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Sleep disruption, gas exchange abnormalities common in CF
Sleep is an issue for many patients with cystic fibrosis, but noninvasive measures can ease at least some of the problem, according to Dr. Eliot Katz of Harvard Medical School, Boston.
A host of factors can disrupt sleep for patients with CF, including chronic cough, hypoventilation, musculoskeletal pain, gastrointestinal reflux, abdominal discomfort, depression, and the need for frequent defecation, Dr. Katz noted. Children with CF may also have obstructive sleep apnea caused by factors such as adenotonsillar hypertrophy and chronic rhinosinusitis. Some patients also take medications that disrupt sleep or struggle to sleep because they receive overnight enteral feeding, he added (Clin. Chest. Med. 2014 [doi:10.1016/j.ccm.2014.06.005]).
Patients also may develop nocturnal hypercapnia and hypoxemia during sleep. Although nocturnal hypoxemia is most common in patients with advanced lung disease (FEV1 less than 65%), it can also affect patients with milder lung disease, Dr. Katz said. "Hypoxemia is not usually associated with arousal from sleep and, therefore, may be unrecognized," he noted.
As their lung disease progresses, patients with CF develop hypercapnia and subsequent dyspnea, which can be relieved, at least in the short term, by noninvasive ventilation to increase tidal volume and minute ventilation during sleep, Dr. Katz said. He noted that in a study of patients with advanced lung disease, minute ventilation was 22% lower during REM sleep than when patients were awake breathing room air, but was only 14% lower when sleeping patients received noninvasive ventilation (Am. J. Respir. Crit. Care. Med. 2001;163;129-34).
Dr. Katz reported no funding sources and had no disclosures.
Sleep is an issue for many patients with cystic fibrosis, but noninvasive measures can ease at least some of the problem, according to Dr. Eliot Katz of Harvard Medical School, Boston.
A host of factors can disrupt sleep for patients with CF, including chronic cough, hypoventilation, musculoskeletal pain, gastrointestinal reflux, abdominal discomfort, depression, and the need for frequent defecation, Dr. Katz noted. Children with CF may also have obstructive sleep apnea caused by factors such as adenotonsillar hypertrophy and chronic rhinosinusitis. Some patients also take medications that disrupt sleep or struggle to sleep because they receive overnight enteral feeding, he added (Clin. Chest. Med. 2014 [doi:10.1016/j.ccm.2014.06.005]).
Patients also may develop nocturnal hypercapnia and hypoxemia during sleep. Although nocturnal hypoxemia is most common in patients with advanced lung disease (FEV1 less than 65%), it can also affect patients with milder lung disease, Dr. Katz said. "Hypoxemia is not usually associated with arousal from sleep and, therefore, may be unrecognized," he noted.
As their lung disease progresses, patients with CF develop hypercapnia and subsequent dyspnea, which can be relieved, at least in the short term, by noninvasive ventilation to increase tidal volume and minute ventilation during sleep, Dr. Katz said. He noted that in a study of patients with advanced lung disease, minute ventilation was 22% lower during REM sleep than when patients were awake breathing room air, but was only 14% lower when sleeping patients received noninvasive ventilation (Am. J. Respir. Crit. Care. Med. 2001;163;129-34).
Dr. Katz reported no funding sources and had no disclosures.
Sleep is an issue for many patients with cystic fibrosis, but noninvasive measures can ease at least some of the problem, according to Dr. Eliot Katz of Harvard Medical School, Boston.
A host of factors can disrupt sleep for patients with CF, including chronic cough, hypoventilation, musculoskeletal pain, gastrointestinal reflux, abdominal discomfort, depression, and the need for frequent defecation, Dr. Katz noted. Children with CF may also have obstructive sleep apnea caused by factors such as adenotonsillar hypertrophy and chronic rhinosinusitis. Some patients also take medications that disrupt sleep or struggle to sleep because they receive overnight enteral feeding, he added (Clin. Chest. Med. 2014 [doi:10.1016/j.ccm.2014.06.005]).
Patients also may develop nocturnal hypercapnia and hypoxemia during sleep. Although nocturnal hypoxemia is most common in patients with advanced lung disease (FEV1 less than 65%), it can also affect patients with milder lung disease, Dr. Katz said. "Hypoxemia is not usually associated with arousal from sleep and, therefore, may be unrecognized," he noted.
As their lung disease progresses, patients with CF develop hypercapnia and subsequent dyspnea, which can be relieved, at least in the short term, by noninvasive ventilation to increase tidal volume and minute ventilation during sleep, Dr. Katz said. He noted that in a study of patients with advanced lung disease, minute ventilation was 22% lower during REM sleep than when patients were awake breathing room air, but was only 14% lower when sleeping patients received noninvasive ventilation (Am. J. Respir. Crit. Care. Med. 2001;163;129-34).
Dr. Katz reported no funding sources and had no disclosures.
FROM CLINICS IN CHEST MEDICINE
Key clinical point: Noninvasive ventilation and supplemental oxygen can mitigate gas exchange abnormalities in patients with cystic fibrosis who have advanced lung disease.
Major finding: Chronic cough, hypoventilation, musculoskeletal pain, gastrointestinal reflux, abdominal discomfort, depression, and the need for frequent defecation all can disrupt sleep for patients with CF.
Data source: A review of 82 articles on cystic fibrosis and sleep.
Disclosures: Dr. Katz reported no funding sources and had no disclosures.
Secondary MERS coronavirus seen in 4% of contacts, with most asymptomatic
The rate of secondary transmission to household members of patients with Middle East respiratory syndrome coronavirus (MERS-CoV) is 4%, a study has shown.
The researchers studied 26 symptomatic index patients in Saudi Arabia along with 280 of their household contacts, and found only 12 with laboratory evidence of infection. The report was published online Aug. 27 in the New England Journal of Medicine.
Dr. Christian Drosten of the University of Bonn (Germany) Medical Center and colleagues found the 12 contacts to have laboratory evidence of MERS-CoV infection a median of 17.5 days from onset of symptoms in the index patients they lived with. Two of the infected household contacts reported having had contact with camels. All 280 contacts were screened via RT-PCR assays on RNA from throat swabs along with serologic testing that included enzyme-linked immunosorbent assays (ELISAs), and immunofluorescence assays.
Of the 12 with evidence of infection, 7 carried MERS-CoV in their upper respiratory tract, generally in low levels. Nonetheless, "positive RT-PCR findings in persons with subclinical infection who are tested soon after exposure should be complemented by serologic analysis," the investigators wrote.
The median age of the household contacts in the study was 29, and of the 12 infected, only one developed symptoms, which were mild. "Persons in the first few decades of life without coexisting illnesses may be able to carry low levels of MERS-CoV RNA without obvious symptoms," the researchers wrote (N. Engl. J. Med. 2014 Aug. 27 [doi:10.1056/NEJMoa1405858]).
The investigators cited as a weakness of their study the fact that only 44 of the 280 contacts originally tested provided follow-up samples. This was because the first round of testing was obligatory in Saudi Arabia, and the second, voluntary. The lack of follow-up testing meant that "we cannot determine whether we may have missed contacts who had delayed seroconversion," they wrote.
Dr. Drosten’s research was funded by the European Commission and German government grants. One coauthor reported receiving payment from the Institute for Experimental Immunology affiliated with EuroIMMUN, a maker of reagents for diagnostics, and another reported support from EuroIMMUN as well as a pending patent related to human betacoronavirus lineage.
The rate of secondary transmission to household members of patients with Middle East respiratory syndrome coronavirus (MERS-CoV) is 4%, a study has shown.
The researchers studied 26 symptomatic index patients in Saudi Arabia along with 280 of their household contacts, and found only 12 with laboratory evidence of infection. The report was published online Aug. 27 in the New England Journal of Medicine.
Dr. Christian Drosten of the University of Bonn (Germany) Medical Center and colleagues found the 12 contacts to have laboratory evidence of MERS-CoV infection a median of 17.5 days from onset of symptoms in the index patients they lived with. Two of the infected household contacts reported having had contact with camels. All 280 contacts were screened via RT-PCR assays on RNA from throat swabs along with serologic testing that included enzyme-linked immunosorbent assays (ELISAs), and immunofluorescence assays.
Of the 12 with evidence of infection, 7 carried MERS-CoV in their upper respiratory tract, generally in low levels. Nonetheless, "positive RT-PCR findings in persons with subclinical infection who are tested soon after exposure should be complemented by serologic analysis," the investigators wrote.
The median age of the household contacts in the study was 29, and of the 12 infected, only one developed symptoms, which were mild. "Persons in the first few decades of life without coexisting illnesses may be able to carry low levels of MERS-CoV RNA without obvious symptoms," the researchers wrote (N. Engl. J. Med. 2014 Aug. 27 [doi:10.1056/NEJMoa1405858]).
The investigators cited as a weakness of their study the fact that only 44 of the 280 contacts originally tested provided follow-up samples. This was because the first round of testing was obligatory in Saudi Arabia, and the second, voluntary. The lack of follow-up testing meant that "we cannot determine whether we may have missed contacts who had delayed seroconversion," they wrote.
Dr. Drosten’s research was funded by the European Commission and German government grants. One coauthor reported receiving payment from the Institute for Experimental Immunology affiliated with EuroIMMUN, a maker of reagents for diagnostics, and another reported support from EuroIMMUN as well as a pending patent related to human betacoronavirus lineage.
The rate of secondary transmission to household members of patients with Middle East respiratory syndrome coronavirus (MERS-CoV) is 4%, a study has shown.
The researchers studied 26 symptomatic index patients in Saudi Arabia along with 280 of their household contacts, and found only 12 with laboratory evidence of infection. The report was published online Aug. 27 in the New England Journal of Medicine.
Dr. Christian Drosten of the University of Bonn (Germany) Medical Center and colleagues found the 12 contacts to have laboratory evidence of MERS-CoV infection a median of 17.5 days from onset of symptoms in the index patients they lived with. Two of the infected household contacts reported having had contact with camels. All 280 contacts were screened via RT-PCR assays on RNA from throat swabs along with serologic testing that included enzyme-linked immunosorbent assays (ELISAs), and immunofluorescence assays.
Of the 12 with evidence of infection, 7 carried MERS-CoV in their upper respiratory tract, generally in low levels. Nonetheless, "positive RT-PCR findings in persons with subclinical infection who are tested soon after exposure should be complemented by serologic analysis," the investigators wrote.
The median age of the household contacts in the study was 29, and of the 12 infected, only one developed symptoms, which were mild. "Persons in the first few decades of life without coexisting illnesses may be able to carry low levels of MERS-CoV RNA without obvious symptoms," the researchers wrote (N. Engl. J. Med. 2014 Aug. 27 [doi:10.1056/NEJMoa1405858]).
The investigators cited as a weakness of their study the fact that only 44 of the 280 contacts originally tested provided follow-up samples. This was because the first round of testing was obligatory in Saudi Arabia, and the second, voluntary. The lack of follow-up testing meant that "we cannot determine whether we may have missed contacts who had delayed seroconversion," they wrote.
Dr. Drosten’s research was funded by the European Commission and German government grants. One coauthor reported receiving payment from the Institute for Experimental Immunology affiliated with EuroIMMUN, a maker of reagents for diagnostics, and another reported support from EuroIMMUN as well as a pending patent related to human betacoronavirus lineage.
FROM THE NEW ENGLAND JOURNAL OF MEDICINE
CPAP, adenotonsillectomy benefits similar for OSA in Down syndrome
Adenotonsillectomy and CPAP achieve similar improvements in apnea-hypopnea index among children with obstructive sleep apnea and Down syndrome or mucopolysaccharidoses, although the benefits from continuous positive airway pressure are more immediate, researchers said.
Dr. Shyam Sudhakar Sudarsan and colleagues from the Madras ENT Research Foundation in Chennai, India, performed a prospective, randomized comparative study in 124 syndromic children aged 6-12 years.
They found that children in both arms of the study achieved a mean apnea-hypopnea index of just over 1 at 12 months after randomization, with no statistically significant difference between CPAP and adenotonsillectomy.
"A striking observation from values of CPAP group was that, the child, if tolerant of a nasal mask/CPAP, showed statistically significant improvement by 6 months itself and this was maintained over a period of time," the authors wrote (Int. J. Pediatr. Otorhinolaryngol. 2014;78:1526-33).
"But in T&A [adenotonsillectomy] group, the improvement was gradual and statistically significant over a period of time," the authors said, suggesting that adenotonsillectomy in this group of high-risk children should be viewed as "beneficial rather than uniformly curative" for obstructive sleep apnea.
There were no conflicts of interest declared.
Adenotonsillectomy and CPAP achieve similar improvements in apnea-hypopnea index among children with obstructive sleep apnea and Down syndrome or mucopolysaccharidoses, although the benefits from continuous positive airway pressure are more immediate, researchers said.
Dr. Shyam Sudhakar Sudarsan and colleagues from the Madras ENT Research Foundation in Chennai, India, performed a prospective, randomized comparative study in 124 syndromic children aged 6-12 years.
They found that children in both arms of the study achieved a mean apnea-hypopnea index of just over 1 at 12 months after randomization, with no statistically significant difference between CPAP and adenotonsillectomy.
"A striking observation from values of CPAP group was that, the child, if tolerant of a nasal mask/CPAP, showed statistically significant improvement by 6 months itself and this was maintained over a period of time," the authors wrote (Int. J. Pediatr. Otorhinolaryngol. 2014;78:1526-33).
"But in T&A [adenotonsillectomy] group, the improvement was gradual and statistically significant over a period of time," the authors said, suggesting that adenotonsillectomy in this group of high-risk children should be viewed as "beneficial rather than uniformly curative" for obstructive sleep apnea.
There were no conflicts of interest declared.
Adenotonsillectomy and CPAP achieve similar improvements in apnea-hypopnea index among children with obstructive sleep apnea and Down syndrome or mucopolysaccharidoses, although the benefits from continuous positive airway pressure are more immediate, researchers said.
Dr. Shyam Sudhakar Sudarsan and colleagues from the Madras ENT Research Foundation in Chennai, India, performed a prospective, randomized comparative study in 124 syndromic children aged 6-12 years.
They found that children in both arms of the study achieved a mean apnea-hypopnea index of just over 1 at 12 months after randomization, with no statistically significant difference between CPAP and adenotonsillectomy.
"A striking observation from values of CPAP group was that, the child, if tolerant of a nasal mask/CPAP, showed statistically significant improvement by 6 months itself and this was maintained over a period of time," the authors wrote (Int. J. Pediatr. Otorhinolaryngol. 2014;78:1526-33).
"But in T&A [adenotonsillectomy] group, the improvement was gradual and statistically significant over a period of time," the authors said, suggesting that adenotonsillectomy in this group of high-risk children should be viewed as "beneficial rather than uniformly curative" for obstructive sleep apnea.
There were no conflicts of interest declared.
FROM INTERNATIONAL JOURNAL OF PEDIATRIC OTORHINOLARYNGOLOGY
Key clinical point: Adenotonsillectomy can be suggested as a first-line treatment in syndromic children.
Major finding: Adenotonsillectomy and CPAP achieve similar improvements in apnea-hypopnea index among children with OSA and Down syndrome, although the improvements following adenotonsillectomy occur more gradually.
Data source: Prospective, randomized comparative study in 124 children with Down syndrome or mucopolysaccharidoses.
Disclosures: No conflicts of interest were declared.
Assess patient’s body clock before treating circadian rhythm sleep disorders
Clinicians should measure dim light melatonin onset before treating patients with circadian rhythm sleep disorders, according to Dr. Henry Keijzer of the University Maastricht (the Netherlands) and his associates.
The test enables clinicians to assess patients’ individual circadian timing, the reviewers said. At least 17 of the 90 scientific studies they reviewed support the use of the test in this context, and it can be performed by collecting saliva at home if in-clinic testing is not feasible, they added.
Although patients are often told to take exogenous melatonin 1-2 hours before bedtime, this generalized approach can lead to adverse phase-shifting effects, the reviewers said (Sleep Med. Rev. 2014;18:333-9).
"Therefore, we believe that the use of melatonin in patients with chronic sleep problems should occur only after their baseline circadian timing information is known, and should be supervised by a health care professional who is familiar with melatonin and its phase-dependent effects," they wrote.
The authors expressed concern that "melatonin is increasingly recommended for patients with insomnia or used by such patients as an over-the-counter self-care medicine."
The investigators reported no funding sources or conflicts of interest.
Clinicians should measure dim light melatonin onset before treating patients with circadian rhythm sleep disorders, according to Dr. Henry Keijzer of the University Maastricht (the Netherlands) and his associates.
The test enables clinicians to assess patients’ individual circadian timing, the reviewers said. At least 17 of the 90 scientific studies they reviewed support the use of the test in this context, and it can be performed by collecting saliva at home if in-clinic testing is not feasible, they added.
Although patients are often told to take exogenous melatonin 1-2 hours before bedtime, this generalized approach can lead to adverse phase-shifting effects, the reviewers said (Sleep Med. Rev. 2014;18:333-9).
"Therefore, we believe that the use of melatonin in patients with chronic sleep problems should occur only after their baseline circadian timing information is known, and should be supervised by a health care professional who is familiar with melatonin and its phase-dependent effects," they wrote.
The authors expressed concern that "melatonin is increasingly recommended for patients with insomnia or used by such patients as an over-the-counter self-care medicine."
The investigators reported no funding sources or conflicts of interest.
Clinicians should measure dim light melatonin onset before treating patients with circadian rhythm sleep disorders, according to Dr. Henry Keijzer of the University Maastricht (the Netherlands) and his associates.
The test enables clinicians to assess patients’ individual circadian timing, the reviewers said. At least 17 of the 90 scientific studies they reviewed support the use of the test in this context, and it can be performed by collecting saliva at home if in-clinic testing is not feasible, they added.
Although patients are often told to take exogenous melatonin 1-2 hours before bedtime, this generalized approach can lead to adverse phase-shifting effects, the reviewers said (Sleep Med. Rev. 2014;18:333-9).
"Therefore, we believe that the use of melatonin in patients with chronic sleep problems should occur only after their baseline circadian timing information is known, and should be supervised by a health care professional who is familiar with melatonin and its phase-dependent effects," they wrote.
The authors expressed concern that "melatonin is increasingly recommended for patients with insomnia or used by such patients as an over-the-counter self-care medicine."
The investigators reported no funding sources or conflicts of interest.
FROM SLEEP MEDICINE REVIEWS
Key clinical point: Clinicians should measure dim light melatonin onset before treating patients with circadian rhythm sleep disorders, and should prescribe melatonin or light therapy based on patients’ individualized DLMO result.
Major finding: Measuring DLMO improves the diagnosis and treatment of circadian rhythm sleep disorders.
Data source: A review of 90 articles on circadian rhythms, sleep disorders, DLMO, and the use of lighting and exogenous melatonin to improve sleep measures.
Disclosures: The authors reported no funding sources or conflicts of interest.
AHA wants e-cigarettes regulated but notes they help some smokers quit
The American Heart Association has released its recommendations regarding the sale and usage of e-cigarettes, cautioning that the devices should be regulated to avoid enticing children to smoke while also saying that e-cigarettes could be used as a way to help some current smokers quit.
The guidelines, published online Aug. 24 in Circulation, state that clinicians "should not discourage" their patients from resorting to e-cigarettes if other approved forms of smoking cessation, such as the nicotine patch, have been exhausted. The AHA warned, however, that further studies are needed to fully understand the effects of e-cigarette usage, stressing that e-cigarettes have not been approved by the Food and Drug Administration as an acceptable smoking cessation device and could contain low amounts of toxic chemicals that would prove more harmful than helpful to patients in the long run.
"E-cigarettes have caused a major shift in the tobacco-control landscape," Aruni Bhatnagar, Ph.D., lead author of the guidelines and chair of cardiovascular medicine at the University of Louisville, Ky., said in a statement. "It’s critical that we rigorously examine the long-term impact of this new technology on public health, cardiovascular disease, and stroke, and pay careful attention to the effect of e-cigarettes on adolescents."
For now, the AHA says that clinicians should tell their patients who use e-cigarettes to set a firm quit date, warning that any device that delivers nicotine into the body is harmful and likely lethal.
"Nicotine is a dangerous and highly addictive chemical no matter what form it takes – conventional cigarettes or some other tobacco product," Dr. Elliott Antman, AHA president, said in the statement. "Every life that has been lost to tobacco addiction" could have been saved, he noted.
To that end, the AHA also called for strong regulations regarding the potential marketability of e-cigarettes to youngsters. The group recommended a federal ban on the sale of e-cigarettes to minors, warning that the devices could become a gateway to actual cigarettes for young people who see e-cigarettes as "high-tech, accessible, and convenient," according to a JAMA Pediatrics study of 40,000 middle and high school students. The AHA also recommended that all existing rules and regulations in place for tobacco-related products should apply to e-cigarettes.
"We must protect future generations from any potential smokescreens in the tobacco product landscape that will cause us to lose precious ground in the fight to make our nation 100% tobacco-free," Dr. Antman said.
According to the AHA, 20 million Americans have lost their lives to tobacco over the last 50 years.
The American Heart Association has released its recommendations regarding the sale and usage of e-cigarettes, cautioning that the devices should be regulated to avoid enticing children to smoke while also saying that e-cigarettes could be used as a way to help some current smokers quit.
The guidelines, published online Aug. 24 in Circulation, state that clinicians "should not discourage" their patients from resorting to e-cigarettes if other approved forms of smoking cessation, such as the nicotine patch, have been exhausted. The AHA warned, however, that further studies are needed to fully understand the effects of e-cigarette usage, stressing that e-cigarettes have not been approved by the Food and Drug Administration as an acceptable smoking cessation device and could contain low amounts of toxic chemicals that would prove more harmful than helpful to patients in the long run.
"E-cigarettes have caused a major shift in the tobacco-control landscape," Aruni Bhatnagar, Ph.D., lead author of the guidelines and chair of cardiovascular medicine at the University of Louisville, Ky., said in a statement. "It’s critical that we rigorously examine the long-term impact of this new technology on public health, cardiovascular disease, and stroke, and pay careful attention to the effect of e-cigarettes on adolescents."
For now, the AHA says that clinicians should tell their patients who use e-cigarettes to set a firm quit date, warning that any device that delivers nicotine into the body is harmful and likely lethal.
"Nicotine is a dangerous and highly addictive chemical no matter what form it takes – conventional cigarettes or some other tobacco product," Dr. Elliott Antman, AHA president, said in the statement. "Every life that has been lost to tobacco addiction" could have been saved, he noted.
To that end, the AHA also called for strong regulations regarding the potential marketability of e-cigarettes to youngsters. The group recommended a federal ban on the sale of e-cigarettes to minors, warning that the devices could become a gateway to actual cigarettes for young people who see e-cigarettes as "high-tech, accessible, and convenient," according to a JAMA Pediatrics study of 40,000 middle and high school students. The AHA also recommended that all existing rules and regulations in place for tobacco-related products should apply to e-cigarettes.
"We must protect future generations from any potential smokescreens in the tobacco product landscape that will cause us to lose precious ground in the fight to make our nation 100% tobacco-free," Dr. Antman said.
According to the AHA, 20 million Americans have lost their lives to tobacco over the last 50 years.
The American Heart Association has released its recommendations regarding the sale and usage of e-cigarettes, cautioning that the devices should be regulated to avoid enticing children to smoke while also saying that e-cigarettes could be used as a way to help some current smokers quit.
The guidelines, published online Aug. 24 in Circulation, state that clinicians "should not discourage" their patients from resorting to e-cigarettes if other approved forms of smoking cessation, such as the nicotine patch, have been exhausted. The AHA warned, however, that further studies are needed to fully understand the effects of e-cigarette usage, stressing that e-cigarettes have not been approved by the Food and Drug Administration as an acceptable smoking cessation device and could contain low amounts of toxic chemicals that would prove more harmful than helpful to patients in the long run.
"E-cigarettes have caused a major shift in the tobacco-control landscape," Aruni Bhatnagar, Ph.D., lead author of the guidelines and chair of cardiovascular medicine at the University of Louisville, Ky., said in a statement. "It’s critical that we rigorously examine the long-term impact of this new technology on public health, cardiovascular disease, and stroke, and pay careful attention to the effect of e-cigarettes on adolescents."
For now, the AHA says that clinicians should tell their patients who use e-cigarettes to set a firm quit date, warning that any device that delivers nicotine into the body is harmful and likely lethal.
"Nicotine is a dangerous and highly addictive chemical no matter what form it takes – conventional cigarettes or some other tobacco product," Dr. Elliott Antman, AHA president, said in the statement. "Every life that has been lost to tobacco addiction" could have been saved, he noted.
To that end, the AHA also called for strong regulations regarding the potential marketability of e-cigarettes to youngsters. The group recommended a federal ban on the sale of e-cigarettes to minors, warning that the devices could become a gateway to actual cigarettes for young people who see e-cigarettes as "high-tech, accessible, and convenient," according to a JAMA Pediatrics study of 40,000 middle and high school students. The AHA also recommended that all existing rules and regulations in place for tobacco-related products should apply to e-cigarettes.
"We must protect future generations from any potential smokescreens in the tobacco product landscape that will cause us to lose precious ground in the fight to make our nation 100% tobacco-free," Dr. Antman said.
According to the AHA, 20 million Americans have lost their lives to tobacco over the last 50 years.
FROM CIRCULATION
Apixaban approved for treating DVT, pulmonary embolism and reducing risk of recurrence
The oral factor Xa inhibitor apixaban is now approved for the treatment of deep vein thrombosis and pulmonary embolism, and for reducing the risk of recurrent DVT and PE following initial treatment, the manufacturers have announced.
This approval is based on the results of the AMPLIFY and AMPLIFY-EXT studies, according to the statement issued by Bristol-Myers Squibb and Pfizer Aug. 21.
Apixaban, initially approved in 2012 and marketed as Eliquis, is already approved for reducing the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation and for the prophylaxis of DVT, "which may lead to PE," after hip or knee surgery. The recommended dose for the treatment of DVT and PE is 10 mg twice a day for 7 days, followed by 5 mg twice a day. The recommended dose for reducing the risk for recurrent DVT and PE, after initial therapy, is 2.5 mg twice a day.
AMPLIFY was a noninferiority study of about 5,200 patients with symptomatic DVT or PE. It compared apixaban (10 mg twice a day for 1 week, followed by 5 mg twice a day for 6 months) with standard care using enoxaparin (1 mg/kg administered subcutaneously twice a day for at least 5 days [until the international normalized ratio was at least 2], followed by warfarin for at least 5 days [target INR range of 2.0-3.0] for 6 months). The primary efficacy endpoint, a composite of recurrent symptomatic VTE or VTE-related death over 6 months, was comparable in the two groups: 2.3% among those on apixaban and 2.7% among those on enoxaparin/warfarin, according to the prescribing information.
The primary safety endpoint, major bleeding, was 0.6% among those on apixaban vs. 1.8% among those on enoxaparin/warfarin, a statistically significant difference. Rates of clinically relevant nonmajor bleeding, a secondary safety endpoint, was 3.9% among those on apixaban vs. 8% among those on enoxaparin/warfarin.
In Amplify-EXT, almost 2,500 patients who had received anticoagulant therapy for DVT and/or PE for 6-12 months and had not had a recurrent event were randomized to treatment with apixaban 2.5 or 5 mg twice a day, or placebo, followed for a mean of almost 1 year. The rate of recurrent VTE or all-cause death was 3.8% among those on 2.5 mg twice daily and 4.2% among those on 5 mg twice daily, vs. 11.6% among those on placebo; the effects of both doses were significantly more effective in reducing risk than was placebo.
In this study, the rate of bleeding-related adverse reactions was 13.3% among those on apixaban vs. 8.7% among those on placebo. The rate of major bleeding was 0.2% among those on the 2.5 mg twice-daily dose and 0.1% among those on the 5 mg twice-daily dose, vs. 0.5% among those on placebo.
The apixaban label includes a boxed warning about an increased risk of spinal/epidural hematoma in patients undergoing neuraxial anesthesia or spinal puncture while on the drug.
Serious adverse events associated with apixaban should be reported to the FDA’s MedWatch program.
The oral factor Xa inhibitor apixaban is now approved for the treatment of deep vein thrombosis and pulmonary embolism, and for reducing the risk of recurrent DVT and PE following initial treatment, the manufacturers have announced.
This approval is based on the results of the AMPLIFY and AMPLIFY-EXT studies, according to the statement issued by Bristol-Myers Squibb and Pfizer Aug. 21.
Apixaban, initially approved in 2012 and marketed as Eliquis, is already approved for reducing the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation and for the prophylaxis of DVT, "which may lead to PE," after hip or knee surgery. The recommended dose for the treatment of DVT and PE is 10 mg twice a day for 7 days, followed by 5 mg twice a day. The recommended dose for reducing the risk for recurrent DVT and PE, after initial therapy, is 2.5 mg twice a day.
AMPLIFY was a noninferiority study of about 5,200 patients with symptomatic DVT or PE. It compared apixaban (10 mg twice a day for 1 week, followed by 5 mg twice a day for 6 months) with standard care using enoxaparin (1 mg/kg administered subcutaneously twice a day for at least 5 days [until the international normalized ratio was at least 2], followed by warfarin for at least 5 days [target INR range of 2.0-3.0] for 6 months). The primary efficacy endpoint, a composite of recurrent symptomatic VTE or VTE-related death over 6 months, was comparable in the two groups: 2.3% among those on apixaban and 2.7% among those on enoxaparin/warfarin, according to the prescribing information.
The primary safety endpoint, major bleeding, was 0.6% among those on apixaban vs. 1.8% among those on enoxaparin/warfarin, a statistically significant difference. Rates of clinically relevant nonmajor bleeding, a secondary safety endpoint, was 3.9% among those on apixaban vs. 8% among those on enoxaparin/warfarin.
In Amplify-EXT, almost 2,500 patients who had received anticoagulant therapy for DVT and/or PE for 6-12 months and had not had a recurrent event were randomized to treatment with apixaban 2.5 or 5 mg twice a day, or placebo, followed for a mean of almost 1 year. The rate of recurrent VTE or all-cause death was 3.8% among those on 2.5 mg twice daily and 4.2% among those on 5 mg twice daily, vs. 11.6% among those on placebo; the effects of both doses were significantly more effective in reducing risk than was placebo.
In this study, the rate of bleeding-related adverse reactions was 13.3% among those on apixaban vs. 8.7% among those on placebo. The rate of major bleeding was 0.2% among those on the 2.5 mg twice-daily dose and 0.1% among those on the 5 mg twice-daily dose, vs. 0.5% among those on placebo.
The apixaban label includes a boxed warning about an increased risk of spinal/epidural hematoma in patients undergoing neuraxial anesthesia or spinal puncture while on the drug.
Serious adverse events associated with apixaban should be reported to the FDA’s MedWatch program.
The oral factor Xa inhibitor apixaban is now approved for the treatment of deep vein thrombosis and pulmonary embolism, and for reducing the risk of recurrent DVT and PE following initial treatment, the manufacturers have announced.
This approval is based on the results of the AMPLIFY and AMPLIFY-EXT studies, according to the statement issued by Bristol-Myers Squibb and Pfizer Aug. 21.
Apixaban, initially approved in 2012 and marketed as Eliquis, is already approved for reducing the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation and for the prophylaxis of DVT, "which may lead to PE," after hip or knee surgery. The recommended dose for the treatment of DVT and PE is 10 mg twice a day for 7 days, followed by 5 mg twice a day. The recommended dose for reducing the risk for recurrent DVT and PE, after initial therapy, is 2.5 mg twice a day.
AMPLIFY was a noninferiority study of about 5,200 patients with symptomatic DVT or PE. It compared apixaban (10 mg twice a day for 1 week, followed by 5 mg twice a day for 6 months) with standard care using enoxaparin (1 mg/kg administered subcutaneously twice a day for at least 5 days [until the international normalized ratio was at least 2], followed by warfarin for at least 5 days [target INR range of 2.0-3.0] for 6 months). The primary efficacy endpoint, a composite of recurrent symptomatic VTE or VTE-related death over 6 months, was comparable in the two groups: 2.3% among those on apixaban and 2.7% among those on enoxaparin/warfarin, according to the prescribing information.
The primary safety endpoint, major bleeding, was 0.6% among those on apixaban vs. 1.8% among those on enoxaparin/warfarin, a statistically significant difference. Rates of clinically relevant nonmajor bleeding, a secondary safety endpoint, was 3.9% among those on apixaban vs. 8% among those on enoxaparin/warfarin.
In Amplify-EXT, almost 2,500 patients who had received anticoagulant therapy for DVT and/or PE for 6-12 months and had not had a recurrent event were randomized to treatment with apixaban 2.5 or 5 mg twice a day, or placebo, followed for a mean of almost 1 year. The rate of recurrent VTE or all-cause death was 3.8% among those on 2.5 mg twice daily and 4.2% among those on 5 mg twice daily, vs. 11.6% among those on placebo; the effects of both doses were significantly more effective in reducing risk than was placebo.
In this study, the rate of bleeding-related adverse reactions was 13.3% among those on apixaban vs. 8.7% among those on placebo. The rate of major bleeding was 0.2% among those on the 2.5 mg twice-daily dose and 0.1% among those on the 5 mg twice-daily dose, vs. 0.5% among those on placebo.
The apixaban label includes a boxed warning about an increased risk of spinal/epidural hematoma in patients undergoing neuraxial anesthesia or spinal puncture while on the drug.
Serious adverse events associated with apixaban should be reported to the FDA’s MedWatch program.
Crises inform updated critical care recommendations for disasters
Care of critically ill or injured patients during Hurricane Sandy, the 2010 earthquake in Haiti, and the H1N1 influenza pandemic in Mexico City played a role in shaping new recommendations from the American College of Chest Physicians.
The 14-part consensus statement from the College’s Task Force for Mass Critical Care updates and expands on the task force’s previous recommendations from 2008, which did not address pediatrics, trauma, subspecialty critical care, or critical care during disasters in the developing world, among other topics.
Although there is some overlap with the 2008 recommendations, "by and large the specific suggestions are all new or have been updated since the 2008 guidelines, based upon lessons learned from pandemics and disasters that have occurred in the interval," Dr. Jeffrey R. Dichter said in an interview.
If history is any guide, clinicians will want to incorporate the 2014 recommendations as a matter of routine instead of placing them on a shelf only to be retrieved in times of crisis, said Dr. Dichter, a member of the task force. He is medical director for intensive care at Unity Hospital, Fridley, Minn., and a past president of the Society of Hospital Medicine.
The task force heard from colleagues that the 2008 document helped them plan to scale up ICU capacity and manage ventilated patients during the 2009 Mexican influenza epidemic – plans that they did not need to implement but that gave them reassurance nonetheless.
Triage guidelines in the 2008 document helped Bellevue Hospital in New York plan for allocating scarce resources such as electricity from backup generators during 2012’s Hurricane Sandy, a hospital physician reported at the College’s 2013 annual meeting. "Simply knowing they had an approach in place to make those decisions if required allowed the staff to focus on dealing with the situation at hand effectively rather than being distracted by the uncertainty of what would happen if the generators failed," Dr. Dichter said.
The 2014 document’s attention to evacuation of critically ill and injured patients should be of particular interest. "We have seen the challenge related to evacuating ICUs, particularly during the New York hurricane," he said.
Another highlight is a focus on developing the capacity to increase health care personnel and supplies when needed (also known as "surge response") to meet disasters of various sizes – small, medium, or large. The previous recommendations covered only the largest, "crisis" care.
The document provides advice for everyone from individual doctors to hospital administrators, regional health systems, and national governments. "While it is important for all health care workers to have a broad understanding of the issues of emergency preparedness, readers can focus on the areas more relevant to them by consulting the tables in each document" that identify the people for whom those recommendations are most relevant, he said.
The recommendations are based on expert consensus because there is no research to support evidence-based guidelines, the task force stated. That should change by the time the recommendations are updated again in the future, Dr. Dichter said. Groups such as the International Forum for Acute Care Trialists and the International Severe Acute Respiratory and Emerging Infection Consortium are starting to conduct research during pandemics and disasters.
And, of course, more crises will occur, providing experience that will help improve planning for the next inevitable disaster.
Dr. Dichter reported having no financial disclosures.
On Twitter @sherryboschert
The consensus statement moves forward planning for critical care during pandemics and disasters in unprecedented ways, Dr. Christian Sandrock said in an editorial.
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The principles behind recommendations for conservation of personnel and supplies (particularly scarce medications and oxygen therapy), substitutions where necessary, and triage "are very relevant today where the resource limitations in West Africa are hindering both patient care and management outbreak," wrote Dr. Sandrock.
Emphases on technology and telemedicine, baseline education plus just-in-time training, and mental health support during disasters should help support health care workers on the front lines to deliver the best care possible, he added.
A goal of "a 20% increase in surge within the health-system resources to a 200% surge with regional and national resource support sets an international benchmark for health-system preparedness," Dr. Sandrock wrote.
Recommendations for coordinating patient flow and resources at a regional level have "not been described elsewhere," and the task force’s vision of incorporating critical care expertise in large, central coordination of patient flow, resources, and care "sets a high but necessary benchmark for public health and government officials," he wrote.
Perhaps the greatest contribution of the document is its attention to potential health care inequities during disasters and the need for ethical and equal care, Dr. Sandrock suggested. The consensus statement provides "a foundation of disaster response for the critically ill that provides justice to the most adversely affected patients," which is an unparalleled accomplishment, he wrote.
Dr. Sandrock is in the division of pulmonary and critical care medicine at the University of California, Davis.
The consensus statement moves forward planning for critical care during pandemics and disasters in unprecedented ways, Dr. Christian Sandrock said in an editorial.
|
|
The principles behind recommendations for conservation of personnel and supplies (particularly scarce medications and oxygen therapy), substitutions where necessary, and triage "are very relevant today where the resource limitations in West Africa are hindering both patient care and management outbreak," wrote Dr. Sandrock.
Emphases on technology and telemedicine, baseline education plus just-in-time training, and mental health support during disasters should help support health care workers on the front lines to deliver the best care possible, he added.
A goal of "a 20% increase in surge within the health-system resources to a 200% surge with regional and national resource support sets an international benchmark for health-system preparedness," Dr. Sandrock wrote.
Recommendations for coordinating patient flow and resources at a regional level have "not been described elsewhere," and the task force’s vision of incorporating critical care expertise in large, central coordination of patient flow, resources, and care "sets a high but necessary benchmark for public health and government officials," he wrote.
Perhaps the greatest contribution of the document is its attention to potential health care inequities during disasters and the need for ethical and equal care, Dr. Sandrock suggested. The consensus statement provides "a foundation of disaster response for the critically ill that provides justice to the most adversely affected patients," which is an unparalleled accomplishment, he wrote.
Dr. Sandrock is in the division of pulmonary and critical care medicine at the University of California, Davis.
The consensus statement moves forward planning for critical care during pandemics and disasters in unprecedented ways, Dr. Christian Sandrock said in an editorial.
|
|
The principles behind recommendations for conservation of personnel and supplies (particularly scarce medications and oxygen therapy), substitutions where necessary, and triage "are very relevant today where the resource limitations in West Africa are hindering both patient care and management outbreak," wrote Dr. Sandrock.
Emphases on technology and telemedicine, baseline education plus just-in-time training, and mental health support during disasters should help support health care workers on the front lines to deliver the best care possible, he added.
A goal of "a 20% increase in surge within the health-system resources to a 200% surge with regional and national resource support sets an international benchmark for health-system preparedness," Dr. Sandrock wrote.
Recommendations for coordinating patient flow and resources at a regional level have "not been described elsewhere," and the task force’s vision of incorporating critical care expertise in large, central coordination of patient flow, resources, and care "sets a high but necessary benchmark for public health and government officials," he wrote.
Perhaps the greatest contribution of the document is its attention to potential health care inequities during disasters and the need for ethical and equal care, Dr. Sandrock suggested. The consensus statement provides "a foundation of disaster response for the critically ill that provides justice to the most adversely affected patients," which is an unparalleled accomplishment, he wrote.
Dr. Sandrock is in the division of pulmonary and critical care medicine at the University of California, Davis.
Care of critically ill or injured patients during Hurricane Sandy, the 2010 earthquake in Haiti, and the H1N1 influenza pandemic in Mexico City played a role in shaping new recommendations from the American College of Chest Physicians.
The 14-part consensus statement from the College’s Task Force for Mass Critical Care updates and expands on the task force’s previous recommendations from 2008, which did not address pediatrics, trauma, subspecialty critical care, or critical care during disasters in the developing world, among other topics.
Although there is some overlap with the 2008 recommendations, "by and large the specific suggestions are all new or have been updated since the 2008 guidelines, based upon lessons learned from pandemics and disasters that have occurred in the interval," Dr. Jeffrey R. Dichter said in an interview.
If history is any guide, clinicians will want to incorporate the 2014 recommendations as a matter of routine instead of placing them on a shelf only to be retrieved in times of crisis, said Dr. Dichter, a member of the task force. He is medical director for intensive care at Unity Hospital, Fridley, Minn., and a past president of the Society of Hospital Medicine.
The task force heard from colleagues that the 2008 document helped them plan to scale up ICU capacity and manage ventilated patients during the 2009 Mexican influenza epidemic – plans that they did not need to implement but that gave them reassurance nonetheless.
Triage guidelines in the 2008 document helped Bellevue Hospital in New York plan for allocating scarce resources such as electricity from backup generators during 2012’s Hurricane Sandy, a hospital physician reported at the College’s 2013 annual meeting. "Simply knowing they had an approach in place to make those decisions if required allowed the staff to focus on dealing with the situation at hand effectively rather than being distracted by the uncertainty of what would happen if the generators failed," Dr. Dichter said.
The 2014 document’s attention to evacuation of critically ill and injured patients should be of particular interest. "We have seen the challenge related to evacuating ICUs, particularly during the New York hurricane," he said.
Another highlight is a focus on developing the capacity to increase health care personnel and supplies when needed (also known as "surge response") to meet disasters of various sizes – small, medium, or large. The previous recommendations covered only the largest, "crisis" care.
The document provides advice for everyone from individual doctors to hospital administrators, regional health systems, and national governments. "While it is important for all health care workers to have a broad understanding of the issues of emergency preparedness, readers can focus on the areas more relevant to them by consulting the tables in each document" that identify the people for whom those recommendations are most relevant, he said.
The recommendations are based on expert consensus because there is no research to support evidence-based guidelines, the task force stated. That should change by the time the recommendations are updated again in the future, Dr. Dichter said. Groups such as the International Forum for Acute Care Trialists and the International Severe Acute Respiratory and Emerging Infection Consortium are starting to conduct research during pandemics and disasters.
And, of course, more crises will occur, providing experience that will help improve planning for the next inevitable disaster.
Dr. Dichter reported having no financial disclosures.
On Twitter @sherryboschert
Care of critically ill or injured patients during Hurricane Sandy, the 2010 earthquake in Haiti, and the H1N1 influenza pandemic in Mexico City played a role in shaping new recommendations from the American College of Chest Physicians.
The 14-part consensus statement from the College’s Task Force for Mass Critical Care updates and expands on the task force’s previous recommendations from 2008, which did not address pediatrics, trauma, subspecialty critical care, or critical care during disasters in the developing world, among other topics.
Although there is some overlap with the 2008 recommendations, "by and large the specific suggestions are all new or have been updated since the 2008 guidelines, based upon lessons learned from pandemics and disasters that have occurred in the interval," Dr. Jeffrey R. Dichter said in an interview.
If history is any guide, clinicians will want to incorporate the 2014 recommendations as a matter of routine instead of placing them on a shelf only to be retrieved in times of crisis, said Dr. Dichter, a member of the task force. He is medical director for intensive care at Unity Hospital, Fridley, Minn., and a past president of the Society of Hospital Medicine.
The task force heard from colleagues that the 2008 document helped them plan to scale up ICU capacity and manage ventilated patients during the 2009 Mexican influenza epidemic – plans that they did not need to implement but that gave them reassurance nonetheless.
Triage guidelines in the 2008 document helped Bellevue Hospital in New York plan for allocating scarce resources such as electricity from backup generators during 2012’s Hurricane Sandy, a hospital physician reported at the College’s 2013 annual meeting. "Simply knowing they had an approach in place to make those decisions if required allowed the staff to focus on dealing with the situation at hand effectively rather than being distracted by the uncertainty of what would happen if the generators failed," Dr. Dichter said.
The 2014 document’s attention to evacuation of critically ill and injured patients should be of particular interest. "We have seen the challenge related to evacuating ICUs, particularly during the New York hurricane," he said.
Another highlight is a focus on developing the capacity to increase health care personnel and supplies when needed (also known as "surge response") to meet disasters of various sizes – small, medium, or large. The previous recommendations covered only the largest, "crisis" care.
The document provides advice for everyone from individual doctors to hospital administrators, regional health systems, and national governments. "While it is important for all health care workers to have a broad understanding of the issues of emergency preparedness, readers can focus on the areas more relevant to them by consulting the tables in each document" that identify the people for whom those recommendations are most relevant, he said.
The recommendations are based on expert consensus because there is no research to support evidence-based guidelines, the task force stated. That should change by the time the recommendations are updated again in the future, Dr. Dichter said. Groups such as the International Forum for Acute Care Trialists and the International Severe Acute Respiratory and Emerging Infection Consortium are starting to conduct research during pandemics and disasters.
And, of course, more crises will occur, providing experience that will help improve planning for the next inevitable disaster.
Dr. Dichter reported having no financial disclosures.
On Twitter @sherryboschert
FROM CHEST
More patients with multidrug-resistant TB respond quickly to bedaquiline
Adding bedaquiline to a five-drug background regimen for multidrug-resistant pulmonary tuberculosis significantly accelerated the time at which sputum cultures showed clearance of Mycobacterium tuberculosis in a phase IIb clinical trial, which was published online Aug. 21 in the New England Journal of Medicine.
The addition of 6 months of oral bedaquiline – a diarylquinoline that inhibits mycobacterial ATP synthase and depletes cellular energy stores – also significantly increased the number of patients who achieved such culture conversion (a surrogate for clinical cure), compared with adding placebo, in this industry-sponsored study. Bedaquiline "is the first antituberculosis drug with a new mechanism of action to be approved for use in multidrug-resistant TB in 40 years," said Dr. Andreas H. Diacon of the National Research Foundation Centre of Excellence for Biomedical Tuberculosis Research at Stellenbosch University in Cape Town, South Africa, and his associates.
Bedaquiline was approved in 2012 under the Food and Drug Administration’s accelerated-approval regulations for drugs that provide meaningful therapeutic benefit over existing therapies for serious or life-threatening conditions. The drug is approved only for patients for whom an effective treatment regimen cannot be constructed without it, a population in whom the benefits outweigh the risks.
In the trial, 160 adults with multidrug-resistant pulmonary TB were randomly assigned to receive either oral bedaquiline (79 patients) or matching placebo tablets (81 patients) for 24 weeks at medical centers in Brazil, India, Latvia, Peru, the Philippines, Russia, South Africa, and Thailand, in addition to the five-drug background regimen of antituberculosis drugs preferred at each location. They continued the background therapy for a further 96 weeks and had a final follow-up exam at the conclusion of that treatment. Sixty participants (38%) left the study prematurely, usually because of withdrawal of consent, adverse effects, nonadherence, loss to follow-up, and pregnancy. The number of and reasons for these discontinuations were similar between the two study groups.
The primary endpoint of the study was the median time to sputum conversion. This was significantly faster in the intervention group (83 days) than in the control group (125 days) in both the modified intention-to-treat analysis and the full intention-to-treat analysis. Moreover, more patients in the intervention group (79%) than in the placebo group (58%) showed culture conversion at 24 weeks, as well as at 120 weeks (62% vs 44%). And according to the World Health Organization definition of "cure," nearly twice as many patients in the bedaquiline group (58%) were cured as in the placebo group (32%) at 120 weeks, Dr. Diacon and his associates said (N. Engl. J. Med. 2014;371:723-32 [doi:10.1056/NEJMoa1313865]).
Fewer patients taking bedaquiline (2) than taking placebo (16) developed new resistance to at least one antituberculosis agent during the study. Rates, types, and severity of adverse events were comparable between the two study groups. However, mortality was significantly higher with bedaquiline (10 patients, 13% mortality) than with placebo (2 patients, 2% mortality), for reasons that are not yet understood but do not appear to be related to the study drug.
"A planned phase III study will enroll a larger number of patients, including HIV-positive patients receiving antiretroviral therapy," the investigators noted.
This study was funded by Janssen Pharmaceuticals. Dr. Diacon reported no financial conflicts of interest. His associates reported ties to Janssen; to its parent company, Johnson & Johnson; and to Quintiles.
The Food and Drug Administration approved the limited use of bedaquiline despite the unexpected finding of significantly increased mortality in the intervention group (13%) than in the control group (2%), because the length of time between the last receipt of bedaquiline and death – 329 days – makes it difficult to discern a mechanism by which the drug could be directly related to the deaths, even if bedaquiline’s long half-life is taken into consideration.
This imbalance in mortality is an important concern. However, 1 of the 10 deaths that occurred in patients who received bedaquiline was because of a motor vehicle accident, 5 were from disease progression (as were both deaths in the placebo group), and the remaining 4 had no apparent commonality. A confirmatory trial, a condition of the accelerated approval of the drug, should shed more light on this discrepancy in mortality.
Dr. Edward Cox and Dr. Katherine Laessig are in the Office of Antimicrobial Products and the Office of New Drugs, the Center for Drug Evaluation and Research, at the FDA. They made their remarks in a perspective piece accompanying Dr. Diacon’s report (N. Engl. J. Med. 2014;371:689-91 [doi:10.1056/NEJMp1314385]). They reported no financial conflicts of interest.
The Food and Drug Administration approved the limited use of bedaquiline despite the unexpected finding of significantly increased mortality in the intervention group (13%) than in the control group (2%), because the length of time between the last receipt of bedaquiline and death – 329 days – makes it difficult to discern a mechanism by which the drug could be directly related to the deaths, even if bedaquiline’s long half-life is taken into consideration.
This imbalance in mortality is an important concern. However, 1 of the 10 deaths that occurred in patients who received bedaquiline was because of a motor vehicle accident, 5 were from disease progression (as were both deaths in the placebo group), and the remaining 4 had no apparent commonality. A confirmatory trial, a condition of the accelerated approval of the drug, should shed more light on this discrepancy in mortality.
Dr. Edward Cox and Dr. Katherine Laessig are in the Office of Antimicrobial Products and the Office of New Drugs, the Center for Drug Evaluation and Research, at the FDA. They made their remarks in a perspective piece accompanying Dr. Diacon’s report (N. Engl. J. Med. 2014;371:689-91 [doi:10.1056/NEJMp1314385]). They reported no financial conflicts of interest.
The Food and Drug Administration approved the limited use of bedaquiline despite the unexpected finding of significantly increased mortality in the intervention group (13%) than in the control group (2%), because the length of time between the last receipt of bedaquiline and death – 329 days – makes it difficult to discern a mechanism by which the drug could be directly related to the deaths, even if bedaquiline’s long half-life is taken into consideration.
This imbalance in mortality is an important concern. However, 1 of the 10 deaths that occurred in patients who received bedaquiline was because of a motor vehicle accident, 5 were from disease progression (as were both deaths in the placebo group), and the remaining 4 had no apparent commonality. A confirmatory trial, a condition of the accelerated approval of the drug, should shed more light on this discrepancy in mortality.
Dr. Edward Cox and Dr. Katherine Laessig are in the Office of Antimicrobial Products and the Office of New Drugs, the Center for Drug Evaluation and Research, at the FDA. They made their remarks in a perspective piece accompanying Dr. Diacon’s report (N. Engl. J. Med. 2014;371:689-91 [doi:10.1056/NEJMp1314385]). They reported no financial conflicts of interest.
Adding bedaquiline to a five-drug background regimen for multidrug-resistant pulmonary tuberculosis significantly accelerated the time at which sputum cultures showed clearance of Mycobacterium tuberculosis in a phase IIb clinical trial, which was published online Aug. 21 in the New England Journal of Medicine.
The addition of 6 months of oral bedaquiline – a diarylquinoline that inhibits mycobacterial ATP synthase and depletes cellular energy stores – also significantly increased the number of patients who achieved such culture conversion (a surrogate for clinical cure), compared with adding placebo, in this industry-sponsored study. Bedaquiline "is the first antituberculosis drug with a new mechanism of action to be approved for use in multidrug-resistant TB in 40 years," said Dr. Andreas H. Diacon of the National Research Foundation Centre of Excellence for Biomedical Tuberculosis Research at Stellenbosch University in Cape Town, South Africa, and his associates.
Bedaquiline was approved in 2012 under the Food and Drug Administration’s accelerated-approval regulations for drugs that provide meaningful therapeutic benefit over existing therapies for serious or life-threatening conditions. The drug is approved only for patients for whom an effective treatment regimen cannot be constructed without it, a population in whom the benefits outweigh the risks.
In the trial, 160 adults with multidrug-resistant pulmonary TB were randomly assigned to receive either oral bedaquiline (79 patients) or matching placebo tablets (81 patients) for 24 weeks at medical centers in Brazil, India, Latvia, Peru, the Philippines, Russia, South Africa, and Thailand, in addition to the five-drug background regimen of antituberculosis drugs preferred at each location. They continued the background therapy for a further 96 weeks and had a final follow-up exam at the conclusion of that treatment. Sixty participants (38%) left the study prematurely, usually because of withdrawal of consent, adverse effects, nonadherence, loss to follow-up, and pregnancy. The number of and reasons for these discontinuations were similar between the two study groups.
The primary endpoint of the study was the median time to sputum conversion. This was significantly faster in the intervention group (83 days) than in the control group (125 days) in both the modified intention-to-treat analysis and the full intention-to-treat analysis. Moreover, more patients in the intervention group (79%) than in the placebo group (58%) showed culture conversion at 24 weeks, as well as at 120 weeks (62% vs 44%). And according to the World Health Organization definition of "cure," nearly twice as many patients in the bedaquiline group (58%) were cured as in the placebo group (32%) at 120 weeks, Dr. Diacon and his associates said (N. Engl. J. Med. 2014;371:723-32 [doi:10.1056/NEJMoa1313865]).
Fewer patients taking bedaquiline (2) than taking placebo (16) developed new resistance to at least one antituberculosis agent during the study. Rates, types, and severity of adverse events were comparable between the two study groups. However, mortality was significantly higher with bedaquiline (10 patients, 13% mortality) than with placebo (2 patients, 2% mortality), for reasons that are not yet understood but do not appear to be related to the study drug.
"A planned phase III study will enroll a larger number of patients, including HIV-positive patients receiving antiretroviral therapy," the investigators noted.
This study was funded by Janssen Pharmaceuticals. Dr. Diacon reported no financial conflicts of interest. His associates reported ties to Janssen; to its parent company, Johnson & Johnson; and to Quintiles.
Adding bedaquiline to a five-drug background regimen for multidrug-resistant pulmonary tuberculosis significantly accelerated the time at which sputum cultures showed clearance of Mycobacterium tuberculosis in a phase IIb clinical trial, which was published online Aug. 21 in the New England Journal of Medicine.
The addition of 6 months of oral bedaquiline – a diarylquinoline that inhibits mycobacterial ATP synthase and depletes cellular energy stores – also significantly increased the number of patients who achieved such culture conversion (a surrogate for clinical cure), compared with adding placebo, in this industry-sponsored study. Bedaquiline "is the first antituberculosis drug with a new mechanism of action to be approved for use in multidrug-resistant TB in 40 years," said Dr. Andreas H. Diacon of the National Research Foundation Centre of Excellence for Biomedical Tuberculosis Research at Stellenbosch University in Cape Town, South Africa, and his associates.
Bedaquiline was approved in 2012 under the Food and Drug Administration’s accelerated-approval regulations for drugs that provide meaningful therapeutic benefit over existing therapies for serious or life-threatening conditions. The drug is approved only for patients for whom an effective treatment regimen cannot be constructed without it, a population in whom the benefits outweigh the risks.
In the trial, 160 adults with multidrug-resistant pulmonary TB were randomly assigned to receive either oral bedaquiline (79 patients) or matching placebo tablets (81 patients) for 24 weeks at medical centers in Brazil, India, Latvia, Peru, the Philippines, Russia, South Africa, and Thailand, in addition to the five-drug background regimen of antituberculosis drugs preferred at each location. They continued the background therapy for a further 96 weeks and had a final follow-up exam at the conclusion of that treatment. Sixty participants (38%) left the study prematurely, usually because of withdrawal of consent, adverse effects, nonadherence, loss to follow-up, and pregnancy. The number of and reasons for these discontinuations were similar between the two study groups.
The primary endpoint of the study was the median time to sputum conversion. This was significantly faster in the intervention group (83 days) than in the control group (125 days) in both the modified intention-to-treat analysis and the full intention-to-treat analysis. Moreover, more patients in the intervention group (79%) than in the placebo group (58%) showed culture conversion at 24 weeks, as well as at 120 weeks (62% vs 44%). And according to the World Health Organization definition of "cure," nearly twice as many patients in the bedaquiline group (58%) were cured as in the placebo group (32%) at 120 weeks, Dr. Diacon and his associates said (N. Engl. J. Med. 2014;371:723-32 [doi:10.1056/NEJMoa1313865]).
Fewer patients taking bedaquiline (2) than taking placebo (16) developed new resistance to at least one antituberculosis agent during the study. Rates, types, and severity of adverse events were comparable between the two study groups. However, mortality was significantly higher with bedaquiline (10 patients, 13% mortality) than with placebo (2 patients, 2% mortality), for reasons that are not yet understood but do not appear to be related to the study drug.
"A planned phase III study will enroll a larger number of patients, including HIV-positive patients receiving antiretroviral therapy," the investigators noted.
This study was funded by Janssen Pharmaceuticals. Dr. Diacon reported no financial conflicts of interest. His associates reported ties to Janssen; to its parent company, Johnson & Johnson; and to Quintiles.
FROM THE NEW ENGLAND JOURNAL OF MEDICINE
Key clinical point: Bedaquiline may offer an alternative treatment for multidrug-resistant TB.
Major finding: The primary endpoint of the study – the median time to sputum conversion – was significantly faster in the intervention group (83 days) than in the control group (125 days), and more patients in the intervention group (79%) than in the placebo group (58%) showed culture conversion at 24 weeks, as well as at 120 weeks (62% vs 44%).
Data source: A randomized, double-blind, placebo-controlled, phase IIb clinical trial involving 160 adults with multidrug-resistant pulmonary TB treated for 24 weeks in eight countries.
Disclosures: This study was funded by Janssen Pharmaceuticals. Dr. Diacon reported no financial conflicts of interest. His associates reported ties to Janssen; to its parent company, Johnson & Johnson; and to Quintiles.
Too much reliance on oximetry in bronchiolitis
Among infants presenting to an emergency department with mild to moderate bronchiolitis, those whose pulse oximetry readings were artificially elevated by 3 percentage points were significantly less likely to be hospitalized than were comparable patients whose pulse oximetry readings were unaltered, according to a report published online Aug. 19 in JAMA.
The accuracy of most oxygen-saturation monitors is approximately plus or minus 2%, and oximetry readings are subject to multiple sources of error, so "the actual physiological difference between the true and altered oximetry measurements in our study was of minor importance. Yet this difference had significant implications for hospitalization," said Dr. Suzanne Schuh, a pediatrician at the Hospital for Sick Children, Toronto, and her associates.
Their findings indicate that clinicians are relying too heavily on tiny differences in a single laboratory parameter – the pulse oximetry reading – to make clinical decisions in this patient population. The results of their experiment further suggest that the monitoring of oxygen saturation in this setting may need to be reevaluated, Dr. Schuh and her colleagues wrote (JAMA 2014;312:712-8).
The introduction and routine use of pulse oximetry in infants with mild to moderate bronchiolitis in the 1980s and 1990s coincided with more than a doubling of hospitalization rates for the disorder. Moreover, the somewhat arbitrary cutoff point of 90% oxygen saturation, which triggers the use of supplemental oxygen, has never been shown to correlate with bronchiolitis progression, yet many mildly ill infants are hospitalized solely because of that reading. Even expert opinion "cautions against overreliance on oximetry and argues for the use of clinical judgment when making disposition determinations," the investigators noted.
They performed this prospective, randomized, double-blind trial to determine whether artificially raising the pulse oximetry reading displayed to emergency department (ED) clinicians by an amount of "minor importance" would lead to fewer unnecessary hospitalizations in 213 otherwise healthy infants (aged 4-12 months) presenting to a single tertiary care pediatric ED during a 5-year period. The 13 physicians who cared for these patients were blinded to the study protocol and ordered supplemental oxygen and pharmacotherapy at their own discretion.
All the infants had a true oxygen saturation of 88% or higher, as well as comparable clinical characteristics and Respiratory Disease Assessment Instrument scores. The oxygen saturation reading was accurately displayed to treating clinicians in the 108 infants who were randomly assigned to "true" oximetry. In the other 105 infants, the oxygen saturation reading was increased by 3 points and displayed to treating clinicians.
All the infants in the study underwent concealed continuous oximetry for safety reasons. These hidden monitors were programmed to sound an alarm if oxygen saturation dropped to less than 92%, which would prompt a thorough clinical reassessment.
The primary outcome of the study was hospitalization for bronchiolitis within 72 hours of enrollment due to concerns about respiratory distress. This occurred in 41% of infants in the true-oximetry group, compared with only 25% of those whose oximetry reading was artificially increased – a significant 16% difference. Yet the medical outcomes of the two study groups were comparable, Dr. Schuh and her associates reported.
"Our results suggest an even lower cutoff might be appropriate and that among children with saturation levels of 88% and higher, disposition determination should be based primarily on the degree of respiratory distress and hydration status, rather than on a particular saturation value," they said.
This study was supported by the Thrasher Research Fund and the Physicians’ Services Incorporated Foundation; Masimo provided the oximeters but played no role in the design or conduct of the study. Dr. Schuh and her associates reported that they had no financial conflicts of interest.
The conditions of this experiment "reflect the experience of many physicians who have been bedeviled by the reliance on oxygen saturation readings to make clinical decisions, particularly for infants with a self-limited disease who are mildly ill," commented Robert Vinci, M.D., and Howard Bauchner, M.D.
"It is now clear that the oxygen saturation reading can influence decision-making in ways that many physicians have thought likely – overreliance on physiologic information of uncertain importance derived from a medical device," they noted.
Dr. Vinci is a pediatrician at Boston Medical Center. Dr. Bauchner is editor in chief of JAMA. They reported no financial conflicts of interest. These remarks were taken from their editorial accompanying Dr. Schuh’s report (JAMA 2014;312:699-700).
The conditions of this experiment "reflect the experience of many physicians who have been bedeviled by the reliance on oxygen saturation readings to make clinical decisions, particularly for infants with a self-limited disease who are mildly ill," commented Robert Vinci, M.D., and Howard Bauchner, M.D.
"It is now clear that the oxygen saturation reading can influence decision-making in ways that many physicians have thought likely – overreliance on physiologic information of uncertain importance derived from a medical device," they noted.
Dr. Vinci is a pediatrician at Boston Medical Center. Dr. Bauchner is editor in chief of JAMA. They reported no financial conflicts of interest. These remarks were taken from their editorial accompanying Dr. Schuh’s report (JAMA 2014;312:699-700).
The conditions of this experiment "reflect the experience of many physicians who have been bedeviled by the reliance on oxygen saturation readings to make clinical decisions, particularly for infants with a self-limited disease who are mildly ill," commented Robert Vinci, M.D., and Howard Bauchner, M.D.
"It is now clear that the oxygen saturation reading can influence decision-making in ways that many physicians have thought likely – overreliance on physiologic information of uncertain importance derived from a medical device," they noted.
Dr. Vinci is a pediatrician at Boston Medical Center. Dr. Bauchner is editor in chief of JAMA. They reported no financial conflicts of interest. These remarks were taken from their editorial accompanying Dr. Schuh’s report (JAMA 2014;312:699-700).
Among infants presenting to an emergency department with mild to moderate bronchiolitis, those whose pulse oximetry readings were artificially elevated by 3 percentage points were significantly less likely to be hospitalized than were comparable patients whose pulse oximetry readings were unaltered, according to a report published online Aug. 19 in JAMA.
The accuracy of most oxygen-saturation monitors is approximately plus or minus 2%, and oximetry readings are subject to multiple sources of error, so "the actual physiological difference between the true and altered oximetry measurements in our study was of minor importance. Yet this difference had significant implications for hospitalization," said Dr. Suzanne Schuh, a pediatrician at the Hospital for Sick Children, Toronto, and her associates.
Their findings indicate that clinicians are relying too heavily on tiny differences in a single laboratory parameter – the pulse oximetry reading – to make clinical decisions in this patient population. The results of their experiment further suggest that the monitoring of oxygen saturation in this setting may need to be reevaluated, Dr. Schuh and her colleagues wrote (JAMA 2014;312:712-8).
The introduction and routine use of pulse oximetry in infants with mild to moderate bronchiolitis in the 1980s and 1990s coincided with more than a doubling of hospitalization rates for the disorder. Moreover, the somewhat arbitrary cutoff point of 90% oxygen saturation, which triggers the use of supplemental oxygen, has never been shown to correlate with bronchiolitis progression, yet many mildly ill infants are hospitalized solely because of that reading. Even expert opinion "cautions against overreliance on oximetry and argues for the use of clinical judgment when making disposition determinations," the investigators noted.
They performed this prospective, randomized, double-blind trial to determine whether artificially raising the pulse oximetry reading displayed to emergency department (ED) clinicians by an amount of "minor importance" would lead to fewer unnecessary hospitalizations in 213 otherwise healthy infants (aged 4-12 months) presenting to a single tertiary care pediatric ED during a 5-year period. The 13 physicians who cared for these patients were blinded to the study protocol and ordered supplemental oxygen and pharmacotherapy at their own discretion.
All the infants had a true oxygen saturation of 88% or higher, as well as comparable clinical characteristics and Respiratory Disease Assessment Instrument scores. The oxygen saturation reading was accurately displayed to treating clinicians in the 108 infants who were randomly assigned to "true" oximetry. In the other 105 infants, the oxygen saturation reading was increased by 3 points and displayed to treating clinicians.
All the infants in the study underwent concealed continuous oximetry for safety reasons. These hidden monitors were programmed to sound an alarm if oxygen saturation dropped to less than 92%, which would prompt a thorough clinical reassessment.
The primary outcome of the study was hospitalization for bronchiolitis within 72 hours of enrollment due to concerns about respiratory distress. This occurred in 41% of infants in the true-oximetry group, compared with only 25% of those whose oximetry reading was artificially increased – a significant 16% difference. Yet the medical outcomes of the two study groups were comparable, Dr. Schuh and her associates reported.
"Our results suggest an even lower cutoff might be appropriate and that among children with saturation levels of 88% and higher, disposition determination should be based primarily on the degree of respiratory distress and hydration status, rather than on a particular saturation value," they said.
This study was supported by the Thrasher Research Fund and the Physicians’ Services Incorporated Foundation; Masimo provided the oximeters but played no role in the design or conduct of the study. Dr. Schuh and her associates reported that they had no financial conflicts of interest.
Among infants presenting to an emergency department with mild to moderate bronchiolitis, those whose pulse oximetry readings were artificially elevated by 3 percentage points were significantly less likely to be hospitalized than were comparable patients whose pulse oximetry readings were unaltered, according to a report published online Aug. 19 in JAMA.
The accuracy of most oxygen-saturation monitors is approximately plus or minus 2%, and oximetry readings are subject to multiple sources of error, so "the actual physiological difference between the true and altered oximetry measurements in our study was of minor importance. Yet this difference had significant implications for hospitalization," said Dr. Suzanne Schuh, a pediatrician at the Hospital for Sick Children, Toronto, and her associates.
Their findings indicate that clinicians are relying too heavily on tiny differences in a single laboratory parameter – the pulse oximetry reading – to make clinical decisions in this patient population. The results of their experiment further suggest that the monitoring of oxygen saturation in this setting may need to be reevaluated, Dr. Schuh and her colleagues wrote (JAMA 2014;312:712-8).
The introduction and routine use of pulse oximetry in infants with mild to moderate bronchiolitis in the 1980s and 1990s coincided with more than a doubling of hospitalization rates for the disorder. Moreover, the somewhat arbitrary cutoff point of 90% oxygen saturation, which triggers the use of supplemental oxygen, has never been shown to correlate with bronchiolitis progression, yet many mildly ill infants are hospitalized solely because of that reading. Even expert opinion "cautions against overreliance on oximetry and argues for the use of clinical judgment when making disposition determinations," the investigators noted.
They performed this prospective, randomized, double-blind trial to determine whether artificially raising the pulse oximetry reading displayed to emergency department (ED) clinicians by an amount of "minor importance" would lead to fewer unnecessary hospitalizations in 213 otherwise healthy infants (aged 4-12 months) presenting to a single tertiary care pediatric ED during a 5-year period. The 13 physicians who cared for these patients were blinded to the study protocol and ordered supplemental oxygen and pharmacotherapy at their own discretion.
All the infants had a true oxygen saturation of 88% or higher, as well as comparable clinical characteristics and Respiratory Disease Assessment Instrument scores. The oxygen saturation reading was accurately displayed to treating clinicians in the 108 infants who were randomly assigned to "true" oximetry. In the other 105 infants, the oxygen saturation reading was increased by 3 points and displayed to treating clinicians.
All the infants in the study underwent concealed continuous oximetry for safety reasons. These hidden monitors were programmed to sound an alarm if oxygen saturation dropped to less than 92%, which would prompt a thorough clinical reassessment.
The primary outcome of the study was hospitalization for bronchiolitis within 72 hours of enrollment due to concerns about respiratory distress. This occurred in 41% of infants in the true-oximetry group, compared with only 25% of those whose oximetry reading was artificially increased – a significant 16% difference. Yet the medical outcomes of the two study groups were comparable, Dr. Schuh and her associates reported.
"Our results suggest an even lower cutoff might be appropriate and that among children with saturation levels of 88% and higher, disposition determination should be based primarily on the degree of respiratory distress and hydration status, rather than on a particular saturation value," they said.
This study was supported by the Thrasher Research Fund and the Physicians’ Services Incorporated Foundation; Masimo provided the oximeters but played no role in the design or conduct of the study. Dr. Schuh and her associates reported that they had no financial conflicts of interest.
FROM JAMA
Key clinical point: Physicians may be overrelying on pulse oximetry readings when evaluating infants who present to the ED with mild to moderate bronchiolitis.
Major finding: The primary outcome of the study – hospitalization for bronchiolitis within 72 hours of enrollment due to concerns about respiratory distress – occurred in 41% of infants in the true-oximetry group, compared with only 25% of those whose oximetry reading was artificially increased.
Data source: A prospective, randomized, double-blind clinical trial assessing the medical management of 213 infants presenting to a single tertiary-care ED with mild to moderate bronchiolitis during a 5-year period.
Disclosures: This study was supported by the Thrasher Research Fund and the Physicians’ Services Incorporated Foundation; Masimo provided the oximeters but played no role in the design or conduct of the study. Dr. Schuh and her associates reported that they had no financial conflicts of interest.
Life expectancy approached 40 years for children born with CF in 2010
Mortality in patients with cystic fibrosis fell by almost 2% per year in the United States between 2000 and 2010, authors of a registry-based study reported online August 18 in Annals of Internal Medicine.
Based on the analyses, patients born and diagnosed in 2010 can expect to live almost 40 years if the state of CF care remains static, and about 56 years if treatments progress at the current rate, said Todd MacKenzie, Ph.D., of Dartmouth School of Medicine in Hanover, N.H., and his associates.
The study included 110 Cystic Fibrosis Foundation–accredited care centers in the United States. Mortality fell by 1.8% per year between 2000 and 2010 (95% confidence interval, 0.5%-2.7%), and males had a 19% lower risk of death than females (95% CI, 13%-24%), the investigators found. The estimated median survival for children born and diagnosed in 2010 was 39 years overall (95% CI, 38-40 years), 37 years for females (95% CI, 35-39 years), and 40 years for males (95% CI, 39-42 years), they reported (Ann. Int. Med. 2014 Aug. 18 [doi:10.7326/M13-0636]).
The results apply only to patients diagnosed in the first year of life, as patients diagnosed later are likely pancreatic-sufficient and therefore have a better prognosis, Dr. MacKenzie and associates noted. Improved survival could partially reflect the increasing diagnosis of patients with less severe phenotypes, they said.
The Cystic Fibrosis Foundation funded the study. Dr. MacKenzie reported no conflicts of interest. Three coauthors reported grant support from the Cystic Fibrosis Foundation.
The findings are "worth celebrating," said Dr. Joseph Pilewski and Dr. Darren Taichman. "However, continuing to ensure the kind of care that has resulted in a growing number of patients with CF living far into adulthood will not be simple or inexpensive."
The patients in the study all were treated at CF Foundation–accredited centers, which provide coordinated, multidisciplinary care, Dr. Pilewski and Dr. Taichman said. And patients with CF often need care from additional specialists for comorbidities such as diabetes, osteoporosis, cancer, depression, and anxiety, they added. "Caring for adults with CF requires a village."
As for other complex primary care scenarios, coordinating and managing the medical and social issues of CF patients can be time-intensive, stressful, and poorly reimbursed, they said. Furthermore, coordinated nursing, social work, nutrition, and respiratory therapy services often are not available outside accredited CF care centers.
"Will the remarkable community support that has enabled progress in CF care and survival continue in an era when growing proportions of patients are no longer children, for whom it may be easier to solicit donations?" Dr. Pilewski and Dr. Taichman asked. "If not, we will need to think hard about other solutions because, although survival of patients with CF has improved, many still suffer and die prematurely."
Dr. Pilewski is codirector of the Adult Cystic Fibrosis program at the University of Pittsburgh Medical Center. Dr. Taichman is executive deputy editor for Annals of Internal Medicine. These remarks were taken from their editorial accompanying Dr. MacKenzie’s report (Ann. Int. Med. 2014 Aug. 18 [doi:10.7326/M14-1534]).
The findings are "worth celebrating," said Dr. Joseph Pilewski and Dr. Darren Taichman. "However, continuing to ensure the kind of care that has resulted in a growing number of patients with CF living far into adulthood will not be simple or inexpensive."
The patients in the study all were treated at CF Foundation–accredited centers, which provide coordinated, multidisciplinary care, Dr. Pilewski and Dr. Taichman said. And patients with CF often need care from additional specialists for comorbidities such as diabetes, osteoporosis, cancer, depression, and anxiety, they added. "Caring for adults with CF requires a village."
As for other complex primary care scenarios, coordinating and managing the medical and social issues of CF patients can be time-intensive, stressful, and poorly reimbursed, they said. Furthermore, coordinated nursing, social work, nutrition, and respiratory therapy services often are not available outside accredited CF care centers.
"Will the remarkable community support that has enabled progress in CF care and survival continue in an era when growing proportions of patients are no longer children, for whom it may be easier to solicit donations?" Dr. Pilewski and Dr. Taichman asked. "If not, we will need to think hard about other solutions because, although survival of patients with CF has improved, many still suffer and die prematurely."
Dr. Pilewski is codirector of the Adult Cystic Fibrosis program at the University of Pittsburgh Medical Center. Dr. Taichman is executive deputy editor for Annals of Internal Medicine. These remarks were taken from their editorial accompanying Dr. MacKenzie’s report (Ann. Int. Med. 2014 Aug. 18 [doi:10.7326/M14-1534]).
The findings are "worth celebrating," said Dr. Joseph Pilewski and Dr. Darren Taichman. "However, continuing to ensure the kind of care that has resulted in a growing number of patients with CF living far into adulthood will not be simple or inexpensive."
The patients in the study all were treated at CF Foundation–accredited centers, which provide coordinated, multidisciplinary care, Dr. Pilewski and Dr. Taichman said. And patients with CF often need care from additional specialists for comorbidities such as diabetes, osteoporosis, cancer, depression, and anxiety, they added. "Caring for adults with CF requires a village."
As for other complex primary care scenarios, coordinating and managing the medical and social issues of CF patients can be time-intensive, stressful, and poorly reimbursed, they said. Furthermore, coordinated nursing, social work, nutrition, and respiratory therapy services often are not available outside accredited CF care centers.
"Will the remarkable community support that has enabled progress in CF care and survival continue in an era when growing proportions of patients are no longer children, for whom it may be easier to solicit donations?" Dr. Pilewski and Dr. Taichman asked. "If not, we will need to think hard about other solutions because, although survival of patients with CF has improved, many still suffer and die prematurely."
Dr. Pilewski is codirector of the Adult Cystic Fibrosis program at the University of Pittsburgh Medical Center. Dr. Taichman is executive deputy editor for Annals of Internal Medicine. These remarks were taken from their editorial accompanying Dr. MacKenzie’s report (Ann. Int. Med. 2014 Aug. 18 [doi:10.7326/M14-1534]).
Mortality in patients with cystic fibrosis fell by almost 2% per year in the United States between 2000 and 2010, authors of a registry-based study reported online August 18 in Annals of Internal Medicine.
Based on the analyses, patients born and diagnosed in 2010 can expect to live almost 40 years if the state of CF care remains static, and about 56 years if treatments progress at the current rate, said Todd MacKenzie, Ph.D., of Dartmouth School of Medicine in Hanover, N.H., and his associates.
The study included 110 Cystic Fibrosis Foundation–accredited care centers in the United States. Mortality fell by 1.8% per year between 2000 and 2010 (95% confidence interval, 0.5%-2.7%), and males had a 19% lower risk of death than females (95% CI, 13%-24%), the investigators found. The estimated median survival for children born and diagnosed in 2010 was 39 years overall (95% CI, 38-40 years), 37 years for females (95% CI, 35-39 years), and 40 years for males (95% CI, 39-42 years), they reported (Ann. Int. Med. 2014 Aug. 18 [doi:10.7326/M13-0636]).
The results apply only to patients diagnosed in the first year of life, as patients diagnosed later are likely pancreatic-sufficient and therefore have a better prognosis, Dr. MacKenzie and associates noted. Improved survival could partially reflect the increasing diagnosis of patients with less severe phenotypes, they said.
The Cystic Fibrosis Foundation funded the study. Dr. MacKenzie reported no conflicts of interest. Three coauthors reported grant support from the Cystic Fibrosis Foundation.
Mortality in patients with cystic fibrosis fell by almost 2% per year in the United States between 2000 and 2010, authors of a registry-based study reported online August 18 in Annals of Internal Medicine.
Based on the analyses, patients born and diagnosed in 2010 can expect to live almost 40 years if the state of CF care remains static, and about 56 years if treatments progress at the current rate, said Todd MacKenzie, Ph.D., of Dartmouth School of Medicine in Hanover, N.H., and his associates.
The study included 110 Cystic Fibrosis Foundation–accredited care centers in the United States. Mortality fell by 1.8% per year between 2000 and 2010 (95% confidence interval, 0.5%-2.7%), and males had a 19% lower risk of death than females (95% CI, 13%-24%), the investigators found. The estimated median survival for children born and diagnosed in 2010 was 39 years overall (95% CI, 38-40 years), 37 years for females (95% CI, 35-39 years), and 40 years for males (95% CI, 39-42 years), they reported (Ann. Int. Med. 2014 Aug. 18 [doi:10.7326/M13-0636]).
The results apply only to patients diagnosed in the first year of life, as patients diagnosed later are likely pancreatic-sufficient and therefore have a better prognosis, Dr. MacKenzie and associates noted. Improved survival could partially reflect the increasing diagnosis of patients with less severe phenotypes, they said.
The Cystic Fibrosis Foundation funded the study. Dr. MacKenzie reported no conflicts of interest. Three coauthors reported grant support from the Cystic Fibrosis Foundation.
FROM ANNALS OF INTERNAL MEDICINE
Key clinical point: Patients with cystic fibrosis who are born and diagnosed in 2010 can expect to live almost 40 years if clinical care remains at its current level.
Major finding: Mortality in patients with cystic fibrosis fell by 1.8% per year between 2000 and 2010 (95% confidence interval, 0.5%-2.7%), and patients born and diagnosed in 2010 were projected to survive 39 years given no further improvements in the current state of care (95% CI, 38-40 years).
Data source: Registry-based study of 110 Cystic Fibrosis Foundation–accredited care centers in the United States.
Disclosures: The Cystic Fibrosis Foundation funded the study. Dr. MacKenzie reported no conflicts of interest. Three coauthors reported grant support from the Cystic Fibrosis Foundation.