The future of GI: A choice between ‘care assembly’ or skilled risk management

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BOSTON – Gastroenterology is becoming a game of risk: It’s either learn to leverage risk through the creation of advanced alternative payment methods (APM) under Medicare’s new Quality Payment Program or risk losing money through the commoditization of the field, according to an expert.

“Our culture right now is one where we get paid for making widgets,” said Lawrence Kosinski, MD, a practice councilor on the American Gastroenterological Association Governing Board and former chairman of its Practice Management and Economics Committee. He made his remarks in an interview in advance of his presentation at the 2017 AGA Tech Summit sponsored by the AGA Center for GI Innovation and Technology. “The more colonoscopies we do, the more money we make. The more we can charge for those colonoscopies and get collected, the better things are for us.”

Eli Zimmerman/Frontline Medical News
Dr. Lawrence Kosinski
However, since the goal of value-based care is to drive down the cost of services – such as by replacing colonoscopies with less expensive alternatives – under the new rules of reimbursement, the gastroenterologist’s competitive advantage is increasingly in the knowledge of how to manage complex chronic diseases.

“Over 80% of the cost of health care is for the management of chronic disease. We happen to have a very expensive set of chronic diseases that we take care of in GI, very complicated illnesses. We need to leverage the management of those patients, but we need to be able to show how our work decreases the overall cost of care so that we can get a piece of that risk premium,” he said.

In his own practice, Dr. Kosinski and his colleagues have created an APM – the first novel APM to be recommended to the Centers for Medicare & Medicaid Services for approval by the Physician-Focused Payment Model Technical Advisory Committee – that is based on better patient risk assessment, combined with earlier patient engagement.

After discovering in 2013 that one of his payers was spending $24,000 annually on patients with inflammatory bowel disease and that two-thirds of patients with inflammatory bowel disease who are admitted to the hospital had not had a CPT code issued in the 30 days before admission, Dr. Kosinski and his colleagues wanted to see if they could offer the insurer value by decreasing that hospitalization rate.

Using proprietary algorithms rooted in thorough patient risk assessment according to published guidelines for the management of patients with Crohn’s disease, they created a patient platform – coined ProjectSonar – that alerts their Crohn’s patients on their smart phones, engages them in a short survey, and provides them with instant feedback on their disease status and care needs based on their responses. Survey results are sent to the Web and to nurse case managers at the practice, who follow up with the patient accordingly.

A year-long pilot program of the patient portal with 50 people in the study population showed more than a 600% return on the cost of investment in the proprietary software, with an average of $6,000 in medical savings for test subjects who responded to texts, compared with controls who did not receive smart phone texts, for a total savings of more than half a million dollars. “All of the savings come from the patients who respond,” Dr. Kosinski said, noting that, in his practice, they now have a sustained patient response rate of more than 80% and that it helps to have the physician emphasize use of the platform to the patient.

“Patients love it. It is almost like chronic disease concierge medicine they don’t have to pay for,” Dr. Kosinski said during his presentation at the meeting, adding that the insurer likes it because it cuts costs, and physicians like it because they don’t have to take less reimbursement to help the insurer realize gains.

“We risk assess every patient, something the majority of doctors don’t do but [that] insurance companies do all the time,” Dr. Kosinski said in an interview after his presentation. “Then we apply the appropriate treatment using the scientific methods in the published guidelines. Then we analyze the data, which helps us refine our assessments and predict our costs of care in this population.”

Knowing the base cost of care for specific patient populations helps define the margin of financial risk he and his colleagues can tolerate. A gastroenterology practice that operates as a risk-bearing entity could theoretically offer to contract with affordable care organizations to manage IBD or other GI-type conditions, he said.

By learning to assess, measure, and leverage risk, gastroenterologists can become sought after for the value they provide rather than for the care they “assemble,” something Dr. Kosinski said is of rising concern as the Affordable Care Act has driven a lot of consolidation, with hospital systems buying up primary care physicians and specialists.

Otherwise, he said, “We’re just going to be commoditized proceduralists.”

Dr. Kosinski is president of SonarMDTM.
 

 

 

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BOSTON – Gastroenterology is becoming a game of risk: It’s either learn to leverage risk through the creation of advanced alternative payment methods (APM) under Medicare’s new Quality Payment Program or risk losing money through the commoditization of the field, according to an expert.

“Our culture right now is one where we get paid for making widgets,” said Lawrence Kosinski, MD, a practice councilor on the American Gastroenterological Association Governing Board and former chairman of its Practice Management and Economics Committee. He made his remarks in an interview in advance of his presentation at the 2017 AGA Tech Summit sponsored by the AGA Center for GI Innovation and Technology. “The more colonoscopies we do, the more money we make. The more we can charge for those colonoscopies and get collected, the better things are for us.”

Eli Zimmerman/Frontline Medical News
Dr. Lawrence Kosinski
However, since the goal of value-based care is to drive down the cost of services – such as by replacing colonoscopies with less expensive alternatives – under the new rules of reimbursement, the gastroenterologist’s competitive advantage is increasingly in the knowledge of how to manage complex chronic diseases.

“Over 80% of the cost of health care is for the management of chronic disease. We happen to have a very expensive set of chronic diseases that we take care of in GI, very complicated illnesses. We need to leverage the management of those patients, but we need to be able to show how our work decreases the overall cost of care so that we can get a piece of that risk premium,” he said.

In his own practice, Dr. Kosinski and his colleagues have created an APM – the first novel APM to be recommended to the Centers for Medicare & Medicaid Services for approval by the Physician-Focused Payment Model Technical Advisory Committee – that is based on better patient risk assessment, combined with earlier patient engagement.

After discovering in 2013 that one of his payers was spending $24,000 annually on patients with inflammatory bowel disease and that two-thirds of patients with inflammatory bowel disease who are admitted to the hospital had not had a CPT code issued in the 30 days before admission, Dr. Kosinski and his colleagues wanted to see if they could offer the insurer value by decreasing that hospitalization rate.

Using proprietary algorithms rooted in thorough patient risk assessment according to published guidelines for the management of patients with Crohn’s disease, they created a patient platform – coined ProjectSonar – that alerts their Crohn’s patients on their smart phones, engages them in a short survey, and provides them with instant feedback on their disease status and care needs based on their responses. Survey results are sent to the Web and to nurse case managers at the practice, who follow up with the patient accordingly.

A year-long pilot program of the patient portal with 50 people in the study population showed more than a 600% return on the cost of investment in the proprietary software, with an average of $6,000 in medical savings for test subjects who responded to texts, compared with controls who did not receive smart phone texts, for a total savings of more than half a million dollars. “All of the savings come from the patients who respond,” Dr. Kosinski said, noting that, in his practice, they now have a sustained patient response rate of more than 80% and that it helps to have the physician emphasize use of the platform to the patient.

“Patients love it. It is almost like chronic disease concierge medicine they don’t have to pay for,” Dr. Kosinski said during his presentation at the meeting, adding that the insurer likes it because it cuts costs, and physicians like it because they don’t have to take less reimbursement to help the insurer realize gains.

“We risk assess every patient, something the majority of doctors don’t do but [that] insurance companies do all the time,” Dr. Kosinski said in an interview after his presentation. “Then we apply the appropriate treatment using the scientific methods in the published guidelines. Then we analyze the data, which helps us refine our assessments and predict our costs of care in this population.”

Knowing the base cost of care for specific patient populations helps define the margin of financial risk he and his colleagues can tolerate. A gastroenterology practice that operates as a risk-bearing entity could theoretically offer to contract with affordable care organizations to manage IBD or other GI-type conditions, he said.

By learning to assess, measure, and leverage risk, gastroenterologists can become sought after for the value they provide rather than for the care they “assemble,” something Dr. Kosinski said is of rising concern as the Affordable Care Act has driven a lot of consolidation, with hospital systems buying up primary care physicians and specialists.

Otherwise, he said, “We’re just going to be commoditized proceduralists.”

Dr. Kosinski is president of SonarMDTM.
 

 

 

 

BOSTON – Gastroenterology is becoming a game of risk: It’s either learn to leverage risk through the creation of advanced alternative payment methods (APM) under Medicare’s new Quality Payment Program or risk losing money through the commoditization of the field, according to an expert.

“Our culture right now is one where we get paid for making widgets,” said Lawrence Kosinski, MD, a practice councilor on the American Gastroenterological Association Governing Board and former chairman of its Practice Management and Economics Committee. He made his remarks in an interview in advance of his presentation at the 2017 AGA Tech Summit sponsored by the AGA Center for GI Innovation and Technology. “The more colonoscopies we do, the more money we make. The more we can charge for those colonoscopies and get collected, the better things are for us.”

Eli Zimmerman/Frontline Medical News
Dr. Lawrence Kosinski
However, since the goal of value-based care is to drive down the cost of services – such as by replacing colonoscopies with less expensive alternatives – under the new rules of reimbursement, the gastroenterologist’s competitive advantage is increasingly in the knowledge of how to manage complex chronic diseases.

“Over 80% of the cost of health care is for the management of chronic disease. We happen to have a very expensive set of chronic diseases that we take care of in GI, very complicated illnesses. We need to leverage the management of those patients, but we need to be able to show how our work decreases the overall cost of care so that we can get a piece of that risk premium,” he said.

In his own practice, Dr. Kosinski and his colleagues have created an APM – the first novel APM to be recommended to the Centers for Medicare & Medicaid Services for approval by the Physician-Focused Payment Model Technical Advisory Committee – that is based on better patient risk assessment, combined with earlier patient engagement.

After discovering in 2013 that one of his payers was spending $24,000 annually on patients with inflammatory bowel disease and that two-thirds of patients with inflammatory bowel disease who are admitted to the hospital had not had a CPT code issued in the 30 days before admission, Dr. Kosinski and his colleagues wanted to see if they could offer the insurer value by decreasing that hospitalization rate.

Using proprietary algorithms rooted in thorough patient risk assessment according to published guidelines for the management of patients with Crohn’s disease, they created a patient platform – coined ProjectSonar – that alerts their Crohn’s patients on their smart phones, engages them in a short survey, and provides them with instant feedback on their disease status and care needs based on their responses. Survey results are sent to the Web and to nurse case managers at the practice, who follow up with the patient accordingly.

A year-long pilot program of the patient portal with 50 people in the study population showed more than a 600% return on the cost of investment in the proprietary software, with an average of $6,000 in medical savings for test subjects who responded to texts, compared with controls who did not receive smart phone texts, for a total savings of more than half a million dollars. “All of the savings come from the patients who respond,” Dr. Kosinski said, noting that, in his practice, they now have a sustained patient response rate of more than 80% and that it helps to have the physician emphasize use of the platform to the patient.

“Patients love it. It is almost like chronic disease concierge medicine they don’t have to pay for,” Dr. Kosinski said during his presentation at the meeting, adding that the insurer likes it because it cuts costs, and physicians like it because they don’t have to take less reimbursement to help the insurer realize gains.

“We risk assess every patient, something the majority of doctors don’t do but [that] insurance companies do all the time,” Dr. Kosinski said in an interview after his presentation. “Then we apply the appropriate treatment using the scientific methods in the published guidelines. Then we analyze the data, which helps us refine our assessments and predict our costs of care in this population.”

Knowing the base cost of care for specific patient populations helps define the margin of financial risk he and his colleagues can tolerate. A gastroenterology practice that operates as a risk-bearing entity could theoretically offer to contract with affordable care organizations to manage IBD or other GI-type conditions, he said.

By learning to assess, measure, and leverage risk, gastroenterologists can become sought after for the value they provide rather than for the care they “assemble,” something Dr. Kosinski said is of rising concern as the Affordable Care Act has driven a lot of consolidation, with hospital systems buying up primary care physicians and specialists.

Otherwise, he said, “We’re just going to be commoditized proceduralists.”

Dr. Kosinski is president of SonarMDTM.
 

 

 

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FROM THE 2017 AGA TECH SUMMIT

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VIDEO: Hidradenitis suppurativa in pediatrics similar to that in adults

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WASHINGTON– Hidradenitis suppurativa (HS) can present early in adolescence, and the approach to treatment in pediatric patients is similar to treatment in adults, “with a few caveats,” A. Yasmine Kirkorian, MD, said in a video interview at an educational session held by George Washington University.

While the literature often states that HS starts in a person’s 20s, it primarily starts in adolescence, sometimes earlier, with a presentation that is similar to that seen in adults and appearing at the same sites, such as the armpits and groin, said Dr. Kirkorian a pediatric dermatologist at Children’s National Health System and George Washington University, Washington.

“As dermatologists, we know this. Kids are getting this disease, but we need to characterize that better in the literature so that we can start to apply the adult style therapeutics in clinical trials ... to children,” she added.

In the interview, Dr. Kirkorian discussed treatment strategies, as well as familial cases of HS and the links between HS and Down syndrome and inflammatory bowel disease in children.

The meeting was sponsored by AbbVie. Dr. Kirkorian had no financial conflicts to disclose.

The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
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WASHINGTON– Hidradenitis suppurativa (HS) can present early in adolescence, and the approach to treatment in pediatric patients is similar to treatment in adults, “with a few caveats,” A. Yasmine Kirkorian, MD, said in a video interview at an educational session held by George Washington University.

While the literature often states that HS starts in a person’s 20s, it primarily starts in adolescence, sometimes earlier, with a presentation that is similar to that seen in adults and appearing at the same sites, such as the armpits and groin, said Dr. Kirkorian a pediatric dermatologist at Children’s National Health System and George Washington University, Washington.

“As dermatologists, we know this. Kids are getting this disease, but we need to characterize that better in the literature so that we can start to apply the adult style therapeutics in clinical trials ... to children,” she added.

In the interview, Dr. Kirkorian discussed treatment strategies, as well as familial cases of HS and the links between HS and Down syndrome and inflammatory bowel disease in children.

The meeting was sponsored by AbbVie. Dr. Kirkorian had no financial conflicts to disclose.

The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel

 

WASHINGTON– Hidradenitis suppurativa (HS) can present early in adolescence, and the approach to treatment in pediatric patients is similar to treatment in adults, “with a few caveats,” A. Yasmine Kirkorian, MD, said in a video interview at an educational session held by George Washington University.

While the literature often states that HS starts in a person’s 20s, it primarily starts in adolescence, sometimes earlier, with a presentation that is similar to that seen in adults and appearing at the same sites, such as the armpits and groin, said Dr. Kirkorian a pediatric dermatologist at Children’s National Health System and George Washington University, Washington.

“As dermatologists, we know this. Kids are getting this disease, but we need to characterize that better in the literature so that we can start to apply the adult style therapeutics in clinical trials ... to children,” she added.

In the interview, Dr. Kirkorian discussed treatment strategies, as well as familial cases of HS and the links between HS and Down syndrome and inflammatory bowel disease in children.

The meeting was sponsored by AbbVie. Dr. Kirkorian had no financial conflicts to disclose.

The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
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In mantle cell lymphoma, triple therapy proves too toxic

Chemo-free treatment: Proceed with caution
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Combined idelalisib, lenalidomide, and rituximab proved excessively toxic for the treatment of relapsed and refractory mantle cell and follicular lymphoma in two phase I trials conducted by the Alliance for Clinical Trials in Oncology.

The unexpected outcome, which led to early study termination, underscores the need for caution as new treatment combinations are proposed, Sonali M. Smith, MD, of the University of Chicago and her colleagues said in The Lancet Haematology.

In four of the first eight patients enrolled in the mantle cell lymphoma (A051201) and follicular lymphoma (A051202) phase I trials between July 9, 2013, and Sept. 30, 2014, unexpected dose-limiting toxicities occurred, including grade 4 sepsis syndrome, grade 4 hypotension with grade 3 rash and fevers, grade 4 aspartate aminotransferase (AST) or alanine aminotransferase (ALT) elevation with fevers, and grade 3 pulmonary infection with grade 3 maculopapular rash.

The adverse events occurred between 9 and 20 days after treatment initiation and coincided with rituximab infusions, the researchers said. No treatment-related deaths occurred (Lancet Haematol. 2017 Apr;4:e176-82).

Overall, 8 of 11 patients were removed from treatment because of an adverse event, and 3 of those required intensive care unit level of care.

Although rituximab was removed in both trials, two of the remaining three patients in the studies, including three with mantle cell lymphoma and eight with follicular lymphoma, experienced grade 3 rashes, and one had grade 3 AST elevations. In those with mantle cell lymphoma, the most common grade 3-4 adverse events were ALT elevations and rash. In those with follicular lymphoma, the most common grade 3-4 adverse events were neutropenia and rash.

“Given the inability to deliver treatment due to toxicity, both studies were permanently closed,” the researchers wrote, noting that the primary endpoint of safety and tolerability was not met.

The trials had the overall goal of developing targeted regimens to replace cytotoxic therapy.

“Both ... trials were designed to capitalize on the clinical synergy of lenalidomide and rituximab observed in previous trials by adding the highly specific PI3K delta inhibitor, idelalisib, for patients with relapsed mantle cell lymphoma and follicular lymphoma,” they said.

Previously available data implied that lenalidomide plus rituximab would be a safe backbone for therapy, and there was clinical rationale for adding idelalisib to that combination, they explained.

“Overall, our brief experience underscores the limited knowledge regarding drug interactions and off-target effects and serves as a cautionary note in developing biological agents in combination and against ad-hoc combinations outside of carefully monitored clinical trials,” they said.

The researchers noted that the nature of the toxicities observed in these trials supports an immune-activated state characterized by excessive inflammation.

“A more detailed assessment of effect on cytokines, T-cell subsets, natural killer cells, and clinical features predictive of toxicity and response should be included in any further testing of these classes of agents, and they should never be combined outside of a carefully designed and diligently monitored clinical trial setting,” they concluded.

The study was funded by the National Cancer Institute. Dr. Smith received research funding and consulting fees from Gilead and Celgene.

Body

 

The findings by Dr. Smith and her colleagues add to several other reported studies that involved unexpected toxicities with various combinations of targeted agents in lymphoid malignancies.

Combinations of B-cell receptor signaling inhibitors can lead to immune dysregulation, which can be acute and severe when combined with immunomodulatory agents.

While the study of rational targeted combinations continues to hold immense potential in both untreated and relapsed/refractory disease, the combination must be thoroughly studied in the context of carefully and conservatively designed clinical trials.

Given the unpredictable nature of adverse events, the use of novel combinations outside of a clinical trial should be strongly discouraged.

Patrick M. Reagan, MD , and Paul M. Barr, MD , are with the James P. Wilmot Cancer Institute, University of Rochester, New York. Dr. Reagan reported having no disclosures. Dr. Barr has consulted for Gilead, Pharmacyclics, AbbVie, and Celgene. They made their remarks in an editorial that accompanied the article.
 

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The findings by Dr. Smith and her colleagues add to several other reported studies that involved unexpected toxicities with various combinations of targeted agents in lymphoid malignancies.

Combinations of B-cell receptor signaling inhibitors can lead to immune dysregulation, which can be acute and severe when combined with immunomodulatory agents.

While the study of rational targeted combinations continues to hold immense potential in both untreated and relapsed/refractory disease, the combination must be thoroughly studied in the context of carefully and conservatively designed clinical trials.

Given the unpredictable nature of adverse events, the use of novel combinations outside of a clinical trial should be strongly discouraged.

Patrick M. Reagan, MD , and Paul M. Barr, MD , are with the James P. Wilmot Cancer Institute, University of Rochester, New York. Dr. Reagan reported having no disclosures. Dr. Barr has consulted for Gilead, Pharmacyclics, AbbVie, and Celgene. They made their remarks in an editorial that accompanied the article.
 

Body

 

The findings by Dr. Smith and her colleagues add to several other reported studies that involved unexpected toxicities with various combinations of targeted agents in lymphoid malignancies.

Combinations of B-cell receptor signaling inhibitors can lead to immune dysregulation, which can be acute and severe when combined with immunomodulatory agents.

While the study of rational targeted combinations continues to hold immense potential in both untreated and relapsed/refractory disease, the combination must be thoroughly studied in the context of carefully and conservatively designed clinical trials.

Given the unpredictable nature of adverse events, the use of novel combinations outside of a clinical trial should be strongly discouraged.

Patrick M. Reagan, MD , and Paul M. Barr, MD , are with the James P. Wilmot Cancer Institute, University of Rochester, New York. Dr. Reagan reported having no disclosures. Dr. Barr has consulted for Gilead, Pharmacyclics, AbbVie, and Celgene. They made their remarks in an editorial that accompanied the article.
 

Title
Chemo-free treatment: Proceed with caution
Chemo-free treatment: Proceed with caution

 

Combined idelalisib, lenalidomide, and rituximab proved excessively toxic for the treatment of relapsed and refractory mantle cell and follicular lymphoma in two phase I trials conducted by the Alliance for Clinical Trials in Oncology.

The unexpected outcome, which led to early study termination, underscores the need for caution as new treatment combinations are proposed, Sonali M. Smith, MD, of the University of Chicago and her colleagues said in The Lancet Haematology.

In four of the first eight patients enrolled in the mantle cell lymphoma (A051201) and follicular lymphoma (A051202) phase I trials between July 9, 2013, and Sept. 30, 2014, unexpected dose-limiting toxicities occurred, including grade 4 sepsis syndrome, grade 4 hypotension with grade 3 rash and fevers, grade 4 aspartate aminotransferase (AST) or alanine aminotransferase (ALT) elevation with fevers, and grade 3 pulmonary infection with grade 3 maculopapular rash.

The adverse events occurred between 9 and 20 days after treatment initiation and coincided with rituximab infusions, the researchers said. No treatment-related deaths occurred (Lancet Haematol. 2017 Apr;4:e176-82).

Overall, 8 of 11 patients were removed from treatment because of an adverse event, and 3 of those required intensive care unit level of care.

Although rituximab was removed in both trials, two of the remaining three patients in the studies, including three with mantle cell lymphoma and eight with follicular lymphoma, experienced grade 3 rashes, and one had grade 3 AST elevations. In those with mantle cell lymphoma, the most common grade 3-4 adverse events were ALT elevations and rash. In those with follicular lymphoma, the most common grade 3-4 adverse events were neutropenia and rash.

“Given the inability to deliver treatment due to toxicity, both studies were permanently closed,” the researchers wrote, noting that the primary endpoint of safety and tolerability was not met.

The trials had the overall goal of developing targeted regimens to replace cytotoxic therapy.

“Both ... trials were designed to capitalize on the clinical synergy of lenalidomide and rituximab observed in previous trials by adding the highly specific PI3K delta inhibitor, idelalisib, for patients with relapsed mantle cell lymphoma and follicular lymphoma,” they said.

Previously available data implied that lenalidomide plus rituximab would be a safe backbone for therapy, and there was clinical rationale for adding idelalisib to that combination, they explained.

“Overall, our brief experience underscores the limited knowledge regarding drug interactions and off-target effects and serves as a cautionary note in developing biological agents in combination and against ad-hoc combinations outside of carefully monitored clinical trials,” they said.

The researchers noted that the nature of the toxicities observed in these trials supports an immune-activated state characterized by excessive inflammation.

“A more detailed assessment of effect on cytokines, T-cell subsets, natural killer cells, and clinical features predictive of toxicity and response should be included in any further testing of these classes of agents, and they should never be combined outside of a carefully designed and diligently monitored clinical trial setting,” they concluded.

The study was funded by the National Cancer Institute. Dr. Smith received research funding and consulting fees from Gilead and Celgene.

 

Combined idelalisib, lenalidomide, and rituximab proved excessively toxic for the treatment of relapsed and refractory mantle cell and follicular lymphoma in two phase I trials conducted by the Alliance for Clinical Trials in Oncology.

The unexpected outcome, which led to early study termination, underscores the need for caution as new treatment combinations are proposed, Sonali M. Smith, MD, of the University of Chicago and her colleagues said in The Lancet Haematology.

In four of the first eight patients enrolled in the mantle cell lymphoma (A051201) and follicular lymphoma (A051202) phase I trials between July 9, 2013, and Sept. 30, 2014, unexpected dose-limiting toxicities occurred, including grade 4 sepsis syndrome, grade 4 hypotension with grade 3 rash and fevers, grade 4 aspartate aminotransferase (AST) or alanine aminotransferase (ALT) elevation with fevers, and grade 3 pulmonary infection with grade 3 maculopapular rash.

The adverse events occurred between 9 and 20 days after treatment initiation and coincided with rituximab infusions, the researchers said. No treatment-related deaths occurred (Lancet Haematol. 2017 Apr;4:e176-82).

Overall, 8 of 11 patients were removed from treatment because of an adverse event, and 3 of those required intensive care unit level of care.

Although rituximab was removed in both trials, two of the remaining three patients in the studies, including three with mantle cell lymphoma and eight with follicular lymphoma, experienced grade 3 rashes, and one had grade 3 AST elevations. In those with mantle cell lymphoma, the most common grade 3-4 adverse events were ALT elevations and rash. In those with follicular lymphoma, the most common grade 3-4 adverse events were neutropenia and rash.

“Given the inability to deliver treatment due to toxicity, both studies were permanently closed,” the researchers wrote, noting that the primary endpoint of safety and tolerability was not met.

The trials had the overall goal of developing targeted regimens to replace cytotoxic therapy.

“Both ... trials were designed to capitalize on the clinical synergy of lenalidomide and rituximab observed in previous trials by adding the highly specific PI3K delta inhibitor, idelalisib, for patients with relapsed mantle cell lymphoma and follicular lymphoma,” they said.

Previously available data implied that lenalidomide plus rituximab would be a safe backbone for therapy, and there was clinical rationale for adding idelalisib to that combination, they explained.

“Overall, our brief experience underscores the limited knowledge regarding drug interactions and off-target effects and serves as a cautionary note in developing biological agents in combination and against ad-hoc combinations outside of carefully monitored clinical trials,” they said.

The researchers noted that the nature of the toxicities observed in these trials supports an immune-activated state characterized by excessive inflammation.

“A more detailed assessment of effect on cytokines, T-cell subsets, natural killer cells, and clinical features predictive of toxicity and response should be included in any further testing of these classes of agents, and they should never be combined outside of a carefully designed and diligently monitored clinical trial setting,” they concluded.

The study was funded by the National Cancer Institute. Dr. Smith received research funding and consulting fees from Gilead and Celgene.

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Key clinical point: Combined idelalisib, lenalidomide, and rituximab for relapsed and refractory lymphoma proved excessively toxic in two phase 1 trials.

Major finding: Of 11 patients, 8 were removed from treatment because of an adverse event, and 3 of those required intensive care unit–level care.

Data source: Two phase I trials involving 11 patients.

Disclosures: The study was funded by the National Cancer Institute. Dr. Smith received research funding and consulting fees from Gilead and Celgene.

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New guideline: Address GTCS frequency to reduce SUDEP risk

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– Generalized tonic-clonic seizures (GTCS) are a major risk factor for sudden unexpected death in epilepsy (SUDEP), which underscores the importance of advising people with epilepsy about controlling such seizures, according to a new practice guideline from the American Academy of Neurology and the American Epilepsy Society.

Though SUDEP is rare, with an incidence rate of 0.22/1,000 patient-years in children with epilepsy and 1.2/1,000 patient-years in adults with epilepsy, the guideline committee found that people with three or more GTCS per year are 15 times more likely to die suddenly than are those without this seizure type. The risk increases with increasing GTCS frequency. This translates to an absolute risk of up to 18 deaths per 1,000 patient-years for people with epilepsy who have frequent GTCS.

Sharon Worcester/Frontline Medical News
Dr. Cynthia Harden
“It is important that the rate of occurrence of SUDEP and the specific risk factors for SUDEP are communicated to persons and families affected by epilepsy,” lead guideline author, Cynthia L. Harden, MD, said during a press conference at the annual meeting of the American Academy of Neurology. “Our guideline brings clarity to the discussion, giving health care providers practical information they can use to help people with epilepsy reduce their risk.”

Specifically, the guideline recommends that health care providers should tell people with epilepsy that controlling seizures, especially GTCS, may reduce the risk of SUDEP, she said, adding that the guideline shows that “being free of seizures, particularly tonic-clonic seizures, is strongly associated with a decreased risk.”

Sharon Worcester/Frontline Medical News
Dr. Elizabeth Donner
“This guideline makes the conversation much easier with information that may motivate people to take their medications on time, to never skip taking their medications, and to learn and manage their seizure triggers so they can work toward reducing seizures. People who follow their medication schedule or pursue other treatments such as epilepsy surgery may be more likely to become seizure free,” said Dr. Harden, director of Epilepsy Services for the Mount Sinai Health System in New York.

Guideline coauthor, Elizabeth Donner, MD, added that, for this reason, the guideline recommends “that health professionals work with people who continue to have, specifically, these kind of seizures to try and reduce them with medications or with epilepsy surgery, actively weighing the risks and benefits of any new approach to seizure management.”

The recommendations are based on moderate (Level B) evidence.

The team also looked at numerous other risk factors for SUDEP and found that the strength of the evidence was too weak to support additional recommendations, said Dr. Donner, director of the comprehensive epilepsy program at The Hospital for Sick Children in Toronto and chair of the American Epilepsy Society SUDEP Task Force.

“More research is now needed to identify other preventable risk factors for SUDEP so that we can focus future studies on finding ways to reduce how often SUDEP occurs,” she added.

While the message regarding the importance of reducing seizure frequency is not new, it is important that this message be reiterated in the context of SUDEP, Dr. Donner said.

“It’s very important for it to be clear that the risk of frequent generalized tonic-clonic seizures – and we’re not talking about really frequent here; we’re talking about significant increased risk of death with only three per year – is not related only to maintaining a driver’s license, maintaining work, or other outcomes like that. It’s actually related to risk of death,” she said, noting that she hopes this is a motivator for pursuing treatments beyond medication when medication isn’t successful for treating seizures.

The guideline, which is endorsed by the International Child Neurology Association, is available online and in print (Neurology. 2017;88:1674–80).

Dr. Harden receives royalties from Wiley and Up-to-Date. Dr. Donner has received research support from the Canadian Institutes of Health Research, Dravet Canada, and SUDEP Aware. Other guideline authors reported numerous disclosures, including many industry sources.

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– Generalized tonic-clonic seizures (GTCS) are a major risk factor for sudden unexpected death in epilepsy (SUDEP), which underscores the importance of advising people with epilepsy about controlling such seizures, according to a new practice guideline from the American Academy of Neurology and the American Epilepsy Society.

Though SUDEP is rare, with an incidence rate of 0.22/1,000 patient-years in children with epilepsy and 1.2/1,000 patient-years in adults with epilepsy, the guideline committee found that people with three or more GTCS per year are 15 times more likely to die suddenly than are those without this seizure type. The risk increases with increasing GTCS frequency. This translates to an absolute risk of up to 18 deaths per 1,000 patient-years for people with epilepsy who have frequent GTCS.

Sharon Worcester/Frontline Medical News
Dr. Cynthia Harden
“It is important that the rate of occurrence of SUDEP and the specific risk factors for SUDEP are communicated to persons and families affected by epilepsy,” lead guideline author, Cynthia L. Harden, MD, said during a press conference at the annual meeting of the American Academy of Neurology. “Our guideline brings clarity to the discussion, giving health care providers practical information they can use to help people with epilepsy reduce their risk.”

Specifically, the guideline recommends that health care providers should tell people with epilepsy that controlling seizures, especially GTCS, may reduce the risk of SUDEP, she said, adding that the guideline shows that “being free of seizures, particularly tonic-clonic seizures, is strongly associated with a decreased risk.”

Sharon Worcester/Frontline Medical News
Dr. Elizabeth Donner
“This guideline makes the conversation much easier with information that may motivate people to take their medications on time, to never skip taking their medications, and to learn and manage their seizure triggers so they can work toward reducing seizures. People who follow their medication schedule or pursue other treatments such as epilepsy surgery may be more likely to become seizure free,” said Dr. Harden, director of Epilepsy Services for the Mount Sinai Health System in New York.

Guideline coauthor, Elizabeth Donner, MD, added that, for this reason, the guideline recommends “that health professionals work with people who continue to have, specifically, these kind of seizures to try and reduce them with medications or with epilepsy surgery, actively weighing the risks and benefits of any new approach to seizure management.”

The recommendations are based on moderate (Level B) evidence.

The team also looked at numerous other risk factors for SUDEP and found that the strength of the evidence was too weak to support additional recommendations, said Dr. Donner, director of the comprehensive epilepsy program at The Hospital for Sick Children in Toronto and chair of the American Epilepsy Society SUDEP Task Force.

“More research is now needed to identify other preventable risk factors for SUDEP so that we can focus future studies on finding ways to reduce how often SUDEP occurs,” she added.

While the message regarding the importance of reducing seizure frequency is not new, it is important that this message be reiterated in the context of SUDEP, Dr. Donner said.

“It’s very important for it to be clear that the risk of frequent generalized tonic-clonic seizures – and we’re not talking about really frequent here; we’re talking about significant increased risk of death with only three per year – is not related only to maintaining a driver’s license, maintaining work, or other outcomes like that. It’s actually related to risk of death,” she said, noting that she hopes this is a motivator for pursuing treatments beyond medication when medication isn’t successful for treating seizures.

The guideline, which is endorsed by the International Child Neurology Association, is available online and in print (Neurology. 2017;88:1674–80).

Dr. Harden receives royalties from Wiley and Up-to-Date. Dr. Donner has received research support from the Canadian Institutes of Health Research, Dravet Canada, and SUDEP Aware. Other guideline authors reported numerous disclosures, including many industry sources.

 

– Generalized tonic-clonic seizures (GTCS) are a major risk factor for sudden unexpected death in epilepsy (SUDEP), which underscores the importance of advising people with epilepsy about controlling such seizures, according to a new practice guideline from the American Academy of Neurology and the American Epilepsy Society.

Though SUDEP is rare, with an incidence rate of 0.22/1,000 patient-years in children with epilepsy and 1.2/1,000 patient-years in adults with epilepsy, the guideline committee found that people with three or more GTCS per year are 15 times more likely to die suddenly than are those without this seizure type. The risk increases with increasing GTCS frequency. This translates to an absolute risk of up to 18 deaths per 1,000 patient-years for people with epilepsy who have frequent GTCS.

Sharon Worcester/Frontline Medical News
Dr. Cynthia Harden
“It is important that the rate of occurrence of SUDEP and the specific risk factors for SUDEP are communicated to persons and families affected by epilepsy,” lead guideline author, Cynthia L. Harden, MD, said during a press conference at the annual meeting of the American Academy of Neurology. “Our guideline brings clarity to the discussion, giving health care providers practical information they can use to help people with epilepsy reduce their risk.”

Specifically, the guideline recommends that health care providers should tell people with epilepsy that controlling seizures, especially GTCS, may reduce the risk of SUDEP, she said, adding that the guideline shows that “being free of seizures, particularly tonic-clonic seizures, is strongly associated with a decreased risk.”

Sharon Worcester/Frontline Medical News
Dr. Elizabeth Donner
“This guideline makes the conversation much easier with information that may motivate people to take their medications on time, to never skip taking their medications, and to learn and manage their seizure triggers so they can work toward reducing seizures. People who follow their medication schedule or pursue other treatments such as epilepsy surgery may be more likely to become seizure free,” said Dr. Harden, director of Epilepsy Services for the Mount Sinai Health System in New York.

Guideline coauthor, Elizabeth Donner, MD, added that, for this reason, the guideline recommends “that health professionals work with people who continue to have, specifically, these kind of seizures to try and reduce them with medications or with epilepsy surgery, actively weighing the risks and benefits of any new approach to seizure management.”

The recommendations are based on moderate (Level B) evidence.

The team also looked at numerous other risk factors for SUDEP and found that the strength of the evidence was too weak to support additional recommendations, said Dr. Donner, director of the comprehensive epilepsy program at The Hospital for Sick Children in Toronto and chair of the American Epilepsy Society SUDEP Task Force.

“More research is now needed to identify other preventable risk factors for SUDEP so that we can focus future studies on finding ways to reduce how often SUDEP occurs,” she added.

While the message regarding the importance of reducing seizure frequency is not new, it is important that this message be reiterated in the context of SUDEP, Dr. Donner said.

“It’s very important for it to be clear that the risk of frequent generalized tonic-clonic seizures – and we’re not talking about really frequent here; we’re talking about significant increased risk of death with only three per year – is not related only to maintaining a driver’s license, maintaining work, or other outcomes like that. It’s actually related to risk of death,” she said, noting that she hopes this is a motivator for pursuing treatments beyond medication when medication isn’t successful for treating seizures.

The guideline, which is endorsed by the International Child Neurology Association, is available online and in print (Neurology. 2017;88:1674–80).

Dr. Harden receives royalties from Wiley and Up-to-Date. Dr. Donner has received research support from the Canadian Institutes of Health Research, Dravet Canada, and SUDEP Aware. Other guideline authors reported numerous disclosures, including many industry sources.

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Apomorphine Reduces Off Time for People with Advanced Parkinson’s Disease

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Patients who received apomorphine reported improvements and were less likely to have worsening of motor function.

BOSTON—Apomorphine may provide relief from off time for people with advanced Parkinson’s disease, according to a study presented at the 69th Annual Meeting of the American Academy of Neurology.

Regina Katzenschlager, MD

“If a person with Parkinson’s disease can reduce their off times, that can have a great impact on their everyday life,” said study author Regina Katzenschlager, MD, of Danube Hospital, which is affiliated with the Medical University of Vienna. “In some patients in the trial, the insecurity of unpredictable periods of incapacity was completely alleviated.”

Open-Label Data Suggest Efficacy

Levodopa has long been the gold standard in the treatment of Parkinson’s disease, but the effects of the medication can partially wear off more quickly as the disease progresses. Patients consequently experience off time that includes symptoms such as slowness and muscle rigidity.

The drug apomorphine, which first was produced in 1865, began to be used to treat advanced Parkinson’s disease in the United States in 1950. Its use grew in the 1990s when European doctors started administering subcutaneous infusions of the drug to treat fluctuations in mobility that could not be controlled by levodopa.

Extensive data from open-label studies of apomorphine have demonstrated its efficacy in reducing off time, dyskinesias, and oral levodopa dose in patients with severe motor fluctuations that are poorly controlled by conventional therapy. Evidence from randomized, blinded studies of the drug has been lacking, however.

Comparing Apomorphine With Placebo

In a double-blind phase III study, researchers recruited 107 people with advanced Parkinson’s disease from 23 centers in seven countries.

Participants were randomized to either apomorphine subcutaneous infusion (≤ 8 mg/h) or a placebo saline infusion. The infusion was administered over a period of 14 to 18 hours each day through a small portable pump similar to the type used in the treatment of type 1 diabetes. The hourly flow rate of the infusion and dose of concomitant antiparkinsonian medication were adjusted during the first four weeks, based on efficacy and tolerability. The study’s primary end point was the absolute change in off time from baseline to Week 12 based on patient diaries.

Patients who received apomorphine had a significantly greater reduction of off time than those who received the placebo infusion. Active patients had, on average, 2.5 hours less off time per day, while participants who received the placebo infusion had an average of 35 minutes less off time per day. This improvement was apparent within the first week of treatment and was sustained over 12 weeks. At the same time, patients who received apomorphine had a significantly greater increase of on time without the dyskinesias that often are observed with levodopa, compared with placebo.

Participants were also asked to evaluate how well they thought the treatment worked. Those who received apomorphine gave their treatment higher scores at week 12 than those who received the placebo infusion. In the apomorphine group, 71% of patients felt improved, compared with 18% of patients who received placebo. Furthermore, 19% of patients worsened on apomorphine, compared with 45% on placebo. Apomorphine was generally well tolerated, and the researchers observed no serious side effects.

“It is our hope that these findings confirming the efficacy of apomorphine infusion will encourage doctors in the United States to offer this treatment to their patients and assess its efficacy in their own clinical practice,” Dr. Katzenschlager
concluded.

The study was supported by Britannia Pharmaceuticals, the maker of apomorphine.

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Patients who received apomorphine reported improvements and were less likely to have worsening of motor function.
Patients who received apomorphine reported improvements and were less likely to have worsening of motor function.

BOSTON—Apomorphine may provide relief from off time for people with advanced Parkinson’s disease, according to a study presented at the 69th Annual Meeting of the American Academy of Neurology.

Regina Katzenschlager, MD

“If a person with Parkinson’s disease can reduce their off times, that can have a great impact on their everyday life,” said study author Regina Katzenschlager, MD, of Danube Hospital, which is affiliated with the Medical University of Vienna. “In some patients in the trial, the insecurity of unpredictable periods of incapacity was completely alleviated.”

Open-Label Data Suggest Efficacy

Levodopa has long been the gold standard in the treatment of Parkinson’s disease, but the effects of the medication can partially wear off more quickly as the disease progresses. Patients consequently experience off time that includes symptoms such as slowness and muscle rigidity.

The drug apomorphine, which first was produced in 1865, began to be used to treat advanced Parkinson’s disease in the United States in 1950. Its use grew in the 1990s when European doctors started administering subcutaneous infusions of the drug to treat fluctuations in mobility that could not be controlled by levodopa.

Extensive data from open-label studies of apomorphine have demonstrated its efficacy in reducing off time, dyskinesias, and oral levodopa dose in patients with severe motor fluctuations that are poorly controlled by conventional therapy. Evidence from randomized, blinded studies of the drug has been lacking, however.

Comparing Apomorphine With Placebo

In a double-blind phase III study, researchers recruited 107 people with advanced Parkinson’s disease from 23 centers in seven countries.

Participants were randomized to either apomorphine subcutaneous infusion (≤ 8 mg/h) or a placebo saline infusion. The infusion was administered over a period of 14 to 18 hours each day through a small portable pump similar to the type used in the treatment of type 1 diabetes. The hourly flow rate of the infusion and dose of concomitant antiparkinsonian medication were adjusted during the first four weeks, based on efficacy and tolerability. The study’s primary end point was the absolute change in off time from baseline to Week 12 based on patient diaries.

Patients who received apomorphine had a significantly greater reduction of off time than those who received the placebo infusion. Active patients had, on average, 2.5 hours less off time per day, while participants who received the placebo infusion had an average of 35 minutes less off time per day. This improvement was apparent within the first week of treatment and was sustained over 12 weeks. At the same time, patients who received apomorphine had a significantly greater increase of on time without the dyskinesias that often are observed with levodopa, compared with placebo.

Participants were also asked to evaluate how well they thought the treatment worked. Those who received apomorphine gave their treatment higher scores at week 12 than those who received the placebo infusion. In the apomorphine group, 71% of patients felt improved, compared with 18% of patients who received placebo. Furthermore, 19% of patients worsened on apomorphine, compared with 45% on placebo. Apomorphine was generally well tolerated, and the researchers observed no serious side effects.

“It is our hope that these findings confirming the efficacy of apomorphine infusion will encourage doctors in the United States to offer this treatment to their patients and assess its efficacy in their own clinical practice,” Dr. Katzenschlager
concluded.

The study was supported by Britannia Pharmaceuticals, the maker of apomorphine.

BOSTON—Apomorphine may provide relief from off time for people with advanced Parkinson’s disease, according to a study presented at the 69th Annual Meeting of the American Academy of Neurology.

Regina Katzenschlager, MD

“If a person with Parkinson’s disease can reduce their off times, that can have a great impact on their everyday life,” said study author Regina Katzenschlager, MD, of Danube Hospital, which is affiliated with the Medical University of Vienna. “In some patients in the trial, the insecurity of unpredictable periods of incapacity was completely alleviated.”

Open-Label Data Suggest Efficacy

Levodopa has long been the gold standard in the treatment of Parkinson’s disease, but the effects of the medication can partially wear off more quickly as the disease progresses. Patients consequently experience off time that includes symptoms such as slowness and muscle rigidity.

The drug apomorphine, which first was produced in 1865, began to be used to treat advanced Parkinson’s disease in the United States in 1950. Its use grew in the 1990s when European doctors started administering subcutaneous infusions of the drug to treat fluctuations in mobility that could not be controlled by levodopa.

Extensive data from open-label studies of apomorphine have demonstrated its efficacy in reducing off time, dyskinesias, and oral levodopa dose in patients with severe motor fluctuations that are poorly controlled by conventional therapy. Evidence from randomized, blinded studies of the drug has been lacking, however.

Comparing Apomorphine With Placebo

In a double-blind phase III study, researchers recruited 107 people with advanced Parkinson’s disease from 23 centers in seven countries.

Participants were randomized to either apomorphine subcutaneous infusion (≤ 8 mg/h) or a placebo saline infusion. The infusion was administered over a period of 14 to 18 hours each day through a small portable pump similar to the type used in the treatment of type 1 diabetes. The hourly flow rate of the infusion and dose of concomitant antiparkinsonian medication were adjusted during the first four weeks, based on efficacy and tolerability. The study’s primary end point was the absolute change in off time from baseline to Week 12 based on patient diaries.

Patients who received apomorphine had a significantly greater reduction of off time than those who received the placebo infusion. Active patients had, on average, 2.5 hours less off time per day, while participants who received the placebo infusion had an average of 35 minutes less off time per day. This improvement was apparent within the first week of treatment and was sustained over 12 weeks. At the same time, patients who received apomorphine had a significantly greater increase of on time without the dyskinesias that often are observed with levodopa, compared with placebo.

Participants were also asked to evaluate how well they thought the treatment worked. Those who received apomorphine gave their treatment higher scores at week 12 than those who received the placebo infusion. In the apomorphine group, 71% of patients felt improved, compared with 18% of patients who received placebo. Furthermore, 19% of patients worsened on apomorphine, compared with 45% on placebo. Apomorphine was generally well tolerated, and the researchers observed no serious side effects.

“It is our hope that these findings confirming the efficacy of apomorphine infusion will encourage doctors in the United States to offer this treatment to their patients and assess its efficacy in their own clinical practice,” Dr. Katzenschlager
concluded.

The study was supported by Britannia Pharmaceuticals, the maker of apomorphine.

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May 2017 Quiz 2

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May 2017 Quiz 2

Q2: Answer: B

Objective: Diagnose HELLP syndrome

Rationale: This patient’s presentation and laboratory findings are consistent with HELLP syndrome – the syndrome of hemolysis, elevated liver enzymes, and low platelets. HELLP is on the preeclampsia spectrum, which encompasses preeclampsia, eclampsia, and HELLP. Patients with HELLP will have hypertension (BP above 140/90), thrombocytopenia to less than 100,000/mm3 and aminotransferase levels above 70 U/L.

The diagnosis can be confirmed with an LDH (lactate dehydrogenase) greater than 600 U/L and microangiopathic hemolytic anemia on peripheral blood smear. On liver biopsy, HELLP is characterized by periportal or focal parenchyma necrosis with hyaline deposition of fibrin material in the sinusoids. However, liver biopsies are rarely performed in this setting as it likely will not change management (delivery of the fetus) and it exposes the mother and fetus to additional risks.

There is significant overlap between HELLP and acute fatty liver of pregnancy, although elevated prothrombin and partial thromboplastin time, severe hypoglycemia, and elevated creatinine are more common in acute fatty liver of pregnancy. Hypertension is more common in HELLP, and therefore this patient’s presentation is more consistent with HELLP.

 

Reference

1. Kia L, Rinella ME. Interpretation and management of hepatic abnormalities in pregnancy. Clin Gastroenterol Hepatol. 2013;11(11):1392-8.

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Q2: Answer: B

Objective: Diagnose HELLP syndrome

Rationale: This patient’s presentation and laboratory findings are consistent with HELLP syndrome – the syndrome of hemolysis, elevated liver enzymes, and low platelets. HELLP is on the preeclampsia spectrum, which encompasses preeclampsia, eclampsia, and HELLP. Patients with HELLP will have hypertension (BP above 140/90), thrombocytopenia to less than 100,000/mm3 and aminotransferase levels above 70 U/L.

The diagnosis can be confirmed with an LDH (lactate dehydrogenase) greater than 600 U/L and microangiopathic hemolytic anemia on peripheral blood smear. On liver biopsy, HELLP is characterized by periportal or focal parenchyma necrosis with hyaline deposition of fibrin material in the sinusoids. However, liver biopsies are rarely performed in this setting as it likely will not change management (delivery of the fetus) and it exposes the mother and fetus to additional risks.

There is significant overlap between HELLP and acute fatty liver of pregnancy, although elevated prothrombin and partial thromboplastin time, severe hypoglycemia, and elevated creatinine are more common in acute fatty liver of pregnancy. Hypertension is more common in HELLP, and therefore this patient’s presentation is more consistent with HELLP.

 

Reference

1. Kia L, Rinella ME. Interpretation and management of hepatic abnormalities in pregnancy. Clin Gastroenterol Hepatol. 2013;11(11):1392-8.

Q2: Answer: B

Objective: Diagnose HELLP syndrome

Rationale: This patient’s presentation and laboratory findings are consistent with HELLP syndrome – the syndrome of hemolysis, elevated liver enzymes, and low platelets. HELLP is on the preeclampsia spectrum, which encompasses preeclampsia, eclampsia, and HELLP. Patients with HELLP will have hypertension (BP above 140/90), thrombocytopenia to less than 100,000/mm3 and aminotransferase levels above 70 U/L.

The diagnosis can be confirmed with an LDH (lactate dehydrogenase) greater than 600 U/L and microangiopathic hemolytic anemia on peripheral blood smear. On liver biopsy, HELLP is characterized by periportal or focal parenchyma necrosis with hyaline deposition of fibrin material in the sinusoids. However, liver biopsies are rarely performed in this setting as it likely will not change management (delivery of the fetus) and it exposes the mother and fetus to additional risks.

There is significant overlap between HELLP and acute fatty liver of pregnancy, although elevated prothrombin and partial thromboplastin time, severe hypoglycemia, and elevated creatinine are more common in acute fatty liver of pregnancy. Hypertension is more common in HELLP, and therefore this patient’s presentation is more consistent with HELLP.

 

Reference

1. Kia L, Rinella ME. Interpretation and management of hepatic abnormalities in pregnancy. Clin Gastroenterol Hepatol. 2013;11(11):1392-8.

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May 2017 Quiz 2
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Q2. A consult is requested for a 32-year-old woman who is 29 weeks pregnant and has presented to the emergency department with nausea, vomiting, and right upper quadrant abdominal pain. She is afebrile, pulse 89, BP 160/105. On exam, she has mild to moderate epigastric and right upper quadrant tenderness. Her labs are notable for WBCs 13.0 x 109/L, Hgb 8.9 g/dL, platelets 55,000 x 109/L, AST 145 U/L, total bilirubin 2.1 mg/dL; PT and PTT are normal, blood glucose is 110 mg/dL. 

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After the epidemic, Ebola’s destructive power still haunts survivors

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– The Ebola crisis may be over in Sierra Leone, but the suffering is not.

The last patient from the epidemic was discharged in February 2016, but 78% of survivors now appear to have one or more sequelae of the infection. Some problems are mild, but some are so debilitating that life may never be the same.

Janet Scott, MD, of the University of Liverpool (England), heads a task force studying Ebola’s lingering aftereffects. These fall into four categories, Dr. Scott said at the European Society of Clinical Microbiology and Infectious Diseases annual congress: musculoskeletal pain, headache, eye problems, and psychological disorders.

They add up to an enormous risk of disability – survivors are more than 200 times more likely than controls to express at least moderate disability.

Courtesy Janet Scott, MD
Dr. Janet Scott, who cared for Ebola patients in Sierra Leone during the epidemic, is now following them to learn about the disease's aftereffects.


Dr. Scott and her team of researchers are partnering with clinicians and data managers in the Ebola treatment unit in the 34th Regimental Military Hospital (MH34) in Freetown, Sierra Leone. In Sierra Leone alone, she said, there were nearly 9,000 cases and 3,500 deaths from the virus; about 5,000 patients survived. So far, Dr. Scott and her team have collected data on about 500 patients for whom they also provide free health care.

The project has six arms, each headed by an expert: clinical care, data collection, disability, neurology, ophthalmology, and psychiatry.

The team sees patients in a large tent sectioned by a plywood wall*. Wireless Internet access, which she said is “enormously expensive” in Sierra Leone, has been donated by Omline Business Communications*. It’s the team’s lifeline, allowing them to transmit data between Freetown and participating units around the world. Members also Skype regularly, talking with patients and with each other.

All patients who come into the clinic have an initial visit that includes collection of demographics, their Ebola and clinical history (including an exploration of comorbidities), a maternal health screening for women, vital signs and symptom assessment, medication dispensing, and a treatment plan.

Then they visit the specialists, either onsite or through local referral*. These specialist modules include joints, eyes, headache, ears, neurology, cardiac, respiratory, gastrointestinal, renal and urologic, reproductive health for both genders, and psychiatry.

Courtesy Janet Scott, MD
The task force sees patients and processes collected data in this large tent.
Patients also fill out a disability questionnaire that asks them to report the presence or absence, and severity, of a variety of issues: visual problems, headache, balance problems, chest and abdominal pain, weakness, and gastrointestinal and urinary problems.

Last year, Dr. Scott published initial data on 44 patients. At ECCMID 2017, she expanded that report to include 203 survivors. They spanned all ages, but about 67% were in their most productive adult years, aged 20-39 years.

Her findings are striking: About 78% report musculoskeletal pain, with many saying they have trouble walking even short distances, climbing stairs, or picking up their children.

Headache was the next most common problem, reported by nearly 40%. About 15% report ocular problems, which include anterior uveitis, cataracts – even in very young children – and retinal lesions. Abdominal and chest pain affect about 10% of the survivors.

Although she didn’t present specific numbers, Dr. Scott also said that many of the survivors experience psychological sequelae, including insomnia, anxiety, and depression. Whether this is related to viral pathology isn’t clear; it could be a not-unexpected response to the trauma of living through the epidemic.

“Many of these people have lost their entire family, and those that are left now shun them,” she said in a live video interview
on Facebook. “It’s almost like a post-traumatic stress reaction.”

The other symptoms probably are related to the disease pathology, she observed. “Unfortunately, we don’t have all the clinical details of the acute phase for everyone, but for those for whom we do have details, we are seeing correlation between some of the problems with viral loads at admission, and even episodes of becoming unconscious during the acute illness.”

Patrick Howlett, MD, of the King’s Sierra Leone Partnership, Freetown, leads the neurology study. So far, the researchers have collected data on 19 patients with severe neurological consequences. Of those, 12 (63%) experienced a period of unconsciousness during their acute Ebola episode. In a comparator group of 21 with nonsevere neurologic sequelae, 33% had experienced unconsciousness.

Headache was present in nine (47%) of the patients. Migraine was the most common diagnosis. “We don’t have money for migraine medications, but fortunately, most of our migraine patients seem to be doing well on beta blockers,” Dr. Scott said.

CT scans were performed on 17 patients: three showed cerebral or cerebellar atrophy and two had confirmed stroke.

The brain injuries were severe in two, including a 42-year-old with extensive gliosis in the left middle cerebral artery region and a dilated left ventricle secondary to loss of volume in that hemisphere. A 12-year-old girl showed extensive parietal and temporal lobe atrophy. She is now so disabled that her family can’t care for her at home.

Other neurological problems include peripheral neuropathy, brachial plexus neuropathy, and asymmetric lower limb muscular atrophy.

Paul Steptoe, MD, an ophthalmic registrar from St. Paul’s Eye Unit at the Royal Liverpool Hospital, heads the eye study. He has observed dense cataracts, even in children, and anterior uveitis that has blinded some patients. There is concern about live virus persisting in vitreal fluid, but two eye taps have been negative, Dr. Scott said.

Courtesy Janet Scott, MD
Ibrahim Bangura (red shirt) is the project's data manager. Idriss Kamara manages data for Sierra Leone's blood bank and transfusion services.
Live virus, however, was recovered in 2015 from the eye of a U.S. physician who volunteered in Sierra Leone during the outbreak. During his recuperation from the illness, he developed an acute inflammatory reaction in one eye and began to lose his vision. His eye and visual acuity recovered after treatment with steroids and an investigational antiviral. A secondary tap was negative.

The most exciting recent finding, however, was made possible by the donation of a digital retinal camera, which “enabled us to get dozens of amazing images,” Dr. Scott said. With it, Dr. Steptoe conducted a case-control study of 81 Ebola survivors and 106 community controls. The findings of this study are potentially very, very important, Dr. Scott said.

“The first thing we found out is that retinal scarring is pervasive in our control patients,” she said. “There is just a lot of it out here in the community. But more interesting is that Dr. Steptoe seems to have identified a characteristic retinal lesion seen only in our survivors. It could be evidence of neurotropic aspects of the Ebola virus.”

The lesions occurred in 12 (15%) of the survivors and none of the controls. They are of a striking and consistent shape: straight-edged and sharply angulated. The lesions are only on the surface of the retina and do not penetrate into deeper levels. Nor do they interfere with vision. Dr. Steptoe has proposed that they take their angular shape from the retina’s underlying structures. His paper documenting this finding has been accepted and will be published shortly in the journal Emerging Infectious Diseases.

All of the post-Ebola sequelae add up to general disability for survivors, Dr. Scott said. Soushieta Jagadesh, of the* Liverpool School of Tropical Medicine, is conducting a disability survey. The comparison between 27 survivors and 54 community controls employed the Washington Group extended disability questionnaire. “We noted major limitations 1 year after discharge in mobility, vision, cognition, and affect,” Dr. Scott said.

The hazard ratios for these issues are enormous: Overall, compared with controls, survivors were 23 times more likely to have some level of disability. They were 94 times more likely to have walking limitations and 65 times more likely to have problems with stairs. Survivors were over 200 times more likely to have moderate disability than were their unaffected neighbors.

If funding for the project is renewed – and Dr. Scott admitted this is an “if,” not a “when” – caring for and studying these survivors will continue. Just in this one city, she said, the need is huge.

According to data from the U.S. Centers for Disease Control and Prevention, more than 17,000 patients in Sierra Leone, Liberia, and Guinea survived the 2014 Ebola outbreak.

If the assessments of Freetown survivors hold true across this population, thousands of survivors face life-limiting sequelae of the disease.

“We still have patients walk in every day with musculoskeletal pain, headaches, and ocular issues,” Dr. Scott said. “At the beginning of the epidemic, we were just focusing on containing it and reducing transmission. Now, we are faced with the long-term consequences.”

The Wellcome Trust supported the study. The authors have been awarded a grant from the Enhancing Research Activity in Epidemic Situations (ERAES) program, funded by the Wellcome Trust to support further research into the sequelae of Ebola virus disease.

The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
 

 

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– The Ebola crisis may be over in Sierra Leone, but the suffering is not.

The last patient from the epidemic was discharged in February 2016, but 78% of survivors now appear to have one or more sequelae of the infection. Some problems are mild, but some are so debilitating that life may never be the same.

Janet Scott, MD, of the University of Liverpool (England), heads a task force studying Ebola’s lingering aftereffects. These fall into four categories, Dr. Scott said at the European Society of Clinical Microbiology and Infectious Diseases annual congress: musculoskeletal pain, headache, eye problems, and psychological disorders.

They add up to an enormous risk of disability – survivors are more than 200 times more likely than controls to express at least moderate disability.

Courtesy Janet Scott, MD
Dr. Janet Scott, who cared for Ebola patients in Sierra Leone during the epidemic, is now following them to learn about the disease's aftereffects.


Dr. Scott and her team of researchers are partnering with clinicians and data managers in the Ebola treatment unit in the 34th Regimental Military Hospital (MH34) in Freetown, Sierra Leone. In Sierra Leone alone, she said, there were nearly 9,000 cases and 3,500 deaths from the virus; about 5,000 patients survived. So far, Dr. Scott and her team have collected data on about 500 patients for whom they also provide free health care.

The project has six arms, each headed by an expert: clinical care, data collection, disability, neurology, ophthalmology, and psychiatry.

The team sees patients in a large tent sectioned by a plywood wall*. Wireless Internet access, which she said is “enormously expensive” in Sierra Leone, has been donated by Omline Business Communications*. It’s the team’s lifeline, allowing them to transmit data between Freetown and participating units around the world. Members also Skype regularly, talking with patients and with each other.

All patients who come into the clinic have an initial visit that includes collection of demographics, their Ebola and clinical history (including an exploration of comorbidities), a maternal health screening for women, vital signs and symptom assessment, medication dispensing, and a treatment plan.

Then they visit the specialists, either onsite or through local referral*. These specialist modules include joints, eyes, headache, ears, neurology, cardiac, respiratory, gastrointestinal, renal and urologic, reproductive health for both genders, and psychiatry.

Courtesy Janet Scott, MD
The task force sees patients and processes collected data in this large tent.
Patients also fill out a disability questionnaire that asks them to report the presence or absence, and severity, of a variety of issues: visual problems, headache, balance problems, chest and abdominal pain, weakness, and gastrointestinal and urinary problems.

Last year, Dr. Scott published initial data on 44 patients. At ECCMID 2017, she expanded that report to include 203 survivors. They spanned all ages, but about 67% were in their most productive adult years, aged 20-39 years.

Her findings are striking: About 78% report musculoskeletal pain, with many saying they have trouble walking even short distances, climbing stairs, or picking up their children.

Headache was the next most common problem, reported by nearly 40%. About 15% report ocular problems, which include anterior uveitis, cataracts – even in very young children – and retinal lesions. Abdominal and chest pain affect about 10% of the survivors.

Although she didn’t present specific numbers, Dr. Scott also said that many of the survivors experience psychological sequelae, including insomnia, anxiety, and depression. Whether this is related to viral pathology isn’t clear; it could be a not-unexpected response to the trauma of living through the epidemic.

“Many of these people have lost their entire family, and those that are left now shun them,” she said in a live video interview
on Facebook. “It’s almost like a post-traumatic stress reaction.”

The other symptoms probably are related to the disease pathology, she observed. “Unfortunately, we don’t have all the clinical details of the acute phase for everyone, but for those for whom we do have details, we are seeing correlation between some of the problems with viral loads at admission, and even episodes of becoming unconscious during the acute illness.”

Patrick Howlett, MD, of the King’s Sierra Leone Partnership, Freetown, leads the neurology study. So far, the researchers have collected data on 19 patients with severe neurological consequences. Of those, 12 (63%) experienced a period of unconsciousness during their acute Ebola episode. In a comparator group of 21 with nonsevere neurologic sequelae, 33% had experienced unconsciousness.

Headache was present in nine (47%) of the patients. Migraine was the most common diagnosis. “We don’t have money for migraine medications, but fortunately, most of our migraine patients seem to be doing well on beta blockers,” Dr. Scott said.

CT scans were performed on 17 patients: three showed cerebral or cerebellar atrophy and two had confirmed stroke.

The brain injuries were severe in two, including a 42-year-old with extensive gliosis in the left middle cerebral artery region and a dilated left ventricle secondary to loss of volume in that hemisphere. A 12-year-old girl showed extensive parietal and temporal lobe atrophy. She is now so disabled that her family can’t care for her at home.

Other neurological problems include peripheral neuropathy, brachial plexus neuropathy, and asymmetric lower limb muscular atrophy.

Paul Steptoe, MD, an ophthalmic registrar from St. Paul’s Eye Unit at the Royal Liverpool Hospital, heads the eye study. He has observed dense cataracts, even in children, and anterior uveitis that has blinded some patients. There is concern about live virus persisting in vitreal fluid, but two eye taps have been negative, Dr. Scott said.

Courtesy Janet Scott, MD
Ibrahim Bangura (red shirt) is the project's data manager. Idriss Kamara manages data for Sierra Leone's blood bank and transfusion services.
Live virus, however, was recovered in 2015 from the eye of a U.S. physician who volunteered in Sierra Leone during the outbreak. During his recuperation from the illness, he developed an acute inflammatory reaction in one eye and began to lose his vision. His eye and visual acuity recovered after treatment with steroids and an investigational antiviral. A secondary tap was negative.

The most exciting recent finding, however, was made possible by the donation of a digital retinal camera, which “enabled us to get dozens of amazing images,” Dr. Scott said. With it, Dr. Steptoe conducted a case-control study of 81 Ebola survivors and 106 community controls. The findings of this study are potentially very, very important, Dr. Scott said.

“The first thing we found out is that retinal scarring is pervasive in our control patients,” she said. “There is just a lot of it out here in the community. But more interesting is that Dr. Steptoe seems to have identified a characteristic retinal lesion seen only in our survivors. It could be evidence of neurotropic aspects of the Ebola virus.”

The lesions occurred in 12 (15%) of the survivors and none of the controls. They are of a striking and consistent shape: straight-edged and sharply angulated. The lesions are only on the surface of the retina and do not penetrate into deeper levels. Nor do they interfere with vision. Dr. Steptoe has proposed that they take their angular shape from the retina’s underlying structures. His paper documenting this finding has been accepted and will be published shortly in the journal Emerging Infectious Diseases.

All of the post-Ebola sequelae add up to general disability for survivors, Dr. Scott said. Soushieta Jagadesh, of the* Liverpool School of Tropical Medicine, is conducting a disability survey. The comparison between 27 survivors and 54 community controls employed the Washington Group extended disability questionnaire. “We noted major limitations 1 year after discharge in mobility, vision, cognition, and affect,” Dr. Scott said.

The hazard ratios for these issues are enormous: Overall, compared with controls, survivors were 23 times more likely to have some level of disability. They were 94 times more likely to have walking limitations and 65 times more likely to have problems with stairs. Survivors were over 200 times more likely to have moderate disability than were their unaffected neighbors.

If funding for the project is renewed – and Dr. Scott admitted this is an “if,” not a “when” – caring for and studying these survivors will continue. Just in this one city, she said, the need is huge.

According to data from the U.S. Centers for Disease Control and Prevention, more than 17,000 patients in Sierra Leone, Liberia, and Guinea survived the 2014 Ebola outbreak.

If the assessments of Freetown survivors hold true across this population, thousands of survivors face life-limiting sequelae of the disease.

“We still have patients walk in every day with musculoskeletal pain, headaches, and ocular issues,” Dr. Scott said. “At the beginning of the epidemic, we were just focusing on containing it and reducing transmission. Now, we are faced with the long-term consequences.”

The Wellcome Trust supported the study. The authors have been awarded a grant from the Enhancing Research Activity in Epidemic Situations (ERAES) program, funded by the Wellcome Trust to support further research into the sequelae of Ebola virus disease.

The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
 

 

 

– The Ebola crisis may be over in Sierra Leone, but the suffering is not.

The last patient from the epidemic was discharged in February 2016, but 78% of survivors now appear to have one or more sequelae of the infection. Some problems are mild, but some are so debilitating that life may never be the same.

Janet Scott, MD, of the University of Liverpool (England), heads a task force studying Ebola’s lingering aftereffects. These fall into four categories, Dr. Scott said at the European Society of Clinical Microbiology and Infectious Diseases annual congress: musculoskeletal pain, headache, eye problems, and psychological disorders.

They add up to an enormous risk of disability – survivors are more than 200 times more likely than controls to express at least moderate disability.

Courtesy Janet Scott, MD
Dr. Janet Scott, who cared for Ebola patients in Sierra Leone during the epidemic, is now following them to learn about the disease's aftereffects.


Dr. Scott and her team of researchers are partnering with clinicians and data managers in the Ebola treatment unit in the 34th Regimental Military Hospital (MH34) in Freetown, Sierra Leone. In Sierra Leone alone, she said, there were nearly 9,000 cases and 3,500 deaths from the virus; about 5,000 patients survived. So far, Dr. Scott and her team have collected data on about 500 patients for whom they also provide free health care.

The project has six arms, each headed by an expert: clinical care, data collection, disability, neurology, ophthalmology, and psychiatry.

The team sees patients in a large tent sectioned by a plywood wall*. Wireless Internet access, which she said is “enormously expensive” in Sierra Leone, has been donated by Omline Business Communications*. It’s the team’s lifeline, allowing them to transmit data between Freetown and participating units around the world. Members also Skype regularly, talking with patients and with each other.

All patients who come into the clinic have an initial visit that includes collection of demographics, their Ebola and clinical history (including an exploration of comorbidities), a maternal health screening for women, vital signs and symptom assessment, medication dispensing, and a treatment plan.

Then they visit the specialists, either onsite or through local referral*. These specialist modules include joints, eyes, headache, ears, neurology, cardiac, respiratory, gastrointestinal, renal and urologic, reproductive health for both genders, and psychiatry.

Courtesy Janet Scott, MD
The task force sees patients and processes collected data in this large tent.
Patients also fill out a disability questionnaire that asks them to report the presence or absence, and severity, of a variety of issues: visual problems, headache, balance problems, chest and abdominal pain, weakness, and gastrointestinal and urinary problems.

Last year, Dr. Scott published initial data on 44 patients. At ECCMID 2017, she expanded that report to include 203 survivors. They spanned all ages, but about 67% were in their most productive adult years, aged 20-39 years.

Her findings are striking: About 78% report musculoskeletal pain, with many saying they have trouble walking even short distances, climbing stairs, or picking up their children.

Headache was the next most common problem, reported by nearly 40%. About 15% report ocular problems, which include anterior uveitis, cataracts – even in very young children – and retinal lesions. Abdominal and chest pain affect about 10% of the survivors.

Although she didn’t present specific numbers, Dr. Scott also said that many of the survivors experience psychological sequelae, including insomnia, anxiety, and depression. Whether this is related to viral pathology isn’t clear; it could be a not-unexpected response to the trauma of living through the epidemic.

“Many of these people have lost their entire family, and those that are left now shun them,” she said in a live video interview
on Facebook. “It’s almost like a post-traumatic stress reaction.”

The other symptoms probably are related to the disease pathology, she observed. “Unfortunately, we don’t have all the clinical details of the acute phase for everyone, but for those for whom we do have details, we are seeing correlation between some of the problems with viral loads at admission, and even episodes of becoming unconscious during the acute illness.”

Patrick Howlett, MD, of the King’s Sierra Leone Partnership, Freetown, leads the neurology study. So far, the researchers have collected data on 19 patients with severe neurological consequences. Of those, 12 (63%) experienced a period of unconsciousness during their acute Ebola episode. In a comparator group of 21 with nonsevere neurologic sequelae, 33% had experienced unconsciousness.

Headache was present in nine (47%) of the patients. Migraine was the most common diagnosis. “We don’t have money for migraine medications, but fortunately, most of our migraine patients seem to be doing well on beta blockers,” Dr. Scott said.

CT scans were performed on 17 patients: three showed cerebral or cerebellar atrophy and two had confirmed stroke.

The brain injuries were severe in two, including a 42-year-old with extensive gliosis in the left middle cerebral artery region and a dilated left ventricle secondary to loss of volume in that hemisphere. A 12-year-old girl showed extensive parietal and temporal lobe atrophy. She is now so disabled that her family can’t care for her at home.

Other neurological problems include peripheral neuropathy, brachial plexus neuropathy, and asymmetric lower limb muscular atrophy.

Paul Steptoe, MD, an ophthalmic registrar from St. Paul’s Eye Unit at the Royal Liverpool Hospital, heads the eye study. He has observed dense cataracts, even in children, and anterior uveitis that has blinded some patients. There is concern about live virus persisting in vitreal fluid, but two eye taps have been negative, Dr. Scott said.

Courtesy Janet Scott, MD
Ibrahim Bangura (red shirt) is the project's data manager. Idriss Kamara manages data for Sierra Leone's blood bank and transfusion services.
Live virus, however, was recovered in 2015 from the eye of a U.S. physician who volunteered in Sierra Leone during the outbreak. During his recuperation from the illness, he developed an acute inflammatory reaction in one eye and began to lose his vision. His eye and visual acuity recovered after treatment with steroids and an investigational antiviral. A secondary tap was negative.

The most exciting recent finding, however, was made possible by the donation of a digital retinal camera, which “enabled us to get dozens of amazing images,” Dr. Scott said. With it, Dr. Steptoe conducted a case-control study of 81 Ebola survivors and 106 community controls. The findings of this study are potentially very, very important, Dr. Scott said.

“The first thing we found out is that retinal scarring is pervasive in our control patients,” she said. “There is just a lot of it out here in the community. But more interesting is that Dr. Steptoe seems to have identified a characteristic retinal lesion seen only in our survivors. It could be evidence of neurotropic aspects of the Ebola virus.”

The lesions occurred in 12 (15%) of the survivors and none of the controls. They are of a striking and consistent shape: straight-edged and sharply angulated. The lesions are only on the surface of the retina and do not penetrate into deeper levels. Nor do they interfere with vision. Dr. Steptoe has proposed that they take their angular shape from the retina’s underlying structures. His paper documenting this finding has been accepted and will be published shortly in the journal Emerging Infectious Diseases.

All of the post-Ebola sequelae add up to general disability for survivors, Dr. Scott said. Soushieta Jagadesh, of the* Liverpool School of Tropical Medicine, is conducting a disability survey. The comparison between 27 survivors and 54 community controls employed the Washington Group extended disability questionnaire. “We noted major limitations 1 year after discharge in mobility, vision, cognition, and affect,” Dr. Scott said.

The hazard ratios for these issues are enormous: Overall, compared with controls, survivors were 23 times more likely to have some level of disability. They were 94 times more likely to have walking limitations and 65 times more likely to have problems with stairs. Survivors were over 200 times more likely to have moderate disability than were their unaffected neighbors.

If funding for the project is renewed – and Dr. Scott admitted this is an “if,” not a “when” – caring for and studying these survivors will continue. Just in this one city, she said, the need is huge.

According to data from the U.S. Centers for Disease Control and Prevention, more than 17,000 patients in Sierra Leone, Liberia, and Guinea survived the 2014 Ebola outbreak.

If the assessments of Freetown survivors hold true across this population, thousands of survivors face life-limiting sequelae of the disease.

“We still have patients walk in every day with musculoskeletal pain, headaches, and ocular issues,” Dr. Scott said. “At the beginning of the epidemic, we were just focusing on containing it and reducing transmission. Now, we are faced with the long-term consequences.”

The Wellcome Trust supported the study. The authors have been awarded a grant from the Enhancing Research Activity in Epidemic Situations (ERAES) program, funded by the Wellcome Trust to support further research into the sequelae of Ebola virus disease.

The video associated with this article is no longer available on this site. Please view all of our videos on the MDedge YouTube channel
 

 

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The Food and Drug Administration issued a postmarketing warning about potential for interaction between sofosbuvir and amiodarone. Nine patients taking sofosbuvir (with other antiviral agents) and amiodarone developed significant bradycardia. Seven patients were on concomitant beta-blockade. One patient died of cardiac arrest while three others required pacemaker placement. Two-thirds of the events occurred within 24 hours of coadministration while the other third occurred within 12 days. Three patients had recurrence of bradycardia with rechallenge of sofosbuvir treatment while on amiodarone. The mechanism of bradycardia is not fully understood. Amiodarone is considered an absolute contraindication to the use of a sofosbuvir-containing regimen. The sofosbuvir-containing regimens listed are endorsed by the AASLD/IDSA joint guidelines for treatment of genotype 1a hepatitis C, as long as the patient is not on amiodarone, although the combination of sofosbuvir and daclatasvir is not FDA approved for genotype 1.

 

Reference

1. Fontaine H, Lazarus A, Pol S, et al.; Cochin Hepatology and Cardiology Group. N Engl J Med. 2015 Nov 5;373(19):1886-8.

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Q1: Answer: A

The Food and Drug Administration issued a postmarketing warning about potential for interaction between sofosbuvir and amiodarone. Nine patients taking sofosbuvir (with other antiviral agents) and amiodarone developed significant bradycardia. Seven patients were on concomitant beta-blockade. One patient died of cardiac arrest while three others required pacemaker placement. Two-thirds of the events occurred within 24 hours of coadministration while the other third occurred within 12 days. Three patients had recurrence of bradycardia with rechallenge of sofosbuvir treatment while on amiodarone. The mechanism of bradycardia is not fully understood. Amiodarone is considered an absolute contraindication to the use of a sofosbuvir-containing regimen. The sofosbuvir-containing regimens listed are endorsed by the AASLD/IDSA joint guidelines for treatment of genotype 1a hepatitis C, as long as the patient is not on amiodarone, although the combination of sofosbuvir and daclatasvir is not FDA approved for genotype 1.

 

Reference

1. Fontaine H, Lazarus A, Pol S, et al.; Cochin Hepatology and Cardiology Group. N Engl J Med. 2015 Nov 5;373(19):1886-8.

Q1: Answer: A

The Food and Drug Administration issued a postmarketing warning about potential for interaction between sofosbuvir and amiodarone. Nine patients taking sofosbuvir (with other antiviral agents) and amiodarone developed significant bradycardia. Seven patients were on concomitant beta-blockade. One patient died of cardiac arrest while three others required pacemaker placement. Two-thirds of the events occurred within 24 hours of coadministration while the other third occurred within 12 days. Three patients had recurrence of bradycardia with rechallenge of sofosbuvir treatment while on amiodarone. The mechanism of bradycardia is not fully understood. Amiodarone is considered an absolute contraindication to the use of a sofosbuvir-containing regimen. The sofosbuvir-containing regimens listed are endorsed by the AASLD/IDSA joint guidelines for treatment of genotype 1a hepatitis C, as long as the patient is not on amiodarone, although the combination of sofosbuvir and daclatasvir is not FDA approved for genotype 1.

 

Reference

1. Fontaine H, Lazarus A, Pol S, et al.; Cochin Hepatology and Cardiology Group. N Engl J Med. 2015 Nov 5;373(19):1886-8.

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Q1. A 58-year-old woman with genotype 1a HCV presents for reevaluation. She is treatment naive and a recent transient elastography reveals stage 3 fibrosis. Her past medical history is notable for atrial fibrillation, hypertension, and dyslipidemia. Medications include amiodarone, lisinopril, and atorvastatin.

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High readmits after peripheral arterial procedures

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– More than one in six patients who undergo a lower extremity arterial endovascular or surgical procedure are readmitted within 30 days, according to a large national study.

The annual total cost of these early readmissions is high, in excess of $360 million. But because there turned out to be surprisingly little difference in readmission rates between hospitals, 30-day readmissions may not be a rational quality measure on which to base institutional reimbursement or withholding of payment for peripheral arterial interventions, Eric A. Secemsky, MD, said at the annual meeting of the American College of Cardiology.

Bruce Jancin/Frontline Medical News
Dr. Eric A. Secemsky
He analyzed 123,799 hospitalizations for lower extremity endovascular or surgical procedures at 1,100 hospitals included in the Nationwide Readmissions Database for January 2013–November 2014. The all-payer database covers 22 states and includes 49% of all U.S. hospitalizations.

Forty-seven percent of patients had an endovascular procedure, 42% had surgery, and the remainder had hybrid procedures in which both endovascular and surgical interventions took place during the same admission. Patients with hybrid procedures contributed data to both treatment groups.

In-hospital mortality occurred in 2.5% of patients.

Of the patients who survived to discharge, 21,589, or 17.4%, were readmitted within 30 days. The early readmission rate was higher following endovascular procedures, at 18.7%, than the 16.1% rate in the surgical group. The average cost of a readmission was $15,876. Death during readmission occurred in 4.2% of patients.

The median rate ratio – a measure of the amount of variance in readmission rates between hospitals – was 1.12. That’s a low figure.

“If the median rate ratio is lower, like here, it says there’s not a lot of interhospital variability across the country. So overall this burden seems to be pretty uniform across the institutions included in our analysis,” Dr. Secemsky explained.

This observation drew the attention of session comoderator Naomi M. Hamburg, MD.

“It’s interesting that you didn’t see a lot of heterogeneity across hospitals, because we often think of readmissions as a potentially modifiable quality metric. Do you think it’s modifiable, or is this just the nature of the disease?” asked Dr. Hamburg of Boston Medical Center.

It’s the disease process, Dr. Secemsky replied.

“We were surprised by the lack of hospital variation,” he added. None of the institutional characteristics examined, including teaching hospital status, bed size, and procedural volume, had a significant impact on readmission rates.

But that doesn’t mean there aren’t opportunities to whittle down those readmissions, according to Dr. Secemsky.

He noted that the high readmission rates were driven by procedural complications such as graft or stent failure. Indeed, procedural complications accounted for 29% of all early readmissions. The procedural complication rate was about 20% following endovascular procedures and 39% after surgery. It’s likely that identification and implementation of best practices could trim those high rates. Unfortunately, however, the nationwide database relies upon ICD-9 codes, which don’t provide the granular level of detail required to home in on specific best practices. That will require further studies, according to Dr. Secemsky.

A distant second on the list of causes of early readmission was peripheral atherosclerosis, meaning persistent claudication or rest pain. This accounted for 8.8% of readmissions. Rounding out the top five causes of readmission were sepsis, which was the reason for 6.7% of readmissions; diabetes with complications, at 4.7%; and heart failure, at 4.6%.

The strongest predictors of readmission included having renal disease at baseline, Medicare rather than private insurance, and discharge to a subacute nursing facility or home with home care.

Dr. Hamburg commented that a focus on reducing readmissions for sepsis as well as for skin and soft tissue infections, which accounted for 2.1% of 30-day hospitalizations, could be fruitful.

Dr. Secemsky reported having no financial conflicts regarding his study.

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– More than one in six patients who undergo a lower extremity arterial endovascular or surgical procedure are readmitted within 30 days, according to a large national study.

The annual total cost of these early readmissions is high, in excess of $360 million. But because there turned out to be surprisingly little difference in readmission rates between hospitals, 30-day readmissions may not be a rational quality measure on which to base institutional reimbursement or withholding of payment for peripheral arterial interventions, Eric A. Secemsky, MD, said at the annual meeting of the American College of Cardiology.

Bruce Jancin/Frontline Medical News
Dr. Eric A. Secemsky
He analyzed 123,799 hospitalizations for lower extremity endovascular or surgical procedures at 1,100 hospitals included in the Nationwide Readmissions Database for January 2013–November 2014. The all-payer database covers 22 states and includes 49% of all U.S. hospitalizations.

Forty-seven percent of patients had an endovascular procedure, 42% had surgery, and the remainder had hybrid procedures in which both endovascular and surgical interventions took place during the same admission. Patients with hybrid procedures contributed data to both treatment groups.

In-hospital mortality occurred in 2.5% of patients.

Of the patients who survived to discharge, 21,589, or 17.4%, were readmitted within 30 days. The early readmission rate was higher following endovascular procedures, at 18.7%, than the 16.1% rate in the surgical group. The average cost of a readmission was $15,876. Death during readmission occurred in 4.2% of patients.

The median rate ratio – a measure of the amount of variance in readmission rates between hospitals – was 1.12. That’s a low figure.

“If the median rate ratio is lower, like here, it says there’s not a lot of interhospital variability across the country. So overall this burden seems to be pretty uniform across the institutions included in our analysis,” Dr. Secemsky explained.

This observation drew the attention of session comoderator Naomi M. Hamburg, MD.

“It’s interesting that you didn’t see a lot of heterogeneity across hospitals, because we often think of readmissions as a potentially modifiable quality metric. Do you think it’s modifiable, or is this just the nature of the disease?” asked Dr. Hamburg of Boston Medical Center.

It’s the disease process, Dr. Secemsky replied.

“We were surprised by the lack of hospital variation,” he added. None of the institutional characteristics examined, including teaching hospital status, bed size, and procedural volume, had a significant impact on readmission rates.

But that doesn’t mean there aren’t opportunities to whittle down those readmissions, according to Dr. Secemsky.

He noted that the high readmission rates were driven by procedural complications such as graft or stent failure. Indeed, procedural complications accounted for 29% of all early readmissions. The procedural complication rate was about 20% following endovascular procedures and 39% after surgery. It’s likely that identification and implementation of best practices could trim those high rates. Unfortunately, however, the nationwide database relies upon ICD-9 codes, which don’t provide the granular level of detail required to home in on specific best practices. That will require further studies, according to Dr. Secemsky.

A distant second on the list of causes of early readmission was peripheral atherosclerosis, meaning persistent claudication or rest pain. This accounted for 8.8% of readmissions. Rounding out the top five causes of readmission were sepsis, which was the reason for 6.7% of readmissions; diabetes with complications, at 4.7%; and heart failure, at 4.6%.

The strongest predictors of readmission included having renal disease at baseline, Medicare rather than private insurance, and discharge to a subacute nursing facility or home with home care.

Dr. Hamburg commented that a focus on reducing readmissions for sepsis as well as for skin and soft tissue infections, which accounted for 2.1% of 30-day hospitalizations, could be fruitful.

Dr. Secemsky reported having no financial conflicts regarding his study.

 

– More than one in six patients who undergo a lower extremity arterial endovascular or surgical procedure are readmitted within 30 days, according to a large national study.

The annual total cost of these early readmissions is high, in excess of $360 million. But because there turned out to be surprisingly little difference in readmission rates between hospitals, 30-day readmissions may not be a rational quality measure on which to base institutional reimbursement or withholding of payment for peripheral arterial interventions, Eric A. Secemsky, MD, said at the annual meeting of the American College of Cardiology.

Bruce Jancin/Frontline Medical News
Dr. Eric A. Secemsky
He analyzed 123,799 hospitalizations for lower extremity endovascular or surgical procedures at 1,100 hospitals included in the Nationwide Readmissions Database for January 2013–November 2014. The all-payer database covers 22 states and includes 49% of all U.S. hospitalizations.

Forty-seven percent of patients had an endovascular procedure, 42% had surgery, and the remainder had hybrid procedures in which both endovascular and surgical interventions took place during the same admission. Patients with hybrid procedures contributed data to both treatment groups.

In-hospital mortality occurred in 2.5% of patients.

Of the patients who survived to discharge, 21,589, or 17.4%, were readmitted within 30 days. The early readmission rate was higher following endovascular procedures, at 18.7%, than the 16.1% rate in the surgical group. The average cost of a readmission was $15,876. Death during readmission occurred in 4.2% of patients.

The median rate ratio – a measure of the amount of variance in readmission rates between hospitals – was 1.12. That’s a low figure.

“If the median rate ratio is lower, like here, it says there’s not a lot of interhospital variability across the country. So overall this burden seems to be pretty uniform across the institutions included in our analysis,” Dr. Secemsky explained.

This observation drew the attention of session comoderator Naomi M. Hamburg, MD.

“It’s interesting that you didn’t see a lot of heterogeneity across hospitals, because we often think of readmissions as a potentially modifiable quality metric. Do you think it’s modifiable, or is this just the nature of the disease?” asked Dr. Hamburg of Boston Medical Center.

It’s the disease process, Dr. Secemsky replied.

“We were surprised by the lack of hospital variation,” he added. None of the institutional characteristics examined, including teaching hospital status, bed size, and procedural volume, had a significant impact on readmission rates.

But that doesn’t mean there aren’t opportunities to whittle down those readmissions, according to Dr. Secemsky.

He noted that the high readmission rates were driven by procedural complications such as graft or stent failure. Indeed, procedural complications accounted for 29% of all early readmissions. The procedural complication rate was about 20% following endovascular procedures and 39% after surgery. It’s likely that identification and implementation of best practices could trim those high rates. Unfortunately, however, the nationwide database relies upon ICD-9 codes, which don’t provide the granular level of detail required to home in on specific best practices. That will require further studies, according to Dr. Secemsky.

A distant second on the list of causes of early readmission was peripheral atherosclerosis, meaning persistent claudication or rest pain. This accounted for 8.8% of readmissions. Rounding out the top five causes of readmission were sepsis, which was the reason for 6.7% of readmissions; diabetes with complications, at 4.7%; and heart failure, at 4.6%.

The strongest predictors of readmission included having renal disease at baseline, Medicare rather than private insurance, and discharge to a subacute nursing facility or home with home care.

Dr. Hamburg commented that a focus on reducing readmissions for sepsis as well as for skin and soft tissue infections, which accounted for 2.1% of 30-day hospitalizations, could be fruitful.

Dr. Secemsky reported having no financial conflicts regarding his study.

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Key clinical point: Rates of early readmission following endovascular or surgical procedures for peripheral arterial disease are consistently high at hospitals across the country.

Major finding: Readmission within 30 days after a peripheral arterial procedure occurred nationally in 17.4% of patients, with little between-hospital variation in rates.

Data source: A retrospective analysis of nearly 124,000 hospital admissions for lower extremity arterial endovascular or surgical procedures.

Disclosures: The study presenter reported having no financial conflicts of interest.

Drugmakers Dramatically Boosted Lobbying Spending In Trump’s First Quarter

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Eight pharmaceutical companies more than doubled their lobbying spending in the first three months of 2017, when the Affordable Care Act was on the chopping block and high drug prices were clearly in the crosshairs of Congress and President Donald Trump.

Congressional records show that those eight, including Celgene and Mylan, kicked in an extra $4.42 million versus that quarter last year. Industry giant Teva Pharmaceutical Industries spent $2.67 million, up 115% from a year ago as several companies embroiled in controversies raised their outlays significantly.

“It’s certainly a rare event” when lobbying dollars double, noted Timothy LaPira, PhD, an associate professor of political science at James Madison University. “These spikes are usually timed when Congress in particular is going to be really hammering home on a particular issue. Right now, that’s health care and taxes.”

Trump has come down hard on drugmakers, stating in a press conference before his inauguration that the industry is “getting away with murder.” He has promised to lower drug prices and increase competition with faster approvals and fewer regulations. Sen. Bernie Sanders (I-Vt.), Sen. John McCain (R-Ariz.), and Rep. Elijah E. Cummings (D-Md.) have introduced bills to allow lower-cost drug imports from Canada or other countries.

Lobbyists weren’t expecting much by way of big policy changes during the comparatively sleepy end of the Obama administration this time last year, but, with a surprise Trump administration and a Republican-controlled House and Senate, trade groups and companies are probably “going all in,” Dr. LaPira said.

Thirty-eight major drugmakers and trade groups spent a total of $50.9 million, up $10.1 million from the first quarter of last year, according to a Kaiser Health News analysis. They deployed 600 lobbyists in all.

PhRMA, the drug industry’s largest trade group, spent $7.98 million during the quarter – more than in any single quarter in almost a decade, congressional records show, topping even its quarterly lobbying ahead of the Affordable Care Act’s passage in 2010.

In their congressional disclosures, companies listed Medicare price negotiation, the American Health Care Act, drug importation, and the orphan drug program as issues they were lobbying for or against. They do not have to disclose on which side of an issue they lobbied.

When Medicare prices are on the table, it should come as no surprise that pharmaceutical companies are interested in influencing congress.

“It’s quite literally hitting their bottom line,” LaPira said.

Drugmakers, under fire, more than doubled their lobbying dollars. Mylan spent $1.45 million during the quarter, up from $610,000 last year. The company’s CEO faced a congressional hearing in the fall when it raised the price of EpiPen to over $600.

Marathon Pharmaceuticals spent $230,000, which was $120,000 more than last year. Marathon was criticized in February after setting the price of Emflaza, a steroid to treat Duchenne muscular dystrophy, at $89,000 a year. That angered advocates, Congress, and patients who had been importing the same drug for as little as $1,000 a year. Marathon has since sold the drug to another company, and the price may come down.

Teva and Shire also more than doubled their spending. Teva was accused, as part of an alleged generic price-fixing scheme in December, and the Federal Trade Commission sued Shire because one of its recently acquired companies allegedly filed “sham” petitions with the Food and Drug Administration to stave off generics.

Companies that make drugs for rare diseases also more than doubled lobbying dollars as congressional leaders and the Government Accountability Office work to determine whether the Orphan Drug Act is being abused. Those firms include BioMarin, Celgene, and Vertex Pharmaceuticals. Celgene, which makes a rare cancer drug, more than tripled its first quarter lobbying to more than $1 million.

Despite efforts to make good on campaign promises to repeal the Affordable Care Act, House Republicans canceled a floor vote on the American Health Care Act in March after multiple studies estimated that millions of people would lose coverage if it passed, and neither Democrats nor ultraconservatives lined up in opposition to the bill’s provisions. Drug prices weren’t a key part of the package.
 

KHN’s coverage of prescription drug development, costs, and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

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Eight pharmaceutical companies more than doubled their lobbying spending in the first three months of 2017, when the Affordable Care Act was on the chopping block and high drug prices were clearly in the crosshairs of Congress and President Donald Trump.

Congressional records show that those eight, including Celgene and Mylan, kicked in an extra $4.42 million versus that quarter last year. Industry giant Teva Pharmaceutical Industries spent $2.67 million, up 115% from a year ago as several companies embroiled in controversies raised their outlays significantly.

“It’s certainly a rare event” when lobbying dollars double, noted Timothy LaPira, PhD, an associate professor of political science at James Madison University. “These spikes are usually timed when Congress in particular is going to be really hammering home on a particular issue. Right now, that’s health care and taxes.”

Trump has come down hard on drugmakers, stating in a press conference before his inauguration that the industry is “getting away with murder.” He has promised to lower drug prices and increase competition with faster approvals and fewer regulations. Sen. Bernie Sanders (I-Vt.), Sen. John McCain (R-Ariz.), and Rep. Elijah E. Cummings (D-Md.) have introduced bills to allow lower-cost drug imports from Canada or other countries.

Lobbyists weren’t expecting much by way of big policy changes during the comparatively sleepy end of the Obama administration this time last year, but, with a surprise Trump administration and a Republican-controlled House and Senate, trade groups and companies are probably “going all in,” Dr. LaPira said.

Thirty-eight major drugmakers and trade groups spent a total of $50.9 million, up $10.1 million from the first quarter of last year, according to a Kaiser Health News analysis. They deployed 600 lobbyists in all.

PhRMA, the drug industry’s largest trade group, spent $7.98 million during the quarter – more than in any single quarter in almost a decade, congressional records show, topping even its quarterly lobbying ahead of the Affordable Care Act’s passage in 2010.

In their congressional disclosures, companies listed Medicare price negotiation, the American Health Care Act, drug importation, and the orphan drug program as issues they were lobbying for or against. They do not have to disclose on which side of an issue they lobbied.

When Medicare prices are on the table, it should come as no surprise that pharmaceutical companies are interested in influencing congress.

“It’s quite literally hitting their bottom line,” LaPira said.

Drugmakers, under fire, more than doubled their lobbying dollars. Mylan spent $1.45 million during the quarter, up from $610,000 last year. The company’s CEO faced a congressional hearing in the fall when it raised the price of EpiPen to over $600.

Marathon Pharmaceuticals spent $230,000, which was $120,000 more than last year. Marathon was criticized in February after setting the price of Emflaza, a steroid to treat Duchenne muscular dystrophy, at $89,000 a year. That angered advocates, Congress, and patients who had been importing the same drug for as little as $1,000 a year. Marathon has since sold the drug to another company, and the price may come down.

Teva and Shire also more than doubled their spending. Teva was accused, as part of an alleged generic price-fixing scheme in December, and the Federal Trade Commission sued Shire because one of its recently acquired companies allegedly filed “sham” petitions with the Food and Drug Administration to stave off generics.

Companies that make drugs for rare diseases also more than doubled lobbying dollars as congressional leaders and the Government Accountability Office work to determine whether the Orphan Drug Act is being abused. Those firms include BioMarin, Celgene, and Vertex Pharmaceuticals. Celgene, which makes a rare cancer drug, more than tripled its first quarter lobbying to more than $1 million.

Despite efforts to make good on campaign promises to repeal the Affordable Care Act, House Republicans canceled a floor vote on the American Health Care Act in March after multiple studies estimated that millions of people would lose coverage if it passed, and neither Democrats nor ultraconservatives lined up in opposition to the bill’s provisions. Drug prices weren’t a key part of the package.
 

KHN’s coverage of prescription drug development, costs, and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

 

Eight pharmaceutical companies more than doubled their lobbying spending in the first three months of 2017, when the Affordable Care Act was on the chopping block and high drug prices were clearly in the crosshairs of Congress and President Donald Trump.

Congressional records show that those eight, including Celgene and Mylan, kicked in an extra $4.42 million versus that quarter last year. Industry giant Teva Pharmaceutical Industries spent $2.67 million, up 115% from a year ago as several companies embroiled in controversies raised their outlays significantly.

“It’s certainly a rare event” when lobbying dollars double, noted Timothy LaPira, PhD, an associate professor of political science at James Madison University. “These spikes are usually timed when Congress in particular is going to be really hammering home on a particular issue. Right now, that’s health care and taxes.”

Trump has come down hard on drugmakers, stating in a press conference before his inauguration that the industry is “getting away with murder.” He has promised to lower drug prices and increase competition with faster approvals and fewer regulations. Sen. Bernie Sanders (I-Vt.), Sen. John McCain (R-Ariz.), and Rep. Elijah E. Cummings (D-Md.) have introduced bills to allow lower-cost drug imports from Canada or other countries.

Lobbyists weren’t expecting much by way of big policy changes during the comparatively sleepy end of the Obama administration this time last year, but, with a surprise Trump administration and a Republican-controlled House and Senate, trade groups and companies are probably “going all in,” Dr. LaPira said.

Thirty-eight major drugmakers and trade groups spent a total of $50.9 million, up $10.1 million from the first quarter of last year, according to a Kaiser Health News analysis. They deployed 600 lobbyists in all.

PhRMA, the drug industry’s largest trade group, spent $7.98 million during the quarter – more than in any single quarter in almost a decade, congressional records show, topping even its quarterly lobbying ahead of the Affordable Care Act’s passage in 2010.

In their congressional disclosures, companies listed Medicare price negotiation, the American Health Care Act, drug importation, and the orphan drug program as issues they were lobbying for or against. They do not have to disclose on which side of an issue they lobbied.

When Medicare prices are on the table, it should come as no surprise that pharmaceutical companies are interested in influencing congress.

“It’s quite literally hitting their bottom line,” LaPira said.

Drugmakers, under fire, more than doubled their lobbying dollars. Mylan spent $1.45 million during the quarter, up from $610,000 last year. The company’s CEO faced a congressional hearing in the fall when it raised the price of EpiPen to over $600.

Marathon Pharmaceuticals spent $230,000, which was $120,000 more than last year. Marathon was criticized in February after setting the price of Emflaza, a steroid to treat Duchenne muscular dystrophy, at $89,000 a year. That angered advocates, Congress, and patients who had been importing the same drug for as little as $1,000 a year. Marathon has since sold the drug to another company, and the price may come down.

Teva and Shire also more than doubled their spending. Teva was accused, as part of an alleged generic price-fixing scheme in December, and the Federal Trade Commission sued Shire because one of its recently acquired companies allegedly filed “sham” petitions with the Food and Drug Administration to stave off generics.

Companies that make drugs for rare diseases also more than doubled lobbying dollars as congressional leaders and the Government Accountability Office work to determine whether the Orphan Drug Act is being abused. Those firms include BioMarin, Celgene, and Vertex Pharmaceuticals. Celgene, which makes a rare cancer drug, more than tripled its first quarter lobbying to more than $1 million.

Despite efforts to make good on campaign promises to repeal the Affordable Care Act, House Republicans canceled a floor vote on the American Health Care Act in March after multiple studies estimated that millions of people would lose coverage if it passed, and neither Democrats nor ultraconservatives lined up in opposition to the bill’s provisions. Drug prices weren’t a key part of the package.
 

KHN’s coverage of prescription drug development, costs, and pricing is supported in part by the Laura and John Arnold Foundation. Kaiser Health News is a national health policy news service that is part of the nonpartisan Henry J. Kaiser Family Foundation.

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