/18/2019 - 16:40

 

– Two drugs that target ion channels in nerves are being used to quiet neurogenic itch.

The powerful anesthetic ketamine and the element strontium have both been formulated into topical compounds that do very well in quelling itches that have been stubbornly resistant to other therapies, Gil Yosipovitch, MD, said at the annual meeting of the American Academy of Dermatology.

Dr. Gil Yosipovitch
Strontium is a calcimimetic that blocks calcium ion channels. It’s been formulated into a 4% gel, which performed very well in two studies, said Dr. Yosipovitch of the University of Miami. “Two double-blind, vehicle-controlled studies demonstrated a reduction in nonhistaminergic, cowhage-induced pruritus. Both showed that strontium 4% was superior to both 2% diphenhydramine and 1% hydrocortisone.”

Both studies employed a 4% strontium hydrogel that is available over the counter (TriCalm). The product is designed to alleviate skin irritation (itching, burning, or stinging sensations), according to the manufacturer’s website.

The first study, published in 2013, comprised 32 healthy subjects in whom itch was induced with cowhage before and after skin treatment with the strontium gel, a control vehicle, topical 1% hydrocortisone, and topical 2% diphenhydramine (Acta Derm Venereol. 2013 Sep 4;93[5]:520-6).

Strontium significantly reduced the peak intensity and duration of itch relative to all three of the comparators.

A confirmatory study was published in 2015. The vehicle-controlled, randomized, crossover study recorded cowhage-induced itch intensity and duration in 48 healthy subjects before and after skin treatment with TriCalm, 2% diphenhydramine, 1% hydrocortisone, and hydrogel vehicle (Clin Cosmet Investig Dermatol. 2015 Apr 24;8:223-9). The results were similar, Dr. Yosipovitch said.

TriCalm effectively reduced peak itch intensity by about 3 points on a visual analog scale – a 41% reduction. Itch duration was reduced by 40%. These results were both clinically and statistically significantly better than those achieved by the other active comparators and the vehicle control.

Dr. Yosipovitch said the gel is most effective on nonhistaminergic itches, including those with a neurogenic component, nummular eczema, and facial itch.

“The most powerful antipruritic we have seen in the last 3 years, however, is topical ketamine,” Dr. Yosipovitch said. Typically formulated in 2%-10% creams, the anesthetic is usually combined with amitriptyline and lidocaine. “I see this as the most effective topical antipruritic and antinociceptive we have been using.”

Ketamine is an antagonist of the n-methyl-D-aspartate (NMDA) glutamate receptor and an ion channel protein. Amitriptyline serves primarily as a voltage-gated sodium channel antagonist, and lidocaine as a local anesthetic.

Mark Davis, MD, professor of dermatology at the Mayo Clinic, Rochester, Minn., and associates initially investigated 0.5% ketamine in a topical combination with amitriptyline 1% in a cream. The compound was remarkably effective for a 41-year-old man with a recalcitrant case of brachioradial pruritus – a neuropathic condition characterized by upper-extremity itching (JAMA Dermatol. 2013;149[2]:148-50). The patient had already failed treatment with halobetasol propionate, pimecrolimus, capsaicin, doxepin hydrochloride creams, and oral hydroxyzine hydrochloride and desloratadine. However, he had complete resolution of the itch soon after using the combination cream two to three times daily. At last follow-up, 4 years later, he was still using it at least once daily and continued to obtain complete relief.

Dr. Yosipovitch said this case was followed by a retrospective study of 16 patients who had used the 0.5% ketamine cream with either 1% or 2% amitriptyline for recalcitrant pruritus. The etiologies included neurodermatitis, pruritus caused by postherpetic neuralgia, nostalgia paresthetica, anesthesia dolorosa, nasal pruritus, and diabetic neuropathy (J Am Acad Dermatol. 2013 Aug;69[2]:320-1).

They used the medication one to five times per day for a mean duration of 10 months. Of the 16 patients, two had complete relief; two had substantial relief; six had some relief; five had no relief; and one reported increased itching.

Most recently, Dr. Yosipovitch and associates reported the results of a retrospective case review of 96 patients with a variety of pruritic conditions. The most frequent indications were neuropathic conditions (29%) and prurigo nodularis (19%). Most patients got a compounded cream of 10% ketamine, 5% amitriptyline, and 2% lidocaine;16 patients got a compound with 5% ketamine. The medication worked quickly, providing itch relief within a median of about 4 minutes, with an average of about a 50% decrease in itch rating (J Am Acad Dermatol. 2017 Apr;76[4]:760-1).

Forty patients participated in a pharmacy-administered telephone survey that assessed medication tolerability and efficacy. Of these, 23 patients (58%) had relief “to a great extent” and 14 (35%) “to a moderate extent.”

There were mild side effects (burning and redness at the application site) in 16 patients. “We attributed this mainly to the lidocaine component,” Dr. Yosipovitch said. “Itch reduction lasted from 30 minutes to 7 hours, so we think this is quite a powerful tool. I now often use this topical for patients with severe intractable itch.”

He added that a case report of encephalopathy associated with the cream has recently surfaced. The patient was an elderly man with Parkinson’s disease who had been using 10% ketamine compounded with amitriptyline and lidocaine for 4 days. He gradually increased the use until he was applying it onto almost all of his upper body. The day after this extensive application, the patient presented to an emergency department with slurred speech, ataxia, and altered mental status (JAMA Dermatol. 2016;152[12]:1390-1).

“So a word of warning here: I don’t recommend using it all over the body,” Dr. Yosipovitch said.

Dr. Yosipovitch has financial relationships with numerous companies that are investigating antipruritic compounds, including strontium.
 

 

 

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– Two drugs that target ion channels in nerves are being used to quiet neurogenic itch.

The powerful anesthetic ketamine and the element strontium have both been formulated into topical compounds that do very well in quelling itches that have been stubbornly resistant to other therapies, Gil Yosipovitch, MD, said at the annual meeting of the American Academy of Dermatology.

Dr. Gil Yosipovitch
Strontium is a calcimimetic that blocks calcium ion channels. It’s been formulated into a 4% gel, which performed very well in two studies, said Dr. Yosipovitch of the University of Miami. “Two double-blind, vehicle-controlled studies demonstrated a reduction in nonhistaminergic, cowhage-induced pruritus. Both showed that strontium 4% was superior to both 2% diphenhydramine and 1% hydrocortisone.”

Both studies employed a 4% strontium hydrogel that is available over the counter (TriCalm). The product is designed to alleviate skin irritation (itching, burning, or stinging sensations), according to the manufacturer’s website.

The first study, published in 2013, comprised 32 healthy subjects in whom itch was induced with cowhage before and after skin treatment with the strontium gel, a control vehicle, topical 1% hydrocortisone, and topical 2% diphenhydramine (Acta Derm Venereol. 2013 Sep 4;93[5]:520-6).

Strontium significantly reduced the peak intensity and duration of itch relative to all three of the comparators.

A confirmatory study was published in 2015. The vehicle-controlled, randomized, crossover study recorded cowhage-induced itch intensity and duration in 48 healthy subjects before and after skin treatment with TriCalm, 2% diphenhydramine, 1% hydrocortisone, and hydrogel vehicle (Clin Cosmet Investig Dermatol. 2015 Apr 24;8:223-9). The results were similar, Dr. Yosipovitch said.

TriCalm effectively reduced peak itch intensity by about 3 points on a visual analog scale – a 41% reduction. Itch duration was reduced by 40%. These results were both clinically and statistically significantly better than those achieved by the other active comparators and the vehicle control.

Dr. Yosipovitch said the gel is most effective on nonhistaminergic itches, including those with a neurogenic component, nummular eczema, and facial itch.

“The most powerful antipruritic we have seen in the last 3 years, however, is topical ketamine,” Dr. Yosipovitch said. Typically formulated in 2%-10% creams, the anesthetic is usually combined with amitriptyline and lidocaine. “I see this as the most effective topical antipruritic and antinociceptive we have been using.”

Ketamine is an antagonist of the n-methyl-D-aspartate (NMDA) glutamate receptor and an ion channel protein. Amitriptyline serves primarily as a voltage-gated sodium channel antagonist, and lidocaine as a local anesthetic.

Mark Davis, MD, professor of dermatology at the Mayo Clinic, Rochester, Minn., and associates initially investigated 0.5% ketamine in a topical combination with amitriptyline 1% in a cream. The compound was remarkably effective for a 41-year-old man with a recalcitrant case of brachioradial pruritus – a neuropathic condition characterized by upper-extremity itching (JAMA Dermatol. 2013;149[2]:148-50). The patient had already failed treatment with halobetasol propionate, pimecrolimus, capsaicin, doxepin hydrochloride creams, and oral hydroxyzine hydrochloride and desloratadine. However, he had complete resolution of the itch soon after using the combination cream two to three times daily. At last follow-up, 4 years later, he was still using it at least once daily and continued to obtain complete relief.

Dr. Yosipovitch said this case was followed by a retrospective study of 16 patients who had used the 0.5% ketamine cream with either 1% or 2% amitriptyline for recalcitrant pruritus. The etiologies included neurodermatitis, pruritus caused by postherpetic neuralgia, nostalgia paresthetica, anesthesia dolorosa, nasal pruritus, and diabetic neuropathy (J Am Acad Dermatol. 2013 Aug;69[2]:320-1).

They used the medication one to five times per day for a mean duration of 10 months. Of the 16 patients, two had complete relief; two had substantial relief; six had some relief; five had no relief; and one reported increased itching.

Most recently, Dr. Yosipovitch and associates reported the results of a retrospective case review of 96 patients with a variety of pruritic conditions. The most frequent indications were neuropathic conditions (29%) and prurigo nodularis (19%). Most patients got a compounded cream of 10% ketamine, 5% amitriptyline, and 2% lidocaine;16 patients got a compound with 5% ketamine. The medication worked quickly, providing itch relief within a median of about 4 minutes, with an average of about a 50% decrease in itch rating (J Am Acad Dermatol. 2017 Apr;76[4]:760-1).

Forty patients participated in a pharmacy-administered telephone survey that assessed medication tolerability and efficacy. Of these, 23 patients (58%) had relief “to a great extent” and 14 (35%) “to a moderate extent.”

There were mild side effects (burning and redness at the application site) in 16 patients. “We attributed this mainly to the lidocaine component,” Dr. Yosipovitch said. “Itch reduction lasted from 30 minutes to 7 hours, so we think this is quite a powerful tool. I now often use this topical for patients with severe intractable itch.”

He added that a case report of encephalopathy associated with the cream has recently surfaced. The patient was an elderly man with Parkinson’s disease who had been using 10% ketamine compounded with amitriptyline and lidocaine for 4 days. He gradually increased the use until he was applying it onto almost all of his upper body. The day after this extensive application, the patient presented to an emergency department with slurred speech, ataxia, and altered mental status (JAMA Dermatol. 2016;152[12]:1390-1).

“So a word of warning here: I don’t recommend using it all over the body,” Dr. Yosipovitch said.

Dr. Yosipovitch has financial relationships with numerous companies that are investigating antipruritic compounds, including strontium.
 

 

 

 

– Two drugs that target ion channels in nerves are being used to quiet neurogenic itch.

The powerful anesthetic ketamine and the element strontium have both been formulated into topical compounds that do very well in quelling itches that have been stubbornly resistant to other therapies, Gil Yosipovitch, MD, said at the annual meeting of the American Academy of Dermatology.

Dr. Gil Yosipovitch
Strontium is a calcimimetic that blocks calcium ion channels. It’s been formulated into a 4% gel, which performed very well in two studies, said Dr. Yosipovitch of the University of Miami. “Two double-blind, vehicle-controlled studies demonstrated a reduction in nonhistaminergic, cowhage-induced pruritus. Both showed that strontium 4% was superior to both 2% diphenhydramine and 1% hydrocortisone.”

Both studies employed a 4% strontium hydrogel that is available over the counter (TriCalm). The product is designed to alleviate skin irritation (itching, burning, or stinging sensations), according to the manufacturer’s website.

The first study, published in 2013, comprised 32 healthy subjects in whom itch was induced with cowhage before and after skin treatment with the strontium gel, a control vehicle, topical 1% hydrocortisone, and topical 2% diphenhydramine (Acta Derm Venereol. 2013 Sep 4;93[5]:520-6).

Strontium significantly reduced the peak intensity and duration of itch relative to all three of the comparators.

A confirmatory study was published in 2015. The vehicle-controlled, randomized, crossover study recorded cowhage-induced itch intensity and duration in 48 healthy subjects before and after skin treatment with TriCalm, 2% diphenhydramine, 1% hydrocortisone, and hydrogel vehicle (Clin Cosmet Investig Dermatol. 2015 Apr 24;8:223-9). The results were similar, Dr. Yosipovitch said.

TriCalm effectively reduced peak itch intensity by about 3 points on a visual analog scale – a 41% reduction. Itch duration was reduced by 40%. These results were both clinically and statistically significantly better than those achieved by the other active comparators and the vehicle control.

Dr. Yosipovitch said the gel is most effective on nonhistaminergic itches, including those with a neurogenic component, nummular eczema, and facial itch.

“The most powerful antipruritic we have seen in the last 3 years, however, is topical ketamine,” Dr. Yosipovitch said. Typically formulated in 2%-10% creams, the anesthetic is usually combined with amitriptyline and lidocaine. “I see this as the most effective topical antipruritic and antinociceptive we have been using.”

Ketamine is an antagonist of the n-methyl-D-aspartate (NMDA) glutamate receptor and an ion channel protein. Amitriptyline serves primarily as a voltage-gated sodium channel antagonist, and lidocaine as a local anesthetic.

Mark Davis, MD, professor of dermatology at the Mayo Clinic, Rochester, Minn., and associates initially investigated 0.5% ketamine in a topical combination with amitriptyline 1% in a cream. The compound was remarkably effective for a 41-year-old man with a recalcitrant case of brachioradial pruritus – a neuropathic condition characterized by upper-extremity itching (JAMA Dermatol. 2013;149[2]:148-50). The patient had already failed treatment with halobetasol propionate, pimecrolimus, capsaicin, doxepin hydrochloride creams, and oral hydroxyzine hydrochloride and desloratadine. However, he had complete resolution of the itch soon after using the combination cream two to three times daily. At last follow-up, 4 years later, he was still using it at least once daily and continued to obtain complete relief.

Dr. Yosipovitch said this case was followed by a retrospective study of 16 patients who had used the 0.5% ketamine cream with either 1% or 2% amitriptyline for recalcitrant pruritus. The etiologies included neurodermatitis, pruritus caused by postherpetic neuralgia, nostalgia paresthetica, anesthesia dolorosa, nasal pruritus, and diabetic neuropathy (J Am Acad Dermatol. 2013 Aug;69[2]:320-1).

They used the medication one to five times per day for a mean duration of 10 months. Of the 16 patients, two had complete relief; two had substantial relief; six had some relief; five had no relief; and one reported increased itching.

Most recently, Dr. Yosipovitch and associates reported the results of a retrospective case review of 96 patients with a variety of pruritic conditions. The most frequent indications were neuropathic conditions (29%) and prurigo nodularis (19%). Most patients got a compounded cream of 10% ketamine, 5% amitriptyline, and 2% lidocaine;16 patients got a compound with 5% ketamine. The medication worked quickly, providing itch relief within a median of about 4 minutes, with an average of about a 50% decrease in itch rating (J Am Acad Dermatol. 2017 Apr;76[4]:760-1).

Forty patients participated in a pharmacy-administered telephone survey that assessed medication tolerability and efficacy. Of these, 23 patients (58%) had relief “to a great extent” and 14 (35%) “to a moderate extent.”

There were mild side effects (burning and redness at the application site) in 16 patients. “We attributed this mainly to the lidocaine component,” Dr. Yosipovitch said. “Itch reduction lasted from 30 minutes to 7 hours, so we think this is quite a powerful tool. I now often use this topical for patients with severe intractable itch.”

He added that a case report of encephalopathy associated with the cream has recently surfaced. The patient was an elderly man with Parkinson’s disease who had been using 10% ketamine compounded with amitriptyline and lidocaine for 4 days. He gradually increased the use until he was applying it onto almost all of his upper body. The day after this extensive application, the patient presented to an emergency department with slurred speech, ataxia, and altered mental status (JAMA Dermatol. 2016;152[12]:1390-1).

“So a word of warning here: I don’t recommend using it all over the body,” Dr. Yosipovitch said.

Dr. Yosipovitch has financial relationships with numerous companies that are investigating antipruritic compounds, including strontium.
 

 

 

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ABIM turns MOC page with open-book 2-year exams

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SAN DIEGO – The way the president of the American Board of Internal Medicine, Richard J. Baron, MD, sees it, maintenance of certification is more important than ever, because trust in the medical profession “is under assault right now in all kinds of ways.”

So, to help “bring clarity to uncertainty,” ABIM is continuing its makeover of the maintenance of certification (MOC) process. Beginning in 2018, an open-book option to test every 2 years will be available for physicians who are certified in internal medicine and for those in the subspecialty of nephrology. These options become available to gastroenterologists in 2019.

Both the 10-year long-form assessment and the shorter 2-year assessment options will be open book, “meaning physicians will have access to an online reference while they’re taking the exam,” said Yul D. Ejnes, MD, who is a member of ABIM’s board of directors and serves on the ABIM’s internal medicine specialty board.

Known as the “Knowledge Check-In,” the 2-year assessment is a shorter, “lower stakes” option that can be taken at home, in an office, or at a testing facility. The check-ins will be scheduled 4-6 times per year, with 10-year exams remaining available twice per year. The open-book 2-year assessments will be about 3 hours in length.

“It’s a more continuous way of learning and assessing, because the way we’ll do feedback is going to change,” explained Dr. Ejnes, who practices in Cranston, R.I. “Specifically, you’ll know right away whether you were successful or not with the assessment, as opposed to having to wait a couple of months, which happens with the 10-year assessment. Then you’ll get more feedback later helping to identify areas where you may be a little weaker and need to work out things.”

“It remains to be seen whether this new system is an improvement for GI learners. AGA’s educators will compare the changes offered by ABIM against our principles for MOC reform,” said Timothy C. Wang, MD, AGAF, President of AGA. “Reforming the MOC process is a high and long-standing priority for AGA. We have pushed ABIM to offer a system that reflects the realities of practice and how adults learn – and we’ll continue to fight for these principles.”

In general, physicians will need to either take the 2-year assessments or pass the 10-year assessment within 10 years of their last pass of the 10-year exam. Those who fail two successive 2-year assessments will have to take the 10-year exam. However, unsuccessful performance on the 2-year assessment in 2018 will not have a negative impact on certification or MOC participation status.

“It won’t count as one of the two opportunities you have before you have to go to the 10-year exam,” Dr. Ejnes said. “It allows people to try it out and lets us learn from what happens and do whatever we need to do to make things better.”

Why a 2-year period instead of a 5-year option, for example? A shorter time frame will allow the ABIM to move to a more modular approach to test material, Dr. Ejnes explained. For now, the 2-year assessments will be breadth-of-discipline exams.

Physicians whose certification expires in 2017 will need to take the 10-year exam – as Dr. Ejnes noted he himself was forced to do. “You cannot wait until 2018,” he cautioned. “That’s important, because if you let your certification lapse, you can’t enter the certification pathway. The prerequisite is that you need to be in good standing with your certification.”

The open-book Knowledge Check-Ins and 10-year assessments are slated to expand to eight subspecialties in 2019 and nine more in 2020.

Linking MOC and trust

Speaking at the annual meeting of the American College of Physicians, Dr. Baron said that false and misleading information circulated widely on Facebook and other social media channels runs the gamut of health issues, from falsified studies about purported links between vaccines and autism and public health scares on impostor websites, to stories of miracle cures for any number of ailments.

“It’s not just vaccines people are questioning,” said Dr. Baron, ABIM’s president and CEO. “There are erosions of trust in government, and there’s the tenacity and power of wildly inaccurate information. You will be dealing with patients who tenaciously believe things that you know not to be true. You will need to find ways to build trust, credibility, and relationships based on their trusting that what you’re saying is really in their interest.”

U.S. physicians aren’t secure in the shaky trust landscape. In fact, globally, the United States ranks 24th in public trust level of physicians by country (N. Eng. J. Med. 2014 Oct 23;371[17]:1570-2).

“The confidence in the medical system today is lower than the confidence in police or in small business,” Dr. Baron said. “That’s [the view] people are bringing into your offices every day. I don’t think we can assume that deference and trust are given to doctors, that the privileged role that society affords us is something that we’re going to have forever. We all have to think how trust is built in the new world.”

 

 

Will patients value MOC?

During a question and answer session at the ACP session, Anne Cummings, MD, an internist who practices in Greenbrae, Calif., asked the ABIM for support in educating the general public about what it means to be treated by a board-certified physician.

“I had a naturopath tell me the other day that she had the same training as I had,” Dr. Cummings said. “I was floored, but I think that patients don’t know the difference [between board-certified and not board-certified].”

Dr. Baron agreed ABIM needs to do more to promote the value of certification among patients. But he also called on board-certified physicians to deliver the value message directly to their own patients.

Other attendees recommended that ABIM expand the number of ways physicians can earn MOC points, and they expressed concern about the time MOC takes away from their daily practice.

For regular updates on the MOC process, physicians can subscribe to the ABIM’s blog at transforming.abim.org.

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SAN DIEGO – The way the president of the American Board of Internal Medicine, Richard J. Baron, MD, sees it, maintenance of certification is more important than ever, because trust in the medical profession “is under assault right now in all kinds of ways.”

So, to help “bring clarity to uncertainty,” ABIM is continuing its makeover of the maintenance of certification (MOC) process. Beginning in 2018, an open-book option to test every 2 years will be available for physicians who are certified in internal medicine and for those in the subspecialty of nephrology. These options become available to gastroenterologists in 2019.

Both the 10-year long-form assessment and the shorter 2-year assessment options will be open book, “meaning physicians will have access to an online reference while they’re taking the exam,” said Yul D. Ejnes, MD, who is a member of ABIM’s board of directors and serves on the ABIM’s internal medicine specialty board.

Known as the “Knowledge Check-In,” the 2-year assessment is a shorter, “lower stakes” option that can be taken at home, in an office, or at a testing facility. The check-ins will be scheduled 4-6 times per year, with 10-year exams remaining available twice per year. The open-book 2-year assessments will be about 3 hours in length.

“It’s a more continuous way of learning and assessing, because the way we’ll do feedback is going to change,” explained Dr. Ejnes, who practices in Cranston, R.I. “Specifically, you’ll know right away whether you were successful or not with the assessment, as opposed to having to wait a couple of months, which happens with the 10-year assessment. Then you’ll get more feedback later helping to identify areas where you may be a little weaker and need to work out things.”

“It remains to be seen whether this new system is an improvement for GI learners. AGA’s educators will compare the changes offered by ABIM against our principles for MOC reform,” said Timothy C. Wang, MD, AGAF, President of AGA. “Reforming the MOC process is a high and long-standing priority for AGA. We have pushed ABIM to offer a system that reflects the realities of practice and how adults learn – and we’ll continue to fight for these principles.”

In general, physicians will need to either take the 2-year assessments or pass the 10-year assessment within 10 years of their last pass of the 10-year exam. Those who fail two successive 2-year assessments will have to take the 10-year exam. However, unsuccessful performance on the 2-year assessment in 2018 will not have a negative impact on certification or MOC participation status.

“It won’t count as one of the two opportunities you have before you have to go to the 10-year exam,” Dr. Ejnes said. “It allows people to try it out and lets us learn from what happens and do whatever we need to do to make things better.”

Why a 2-year period instead of a 5-year option, for example? A shorter time frame will allow the ABIM to move to a more modular approach to test material, Dr. Ejnes explained. For now, the 2-year assessments will be breadth-of-discipline exams.

Physicians whose certification expires in 2017 will need to take the 10-year exam – as Dr. Ejnes noted he himself was forced to do. “You cannot wait until 2018,” he cautioned. “That’s important, because if you let your certification lapse, you can’t enter the certification pathway. The prerequisite is that you need to be in good standing with your certification.”

The open-book Knowledge Check-Ins and 10-year assessments are slated to expand to eight subspecialties in 2019 and nine more in 2020.

Linking MOC and trust

Speaking at the annual meeting of the American College of Physicians, Dr. Baron said that false and misleading information circulated widely on Facebook and other social media channels runs the gamut of health issues, from falsified studies about purported links between vaccines and autism and public health scares on impostor websites, to stories of miracle cures for any number of ailments.

“It’s not just vaccines people are questioning,” said Dr. Baron, ABIM’s president and CEO. “There are erosions of trust in government, and there’s the tenacity and power of wildly inaccurate information. You will be dealing with patients who tenaciously believe things that you know not to be true. You will need to find ways to build trust, credibility, and relationships based on their trusting that what you’re saying is really in their interest.”

U.S. physicians aren’t secure in the shaky trust landscape. In fact, globally, the United States ranks 24th in public trust level of physicians by country (N. Eng. J. Med. 2014 Oct 23;371[17]:1570-2).

“The confidence in the medical system today is lower than the confidence in police or in small business,” Dr. Baron said. “That’s [the view] people are bringing into your offices every day. I don’t think we can assume that deference and trust are given to doctors, that the privileged role that society affords us is something that we’re going to have forever. We all have to think how trust is built in the new world.”

 

 

Will patients value MOC?

During a question and answer session at the ACP session, Anne Cummings, MD, an internist who practices in Greenbrae, Calif., asked the ABIM for support in educating the general public about what it means to be treated by a board-certified physician.

“I had a naturopath tell me the other day that she had the same training as I had,” Dr. Cummings said. “I was floored, but I think that patients don’t know the difference [between board-certified and not board-certified].”

Dr. Baron agreed ABIM needs to do more to promote the value of certification among patients. But he also called on board-certified physicians to deliver the value message directly to their own patients.

Other attendees recommended that ABIM expand the number of ways physicians can earn MOC points, and they expressed concern about the time MOC takes away from their daily practice.

For regular updates on the MOC process, physicians can subscribe to the ABIM’s blog at transforming.abim.org.

 

SAN DIEGO – The way the president of the American Board of Internal Medicine, Richard J. Baron, MD, sees it, maintenance of certification is more important than ever, because trust in the medical profession “is under assault right now in all kinds of ways.”

So, to help “bring clarity to uncertainty,” ABIM is continuing its makeover of the maintenance of certification (MOC) process. Beginning in 2018, an open-book option to test every 2 years will be available for physicians who are certified in internal medicine and for those in the subspecialty of nephrology. These options become available to gastroenterologists in 2019.

Both the 10-year long-form assessment and the shorter 2-year assessment options will be open book, “meaning physicians will have access to an online reference while they’re taking the exam,” said Yul D. Ejnes, MD, who is a member of ABIM’s board of directors and serves on the ABIM’s internal medicine specialty board.

Known as the “Knowledge Check-In,” the 2-year assessment is a shorter, “lower stakes” option that can be taken at home, in an office, or at a testing facility. The check-ins will be scheduled 4-6 times per year, with 10-year exams remaining available twice per year. The open-book 2-year assessments will be about 3 hours in length.

“It’s a more continuous way of learning and assessing, because the way we’ll do feedback is going to change,” explained Dr. Ejnes, who practices in Cranston, R.I. “Specifically, you’ll know right away whether you were successful or not with the assessment, as opposed to having to wait a couple of months, which happens with the 10-year assessment. Then you’ll get more feedback later helping to identify areas where you may be a little weaker and need to work out things.”

“It remains to be seen whether this new system is an improvement for GI learners. AGA’s educators will compare the changes offered by ABIM against our principles for MOC reform,” said Timothy C. Wang, MD, AGAF, President of AGA. “Reforming the MOC process is a high and long-standing priority for AGA. We have pushed ABIM to offer a system that reflects the realities of practice and how adults learn – and we’ll continue to fight for these principles.”

In general, physicians will need to either take the 2-year assessments or pass the 10-year assessment within 10 years of their last pass of the 10-year exam. Those who fail two successive 2-year assessments will have to take the 10-year exam. However, unsuccessful performance on the 2-year assessment in 2018 will not have a negative impact on certification or MOC participation status.

“It won’t count as one of the two opportunities you have before you have to go to the 10-year exam,” Dr. Ejnes said. “It allows people to try it out and lets us learn from what happens and do whatever we need to do to make things better.”

Why a 2-year period instead of a 5-year option, for example? A shorter time frame will allow the ABIM to move to a more modular approach to test material, Dr. Ejnes explained. For now, the 2-year assessments will be breadth-of-discipline exams.

Physicians whose certification expires in 2017 will need to take the 10-year exam – as Dr. Ejnes noted he himself was forced to do. “You cannot wait until 2018,” he cautioned. “That’s important, because if you let your certification lapse, you can’t enter the certification pathway. The prerequisite is that you need to be in good standing with your certification.”

The open-book Knowledge Check-Ins and 10-year assessments are slated to expand to eight subspecialties in 2019 and nine more in 2020.

Linking MOC and trust

Speaking at the annual meeting of the American College of Physicians, Dr. Baron said that false and misleading information circulated widely on Facebook and other social media channels runs the gamut of health issues, from falsified studies about purported links between vaccines and autism and public health scares on impostor websites, to stories of miracle cures for any number of ailments.

“It’s not just vaccines people are questioning,” said Dr. Baron, ABIM’s president and CEO. “There are erosions of trust in government, and there’s the tenacity and power of wildly inaccurate information. You will be dealing with patients who tenaciously believe things that you know not to be true. You will need to find ways to build trust, credibility, and relationships based on their trusting that what you’re saying is really in their interest.”

U.S. physicians aren’t secure in the shaky trust landscape. In fact, globally, the United States ranks 24th in public trust level of physicians by country (N. Eng. J. Med. 2014 Oct 23;371[17]:1570-2).

“The confidence in the medical system today is lower than the confidence in police or in small business,” Dr. Baron said. “That’s [the view] people are bringing into your offices every day. I don’t think we can assume that deference and trust are given to doctors, that the privileged role that society affords us is something that we’re going to have forever. We all have to think how trust is built in the new world.”

 

 

Will patients value MOC?

During a question and answer session at the ACP session, Anne Cummings, MD, an internist who practices in Greenbrae, Calif., asked the ABIM for support in educating the general public about what it means to be treated by a board-certified physician.

“I had a naturopath tell me the other day that she had the same training as I had,” Dr. Cummings said. “I was floored, but I think that patients don’t know the difference [between board-certified and not board-certified].”

Dr. Baron agreed ABIM needs to do more to promote the value of certification among patients. But he also called on board-certified physicians to deliver the value message directly to their own patients.

Other attendees recommended that ABIM expand the number of ways physicians can earn MOC points, and they expressed concern about the time MOC takes away from their daily practice.

For regular updates on the MOC process, physicians can subscribe to the ABIM’s blog at transforming.abim.org.

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First treat-to-target gout trial supports allopurinol dose escalation

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Gradual dose escalation of allopurinol achieved target serum urate levels without causing excess adverse effects in patients with gout who tolerated but responded inadequately to creatinine clearance–based dosing, including in patients with chronic kidney disease, investigators reported in Annals of the Rheumatic Diseases.

 

 

Medical Illustrations, Canterbury district Health Board
Dr. Lisa Stamp
Managing serum urate levels is the cornerstone of treating gout, and international guidelines recommend targets of less than 6 mg/dL, or less than 5 mg/dL in the presence of tophi. Allopurinol is the most commonly used urate-lowering therapy worldwide, but concerns about adverse effects have inspired debates about dosing strategies for years, noted Dr. Stamp of the University of Otago, Christchurch, New Zealand. For example, the American College of Rheumatology recommends gradual, treat-to-target dose escalation of allopurinol regardless of renal status, the European League Against Rheumatism (EULAR) suggests creatinine clearance–based dosing in renally impaired patients, and the American College of Physicians (ACP) advocates for corticosteroids, nonsteroidal anti-inflammatory drugs, and colchicine over urate-lowering therapy.

To help settle these debates, Dr. Stamp and her associates recruited 183 patients with gout whose serum urate levels averaged 7.15 mg/dL (standard deviation, 1.6 mg/dL) despite at least 1 month of at least a creatinine clearance–based allopurinol dose (average, 269 mg/day). For the next 12 months, patients either continued this dose or increased it monthly until serum urate fell below 6 mg/dL (Ann Rheum Dis. 2017 March 17. doi: 10.1136/annrheumdis-2016-210872).

At month 12, 69% of dose-escalation patients and 32% of controls reached this target (P less than .001; odds ratio, 4.3; 95% confidence interval, 2.4-7.9). Serum urate levels dropped by a mean of 1.5 mg/dL with dose escalation and by 0.34 mg/dL in controls (P less than .001). Thus, dose escalation cut serum urate levels by an additional 1.2 mg/dL (95% CI, 0.67-1.5 mg/dL; P less than .001). The average final daily allopurinol dose was 413 mg (range, 0-900 mg) with dose escalation and 288 mg (0-600 mg) for controls.

Dr. Kenneth G. Saag
Gradually ramping up the dose of allopurinol did not significantly increase the risk of rash or other adverse events, compared with continuing at the lower dose, although the study was not powered to detect the rare allopurinol hypersensitivity syndrome, Dr. Stamp said. The only serious treatment-related adverse event was increased international normalized ratio (INR) in a dose-escalation patient who received warfarin after elective mitral valve replacement. There were five deaths in each study arm, none of which were considered treatment related.

Notably, renal function measures did not differ between arms. “There has been long-standing concern about whether it is safe to escalate allopurinol doses in persons with chronic kidney disease,” said Kenneth Saag, MD, of the University of Alabama at Birmingham, who was not involved in the trial. “This study contributes to the limited literature supporting the practice of treating to serum urate targets, even in patients with underlying kidney disease.”

Fully 52% of trial participants had chronic kidney disease, and 44% had tophi. “Thus, our population is representative of people with gout, represents real-life clinical practice, and the results are generalizable to other gout populations,” the investigators wrote.

The findings support the ACR recommendation to gradually titrate urate-lowering therapy while performing close laboratory monitoring, said Tuhina Neogi, MD, of Boston University, who was not involved in the study. This approach is “efficacious and relatively safe for patients who have already tolerated lower doses of allopurinol, but have not yet achieved their serum urate target,” she said. “This is akin to finding the right regimen and titrating doses of medications for patients with diabetes or hypertension in a patient-centered manner.”

Dr. Tuhina Neogi
Dr. Saag agreed. “Rheumatologists and other physicians who commonly treat gout should treat to target in all their patients,” he said. “In patients with chronic kidney disease, providers should more slowly escalate the allopurinol dose, as slowly as by 50 mg every month.”

Patients in both groups developed mild increases in liver function tests, and a few in the dose escalation group developed more pronounced rises in gamma glutamyl transferase (GGT), as previously reported in the LASSO trial (Semin Arthritis Rheum. 2015 Oct;45[2]:174-83). Allopurinol has been linked to liver enzyme abnormalities, and the GGT finding is of unclear significance, but laboratory monitoring is important, Dr. Neogi said. If primary care providers who treat gout cannot routinely measure serum urate and other relevant laboratory measures, they should consider referring patients to a rheumatologist, she added.

Gradual dose escalation meant that patients did not reach the serum urate target until month 7, Dr. Neogi added. “Since the trial was 12 months long, the time over which serum urate was less than 6 mg/dL was not long enough to demonstrate the benefits on flares and tophi,” she said. The open-label extension phase of the trial should shed more insight on these important questions, she added.

For now, providers should know that “patients with gout are not well served if their serum urate levels remain elevated, as that means they are at ongoing risk for flares and tophi,” Dr. Neogi said. “This trial provides evidence and further support for the feasibility, efficacy, and relative safety of escalating allopurinol beyond creatinine-clearance dosing to achieve this important and critical goal for gout management.”

The Health Research Council of New Zealand funded the study. Dr. Stamp and two coinvestigators disclosed grant support from the Health Research Council of New Zealand. Dr. Stamp also disclosed grants from Ardea Biosciences and the Health Research Council of New Zealand outside the submitted work, and her coinvestigators disclosed grants and personal fees from AstraZeneca, Ardea Biosciences, Takeda, and several other pharmaceutical companies. Dr. Neogi had no disclosures. Dr. Saag has received meal compensation from Eli Lilly.

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Gradual dose escalation of allopurinol achieved target serum urate levels without causing excess adverse effects in patients with gout who tolerated but responded inadequately to creatinine clearance–based dosing, including in patients with chronic kidney disease, investigators reported in Annals of the Rheumatic Diseases.

 

 

Medical Illustrations, Canterbury district Health Board
Dr. Lisa Stamp
Managing serum urate levels is the cornerstone of treating gout, and international guidelines recommend targets of less than 6 mg/dL, or less than 5 mg/dL in the presence of tophi. Allopurinol is the most commonly used urate-lowering therapy worldwide, but concerns about adverse effects have inspired debates about dosing strategies for years, noted Dr. Stamp of the University of Otago, Christchurch, New Zealand. For example, the American College of Rheumatology recommends gradual, treat-to-target dose escalation of allopurinol regardless of renal status, the European League Against Rheumatism (EULAR) suggests creatinine clearance–based dosing in renally impaired patients, and the American College of Physicians (ACP) advocates for corticosteroids, nonsteroidal anti-inflammatory drugs, and colchicine over urate-lowering therapy.

To help settle these debates, Dr. Stamp and her associates recruited 183 patients with gout whose serum urate levels averaged 7.15 mg/dL (standard deviation, 1.6 mg/dL) despite at least 1 month of at least a creatinine clearance–based allopurinol dose (average, 269 mg/day). For the next 12 months, patients either continued this dose or increased it monthly until serum urate fell below 6 mg/dL (Ann Rheum Dis. 2017 March 17. doi: 10.1136/annrheumdis-2016-210872).

At month 12, 69% of dose-escalation patients and 32% of controls reached this target (P less than .001; odds ratio, 4.3; 95% confidence interval, 2.4-7.9). Serum urate levels dropped by a mean of 1.5 mg/dL with dose escalation and by 0.34 mg/dL in controls (P less than .001). Thus, dose escalation cut serum urate levels by an additional 1.2 mg/dL (95% CI, 0.67-1.5 mg/dL; P less than .001). The average final daily allopurinol dose was 413 mg (range, 0-900 mg) with dose escalation and 288 mg (0-600 mg) for controls.

Dr. Kenneth G. Saag
Gradually ramping up the dose of allopurinol did not significantly increase the risk of rash or other adverse events, compared with continuing at the lower dose, although the study was not powered to detect the rare allopurinol hypersensitivity syndrome, Dr. Stamp said. The only serious treatment-related adverse event was increased international normalized ratio (INR) in a dose-escalation patient who received warfarin after elective mitral valve replacement. There were five deaths in each study arm, none of which were considered treatment related.

Notably, renal function measures did not differ between arms. “There has been long-standing concern about whether it is safe to escalate allopurinol doses in persons with chronic kidney disease,” said Kenneth Saag, MD, of the University of Alabama at Birmingham, who was not involved in the trial. “This study contributes to the limited literature supporting the practice of treating to serum urate targets, even in patients with underlying kidney disease.”

Fully 52% of trial participants had chronic kidney disease, and 44% had tophi. “Thus, our population is representative of people with gout, represents real-life clinical practice, and the results are generalizable to other gout populations,” the investigators wrote.

The findings support the ACR recommendation to gradually titrate urate-lowering therapy while performing close laboratory monitoring, said Tuhina Neogi, MD, of Boston University, who was not involved in the study. This approach is “efficacious and relatively safe for patients who have already tolerated lower doses of allopurinol, but have not yet achieved their serum urate target,” she said. “This is akin to finding the right regimen and titrating doses of medications for patients with diabetes or hypertension in a patient-centered manner.”

Dr. Tuhina Neogi
Dr. Saag agreed. “Rheumatologists and other physicians who commonly treat gout should treat to target in all their patients,” he said. “In patients with chronic kidney disease, providers should more slowly escalate the allopurinol dose, as slowly as by 50 mg every month.”

Patients in both groups developed mild increases in liver function tests, and a few in the dose escalation group developed more pronounced rises in gamma glutamyl transferase (GGT), as previously reported in the LASSO trial (Semin Arthritis Rheum. 2015 Oct;45[2]:174-83). Allopurinol has been linked to liver enzyme abnormalities, and the GGT finding is of unclear significance, but laboratory monitoring is important, Dr. Neogi said. If primary care providers who treat gout cannot routinely measure serum urate and other relevant laboratory measures, they should consider referring patients to a rheumatologist, she added.

Gradual dose escalation meant that patients did not reach the serum urate target until month 7, Dr. Neogi added. “Since the trial was 12 months long, the time over which serum urate was less than 6 mg/dL was not long enough to demonstrate the benefits on flares and tophi,” she said. The open-label extension phase of the trial should shed more insight on these important questions, she added.

For now, providers should know that “patients with gout are not well served if their serum urate levels remain elevated, as that means they are at ongoing risk for flares and tophi,” Dr. Neogi said. “This trial provides evidence and further support for the feasibility, efficacy, and relative safety of escalating allopurinol beyond creatinine-clearance dosing to achieve this important and critical goal for gout management.”

The Health Research Council of New Zealand funded the study. Dr. Stamp and two coinvestigators disclosed grant support from the Health Research Council of New Zealand. Dr. Stamp also disclosed grants from Ardea Biosciences and the Health Research Council of New Zealand outside the submitted work, and her coinvestigators disclosed grants and personal fees from AstraZeneca, Ardea Biosciences, Takeda, and several other pharmaceutical companies. Dr. Neogi had no disclosures. Dr. Saag has received meal compensation from Eli Lilly.

 

Gradual dose escalation of allopurinol achieved target serum urate levels without causing excess adverse effects in patients with gout who tolerated but responded inadequately to creatinine clearance–based dosing, including in patients with chronic kidney disease, investigators reported in Annals of the Rheumatic Diseases.

 

 

Medical Illustrations, Canterbury district Health Board
Dr. Lisa Stamp
Managing serum urate levels is the cornerstone of treating gout, and international guidelines recommend targets of less than 6 mg/dL, or less than 5 mg/dL in the presence of tophi. Allopurinol is the most commonly used urate-lowering therapy worldwide, but concerns about adverse effects have inspired debates about dosing strategies for years, noted Dr. Stamp of the University of Otago, Christchurch, New Zealand. For example, the American College of Rheumatology recommends gradual, treat-to-target dose escalation of allopurinol regardless of renal status, the European League Against Rheumatism (EULAR) suggests creatinine clearance–based dosing in renally impaired patients, and the American College of Physicians (ACP) advocates for corticosteroids, nonsteroidal anti-inflammatory drugs, and colchicine over urate-lowering therapy.

To help settle these debates, Dr. Stamp and her associates recruited 183 patients with gout whose serum urate levels averaged 7.15 mg/dL (standard deviation, 1.6 mg/dL) despite at least 1 month of at least a creatinine clearance–based allopurinol dose (average, 269 mg/day). For the next 12 months, patients either continued this dose or increased it monthly until serum urate fell below 6 mg/dL (Ann Rheum Dis. 2017 March 17. doi: 10.1136/annrheumdis-2016-210872).

At month 12, 69% of dose-escalation patients and 32% of controls reached this target (P less than .001; odds ratio, 4.3; 95% confidence interval, 2.4-7.9). Serum urate levels dropped by a mean of 1.5 mg/dL with dose escalation and by 0.34 mg/dL in controls (P less than .001). Thus, dose escalation cut serum urate levels by an additional 1.2 mg/dL (95% CI, 0.67-1.5 mg/dL; P less than .001). The average final daily allopurinol dose was 413 mg (range, 0-900 mg) with dose escalation and 288 mg (0-600 mg) for controls.

Dr. Kenneth G. Saag
Gradually ramping up the dose of allopurinol did not significantly increase the risk of rash or other adverse events, compared with continuing at the lower dose, although the study was not powered to detect the rare allopurinol hypersensitivity syndrome, Dr. Stamp said. The only serious treatment-related adverse event was increased international normalized ratio (INR) in a dose-escalation patient who received warfarin after elective mitral valve replacement. There were five deaths in each study arm, none of which were considered treatment related.

Notably, renal function measures did not differ between arms. “There has been long-standing concern about whether it is safe to escalate allopurinol doses in persons with chronic kidney disease,” said Kenneth Saag, MD, of the University of Alabama at Birmingham, who was not involved in the trial. “This study contributes to the limited literature supporting the practice of treating to serum urate targets, even in patients with underlying kidney disease.”

Fully 52% of trial participants had chronic kidney disease, and 44% had tophi. “Thus, our population is representative of people with gout, represents real-life clinical practice, and the results are generalizable to other gout populations,” the investigators wrote.

The findings support the ACR recommendation to gradually titrate urate-lowering therapy while performing close laboratory monitoring, said Tuhina Neogi, MD, of Boston University, who was not involved in the study. This approach is “efficacious and relatively safe for patients who have already tolerated lower doses of allopurinol, but have not yet achieved their serum urate target,” she said. “This is akin to finding the right regimen and titrating doses of medications for patients with diabetes or hypertension in a patient-centered manner.”

Dr. Tuhina Neogi
Dr. Saag agreed. “Rheumatologists and other physicians who commonly treat gout should treat to target in all their patients,” he said. “In patients with chronic kidney disease, providers should more slowly escalate the allopurinol dose, as slowly as by 50 mg every month.”

Patients in both groups developed mild increases in liver function tests, and a few in the dose escalation group developed more pronounced rises in gamma glutamyl transferase (GGT), as previously reported in the LASSO trial (Semin Arthritis Rheum. 2015 Oct;45[2]:174-83). Allopurinol has been linked to liver enzyme abnormalities, and the GGT finding is of unclear significance, but laboratory monitoring is important, Dr. Neogi said. If primary care providers who treat gout cannot routinely measure serum urate and other relevant laboratory measures, they should consider referring patients to a rheumatologist, she added.

Gradual dose escalation meant that patients did not reach the serum urate target until month 7, Dr. Neogi added. “Since the trial was 12 months long, the time over which serum urate was less than 6 mg/dL was not long enough to demonstrate the benefits on flares and tophi,” she said. The open-label extension phase of the trial should shed more insight on these important questions, she added.

For now, providers should know that “patients with gout are not well served if their serum urate levels remain elevated, as that means they are at ongoing risk for flares and tophi,” Dr. Neogi said. “This trial provides evidence and further support for the feasibility, efficacy, and relative safety of escalating allopurinol beyond creatinine-clearance dosing to achieve this important and critical goal for gout management.”

The Health Research Council of New Zealand funded the study. Dr. Stamp and two coinvestigators disclosed grant support from the Health Research Council of New Zealand. Dr. Stamp also disclosed grants from Ardea Biosciences and the Health Research Council of New Zealand outside the submitted work, and her coinvestigators disclosed grants and personal fees from AstraZeneca, Ardea Biosciences, Takeda, and several other pharmaceutical companies. Dr. Neogi had no disclosures. Dr. Saag has received meal compensation from Eli Lilly.

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Key clinical point: It is safe to gradually increase the dose of allopurinol to achieve serum urate targets in gout patients who tolerate the drug, even in those with chronic kidney disease.

Major finding: At month 12, 69% of dose escalation patients and 32% of controls had serum urate levels below 6 mg/dL (P less than .001). Dose escalation did not increase the risk of serious adverse effects.

Data source: A 12-month, open-label, randomized, controlled, parallel-group, comparative trial of 186 patients with gout.

Disclosures: The Health Research Council of New Zealand funded the study. Dr. Stamp and two coinvestigators disclosed grant support from Health Research Council of New Zealand. Dr. Stamp also disclosed grants from Ardea Biosciences and Health Research Council of New Zealand outside the submitted work, and her coinvestigators disclosed grants and personal fees from AstraZeneca, Ardea Biosciences, Takeda, and several other pharmaceutical companies. Dr. Neogi had no disclosures. Dr. Saag has received meal compensation from Eli Lilly.

Omalizumab effects rapid, often complete, clearance of refractory bullous pemphigoid

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Fri, 01/18/2019 - 16:40

 

– Omalizumab, a monoclonal anti-IgE antibody, may be a good option for patients with treatment-refractory bullous pemphigoid.

Patients who received omalizumab (Xolair) experienced rapid improvements, with 30%-50% lesion clearance within a week and complete clearance by 3 weeks, Kenneth Yu, MD, said at the annual meeting of the American Academy of Dermatology. With regular injections, they were kept symptom free for months. Some patients did flare, but were then easily controlled on standard treatment. Omalizumab, approved by the Food and Drug Administration in 2003, is indicated for moderate to severe persistent asthma and chronic idiopathic urticaria.

Dr. Kenneth Yu


“We have now treated six patients with omalizumab with very good results with five of them. These are not your garden-variety BP patients, but people with very treatment-resistant disease who have failed treatment with corticosteroids alone, and in combination with other immunosuppressants.”

The rapid clinical improvements, along with observations that eosinophilia decreased with treatment, “strengthen the evidence that BP is an IgE-mediated, organ-specific autoimmune disease,” said Dr. Yu, senior resident in dermatology at the University of Michigan, Ann Arbor.

“Would I use this as a first-line treatment for BP? Probably not. But if you are seeing someone who’s nonresponsive to therapy, you might want to check IgE and eosinophil levels and if those are elevated, you might consider omalizumab as an adjunct treatment – and you might observe a fairly dramatic response.”

Three of Dr. Yu’s patients received omalizumab as monotherapy, and three received it in conjunction with other immunosuppressants. He described their disease presentation, treatment, and progression.

In general, Dr. Yu reserves omalizumab for patients with refractory disease and two particular clinical characteristics: high eosinophil count and elevated serum IgE. The initial dosing is based on the asthma treatment nomogram for the drug; he titrates it according to clinical response. “We don’t alter the total dose given, but we do adjust the frequency with which we give it.”

His first patient was a 70-year-old woman with a 1-year history of poorly controlled BP; she had failed prednisone, azathioprine, and minocycline. She also had a history of steroid-related vertebral compression fractures. She presented with an eosinophil count of over 400 cells/microL.

“We treated her with subcutaneous injections of 300 mg every 2 weeks for 16 weeks,” Dr. Yu said. Within 1 week, she had a 44% reduction in blisters; within 4 weeks, she had gone from 50% body surface area involvement to 5%.

After eight injections, the patient was disease free and Dr. Yu discontinued treatment. She remained clear until week 32 after treatment initiation; she had a flare manifested by increased pruritus and recurrence of lesions. Dr. Yu restarted omalizumab and the lesions cleared within 2 weeks. From weeks 35-72, the patient received five more injections and remained disease free.

“After that, she did have another flare, so we used omalizumab again,” but without the same excellent results. “She had an initial decrease in pruritus, and symptom improvement, but her disease subsequently worsened. We restarted her on prednisone and azathioprine and she has done well.”

Dr. Yu said he made “a couple of interesting observations on this case.”

“We saw no real correlation between disease activity score, and the levels of serum IgG antibodies. But we did notice a parallel correlation with the level of eosinophil and disease severity and also treatment response,” he said. “It was quite clear that immediately after injection, she had a dramatic drop in eosinophils” from 1,600 to 60 cells/microL within 24 hours.

His next case was a 72-year-old woman with a history of somewhat controlled essential tremor, and 6 months of highly pruritic BP blistering. She had been treated with 60 mg/day prednisone, but didn’t tolerate it well, developing steroid-induced psychosis with agitation and violence, and a worsening of her tremor. The steroid was tapered to 40 mg/day and azathioprine was added, but she was did not respond to this change and continued to develop new blisters each day. She was admitted to the hospital for plasmapheresis, which was not helpful. Nor did she respond to six cycles of cyclophosphamide.

At that point, Dr. Yu drew IgE and eosinophil levels: Her absolute eosinophil count was 1,600 cells/microL and IgE was 287 units/mL. He then gave the patient 300 mg omalizumab subcutaneously.

“Ten days after a single injection, her blisters had almost completely resolved,” he said. “To briefly describe her disease course, the blisters went away, and she had resolution of her pruritus. She was discharged with 1 month of prednisone, but we tapered that and have been able to maintain her on omalizumab alone. She had one mild flare, which was readily controlled with prednisone. The last time we saw her, she was disease free.”

He also described four other steroid-refractory BP patients treated with omalizumab.“Their commonalities were that they all had steroid-refractory disease that was resistant to immunosuppressants, had a high level of IgE, and most of them also had eosinophilia.”

Dr. Yu’s descriptions:
 

 

 

• A 78-year-old woman with refractory BP of 1.5 years responded well to three initial injections spaced 6 and 4 weeks apart, and has been well maintained for 20 months with 300-mL injections administered once a month. One relapse was easily controlled.

• A 72-year-old woman with 3.5 years of refractory BP responded well to 375 mg injections every 4 weeks and has been symptom free for a year on that maintenance dose.

• A 55-year-old woman with a 7-month history of refractory BP experienced a 30% reduction in body surface area blistering within 1 week of her first 375-mg injection. By 3 weeks, she was clear. She had three injections, 2 weeks apart, and was disease free for 3 months.

• An 86-year-old woman with longstanding refractory BP experienced a 22% reduction in blister count within a week of her first 375-mL injection. After a series of injections every 2 weeks, however, she developed an exacerbation of her preexisting chronic obstructive pulmonary disease, which was due primarily to tapering her prednisone. However, she no longer uses omalizumab.

“It is difficult to make recommendations because of the limitations of our data,” Dr. Yu said. “But based on the small number of patients we have treated, I would consider using omalizumab in patients with resistant disease who have an elevated IgE and eosinophil count. The optimal dosing regimen is not yet determined. Our approach is to start out with the asthma dosing and titrate until we see improvement. We use the highest dose indicated for the patient’s weight and IgE levels, typically 300-375 mg subcutaneously every 2-8 weeks, and start tapering when the patient gets control.”

He had no financial disclosures.

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– Omalizumab, a monoclonal anti-IgE antibody, may be a good option for patients with treatment-refractory bullous pemphigoid.

Patients who received omalizumab (Xolair) experienced rapid improvements, with 30%-50% lesion clearance within a week and complete clearance by 3 weeks, Kenneth Yu, MD, said at the annual meeting of the American Academy of Dermatology. With regular injections, they were kept symptom free for months. Some patients did flare, but were then easily controlled on standard treatment. Omalizumab, approved by the Food and Drug Administration in 2003, is indicated for moderate to severe persistent asthma and chronic idiopathic urticaria.

Dr. Kenneth Yu


“We have now treated six patients with omalizumab with very good results with five of them. These are not your garden-variety BP patients, but people with very treatment-resistant disease who have failed treatment with corticosteroids alone, and in combination with other immunosuppressants.”

The rapid clinical improvements, along with observations that eosinophilia decreased with treatment, “strengthen the evidence that BP is an IgE-mediated, organ-specific autoimmune disease,” said Dr. Yu, senior resident in dermatology at the University of Michigan, Ann Arbor.

“Would I use this as a first-line treatment for BP? Probably not. But if you are seeing someone who’s nonresponsive to therapy, you might want to check IgE and eosinophil levels and if those are elevated, you might consider omalizumab as an adjunct treatment – and you might observe a fairly dramatic response.”

Three of Dr. Yu’s patients received omalizumab as monotherapy, and three received it in conjunction with other immunosuppressants. He described their disease presentation, treatment, and progression.

In general, Dr. Yu reserves omalizumab for patients with refractory disease and two particular clinical characteristics: high eosinophil count and elevated serum IgE. The initial dosing is based on the asthma treatment nomogram for the drug; he titrates it according to clinical response. “We don’t alter the total dose given, but we do adjust the frequency with which we give it.”

His first patient was a 70-year-old woman with a 1-year history of poorly controlled BP; she had failed prednisone, azathioprine, and minocycline. She also had a history of steroid-related vertebral compression fractures. She presented with an eosinophil count of over 400 cells/microL.

“We treated her with subcutaneous injections of 300 mg every 2 weeks for 16 weeks,” Dr. Yu said. Within 1 week, she had a 44% reduction in blisters; within 4 weeks, she had gone from 50% body surface area involvement to 5%.

After eight injections, the patient was disease free and Dr. Yu discontinued treatment. She remained clear until week 32 after treatment initiation; she had a flare manifested by increased pruritus and recurrence of lesions. Dr. Yu restarted omalizumab and the lesions cleared within 2 weeks. From weeks 35-72, the patient received five more injections and remained disease free.

“After that, she did have another flare, so we used omalizumab again,” but without the same excellent results. “She had an initial decrease in pruritus, and symptom improvement, but her disease subsequently worsened. We restarted her on prednisone and azathioprine and she has done well.”

Dr. Yu said he made “a couple of interesting observations on this case.”

“We saw no real correlation between disease activity score, and the levels of serum IgG antibodies. But we did notice a parallel correlation with the level of eosinophil and disease severity and also treatment response,” he said. “It was quite clear that immediately after injection, she had a dramatic drop in eosinophils” from 1,600 to 60 cells/microL within 24 hours.

His next case was a 72-year-old woman with a history of somewhat controlled essential tremor, and 6 months of highly pruritic BP blistering. She had been treated with 60 mg/day prednisone, but didn’t tolerate it well, developing steroid-induced psychosis with agitation and violence, and a worsening of her tremor. The steroid was tapered to 40 mg/day and azathioprine was added, but she was did not respond to this change and continued to develop new blisters each day. She was admitted to the hospital for plasmapheresis, which was not helpful. Nor did she respond to six cycles of cyclophosphamide.

At that point, Dr. Yu drew IgE and eosinophil levels: Her absolute eosinophil count was 1,600 cells/microL and IgE was 287 units/mL. He then gave the patient 300 mg omalizumab subcutaneously.

“Ten days after a single injection, her blisters had almost completely resolved,” he said. “To briefly describe her disease course, the blisters went away, and she had resolution of her pruritus. She was discharged with 1 month of prednisone, but we tapered that and have been able to maintain her on omalizumab alone. She had one mild flare, which was readily controlled with prednisone. The last time we saw her, she was disease free.”

He also described four other steroid-refractory BP patients treated with omalizumab.“Their commonalities were that they all had steroid-refractory disease that was resistant to immunosuppressants, had a high level of IgE, and most of them also had eosinophilia.”

Dr. Yu’s descriptions:
 

 

 

• A 78-year-old woman with refractory BP of 1.5 years responded well to three initial injections spaced 6 and 4 weeks apart, and has been well maintained for 20 months with 300-mL injections administered once a month. One relapse was easily controlled.

• A 72-year-old woman with 3.5 years of refractory BP responded well to 375 mg injections every 4 weeks and has been symptom free for a year on that maintenance dose.

• A 55-year-old woman with a 7-month history of refractory BP experienced a 30% reduction in body surface area blistering within 1 week of her first 375-mg injection. By 3 weeks, she was clear. She had three injections, 2 weeks apart, and was disease free for 3 months.

• An 86-year-old woman with longstanding refractory BP experienced a 22% reduction in blister count within a week of her first 375-mL injection. After a series of injections every 2 weeks, however, she developed an exacerbation of her preexisting chronic obstructive pulmonary disease, which was due primarily to tapering her prednisone. However, she no longer uses omalizumab.

“It is difficult to make recommendations because of the limitations of our data,” Dr. Yu said. “But based on the small number of patients we have treated, I would consider using omalizumab in patients with resistant disease who have an elevated IgE and eosinophil count. The optimal dosing regimen is not yet determined. Our approach is to start out with the asthma dosing and titrate until we see improvement. We use the highest dose indicated for the patient’s weight and IgE levels, typically 300-375 mg subcutaneously every 2-8 weeks, and start tapering when the patient gets control.”

He had no financial disclosures.

 

– Omalizumab, a monoclonal anti-IgE antibody, may be a good option for patients with treatment-refractory bullous pemphigoid.

Patients who received omalizumab (Xolair) experienced rapid improvements, with 30%-50% lesion clearance within a week and complete clearance by 3 weeks, Kenneth Yu, MD, said at the annual meeting of the American Academy of Dermatology. With regular injections, they were kept symptom free for months. Some patients did flare, but were then easily controlled on standard treatment. Omalizumab, approved by the Food and Drug Administration in 2003, is indicated for moderate to severe persistent asthma and chronic idiopathic urticaria.

Dr. Kenneth Yu


“We have now treated six patients with omalizumab with very good results with five of them. These are not your garden-variety BP patients, but people with very treatment-resistant disease who have failed treatment with corticosteroids alone, and in combination with other immunosuppressants.”

The rapid clinical improvements, along with observations that eosinophilia decreased with treatment, “strengthen the evidence that BP is an IgE-mediated, organ-specific autoimmune disease,” said Dr. Yu, senior resident in dermatology at the University of Michigan, Ann Arbor.

“Would I use this as a first-line treatment for BP? Probably not. But if you are seeing someone who’s nonresponsive to therapy, you might want to check IgE and eosinophil levels and if those are elevated, you might consider omalizumab as an adjunct treatment – and you might observe a fairly dramatic response.”

Three of Dr. Yu’s patients received omalizumab as monotherapy, and three received it in conjunction with other immunosuppressants. He described their disease presentation, treatment, and progression.

In general, Dr. Yu reserves omalizumab for patients with refractory disease and two particular clinical characteristics: high eosinophil count and elevated serum IgE. The initial dosing is based on the asthma treatment nomogram for the drug; he titrates it according to clinical response. “We don’t alter the total dose given, but we do adjust the frequency with which we give it.”

His first patient was a 70-year-old woman with a 1-year history of poorly controlled BP; she had failed prednisone, azathioprine, and minocycline. She also had a history of steroid-related vertebral compression fractures. She presented with an eosinophil count of over 400 cells/microL.

“We treated her with subcutaneous injections of 300 mg every 2 weeks for 16 weeks,” Dr. Yu said. Within 1 week, she had a 44% reduction in blisters; within 4 weeks, she had gone from 50% body surface area involvement to 5%.

After eight injections, the patient was disease free and Dr. Yu discontinued treatment. She remained clear until week 32 after treatment initiation; she had a flare manifested by increased pruritus and recurrence of lesions. Dr. Yu restarted omalizumab and the lesions cleared within 2 weeks. From weeks 35-72, the patient received five more injections and remained disease free.

“After that, she did have another flare, so we used omalizumab again,” but without the same excellent results. “She had an initial decrease in pruritus, and symptom improvement, but her disease subsequently worsened. We restarted her on prednisone and azathioprine and she has done well.”

Dr. Yu said he made “a couple of interesting observations on this case.”

“We saw no real correlation between disease activity score, and the levels of serum IgG antibodies. But we did notice a parallel correlation with the level of eosinophil and disease severity and also treatment response,” he said. “It was quite clear that immediately after injection, she had a dramatic drop in eosinophils” from 1,600 to 60 cells/microL within 24 hours.

His next case was a 72-year-old woman with a history of somewhat controlled essential tremor, and 6 months of highly pruritic BP blistering. She had been treated with 60 mg/day prednisone, but didn’t tolerate it well, developing steroid-induced psychosis with agitation and violence, and a worsening of her tremor. The steroid was tapered to 40 mg/day and azathioprine was added, but she was did not respond to this change and continued to develop new blisters each day. She was admitted to the hospital for plasmapheresis, which was not helpful. Nor did she respond to six cycles of cyclophosphamide.

At that point, Dr. Yu drew IgE and eosinophil levels: Her absolute eosinophil count was 1,600 cells/microL and IgE was 287 units/mL. He then gave the patient 300 mg omalizumab subcutaneously.

“Ten days after a single injection, her blisters had almost completely resolved,” he said. “To briefly describe her disease course, the blisters went away, and she had resolution of her pruritus. She was discharged with 1 month of prednisone, but we tapered that and have been able to maintain her on omalizumab alone. She had one mild flare, which was readily controlled with prednisone. The last time we saw her, she was disease free.”

He also described four other steroid-refractory BP patients treated with omalizumab.“Their commonalities were that they all had steroid-refractory disease that was resistant to immunosuppressants, had a high level of IgE, and most of them also had eosinophilia.”

Dr. Yu’s descriptions:
 

 

 

• A 78-year-old woman with refractory BP of 1.5 years responded well to three initial injections spaced 6 and 4 weeks apart, and has been well maintained for 20 months with 300-mL injections administered once a month. One relapse was easily controlled.

• A 72-year-old woman with 3.5 years of refractory BP responded well to 375 mg injections every 4 weeks and has been symptom free for a year on that maintenance dose.

• A 55-year-old woman with a 7-month history of refractory BP experienced a 30% reduction in body surface area blistering within 1 week of her first 375-mg injection. By 3 weeks, she was clear. She had three injections, 2 weeks apart, and was disease free for 3 months.

• An 86-year-old woman with longstanding refractory BP experienced a 22% reduction in blister count within a week of her first 375-mL injection. After a series of injections every 2 weeks, however, she developed an exacerbation of her preexisting chronic obstructive pulmonary disease, which was due primarily to tapering her prednisone. However, she no longer uses omalizumab.

“It is difficult to make recommendations because of the limitations of our data,” Dr. Yu said. “But based on the small number of patients we have treated, I would consider using omalizumab in patients with resistant disease who have an elevated IgE and eosinophil count. The optimal dosing regimen is not yet determined. Our approach is to start out with the asthma dosing and titrate until we see improvement. We use the highest dose indicated for the patient’s weight and IgE levels, typically 300-375 mg subcutaneously every 2-8 weeks, and start tapering when the patient gets control.”

He had no financial disclosures.

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Psoriasis Symptoms With the Greatest Impact on Patients

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Flaking/scaling and itching, followed by dry cracked skin that may bleed, pain or soreness, and burning/stinging were noted by psoriasis patients as the symptoms with the most significant impact on daily life in a public meeting hosted by the US Food and Drug Administration (FDA) to hear patient perspectives on psoriasis. Approximately 70 psoriasis patients or patient representatives attended the meeting in person and others attended through a live webcast.

More than two-thirds of respondents identified flaking/scaling as one of their most significant symptoms of psoriasis, either localized to psoriasis-prone areas such as the elbows and knees or more widespread. Patients reported that this symptom is constant, leaving them to absentmindedly rub certain areas of the skin.

A similar number of respondents indicated that itching was their most significant symptom. One patient called it “an intense subcutaneous itch… deep down in the skin,” a description that resonated with other patients in the room.

Nearly 40% identified dry cracked skin that may bleed as a significant symptom, noting that areas where skin is thinner are affected more, such as the folds of the body. Patients described this symptom as interrelated with other symptoms such as itching. “The thicker the scales get on my skin, the more they itch, and the more they itch, the more I am likely to scratch them, and the more I scratch them, the more they start to crack, and then more come back and it keeps going and going,” one patient said.

More than one-quarter of respondents indicated that pain, soreness, or burning/stinging were the most significant symptoms. Patients indicated that the stinging/burning was more episodic, while the pain was more constant, with the pain being under the skin.

Triggers of these symptoms included stress (primary trigger), changes in weather, hormonal changes, diet, lotions, prolonged exposure to sunlight, sweat, aging, and other medical conditions.

Dermatologists may use these patient insights to prescribe therapies that target these symptoms.

The psoriasis public meeting in March 2016 was the FDA’s 18th patient-focused drug development meeting. The FDA sought this information to have a greater understanding of the burden of psoriasis on patients and the treatments currently used to treat psoriasis and its symptoms. This information will help guide the FDA as they consider future drug approvals.

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Flaking/scaling and itching, followed by dry cracked skin that may bleed, pain or soreness, and burning/stinging were noted by psoriasis patients as the symptoms with the most significant impact on daily life in a public meeting hosted by the US Food and Drug Administration (FDA) to hear patient perspectives on psoriasis. Approximately 70 psoriasis patients or patient representatives attended the meeting in person and others attended through a live webcast.

More than two-thirds of respondents identified flaking/scaling as one of their most significant symptoms of psoriasis, either localized to psoriasis-prone areas such as the elbows and knees or more widespread. Patients reported that this symptom is constant, leaving them to absentmindedly rub certain areas of the skin.

A similar number of respondents indicated that itching was their most significant symptom. One patient called it “an intense subcutaneous itch… deep down in the skin,” a description that resonated with other patients in the room.

Nearly 40% identified dry cracked skin that may bleed as a significant symptom, noting that areas where skin is thinner are affected more, such as the folds of the body. Patients described this symptom as interrelated with other symptoms such as itching. “The thicker the scales get on my skin, the more they itch, and the more they itch, the more I am likely to scratch them, and the more I scratch them, the more they start to crack, and then more come back and it keeps going and going,” one patient said.

More than one-quarter of respondents indicated that pain, soreness, or burning/stinging were the most significant symptoms. Patients indicated that the stinging/burning was more episodic, while the pain was more constant, with the pain being under the skin.

Triggers of these symptoms included stress (primary trigger), changes in weather, hormonal changes, diet, lotions, prolonged exposure to sunlight, sweat, aging, and other medical conditions.

Dermatologists may use these patient insights to prescribe therapies that target these symptoms.

The psoriasis public meeting in March 2016 was the FDA’s 18th patient-focused drug development meeting. The FDA sought this information to have a greater understanding of the burden of psoriasis on patients and the treatments currently used to treat psoriasis and its symptoms. This information will help guide the FDA as they consider future drug approvals.

Flaking/scaling and itching, followed by dry cracked skin that may bleed, pain or soreness, and burning/stinging were noted by psoriasis patients as the symptoms with the most significant impact on daily life in a public meeting hosted by the US Food and Drug Administration (FDA) to hear patient perspectives on psoriasis. Approximately 70 psoriasis patients or patient representatives attended the meeting in person and others attended through a live webcast.

More than two-thirds of respondents identified flaking/scaling as one of their most significant symptoms of psoriasis, either localized to psoriasis-prone areas such as the elbows and knees or more widespread. Patients reported that this symptom is constant, leaving them to absentmindedly rub certain areas of the skin.

A similar number of respondents indicated that itching was their most significant symptom. One patient called it “an intense subcutaneous itch… deep down in the skin,” a description that resonated with other patients in the room.

Nearly 40% identified dry cracked skin that may bleed as a significant symptom, noting that areas where skin is thinner are affected more, such as the folds of the body. Patients described this symptom as interrelated with other symptoms such as itching. “The thicker the scales get on my skin, the more they itch, and the more they itch, the more I am likely to scratch them, and the more I scratch them, the more they start to crack, and then more come back and it keeps going and going,” one patient said.

More than one-quarter of respondents indicated that pain, soreness, or burning/stinging were the most significant symptoms. Patients indicated that the stinging/burning was more episodic, while the pain was more constant, with the pain being under the skin.

Triggers of these symptoms included stress (primary trigger), changes in weather, hormonal changes, diet, lotions, prolonged exposure to sunlight, sweat, aging, and other medical conditions.

Dermatologists may use these patient insights to prescribe therapies that target these symptoms.

The psoriasis public meeting in March 2016 was the FDA’s 18th patient-focused drug development meeting. The FDA sought this information to have a greater understanding of the burden of psoriasis on patients and the treatments currently used to treat psoriasis and its symptoms. This information will help guide the FDA as they consider future drug approvals.

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Tocilizumab shows promise for GVHD prevention

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– Tocilizumab plus standard immune suppression appears to drive down the risk for graft-versus-host disease (GVHD), according to results from a phase II study of 35 adults undergoing allogeneic stem cell transplants.

The effect was particularly pronounced for prevention of GVHD in the colon, William Drobyski, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The incidence rate of grades II-IV and III-IV acute GVHD was 12% at day 100 in patients given standard prophylaxis of tacrolimus/methotrexate (Tac/MTX) and 3% in patients given Tac/MTX plus 8 mg/kg of tocilizumab (Toc, capped at 800 mg), said Dr. Drobyski of the Medical College of Wisconsin, Milwaukee.

To provide further context to the results, Dr. Drobyski and his colleagues performed a matched case-control analysis using contemporary controls in the Center for International Blood & Marrow Transplant Research from 2000 to 2014. The same eligibility criteria used for the trial were applied to the matched controls except for the use of Tac/MTX as GVHD prophylaxis. Patients were otherwise matched based on age, performance score, disease, and donor type.

The incidence of grades II-IV acute GVHD at day 100 was significantly lower in the Toc/Tac/MTX group than in the Tac/MTX control population (12% vs. 41%). The incidence of grades III-IV acute GVHD was slightly lower with tocilizumab, but the difference between the groups was not statistically significant, Dr. Drobyski said.

The probability of grade II-IV acute GVHD–free survival, which was the primary endpoint of the study, was significantly higher in the Toc/Tac/MTX group (79% vs 52%), he said.

Five patients developed grade 2 acute GVHD of the skin or upper GI tract, and one patient died of grade 4 acute GVHD of the skin in the first 100 days. Notably, there were no cases of acute GVHD of the lower GI tract during that time, although two cases did occur between days 130 and 180, he said.

“There was no difference in transplant-related mortality, relapse, disease-free survival, or overall survival,” he said, adding that preliminary data suggest there were no differences in chronic GVHD between the groups.

Causes of death also were similar between the two cohorts with respect to disease- and transplant-related complications.

Patients in the tocilizumab study were enrolled between January 2015 and June 2016; the median age was 66 years. Diseases represented in the cohort included de novo acute myeloid leukemia (13 patients), AML (6 patients), chronic myelomonocytic leukemia (6 patients), acute lymphoblastic leukemia (4 patients), myelodysplastic syndrome (3 patients), and T-cell lymphoma, chronic myeloid leukemia, and NK/T cell lymphoma (in 1 patient each). Most patients were classified as high risk (9 patients) or intermediate risk (22 patients) by the disease risk index.

Conditioning was entirely busulfan based. Myeloablative conditioning was with busulfan and cyclophosphamide (Cytoxan) in 5 patients, or fludarabine and 4 days of busulfan in 10 patients, and reduced-intensity conditioning was with fludarabine and 2 days of busulfan in 18 patients. Transplants were with either HLA-matched related or unrelated donor grafts. Most patients (29 of 35) received peripheral stem cell grafts.

Tocilizumab, an interleuken-6 receptor blocker that is approved for treatment of rheumatoid arthritis, was administered after completion of conditioning and on the day prior to stem cell infusion.

In a pilot clinical trial of tocilizumab for the treatment of steroid-resistant acute GVHD in patients who had primarily had lower GI tract disease, “we were able to demonstrate responses in a majority of these patients,” Dr. Drobyski said, noting that a recent study presented at the 2016 annual meeting of the American Society of Hematology also showed efficacy in the treatment of lower tract GI GVHD, “providing evidence that tocilizumab had activity in acute GVHD, and perhaps in the treatment of steroid-refractory lower GI GVHD.”

Elevated IL-6 levels in the peripheral blood are correlated with an increased incidence and severity of GVHD; administration of an anti-IL-6 receptor antibody has been shown in preclinical studies to protect mice from lethal GVHD. The current open-label study was performed to “try to advance this concept” by assessing whether inhibition of IL-6 signaling could also prevent acute GVHD.

The findings confirm those of a 2014 study by Kennedy et al. in Lancet Oncology (2014;15:1451-9), and imply that tocilizumab warrants a randomized trial as prophylaxis for acute GVHD, he concluded.

Dr. Drobyski reported having no disclosures.

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– Tocilizumab plus standard immune suppression appears to drive down the risk for graft-versus-host disease (GVHD), according to results from a phase II study of 35 adults undergoing allogeneic stem cell transplants.

The effect was particularly pronounced for prevention of GVHD in the colon, William Drobyski, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The incidence rate of grades II-IV and III-IV acute GVHD was 12% at day 100 in patients given standard prophylaxis of tacrolimus/methotrexate (Tac/MTX) and 3% in patients given Tac/MTX plus 8 mg/kg of tocilizumab (Toc, capped at 800 mg), said Dr. Drobyski of the Medical College of Wisconsin, Milwaukee.

To provide further context to the results, Dr. Drobyski and his colleagues performed a matched case-control analysis using contemporary controls in the Center for International Blood & Marrow Transplant Research from 2000 to 2014. The same eligibility criteria used for the trial were applied to the matched controls except for the use of Tac/MTX as GVHD prophylaxis. Patients were otherwise matched based on age, performance score, disease, and donor type.

The incidence of grades II-IV acute GVHD at day 100 was significantly lower in the Toc/Tac/MTX group than in the Tac/MTX control population (12% vs. 41%). The incidence of grades III-IV acute GVHD was slightly lower with tocilizumab, but the difference between the groups was not statistically significant, Dr. Drobyski said.

The probability of grade II-IV acute GVHD–free survival, which was the primary endpoint of the study, was significantly higher in the Toc/Tac/MTX group (79% vs 52%), he said.

Five patients developed grade 2 acute GVHD of the skin or upper GI tract, and one patient died of grade 4 acute GVHD of the skin in the first 100 days. Notably, there were no cases of acute GVHD of the lower GI tract during that time, although two cases did occur between days 130 and 180, he said.

“There was no difference in transplant-related mortality, relapse, disease-free survival, or overall survival,” he said, adding that preliminary data suggest there were no differences in chronic GVHD between the groups.

Causes of death also were similar between the two cohorts with respect to disease- and transplant-related complications.

Patients in the tocilizumab study were enrolled between January 2015 and June 2016; the median age was 66 years. Diseases represented in the cohort included de novo acute myeloid leukemia (13 patients), AML (6 patients), chronic myelomonocytic leukemia (6 patients), acute lymphoblastic leukemia (4 patients), myelodysplastic syndrome (3 patients), and T-cell lymphoma, chronic myeloid leukemia, and NK/T cell lymphoma (in 1 patient each). Most patients were classified as high risk (9 patients) or intermediate risk (22 patients) by the disease risk index.

Conditioning was entirely busulfan based. Myeloablative conditioning was with busulfan and cyclophosphamide (Cytoxan) in 5 patients, or fludarabine and 4 days of busulfan in 10 patients, and reduced-intensity conditioning was with fludarabine and 2 days of busulfan in 18 patients. Transplants were with either HLA-matched related or unrelated donor grafts. Most patients (29 of 35) received peripheral stem cell grafts.

Tocilizumab, an interleuken-6 receptor blocker that is approved for treatment of rheumatoid arthritis, was administered after completion of conditioning and on the day prior to stem cell infusion.

In a pilot clinical trial of tocilizumab for the treatment of steroid-resistant acute GVHD in patients who had primarily had lower GI tract disease, “we were able to demonstrate responses in a majority of these patients,” Dr. Drobyski said, noting that a recent study presented at the 2016 annual meeting of the American Society of Hematology also showed efficacy in the treatment of lower tract GI GVHD, “providing evidence that tocilizumab had activity in acute GVHD, and perhaps in the treatment of steroid-refractory lower GI GVHD.”

Elevated IL-6 levels in the peripheral blood are correlated with an increased incidence and severity of GVHD; administration of an anti-IL-6 receptor antibody has been shown in preclinical studies to protect mice from lethal GVHD. The current open-label study was performed to “try to advance this concept” by assessing whether inhibition of IL-6 signaling could also prevent acute GVHD.

The findings confirm those of a 2014 study by Kennedy et al. in Lancet Oncology (2014;15:1451-9), and imply that tocilizumab warrants a randomized trial as prophylaxis for acute GVHD, he concluded.

Dr. Drobyski reported having no disclosures.

 

– Tocilizumab plus standard immune suppression appears to drive down the risk for graft-versus-host disease (GVHD), according to results from a phase II study of 35 adults undergoing allogeneic stem cell transplants.

The effect was particularly pronounced for prevention of GVHD in the colon, William Drobyski, MD, reported at the combined annual meetings of the Center for International Blood & Marrow Transplant Research and the American Society for Blood and Marrow Transplantation.

The incidence rate of grades II-IV and III-IV acute GVHD was 12% at day 100 in patients given standard prophylaxis of tacrolimus/methotrexate (Tac/MTX) and 3% in patients given Tac/MTX plus 8 mg/kg of tocilizumab (Toc, capped at 800 mg), said Dr. Drobyski of the Medical College of Wisconsin, Milwaukee.

To provide further context to the results, Dr. Drobyski and his colleagues performed a matched case-control analysis using contemporary controls in the Center for International Blood & Marrow Transplant Research from 2000 to 2014. The same eligibility criteria used for the trial were applied to the matched controls except for the use of Tac/MTX as GVHD prophylaxis. Patients were otherwise matched based on age, performance score, disease, and donor type.

The incidence of grades II-IV acute GVHD at day 100 was significantly lower in the Toc/Tac/MTX group than in the Tac/MTX control population (12% vs. 41%). The incidence of grades III-IV acute GVHD was slightly lower with tocilizumab, but the difference between the groups was not statistically significant, Dr. Drobyski said.

The probability of grade II-IV acute GVHD–free survival, which was the primary endpoint of the study, was significantly higher in the Toc/Tac/MTX group (79% vs 52%), he said.

Five patients developed grade 2 acute GVHD of the skin or upper GI tract, and one patient died of grade 4 acute GVHD of the skin in the first 100 days. Notably, there were no cases of acute GVHD of the lower GI tract during that time, although two cases did occur between days 130 and 180, he said.

“There was no difference in transplant-related mortality, relapse, disease-free survival, or overall survival,” he said, adding that preliminary data suggest there were no differences in chronic GVHD between the groups.

Causes of death also were similar between the two cohorts with respect to disease- and transplant-related complications.

Patients in the tocilizumab study were enrolled between January 2015 and June 2016; the median age was 66 years. Diseases represented in the cohort included de novo acute myeloid leukemia (13 patients), AML (6 patients), chronic myelomonocytic leukemia (6 patients), acute lymphoblastic leukemia (4 patients), myelodysplastic syndrome (3 patients), and T-cell lymphoma, chronic myeloid leukemia, and NK/T cell lymphoma (in 1 patient each). Most patients were classified as high risk (9 patients) or intermediate risk (22 patients) by the disease risk index.

Conditioning was entirely busulfan based. Myeloablative conditioning was with busulfan and cyclophosphamide (Cytoxan) in 5 patients, or fludarabine and 4 days of busulfan in 10 patients, and reduced-intensity conditioning was with fludarabine and 2 days of busulfan in 18 patients. Transplants were with either HLA-matched related or unrelated donor grafts. Most patients (29 of 35) received peripheral stem cell grafts.

Tocilizumab, an interleuken-6 receptor blocker that is approved for treatment of rheumatoid arthritis, was administered after completion of conditioning and on the day prior to stem cell infusion.

In a pilot clinical trial of tocilizumab for the treatment of steroid-resistant acute GVHD in patients who had primarily had lower GI tract disease, “we were able to demonstrate responses in a majority of these patients,” Dr. Drobyski said, noting that a recent study presented at the 2016 annual meeting of the American Society of Hematology also showed efficacy in the treatment of lower tract GI GVHD, “providing evidence that tocilizumab had activity in acute GVHD, and perhaps in the treatment of steroid-refractory lower GI GVHD.”

Elevated IL-6 levels in the peripheral blood are correlated with an increased incidence and severity of GVHD; administration of an anti-IL-6 receptor antibody has been shown in preclinical studies to protect mice from lethal GVHD. The current open-label study was performed to “try to advance this concept” by assessing whether inhibition of IL-6 signaling could also prevent acute GVHD.

The findings confirm those of a 2014 study by Kennedy et al. in Lancet Oncology (2014;15:1451-9), and imply that tocilizumab warrants a randomized trial as prophylaxis for acute GVHD, he concluded.

Dr. Drobyski reported having no disclosures.

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Key clinical point: Tocilizumab shows promising activity for preventing GVHD when added to standard immune suppression for prophylaxis in stem cell transplant patients.

Major finding: The probability of grade II-IV acute GVHD-free survival was 79% vs. 52% in the tocilizumab group vs. age-matched controls.

Data source: An open-label phase II study of 35 patients.

Disclosures: Dr. Drobyski reported having no disclosures.

Hepatitis B, C appear to raise Parkinson’s risk

Further research justified
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Hepatitis B and C appear to raise the risk of later Parkinson’s disease (PD), according to a report published in Neurology.

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The findings presented by Dr. Pakpoor and her associates justify performing deep-sequencing studies in brain tissue samples at autopsy or in cerebrospinal fluid samples from patients with PD, to detect possible links with infectious agents such as viral hepatitis.

However, for any such link to be considered conclusive, future research also must show that direct-acting antiviral therapies for chronic HCV improve PD symptoms, or epidemiology studies must demonstrate a strong association with specific hepatitis virus.
 

Julian Benito-Leon, MD, PhD, is in the department of neurology at Complutense University Hospital, Madrid. He reported having no relevant financial disclosures. Dr. Benito-Leon made these remarks in an editorial accompanying Dr. Pakpoor’s report (Neurology. 2017;88:1-2).

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The findings presented by Dr. Pakpoor and her associates justify performing deep-sequencing studies in brain tissue samples at autopsy or in cerebrospinal fluid samples from patients with PD, to detect possible links with infectious agents such as viral hepatitis.

However, for any such link to be considered conclusive, future research also must show that direct-acting antiviral therapies for chronic HCV improve PD symptoms, or epidemiology studies must demonstrate a strong association with specific hepatitis virus.
 

Julian Benito-Leon, MD, PhD, is in the department of neurology at Complutense University Hospital, Madrid. He reported having no relevant financial disclosures. Dr. Benito-Leon made these remarks in an editorial accompanying Dr. Pakpoor’s report (Neurology. 2017;88:1-2).

Body

 

The findings presented by Dr. Pakpoor and her associates justify performing deep-sequencing studies in brain tissue samples at autopsy or in cerebrospinal fluid samples from patients with PD, to detect possible links with infectious agents such as viral hepatitis.

However, for any such link to be considered conclusive, future research also must show that direct-acting antiviral therapies for chronic HCV improve PD symptoms, or epidemiology studies must demonstrate a strong association with specific hepatitis virus.
 

Julian Benito-Leon, MD, PhD, is in the department of neurology at Complutense University Hospital, Madrid. He reported having no relevant financial disclosures. Dr. Benito-Leon made these remarks in an editorial accompanying Dr. Pakpoor’s report (Neurology. 2017;88:1-2).

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Further research justified
Further research justified

 

Hepatitis B and C appear to raise the risk of later Parkinson’s disease (PD), according to a report published in Neurology.

 

Hepatitis B and C appear to raise the risk of later Parkinson’s disease (PD), according to a report published in Neurology.

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Key clinical point: Hepatitis B and C appear to raise the risk of later Parkinson’s disease.

Major finding: The risk of developing PD was significantly elevated for only 1 or more years following hospitalization for hepatitis B (RR, 1.76) or hepatitis C (RR, 1.51).

Data source: A retrospective cohort study involving 70,061 people in the general U.K. population with hepatitis B or C, 6,225 with autoimmune hepatitis, 4,234 with chronic active hepatitis, 19,870 with HIV, and 6,132,124 control subjects hospitalized during 1999-2011.

Disclosures: The English National Institute for Health Research supported the study. Dr. Pakpoor and her associates reported having no relevant financial disclosures.