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Mitchel is a reporter for MDedge based in the Philadelphia area. He started with the company in 1992, when it was International Medical News Group (IMNG), and has since covered a range of medical specialties. Mitchel trained as a virologist at Roswell Park Memorial Institute in Buffalo, and then worked briefly as a researcher at Boston Children's Hospital before pivoting to journalism as a AAAS Mass Media Fellow in 1980. His first reporting job was with Science Digest magazine, and from the mid-1980s to early-1990s he was a reporter with Medical World News. @mitchelzoler
Vedolizumab shows safety, efficacy for pediatric IBD
MONTREAL – Vedolizumab, 2 years out from its entry onto the U.S. market as an option for treating adults with inflammatory bowel disease, also has shown early safety and efficacy in 52 pediatric patients with ulcerative colitis or Crohn’s disease.
Retrospective review of a patients series assembled from three U.S. centers showed 13 (76%) ulcerative colitis patients in remission among 17 treated and followed for 14 weeks, the study’s primary outcome. Among 24 Crohn’s disease patients treated and followed for 14 weeks, 10 (42%) had remissions, Namita Singh, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition.
Treatment with vedolizumab (Entyvio), an anti-integrin with highly gut-specific activity that limits its systemic effects, was especially potent for the five patients in the series who were naive to treatment with an anti–tumor necrosis factor (TNF) agent. All five patients were in remission at 14 weeks, said Dr. Singh, a pediatric gastroenterologist at Cedars-Sinai Medical Center, Los Angeles.
“From this and other data it seems like there is a potential role for vedolizumab as first-line treatment” for selected patients, Dr. Singh said in an interview. “The attraction of vedolizumab is its gut selectivity.”
The American Gastroenterological Association lists vedolizumab as an equal alternative to an anti-TNF agent in its recommended algorithm for treating ulcerative colitis, but vedolizumab is not mentioned in the association’s posted guidance for treating Crohn’s disease. ”Prospective studies are needed” to generate more definitive evidence on how to use vedolizumab, she acknowledged, but added that pediatric gastroenterological societies “need to come up with guidelines on where to place vedolizumab.”Dr. Singh said she discusses with patients and their families the potential risks and benefits of the various treatment options available when inflammatory bowel disease (IBD) doesn’t respond to mesalamine: an anti-TNF agent, an immunomodulator like methotrexate or azathioprine, or vedolizumab. One important issue when deciding which drug class to try first is the cost of treatment and whether it will be covered by insurance.
The combined series included pediatric IBD patients less than 18 years old, with an actual median age of just under 15 years. They had been diagnosed with ulcerative colitis or Crohn’s disease for a median of 3 years. The 30 Crohn’s disease patients had received a median of two anti-TNF agents prior to starting vedolizumab, while the ulcerative colitis patients had received a median of one anti-TNF drug before vedolizumab. Three-quarters of the patients received the adult dose of 300 mg per infusion. A fifth of the patients received a dosage of 6 mg/kg, and the remaining patients received 5 mg/kg.
Although the 24 Crohn’s disease patients followed through 14 weeks of therapy had a 42% remission rate, the remission rate jumped sharply to more than 70% among the 11 patients followed to 30 weeks.
One limitation to vedolizumab is its relatively slow onset of action, slower than anti-TNF agents, which makes vedolizumab less suitable for patients with acute, severe colitis, although short-term improvement of acute colitis can be achieved with a corticosteroid when starting a patient on vedolizumab, Dr. Singh said. A trial currently underway is collecting prospective data on vedolizumab in pediatric IBD patients that could result in pediatric labeling for the drug, she added.
Until those data are available, current experience suggests vedolizumab “is good for ulcerative colitis patients,” she said. “Our data encourage us to continue” offering vedolizumab to selected pediatric IBD patients.
Dr. Singh has been a consultant to Janssen and Prometheus and received grant support from Janssen.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
MONTREAL – Vedolizumab, 2 years out from its entry onto the U.S. market as an option for treating adults with inflammatory bowel disease, also has shown early safety and efficacy in 52 pediatric patients with ulcerative colitis or Crohn’s disease.
Retrospective review of a patients series assembled from three U.S. centers showed 13 (76%) ulcerative colitis patients in remission among 17 treated and followed for 14 weeks, the study’s primary outcome. Among 24 Crohn’s disease patients treated and followed for 14 weeks, 10 (42%) had remissions, Namita Singh, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition.
Treatment with vedolizumab (Entyvio), an anti-integrin with highly gut-specific activity that limits its systemic effects, was especially potent for the five patients in the series who were naive to treatment with an anti–tumor necrosis factor (TNF) agent. All five patients were in remission at 14 weeks, said Dr. Singh, a pediatric gastroenterologist at Cedars-Sinai Medical Center, Los Angeles.
“From this and other data it seems like there is a potential role for vedolizumab as first-line treatment” for selected patients, Dr. Singh said in an interview. “The attraction of vedolizumab is its gut selectivity.”
The American Gastroenterological Association lists vedolizumab as an equal alternative to an anti-TNF agent in its recommended algorithm for treating ulcerative colitis, but vedolizumab is not mentioned in the association’s posted guidance for treating Crohn’s disease. ”Prospective studies are needed” to generate more definitive evidence on how to use vedolizumab, she acknowledged, but added that pediatric gastroenterological societies “need to come up with guidelines on where to place vedolizumab.”Dr. Singh said she discusses with patients and their families the potential risks and benefits of the various treatment options available when inflammatory bowel disease (IBD) doesn’t respond to mesalamine: an anti-TNF agent, an immunomodulator like methotrexate or azathioprine, or vedolizumab. One important issue when deciding which drug class to try first is the cost of treatment and whether it will be covered by insurance.
The combined series included pediatric IBD patients less than 18 years old, with an actual median age of just under 15 years. They had been diagnosed with ulcerative colitis or Crohn’s disease for a median of 3 years. The 30 Crohn’s disease patients had received a median of two anti-TNF agents prior to starting vedolizumab, while the ulcerative colitis patients had received a median of one anti-TNF drug before vedolizumab. Three-quarters of the patients received the adult dose of 300 mg per infusion. A fifth of the patients received a dosage of 6 mg/kg, and the remaining patients received 5 mg/kg.
Although the 24 Crohn’s disease patients followed through 14 weeks of therapy had a 42% remission rate, the remission rate jumped sharply to more than 70% among the 11 patients followed to 30 weeks.
One limitation to vedolizumab is its relatively slow onset of action, slower than anti-TNF agents, which makes vedolizumab less suitable for patients with acute, severe colitis, although short-term improvement of acute colitis can be achieved with a corticosteroid when starting a patient on vedolizumab, Dr. Singh said. A trial currently underway is collecting prospective data on vedolizumab in pediatric IBD patients that could result in pediatric labeling for the drug, she added.
Until those data are available, current experience suggests vedolizumab “is good for ulcerative colitis patients,” she said. “Our data encourage us to continue” offering vedolizumab to selected pediatric IBD patients.
Dr. Singh has been a consultant to Janssen and Prometheus and received grant support from Janssen.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
MONTREAL – Vedolizumab, 2 years out from its entry onto the U.S. market as an option for treating adults with inflammatory bowel disease, also has shown early safety and efficacy in 52 pediatric patients with ulcerative colitis or Crohn’s disease.
Retrospective review of a patients series assembled from three U.S. centers showed 13 (76%) ulcerative colitis patients in remission among 17 treated and followed for 14 weeks, the study’s primary outcome. Among 24 Crohn’s disease patients treated and followed for 14 weeks, 10 (42%) had remissions, Namita Singh, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition.
Treatment with vedolizumab (Entyvio), an anti-integrin with highly gut-specific activity that limits its systemic effects, was especially potent for the five patients in the series who were naive to treatment with an anti–tumor necrosis factor (TNF) agent. All five patients were in remission at 14 weeks, said Dr. Singh, a pediatric gastroenterologist at Cedars-Sinai Medical Center, Los Angeles.
“From this and other data it seems like there is a potential role for vedolizumab as first-line treatment” for selected patients, Dr. Singh said in an interview. “The attraction of vedolizumab is its gut selectivity.”
The American Gastroenterological Association lists vedolizumab as an equal alternative to an anti-TNF agent in its recommended algorithm for treating ulcerative colitis, but vedolizumab is not mentioned in the association’s posted guidance for treating Crohn’s disease. ”Prospective studies are needed” to generate more definitive evidence on how to use vedolizumab, she acknowledged, but added that pediatric gastroenterological societies “need to come up with guidelines on where to place vedolizumab.”Dr. Singh said she discusses with patients and their families the potential risks and benefits of the various treatment options available when inflammatory bowel disease (IBD) doesn’t respond to mesalamine: an anti-TNF agent, an immunomodulator like methotrexate or azathioprine, or vedolizumab. One important issue when deciding which drug class to try first is the cost of treatment and whether it will be covered by insurance.
The combined series included pediatric IBD patients less than 18 years old, with an actual median age of just under 15 years. They had been diagnosed with ulcerative colitis or Crohn’s disease for a median of 3 years. The 30 Crohn’s disease patients had received a median of two anti-TNF agents prior to starting vedolizumab, while the ulcerative colitis patients had received a median of one anti-TNF drug before vedolizumab. Three-quarters of the patients received the adult dose of 300 mg per infusion. A fifth of the patients received a dosage of 6 mg/kg, and the remaining patients received 5 mg/kg.
Although the 24 Crohn’s disease patients followed through 14 weeks of therapy had a 42% remission rate, the remission rate jumped sharply to more than 70% among the 11 patients followed to 30 weeks.
One limitation to vedolizumab is its relatively slow onset of action, slower than anti-TNF agents, which makes vedolizumab less suitable for patients with acute, severe colitis, although short-term improvement of acute colitis can be achieved with a corticosteroid when starting a patient on vedolizumab, Dr. Singh said. A trial currently underway is collecting prospective data on vedolizumab in pediatric IBD patients that could result in pediatric labeling for the drug, she added.
Until those data are available, current experience suggests vedolizumab “is good for ulcerative colitis patients,” she said. “Our data encourage us to continue” offering vedolizumab to selected pediatric IBD patients.
Dr. Singh has been a consultant to Janssen and Prometheus and received grant support from Janssen.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
AT WCPGHAN 2016
Key clinical point:
Major finding: Fourteen weeks of treatment with vedolizumab produced remission in 76% of ulcerative colitis patients and 42% of Crohn’s disease patients.
Data source: Retrospective review of 52 pediatric patients with inflammatory bowel disease at seen at any of three U.S. centers.
Disclosures: Dr. Singh has been a consultant to Janssen and Prometheus and received grant support from Janssen.
CD64 validated as biomarker for pediatric Crohn’s disease
MONTREAL – Blood levels of a neutrophil receptor protein, CD64, proved to be a reliable, noninvasive marker of both Crohn’s disease activity and the risk for relapse from remission in children and adolescents in a pair of single-center studies with a total of 140 patients.
An elevation in blood levels of CD64, a marker for inflammation, in asymptomatic patients with Crohn’s disease “is a significant risk factor for treatment failure or complications during infliximab maintenance,” Phillip Minar, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology and Nutrition. Although Dr. Minar acknowledged that larger validation studies are still needed, neutrophil CD64 levels can potentially serve as a “treat-to-target” biomarker of disease status in selected pediatric Crohn’s disease patients.
Dr. Minar cautioned that in some pediatric patients with Crohn’s disease CD64 is not an effective marker for inflammation and a change in their Crohn’s disease status. In his study, the sensitivity of an elevated CD64 level was 64% as a surrogate marker for mucosal damage seen with endoscopy.
“I get a CD64 level at the time we diagnose Crohn’s disease. If it is elevated, then I will follow it; if it is not elevated, then I won’t use it for that patient. It’s patient specific,” he explained in an interview.
Dr. Minar and his associates first established the prognostic value of elevated CD64 levels in patients with Crohn’s disease in a study with 208 pediatric patients with inflammatory bowel disease and 43 controls (Inflam Bowel Dis. 2014, Jun;20[6]:1037-48). His new validation study included 105 pediatric patients with Crohn’s disease, of whom 54 were newly diagnosed. Among the 51 previously diagnosed patients, 18 had inactive disease. The patients averaged 14 years old, and all 105 underwent endoscopy to directly examine their Crohn’s disease activity.
The results showed clear and statistically significant correlations among the average CD64 levels in the patients and the blinded endoscopic evaluations that categorized the patients as having inactive Crohn’s disease, mild disease, or moderate to severe disease. The results also suggested that a useful dichotomous cut point for CD64 was an index of 1. Among patients with a level above 1, diagnostic sensitivity for mucosal damage was 64% and specificity was 100%, he reported. In these studies as well as their routine practice, Dr. Minar and his associates use a commercially available immunoassay for quantifying blood levels of CD64.
The second study he reported on assessed the ability of CD64 levels to predict a patient’s status on infliximab (Remicade) maintenance treatment. This study enrolled 35 pediatric patients, who averaged about 15 years old, had been diagnosed with Crohn’s disease for an average of about 2 years and were in remission after having received at least four serial infliximab doses. During 1 year of follow-up, 15 patients relapsed and 21 remained in remission.
The researchers measured CD64 levels at baseline and found that, during the next year, those who had a CD64 index of less than 1 at baseline had a relapse rate of less than 40% during follow-up, while those with a CD64 index of 1 or greater at baseline had a relapse rate of more than 70% during follow-up, a statistically significant difference between the two subgroups. The analysis also showed that lower CD64 levels linked with higher trough levels of infliximab.
A multivariate analysis showed that a CD64 index level of 1 or greater at baseline linked with a statistically significant, 4.5-fold increased risk for relapse, compared with patients with a baseline CD64 level below 1. This analysis identified three additional significant correlates of an elevated risk for relapse: nonwhite race, a baseline serum albumin level of less than 3.9 g/dL, and a baseline infliximab serum level of less than 5 mcg/mL.
The CD64 test that his group has been using typically has a work week turnaround time of about an hour, and costs less than $100 per test per patient. Blood levels of CD64 are stable for 48 hours in the refrigerator, so specimens can sit over a weekend without compromising results. The Cincinnati group is planning to soon change to an in-house test that will cost about $10-$20 per test per patient, Dr. Minar said.
Dr. Minar had no relevant financial disclosures.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
We are in desperate need of more reliable biomarkers of disease activity in patients with Crohn’s disease. Identifying effective noninvasive biomarkers has been a holy grail that we have pursued for many years because what we currently have is imperfect. CD64 appears to be a very reliable and specific biomarker of disease activity.
I think pediatric gastroenterologists will pay attention to Dr. Minar’s report. The entire community is very interested in this and will be watching the evolution of the science behind CD64 assessment.
John A. Barnard, MD , is chief of pediatrics at Nationwide Children’s Hospital and professor and chairman of pediatrics at Ohio State University, both in Columbus. He had no relevant disclosures. He made these comments in an interview.
We are in desperate need of more reliable biomarkers of disease activity in patients with Crohn’s disease. Identifying effective noninvasive biomarkers has been a holy grail that we have pursued for many years because what we currently have is imperfect. CD64 appears to be a very reliable and specific biomarker of disease activity.
I think pediatric gastroenterologists will pay attention to Dr. Minar’s report. The entire community is very interested in this and will be watching the evolution of the science behind CD64 assessment.
John A. Barnard, MD , is chief of pediatrics at Nationwide Children’s Hospital and professor and chairman of pediatrics at Ohio State University, both in Columbus. He had no relevant disclosures. He made these comments in an interview.
We are in desperate need of more reliable biomarkers of disease activity in patients with Crohn’s disease. Identifying effective noninvasive biomarkers has been a holy grail that we have pursued for many years because what we currently have is imperfect. CD64 appears to be a very reliable and specific biomarker of disease activity.
I think pediatric gastroenterologists will pay attention to Dr. Minar’s report. The entire community is very interested in this and will be watching the evolution of the science behind CD64 assessment.
John A. Barnard, MD , is chief of pediatrics at Nationwide Children’s Hospital and professor and chairman of pediatrics at Ohio State University, both in Columbus. He had no relevant disclosures. He made these comments in an interview.
MONTREAL – Blood levels of a neutrophil receptor protein, CD64, proved to be a reliable, noninvasive marker of both Crohn’s disease activity and the risk for relapse from remission in children and adolescents in a pair of single-center studies with a total of 140 patients.
An elevation in blood levels of CD64, a marker for inflammation, in asymptomatic patients with Crohn’s disease “is a significant risk factor for treatment failure or complications during infliximab maintenance,” Phillip Minar, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology and Nutrition. Although Dr. Minar acknowledged that larger validation studies are still needed, neutrophil CD64 levels can potentially serve as a “treat-to-target” biomarker of disease status in selected pediatric Crohn’s disease patients.
Dr. Minar cautioned that in some pediatric patients with Crohn’s disease CD64 is not an effective marker for inflammation and a change in their Crohn’s disease status. In his study, the sensitivity of an elevated CD64 level was 64% as a surrogate marker for mucosal damage seen with endoscopy.
“I get a CD64 level at the time we diagnose Crohn’s disease. If it is elevated, then I will follow it; if it is not elevated, then I won’t use it for that patient. It’s patient specific,” he explained in an interview.
Dr. Minar and his associates first established the prognostic value of elevated CD64 levels in patients with Crohn’s disease in a study with 208 pediatric patients with inflammatory bowel disease and 43 controls (Inflam Bowel Dis. 2014, Jun;20[6]:1037-48). His new validation study included 105 pediatric patients with Crohn’s disease, of whom 54 were newly diagnosed. Among the 51 previously diagnosed patients, 18 had inactive disease. The patients averaged 14 years old, and all 105 underwent endoscopy to directly examine their Crohn’s disease activity.
The results showed clear and statistically significant correlations among the average CD64 levels in the patients and the blinded endoscopic evaluations that categorized the patients as having inactive Crohn’s disease, mild disease, or moderate to severe disease. The results also suggested that a useful dichotomous cut point for CD64 was an index of 1. Among patients with a level above 1, diagnostic sensitivity for mucosal damage was 64% and specificity was 100%, he reported. In these studies as well as their routine practice, Dr. Minar and his associates use a commercially available immunoassay for quantifying blood levels of CD64.
The second study he reported on assessed the ability of CD64 levels to predict a patient’s status on infliximab (Remicade) maintenance treatment. This study enrolled 35 pediatric patients, who averaged about 15 years old, had been diagnosed with Crohn’s disease for an average of about 2 years and were in remission after having received at least four serial infliximab doses. During 1 year of follow-up, 15 patients relapsed and 21 remained in remission.
The researchers measured CD64 levels at baseline and found that, during the next year, those who had a CD64 index of less than 1 at baseline had a relapse rate of less than 40% during follow-up, while those with a CD64 index of 1 or greater at baseline had a relapse rate of more than 70% during follow-up, a statistically significant difference between the two subgroups. The analysis also showed that lower CD64 levels linked with higher trough levels of infliximab.
A multivariate analysis showed that a CD64 index level of 1 or greater at baseline linked with a statistically significant, 4.5-fold increased risk for relapse, compared with patients with a baseline CD64 level below 1. This analysis identified three additional significant correlates of an elevated risk for relapse: nonwhite race, a baseline serum albumin level of less than 3.9 g/dL, and a baseline infliximab serum level of less than 5 mcg/mL.
The CD64 test that his group has been using typically has a work week turnaround time of about an hour, and costs less than $100 per test per patient. Blood levels of CD64 are stable for 48 hours in the refrigerator, so specimens can sit over a weekend without compromising results. The Cincinnati group is planning to soon change to an in-house test that will cost about $10-$20 per test per patient, Dr. Minar said.
Dr. Minar had no relevant financial disclosures.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
MONTREAL – Blood levels of a neutrophil receptor protein, CD64, proved to be a reliable, noninvasive marker of both Crohn’s disease activity and the risk for relapse from remission in children and adolescents in a pair of single-center studies with a total of 140 patients.
An elevation in blood levels of CD64, a marker for inflammation, in asymptomatic patients with Crohn’s disease “is a significant risk factor for treatment failure or complications during infliximab maintenance,” Phillip Minar, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology and Nutrition. Although Dr. Minar acknowledged that larger validation studies are still needed, neutrophil CD64 levels can potentially serve as a “treat-to-target” biomarker of disease status in selected pediatric Crohn’s disease patients.
Dr. Minar cautioned that in some pediatric patients with Crohn’s disease CD64 is not an effective marker for inflammation and a change in their Crohn’s disease status. In his study, the sensitivity of an elevated CD64 level was 64% as a surrogate marker for mucosal damage seen with endoscopy.
“I get a CD64 level at the time we diagnose Crohn’s disease. If it is elevated, then I will follow it; if it is not elevated, then I won’t use it for that patient. It’s patient specific,” he explained in an interview.
Dr. Minar and his associates first established the prognostic value of elevated CD64 levels in patients with Crohn’s disease in a study with 208 pediatric patients with inflammatory bowel disease and 43 controls (Inflam Bowel Dis. 2014, Jun;20[6]:1037-48). His new validation study included 105 pediatric patients with Crohn’s disease, of whom 54 were newly diagnosed. Among the 51 previously diagnosed patients, 18 had inactive disease. The patients averaged 14 years old, and all 105 underwent endoscopy to directly examine their Crohn’s disease activity.
The results showed clear and statistically significant correlations among the average CD64 levels in the patients and the blinded endoscopic evaluations that categorized the patients as having inactive Crohn’s disease, mild disease, or moderate to severe disease. The results also suggested that a useful dichotomous cut point for CD64 was an index of 1. Among patients with a level above 1, diagnostic sensitivity for mucosal damage was 64% and specificity was 100%, he reported. In these studies as well as their routine practice, Dr. Minar and his associates use a commercially available immunoassay for quantifying blood levels of CD64.
The second study he reported on assessed the ability of CD64 levels to predict a patient’s status on infliximab (Remicade) maintenance treatment. This study enrolled 35 pediatric patients, who averaged about 15 years old, had been diagnosed with Crohn’s disease for an average of about 2 years and were in remission after having received at least four serial infliximab doses. During 1 year of follow-up, 15 patients relapsed and 21 remained in remission.
The researchers measured CD64 levels at baseline and found that, during the next year, those who had a CD64 index of less than 1 at baseline had a relapse rate of less than 40% during follow-up, while those with a CD64 index of 1 or greater at baseline had a relapse rate of more than 70% during follow-up, a statistically significant difference between the two subgroups. The analysis also showed that lower CD64 levels linked with higher trough levels of infliximab.
A multivariate analysis showed that a CD64 index level of 1 or greater at baseline linked with a statistically significant, 4.5-fold increased risk for relapse, compared with patients with a baseline CD64 level below 1. This analysis identified three additional significant correlates of an elevated risk for relapse: nonwhite race, a baseline serum albumin level of less than 3.9 g/dL, and a baseline infliximab serum level of less than 5 mcg/mL.
The CD64 test that his group has been using typically has a work week turnaround time of about an hour, and costs less than $100 per test per patient. Blood levels of CD64 are stable for 48 hours in the refrigerator, so specimens can sit over a weekend without compromising results. The Cincinnati group is planning to soon change to an in-house test that will cost about $10-$20 per test per patient, Dr. Minar said.
Dr. Minar had no relevant financial disclosures.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
AT WCPGHAN 2016
Key clinical point: Results from two studies further validated neutrophil CD64 as a highly specific biomarker for Crohn’s disease severity in children and adolescents and suggested that CD64 could serve as a treat-to-target guide for infliximab treatment.
Major finding: During infliximab maintenance, relapses occurred in fewer than 40% of pediatric Crohn’s disease patients with low CD64 and in more than 70% with high CD64.
Data source: A single-center study of 105 pediatric patients with Crohn’s disease to assess disease severity correlates, and 35 patients in remission on infliximab to assess predicted efficacy.
Disclosures: Dr. Minar had no relevant financial disclosures.
Palliative care boosts heart failure patient outcomes
ORLANDO – Systematic introduction of palliative care interventions for patients with advanced heart failure improved patients’ quality of life and spurred their development of advanced-care preferences in a pair of independently performed, controlled, pilot studies.
But, despite demonstrating the ability of palliative-care interventions to help heart failure patients during their final months of life, the findings raised questions about the generalizability and reproducibility of palliative-care interventions that may depend on the skills and experience of the individual specialists who deliver the palliative care.
“Palliative care for patients with cardiovascular disease is in desperate need of good-quality evidence,” commented Larry A, Allen, MD, a heart failure cardiologist at the University of Colorado in Aurora and designated discussant for one of the two studies presented at the meeting. “We need large, randomized trials with clinical outcomes to look at patient outcomes from palliative-care interventions.”
The patients average 71 years old, about half were women, and about 40% were African Americans. They had been diagnosed with heart failure for an average of more than 5 years, all had advanced heart failure, about 60% spent at least half of their time awake immobilized in a bed or chair, and they had average NT-proBNP blood levels of greater than 10,000 pg/mL.
After 24 weeks of intervention, the palliative-care program produced both statistically significant and clinically meaningful improvements in two different measures of health-related quality of life, the Kansas City Cardiomyopathy Questionnaire (KCCQ) and the Functional Assessment of Chronic Illness Therapy – Palliative Care (FACIT-PAL). The KCCQ showed the palliative care intervention linked with an average rise of more than 9 points compared with patients in the control arm after adjustment for age and sex, a statistically significant increase on a scale where a 5-point rise is considered clinically meaningful. The FACIT-PAL showed an average, adjusted 11-point rise linked with the intervention, a statistically significant increase on a scale where an increase of at least 10 is judged clinically meaningful, reported Dr. Rogers, a heart failure cardiologist and professor of medicine at Duke University.
The palliative-care intervention also led to significant improvements in measures of spirituality, depression, and anxiety, but intervention had no impact on mortality.
“I like these endpoints and the idea that we can make quality-of-life better. These are very sick patients, with a predicted 6-month mortality of 50%. Patients reach a time when they don’t want to live longer but want better life quality for the days they still have,” he said in an interview.
The second report came from a single-center pilot study of 50 patients enrolled when they were hospitalized for acute decompensated heart failure and had at least one addition risk factor for poor prognosis such as age of at least 81 years, renal dysfunction, or a prior heart failure hospitalization within the past year. Patients randomized to the intervention arm underwent a structured evaluation based on the Serious Illness Conversation Guide and performed by a social worker experienced in palliative care and embedded in the heart failure clinical team. The primary endpoint of the SWAP-HF (Social Worker–Aided Palliative Care Intervention in High Risk Patients with Heart Failure) study was clinical-level documentation of advanced-care preferences by 6 months after the program began.
“Although more comprehensive, multidisciplinary palliative care interventions may also be effective, the focused approach [used in this study] may represent a cost-effective and scalable method for shepherding limited specialty resources to enhance the delivery of patient-centered care,” Dr. Desai said. In other words, a program with a social worker costs less than a two-person staff with a palliative-care physician and nurse practitioner.
Despite its relative simplicity, the SWAP-HF intervention had some unique aspects that make it generalizability uncertain, commented Dr. Allen. The embedding of a social worker on the heart failure team placed a professional with a “good understanding of social context” right on the scene with everyone else delivering care to the heart failure patient, a good strategy for minimizing fragmentation, he said. In addition, the place where the study was done, Brigham and Women’s Hospital, “is not your average hospital,” he noted,
In addition, the timing of the intervention studied during hospitalization may be problematic. Clinicians need to “be careful about patients making long-term decisions” about their care while they are hospitalized, a time when patients can be “ill, confused, and scared.” He cited recent findings from a study of hospital-based palliative-care interventions for family members of patients with chronic critical illness that did not reduce anxiety or depression symptoms among the treated family members and may have increased symptoms of posttraumatic stress disorder (JAMA. 2016 July 5;374[1]:51-62).
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
It’s very exciting to have these two studies presented at the Heart Failure Society of America’s annual meeting. Palliative-care research now receives funding from the National Institutes of Health, but consistently and successfully integrating palliative care into heart failure management still has a long way to go. In 2004, my colleagues and I published a set of consensus recommendations on how to apply palliative care methods to patients with advanced heart failure and what research needs existed for the field (J Card Fail. 2004 June;10[3]:200-9). Today, 12 years later, many of those research needs remain inadequately addressed.
Sarah J. Goodlin, MD , is chief of geriatrics at the Portland (Ore.) VA Medical Center. She had no disclosures. She made these comments as the designated discussant for Dr. Rogers’ report.
It’s very exciting to have these two studies presented at the Heart Failure Society of America’s annual meeting. Palliative-care research now receives funding from the National Institutes of Health, but consistently and successfully integrating palliative care into heart failure management still has a long way to go. In 2004, my colleagues and I published a set of consensus recommendations on how to apply palliative care methods to patients with advanced heart failure and what research needs existed for the field (J Card Fail. 2004 June;10[3]:200-9). Today, 12 years later, many of those research needs remain inadequately addressed.
Sarah J. Goodlin, MD , is chief of geriatrics at the Portland (Ore.) VA Medical Center. She had no disclosures. She made these comments as the designated discussant for Dr. Rogers’ report.
It’s very exciting to have these two studies presented at the Heart Failure Society of America’s annual meeting. Palliative-care research now receives funding from the National Institutes of Health, but consistently and successfully integrating palliative care into heart failure management still has a long way to go. In 2004, my colleagues and I published a set of consensus recommendations on how to apply palliative care methods to patients with advanced heart failure and what research needs existed for the field (J Card Fail. 2004 June;10[3]:200-9). Today, 12 years later, many of those research needs remain inadequately addressed.
Sarah J. Goodlin, MD , is chief of geriatrics at the Portland (Ore.) VA Medical Center. She had no disclosures. She made these comments as the designated discussant for Dr. Rogers’ report.
ORLANDO – Systematic introduction of palliative care interventions for patients with advanced heart failure improved patients’ quality of life and spurred their development of advanced-care preferences in a pair of independently performed, controlled, pilot studies.
But, despite demonstrating the ability of palliative-care interventions to help heart failure patients during their final months of life, the findings raised questions about the generalizability and reproducibility of palliative-care interventions that may depend on the skills and experience of the individual specialists who deliver the palliative care.
“Palliative care for patients with cardiovascular disease is in desperate need of good-quality evidence,” commented Larry A, Allen, MD, a heart failure cardiologist at the University of Colorado in Aurora and designated discussant for one of the two studies presented at the meeting. “We need large, randomized trials with clinical outcomes to look at patient outcomes from palliative-care interventions.”
The patients average 71 years old, about half were women, and about 40% were African Americans. They had been diagnosed with heart failure for an average of more than 5 years, all had advanced heart failure, about 60% spent at least half of their time awake immobilized in a bed or chair, and they had average NT-proBNP blood levels of greater than 10,000 pg/mL.
After 24 weeks of intervention, the palliative-care program produced both statistically significant and clinically meaningful improvements in two different measures of health-related quality of life, the Kansas City Cardiomyopathy Questionnaire (KCCQ) and the Functional Assessment of Chronic Illness Therapy – Palliative Care (FACIT-PAL). The KCCQ showed the palliative care intervention linked with an average rise of more than 9 points compared with patients in the control arm after adjustment for age and sex, a statistically significant increase on a scale where a 5-point rise is considered clinically meaningful. The FACIT-PAL showed an average, adjusted 11-point rise linked with the intervention, a statistically significant increase on a scale where an increase of at least 10 is judged clinically meaningful, reported Dr. Rogers, a heart failure cardiologist and professor of medicine at Duke University.
The palliative-care intervention also led to significant improvements in measures of spirituality, depression, and anxiety, but intervention had no impact on mortality.
“I like these endpoints and the idea that we can make quality-of-life better. These are very sick patients, with a predicted 6-month mortality of 50%. Patients reach a time when they don’t want to live longer but want better life quality for the days they still have,” he said in an interview.
The second report came from a single-center pilot study of 50 patients enrolled when they were hospitalized for acute decompensated heart failure and had at least one addition risk factor for poor prognosis such as age of at least 81 years, renal dysfunction, or a prior heart failure hospitalization within the past year. Patients randomized to the intervention arm underwent a structured evaluation based on the Serious Illness Conversation Guide and performed by a social worker experienced in palliative care and embedded in the heart failure clinical team. The primary endpoint of the SWAP-HF (Social Worker–Aided Palliative Care Intervention in High Risk Patients with Heart Failure) study was clinical-level documentation of advanced-care preferences by 6 months after the program began.
“Although more comprehensive, multidisciplinary palliative care interventions may also be effective, the focused approach [used in this study] may represent a cost-effective and scalable method for shepherding limited specialty resources to enhance the delivery of patient-centered care,” Dr. Desai said. In other words, a program with a social worker costs less than a two-person staff with a palliative-care physician and nurse practitioner.
Despite its relative simplicity, the SWAP-HF intervention had some unique aspects that make it generalizability uncertain, commented Dr. Allen. The embedding of a social worker on the heart failure team placed a professional with a “good understanding of social context” right on the scene with everyone else delivering care to the heart failure patient, a good strategy for minimizing fragmentation, he said. In addition, the place where the study was done, Brigham and Women’s Hospital, “is not your average hospital,” he noted,
In addition, the timing of the intervention studied during hospitalization may be problematic. Clinicians need to “be careful about patients making long-term decisions” about their care while they are hospitalized, a time when patients can be “ill, confused, and scared.” He cited recent findings from a study of hospital-based palliative-care interventions for family members of patients with chronic critical illness that did not reduce anxiety or depression symptoms among the treated family members and may have increased symptoms of posttraumatic stress disorder (JAMA. 2016 July 5;374[1]:51-62).
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
ORLANDO – Systematic introduction of palliative care interventions for patients with advanced heart failure improved patients’ quality of life and spurred their development of advanced-care preferences in a pair of independently performed, controlled, pilot studies.
But, despite demonstrating the ability of palliative-care interventions to help heart failure patients during their final months of life, the findings raised questions about the generalizability and reproducibility of palliative-care interventions that may depend on the skills and experience of the individual specialists who deliver the palliative care.
“Palliative care for patients with cardiovascular disease is in desperate need of good-quality evidence,” commented Larry A, Allen, MD, a heart failure cardiologist at the University of Colorado in Aurora and designated discussant for one of the two studies presented at the meeting. “We need large, randomized trials with clinical outcomes to look at patient outcomes from palliative-care interventions.”
The patients average 71 years old, about half were women, and about 40% were African Americans. They had been diagnosed with heart failure for an average of more than 5 years, all had advanced heart failure, about 60% spent at least half of their time awake immobilized in a bed or chair, and they had average NT-proBNP blood levels of greater than 10,000 pg/mL.
After 24 weeks of intervention, the palliative-care program produced both statistically significant and clinically meaningful improvements in two different measures of health-related quality of life, the Kansas City Cardiomyopathy Questionnaire (KCCQ) and the Functional Assessment of Chronic Illness Therapy – Palliative Care (FACIT-PAL). The KCCQ showed the palliative care intervention linked with an average rise of more than 9 points compared with patients in the control arm after adjustment for age and sex, a statistically significant increase on a scale where a 5-point rise is considered clinically meaningful. The FACIT-PAL showed an average, adjusted 11-point rise linked with the intervention, a statistically significant increase on a scale where an increase of at least 10 is judged clinically meaningful, reported Dr. Rogers, a heart failure cardiologist and professor of medicine at Duke University.
The palliative-care intervention also led to significant improvements in measures of spirituality, depression, and anxiety, but intervention had no impact on mortality.
“I like these endpoints and the idea that we can make quality-of-life better. These are very sick patients, with a predicted 6-month mortality of 50%. Patients reach a time when they don’t want to live longer but want better life quality for the days they still have,” he said in an interview.
The second report came from a single-center pilot study of 50 patients enrolled when they were hospitalized for acute decompensated heart failure and had at least one addition risk factor for poor prognosis such as age of at least 81 years, renal dysfunction, or a prior heart failure hospitalization within the past year. Patients randomized to the intervention arm underwent a structured evaluation based on the Serious Illness Conversation Guide and performed by a social worker experienced in palliative care and embedded in the heart failure clinical team. The primary endpoint of the SWAP-HF (Social Worker–Aided Palliative Care Intervention in High Risk Patients with Heart Failure) study was clinical-level documentation of advanced-care preferences by 6 months after the program began.
“Although more comprehensive, multidisciplinary palliative care interventions may also be effective, the focused approach [used in this study] may represent a cost-effective and scalable method for shepherding limited specialty resources to enhance the delivery of patient-centered care,” Dr. Desai said. In other words, a program with a social worker costs less than a two-person staff with a palliative-care physician and nurse practitioner.
Despite its relative simplicity, the SWAP-HF intervention had some unique aspects that make it generalizability uncertain, commented Dr. Allen. The embedding of a social worker on the heart failure team placed a professional with a “good understanding of social context” right on the scene with everyone else delivering care to the heart failure patient, a good strategy for minimizing fragmentation, he said. In addition, the place where the study was done, Brigham and Women’s Hospital, “is not your average hospital,” he noted,
In addition, the timing of the intervention studied during hospitalization may be problematic. Clinicians need to “be careful about patients making long-term decisions” about their care while they are hospitalized, a time when patients can be “ill, confused, and scared.” He cited recent findings from a study of hospital-based palliative-care interventions for family members of patients with chronic critical illness that did not reduce anxiety or depression symptoms among the treated family members and may have increased symptoms of posttraumatic stress disorder (JAMA. 2016 July 5;374[1]:51-62).
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
AT THE HFSA ANNUAL SCIENTIFIC MEETING
Key clinical point:
Major finding: Palliative care measures boosted patients’ Kansas City Cardiomyopathy Questionnaire score by an average of 9 points over that of controls.
Data source: PAL-HF, a single-center study with 150 randomized patients with heart failure and SWAP-HF, a single-center study with 50 randomized patients.
Disclosures: Dr. Rogers, Dr. Allen, and Dr. Desai had no relevant disclosures.
Cow’s milk allergy appears to affect more U.S. infants than thought
MONTREAL – The incidence of cow’s milk protein allergy during the first few months of life may be much more common than suggested by published studies, based on what was found is a prospective study with 700 infants seen regularly at a single, general pediatrics practice in suburban Massachusetts.
Among the 700 infants enrolled in this series, 105 (15%) were diagnosed with cow’s milk protein allergy (CMPA) when they were 5-163 days old, with a median age at diagnosis of 33 days, Victoria J. Martin, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition. She and her associates confirmed that all these infants had true CMPA episodes of proctocolitis by requiring detection of blood in the stool of affected children.
The study results also suggested a protective effect against CMPA when infants received some amount of early breastfeeding, and a pilot substudy run in 47 of the enrolled infants also suggested a link between development of CMPA and abnormalities in the microbiome composition of affected infants, she reported.
While the 15% incidence rate was unexpectedly high, it “absolutely feels like what we see in routine clinical practice,” Dr. Martin said in an interview. She chalked up the much-lower figure cited in the pediatric literature as relying on strict follow-up confirmation by rechallenge of the child with cow’s milk, a step often not taken by busy clinicians. Deferring formal confirmation also often means delayed reintroduction of cow’s milk into the infant’s diet, with restriction often continuing for perhaps a year following the index episode of CMPA. Although such unnecessarily long delays in milk reintroduction have largely been considered benign, recent findings from the Learning Early About Peanut Allergy (LEAP) trial that withholding peanut exposure can increase development of peanut allergies suggests that children also might receive long-term benefit from quicker reintroduction of milk in terms of better development of the immune system and microbiome, she said.
“If we rechallenged all these infants after 1 month, I think we’d find a CMPA rate closer to 3%. Leaving infants on a mild restricted diet for 12 months is a mistake,” she added.
The Gastrointestinal Microbiome & Proctocolitis (GMAP) study enrolled 700 infants seen at a single general practice pediatric practice in suburban Massachusetts at the time of their first well-baby visit, at a median age of 8 days. During 2 years of follow-up, the researchers collected stool specimens from the enrolled children at each of up to five scheduled visits during the first 4 months. They also kept track of when children received a CMPA diagnosis confirmed by at least one bloody stool.
Analysis of CMPA correlates showed that, among infants who developed it, 17% had not received any breastfeeding soon after birth, while among infants who did not develop CMPA, 8% did not undergo early breastfeeding. The incidence of CMPA was roughly similar among infants who received an early combination of breast milk and formula and in those who received exclusively breast milk during the first days of life, showing that even partial breastfeeding is better than no breastfeeding, Dr. Martin noted.
Her analysis also includes initial results from microbial assessment of the collected serial stool specimens from a subgroup of 24 infants who developed CMPA and 23 who did not, with a total of 223 total specimens evaluated. These studies showed that the infants who developed CMPA significantly lagged in their colonization with Bifidobacteria, had significantly higher colonization levels with Enterobacteriaceae, and that in infants who did develop CMPA, their gut level of Clostridia significantly increased as their proctocolitis resolved.
Dr. Martin had no relevant financial disclosures.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
MONTREAL – The incidence of cow’s milk protein allergy during the first few months of life may be much more common than suggested by published studies, based on what was found is a prospective study with 700 infants seen regularly at a single, general pediatrics practice in suburban Massachusetts.
Among the 700 infants enrolled in this series, 105 (15%) were diagnosed with cow’s milk protein allergy (CMPA) when they were 5-163 days old, with a median age at diagnosis of 33 days, Victoria J. Martin, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition. She and her associates confirmed that all these infants had true CMPA episodes of proctocolitis by requiring detection of blood in the stool of affected children.
The study results also suggested a protective effect against CMPA when infants received some amount of early breastfeeding, and a pilot substudy run in 47 of the enrolled infants also suggested a link between development of CMPA and abnormalities in the microbiome composition of affected infants, she reported.
While the 15% incidence rate was unexpectedly high, it “absolutely feels like what we see in routine clinical practice,” Dr. Martin said in an interview. She chalked up the much-lower figure cited in the pediatric literature as relying on strict follow-up confirmation by rechallenge of the child with cow’s milk, a step often not taken by busy clinicians. Deferring formal confirmation also often means delayed reintroduction of cow’s milk into the infant’s diet, with restriction often continuing for perhaps a year following the index episode of CMPA. Although such unnecessarily long delays in milk reintroduction have largely been considered benign, recent findings from the Learning Early About Peanut Allergy (LEAP) trial that withholding peanut exposure can increase development of peanut allergies suggests that children also might receive long-term benefit from quicker reintroduction of milk in terms of better development of the immune system and microbiome, she said.
“If we rechallenged all these infants after 1 month, I think we’d find a CMPA rate closer to 3%. Leaving infants on a mild restricted diet for 12 months is a mistake,” she added.
The Gastrointestinal Microbiome & Proctocolitis (GMAP) study enrolled 700 infants seen at a single general practice pediatric practice in suburban Massachusetts at the time of their first well-baby visit, at a median age of 8 days. During 2 years of follow-up, the researchers collected stool specimens from the enrolled children at each of up to five scheduled visits during the first 4 months. They also kept track of when children received a CMPA diagnosis confirmed by at least one bloody stool.
Analysis of CMPA correlates showed that, among infants who developed it, 17% had not received any breastfeeding soon after birth, while among infants who did not develop CMPA, 8% did not undergo early breastfeeding. The incidence of CMPA was roughly similar among infants who received an early combination of breast milk and formula and in those who received exclusively breast milk during the first days of life, showing that even partial breastfeeding is better than no breastfeeding, Dr. Martin noted.
Her analysis also includes initial results from microbial assessment of the collected serial stool specimens from a subgroup of 24 infants who developed CMPA and 23 who did not, with a total of 223 total specimens evaluated. These studies showed that the infants who developed CMPA significantly lagged in their colonization with Bifidobacteria, had significantly higher colonization levels with Enterobacteriaceae, and that in infants who did develop CMPA, their gut level of Clostridia significantly increased as their proctocolitis resolved.
Dr. Martin had no relevant financial disclosures.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
MONTREAL – The incidence of cow’s milk protein allergy during the first few months of life may be much more common than suggested by published studies, based on what was found is a prospective study with 700 infants seen regularly at a single, general pediatrics practice in suburban Massachusetts.
Among the 700 infants enrolled in this series, 105 (15%) were diagnosed with cow’s milk protein allergy (CMPA) when they were 5-163 days old, with a median age at diagnosis of 33 days, Victoria J. Martin, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition. She and her associates confirmed that all these infants had true CMPA episodes of proctocolitis by requiring detection of blood in the stool of affected children.
The study results also suggested a protective effect against CMPA when infants received some amount of early breastfeeding, and a pilot substudy run in 47 of the enrolled infants also suggested a link between development of CMPA and abnormalities in the microbiome composition of affected infants, she reported.
While the 15% incidence rate was unexpectedly high, it “absolutely feels like what we see in routine clinical practice,” Dr. Martin said in an interview. She chalked up the much-lower figure cited in the pediatric literature as relying on strict follow-up confirmation by rechallenge of the child with cow’s milk, a step often not taken by busy clinicians. Deferring formal confirmation also often means delayed reintroduction of cow’s milk into the infant’s diet, with restriction often continuing for perhaps a year following the index episode of CMPA. Although such unnecessarily long delays in milk reintroduction have largely been considered benign, recent findings from the Learning Early About Peanut Allergy (LEAP) trial that withholding peanut exposure can increase development of peanut allergies suggests that children also might receive long-term benefit from quicker reintroduction of milk in terms of better development of the immune system and microbiome, she said.
“If we rechallenged all these infants after 1 month, I think we’d find a CMPA rate closer to 3%. Leaving infants on a mild restricted diet for 12 months is a mistake,” she added.
The Gastrointestinal Microbiome & Proctocolitis (GMAP) study enrolled 700 infants seen at a single general practice pediatric practice in suburban Massachusetts at the time of their first well-baby visit, at a median age of 8 days. During 2 years of follow-up, the researchers collected stool specimens from the enrolled children at each of up to five scheduled visits during the first 4 months. They also kept track of when children received a CMPA diagnosis confirmed by at least one bloody stool.
Analysis of CMPA correlates showed that, among infants who developed it, 17% had not received any breastfeeding soon after birth, while among infants who did not develop CMPA, 8% did not undergo early breastfeeding. The incidence of CMPA was roughly similar among infants who received an early combination of breast milk and formula and in those who received exclusively breast milk during the first days of life, showing that even partial breastfeeding is better than no breastfeeding, Dr. Martin noted.
Her analysis also includes initial results from microbial assessment of the collected serial stool specimens from a subgroup of 24 infants who developed CMPA and 23 who did not, with a total of 223 total specimens evaluated. These studies showed that the infants who developed CMPA significantly lagged in their colonization with Bifidobacteria, had significantly higher colonization levels with Enterobacteriaceae, and that in infants who did develop CMPA, their gut level of Clostridia significantly increased as their proctocolitis resolved.
Dr. Martin had no relevant financial disclosures.
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
AT WCPGHAN 2016
Key clinical point:
Major finding: Among 700 enrolled well infants, aged 5-163 days, 105 (15%) developed proctocolitis linked with cow’s milk.
Data source: Prospective observational study of 700 healthy neonates seen at a single U.S. pediatric practice.
Disclosures: Dr. Martin had no relevant financial disclosures.
VIDEO: Harvoni shows safety, efficacy in adolescents for hepatitis C
MONTREAL – One of the antiviral drug combinations that has revolutionized treatment of hepatitis C virus in adults has for the first time been shown safe and effective against genotype 1 infections in adolescents aged 12-17 years old, paving the way to new regulatory labeling followed by easier and more reliable payer coverage for definitive hepatitis C treatment in this age group.
“I think having clear data on safety and efficacy and FDA [Food and Drug Administration] approval will greatly help getting insurance coverage,” for the tested combination of ledipasvir/sofosbuvir (Harvoni) Karen F. Murray, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition.
The results showed that in adolescents the ledipasvir/sofosbuvir formulation tested, at the same dosage approved for adults, was “very potent for genotype 1 hepatitis C, and was not only well tolerated but very easy” when given for 12 weeks, Dr. Murray said in an interview. The nearly perfect score for sustained virologic responses was “spectacular,” she added.
Ongoing studies that should finish soon are also looking at the safety and efficacy of ledipasvir/sofosbuvir in children aged 3-11 years old and in children and adolescents infected by other hepatitis C genotypes, specifically 4, 5, and 6. Taking on genotypes 2 and 3 will require additional treatment with ribavirin, she noted. Subsequent reports will also document patient outcomes 24 weeks from the start of treatment, after they’ve been off their completed regimen for 12 weeks. Gilead staffers have told Dr. Murray that they anticipate asking the FDA before the end of 2016 for approval to relabel the ledipasvir/sofosbuvir formulation they market to include adolescents, and possibly children too, depending on the outcome of studies still underway.
The result she reported came from 100 patients enrolled at 24 centers in the United States, Canada, and Europe. They averaged 15 years old, nearly two-thirds were girls, and 90% were white. One-fifth of the patients had been previously treated, and 81% were infected by genotype 1a hepatitis C with the remaining 19% infected with genotype 1b. Patients received the conventional, marketed formulation of ledipasvir/sofosbuvir, 90/400 mg, orally once daily.
Ninety-eight of the patients had no detectable hepatitis C virus in their blood at the end of 12 weeks of treatment. The other two patients were lost to follow-up and did not undergo virologic testing at the end of treatment and conservatively were tallied as nonresponders, she reported. A pharmacokinetic study done in a subgroup of patients showed plasma drug levels comparable with those seen in adults.
Although 71% of the patients reported having some adverse effect, no patient reported a serious or grade 3 or 4 adverse effect and no patient stopped treatment because of adverse effects. Nine patients had a grade 3 or 4 laboratory abnormality on treatment. The only lab abnormality to occur in more than one patient was a transient rise in amylase levels, which happened in three patients. The regimen’s overall performance in adolescents closely tracked what’s been seen in adults, Dr. Murray said.
“These data will lead to FDA approval” of the regimen for adolescents, she said confidently, and that will ease insurance coverage. “There will still be hoops to jump through, but with approval and once written into guidelines, insurers will be under pressure to reimburse for it,” she noted. “We find that, because of the cost, insurers resist or refuse to pay for these medications. The idea of treating only patients with advanced liver disease is morally inappropriate. These children must be treated before they develop significant or irreversible liver 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
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
MONTREAL – One of the antiviral drug combinations that has revolutionized treatment of hepatitis C virus in adults has for the first time been shown safe and effective against genotype 1 infections in adolescents aged 12-17 years old, paving the way to new regulatory labeling followed by easier and more reliable payer coverage for definitive hepatitis C treatment in this age group.
“I think having clear data on safety and efficacy and FDA [Food and Drug Administration] approval will greatly help getting insurance coverage,” for the tested combination of ledipasvir/sofosbuvir (Harvoni) Karen F. Murray, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition.
The results showed that in adolescents the ledipasvir/sofosbuvir formulation tested, at the same dosage approved for adults, was “very potent for genotype 1 hepatitis C, and was not only well tolerated but very easy” when given for 12 weeks, Dr. Murray said in an interview. The nearly perfect score for sustained virologic responses was “spectacular,” she added.
Ongoing studies that should finish soon are also looking at the safety and efficacy of ledipasvir/sofosbuvir in children aged 3-11 years old and in children and adolescents infected by other hepatitis C genotypes, specifically 4, 5, and 6. Taking on genotypes 2 and 3 will require additional treatment with ribavirin, she noted. Subsequent reports will also document patient outcomes 24 weeks from the start of treatment, after they’ve been off their completed regimen for 12 weeks. Gilead staffers have told Dr. Murray that they anticipate asking the FDA before the end of 2016 for approval to relabel the ledipasvir/sofosbuvir formulation they market to include adolescents, and possibly children too, depending on the outcome of studies still underway.
The result she reported came from 100 patients enrolled at 24 centers in the United States, Canada, and Europe. They averaged 15 years old, nearly two-thirds were girls, and 90% were white. One-fifth of the patients had been previously treated, and 81% were infected by genotype 1a hepatitis C with the remaining 19% infected with genotype 1b. Patients received the conventional, marketed formulation of ledipasvir/sofosbuvir, 90/400 mg, orally once daily.
Ninety-eight of the patients had no detectable hepatitis C virus in their blood at the end of 12 weeks of treatment. The other two patients were lost to follow-up and did not undergo virologic testing at the end of treatment and conservatively were tallied as nonresponders, she reported. A pharmacokinetic study done in a subgroup of patients showed plasma drug levels comparable with those seen in adults.
Although 71% of the patients reported having some adverse effect, no patient reported a serious or grade 3 or 4 adverse effect and no patient stopped treatment because of adverse effects. Nine patients had a grade 3 or 4 laboratory abnormality on treatment. The only lab abnormality to occur in more than one patient was a transient rise in amylase levels, which happened in three patients. The regimen’s overall performance in adolescents closely tracked what’s been seen in adults, Dr. Murray said.
“These data will lead to FDA approval” of the regimen for adolescents, she said confidently, and that will ease insurance coverage. “There will still be hoops to jump through, but with approval and once written into guidelines, insurers will be under pressure to reimburse for it,” she noted. “We find that, because of the cost, insurers resist or refuse to pay for these medications. The idea of treating only patients with advanced liver disease is morally inappropriate. These children must be treated before they develop significant or irreversible liver 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
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
MONTREAL – One of the antiviral drug combinations that has revolutionized treatment of hepatitis C virus in adults has for the first time been shown safe and effective against genotype 1 infections in adolescents aged 12-17 years old, paving the way to new regulatory labeling followed by easier and more reliable payer coverage for definitive hepatitis C treatment in this age group.
“I think having clear data on safety and efficacy and FDA [Food and Drug Administration] approval will greatly help getting insurance coverage,” for the tested combination of ledipasvir/sofosbuvir (Harvoni) Karen F. Murray, MD, said at the World Congress of Pediatric Gastroenterology, Hepatology, and Nutrition.
The results showed that in adolescents the ledipasvir/sofosbuvir formulation tested, at the same dosage approved for adults, was “very potent for genotype 1 hepatitis C, and was not only well tolerated but very easy” when given for 12 weeks, Dr. Murray said in an interview. The nearly perfect score for sustained virologic responses was “spectacular,” she added.
Ongoing studies that should finish soon are also looking at the safety and efficacy of ledipasvir/sofosbuvir in children aged 3-11 years old and in children and adolescents infected by other hepatitis C genotypes, specifically 4, 5, and 6. Taking on genotypes 2 and 3 will require additional treatment with ribavirin, she noted. Subsequent reports will also document patient outcomes 24 weeks from the start of treatment, after they’ve been off their completed regimen for 12 weeks. Gilead staffers have told Dr. Murray that they anticipate asking the FDA before the end of 2016 for approval to relabel the ledipasvir/sofosbuvir formulation they market to include adolescents, and possibly children too, depending on the outcome of studies still underway.
The result she reported came from 100 patients enrolled at 24 centers in the United States, Canada, and Europe. They averaged 15 years old, nearly two-thirds were girls, and 90% were white. One-fifth of the patients had been previously treated, and 81% were infected by genotype 1a hepatitis C with the remaining 19% infected with genotype 1b. Patients received the conventional, marketed formulation of ledipasvir/sofosbuvir, 90/400 mg, orally once daily.
Ninety-eight of the patients had no detectable hepatitis C virus in their blood at the end of 12 weeks of treatment. The other two patients were lost to follow-up and did not undergo virologic testing at the end of treatment and conservatively were tallied as nonresponders, she reported. A pharmacokinetic study done in a subgroup of patients showed plasma drug levels comparable with those seen in adults.
Although 71% of the patients reported having some adverse effect, no patient reported a serious or grade 3 or 4 adverse effect and no patient stopped treatment because of adverse effects. Nine patients had a grade 3 or 4 laboratory abnormality on treatment. The only lab abnormality to occur in more than one patient was a transient rise in amylase levels, which happened in three patients. The regimen’s overall performance in adolescents closely tracked what’s been seen in adults, Dr. Murray said.
“These data will lead to FDA approval” of the regimen for adolescents, she said confidently, and that will ease insurance coverage. “There will still be hoops to jump through, but with approval and once written into guidelines, insurers will be under pressure to reimburse for it,” she noted. “We find that, because of the cost, insurers resist or refuse to pay for these medications. The idea of treating only patients with advanced liver disease is morally inappropriate. These children must be treated before they develop significant or irreversible liver 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
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
AT WCPGHAN 2016
Key clinical point:
Major finding: Hepatitis C virus was undetectable after 12 weeks of treatment in 98%; the remaining two patients were lost to follow-up.
Data source: A multicenter, open-label study with 100 patients, aged 12-17 years old, and chronically infected with hepatitis C genotype 1.
Disclosures: The study was sponsored by Gilead, the company that markets ledipasvir/sofosbuvir (Harvoni). Dr. Murray has received research support from Gilead and is a shareholder in Merck.
FDA reaffirms rivaroxaban’s atrial fib efficacy in ROCKET AF
The Food and Drug Administration reaffirmed its confidence in the data supporting the claim that rivaroxaban (Xarelto) is a safe and effective alternative to warfarin for preventing strokes and blood clots in patients with nonvalvular atrial fibrillation.
“The FDA concludes that Xarelto is a safe and effective alternative to warfarin in patients with atrial fibrillation,” the agency said in a statement released on Oct. 11.
In response to these events the FDA “completed a variety of analyses to assess the impact that this faulty monitoring device had on the ROCKET AF study results. The agency has determined that effects on strokes or bleeding, including bleeding in the head, were minimal,” the agency said in its statement.
Researchers associated with ROCKET AF published their own analysis of the impact of the faulty device on bleeding rates among patients treated with warfarin in the trial and concluded that device malfunction did not appear to influence the results (N Engl J Med. 2016 Feb 25;374[8]:785-8).
Rivaroxaban is one of four new oral anticoagulants (NOACs) on the U.S. market that are alternatives to warfarin for stroke and clot prevention in patients with nonvalvular atrial fibrillation. An analysis of 2014 data on U.S. office-based prescriptions for NOACs in atrial fibrillation patients showed that rivaroxaban was by far the most commonly prescribed drug in the class, prescribed for patients during 48% of physician office visits that led to a NOAC prescription (Am J Med. 2015 Dec;128[12]:1300-5).
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
The Food and Drug Administration reaffirmed its confidence in the data supporting the claim that rivaroxaban (Xarelto) is a safe and effective alternative to warfarin for preventing strokes and blood clots in patients with nonvalvular atrial fibrillation.
“The FDA concludes that Xarelto is a safe and effective alternative to warfarin in patients with atrial fibrillation,” the agency said in a statement released on Oct. 11.
In response to these events the FDA “completed a variety of analyses to assess the impact that this faulty monitoring device had on the ROCKET AF study results. The agency has determined that effects on strokes or bleeding, including bleeding in the head, were minimal,” the agency said in its statement.
Researchers associated with ROCKET AF published their own analysis of the impact of the faulty device on bleeding rates among patients treated with warfarin in the trial and concluded that device malfunction did not appear to influence the results (N Engl J Med. 2016 Feb 25;374[8]:785-8).
Rivaroxaban is one of four new oral anticoagulants (NOACs) on the U.S. market that are alternatives to warfarin for stroke and clot prevention in patients with nonvalvular atrial fibrillation. An analysis of 2014 data on U.S. office-based prescriptions for NOACs in atrial fibrillation patients showed that rivaroxaban was by far the most commonly prescribed drug in the class, prescribed for patients during 48% of physician office visits that led to a NOAC prescription (Am J Med. 2015 Dec;128[12]:1300-5).
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
The Food and Drug Administration reaffirmed its confidence in the data supporting the claim that rivaroxaban (Xarelto) is a safe and effective alternative to warfarin for preventing strokes and blood clots in patients with nonvalvular atrial fibrillation.
“The FDA concludes that Xarelto is a safe and effective alternative to warfarin in patients with atrial fibrillation,” the agency said in a statement released on Oct. 11.
In response to these events the FDA “completed a variety of analyses to assess the impact that this faulty monitoring device had on the ROCKET AF study results. The agency has determined that effects on strokes or bleeding, including bleeding in the head, were minimal,” the agency said in its statement.
Researchers associated with ROCKET AF published their own analysis of the impact of the faulty device on bleeding rates among patients treated with warfarin in the trial and concluded that device malfunction did not appear to influence the results (N Engl J Med. 2016 Feb 25;374[8]:785-8).
Rivaroxaban is one of four new oral anticoagulants (NOACs) on the U.S. market that are alternatives to warfarin for stroke and clot prevention in patients with nonvalvular atrial fibrillation. An analysis of 2014 data on U.S. office-based prescriptions for NOACs in atrial fibrillation patients showed that rivaroxaban was by far the most commonly prescribed drug in the class, prescribed for patients during 48% of physician office visits that led to a NOAC prescription (Am J Med. 2015 Dec;128[12]:1300-5).
mzoler@frontlinemedcom.com
On Twitter @mitchelzoler
October 2016: Click for Credit
Here are 5 articles in the October issue of Clinician Reviews (individual articles are valid for one year from date of publication—expiration dates below):
1. Autism Follow-up Screening by PCPs Yields High Accuracy
To take the posttest, go to: http://bit.ly/2bTLhFS
Expires August 19, 2017
VITALS
Key clinical point:
Primary care providers can conduct the M-CHAT/F following a positive M-CHAT screening for autism spectrum disorders.
Major finding:
Primary care providers and trained interviewers agreed 86.6% of the time on the screening results of the M-CHAT/F for ASDs.
Data source:
A cohort study of 5,071 children, mean age 23 months, screened with the M-CHAT, and a subsequent 197 children screened with the M-CHAT/F in 22 Maryland primary care practices.
Disclosures:
The National Institutes of Mental Health funded the research. Dr. Sturner is director of Total Child Health (TCH), a for-profit subsidiary of the Center for Promotion of Child Development through Primary Care, which conducted the study. Barbara Howard, MD, is president of TCH. Tanya Morrel, PhD, is an employee of and stockholder in TCH, and Paul Bergmann has consulted for the company. The remaining authors had no relevant disclosures.
2. Gallstone Disease Boosts Heart Risk
To take the posttest, go to: http://bit.ly/2c7TP7D
Expires August 18, 2017
VITALS
Key clinical point:
Gallstone disease is associated with an increased risk for coronary heart disease; preventing the former can help mitigate chances of developing the latter.
Major finding:
A meta-analysis revealed a 23% increased chance of CHD in gallstone disease patients.
Data source:
A meta-analysis of seven studies involving 842,553 subjects, and a prospective cohort study of 269,142 participants in three separate studies that took place from 1980 to 2011.
Disclosures:
Funding provided by NIH, Boston Obesity Nutrition Research Center, and United States-Israel Binational Science Foundation. The authors had no relevant financial disclosures.
3. New HER2-testing Guidelines Result in More Women Eligible for Directed Treatment
To take the posttest, go to: http://bit.ly/2cd9llO
Expires July 25, 2017
VITALS
Key clinical point:
New IHC and FISH pathology guidelines categorize more breast cancers as "equivocal" regarding HER2 positivity and ultimately lead to identifying more of them as HER2 positive.
Major finding:
By using 2013 guidelines, 358 additional tumors were interpreted as positive, compared with the 2007 guidelines and 298 additional tumors were considered positive, compared with the FDA criteria.
Data source:
A cohort study involving 2,851 breast cancer samples analyzed according to three different pathology guidelines during a 1-year period.
Disclosures:
This study was supported by the Mayo Clinic. Dr. Shah reported having no relevant financial disclosures; his associates reported ties to Merck, Hospira, Ariad Pharmaceuticals, Abbott Molecular, and Genome Diagnostics.
4. Extreme Alcohol Use Worsens HIV Disease
To take the posttest, go to: http://bit.ly/2coIzG3
Expires August 14, 2017
VITALS
Key clinical point:
A pattern of heavy alcohol use over time in HIV-infected patients was associated with accelerated HIV disease progression.
Major finding:
Long-term heavy alcohol use by middle-aged, HIV-infected military veterans was associated with a 1.83-fold increased likelihood of also being in the highest-risk group for accelerated progression of HIV disease.
Data source:
This study included 3,539 U.S. military veterans receiving care for HIV infection at eight VA centers. The impact of their long-term pattern of alcohol use on HIV disease progression was assessed over an 8-year period by annual assessments using validated instruments.
Disclosures:
The presenter reported having no financial conflicts of interest regarding the study, funded by the National Institute on Alcohol Abuse and Alcoholism and the National Institute of Allergy and Infectious Diseases.
5. Weight Loss Boosts TNFis' Psoriatic Arthritis Efficacy
To take the posttest, go to: http://bit.ly/2chD4M1
Expires July 23, 2017
Here are 5 articles in the October issue of Clinician Reviews (individual articles are valid for one year from date of publication—expiration dates below):
1. Autism Follow-up Screening by PCPs Yields High Accuracy
To take the posttest, go to: http://bit.ly/2bTLhFS
Expires August 19, 2017
VITALS
Key clinical point:
Primary care providers can conduct the M-CHAT/F following a positive M-CHAT screening for autism spectrum disorders.
Major finding:
Primary care providers and trained interviewers agreed 86.6% of the time on the screening results of the M-CHAT/F for ASDs.
Data source:
A cohort study of 5,071 children, mean age 23 months, screened with the M-CHAT, and a subsequent 197 children screened with the M-CHAT/F in 22 Maryland primary care practices.
Disclosures:
The National Institutes of Mental Health funded the research. Dr. Sturner is director of Total Child Health (TCH), a for-profit subsidiary of the Center for Promotion of Child Development through Primary Care, which conducted the study. Barbara Howard, MD, is president of TCH. Tanya Morrel, PhD, is an employee of and stockholder in TCH, and Paul Bergmann has consulted for the company. The remaining authors had no relevant disclosures.
2. Gallstone Disease Boosts Heart Risk
To take the posttest, go to: http://bit.ly/2c7TP7D
Expires August 18, 2017
VITALS
Key clinical point:
Gallstone disease is associated with an increased risk for coronary heart disease; preventing the former can help mitigate chances of developing the latter.
Major finding:
A meta-analysis revealed a 23% increased chance of CHD in gallstone disease patients.
Data source:
A meta-analysis of seven studies involving 842,553 subjects, and a prospective cohort study of 269,142 participants in three separate studies that took place from 1980 to 2011.
Disclosures:
Funding provided by NIH, Boston Obesity Nutrition Research Center, and United States-Israel Binational Science Foundation. The authors had no relevant financial disclosures.
3. New HER2-testing Guidelines Result in More Women Eligible for Directed Treatment
To take the posttest, go to: http://bit.ly/2cd9llO
Expires July 25, 2017
VITALS
Key clinical point:
New IHC and FISH pathology guidelines categorize more breast cancers as "equivocal" regarding HER2 positivity and ultimately lead to identifying more of them as HER2 positive.
Major finding:
By using 2013 guidelines, 358 additional tumors were interpreted as positive, compared with the 2007 guidelines and 298 additional tumors were considered positive, compared with the FDA criteria.
Data source:
A cohort study involving 2,851 breast cancer samples analyzed according to three different pathology guidelines during a 1-year period.
Disclosures:
This study was supported by the Mayo Clinic. Dr. Shah reported having no relevant financial disclosures; his associates reported ties to Merck, Hospira, Ariad Pharmaceuticals, Abbott Molecular, and Genome Diagnostics.
4. Extreme Alcohol Use Worsens HIV Disease
To take the posttest, go to: http://bit.ly/2coIzG3
Expires August 14, 2017
VITALS
Key clinical point:
A pattern of heavy alcohol use over time in HIV-infected patients was associated with accelerated HIV disease progression.
Major finding:
Long-term heavy alcohol use by middle-aged, HIV-infected military veterans was associated with a 1.83-fold increased likelihood of also being in the highest-risk group for accelerated progression of HIV disease.
Data source:
This study included 3,539 U.S. military veterans receiving care for HIV infection at eight VA centers. The impact of their long-term pattern of alcohol use on HIV disease progression was assessed over an 8-year period by annual assessments using validated instruments.
Disclosures:
The presenter reported having no financial conflicts of interest regarding the study, funded by the National Institute on Alcohol Abuse and Alcoholism and the National Institute of Allergy and Infectious Diseases.
5. Weight Loss Boosts TNFis' Psoriatic Arthritis Efficacy
To take the posttest, go to: http://bit.ly/2chD4M1
Expires July 23, 2017
Here are 5 articles in the October issue of Clinician Reviews (individual articles are valid for one year from date of publication—expiration dates below):
1. Autism Follow-up Screening by PCPs Yields High Accuracy
To take the posttest, go to: http://bit.ly/2bTLhFS
Expires August 19, 2017
VITALS
Key clinical point:
Primary care providers can conduct the M-CHAT/F following a positive M-CHAT screening for autism spectrum disorders.
Major finding:
Primary care providers and trained interviewers agreed 86.6% of the time on the screening results of the M-CHAT/F for ASDs.
Data source:
A cohort study of 5,071 children, mean age 23 months, screened with the M-CHAT, and a subsequent 197 children screened with the M-CHAT/F in 22 Maryland primary care practices.
Disclosures:
The National Institutes of Mental Health funded the research. Dr. Sturner is director of Total Child Health (TCH), a for-profit subsidiary of the Center for Promotion of Child Development through Primary Care, which conducted the study. Barbara Howard, MD, is president of TCH. Tanya Morrel, PhD, is an employee of and stockholder in TCH, and Paul Bergmann has consulted for the company. The remaining authors had no relevant disclosures.
2. Gallstone Disease Boosts Heart Risk
To take the posttest, go to: http://bit.ly/2c7TP7D
Expires August 18, 2017
VITALS
Key clinical point:
Gallstone disease is associated with an increased risk for coronary heart disease; preventing the former can help mitigate chances of developing the latter.
Major finding:
A meta-analysis revealed a 23% increased chance of CHD in gallstone disease patients.
Data source:
A meta-analysis of seven studies involving 842,553 subjects, and a prospective cohort study of 269,142 participants in three separate studies that took place from 1980 to 2011.
Disclosures:
Funding provided by NIH, Boston Obesity Nutrition Research Center, and United States-Israel Binational Science Foundation. The authors had no relevant financial disclosures.
3. New HER2-testing Guidelines Result in More Women Eligible for Directed Treatment
To take the posttest, go to: http://bit.ly/2cd9llO
Expires July 25, 2017
VITALS
Key clinical point:
New IHC and FISH pathology guidelines categorize more breast cancers as "equivocal" regarding HER2 positivity and ultimately lead to identifying more of them as HER2 positive.
Major finding:
By using 2013 guidelines, 358 additional tumors were interpreted as positive, compared with the 2007 guidelines and 298 additional tumors were considered positive, compared with the FDA criteria.
Data source:
A cohort study involving 2,851 breast cancer samples analyzed according to three different pathology guidelines during a 1-year period.
Disclosures:
This study was supported by the Mayo Clinic. Dr. Shah reported having no relevant financial disclosures; his associates reported ties to Merck, Hospira, Ariad Pharmaceuticals, Abbott Molecular, and Genome Diagnostics.
4. Extreme Alcohol Use Worsens HIV Disease
To take the posttest, go to: http://bit.ly/2coIzG3
Expires August 14, 2017
VITALS
Key clinical point:
A pattern of heavy alcohol use over time in HIV-infected patients was associated with accelerated HIV disease progression.
Major finding:
Long-term heavy alcohol use by middle-aged, HIV-infected military veterans was associated with a 1.83-fold increased likelihood of also being in the highest-risk group for accelerated progression of HIV disease.
Data source:
This study included 3,539 U.S. military veterans receiving care for HIV infection at eight VA centers. The impact of their long-term pattern of alcohol use on HIV disease progression was assessed over an 8-year period by annual assessments using validated instruments.
Disclosures:
The presenter reported having no financial conflicts of interest regarding the study, funded by the National Institute on Alcohol Abuse and Alcoholism and the National Institute of Allergy and Infectious Diseases.
5. Weight Loss Boosts TNFis' Psoriatic Arthritis Efficacy
To take the posttest, go to: http://bit.ly/2chD4M1
Expires July 23, 2017
Reimbursement hurdles hinder Entresto use in HFrEF
ORLANDO – Use of sacubitril/valsartan to treat patients with heart failure with reduced ejection fraction (HFrEF) became a class I recommendation in both the U.S. and European heart failure guidelines in May 2016, but virtually all U.S. health insurers continue to regard the potent and effective sacubitril/valsartan formulation as a second-line treatment that needs special preauthorization before patients receive reimbursement for the prescription.
“What is really morally sad is that U.S. payers are requiring physicians to fill out extensive, patient-by-patient paperwork” to allow patients with HFrEF to receive health insurance coverage for sacubitril/valsartan (Entresto), Milton Packer, MD, said at the annual scientific meeting of the Heart Failure Society of America.
“It is very difficult to understand why third-party payers would intentionally try to slow adoption of this life-saving drug simply because it is considered expensive. It is much cheaper than many drugs they cover for patients with cancer that don’t work half as well,” said Dr. Packer, a cardiologist and heart failure specialist at Baylor University Medical Center in Dallas.
The “excessive paperwork” for insurers when starting patients on sacubitril/valsartan is a “new and unique phenomenon among the cardiovascular drugs I prescribe,” agreed Nancy K. Sweitzer, MD, PhD, professor and chief of cardiology at the University of Arizona in Tuscon. “This approach by insurers seems based on cost; they do not want to pay” for sacubitril/valsartan, and to successfully arrange for coverage patients need to exactly match the enrollment criteria used in the PARADIGM-HF (Prospective Comparison of ARNI [Angiotensin Receptor – Neprilysin Inhibitor] with ACEI [Angiotensin-Converting Enzyme Inhibitor] to Determine Impact on Global Mortality and Morbidity in Heart Failure Trial ), the pivotal study that supplied the evidence base for making sacubitril/valsartan a class I agent for treating HFrEF.
“I don’t put some HFrEF patients on sacubitil/valsartan just because they don’t meet the trial’s entry criteria,” Dr. Sweitzer said in an interview. “Insurers seem to scrutinize every single parameter to make sure patients match the PARADIGM-HF patients. Coverage is denied if their BNP [brain natriuretic peptide] level is too low.” Dr. Sweitzer added that in one instance she had to submit a second preauthorization to simply uptitrate the dosage of sacubitril/valsartan she wanted a patient to receive.
“Based on the data it seems like you could easily identify HFrEF patients who are good candidates for sacubitril/valsartan, but your hands are tied by payers because you can’t prescribe it until you’ve first tried something else, and even then you still need to deal with a lot of paperwork,” agreed Robert O. Bonow, MD, professor of medicine at Northwestern University in Chicago. “The paperwork burden is really cumbersome for physicians with busy practices; it impedes taking care of patients,” Dr. Bonow said in an interview.
Sales figures for sacubitril/valsartan that the drug’s manufacturer, Novartis, has reported since the agent received U.S. marketing approval a little over a year ago reflect these challenges in prescribing the compound to patients. During the first quarter of 2016, Novartis reported $17 million in worldwide sales of the agent, followed by $32 million in worldwide sales during the second quarter of 2016, through June 30. With a total of $49 million in sacubitril/valsartan sales during the first 6 months of 2016, it seems like Novartis may be challenged to meet its stated target of $200 million in total sales of the compound during 2016. In April, one commentator called the $17 million sales figure for first quarter 2016 “an astonishingly small amount for a drug that was widely expected to be a blockbuster.”
Dr. Packer has in the past been a consultant to Novartis and was one of the lead investigators for the PARADIGM-HF trial. He said that currently he has no financial relationship with Novartis but he does serve as a consultant to several other drug companies. Dr. Sweitzer has received research support from Novartis and was an investigator for PARADIGM-HF. Dr. Bonow has been a consultant to Gilead.
On Twitter @mitchelzoler
ORLANDO – Use of sacubitril/valsartan to treat patients with heart failure with reduced ejection fraction (HFrEF) became a class I recommendation in both the U.S. and European heart failure guidelines in May 2016, but virtually all U.S. health insurers continue to regard the potent and effective sacubitril/valsartan formulation as a second-line treatment that needs special preauthorization before patients receive reimbursement for the prescription.
“What is really morally sad is that U.S. payers are requiring physicians to fill out extensive, patient-by-patient paperwork” to allow patients with HFrEF to receive health insurance coverage for sacubitril/valsartan (Entresto), Milton Packer, MD, said at the annual scientific meeting of the Heart Failure Society of America.
“It is very difficult to understand why third-party payers would intentionally try to slow adoption of this life-saving drug simply because it is considered expensive. It is much cheaper than many drugs they cover for patients with cancer that don’t work half as well,” said Dr. Packer, a cardiologist and heart failure specialist at Baylor University Medical Center in Dallas.
The “excessive paperwork” for insurers when starting patients on sacubitril/valsartan is a “new and unique phenomenon among the cardiovascular drugs I prescribe,” agreed Nancy K. Sweitzer, MD, PhD, professor and chief of cardiology at the University of Arizona in Tuscon. “This approach by insurers seems based on cost; they do not want to pay” for sacubitril/valsartan, and to successfully arrange for coverage patients need to exactly match the enrollment criteria used in the PARADIGM-HF (Prospective Comparison of ARNI [Angiotensin Receptor – Neprilysin Inhibitor] with ACEI [Angiotensin-Converting Enzyme Inhibitor] to Determine Impact on Global Mortality and Morbidity in Heart Failure Trial ), the pivotal study that supplied the evidence base for making sacubitril/valsartan a class I agent for treating HFrEF.
“I don’t put some HFrEF patients on sacubitil/valsartan just because they don’t meet the trial’s entry criteria,” Dr. Sweitzer said in an interview. “Insurers seem to scrutinize every single parameter to make sure patients match the PARADIGM-HF patients. Coverage is denied if their BNP [brain natriuretic peptide] level is too low.” Dr. Sweitzer added that in one instance she had to submit a second preauthorization to simply uptitrate the dosage of sacubitril/valsartan she wanted a patient to receive.
“Based on the data it seems like you could easily identify HFrEF patients who are good candidates for sacubitril/valsartan, but your hands are tied by payers because you can’t prescribe it until you’ve first tried something else, and even then you still need to deal with a lot of paperwork,” agreed Robert O. Bonow, MD, professor of medicine at Northwestern University in Chicago. “The paperwork burden is really cumbersome for physicians with busy practices; it impedes taking care of patients,” Dr. Bonow said in an interview.
Sales figures for sacubitril/valsartan that the drug’s manufacturer, Novartis, has reported since the agent received U.S. marketing approval a little over a year ago reflect these challenges in prescribing the compound to patients. During the first quarter of 2016, Novartis reported $17 million in worldwide sales of the agent, followed by $32 million in worldwide sales during the second quarter of 2016, through June 30. With a total of $49 million in sacubitril/valsartan sales during the first 6 months of 2016, it seems like Novartis may be challenged to meet its stated target of $200 million in total sales of the compound during 2016. In April, one commentator called the $17 million sales figure for first quarter 2016 “an astonishingly small amount for a drug that was widely expected to be a blockbuster.”
Dr. Packer has in the past been a consultant to Novartis and was one of the lead investigators for the PARADIGM-HF trial. He said that currently he has no financial relationship with Novartis but he does serve as a consultant to several other drug companies. Dr. Sweitzer has received research support from Novartis and was an investigator for PARADIGM-HF. Dr. Bonow has been a consultant to Gilead.
On Twitter @mitchelzoler
ORLANDO – Use of sacubitril/valsartan to treat patients with heart failure with reduced ejection fraction (HFrEF) became a class I recommendation in both the U.S. and European heart failure guidelines in May 2016, but virtually all U.S. health insurers continue to regard the potent and effective sacubitril/valsartan formulation as a second-line treatment that needs special preauthorization before patients receive reimbursement for the prescription.
“What is really morally sad is that U.S. payers are requiring physicians to fill out extensive, patient-by-patient paperwork” to allow patients with HFrEF to receive health insurance coverage for sacubitril/valsartan (Entresto), Milton Packer, MD, said at the annual scientific meeting of the Heart Failure Society of America.
“It is very difficult to understand why third-party payers would intentionally try to slow adoption of this life-saving drug simply because it is considered expensive. It is much cheaper than many drugs they cover for patients with cancer that don’t work half as well,” said Dr. Packer, a cardiologist and heart failure specialist at Baylor University Medical Center in Dallas.
The “excessive paperwork” for insurers when starting patients on sacubitril/valsartan is a “new and unique phenomenon among the cardiovascular drugs I prescribe,” agreed Nancy K. Sweitzer, MD, PhD, professor and chief of cardiology at the University of Arizona in Tuscon. “This approach by insurers seems based on cost; they do not want to pay” for sacubitril/valsartan, and to successfully arrange for coverage patients need to exactly match the enrollment criteria used in the PARADIGM-HF (Prospective Comparison of ARNI [Angiotensin Receptor – Neprilysin Inhibitor] with ACEI [Angiotensin-Converting Enzyme Inhibitor] to Determine Impact on Global Mortality and Morbidity in Heart Failure Trial ), the pivotal study that supplied the evidence base for making sacubitril/valsartan a class I agent for treating HFrEF.
“I don’t put some HFrEF patients on sacubitil/valsartan just because they don’t meet the trial’s entry criteria,” Dr. Sweitzer said in an interview. “Insurers seem to scrutinize every single parameter to make sure patients match the PARADIGM-HF patients. Coverage is denied if their BNP [brain natriuretic peptide] level is too low.” Dr. Sweitzer added that in one instance she had to submit a second preauthorization to simply uptitrate the dosage of sacubitril/valsartan she wanted a patient to receive.
“Based on the data it seems like you could easily identify HFrEF patients who are good candidates for sacubitril/valsartan, but your hands are tied by payers because you can’t prescribe it until you’ve first tried something else, and even then you still need to deal with a lot of paperwork,” agreed Robert O. Bonow, MD, professor of medicine at Northwestern University in Chicago. “The paperwork burden is really cumbersome for physicians with busy practices; it impedes taking care of patients,” Dr. Bonow said in an interview.
Sales figures for sacubitril/valsartan that the drug’s manufacturer, Novartis, has reported since the agent received U.S. marketing approval a little over a year ago reflect these challenges in prescribing the compound to patients. During the first quarter of 2016, Novartis reported $17 million in worldwide sales of the agent, followed by $32 million in worldwide sales during the second quarter of 2016, through June 30. With a total of $49 million in sacubitril/valsartan sales during the first 6 months of 2016, it seems like Novartis may be challenged to meet its stated target of $200 million in total sales of the compound during 2016. In April, one commentator called the $17 million sales figure for first quarter 2016 “an astonishingly small amount for a drug that was widely expected to be a blockbuster.”
Dr. Packer has in the past been a consultant to Novartis and was one of the lead investigators for the PARADIGM-HF trial. He said that currently he has no financial relationship with Novartis but he does serve as a consultant to several other drug companies. Dr. Sweitzer has received research support from Novartis and was an investigator for PARADIGM-HF. Dr. Bonow has been a consultant to Gilead.
On Twitter @mitchelzoler
EXPERT ANALYSIS FROM THE HFSA ANNUAL SCIENTIFIC MEETING
NOACs outpace warfarin for afib anticoagulation
The NOAC revolution has happened.
New oral anticoagulants (NOACs) now claim the majority of the oral anticoagulant market in patients with new atrial fibrillation, wresting the advantage from warfarin in the U.S. and globally. This is the promise NOACs held even while still in development, the potential to whittle warfarin use down to a shadow of what it was. Despite a sputtering reception when the first NOAC, dabigatran (Pradaxa), came onto the U.S. market in late 2010, the four NOACs (also apixaban [Eliquis], edoxaban [Savaysa], and rivaroxaban [Xarelto]) now available in the United States and elsewhere gradually gained traction and today they collectively form the most widely used oral anticoagulant option for patients newly diagnosed with atrial fibrillation.
Data reported in August at the annual congress of the European Society of Cardiology showed that in Denmark during the first half of 2015, NOACs accounted for 73% of oral anticoagulant prescriptions for patients with newly diagnosed atrial fibrillation, Kasper Gadsboll, MD, reported. He and his associates studied NOAC and warfarin use in a total of 108,410 atrial fibrillation patients newly diagnosed starting in 2005. Through 2010, warfarin was the only option, but starting in early 2011 when the first NOAC became available in Denmark, use of the class rose sharply with a corresponding plummet in warfarin prescriptions. Not only did the NOACs largely supplant warfarin during 2011-2015, but they also powered an overall rise in the percentage of atrial fibrillation patients treated with an oral anticoagulant, boosting the rate by a relative 75% between the end of 2009 and mid-2015.
“People were reluctant to start an oral anticoagulant because they feared bleeding. Now with NOACs they are not as fearful,” said Dr. Gadsboll, a cardiology researcher at Gentofte Hospital in Hellerup, Denmark. He also noted that in Denmark the national health system pays for prescribed drugs, so the increased direct cost for NOAC treatment in place of warfarin is covered by the Danish government.
“NOAC uptake is gathering steam,” commented Stuart J. Connolly, MD, an electrophysiologist and professor of medicine at McMaster University in Hamilton, Ontario. “When the government pays for it, people prescribe it more, but increased NOAC use is happening everywhere,” said Dr. Connolly, who led major trials that assessed dabigatran and apixaban in atrial fibrillation patients. “I’ve seen Canadian data that show warfarin use is falling and NOAC use is increasing. People are more willing to prescribe NOACs because they are safer,” he said in an interview.
The most current U.S. data I found came from the IMS Health National Disease and Therapeutic Index in 2014, based on a survey of a representative sample of about 4,800 U.S. office-based physicians. The results showed that the NOAC share of oral anticoagulant use in patients who had office visits for atrial fibrillation jumped from 6% of patients in early 2011 to roughly half of all atrial fibrillation patients, 48%, during 2014 (Am J Med. 2015 Dec;128[12]:1300-5). That trajectory makes it likely that by now NOACs have a clear lead.
It’s a similar story in several European countries, such as in Belgium where NOACs now account for about 70% of new prescriptions for atrial fibrillation patients, commented Freek W.A. Verheugt, MD. In the Netherlands, however, NOACs have not taken off, in large part because a popular and entrenched thrombosis clinic system exists that employs thousands of Dutch workers, thereby making choice of an anticoagulant drug a social and political question as well as a medical one, he said. “Warfarin is still indicated for patients with an artificial heart valve or poor kidney function,” noted Dr. Verheugt, professor of cardiology at the University of Nijmegen in the Netherlands, “but a majority of atrial fibrillation patients will eventually change to a NOAC. Even when treatment with warfarin has a good time-in-therapeutic-range, NOACs are still better. They are so much safer.”
On Twitter @mitchelzoler
The NOAC revolution has happened.
New oral anticoagulants (NOACs) now claim the majority of the oral anticoagulant market in patients with new atrial fibrillation, wresting the advantage from warfarin in the U.S. and globally. This is the promise NOACs held even while still in development, the potential to whittle warfarin use down to a shadow of what it was. Despite a sputtering reception when the first NOAC, dabigatran (Pradaxa), came onto the U.S. market in late 2010, the four NOACs (also apixaban [Eliquis], edoxaban [Savaysa], and rivaroxaban [Xarelto]) now available in the United States and elsewhere gradually gained traction and today they collectively form the most widely used oral anticoagulant option for patients newly diagnosed with atrial fibrillation.
Data reported in August at the annual congress of the European Society of Cardiology showed that in Denmark during the first half of 2015, NOACs accounted for 73% of oral anticoagulant prescriptions for patients with newly diagnosed atrial fibrillation, Kasper Gadsboll, MD, reported. He and his associates studied NOAC and warfarin use in a total of 108,410 atrial fibrillation patients newly diagnosed starting in 2005. Through 2010, warfarin was the only option, but starting in early 2011 when the first NOAC became available in Denmark, use of the class rose sharply with a corresponding plummet in warfarin prescriptions. Not only did the NOACs largely supplant warfarin during 2011-2015, but they also powered an overall rise in the percentage of atrial fibrillation patients treated with an oral anticoagulant, boosting the rate by a relative 75% between the end of 2009 and mid-2015.
“People were reluctant to start an oral anticoagulant because they feared bleeding. Now with NOACs they are not as fearful,” said Dr. Gadsboll, a cardiology researcher at Gentofte Hospital in Hellerup, Denmark. He also noted that in Denmark the national health system pays for prescribed drugs, so the increased direct cost for NOAC treatment in place of warfarin is covered by the Danish government.
“NOAC uptake is gathering steam,” commented Stuart J. Connolly, MD, an electrophysiologist and professor of medicine at McMaster University in Hamilton, Ontario. “When the government pays for it, people prescribe it more, but increased NOAC use is happening everywhere,” said Dr. Connolly, who led major trials that assessed dabigatran and apixaban in atrial fibrillation patients. “I’ve seen Canadian data that show warfarin use is falling and NOAC use is increasing. People are more willing to prescribe NOACs because they are safer,” he said in an interview.
The most current U.S. data I found came from the IMS Health National Disease and Therapeutic Index in 2014, based on a survey of a representative sample of about 4,800 U.S. office-based physicians. The results showed that the NOAC share of oral anticoagulant use in patients who had office visits for atrial fibrillation jumped from 6% of patients in early 2011 to roughly half of all atrial fibrillation patients, 48%, during 2014 (Am J Med. 2015 Dec;128[12]:1300-5). That trajectory makes it likely that by now NOACs have a clear lead.
It’s a similar story in several European countries, such as in Belgium where NOACs now account for about 70% of new prescriptions for atrial fibrillation patients, commented Freek W.A. Verheugt, MD. In the Netherlands, however, NOACs have not taken off, in large part because a popular and entrenched thrombosis clinic system exists that employs thousands of Dutch workers, thereby making choice of an anticoagulant drug a social and political question as well as a medical one, he said. “Warfarin is still indicated for patients with an artificial heart valve or poor kidney function,” noted Dr. Verheugt, professor of cardiology at the University of Nijmegen in the Netherlands, “but a majority of atrial fibrillation patients will eventually change to a NOAC. Even when treatment with warfarin has a good time-in-therapeutic-range, NOACs are still better. They are so much safer.”
On Twitter @mitchelzoler
The NOAC revolution has happened.
New oral anticoagulants (NOACs) now claim the majority of the oral anticoagulant market in patients with new atrial fibrillation, wresting the advantage from warfarin in the U.S. and globally. This is the promise NOACs held even while still in development, the potential to whittle warfarin use down to a shadow of what it was. Despite a sputtering reception when the first NOAC, dabigatran (Pradaxa), came onto the U.S. market in late 2010, the four NOACs (also apixaban [Eliquis], edoxaban [Savaysa], and rivaroxaban [Xarelto]) now available in the United States and elsewhere gradually gained traction and today they collectively form the most widely used oral anticoagulant option for patients newly diagnosed with atrial fibrillation.
Data reported in August at the annual congress of the European Society of Cardiology showed that in Denmark during the first half of 2015, NOACs accounted for 73% of oral anticoagulant prescriptions for patients with newly diagnosed atrial fibrillation, Kasper Gadsboll, MD, reported. He and his associates studied NOAC and warfarin use in a total of 108,410 atrial fibrillation patients newly diagnosed starting in 2005. Through 2010, warfarin was the only option, but starting in early 2011 when the first NOAC became available in Denmark, use of the class rose sharply with a corresponding plummet in warfarin prescriptions. Not only did the NOACs largely supplant warfarin during 2011-2015, but they also powered an overall rise in the percentage of atrial fibrillation patients treated with an oral anticoagulant, boosting the rate by a relative 75% between the end of 2009 and mid-2015.
“People were reluctant to start an oral anticoagulant because they feared bleeding. Now with NOACs they are not as fearful,” said Dr. Gadsboll, a cardiology researcher at Gentofte Hospital in Hellerup, Denmark. He also noted that in Denmark the national health system pays for prescribed drugs, so the increased direct cost for NOAC treatment in place of warfarin is covered by the Danish government.
“NOAC uptake is gathering steam,” commented Stuart J. Connolly, MD, an electrophysiologist and professor of medicine at McMaster University in Hamilton, Ontario. “When the government pays for it, people prescribe it more, but increased NOAC use is happening everywhere,” said Dr. Connolly, who led major trials that assessed dabigatran and apixaban in atrial fibrillation patients. “I’ve seen Canadian data that show warfarin use is falling and NOAC use is increasing. People are more willing to prescribe NOACs because they are safer,” he said in an interview.
The most current U.S. data I found came from the IMS Health National Disease and Therapeutic Index in 2014, based on a survey of a representative sample of about 4,800 U.S. office-based physicians. The results showed that the NOAC share of oral anticoagulant use in patients who had office visits for atrial fibrillation jumped from 6% of patients in early 2011 to roughly half of all atrial fibrillation patients, 48%, during 2014 (Am J Med. 2015 Dec;128[12]:1300-5). That trajectory makes it likely that by now NOACs have a clear lead.
It’s a similar story in several European countries, such as in Belgium where NOACs now account for about 70% of new prescriptions for atrial fibrillation patients, commented Freek W.A. Verheugt, MD. In the Netherlands, however, NOACs have not taken off, in large part because a popular and entrenched thrombosis clinic system exists that employs thousands of Dutch workers, thereby making choice of an anticoagulant drug a social and political question as well as a medical one, he said. “Warfarin is still indicated for patients with an artificial heart valve or poor kidney function,” noted Dr. Verheugt, professor of cardiology at the University of Nijmegen in the Netherlands, “but a majority of atrial fibrillation patients will eventually change to a NOAC. Even when treatment with warfarin has a good time-in-therapeutic-range, NOACs are still better. They are so much safer.”
On Twitter @mitchelzoler
CardioMEMS shows real-world heart failure benefit
ORLANDO – Pulmonary artery pressure monitoring using an implanted device was even more effective for controlling pulmonary artery pressures in 2,000 real-world U.S. heart failure patients than it was in the pivotal trial that led to the device’s regulatory approval.
Data from the first 2,000 U.S. heart failure patients to receive the CardioMEMS pulmonary artery (PA) pressure monitoring device and have at least 6 months of follow-up data since the device received Food and Drug Administration approval in 2014 showed that cumulative PA pressure reductions in these patients during the first 6 months of use averaged 434 mm Hg per patient when compared with their baseline PA pressure when they first received the device. This was nearly threefold better than the average 150–mm Hg cumulative reduction in PA pressure per patient during 6 months of use seen in the CHAMPION trial, Dr. William T. Abraham reported at the annual scientific meeting of the Heart Failure Society of America.
Although this analysis of data from the registry maintained for U.S. patients who receive the CardioMEMS device does not yet include information on how these patients fared clinically, and specifically how often they required rehospitalization for heart failure, the strikingly high level of PA pressure control seen in the first 2,000 U.S. patients bodes well for what the clinical findings will show once they are available.
“In our experience with PA pressure monitoring, there is almost a linear relationship between reduced PA pressures and reduced numbers of events” in the form of rehospitalizations for heart failure, said Dr. Abraham, professor of medicine and director of cardiovascular medicine at Ohio State University in Columbus. Once data on outcomes are analyzed for the registry patients, “I think they will be even better than they were in the trial,” he said in an interview.
The PA pressure data in these initial patients “are very important because they tell us that in general use, clinicians – many of whom are at community hospitals – are very capable of using the CardioMEMS data to control patient pressures, and in CHAMPION we showed that there is a relationship between controlled pressures and improved outcomes,” he said. The findings also help allay a key concern about the potential benefit from implanting a device to monitor PA pressure, which is that clinicians must respond to the information and tweak a patient’s diuretic and vasodilator treatments in order for pressure monitoring to have an effect on heart failure outcomes.
“These data clearly refute that concern,” Dr. Abraham said.
He expressed some surprise that PA pressure control with monitoring was so much more effective in real-world use than in the CHAMPION pivotal trial. “In the trial, it was a paradigm shift to manage heart failure patients based on their PA pressures and not according to their symptoms,” he said. With CardioMEMS pressure monitoring, clinicians are supposed to treat high PA pressure with dose adjustments even if the patient feels okay. The new data suggest that clinicians now using the device “have gotten the message that if you don’t do something with the data the patients won’t improve.”
The registry patients came from 47 states and 427 unique physicians who worked in a range of settings including large and small centers, and academic and nonacademic community centers. The patients averaged 70 years, 40% were women, a third had a left ventricular ejection fraction at or above 40%, and their average PA pressure at the time they had their device implanted was 34.9 mm Hg. This pressure was notably higher than the average 31.6 mm Hg pressure among patients enrolled in CHAMPION, a fact that also helps explain why the registry patients received a larger pressure-reduction benefit: They started from a higher level than the trial patients, and during follow-up, their achieved pressures were always compared back to their high baseline pressures.
The registry patients were also substantially older than the trial patients, who had averaged 62 years, and the registry included substantially more women and more patients with higher ejection fractions. Dr. Abraham did not report data on their New York Heart Association class at entry, but labeling for CardioMEMS specifies that patients should have class III heart failure as well as a recent heart failure hospitalization.
Dr. Abraham’s analysis also showed that the greatest degree of PA pressure control occurred in the patients who began device-based treatment with the highest PA pressures. Nearly half the 2,000 registry patients had an entry PA pressure at or above 35 mm Hg, and over a period of 6 months, they averaged a cumulative 876–mm Hg reduction in their PA pressure relative to their baseline level. The third of patients who began with a PA pressure of 25-34 mm Hg had an average 169–mm Hg cumulative pressure reduction over the 6 month period, and the 18% of patients who began with a PA pressure of less than 25 mm Hg actually had an average cumulative increase in the PA pressure of 163 mm Hg. Target PA pressures are usually in the normal range of 18-25 mm Hg.
The analyses also showed that the impact of PA pressure monitoring on pressure was roughly similar regardless of the left ventricular ejection fraction patients had at baseline, and regardless of their sex.
The registry data were collected by St. Jude, the company that markets the CardioMEMS device. Dr. Abraham is a consultant to St. Jude and was lead investigator for the CHAMPION pivotal trial.
On Twitter @mitchelzoler
ORLANDO – Pulmonary artery pressure monitoring using an implanted device was even more effective for controlling pulmonary artery pressures in 2,000 real-world U.S. heart failure patients than it was in the pivotal trial that led to the device’s regulatory approval.
Data from the first 2,000 U.S. heart failure patients to receive the CardioMEMS pulmonary artery (PA) pressure monitoring device and have at least 6 months of follow-up data since the device received Food and Drug Administration approval in 2014 showed that cumulative PA pressure reductions in these patients during the first 6 months of use averaged 434 mm Hg per patient when compared with their baseline PA pressure when they first received the device. This was nearly threefold better than the average 150–mm Hg cumulative reduction in PA pressure per patient during 6 months of use seen in the CHAMPION trial, Dr. William T. Abraham reported at the annual scientific meeting of the Heart Failure Society of America.
Although this analysis of data from the registry maintained for U.S. patients who receive the CardioMEMS device does not yet include information on how these patients fared clinically, and specifically how often they required rehospitalization for heart failure, the strikingly high level of PA pressure control seen in the first 2,000 U.S. patients bodes well for what the clinical findings will show once they are available.
“In our experience with PA pressure monitoring, there is almost a linear relationship between reduced PA pressures and reduced numbers of events” in the form of rehospitalizations for heart failure, said Dr. Abraham, professor of medicine and director of cardiovascular medicine at Ohio State University in Columbus. Once data on outcomes are analyzed for the registry patients, “I think they will be even better than they were in the trial,” he said in an interview.
The PA pressure data in these initial patients “are very important because they tell us that in general use, clinicians – many of whom are at community hospitals – are very capable of using the CardioMEMS data to control patient pressures, and in CHAMPION we showed that there is a relationship between controlled pressures and improved outcomes,” he said. The findings also help allay a key concern about the potential benefit from implanting a device to monitor PA pressure, which is that clinicians must respond to the information and tweak a patient’s diuretic and vasodilator treatments in order for pressure monitoring to have an effect on heart failure outcomes.
“These data clearly refute that concern,” Dr. Abraham said.
He expressed some surprise that PA pressure control with monitoring was so much more effective in real-world use than in the CHAMPION pivotal trial. “In the trial, it was a paradigm shift to manage heart failure patients based on their PA pressures and not according to their symptoms,” he said. With CardioMEMS pressure monitoring, clinicians are supposed to treat high PA pressure with dose adjustments even if the patient feels okay. The new data suggest that clinicians now using the device “have gotten the message that if you don’t do something with the data the patients won’t improve.”
The registry patients came from 47 states and 427 unique physicians who worked in a range of settings including large and small centers, and academic and nonacademic community centers. The patients averaged 70 years, 40% were women, a third had a left ventricular ejection fraction at or above 40%, and their average PA pressure at the time they had their device implanted was 34.9 mm Hg. This pressure was notably higher than the average 31.6 mm Hg pressure among patients enrolled in CHAMPION, a fact that also helps explain why the registry patients received a larger pressure-reduction benefit: They started from a higher level than the trial patients, and during follow-up, their achieved pressures were always compared back to their high baseline pressures.
The registry patients were also substantially older than the trial patients, who had averaged 62 years, and the registry included substantially more women and more patients with higher ejection fractions. Dr. Abraham did not report data on their New York Heart Association class at entry, but labeling for CardioMEMS specifies that patients should have class III heart failure as well as a recent heart failure hospitalization.
Dr. Abraham’s analysis also showed that the greatest degree of PA pressure control occurred in the patients who began device-based treatment with the highest PA pressures. Nearly half the 2,000 registry patients had an entry PA pressure at or above 35 mm Hg, and over a period of 6 months, they averaged a cumulative 876–mm Hg reduction in their PA pressure relative to their baseline level. The third of patients who began with a PA pressure of 25-34 mm Hg had an average 169–mm Hg cumulative pressure reduction over the 6 month period, and the 18% of patients who began with a PA pressure of less than 25 mm Hg actually had an average cumulative increase in the PA pressure of 163 mm Hg. Target PA pressures are usually in the normal range of 18-25 mm Hg.
The analyses also showed that the impact of PA pressure monitoring on pressure was roughly similar regardless of the left ventricular ejection fraction patients had at baseline, and regardless of their sex.
The registry data were collected by St. Jude, the company that markets the CardioMEMS device. Dr. Abraham is a consultant to St. Jude and was lead investigator for the CHAMPION pivotal trial.
On Twitter @mitchelzoler
ORLANDO – Pulmonary artery pressure monitoring using an implanted device was even more effective for controlling pulmonary artery pressures in 2,000 real-world U.S. heart failure patients than it was in the pivotal trial that led to the device’s regulatory approval.
Data from the first 2,000 U.S. heart failure patients to receive the CardioMEMS pulmonary artery (PA) pressure monitoring device and have at least 6 months of follow-up data since the device received Food and Drug Administration approval in 2014 showed that cumulative PA pressure reductions in these patients during the first 6 months of use averaged 434 mm Hg per patient when compared with their baseline PA pressure when they first received the device. This was nearly threefold better than the average 150–mm Hg cumulative reduction in PA pressure per patient during 6 months of use seen in the CHAMPION trial, Dr. William T. Abraham reported at the annual scientific meeting of the Heart Failure Society of America.
Although this analysis of data from the registry maintained for U.S. patients who receive the CardioMEMS device does not yet include information on how these patients fared clinically, and specifically how often they required rehospitalization for heart failure, the strikingly high level of PA pressure control seen in the first 2,000 U.S. patients bodes well for what the clinical findings will show once they are available.
“In our experience with PA pressure monitoring, there is almost a linear relationship between reduced PA pressures and reduced numbers of events” in the form of rehospitalizations for heart failure, said Dr. Abraham, professor of medicine and director of cardiovascular medicine at Ohio State University in Columbus. Once data on outcomes are analyzed for the registry patients, “I think they will be even better than they were in the trial,” he said in an interview.
The PA pressure data in these initial patients “are very important because they tell us that in general use, clinicians – many of whom are at community hospitals – are very capable of using the CardioMEMS data to control patient pressures, and in CHAMPION we showed that there is a relationship between controlled pressures and improved outcomes,” he said. The findings also help allay a key concern about the potential benefit from implanting a device to monitor PA pressure, which is that clinicians must respond to the information and tweak a patient’s diuretic and vasodilator treatments in order for pressure monitoring to have an effect on heart failure outcomes.
“These data clearly refute that concern,” Dr. Abraham said.
He expressed some surprise that PA pressure control with monitoring was so much more effective in real-world use than in the CHAMPION pivotal trial. “In the trial, it was a paradigm shift to manage heart failure patients based on their PA pressures and not according to their symptoms,” he said. With CardioMEMS pressure monitoring, clinicians are supposed to treat high PA pressure with dose adjustments even if the patient feels okay. The new data suggest that clinicians now using the device “have gotten the message that if you don’t do something with the data the patients won’t improve.”
The registry patients came from 47 states and 427 unique physicians who worked in a range of settings including large and small centers, and academic and nonacademic community centers. The patients averaged 70 years, 40% were women, a third had a left ventricular ejection fraction at or above 40%, and their average PA pressure at the time they had their device implanted was 34.9 mm Hg. This pressure was notably higher than the average 31.6 mm Hg pressure among patients enrolled in CHAMPION, a fact that also helps explain why the registry patients received a larger pressure-reduction benefit: They started from a higher level than the trial patients, and during follow-up, their achieved pressures were always compared back to their high baseline pressures.
The registry patients were also substantially older than the trial patients, who had averaged 62 years, and the registry included substantially more women and more patients with higher ejection fractions. Dr. Abraham did not report data on their New York Heart Association class at entry, but labeling for CardioMEMS specifies that patients should have class III heart failure as well as a recent heart failure hospitalization.
Dr. Abraham’s analysis also showed that the greatest degree of PA pressure control occurred in the patients who began device-based treatment with the highest PA pressures. Nearly half the 2,000 registry patients had an entry PA pressure at or above 35 mm Hg, and over a period of 6 months, they averaged a cumulative 876–mm Hg reduction in their PA pressure relative to their baseline level. The third of patients who began with a PA pressure of 25-34 mm Hg had an average 169–mm Hg cumulative pressure reduction over the 6 month period, and the 18% of patients who began with a PA pressure of less than 25 mm Hg actually had an average cumulative increase in the PA pressure of 163 mm Hg. Target PA pressures are usually in the normal range of 18-25 mm Hg.
The analyses also showed that the impact of PA pressure monitoring on pressure was roughly similar regardless of the left ventricular ejection fraction patients had at baseline, and regardless of their sex.
The registry data were collected by St. Jude, the company that markets the CardioMEMS device. Dr. Abraham is a consultant to St. Jude and was lead investigator for the CHAMPION pivotal trial.
On Twitter @mitchelzoler
AT THE HFSA ANNUAL SCIENTIFIC MEETING
Key clinical point: Registry data from the first 2,000 U.S. heart failure patients who received an implanted pulmonary artery pressure monitor showed a level of pressure control over a period of 6 months nearly triple that seen in the pivotal trial.
Major finding: Cumulative pulmonary artery pressure reductions averaged 434 mm Hg over a period of 6 months, compared with an average reduction of 150 mm Hg in the pivotal trial.
Data source: The first 2,000 U.S. patients who received a CardioMEMS device and were followed for at least 6 months.
Disclosures: The registry data were collected by St. Jude, the company that markets the CardioMEMS device. Dr. Abraham is a consultant to St. Jude and was lead investigator for the CHAMPION pivotal trial.
