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Tocilizumab preserves lung function in systemic sclerosis
MADRID – , according to a secondary endpoint analysis of the phase 3, double-blind, randomized, controlled focuSSced trial.
After 48 weeks, a significantly lower proportion of patients treated with tocilizumab than placebo experienced any decline in lung function from baseline (50.5% versus 70.3% (P = .015), as defined by the percentage increase in predicted forced vital capacity (%pFVC). When only patients with interstitial lung disease (ILD) were considered, the respective percentages were 51.7% and 75.5% (P = .003).
In SSc-ILD patients, a clinically meaningful decline of 10% or more of the %pFVC in lung function was seen in 24.5% given placebo but in just 8.6% of those treated with tocilizumab.
“ILD is a major complication of scleroderma; it has high morbidity and mortality ... and it’s largely irreversible,” Dinesh Khanna, MD, said at the European Congress of Rheumatology.
“In this day and age, when we treat ILD, we wait for a patient to develop clinical ILD,” added Dr. Khanna, director of the scleroderma program at the University of Michigan, Ann Arbor. Clinical ILD can be defined by symptoms, abnormal pulmonary function tests, and marked abnormalities on high resolution computed tomography (HRCT) scans. He indicated that if improving ILD was not possible, then the next best thing would be to stabilize the disease and ensure there was no worsening in lung function.
As yet, there are no disease-modifying treatments available to treat SSc but there are “ample data that interleukin-6 plays a very important role in the pathogenesis of scleroderma,” Dr. Khanna observed. Tocilizumab is a humanized monoclonal antibody against the interleukin-6 receptor.
Data from the phase 2 faSScinate trial showed initial promise for the drug in SSc where a numerical, but not statistically significant, improvement in skin thickening was seen, and the results had hinted at a possible benefit on lung function (Lancet. 2016 Jun 25;387:2630-40).
However, in the phase 3 focuSSced trial, there was no statistically significant difference in the change from baseline to week 48 modified Rodnan skin score (mRSS) between tocilizumab and placebo, which was the primary endpoint. The least square mean change in mRSS was –6.14 for tocilizumab and –4.41 for placebo (P = .0983).
A total of 205 patients with SSc were studied and randomized, 1:1 in a double-blind fashion, to receive either a once-weekly, subcutaneous dose of 162 mg tocilizumab or a weekly subcutaneous placebo injection for 48 weeks.
For inclusion in the study, patients had to have SSc that met American College of Rheumatology and European League Against Rheumatism (EULAR) criteria and be diagnosed less than 60 months previously. Patients had to have an mRSS of 10-35 units and active disease with one or more of the following: C-reactive protein of 6 mg/L or higher; erythrocyte sedimentation rate of 28 mm/h or higher; and platelet count of330 x 109 L.
“What was astonishing in the trial was that every patient had HRCT at baseline and at the end of the study,” Dr. Khanna reported. These scans showed that 64% of patients had evidence of ILD at baseline and that those treated with tocilizumab had less evidence of fibrosis at week 48 versus placebo, indicating a stabilization rather than worsening of disease.
A time to treatment failure analysis also favored tocilizumab over placebo, but there were no significant changes in patient-reported outcomes.
Dr. Khanna’s slides stated that “given that the primary endpoint for mRSS was not met, all other P values are presented for information purposes only and cannot be considered statistically significant despite the strength of the evidence.” During the Q&A after his presentation, he noted that it was unlikely that the study’s sponsors (Roche/Genentech) will now pursue a license for tocilizumab in SSc.
Nevertheless, Dr. Khanna concluded, “we have the opportunity, based on these data, to treat these patients early on, where you can preserve the lung function, which is a paradigm shift versus waiting for the lung function to decline, become clinically meaningful, significant, and then treat this patient population.”
Roche/Genentech sponsored the study. Dr. Khanna acts as a consultant to Roche/Genentech and eight other pharmaceutical companies. He owns stock in Eicos Sciences.
SOURCE: Khanna D et al. Ann Rheum Dis. Jun 2019;78(Suppl 2):202-3. Abstract OP0245, doi: 10.1136/annrheumdis-2019-eular.2120
MADRID – , according to a secondary endpoint analysis of the phase 3, double-blind, randomized, controlled focuSSced trial.
After 48 weeks, a significantly lower proportion of patients treated with tocilizumab than placebo experienced any decline in lung function from baseline (50.5% versus 70.3% (P = .015), as defined by the percentage increase in predicted forced vital capacity (%pFVC). When only patients with interstitial lung disease (ILD) were considered, the respective percentages were 51.7% and 75.5% (P = .003).
In SSc-ILD patients, a clinically meaningful decline of 10% or more of the %pFVC in lung function was seen in 24.5% given placebo but in just 8.6% of those treated with tocilizumab.
“ILD is a major complication of scleroderma; it has high morbidity and mortality ... and it’s largely irreversible,” Dinesh Khanna, MD, said at the European Congress of Rheumatology.
“In this day and age, when we treat ILD, we wait for a patient to develop clinical ILD,” added Dr. Khanna, director of the scleroderma program at the University of Michigan, Ann Arbor. Clinical ILD can be defined by symptoms, abnormal pulmonary function tests, and marked abnormalities on high resolution computed tomography (HRCT) scans. He indicated that if improving ILD was not possible, then the next best thing would be to stabilize the disease and ensure there was no worsening in lung function.
As yet, there are no disease-modifying treatments available to treat SSc but there are “ample data that interleukin-6 plays a very important role in the pathogenesis of scleroderma,” Dr. Khanna observed. Tocilizumab is a humanized monoclonal antibody against the interleukin-6 receptor.
Data from the phase 2 faSScinate trial showed initial promise for the drug in SSc where a numerical, but not statistically significant, improvement in skin thickening was seen, and the results had hinted at a possible benefit on lung function (Lancet. 2016 Jun 25;387:2630-40).
However, in the phase 3 focuSSced trial, there was no statistically significant difference in the change from baseline to week 48 modified Rodnan skin score (mRSS) between tocilizumab and placebo, which was the primary endpoint. The least square mean change in mRSS was –6.14 for tocilizumab and –4.41 for placebo (P = .0983).
A total of 205 patients with SSc were studied and randomized, 1:1 in a double-blind fashion, to receive either a once-weekly, subcutaneous dose of 162 mg tocilizumab or a weekly subcutaneous placebo injection for 48 weeks.
For inclusion in the study, patients had to have SSc that met American College of Rheumatology and European League Against Rheumatism (EULAR) criteria and be diagnosed less than 60 months previously. Patients had to have an mRSS of 10-35 units and active disease with one or more of the following: C-reactive protein of 6 mg/L or higher; erythrocyte sedimentation rate of 28 mm/h or higher; and platelet count of330 x 109 L.
“What was astonishing in the trial was that every patient had HRCT at baseline and at the end of the study,” Dr. Khanna reported. These scans showed that 64% of patients had evidence of ILD at baseline and that those treated with tocilizumab had less evidence of fibrosis at week 48 versus placebo, indicating a stabilization rather than worsening of disease.
A time to treatment failure analysis also favored tocilizumab over placebo, but there were no significant changes in patient-reported outcomes.
Dr. Khanna’s slides stated that “given that the primary endpoint for mRSS was not met, all other P values are presented for information purposes only and cannot be considered statistically significant despite the strength of the evidence.” During the Q&A after his presentation, he noted that it was unlikely that the study’s sponsors (Roche/Genentech) will now pursue a license for tocilizumab in SSc.
Nevertheless, Dr. Khanna concluded, “we have the opportunity, based on these data, to treat these patients early on, where you can preserve the lung function, which is a paradigm shift versus waiting for the lung function to decline, become clinically meaningful, significant, and then treat this patient population.”
Roche/Genentech sponsored the study. Dr. Khanna acts as a consultant to Roche/Genentech and eight other pharmaceutical companies. He owns stock in Eicos Sciences.
SOURCE: Khanna D et al. Ann Rheum Dis. Jun 2019;78(Suppl 2):202-3. Abstract OP0245, doi: 10.1136/annrheumdis-2019-eular.2120
MADRID – , according to a secondary endpoint analysis of the phase 3, double-blind, randomized, controlled focuSSced trial.
After 48 weeks, a significantly lower proportion of patients treated with tocilizumab than placebo experienced any decline in lung function from baseline (50.5% versus 70.3% (P = .015), as defined by the percentage increase in predicted forced vital capacity (%pFVC). When only patients with interstitial lung disease (ILD) were considered, the respective percentages were 51.7% and 75.5% (P = .003).
In SSc-ILD patients, a clinically meaningful decline of 10% or more of the %pFVC in lung function was seen in 24.5% given placebo but in just 8.6% of those treated with tocilizumab.
“ILD is a major complication of scleroderma; it has high morbidity and mortality ... and it’s largely irreversible,” Dinesh Khanna, MD, said at the European Congress of Rheumatology.
“In this day and age, when we treat ILD, we wait for a patient to develop clinical ILD,” added Dr. Khanna, director of the scleroderma program at the University of Michigan, Ann Arbor. Clinical ILD can be defined by symptoms, abnormal pulmonary function tests, and marked abnormalities on high resolution computed tomography (HRCT) scans. He indicated that if improving ILD was not possible, then the next best thing would be to stabilize the disease and ensure there was no worsening in lung function.
As yet, there are no disease-modifying treatments available to treat SSc but there are “ample data that interleukin-6 plays a very important role in the pathogenesis of scleroderma,” Dr. Khanna observed. Tocilizumab is a humanized monoclonal antibody against the interleukin-6 receptor.
Data from the phase 2 faSScinate trial showed initial promise for the drug in SSc where a numerical, but not statistically significant, improvement in skin thickening was seen, and the results had hinted at a possible benefit on lung function (Lancet. 2016 Jun 25;387:2630-40).
However, in the phase 3 focuSSced trial, there was no statistically significant difference in the change from baseline to week 48 modified Rodnan skin score (mRSS) between tocilizumab and placebo, which was the primary endpoint. The least square mean change in mRSS was –6.14 for tocilizumab and –4.41 for placebo (P = .0983).
A total of 205 patients with SSc were studied and randomized, 1:1 in a double-blind fashion, to receive either a once-weekly, subcutaneous dose of 162 mg tocilizumab or a weekly subcutaneous placebo injection for 48 weeks.
For inclusion in the study, patients had to have SSc that met American College of Rheumatology and European League Against Rheumatism (EULAR) criteria and be diagnosed less than 60 months previously. Patients had to have an mRSS of 10-35 units and active disease with one or more of the following: C-reactive protein of 6 mg/L or higher; erythrocyte sedimentation rate of 28 mm/h or higher; and platelet count of330 x 109 L.
“What was astonishing in the trial was that every patient had HRCT at baseline and at the end of the study,” Dr. Khanna reported. These scans showed that 64% of patients had evidence of ILD at baseline and that those treated with tocilizumab had less evidence of fibrosis at week 48 versus placebo, indicating a stabilization rather than worsening of disease.
A time to treatment failure analysis also favored tocilizumab over placebo, but there were no significant changes in patient-reported outcomes.
Dr. Khanna’s slides stated that “given that the primary endpoint for mRSS was not met, all other P values are presented for information purposes only and cannot be considered statistically significant despite the strength of the evidence.” During the Q&A after his presentation, he noted that it was unlikely that the study’s sponsors (Roche/Genentech) will now pursue a license for tocilizumab in SSc.
Nevertheless, Dr. Khanna concluded, “we have the opportunity, based on these data, to treat these patients early on, where you can preserve the lung function, which is a paradigm shift versus waiting for the lung function to decline, become clinically meaningful, significant, and then treat this patient population.”
Roche/Genentech sponsored the study. Dr. Khanna acts as a consultant to Roche/Genentech and eight other pharmaceutical companies. He owns stock in Eicos Sciences.
SOURCE: Khanna D et al. Ann Rheum Dis. Jun 2019;78(Suppl 2):202-3. Abstract OP0245, doi: 10.1136/annrheumdis-2019-eular.2120
REPORTING FROM THE EULAR 2019 CONGRESS
Medicare may best Medicare Advantage at reducing readmissions
Although earlier research may suggest otherwise, traditional new research suggests.
Researchers used what they described as “a novel data linkage” comparing 30-day readmission rates after hospitalization for three major conditions in the Hospital Readmissions Reduction Program for patients using traditional Medicare versus Medicare Advantage. Those conditions included acute MI, heart failure, and pneumonia.
“Our results contrast with those of previous studies that have reported lower or statistically similar readmission rates for Medicare Advantage beneficiaries,” Orestis A. Panagiotou, MD, of Brown University, Providence, R.I., and colleagues wrote in a research report published in Annals of Internal Medicine.
In this retrospective cohort study, the researchers linked data from 2011 to 2014 from the Medicare Provider Analysis and Review (MedPAR) file to the Healthcare Effectiveness Data and Information Set (HEDIS).
The novel linkage found that HEDIS data underreported hospital admissions for acute MI, heart failure, and pneumonia, the researchers stated. “Plans incorrectly excluded hospitalizations that should have qualified for the readmission measure, and readmission rates were substantially higher among incorrectly excluded hospitalizations.”
Despite this, in analyses using the linkage of HEDIS and MedPAR, “Medicare Advantage beneficiaries had higher 30-day risk-adjusted readmission rates after [acute MI, heart failure, and pneumonia] than did traditional Medicare beneficiaries,” the investigators noted.
Patients in Medicare Advantage had lower unadjusted readmission rates compared with those in traditional Medicare (16.6% vs. 17.1% for acute MI; 21.4% vs. 21.7% for heart failure; and 16.3% vs. 16.4% for pneumonia). After standardization, Medicare Advantage patients had higher readmission rates, compared with those in traditional Medicare (17.2% vs. 16.9% for acute MI; 21.7% vs. 21.4% for heart failure; and 16.5% vs. 16.0% for pneumonia).
The study authors added that, while unadjusted readmission rates were higher for traditional Medicare beneficiaries, “the direction of the difference reversed after standardization. This occurred because Medicare Advantage beneficiaries have, on average, a lower expected readmission risk [that is, they are ‘healthier’].” Prior studies have documented that Medicare Advantage plans enroll beneficiaries with fewer comorbid conditions and that high-cost beneficiaries switch out of Medicare Advantage and into traditional Medicare.
The researchers suggested four reasons for the differences between the results in this study versus others that compared patients using Medicare with those using Medicare Advantage. These were that the new study included a more comprehensive data set, analyses with comorbid conditions “from a well-validated model applied by CMS [Centers for Medicare & Medicaid Services],” national data focused on three conditions included in the Hospital Readmissions Reduction Program, and patients discharged to places other than skilled nursing facilities and inpatient rehabilitation facilities.
Authors of an accompanying editorial called for caution to be used in interpreting Medicare Advantage enrollment as causing an increased readmission risk.
“[The] results are sensitive to adjustment for case mix,” wrote Peter Huckfeldt, PhD, of the University of Minnesota, Minneapolis, and Neeraj Sood, PhD, of the University of Southern California, Los Angeles, in the editorial published in Annals of Internal Medicine (2019 June 25. doi:10.7326/M19-1599) “Using diagnosis codes on hospital claims for case-mix adjustments may be increasingly perilous. ... To our knowledge, there is no recent evidence comparing the intensity of diagnostic coding between clinically similar [traditional Medicare] and [Medicare Advantage] hospital admissions, but if [traditional Medicare] enrollees were coded more intensively than [Medicare Advantage] enrollees, this could lead to [traditional Medicare] enrollees having lower risk-adjusted readmission rares due to coding practices.”
The editorialists added that using a cross-sectional comparison of Medicare Advantage and traditional Medicare patients is concerning because a “key challenge in estimating the effect of [Medicare Advantage] is that enrollment is voluntary,” which can lead to a number of analytical concerns.
The researchers concluded that their findings “are concerning because CMS uses HEDIS performance to construct composite quality ratings and assign payment bonuses to Medicare Advantage plans.
“Our study suggests a need for improved monitoring of the accuracy of HEDIS data,” they noted.
The National Institute on Aging provided the primary funding for this study. A number of the authors received grants from the National Institutes of Health during the conduct of the study. No other relevant disclosures were reported.
SOURCE: Panagiotou OA et al. Ann Intern Med. 2019 Jun 25. doi: 10.7326/M18-1795.
Although earlier research may suggest otherwise, traditional new research suggests.
Researchers used what they described as “a novel data linkage” comparing 30-day readmission rates after hospitalization for three major conditions in the Hospital Readmissions Reduction Program for patients using traditional Medicare versus Medicare Advantage. Those conditions included acute MI, heart failure, and pneumonia.
“Our results contrast with those of previous studies that have reported lower or statistically similar readmission rates for Medicare Advantage beneficiaries,” Orestis A. Panagiotou, MD, of Brown University, Providence, R.I., and colleagues wrote in a research report published in Annals of Internal Medicine.
In this retrospective cohort study, the researchers linked data from 2011 to 2014 from the Medicare Provider Analysis and Review (MedPAR) file to the Healthcare Effectiveness Data and Information Set (HEDIS).
The novel linkage found that HEDIS data underreported hospital admissions for acute MI, heart failure, and pneumonia, the researchers stated. “Plans incorrectly excluded hospitalizations that should have qualified for the readmission measure, and readmission rates were substantially higher among incorrectly excluded hospitalizations.”
Despite this, in analyses using the linkage of HEDIS and MedPAR, “Medicare Advantage beneficiaries had higher 30-day risk-adjusted readmission rates after [acute MI, heart failure, and pneumonia] than did traditional Medicare beneficiaries,” the investigators noted.
Patients in Medicare Advantage had lower unadjusted readmission rates compared with those in traditional Medicare (16.6% vs. 17.1% for acute MI; 21.4% vs. 21.7% for heart failure; and 16.3% vs. 16.4% for pneumonia). After standardization, Medicare Advantage patients had higher readmission rates, compared with those in traditional Medicare (17.2% vs. 16.9% for acute MI; 21.7% vs. 21.4% for heart failure; and 16.5% vs. 16.0% for pneumonia).
The study authors added that, while unadjusted readmission rates were higher for traditional Medicare beneficiaries, “the direction of the difference reversed after standardization. This occurred because Medicare Advantage beneficiaries have, on average, a lower expected readmission risk [that is, they are ‘healthier’].” Prior studies have documented that Medicare Advantage plans enroll beneficiaries with fewer comorbid conditions and that high-cost beneficiaries switch out of Medicare Advantage and into traditional Medicare.
The researchers suggested four reasons for the differences between the results in this study versus others that compared patients using Medicare with those using Medicare Advantage. These were that the new study included a more comprehensive data set, analyses with comorbid conditions “from a well-validated model applied by CMS [Centers for Medicare & Medicaid Services],” national data focused on three conditions included in the Hospital Readmissions Reduction Program, and patients discharged to places other than skilled nursing facilities and inpatient rehabilitation facilities.
Authors of an accompanying editorial called for caution to be used in interpreting Medicare Advantage enrollment as causing an increased readmission risk.
“[The] results are sensitive to adjustment for case mix,” wrote Peter Huckfeldt, PhD, of the University of Minnesota, Minneapolis, and Neeraj Sood, PhD, of the University of Southern California, Los Angeles, in the editorial published in Annals of Internal Medicine (2019 June 25. doi:10.7326/M19-1599) “Using diagnosis codes on hospital claims for case-mix adjustments may be increasingly perilous. ... To our knowledge, there is no recent evidence comparing the intensity of diagnostic coding between clinically similar [traditional Medicare] and [Medicare Advantage] hospital admissions, but if [traditional Medicare] enrollees were coded more intensively than [Medicare Advantage] enrollees, this could lead to [traditional Medicare] enrollees having lower risk-adjusted readmission rares due to coding practices.”
The editorialists added that using a cross-sectional comparison of Medicare Advantage and traditional Medicare patients is concerning because a “key challenge in estimating the effect of [Medicare Advantage] is that enrollment is voluntary,” which can lead to a number of analytical concerns.
The researchers concluded that their findings “are concerning because CMS uses HEDIS performance to construct composite quality ratings and assign payment bonuses to Medicare Advantage plans.
“Our study suggests a need for improved monitoring of the accuracy of HEDIS data,” they noted.
The National Institute on Aging provided the primary funding for this study. A number of the authors received grants from the National Institutes of Health during the conduct of the study. No other relevant disclosures were reported.
SOURCE: Panagiotou OA et al. Ann Intern Med. 2019 Jun 25. doi: 10.7326/M18-1795.
Although earlier research may suggest otherwise, traditional new research suggests.
Researchers used what they described as “a novel data linkage” comparing 30-day readmission rates after hospitalization for three major conditions in the Hospital Readmissions Reduction Program for patients using traditional Medicare versus Medicare Advantage. Those conditions included acute MI, heart failure, and pneumonia.
“Our results contrast with those of previous studies that have reported lower or statistically similar readmission rates for Medicare Advantage beneficiaries,” Orestis A. Panagiotou, MD, of Brown University, Providence, R.I., and colleagues wrote in a research report published in Annals of Internal Medicine.
In this retrospective cohort study, the researchers linked data from 2011 to 2014 from the Medicare Provider Analysis and Review (MedPAR) file to the Healthcare Effectiveness Data and Information Set (HEDIS).
The novel linkage found that HEDIS data underreported hospital admissions for acute MI, heart failure, and pneumonia, the researchers stated. “Plans incorrectly excluded hospitalizations that should have qualified for the readmission measure, and readmission rates were substantially higher among incorrectly excluded hospitalizations.”
Despite this, in analyses using the linkage of HEDIS and MedPAR, “Medicare Advantage beneficiaries had higher 30-day risk-adjusted readmission rates after [acute MI, heart failure, and pneumonia] than did traditional Medicare beneficiaries,” the investigators noted.
Patients in Medicare Advantage had lower unadjusted readmission rates compared with those in traditional Medicare (16.6% vs. 17.1% for acute MI; 21.4% vs. 21.7% for heart failure; and 16.3% vs. 16.4% for pneumonia). After standardization, Medicare Advantage patients had higher readmission rates, compared with those in traditional Medicare (17.2% vs. 16.9% for acute MI; 21.7% vs. 21.4% for heart failure; and 16.5% vs. 16.0% for pneumonia).
The study authors added that, while unadjusted readmission rates were higher for traditional Medicare beneficiaries, “the direction of the difference reversed after standardization. This occurred because Medicare Advantage beneficiaries have, on average, a lower expected readmission risk [that is, they are ‘healthier’].” Prior studies have documented that Medicare Advantage plans enroll beneficiaries with fewer comorbid conditions and that high-cost beneficiaries switch out of Medicare Advantage and into traditional Medicare.
The researchers suggested four reasons for the differences between the results in this study versus others that compared patients using Medicare with those using Medicare Advantage. These were that the new study included a more comprehensive data set, analyses with comorbid conditions “from a well-validated model applied by CMS [Centers for Medicare & Medicaid Services],” national data focused on three conditions included in the Hospital Readmissions Reduction Program, and patients discharged to places other than skilled nursing facilities and inpatient rehabilitation facilities.
Authors of an accompanying editorial called for caution to be used in interpreting Medicare Advantage enrollment as causing an increased readmission risk.
“[The] results are sensitive to adjustment for case mix,” wrote Peter Huckfeldt, PhD, of the University of Minnesota, Minneapolis, and Neeraj Sood, PhD, of the University of Southern California, Los Angeles, in the editorial published in Annals of Internal Medicine (2019 June 25. doi:10.7326/M19-1599) “Using diagnosis codes on hospital claims for case-mix adjustments may be increasingly perilous. ... To our knowledge, there is no recent evidence comparing the intensity of diagnostic coding between clinically similar [traditional Medicare] and [Medicare Advantage] hospital admissions, but if [traditional Medicare] enrollees were coded more intensively than [Medicare Advantage] enrollees, this could lead to [traditional Medicare] enrollees having lower risk-adjusted readmission rares due to coding practices.”
The editorialists added that using a cross-sectional comparison of Medicare Advantage and traditional Medicare patients is concerning because a “key challenge in estimating the effect of [Medicare Advantage] is that enrollment is voluntary,” which can lead to a number of analytical concerns.
The researchers concluded that their findings “are concerning because CMS uses HEDIS performance to construct composite quality ratings and assign payment bonuses to Medicare Advantage plans.
“Our study suggests a need for improved monitoring of the accuracy of HEDIS data,” they noted.
The National Institute on Aging provided the primary funding for this study. A number of the authors received grants from the National Institutes of Health during the conduct of the study. No other relevant disclosures were reported.
SOURCE: Panagiotou OA et al. Ann Intern Med. 2019 Jun 25. doi: 10.7326/M18-1795.
FROM ANNALS OF INTERNAL MEDICINE
Trump administration seeks more health care cost details for consumers
Anyone who has tried to “shop” for hospital services knows one thing: It’s hard to get prices.
President Donald Trump on Monday signed an executive order he said would make it easier.
The order directs agencies to draw up rules requiring hospitals and insurers to make public more information on the negotiated prices they hammer out in contract negotiations. Also, hospitals and insurers would have to give estimates to patients on out-of-pocket costs before they go in for nonemergency medical care.
The move, which officials said will help address skyrocketing health care costs, comes amid other efforts by the administration to elicit more price transparency for medical care and initiatives by Congress to limit so-called surprise bills. These are the often-expensive bills consumers get when they unwittingly receive care that is not covered by their insurers.
“This will put American patients in control and address fundamental drivers of health care costs in a way no president has done before,” said Health & Human Services Secretary Alex Azar during a press briefing on Monday.
The proposal is likely to run into opposition from some hospitals and insurers who say disclosing negotiated rates could instead drive up costs.
Just how useful the effort will prove for consumers is unclear.
Much depends on how the administration writes the rules governing what information must be provided, such as whether it will include hospital-specific prices, regional averages, or other measures. While the administration calls for a “consumer-friendly” format, it’s not clear how such a massive amount of data – potentially negotiated price information from thousands of hospitals and insurers for tens of thousands of services – will be presented to consumers.
“It’s well intended, but may grossly overestimate the ability of the average patient to decipher this information overload,” said Dan Ward, a vice president at Waystar, a health care payments service.
So, does this new development advance efforts to better arm consumers with pricing information? Some key point to consider:
Q: What does the order do?
It may expand on price information consumers receive.
The order directs agencies to develop rules to require hospitals and insurers to provide information “based on negotiated rates” to the public.
Currently, such rates are hard to get, even for patients, until after medical care is provided. That’s when insured patients get an “explanation of benefits [EOBs],” which shows how much the hospital charged, how much of a discount their insurer received, and the amount a patient may owe.
In addition to consumers being unable to get price information upfront in many cases, hospital list prices and negotiated discount rates vary widely by hospital and insurer, even in a region. Uninsured patients often are charged the full amounts.
“People are sick and tired of hospitals playing these games with prices,” said George Nation, a business professor at Lehigh University in Bethlehem, Pa. who studies hospital contract law. “That’s what’s driving all of this.”
Some insurers and hospitals do provide online tools or apps that can help individual patients estimate out-of-pocket costs for a service or procedure ahead of time, but research shows few patients use such tools. Also, many medical services are needed without much notice – think of a heart attack or a broken leg – so shopping simply isn’t possible.
Administration officials say they want patients to have access to more information, including “advance EOBs” outlining anticipated costs before patients get nonemergency medical care. In theory, that would allow consumers to shop around for lower-cost care.
Q: Isn’t this information already available?
Not exactly. In January, new rules took effect under the Affordable Care Act that require hospitals to post online their “list prices,” which hospitals set themselves and have little relation to actual costs or what insurers actually pay.
What resulted are often confusing spreadsheets that contain thousands of a la carte charges – ranging from the price of medicines and sutures to room costs, among other things – that patients have to piece together if they can to estimate their total bill. Also, those list charges don’t reflect the discounted rates insurers have negotiated, so they are of little use to insured patients who might want to compare prices hospital to hospital.
The information that would result from President Trump’s executive order would provide more detail based on negotiated, discounted rates.
A senior administration official at the press briefing said details about whether the rates would be aggregated or relate to individual hospitals would be spelled out only when the administration puts forward proposed rules to implement the order later this year. It also is unclear how the administration would enforce the rules.
Another limitation: The order applies only to hospitals and the medical staff they employ. Many hospitals, however, are staffed by doctors who are not directly employed, or laboratories that are also separate. That means negotiated prices for services provided by such laboratories or physicians would not have to be disclosed.
Q: How could consumers use this information?
In theory, consumers could get information allowing them to compare prices for, say, a hip replacement or knee surgery in advance.
But that could prove difficult if the rates were not fairly hospital specific, or if they were not lumped in with all the care needed for a specific procedure or surgery.
“They could take the top 20 common procedures the hospital does, for example, and put negotiated prices on them,” said Mr. Nation. “It makes sense to do an average for that particular hospital, so I can see how much it’s going to cost to have my knee replaced at St. Joe’s versus St. Anne’s.”
Having advance notice of out-of-pocket costs could also help patients who have high-deductible plans.
“Patients are increasingly subject to insurance deductibles and other forms of substantial cost sharing. For a subset of so-called shoppable services, patients would benefit from price estimates in advance that allow them to compare options and plan financially for their care,” said John Rother, president and CEO at the advocacy group National Coalition on Health Care.
Q: Will this push consumers to shop for health care?
The short answer is maybe. Right now, it’s difficult, even with some of the tools available, said Lovisa Gustafsson, assistant vice president at the Commonwealth Fund, which has looked at whether patients use existing tools or the list price information hospitals must post online.
“The evidence to date shows patients aren’t necessarily the best shoppers, but we haven’t given them the best tools to be shoppers,” she said.
Posting negotiated rates might be a step forward, she said, but only if it is easily understandable.
It’s possible that insurers, physician offices, consumer groups, or online businesses may find ways to help direct patients to the most cost-effective locations for surgeries, tests or other procedures based on the information.
“Institutions like Consumer Reports or Consumer Checkbook could do some kind of high-level comparison between facilities or doctors, giving some general information that might be useful for consumers,” said Tim Jost, a professor emeritus at the Washington and Lee University School of Law in Lexington, Va.
But some hospitals and insurers maintain that disclosing specific rates could backfire.
Hospitals charging lower rates, for example, might raise them if they see competitors are getting higher reimbursement from insurers, they say. Insurers say they might be hampered in their ability to negotiate if rivals all know what they each pay.
“We also agree that patients should have accurate, real-time information about costs so they can make the best, most informed decisions about their care,” said a statement from lobbying group America’s Health Insurance Plans. “But publicly disclosing competitively negotiated, proprietary rates will reduce competition and push prices higher – not lower – for consumers, patients, and taxpayers.”
Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
Anyone who has tried to “shop” for hospital services knows one thing: It’s hard to get prices.
President Donald Trump on Monday signed an executive order he said would make it easier.
The order directs agencies to draw up rules requiring hospitals and insurers to make public more information on the negotiated prices they hammer out in contract negotiations. Also, hospitals and insurers would have to give estimates to patients on out-of-pocket costs before they go in for nonemergency medical care.
The move, which officials said will help address skyrocketing health care costs, comes amid other efforts by the administration to elicit more price transparency for medical care and initiatives by Congress to limit so-called surprise bills. These are the often-expensive bills consumers get when they unwittingly receive care that is not covered by their insurers.
“This will put American patients in control and address fundamental drivers of health care costs in a way no president has done before,” said Health & Human Services Secretary Alex Azar during a press briefing on Monday.
The proposal is likely to run into opposition from some hospitals and insurers who say disclosing negotiated rates could instead drive up costs.
Just how useful the effort will prove for consumers is unclear.
Much depends on how the administration writes the rules governing what information must be provided, such as whether it will include hospital-specific prices, regional averages, or other measures. While the administration calls for a “consumer-friendly” format, it’s not clear how such a massive amount of data – potentially negotiated price information from thousands of hospitals and insurers for tens of thousands of services – will be presented to consumers.
“It’s well intended, but may grossly overestimate the ability of the average patient to decipher this information overload,” said Dan Ward, a vice president at Waystar, a health care payments service.
So, does this new development advance efforts to better arm consumers with pricing information? Some key point to consider:
Q: What does the order do?
It may expand on price information consumers receive.
The order directs agencies to develop rules to require hospitals and insurers to provide information “based on negotiated rates” to the public.
Currently, such rates are hard to get, even for patients, until after medical care is provided. That’s when insured patients get an “explanation of benefits [EOBs],” which shows how much the hospital charged, how much of a discount their insurer received, and the amount a patient may owe.
In addition to consumers being unable to get price information upfront in many cases, hospital list prices and negotiated discount rates vary widely by hospital and insurer, even in a region. Uninsured patients often are charged the full amounts.
“People are sick and tired of hospitals playing these games with prices,” said George Nation, a business professor at Lehigh University in Bethlehem, Pa. who studies hospital contract law. “That’s what’s driving all of this.”
Some insurers and hospitals do provide online tools or apps that can help individual patients estimate out-of-pocket costs for a service or procedure ahead of time, but research shows few patients use such tools. Also, many medical services are needed without much notice – think of a heart attack or a broken leg – so shopping simply isn’t possible.
Administration officials say they want patients to have access to more information, including “advance EOBs” outlining anticipated costs before patients get nonemergency medical care. In theory, that would allow consumers to shop around for lower-cost care.
Q: Isn’t this information already available?
Not exactly. In January, new rules took effect under the Affordable Care Act that require hospitals to post online their “list prices,” which hospitals set themselves and have little relation to actual costs or what insurers actually pay.
What resulted are often confusing spreadsheets that contain thousands of a la carte charges – ranging from the price of medicines and sutures to room costs, among other things – that patients have to piece together if they can to estimate their total bill. Also, those list charges don’t reflect the discounted rates insurers have negotiated, so they are of little use to insured patients who might want to compare prices hospital to hospital.
The information that would result from President Trump’s executive order would provide more detail based on negotiated, discounted rates.
A senior administration official at the press briefing said details about whether the rates would be aggregated or relate to individual hospitals would be spelled out only when the administration puts forward proposed rules to implement the order later this year. It also is unclear how the administration would enforce the rules.
Another limitation: The order applies only to hospitals and the medical staff they employ. Many hospitals, however, are staffed by doctors who are not directly employed, or laboratories that are also separate. That means negotiated prices for services provided by such laboratories or physicians would not have to be disclosed.
Q: How could consumers use this information?
In theory, consumers could get information allowing them to compare prices for, say, a hip replacement or knee surgery in advance.
But that could prove difficult if the rates were not fairly hospital specific, or if they were not lumped in with all the care needed for a specific procedure or surgery.
“They could take the top 20 common procedures the hospital does, for example, and put negotiated prices on them,” said Mr. Nation. “It makes sense to do an average for that particular hospital, so I can see how much it’s going to cost to have my knee replaced at St. Joe’s versus St. Anne’s.”
Having advance notice of out-of-pocket costs could also help patients who have high-deductible plans.
“Patients are increasingly subject to insurance deductibles and other forms of substantial cost sharing. For a subset of so-called shoppable services, patients would benefit from price estimates in advance that allow them to compare options and plan financially for their care,” said John Rother, president and CEO at the advocacy group National Coalition on Health Care.
Q: Will this push consumers to shop for health care?
The short answer is maybe. Right now, it’s difficult, even with some of the tools available, said Lovisa Gustafsson, assistant vice president at the Commonwealth Fund, which has looked at whether patients use existing tools or the list price information hospitals must post online.
“The evidence to date shows patients aren’t necessarily the best shoppers, but we haven’t given them the best tools to be shoppers,” she said.
Posting negotiated rates might be a step forward, she said, but only if it is easily understandable.
It’s possible that insurers, physician offices, consumer groups, or online businesses may find ways to help direct patients to the most cost-effective locations for surgeries, tests or other procedures based on the information.
“Institutions like Consumer Reports or Consumer Checkbook could do some kind of high-level comparison between facilities or doctors, giving some general information that might be useful for consumers,” said Tim Jost, a professor emeritus at the Washington and Lee University School of Law in Lexington, Va.
But some hospitals and insurers maintain that disclosing specific rates could backfire.
Hospitals charging lower rates, for example, might raise them if they see competitors are getting higher reimbursement from insurers, they say. Insurers say they might be hampered in their ability to negotiate if rivals all know what they each pay.
“We also agree that patients should have accurate, real-time information about costs so they can make the best, most informed decisions about their care,” said a statement from lobbying group America’s Health Insurance Plans. “But publicly disclosing competitively negotiated, proprietary rates will reduce competition and push prices higher – not lower – for consumers, patients, and taxpayers.”
Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
Anyone who has tried to “shop” for hospital services knows one thing: It’s hard to get prices.
President Donald Trump on Monday signed an executive order he said would make it easier.
The order directs agencies to draw up rules requiring hospitals and insurers to make public more information on the negotiated prices they hammer out in contract negotiations. Also, hospitals and insurers would have to give estimates to patients on out-of-pocket costs before they go in for nonemergency medical care.
The move, which officials said will help address skyrocketing health care costs, comes amid other efforts by the administration to elicit more price transparency for medical care and initiatives by Congress to limit so-called surprise bills. These are the often-expensive bills consumers get when they unwittingly receive care that is not covered by their insurers.
“This will put American patients in control and address fundamental drivers of health care costs in a way no president has done before,” said Health & Human Services Secretary Alex Azar during a press briefing on Monday.
The proposal is likely to run into opposition from some hospitals and insurers who say disclosing negotiated rates could instead drive up costs.
Just how useful the effort will prove for consumers is unclear.
Much depends on how the administration writes the rules governing what information must be provided, such as whether it will include hospital-specific prices, regional averages, or other measures. While the administration calls for a “consumer-friendly” format, it’s not clear how such a massive amount of data – potentially negotiated price information from thousands of hospitals and insurers for tens of thousands of services – will be presented to consumers.
“It’s well intended, but may grossly overestimate the ability of the average patient to decipher this information overload,” said Dan Ward, a vice president at Waystar, a health care payments service.
So, does this new development advance efforts to better arm consumers with pricing information? Some key point to consider:
Q: What does the order do?
It may expand on price information consumers receive.
The order directs agencies to develop rules to require hospitals and insurers to provide information “based on negotiated rates” to the public.
Currently, such rates are hard to get, even for patients, until after medical care is provided. That’s when insured patients get an “explanation of benefits [EOBs],” which shows how much the hospital charged, how much of a discount their insurer received, and the amount a patient may owe.
In addition to consumers being unable to get price information upfront in many cases, hospital list prices and negotiated discount rates vary widely by hospital and insurer, even in a region. Uninsured patients often are charged the full amounts.
“People are sick and tired of hospitals playing these games with prices,” said George Nation, a business professor at Lehigh University in Bethlehem, Pa. who studies hospital contract law. “That’s what’s driving all of this.”
Some insurers and hospitals do provide online tools or apps that can help individual patients estimate out-of-pocket costs for a service or procedure ahead of time, but research shows few patients use such tools. Also, many medical services are needed without much notice – think of a heart attack or a broken leg – so shopping simply isn’t possible.
Administration officials say they want patients to have access to more information, including “advance EOBs” outlining anticipated costs before patients get nonemergency medical care. In theory, that would allow consumers to shop around for lower-cost care.
Q: Isn’t this information already available?
Not exactly. In January, new rules took effect under the Affordable Care Act that require hospitals to post online their “list prices,” which hospitals set themselves and have little relation to actual costs or what insurers actually pay.
What resulted are often confusing spreadsheets that contain thousands of a la carte charges – ranging from the price of medicines and sutures to room costs, among other things – that patients have to piece together if they can to estimate their total bill. Also, those list charges don’t reflect the discounted rates insurers have negotiated, so they are of little use to insured patients who might want to compare prices hospital to hospital.
The information that would result from President Trump’s executive order would provide more detail based on negotiated, discounted rates.
A senior administration official at the press briefing said details about whether the rates would be aggregated or relate to individual hospitals would be spelled out only when the administration puts forward proposed rules to implement the order later this year. It also is unclear how the administration would enforce the rules.
Another limitation: The order applies only to hospitals and the medical staff they employ. Many hospitals, however, are staffed by doctors who are not directly employed, or laboratories that are also separate. That means negotiated prices for services provided by such laboratories or physicians would not have to be disclosed.
Q: How could consumers use this information?
In theory, consumers could get information allowing them to compare prices for, say, a hip replacement or knee surgery in advance.
But that could prove difficult if the rates were not fairly hospital specific, or if they were not lumped in with all the care needed for a specific procedure or surgery.
“They could take the top 20 common procedures the hospital does, for example, and put negotiated prices on them,” said Mr. Nation. “It makes sense to do an average for that particular hospital, so I can see how much it’s going to cost to have my knee replaced at St. Joe’s versus St. Anne’s.”
Having advance notice of out-of-pocket costs could also help patients who have high-deductible plans.
“Patients are increasingly subject to insurance deductibles and other forms of substantial cost sharing. For a subset of so-called shoppable services, patients would benefit from price estimates in advance that allow them to compare options and plan financially for their care,” said John Rother, president and CEO at the advocacy group National Coalition on Health Care.
Q: Will this push consumers to shop for health care?
The short answer is maybe. Right now, it’s difficult, even with some of the tools available, said Lovisa Gustafsson, assistant vice president at the Commonwealth Fund, which has looked at whether patients use existing tools or the list price information hospitals must post online.
“The evidence to date shows patients aren’t necessarily the best shoppers, but we haven’t given them the best tools to be shoppers,” she said.
Posting negotiated rates might be a step forward, she said, but only if it is easily understandable.
It’s possible that insurers, physician offices, consumer groups, or online businesses may find ways to help direct patients to the most cost-effective locations for surgeries, tests or other procedures based on the information.
“Institutions like Consumer Reports or Consumer Checkbook could do some kind of high-level comparison between facilities or doctors, giving some general information that might be useful for consumers,” said Tim Jost, a professor emeritus at the Washington and Lee University School of Law in Lexington, Va.
But some hospitals and insurers maintain that disclosing specific rates could backfire.
Hospitals charging lower rates, for example, might raise them if they see competitors are getting higher reimbursement from insurers, they say. Insurers say they might be hampered in their ability to negotiate if rivals all know what they each pay.
“We also agree that patients should have accurate, real-time information about costs so they can make the best, most informed decisions about their care,” said a statement from lobbying group America’s Health Insurance Plans. “But publicly disclosing competitively negotiated, proprietary rates will reduce competition and push prices higher – not lower – for consumers, patients, and taxpayers.”
Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.
Highlights of the 2019 Update to the Global Initiative for Chronic Obstructive Lung Disease (GOLD) Report and Their Application in Practice
Click here to read the supplement
Key Points
- The Global Initiative for Chronic Obstructive Lung Disease (GOLD) program releases consensus reports to provide evidence-based recommendations about the management and prevention of chronic obstructive pulmonary disease (COPD);
- The most recent major update occurred in November 2018.
- The 2019 GOLD strategy for COPD assessment now separates lung function measures from respiratory symptom scores because lung function is only weakly correlated to symptoms and health status impairment.
- The updated strategy now provides recommendations for initiation and maintenance of pharmacologic therapy in patients based on exacerbation risk and symptom scores, with consideration of blood eosinophil counts.
- Management of exacerbations now includes the prevention of future exacerbations, which may require adding another medication to the patient's maintenance regimen.
About the Authors:
Jennifer Banfield, APRN, FNP
Clinical Research Coordinator
Boys Town National Research Hospital
Boys Town, Nebraska
Kevin R. Murphy, MD
Director of Allergy, Asthma, and Pulmonary Research
Boys Town National Research Hospital
Boys Town, Nebraska
University of Nebraska Medical Center
Creighton University School of Medicine
Omaha, Nebraska
Click here to read the supplement
Key Points
- The Global Initiative for Chronic Obstructive Lung Disease (GOLD) program releases consensus reports to provide evidence-based recommendations about the management and prevention of chronic obstructive pulmonary disease (COPD);
- The most recent major update occurred in November 2018.
- The 2019 GOLD strategy for COPD assessment now separates lung function measures from respiratory symptom scores because lung function is only weakly correlated to symptoms and health status impairment.
- The updated strategy now provides recommendations for initiation and maintenance of pharmacologic therapy in patients based on exacerbation risk and symptom scores, with consideration of blood eosinophil counts.
- Management of exacerbations now includes the prevention of future exacerbations, which may require adding another medication to the patient's maintenance regimen.
About the Authors:
Jennifer Banfield, APRN, FNP
Clinical Research Coordinator
Boys Town National Research Hospital
Boys Town, Nebraska
Kevin R. Murphy, MD
Director of Allergy, Asthma, and Pulmonary Research
Boys Town National Research Hospital
Boys Town, Nebraska
University of Nebraska Medical Center
Creighton University School of Medicine
Omaha, Nebraska
Click here to read the supplement
Key Points
- The Global Initiative for Chronic Obstructive Lung Disease (GOLD) program releases consensus reports to provide evidence-based recommendations about the management and prevention of chronic obstructive pulmonary disease (COPD);
- The most recent major update occurred in November 2018.
- The 2019 GOLD strategy for COPD assessment now separates lung function measures from respiratory symptom scores because lung function is only weakly correlated to symptoms and health status impairment.
- The updated strategy now provides recommendations for initiation and maintenance of pharmacologic therapy in patients based on exacerbation risk and symptom scores, with consideration of blood eosinophil counts.
- Management of exacerbations now includes the prevention of future exacerbations, which may require adding another medication to the patient's maintenance regimen.
About the Authors:
Jennifer Banfield, APRN, FNP
Clinical Research Coordinator
Boys Town National Research Hospital
Boys Town, Nebraska
Kevin R. Murphy, MD
Director of Allergy, Asthma, and Pulmonary Research
Boys Town National Research Hospital
Boys Town, Nebraska
University of Nebraska Medical Center
Creighton University School of Medicine
Omaha, Nebraska
Penicillin-susceptible Streptococcus pneumoniae most common cause of bacteremic CAP
A study found that only 2% of children hospitalized with community-acquired pneumonia (CAP) actually had any causative pathogen in their blood culture results, despite national guidelines that recommend blood cultures for all children hospitalized with moderate to severe CAP.
The guidelines are the 2011 guidelines for managing CAP published by the Pediatric Infectious Diseases Society (PIDS) and the Infectious Diseases Society of America (IDSA) (Clin Infect Dis. 2011 Oct;53[7]:617-30).
Cristin O. Fritz, MD, of the Children’s Hospital of Colorado, Aurora, and associates conducted a data analysis of the EPIC (Etiology of Pneumonia in the Community) study to estimate prevalence, risk factors, and clinical outcomes in children hospitalized with bacteremic CAP and to evaluate the relationship between positive blood culture results, empirical antibiotics, and changes in antibiotic treatment regimens.
Data were collected at two Tennessee hospitals and one Utah hospital during Jan. 1, 2010–June 30, 2012. Of the 2,358 children with CAP enrolled in the study, 2,143 (91%) with blood cultures were included in Dr. Fritz’s analysis. Of the 53 patients presenting with positive blood culture results, 46 (2%; 95% confidence interval: 1.6%-2.9%) were identified as having bacteremia. Half of all cases observed were caused by Streptococcus pneumoniae, with Staphylococcus aureus and Streptococcus pyogenes noted less frequently, according to the study published in Pediatrics.
A previous meta-analysis of smaller studies also found that children with CAP rarely had positive blood culture results, a pooled prevalence of 5% (Pediatr Infect Dis J. 2013;32[7]:736-40). Although it is believed that positive blood culture results are key to narrowing the choice of antibiotic and predicting treatment outcomes, the literature – to date – reveals a paucity of data supporting this assumption.
Overall, children in the study presenting with bacteremia experienced more severe clinical outcomes, including longer length of stay, greater likelihood of ICU admission, and invasive mechanical ventilation and/or shock. The authors also observed that bacteremia was less likely to be detected in children given antibiotics after admission but before cultures were obtained (0.8% vs 3%; P = .021). Pleural effusion detected with chest radiograph also consistently indicated bacteremic pneumonia, an observation made within this and other similar studies.
Also of note in detection is the biomarker procalcitonin, which is typically present with bacterial disease. Dr. Fritz and colleagues stressed that because the procalcitonin rate was higher in patients presenting with bacteremia, “this information could influence decisions around culturing if results are rapidly available.” Risk-stratification tools also might serve a valuable purpose in ferreting out those patients presenting with moderate to severe pneumonia most at increased risk for bacterial CAP.
Compared with other studies reporting prevalence ranges of 1%-7%, the prevalence of bacteremia in this study is lower at 2%. The authors attributed the difference to a possible potential limitation with the other studies, for which culture data was only available for a median 47% of enrollees. Dr. Fritz and her colleagues caution that “because cultures were obtained at the discretion of the treating clinician in a majority of studies, blood cultures were likely obtained more often in those with more severe illness or who had not already received antibiotics.” In this scenario, the likelihood that prevalence of bacteremia was overestimated is noteworthy.
The authors observed that penicillin-susceptible S. pneumonia was the most common cause of bacteremic CAP. They further acknowledged that their study and findings by Neuman et al. in 2017 give credence to the joint 2011 PIDS/IDSA guideline recommending narrow-spectrum aminopenicillins specifically to treat children hospitalized due to suspected bacterial CAP.
Despite its small sample size, the results of this study clearly demonstrate that children with bacteremia because of S. pyogenes or S. aureus experience increased morbidity, compared with children with S. pneumoniae, they said
While this is acknowledged to be one of the largest studies of its kind to date, a key limitation was the small number of observable patients with bacteremia, which prevented the researchers from conducting a more in-depth analysis of risk factors and pathogen-specific differences. That one-fourth of patients received in-patient antibiotics before cultures could be collected also likely led to an underestimation of risk factors and misclassification bias. Lastly, the use of blood culture instead of whole-blood polymerase chain reaction, which is known to be more sensitive, also may have led to underestimation of overall bacteremia prevalence.
“In an era with widespread pneumococcal vaccination and low prevalence of bacteremia in the United States, noted Dr. Fritz and associates.
Dr. Fritz had no conflicts of interest to report. Some coauthors cited multiple sources of potential conflict of interest related to consulting fees, grant support, and research support from various pharmaceutical companies and agencies. The study was funded by the National Institutes of Health and in part by a grant from the National Institute of Allergy and Infectious Diseases.
SOURCE: Fritz C et al. Pediatrics. 2019;144(1):e20183090.
A study found that only 2% of children hospitalized with community-acquired pneumonia (CAP) actually had any causative pathogen in their blood culture results, despite national guidelines that recommend blood cultures for all children hospitalized with moderate to severe CAP.
The guidelines are the 2011 guidelines for managing CAP published by the Pediatric Infectious Diseases Society (PIDS) and the Infectious Diseases Society of America (IDSA) (Clin Infect Dis. 2011 Oct;53[7]:617-30).
Cristin O. Fritz, MD, of the Children’s Hospital of Colorado, Aurora, and associates conducted a data analysis of the EPIC (Etiology of Pneumonia in the Community) study to estimate prevalence, risk factors, and clinical outcomes in children hospitalized with bacteremic CAP and to evaluate the relationship between positive blood culture results, empirical antibiotics, and changes in antibiotic treatment regimens.
Data were collected at two Tennessee hospitals and one Utah hospital during Jan. 1, 2010–June 30, 2012. Of the 2,358 children with CAP enrolled in the study, 2,143 (91%) with blood cultures were included in Dr. Fritz’s analysis. Of the 53 patients presenting with positive blood culture results, 46 (2%; 95% confidence interval: 1.6%-2.9%) were identified as having bacteremia. Half of all cases observed were caused by Streptococcus pneumoniae, with Staphylococcus aureus and Streptococcus pyogenes noted less frequently, according to the study published in Pediatrics.
A previous meta-analysis of smaller studies also found that children with CAP rarely had positive blood culture results, a pooled prevalence of 5% (Pediatr Infect Dis J. 2013;32[7]:736-40). Although it is believed that positive blood culture results are key to narrowing the choice of antibiotic and predicting treatment outcomes, the literature – to date – reveals a paucity of data supporting this assumption.
Overall, children in the study presenting with bacteremia experienced more severe clinical outcomes, including longer length of stay, greater likelihood of ICU admission, and invasive mechanical ventilation and/or shock. The authors also observed that bacteremia was less likely to be detected in children given antibiotics after admission but before cultures were obtained (0.8% vs 3%; P = .021). Pleural effusion detected with chest radiograph also consistently indicated bacteremic pneumonia, an observation made within this and other similar studies.
Also of note in detection is the biomarker procalcitonin, which is typically present with bacterial disease. Dr. Fritz and colleagues stressed that because the procalcitonin rate was higher in patients presenting with bacteremia, “this information could influence decisions around culturing if results are rapidly available.” Risk-stratification tools also might serve a valuable purpose in ferreting out those patients presenting with moderate to severe pneumonia most at increased risk for bacterial CAP.
Compared with other studies reporting prevalence ranges of 1%-7%, the prevalence of bacteremia in this study is lower at 2%. The authors attributed the difference to a possible potential limitation with the other studies, for which culture data was only available for a median 47% of enrollees. Dr. Fritz and her colleagues caution that “because cultures were obtained at the discretion of the treating clinician in a majority of studies, blood cultures were likely obtained more often in those with more severe illness or who had not already received antibiotics.” In this scenario, the likelihood that prevalence of bacteremia was overestimated is noteworthy.
The authors observed that penicillin-susceptible S. pneumonia was the most common cause of bacteremic CAP. They further acknowledged that their study and findings by Neuman et al. in 2017 give credence to the joint 2011 PIDS/IDSA guideline recommending narrow-spectrum aminopenicillins specifically to treat children hospitalized due to suspected bacterial CAP.
Despite its small sample size, the results of this study clearly demonstrate that children with bacteremia because of S. pyogenes or S. aureus experience increased morbidity, compared with children with S. pneumoniae, they said
While this is acknowledged to be one of the largest studies of its kind to date, a key limitation was the small number of observable patients with bacteremia, which prevented the researchers from conducting a more in-depth analysis of risk factors and pathogen-specific differences. That one-fourth of patients received in-patient antibiotics before cultures could be collected also likely led to an underestimation of risk factors and misclassification bias. Lastly, the use of blood culture instead of whole-blood polymerase chain reaction, which is known to be more sensitive, also may have led to underestimation of overall bacteremia prevalence.
“In an era with widespread pneumococcal vaccination and low prevalence of bacteremia in the United States, noted Dr. Fritz and associates.
Dr. Fritz had no conflicts of interest to report. Some coauthors cited multiple sources of potential conflict of interest related to consulting fees, grant support, and research support from various pharmaceutical companies and agencies. The study was funded by the National Institutes of Health and in part by a grant from the National Institute of Allergy and Infectious Diseases.
SOURCE: Fritz C et al. Pediatrics. 2019;144(1):e20183090.
A study found that only 2% of children hospitalized with community-acquired pneumonia (CAP) actually had any causative pathogen in their blood culture results, despite national guidelines that recommend blood cultures for all children hospitalized with moderate to severe CAP.
The guidelines are the 2011 guidelines for managing CAP published by the Pediatric Infectious Diseases Society (PIDS) and the Infectious Diseases Society of America (IDSA) (Clin Infect Dis. 2011 Oct;53[7]:617-30).
Cristin O. Fritz, MD, of the Children’s Hospital of Colorado, Aurora, and associates conducted a data analysis of the EPIC (Etiology of Pneumonia in the Community) study to estimate prevalence, risk factors, and clinical outcomes in children hospitalized with bacteremic CAP and to evaluate the relationship between positive blood culture results, empirical antibiotics, and changes in antibiotic treatment regimens.
Data were collected at two Tennessee hospitals and one Utah hospital during Jan. 1, 2010–June 30, 2012. Of the 2,358 children with CAP enrolled in the study, 2,143 (91%) with blood cultures were included in Dr. Fritz’s analysis. Of the 53 patients presenting with positive blood culture results, 46 (2%; 95% confidence interval: 1.6%-2.9%) were identified as having bacteremia. Half of all cases observed were caused by Streptococcus pneumoniae, with Staphylococcus aureus and Streptococcus pyogenes noted less frequently, according to the study published in Pediatrics.
A previous meta-analysis of smaller studies also found that children with CAP rarely had positive blood culture results, a pooled prevalence of 5% (Pediatr Infect Dis J. 2013;32[7]:736-40). Although it is believed that positive blood culture results are key to narrowing the choice of antibiotic and predicting treatment outcomes, the literature – to date – reveals a paucity of data supporting this assumption.
Overall, children in the study presenting with bacteremia experienced more severe clinical outcomes, including longer length of stay, greater likelihood of ICU admission, and invasive mechanical ventilation and/or shock. The authors also observed that bacteremia was less likely to be detected in children given antibiotics after admission but before cultures were obtained (0.8% vs 3%; P = .021). Pleural effusion detected with chest radiograph also consistently indicated bacteremic pneumonia, an observation made within this and other similar studies.
Also of note in detection is the biomarker procalcitonin, which is typically present with bacterial disease. Dr. Fritz and colleagues stressed that because the procalcitonin rate was higher in patients presenting with bacteremia, “this information could influence decisions around culturing if results are rapidly available.” Risk-stratification tools also might serve a valuable purpose in ferreting out those patients presenting with moderate to severe pneumonia most at increased risk for bacterial CAP.
Compared with other studies reporting prevalence ranges of 1%-7%, the prevalence of bacteremia in this study is lower at 2%. The authors attributed the difference to a possible potential limitation with the other studies, for which culture data was only available for a median 47% of enrollees. Dr. Fritz and her colleagues caution that “because cultures were obtained at the discretion of the treating clinician in a majority of studies, blood cultures were likely obtained more often in those with more severe illness or who had not already received antibiotics.” In this scenario, the likelihood that prevalence of bacteremia was overestimated is noteworthy.
The authors observed that penicillin-susceptible S. pneumonia was the most common cause of bacteremic CAP. They further acknowledged that their study and findings by Neuman et al. in 2017 give credence to the joint 2011 PIDS/IDSA guideline recommending narrow-spectrum aminopenicillins specifically to treat children hospitalized due to suspected bacterial CAP.
Despite its small sample size, the results of this study clearly demonstrate that children with bacteremia because of S. pyogenes or S. aureus experience increased morbidity, compared with children with S. pneumoniae, they said
While this is acknowledged to be one of the largest studies of its kind to date, a key limitation was the small number of observable patients with bacteremia, which prevented the researchers from conducting a more in-depth analysis of risk factors and pathogen-specific differences. That one-fourth of patients received in-patient antibiotics before cultures could be collected also likely led to an underestimation of risk factors and misclassification bias. Lastly, the use of blood culture instead of whole-blood polymerase chain reaction, which is known to be more sensitive, also may have led to underestimation of overall bacteremia prevalence.
“In an era with widespread pneumococcal vaccination and low prevalence of bacteremia in the United States, noted Dr. Fritz and associates.
Dr. Fritz had no conflicts of interest to report. Some coauthors cited multiple sources of potential conflict of interest related to consulting fees, grant support, and research support from various pharmaceutical companies and agencies. The study was funded by the National Institutes of Health and in part by a grant from the National Institute of Allergy and Infectious Diseases.
SOURCE: Fritz C et al. Pediatrics. 2019;144(1):e20183090.
FROM PEDIATRICS
New single-dose influenza therapy effective among outpatients
Clinical question: Is baloxavir marboxil, a selective inhibitor of influenza cap-dependent endonuclease, a safe and effective treatment for acute uncomplicated influenza?
Background: The emergence of oseltamivir-resistant influenza A(H1NI) infection in 2007 highlights the risk of future neuraminidase-resistant global pandemics. Baloxavir represents a new class of antiviral agent that may help treat such outbreaks.
Study design: Phase 3 randomized, double-blind, placebo-controlled trial.
Setting: Outpatients in the United States and Japan.
Synopsis: The trial recruited 1,436 otherwise healthy patients aged 12-64 years of age (median age, 33 years) with a clinical diagnosis of acute uncomplicated influenza pneumonia. The patients were randomly assigned to receive either a single dose of oral baloxavir, oseltamivir 75 mg twice daily for 5 days, or matching placebo within 48 hours of symptom onset. The primary outcome was patient self-assessment of symptomatology.
Among the 1,064 adult patients (age 20-64) with influenza diagnosis confirmed by reverse transcription polymerase chain reaction (RT-PCR), the median time to alleviation of symptoms was lower in the baloxavir group than it was in the placebo group (53.7 hours vs. 80.2 hours; P less than .001). There was no significant difference in time to alleviation of symptoms in the baloxavir group when compared with the oseltamivir group. Adverse events were reported in 21% of baloxavir patients, 25% of placebo patients, and 25% of oseltamivir patients.
The enrolled patients were predominantly young, healthy, and treated as an outpatient. Patients hospitalized with influenza pneumonia are often older, have significant comorbidities, and are at higher risk of poor outcomes. This trial does not directly support the safety or efficacy of baloxavir in this population.
Bottom line: A single dose of baloxavir provides similar clinical benefit as 5 days of oseltamivir therapy in the early treatment of healthy patients with acute influenza.
Citation: Hayden FG et al. Baloxavir marboxil for uncomplicated influenza in adults and adolescents. N Eng J Med. 2018:379(10):914-23.
Dr. Holzer is an assistant professor of medicine in the division of hospital medicine at Mount Sinai Hospital, New York.
Clinical question: Is baloxavir marboxil, a selective inhibitor of influenza cap-dependent endonuclease, a safe and effective treatment for acute uncomplicated influenza?
Background: The emergence of oseltamivir-resistant influenza A(H1NI) infection in 2007 highlights the risk of future neuraminidase-resistant global pandemics. Baloxavir represents a new class of antiviral agent that may help treat such outbreaks.
Study design: Phase 3 randomized, double-blind, placebo-controlled trial.
Setting: Outpatients in the United States and Japan.
Synopsis: The trial recruited 1,436 otherwise healthy patients aged 12-64 years of age (median age, 33 years) with a clinical diagnosis of acute uncomplicated influenza pneumonia. The patients were randomly assigned to receive either a single dose of oral baloxavir, oseltamivir 75 mg twice daily for 5 days, or matching placebo within 48 hours of symptom onset. The primary outcome was patient self-assessment of symptomatology.
Among the 1,064 adult patients (age 20-64) with influenza diagnosis confirmed by reverse transcription polymerase chain reaction (RT-PCR), the median time to alleviation of symptoms was lower in the baloxavir group than it was in the placebo group (53.7 hours vs. 80.2 hours; P less than .001). There was no significant difference in time to alleviation of symptoms in the baloxavir group when compared with the oseltamivir group. Adverse events were reported in 21% of baloxavir patients, 25% of placebo patients, and 25% of oseltamivir patients.
The enrolled patients were predominantly young, healthy, and treated as an outpatient. Patients hospitalized with influenza pneumonia are often older, have significant comorbidities, and are at higher risk of poor outcomes. This trial does not directly support the safety or efficacy of baloxavir in this population.
Bottom line: A single dose of baloxavir provides similar clinical benefit as 5 days of oseltamivir therapy in the early treatment of healthy patients with acute influenza.
Citation: Hayden FG et al. Baloxavir marboxil for uncomplicated influenza in adults and adolescents. N Eng J Med. 2018:379(10):914-23.
Dr. Holzer is an assistant professor of medicine in the division of hospital medicine at Mount Sinai Hospital, New York.
Clinical question: Is baloxavir marboxil, a selective inhibitor of influenza cap-dependent endonuclease, a safe and effective treatment for acute uncomplicated influenza?
Background: The emergence of oseltamivir-resistant influenza A(H1NI) infection in 2007 highlights the risk of future neuraminidase-resistant global pandemics. Baloxavir represents a new class of antiviral agent that may help treat such outbreaks.
Study design: Phase 3 randomized, double-blind, placebo-controlled trial.
Setting: Outpatients in the United States and Japan.
Synopsis: The trial recruited 1,436 otherwise healthy patients aged 12-64 years of age (median age, 33 years) with a clinical diagnosis of acute uncomplicated influenza pneumonia. The patients were randomly assigned to receive either a single dose of oral baloxavir, oseltamivir 75 mg twice daily for 5 days, or matching placebo within 48 hours of symptom onset. The primary outcome was patient self-assessment of symptomatology.
Among the 1,064 adult patients (age 20-64) with influenza diagnosis confirmed by reverse transcription polymerase chain reaction (RT-PCR), the median time to alleviation of symptoms was lower in the baloxavir group than it was in the placebo group (53.7 hours vs. 80.2 hours; P less than .001). There was no significant difference in time to alleviation of symptoms in the baloxavir group when compared with the oseltamivir group. Adverse events were reported in 21% of baloxavir patients, 25% of placebo patients, and 25% of oseltamivir patients.
The enrolled patients were predominantly young, healthy, and treated as an outpatient. Patients hospitalized with influenza pneumonia are often older, have significant comorbidities, and are at higher risk of poor outcomes. This trial does not directly support the safety or efficacy of baloxavir in this population.
Bottom line: A single dose of baloxavir provides similar clinical benefit as 5 days of oseltamivir therapy in the early treatment of healthy patients with acute influenza.
Citation: Hayden FG et al. Baloxavir marboxil for uncomplicated influenza in adults and adolescents. N Eng J Med. 2018:379(10):914-23.
Dr. Holzer is an assistant professor of medicine in the division of hospital medicine at Mount Sinai Hospital, New York.
Elevated monocyte count predicts poor outcomes in idiopathic pulmonary fibrosis
, including hypertrophic cardiomyopathy, systemic sclerosis, and myelofibrosis, according to research published in The Lancet Respiratory Medicine.
The data indicate that “a single threshold value of absolute monocyte counts of 0.95 K/mcL could be used to identify high-risk patients with a fibrotic disease,” said Madeleine K. D. Scott, a researcher at Stanford (Calif.) University, and coauthors. The results “suggest that monocyte count should be incorporated into the clinical assessment” and may “enable more conscientious allocation of scarce resources, including lung transplantations,” they said.
While other published biomarkers – including gene panels and multicytokine signatures – may be expensive and not readily available, “absolute monocyte count is routinely measured as part of a complete blood count, an inexpensive test used in clinical practice worldwide,” the authors said.
Further study of monocytes’ mechanistic role in fibrosis ultimately could point to new treatment approaches.
A retrospective multicenter cohort study
To assess whether immune cells may identify patients with idiopathic pulmonary fibrosis at greater risk of poor outcomes, Ms. Scott and her collaborators conducted a retrospective multicenter cohort study.
They first analyzed transcriptome data from 120 peripheral blood mononuclear cell samples of patients with idiopathic pulmonary fibrosis, which they obtained from the Gene Expression Omnibus at the National Center for Biotechnology Information. They used statistical deconvolution to estimate percentages of 13 immune cell types and examined their associations with transplant-free survival. Their discovery analysis found that estimated CD14+ classical monocyte percentages above the mean correlated with shorter transplant-free survival times (hazard ratio, 1.82), but percentages of T cells and B cells did not.
The researchers then validated these results using samples from patients with idiopathic pulmonary fibrosis in two independent cohorts. In the COMET validation cohort, which included 45 patients with idiopathic pulmonary fibrosis whose monocyte counts were measured using flow cytometry, higher monocyte counts were significantly associated with greater risk of disease progression. In the Yale cohort, which included 15 patients with idiopathic pulmonary fibrosis, the 6 patients who were classified as high risk on the basis of a 52-gene signature had more CD14+ monocytes than the 9 low-risk patients did.
In addition, Ms. Scott and her collaborators looked at complete blood count values in the electronic health records of 45,068 patients with idiopathic pulmonary fibrosis, systemic sclerosis, hypertrophic cardiomyopathy, or myelofibrosis in Stanford, Northwestern, Vanderbilt, and Optum Clinformatics Data Mart cohorts.
Among patients in the COMET, Stanford, and Northwestern datasets, monocyte counts of 0.95 K/mcL or greater were associated with mortality after adjustment for forced vital capacity (HR, 2.47) and the gender, age, and physiology index (HR, 2.06). Data from 7,459 patients with idiopathic pulmonary fibrosis “showed that patients with monocyte counts of 0.95 K/mcL or greater were at increased risk of mortality with lung transplantation as a censoring event, after adjusting for age at diagnosis and sex” in the Stanford (HR, 2.30), Vanderbilt (HR, 1.52), and Optum (HR, 1.74) cohorts. “Likewise, higher absolute monocyte count was associated with shortened survival in patients with hypertrophic cardiomyopathy across all three cohorts, and in patients with systemic sclerosis or myelofibrosis in two of the three cohorts,” the researchers said.
The study was funded by grants from the Bill & Melinda Gates Foundation, U.S. National Institute of Allergy and Infectious Diseases, and U.S. National Library of Medicine. Ms. Scott had no competing interests. Coauthors disclosed grants, compensation, and support from foundations, agencies, and companies.
SOURCE: Scott MKD et al. Lancet Respir Med. 2019 Jun. doi: 10.1016/S2213-2600(18)30508-3.
The study by Scott et al. provides evidence that monocyte count may be a “novel, simple, and inexpensive prognostic biomarker in idiopathic pulmonary fibrosis,” according to an accompanying editorial.
Progress has been made in the treatment of idiopathic pulmonary fibrosis, but patient prognosis remains “challenging to predict,” wrote Michael Kreuter, MD, of University of Heidelberg, Germany, and Toby M. Maher, MB, MSc, PhD, of Royal Brompton Hospital in London and Imperial College London. “One lesson that can be learned from other respiratory disorders is that routinely measured cellular biomarkers, such as blood eosinophil counts in chronic obstructive pulmonary disease (COPD), can predict treatment responses” (Lancet Respir Med. 2019 Jun. doi: 10.1016/S2213-2600[19]30050-5).
Increased blood monocyte counts in idiopathic pulmonary fibrosis may reflect disease activity, which “could explain the outcome differences,” said Dr. Kreuter and Dr. Maher. “As highlighted by the investigators themselves, before introducing assessment of monocyte counts as part of routine clinical care for individuals with idiopathic pulmonary fibrosis, the limitations of this research should be taken into account. These include uncertainty around diagnosis and disease severity in a substantial subset of the patients, and the unknown effect of medical therapies (including corticosteroids and immunosuppressant and antifibrotic drugs) on monocyte counts and prognosis.” Researchers should validate the clinical value of blood monocyte counts in existing and future cohorts and evaluate the biomarker in clinical trials.
The editorialists have received compensation and funding from various pharmaceutical companies.
The study by Scott et al. provides evidence that monocyte count may be a “novel, simple, and inexpensive prognostic biomarker in idiopathic pulmonary fibrosis,” according to an accompanying editorial.
Progress has been made in the treatment of idiopathic pulmonary fibrosis, but patient prognosis remains “challenging to predict,” wrote Michael Kreuter, MD, of University of Heidelberg, Germany, and Toby M. Maher, MB, MSc, PhD, of Royal Brompton Hospital in London and Imperial College London. “One lesson that can be learned from other respiratory disorders is that routinely measured cellular biomarkers, such as blood eosinophil counts in chronic obstructive pulmonary disease (COPD), can predict treatment responses” (Lancet Respir Med. 2019 Jun. doi: 10.1016/S2213-2600[19]30050-5).
Increased blood monocyte counts in idiopathic pulmonary fibrosis may reflect disease activity, which “could explain the outcome differences,” said Dr. Kreuter and Dr. Maher. “As highlighted by the investigators themselves, before introducing assessment of monocyte counts as part of routine clinical care for individuals with idiopathic pulmonary fibrosis, the limitations of this research should be taken into account. These include uncertainty around diagnosis and disease severity in a substantial subset of the patients, and the unknown effect of medical therapies (including corticosteroids and immunosuppressant and antifibrotic drugs) on monocyte counts and prognosis.” Researchers should validate the clinical value of blood monocyte counts in existing and future cohorts and evaluate the biomarker in clinical trials.
The editorialists have received compensation and funding from various pharmaceutical companies.
The study by Scott et al. provides evidence that monocyte count may be a “novel, simple, and inexpensive prognostic biomarker in idiopathic pulmonary fibrosis,” according to an accompanying editorial.
Progress has been made in the treatment of idiopathic pulmonary fibrosis, but patient prognosis remains “challenging to predict,” wrote Michael Kreuter, MD, of University of Heidelberg, Germany, and Toby M. Maher, MB, MSc, PhD, of Royal Brompton Hospital in London and Imperial College London. “One lesson that can be learned from other respiratory disorders is that routinely measured cellular biomarkers, such as blood eosinophil counts in chronic obstructive pulmonary disease (COPD), can predict treatment responses” (Lancet Respir Med. 2019 Jun. doi: 10.1016/S2213-2600[19]30050-5).
Increased blood monocyte counts in idiopathic pulmonary fibrosis may reflect disease activity, which “could explain the outcome differences,” said Dr. Kreuter and Dr. Maher. “As highlighted by the investigators themselves, before introducing assessment of monocyte counts as part of routine clinical care for individuals with idiopathic pulmonary fibrosis, the limitations of this research should be taken into account. These include uncertainty around diagnosis and disease severity in a substantial subset of the patients, and the unknown effect of medical therapies (including corticosteroids and immunosuppressant and antifibrotic drugs) on monocyte counts and prognosis.” Researchers should validate the clinical value of blood monocyte counts in existing and future cohorts and evaluate the biomarker in clinical trials.
The editorialists have received compensation and funding from various pharmaceutical companies.
, including hypertrophic cardiomyopathy, systemic sclerosis, and myelofibrosis, according to research published in The Lancet Respiratory Medicine.
The data indicate that “a single threshold value of absolute monocyte counts of 0.95 K/mcL could be used to identify high-risk patients with a fibrotic disease,” said Madeleine K. D. Scott, a researcher at Stanford (Calif.) University, and coauthors. The results “suggest that monocyte count should be incorporated into the clinical assessment” and may “enable more conscientious allocation of scarce resources, including lung transplantations,” they said.
While other published biomarkers – including gene panels and multicytokine signatures – may be expensive and not readily available, “absolute monocyte count is routinely measured as part of a complete blood count, an inexpensive test used in clinical practice worldwide,” the authors said.
Further study of monocytes’ mechanistic role in fibrosis ultimately could point to new treatment approaches.
A retrospective multicenter cohort study
To assess whether immune cells may identify patients with idiopathic pulmonary fibrosis at greater risk of poor outcomes, Ms. Scott and her collaborators conducted a retrospective multicenter cohort study.
They first analyzed transcriptome data from 120 peripheral blood mononuclear cell samples of patients with idiopathic pulmonary fibrosis, which they obtained from the Gene Expression Omnibus at the National Center for Biotechnology Information. They used statistical deconvolution to estimate percentages of 13 immune cell types and examined their associations with transplant-free survival. Their discovery analysis found that estimated CD14+ classical monocyte percentages above the mean correlated with shorter transplant-free survival times (hazard ratio, 1.82), but percentages of T cells and B cells did not.
The researchers then validated these results using samples from patients with idiopathic pulmonary fibrosis in two independent cohorts. In the COMET validation cohort, which included 45 patients with idiopathic pulmonary fibrosis whose monocyte counts were measured using flow cytometry, higher monocyte counts were significantly associated with greater risk of disease progression. In the Yale cohort, which included 15 patients with idiopathic pulmonary fibrosis, the 6 patients who were classified as high risk on the basis of a 52-gene signature had more CD14+ monocytes than the 9 low-risk patients did.
In addition, Ms. Scott and her collaborators looked at complete blood count values in the electronic health records of 45,068 patients with idiopathic pulmonary fibrosis, systemic sclerosis, hypertrophic cardiomyopathy, or myelofibrosis in Stanford, Northwestern, Vanderbilt, and Optum Clinformatics Data Mart cohorts.
Among patients in the COMET, Stanford, and Northwestern datasets, monocyte counts of 0.95 K/mcL or greater were associated with mortality after adjustment for forced vital capacity (HR, 2.47) and the gender, age, and physiology index (HR, 2.06). Data from 7,459 patients with idiopathic pulmonary fibrosis “showed that patients with monocyte counts of 0.95 K/mcL or greater were at increased risk of mortality with lung transplantation as a censoring event, after adjusting for age at diagnosis and sex” in the Stanford (HR, 2.30), Vanderbilt (HR, 1.52), and Optum (HR, 1.74) cohorts. “Likewise, higher absolute monocyte count was associated with shortened survival in patients with hypertrophic cardiomyopathy across all three cohorts, and in patients with systemic sclerosis or myelofibrosis in two of the three cohorts,” the researchers said.
The study was funded by grants from the Bill & Melinda Gates Foundation, U.S. National Institute of Allergy and Infectious Diseases, and U.S. National Library of Medicine. Ms. Scott had no competing interests. Coauthors disclosed grants, compensation, and support from foundations, agencies, and companies.
SOURCE: Scott MKD et al. Lancet Respir Med. 2019 Jun. doi: 10.1016/S2213-2600(18)30508-3.
, including hypertrophic cardiomyopathy, systemic sclerosis, and myelofibrosis, according to research published in The Lancet Respiratory Medicine.
The data indicate that “a single threshold value of absolute monocyte counts of 0.95 K/mcL could be used to identify high-risk patients with a fibrotic disease,” said Madeleine K. D. Scott, a researcher at Stanford (Calif.) University, and coauthors. The results “suggest that monocyte count should be incorporated into the clinical assessment” and may “enable more conscientious allocation of scarce resources, including lung transplantations,” they said.
While other published biomarkers – including gene panels and multicytokine signatures – may be expensive and not readily available, “absolute monocyte count is routinely measured as part of a complete blood count, an inexpensive test used in clinical practice worldwide,” the authors said.
Further study of monocytes’ mechanistic role in fibrosis ultimately could point to new treatment approaches.
A retrospective multicenter cohort study
To assess whether immune cells may identify patients with idiopathic pulmonary fibrosis at greater risk of poor outcomes, Ms. Scott and her collaborators conducted a retrospective multicenter cohort study.
They first analyzed transcriptome data from 120 peripheral blood mononuclear cell samples of patients with idiopathic pulmonary fibrosis, which they obtained from the Gene Expression Omnibus at the National Center for Biotechnology Information. They used statistical deconvolution to estimate percentages of 13 immune cell types and examined their associations with transplant-free survival. Their discovery analysis found that estimated CD14+ classical monocyte percentages above the mean correlated with shorter transplant-free survival times (hazard ratio, 1.82), but percentages of T cells and B cells did not.
The researchers then validated these results using samples from patients with idiopathic pulmonary fibrosis in two independent cohorts. In the COMET validation cohort, which included 45 patients with idiopathic pulmonary fibrosis whose monocyte counts were measured using flow cytometry, higher monocyte counts were significantly associated with greater risk of disease progression. In the Yale cohort, which included 15 patients with idiopathic pulmonary fibrosis, the 6 patients who were classified as high risk on the basis of a 52-gene signature had more CD14+ monocytes than the 9 low-risk patients did.
In addition, Ms. Scott and her collaborators looked at complete blood count values in the electronic health records of 45,068 patients with idiopathic pulmonary fibrosis, systemic sclerosis, hypertrophic cardiomyopathy, or myelofibrosis in Stanford, Northwestern, Vanderbilt, and Optum Clinformatics Data Mart cohorts.
Among patients in the COMET, Stanford, and Northwestern datasets, monocyte counts of 0.95 K/mcL or greater were associated with mortality after adjustment for forced vital capacity (HR, 2.47) and the gender, age, and physiology index (HR, 2.06). Data from 7,459 patients with idiopathic pulmonary fibrosis “showed that patients with monocyte counts of 0.95 K/mcL or greater were at increased risk of mortality with lung transplantation as a censoring event, after adjusting for age at diagnosis and sex” in the Stanford (HR, 2.30), Vanderbilt (HR, 1.52), and Optum (HR, 1.74) cohorts. “Likewise, higher absolute monocyte count was associated with shortened survival in patients with hypertrophic cardiomyopathy across all three cohorts, and in patients with systemic sclerosis or myelofibrosis in two of the three cohorts,” the researchers said.
The study was funded by grants from the Bill & Melinda Gates Foundation, U.S. National Institute of Allergy and Infectious Diseases, and U.S. National Library of Medicine. Ms. Scott had no competing interests. Coauthors disclosed grants, compensation, and support from foundations, agencies, and companies.
SOURCE: Scott MKD et al. Lancet Respir Med. 2019 Jun. doi: 10.1016/S2213-2600(18)30508-3.
FROM THE LANCET RESPIRATORY MEDICINE
Key clinical point: An increased monocyte count predicts poor outcomes among patients with idiopathic pulmonary fibrosis and other fibrotic diseases.
Major finding: Among patients in three cohorts, monocyte counts of 0.95 K/mcL or greater were associated with mortality after adjustment for forced vital capacity (hazard ratio, 2.47) and the gender, age, and physiology index (HR, 2.06).
Study details: A retrospective analysis of data from 7,000 patients with idiopathic pulmonary fibrosis from five independent cohorts.
Disclosures: The study was funded by grants from the Bill & Melinda Gates Foundation, U.S. National Institute of Allergy and Infectious Diseases, and U.S. National Library of Medicine. Ms. Scott had no competing interests. Coauthors disclosed grants, compensation, and support from foundations, agencies, and companies.
Source: Scott MKD et al. Lancet Respir Med. 2019 Jun. doi: 10.1016/S2213-2600(18)30508-3.
CF drug picks up indication for children as young as 6
to include children as young as 6 years old who have cystic fibrosis.
The drug was approved in 2018 for patients aged 12 years and older who have the most common cause of the disease, two alleles for the F508del mutation in the gene that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, or at least one other CFTR mutation responsive to the combination, as listed in labeling.
The original approval was based on three phase 3, double blind, placebo-controlled trials, which demonstrated improvements in lung function and other key measures of the disease. One trial that found a 6.8% mean improvement in lung function testing over placebo at 8 weeks, and another that found a 4% improvement at 24 weeks, with fewer respiratory exacerbations and improved respiratory-related quality of life. A third trial in patients without the indicated genetic mutations was ended early for futility.
The efficacy in children under 12 years was extrapolated from those trials, plus an open-label study that found similar effects.
Labeling warns of elevated liver enzymes and cataracts in children, and notes that the drug should be taken with food that contains fat. Labeling also recommends against use with strong cytochrome P450 3A4 (CYP3A) inducers – rifampin, phenobarbital, St. John’s wort, among others – because they might reduce efficacy, and against use with CYP3A inhibitors – ketoconazole, clarithromycin, Seville oranges, grapefruit juice, etc. – because of the risk of increased exposure.
The most common side effects are headache, nausea, sinus congestion, and dizziness. The FDA has cleared a CF gene test to check for the required mutations. Symdeko is marketed by Vertex Pharmaceuticals.
to include children as young as 6 years old who have cystic fibrosis.
The drug was approved in 2018 for patients aged 12 years and older who have the most common cause of the disease, two alleles for the F508del mutation in the gene that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, or at least one other CFTR mutation responsive to the combination, as listed in labeling.
The original approval was based on three phase 3, double blind, placebo-controlled trials, which demonstrated improvements in lung function and other key measures of the disease. One trial that found a 6.8% mean improvement in lung function testing over placebo at 8 weeks, and another that found a 4% improvement at 24 weeks, with fewer respiratory exacerbations and improved respiratory-related quality of life. A third trial in patients without the indicated genetic mutations was ended early for futility.
The efficacy in children under 12 years was extrapolated from those trials, plus an open-label study that found similar effects.
Labeling warns of elevated liver enzymes and cataracts in children, and notes that the drug should be taken with food that contains fat. Labeling also recommends against use with strong cytochrome P450 3A4 (CYP3A) inducers – rifampin, phenobarbital, St. John’s wort, among others – because they might reduce efficacy, and against use with CYP3A inhibitors – ketoconazole, clarithromycin, Seville oranges, grapefruit juice, etc. – because of the risk of increased exposure.
The most common side effects are headache, nausea, sinus congestion, and dizziness. The FDA has cleared a CF gene test to check for the required mutations. Symdeko is marketed by Vertex Pharmaceuticals.
to include children as young as 6 years old who have cystic fibrosis.
The drug was approved in 2018 for patients aged 12 years and older who have the most common cause of the disease, two alleles for the F508del mutation in the gene that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein, or at least one other CFTR mutation responsive to the combination, as listed in labeling.
The original approval was based on three phase 3, double blind, placebo-controlled trials, which demonstrated improvements in lung function and other key measures of the disease. One trial that found a 6.8% mean improvement in lung function testing over placebo at 8 weeks, and another that found a 4% improvement at 24 weeks, with fewer respiratory exacerbations and improved respiratory-related quality of life. A third trial in patients without the indicated genetic mutations was ended early for futility.
The efficacy in children under 12 years was extrapolated from those trials, plus an open-label study that found similar effects.
Labeling warns of elevated liver enzymes and cataracts in children, and notes that the drug should be taken with food that contains fat. Labeling also recommends against use with strong cytochrome P450 3A4 (CYP3A) inducers – rifampin, phenobarbital, St. John’s wort, among others – because they might reduce efficacy, and against use with CYP3A inhibitors – ketoconazole, clarithromycin, Seville oranges, grapefruit juice, etc. – because of the risk of increased exposure.
The most common side effects are headache, nausea, sinus congestion, and dizziness. The FDA has cleared a CF gene test to check for the required mutations. Symdeko is marketed by Vertex Pharmaceuticals.
Cannabis vaping among teens tied to tobacco use
and that practice is associated with cigars, waterpipe and e-cigarette use, findings from a survey of nearly 3,000 adolescents have shown.
“Although the prevalence of e-cigarette use among youth has increased dramatically in the past decade, little epidemiologic data exist on the prevalence of using e-cigarette devices or other specialised devices to vaporise (‘vape’) cannabis in the form of hash oil, tetrahydrocannabinol (THC) wax or oil, or dried cannabis buds or leaves,” wrote Sarah D. Kowitt, PhD, of the University of North Carolina, Chapel Hill, and colleagues. “This is surprising given that (1) cannabis (also referred to as marijuana) and e-cigarettes are the most commonly used substances by adolescents in the USA, (2) evidence exists that adolescents dual use both tobacco e-cigarettes and cannabis, and (3) longitudinal research suggests that use of e-cigarettes is associated with progression to use of cannabis.”
In a study published in BMJ Open, the researchers used data from the 2017 North Carolina Youth Tobacco Survey, a school-based survey of students in grades 6-12. The study population included 2,835 adolescents in grades 9-12.
Overall, 9.6% of students reported ever vaping cannabis. In multivariate analysis, cannabis vaping was significantly more likely among adolescents who reported using e-cigarettes (adjusted odds ratio 3.18), cigars (aOR 3.76), or water pipes (aOR 2.32) in the past 30 days, compared with peers who didn’t use tobacco.
The researchers found no significant association between smokeless tobacco use or traditional cigarette use in the past 30 days and vaping cannabis.
In a bivariate analysis, vaping cannabis was significantly more common among males vs. females (11% vs. 8.2%) and among non-Hispanic white students (11.3%), Hispanic students (10.5%), and other non-Hispanic students (11.8%) compared with non-Hispanic black students (5.0%).
In addition, prevalence of cannabis vaping increased with grade level, from 4.7% of 9th graders to 15.5% of 12th graders.
The health impacts of vaping cannabis are not well researched, but the researchers note that among the potential safety issues are earlier initiation of tobacco or cannabis use, concomitant tobacco and cannabis use, increased frequency of use or misuse of tobacco or cannabis, or increased potency of cannabis.
The results of the study were limited by several factors including the use of data only from the state of North Carolina, the lack of data on frequency or current vaping cannabis behavior, lack of data on specific products, and lack of data on whether teens used specialized devices or e-cigarettes for cannabis vaping. However, the findings are consistent with studies on prevalence of cannabis vaping in other states such as Connecticut and California. “No studies to our knowledge have examined how adolescents who vape cannabis use other specific tobacco products (i.e., cigarettes, cigars, waterpipe, smokeless tobacco),” the researchers wrote.
The findings confirm that a large number of adolescents who use tobacco products have vaped cannabis as well, and this growing public health issue “is likely to affect and be affected by tobacco control and cannabis policies in states and at the federal level in the USA,” the researchers concluded.
“Increased research investigating how youth use e-cigarette devices for other purposes beyond vaping nicotine, like the current study, is needed,” they added.
The study was supported in part by the National Cancer Institute and the Food and Drug Administration’s Center for Tobacco Products. The researchers had no financial conflicts to disclose.
SOURCE: Kowitt SD et al. BMJ Open. 2019 Jun 13. doi: 10.1136/bmjopen-2018-028535.
and that practice is associated with cigars, waterpipe and e-cigarette use, findings from a survey of nearly 3,000 adolescents have shown.
“Although the prevalence of e-cigarette use among youth has increased dramatically in the past decade, little epidemiologic data exist on the prevalence of using e-cigarette devices or other specialised devices to vaporise (‘vape’) cannabis in the form of hash oil, tetrahydrocannabinol (THC) wax or oil, or dried cannabis buds or leaves,” wrote Sarah D. Kowitt, PhD, of the University of North Carolina, Chapel Hill, and colleagues. “This is surprising given that (1) cannabis (also referred to as marijuana) and e-cigarettes are the most commonly used substances by adolescents in the USA, (2) evidence exists that adolescents dual use both tobacco e-cigarettes and cannabis, and (3) longitudinal research suggests that use of e-cigarettes is associated with progression to use of cannabis.”
In a study published in BMJ Open, the researchers used data from the 2017 North Carolina Youth Tobacco Survey, a school-based survey of students in grades 6-12. The study population included 2,835 adolescents in grades 9-12.
Overall, 9.6% of students reported ever vaping cannabis. In multivariate analysis, cannabis vaping was significantly more likely among adolescents who reported using e-cigarettes (adjusted odds ratio 3.18), cigars (aOR 3.76), or water pipes (aOR 2.32) in the past 30 days, compared with peers who didn’t use tobacco.
The researchers found no significant association between smokeless tobacco use or traditional cigarette use in the past 30 days and vaping cannabis.
In a bivariate analysis, vaping cannabis was significantly more common among males vs. females (11% vs. 8.2%) and among non-Hispanic white students (11.3%), Hispanic students (10.5%), and other non-Hispanic students (11.8%) compared with non-Hispanic black students (5.0%).
In addition, prevalence of cannabis vaping increased with grade level, from 4.7% of 9th graders to 15.5% of 12th graders.
The health impacts of vaping cannabis are not well researched, but the researchers note that among the potential safety issues are earlier initiation of tobacco or cannabis use, concomitant tobacco and cannabis use, increased frequency of use or misuse of tobacco or cannabis, or increased potency of cannabis.
The results of the study were limited by several factors including the use of data only from the state of North Carolina, the lack of data on frequency or current vaping cannabis behavior, lack of data on specific products, and lack of data on whether teens used specialized devices or e-cigarettes for cannabis vaping. However, the findings are consistent with studies on prevalence of cannabis vaping in other states such as Connecticut and California. “No studies to our knowledge have examined how adolescents who vape cannabis use other specific tobacco products (i.e., cigarettes, cigars, waterpipe, smokeless tobacco),” the researchers wrote.
The findings confirm that a large number of adolescents who use tobacco products have vaped cannabis as well, and this growing public health issue “is likely to affect and be affected by tobacco control and cannabis policies in states and at the federal level in the USA,” the researchers concluded.
“Increased research investigating how youth use e-cigarette devices for other purposes beyond vaping nicotine, like the current study, is needed,” they added.
The study was supported in part by the National Cancer Institute and the Food and Drug Administration’s Center for Tobacco Products. The researchers had no financial conflicts to disclose.
SOURCE: Kowitt SD et al. BMJ Open. 2019 Jun 13. doi: 10.1136/bmjopen-2018-028535.
and that practice is associated with cigars, waterpipe and e-cigarette use, findings from a survey of nearly 3,000 adolescents have shown.
“Although the prevalence of e-cigarette use among youth has increased dramatically in the past decade, little epidemiologic data exist on the prevalence of using e-cigarette devices or other specialised devices to vaporise (‘vape’) cannabis in the form of hash oil, tetrahydrocannabinol (THC) wax or oil, or dried cannabis buds or leaves,” wrote Sarah D. Kowitt, PhD, of the University of North Carolina, Chapel Hill, and colleagues. “This is surprising given that (1) cannabis (also referred to as marijuana) and e-cigarettes are the most commonly used substances by adolescents in the USA, (2) evidence exists that adolescents dual use both tobacco e-cigarettes and cannabis, and (3) longitudinal research suggests that use of e-cigarettes is associated with progression to use of cannabis.”
In a study published in BMJ Open, the researchers used data from the 2017 North Carolina Youth Tobacco Survey, a school-based survey of students in grades 6-12. The study population included 2,835 adolescents in grades 9-12.
Overall, 9.6% of students reported ever vaping cannabis. In multivariate analysis, cannabis vaping was significantly more likely among adolescents who reported using e-cigarettes (adjusted odds ratio 3.18), cigars (aOR 3.76), or water pipes (aOR 2.32) in the past 30 days, compared with peers who didn’t use tobacco.
The researchers found no significant association between smokeless tobacco use or traditional cigarette use in the past 30 days and vaping cannabis.
In a bivariate analysis, vaping cannabis was significantly more common among males vs. females (11% vs. 8.2%) and among non-Hispanic white students (11.3%), Hispanic students (10.5%), and other non-Hispanic students (11.8%) compared with non-Hispanic black students (5.0%).
In addition, prevalence of cannabis vaping increased with grade level, from 4.7% of 9th graders to 15.5% of 12th graders.
The health impacts of vaping cannabis are not well researched, but the researchers note that among the potential safety issues are earlier initiation of tobacco or cannabis use, concomitant tobacco and cannabis use, increased frequency of use or misuse of tobacco or cannabis, or increased potency of cannabis.
The results of the study were limited by several factors including the use of data only from the state of North Carolina, the lack of data on frequency or current vaping cannabis behavior, lack of data on specific products, and lack of data on whether teens used specialized devices or e-cigarettes for cannabis vaping. However, the findings are consistent with studies on prevalence of cannabis vaping in other states such as Connecticut and California. “No studies to our knowledge have examined how adolescents who vape cannabis use other specific tobacco products (i.e., cigarettes, cigars, waterpipe, smokeless tobacco),” the researchers wrote.
The findings confirm that a large number of adolescents who use tobacco products have vaped cannabis as well, and this growing public health issue “is likely to affect and be affected by tobacco control and cannabis policies in states and at the federal level in the USA,” the researchers concluded.
“Increased research investigating how youth use e-cigarette devices for other purposes beyond vaping nicotine, like the current study, is needed,” they added.
The study was supported in part by the National Cancer Institute and the Food and Drug Administration’s Center for Tobacco Products. The researchers had no financial conflicts to disclose.
SOURCE: Kowitt SD et al. BMJ Open. 2019 Jun 13. doi: 10.1136/bmjopen-2018-028535.
FROM BMJ OPEN
Key clinical point: Use of tobacco products was significantly associated with cannabis vaping in teens.
Major finding: Approximately 10% of adolescents reported vaping cannabis.
Study details: The data come from a survey of 2,835 adolescents in North Carolina.
Disclosures: The study was supported in part by the National Cancer Institute and the FDA Center for Tobacco Products. The researchers had no financial conflicts to disclose.
Source: Kowitt SD et al. BMJ Open. 2019 Jun 13. doi: 10.1136/bmjopen-2018-028535.
Children with Down syndrome may need more screening for sleep-disordered breathing
because the condition frequently persists and recurs.
“Current screening recommendations to assess for SDB at a particular age may not be adequate in this population,” the authors of the study stated, adding that “persistence/recurrence of SDB is not easily predicted.”
The study, led by Joy Nehme, BSc, of Children’s Hospital of Eastern Ontario and the University of Ottawa, was published in Pediatric Pulmonology.
According to the study, research suggests that 43%-66% of children with Down syndrome have SDB, a category that encompasses sleep apnea (both obstructive and central) and hypoventilation. Those numbers are several times higher than the prevalence of SDB in children in the general population (1%-5%).
“Because SDB is associated with cardiometabolic and neurocognitive morbidity, its prompt and accurate diagnosis is important,” the researchers wrote. However, diagnosis requires a sleep study, which is not always performed although the American Academy of Pediatrics recommends children with Down syndrome undergo one by age 4.
Treatments include adenotonsillectomy (considered first-line), positive airway pressure, and lingual tonsillectomy.
The study aims to fill in gaps in knowledge about the condition over the long term since “there is little available literature on the trajectory of SDB in children and youth with Down syndrome over time.”
The researchers launched a retrospective study of 560 children with Down syndrome who were treated from 2004 to 2015 at Children’s Hospital of Eastern Ontario. Of those, 120 showed signs of SDB and underwent sleep studies (48% male, median age 6.6 years [range 4.5-10.5], median total apnea‐hypopnea index events per hour = 3.4 [1.6-10.8]).
Of the 120 children, 67 (56%) had obstructive-mixed SDB, 9 (8%) had central sleep apnea, and 5 (4%) had hypoventilation. The others (39, 32%) had no SDB.
Fifty-four children underwent at least two sleep studies during the period of the study, with at least one undergoing seven.
Researchers found weak, nonsignificant evidence that SDB persistence/occurrence varied by age (odds ratio per year = 1.15; 95% confidence interval, 0.96-1.41; P = .13).
As for treatment, adenotonsillectomy was most common, although “previous studies have ... shown that moderate to severe OSA in children with Down syndrome is likely to persist after a tonsillectomy.”
In regard to obstructive sleep apnea (OSA) specifically, the authors wrote, “our study ... showed that OSA‐SDB persisted or recurred in the vast majority of children. Further, persistence/recurrence could not be predicted by clinical features or SDB severity in our study. This, therefore, highlights the need for serial longitudinal screening for SDB in this population and for follow‐up PSG to ensure the success of treatment interventions.”
No study funding was reported. The study authors reported no disclosures.
SOURCE: Nehme J et al. Pediatr Pulmonol. 2019 Jun 6. doi: 10.1002/ppul.24380.
because the condition frequently persists and recurs.
“Current screening recommendations to assess for SDB at a particular age may not be adequate in this population,” the authors of the study stated, adding that “persistence/recurrence of SDB is not easily predicted.”
The study, led by Joy Nehme, BSc, of Children’s Hospital of Eastern Ontario and the University of Ottawa, was published in Pediatric Pulmonology.
According to the study, research suggests that 43%-66% of children with Down syndrome have SDB, a category that encompasses sleep apnea (both obstructive and central) and hypoventilation. Those numbers are several times higher than the prevalence of SDB in children in the general population (1%-5%).
“Because SDB is associated with cardiometabolic and neurocognitive morbidity, its prompt and accurate diagnosis is important,” the researchers wrote. However, diagnosis requires a sleep study, which is not always performed although the American Academy of Pediatrics recommends children with Down syndrome undergo one by age 4.
Treatments include adenotonsillectomy (considered first-line), positive airway pressure, and lingual tonsillectomy.
The study aims to fill in gaps in knowledge about the condition over the long term since “there is little available literature on the trajectory of SDB in children and youth with Down syndrome over time.”
The researchers launched a retrospective study of 560 children with Down syndrome who were treated from 2004 to 2015 at Children’s Hospital of Eastern Ontario. Of those, 120 showed signs of SDB and underwent sleep studies (48% male, median age 6.6 years [range 4.5-10.5], median total apnea‐hypopnea index events per hour = 3.4 [1.6-10.8]).
Of the 120 children, 67 (56%) had obstructive-mixed SDB, 9 (8%) had central sleep apnea, and 5 (4%) had hypoventilation. The others (39, 32%) had no SDB.
Fifty-four children underwent at least two sleep studies during the period of the study, with at least one undergoing seven.
Researchers found weak, nonsignificant evidence that SDB persistence/occurrence varied by age (odds ratio per year = 1.15; 95% confidence interval, 0.96-1.41; P = .13).
As for treatment, adenotonsillectomy was most common, although “previous studies have ... shown that moderate to severe OSA in children with Down syndrome is likely to persist after a tonsillectomy.”
In regard to obstructive sleep apnea (OSA) specifically, the authors wrote, “our study ... showed that OSA‐SDB persisted or recurred in the vast majority of children. Further, persistence/recurrence could not be predicted by clinical features or SDB severity in our study. This, therefore, highlights the need for serial longitudinal screening for SDB in this population and for follow‐up PSG to ensure the success of treatment interventions.”
No study funding was reported. The study authors reported no disclosures.
SOURCE: Nehme J et al. Pediatr Pulmonol. 2019 Jun 6. doi: 10.1002/ppul.24380.
because the condition frequently persists and recurs.
“Current screening recommendations to assess for SDB at a particular age may not be adequate in this population,” the authors of the study stated, adding that “persistence/recurrence of SDB is not easily predicted.”
The study, led by Joy Nehme, BSc, of Children’s Hospital of Eastern Ontario and the University of Ottawa, was published in Pediatric Pulmonology.
According to the study, research suggests that 43%-66% of children with Down syndrome have SDB, a category that encompasses sleep apnea (both obstructive and central) and hypoventilation. Those numbers are several times higher than the prevalence of SDB in children in the general population (1%-5%).
“Because SDB is associated with cardiometabolic and neurocognitive morbidity, its prompt and accurate diagnosis is important,” the researchers wrote. However, diagnosis requires a sleep study, which is not always performed although the American Academy of Pediatrics recommends children with Down syndrome undergo one by age 4.
Treatments include adenotonsillectomy (considered first-line), positive airway pressure, and lingual tonsillectomy.
The study aims to fill in gaps in knowledge about the condition over the long term since “there is little available literature on the trajectory of SDB in children and youth with Down syndrome over time.”
The researchers launched a retrospective study of 560 children with Down syndrome who were treated from 2004 to 2015 at Children’s Hospital of Eastern Ontario. Of those, 120 showed signs of SDB and underwent sleep studies (48% male, median age 6.6 years [range 4.5-10.5], median total apnea‐hypopnea index events per hour = 3.4 [1.6-10.8]).
Of the 120 children, 67 (56%) had obstructive-mixed SDB, 9 (8%) had central sleep apnea, and 5 (4%) had hypoventilation. The others (39, 32%) had no SDB.
Fifty-four children underwent at least two sleep studies during the period of the study, with at least one undergoing seven.
Researchers found weak, nonsignificant evidence that SDB persistence/occurrence varied by age (odds ratio per year = 1.15; 95% confidence interval, 0.96-1.41; P = .13).
As for treatment, adenotonsillectomy was most common, although “previous studies have ... shown that moderate to severe OSA in children with Down syndrome is likely to persist after a tonsillectomy.”
In regard to obstructive sleep apnea (OSA) specifically, the authors wrote, “our study ... showed that OSA‐SDB persisted or recurred in the vast majority of children. Further, persistence/recurrence could not be predicted by clinical features or SDB severity in our study. This, therefore, highlights the need for serial longitudinal screening for SDB in this population and for follow‐up PSG to ensure the success of treatment interventions.”
No study funding was reported. The study authors reported no disclosures.
SOURCE: Nehme J et al. Pediatr Pulmonol. 2019 Jun 6. doi: 10.1002/ppul.24380.
FROM PEDIATRIC PULMONOLOGY
Key clinical point: Sleep-disordered breathing (SDB) can be persistent and recurrent in children with Down syndrome, and long-term monitoring is warranted.
Major finding: SDB persistence/recurrence did not vary by age (odds ratio per year = 1.15; 95% confidence interval, 0.96-1.41; P = .13).
Study details: Retrospective cohort analysis of 120 children with Down syndrome tested via sleep study at least once for SDB (48% male, median age 6.6 years).
Disclosures: No study funding or author disclosures were reported.
Source: Nehme J et al. Pediatr Pulmonol. 2019 Jun 6. doi: 10.1002/ppul.24380.