To provide the best clinical practice guidance for surfactant use in preterm neonates with respiratory distress syndrome (RDS). The RDS-Neonatal Expert Taskforce (RDS-NExT) initiative was intended to ...add to existing evidence and clinical guidelines, where evidence is lacking, with input from an expert panel.
An expert panel of healthcare providers specializing in neonatal intensive care was convened and administered a survey questionnaire, followed by 3 virtual workshops. A modified Delphi method was used to obtain consensus around topics in surfactant use in neonatal RDS.
Statements focused on establishing RDS diagnosis and indicators for surfactant administration, surfactant administration methods and techniques, and other considerations. After discussion and voting, consensus was achieved on 20 statements.
These consensus statements provide practical guidance for surfactant administration in preterm neonates with RDS, with a goal to contribute to improving the care of neonates and providing a stimulus for further investigation to bridge existing knowledge gaps.
Short-acting β-agonist (SABA) use is well established in predicting asthma events in adults. However, this predictive ability has yet to be established in a pediatric population together with an ...assessment of amount of use.
To identify the number of SABA canisters that best predicts future asthma-related exacerbations and the optimal length of time for measurement of SABA use in pediatric and adult asthma patients.
Asthma patients were identified from a Medicaid and a commercially insured database (January 1, 2004, through December 31, 2005, and January 1, 2004, through June 30, 2006, respectively). Following the date of first asthma medication, an assessment period (3, 6, or 12 months) was used to measure SABA use. Asthma-related exacerbations were identified in the subsequent 12-month period. Receiver operating characteristic curve analyses and logistic regression were used to select the critical values of SABA use and optimal assessment periods and to conduct incremental analysis, respectively.
A total of 33,793 Medicaid and 101,437 commercial patients met the study criteria. Use of 3 or more SABA canisters during 12 months was identified in both pediatric Medicaid and commercial populations to best predict an increased risk of an asthma-related exacerbation. For adults, use of 2 or more SABA canisters was found as the critical value with shorter optimal assessment periods of 3 and 6 months. Each additional SABA canister resulted in an 8% to 14% and 14% to 18% increase in risk of an asthma-related exacerbation in children and adults, respectively.
The study identified critical values of SABA use that predict future asthma events. Each additional SABA canister predicted increases in exacerbation risk in children and adults.
This study assessed patient-level characteristics and patterns of medication use in patients with chronic obstructive pulmonary disease (COPD) before initiation of multiple inhaler triple therapy ...(MITT; long-acting muscarinic antagonist/long-acting β2-agonist/inhaled corticosteroid ICS/LAMA/LABA combination).
This retrospective study was conducted using the Optum Research Database. Patients enrolled in commercial or Medicare Advantage Prescription Drug plans, with a COPD diagnosis and >1 prescription for a COPD medication between January 2014 and March 2016 were included. The dispensing date for the first pharmacy prescription completing MITT with at least 1 day of overlap was the patient's index date. The 12 months prior to this date were used to assess patient characteristics, exacerbations, eosinophil counts and changes in medication.
The study population comprised 13,701 patients. At the index date, most patients were using a LAMA (n = 13,353 97.5%) and combination ICS/LABA (n = 13,292 97.0%) MITT. Overall, 90.4% of patients used a LABA, LAMA, LAMA/LABA, or ICS/LABA or had a moderate or severe exacerbation at any time during the baseline period, indicating that approximately 10% of patients initiated MITT without prior bronchodilator use or exacerbation history. Over 65% of patients with an eosinophil measurement had a value ≥ 150 cells/μL.
Overall, it appears that in this patient population, ICS/LAMA/LABA as triple therapy is being initiated after use of a bronchodilator and/or after an exacerbation event, in accordance with accepted treatment recommendations.
•59.8% of patients had a moderate/severe exacerbation prior to initiating MITT.•90.4% of patients were on COPD medication or had an exacerbation prior to MITT.•9.6% of patients initiated MITT without prior COPD medication use or exacerbations.•Eosinophil results were available for 3109 patients and 79.5% had normal counts.
This longitudinal, retrospective cohort study of patients with COPD describes baseline characteristics, adherence, and persistence following initiation of inhaled corticosteroids (ICS)/long-acting β
...-agonists (LABA)/long-acting muscarinic antagonists (LAMA) from multiple inhaler triple therapy (MITT).
Patients aged ≥40 years receiving MITT between January 2012 and September 2015 were identified from the IQVIA™ Real-world Data Adjudicated Claims-USA database. MITT was defined as subjects with ≥1 overlapping days' supply of three COPD medications (ICS, LABA, and LAMA). Adherence (proportion of days covered, PDC) and discontinuation (defined as a gap of 1, 30, 60, or 90 days of supply in any of the three components of the triple therapy) were calculated for each patient over 12 months of follow-up. In addition, analyses were stratified by number of inhalers.
In total, 14,635 MITT users were identified (mean age, 62 years). Mean PDC for MITT at 12 months was 0.37%. Mean PDC for the ICS/LABA and LAMA component at 12 months was 49% (0.49±0.31; median, 0.47) and 54% (0.54±0.33; 0.56), respectively. The proportion of adherent patients (PDC ≥0.8) at 12 months was 14% for MITT. Allowing for a 30-day gap from last day of therapy, 86% of MITT users discontinued therapy during follow-up.
Patients with COPD had low adherence to and persistence with MITT in a real-world setting. Mean PDC for each single inhaler component was higher than the mean PDC observed with MITT. Reducing the number of inhalers may improve overall adherence to intended triple therapy.
Background The Rhinitis Control Assessment Test (RCAT) is a brief, patient-completed tool to evaluate rhinitis symptom control. Objective We sought to test the reliability, validity, and ...responsiveness of RCAT and to estimate a cut-point score and minimal important difference (MID). Methods A total of 402 patients 12 years of age and older with allergic or nonallergic rhinitis were enrolled in a noninterventional study. Patients completed the RCAT (6 items; score range, 6-30) and had Total Nasal Symptom Scores (TNSSs) measured at baseline and 2 weeks later. Physicians completed a global assessment of rhinitis symptom control (Physician's Global Assessment) and disease severity. Internal consistency, test-retest reliability, convergent validity, known-groups validity, and responsiveness were evaluated. The MID was determined by using distribution- and anchor-based methods. Content validity of the RCAT was assessed in individual interviews with a separate group of 58 adult patients. Results Internal consistency and test-retest reliability of RCAT scores were 0.77 and 0.78, respectively. Convergent validity correlation between RCAT and TNSS scores was 0.57, and that between RCAT and Physician's Global Assessment scores was 0.34. Mean RCAT scores differed significantly ( P < .001) across patient groups, differing in TNSS (F = 72.7), Physician's Global Assessment score (F = 28.6), and disease severity (F = 34.1) in the hypothesized direction. Results suggested a cut-point score of 21 or less can be used to identify patients who are experiencing rhinitis symptom control problems. The preliminary estimate of the MID was 3 points. Patients found RCAT items comprehensive, easy to understand, and relevant. Conclusion The RCAT demonstrated adequate reliability, validity, and responsiveness and was deemed acceptable and appropriate by patients. This tool can facilitate the detection of rhinitis symptom control problems, and its brevity supports its usefulness in clinical care.
This real-world observational study compared medication adherence and persistence among patients with asthma receiving the once-daily inhaled corticosteroid/long-acting β
2
-agonist (ICS/LABA) ...fluticasone furoate/vilanterol (FF/VI) versus the twice-daily ICS/LABAs budesonide/formoterol (B/F) and fluticasone propionate/salmeterol (FP/SAL).
This retrospective cohort study conducted using IQVIA
TM
Health Plan Claims Data included patients with asthma ≥18 years of age initiating ICS/LABA therapy with FF/VI, B/F, or FP/SAL between January 1, 2014 and June 30, 2016 (index date). Patients had ≥12 months and ≥3 months of continuous eligibility pre- and post-index date, respectively. Patients receiving FF/VI were separately matched 1:1 with patients receiving B/F or FP/SAL using propensity score matching (PSM) and multivariable regression to balance baseline covariates between cohorts. The primary endpoint was medication adherence, measured by proportion of days covered (PDC). Secondary endpoints included proportion of patients achieving PDC ≥ 0.5 and PDC ≥ 0.8 and persistence with index medication, measured by time to discontinuation (>45-day gap in therapy).
After PSM, 3,764 and 3,339 patients receiving FF/VI were matched with patients receiving B/F or FP/SAL, respectively. Mean PDC was significantly higher for FF/VI versus B/F (0.453 vs 0.345; adjusted p < 0.001) and FP/SAL (0.446 vs 0.341; adjusted p < 0.001). The proportion of patients achieving PDC ≥ 0.5 or PDC ≥ 0.8, and treatment persistence were significantly higher for FF/VI versus B/F and FP/SAL (all p < 0.001).
In this real-world study, patients initiating FF/VI had better adherence and lower risk of discontinuing treatment versus B/F or FP/SAL, suggesting that once-daily ICS/LABA treatment might improve adherence and persistence compared with twice-daily alternatives.
It is unknown whether there is a benefit to initiating triple therapy (TT; inhaled corticosteroids combined with long-acting β2-agonists and long-acting muscarinic antagonists) promptly (within 30 ...days) following a chronic obstructive pulmonary disease (COPD)-related hospitalization or emergency-department (ED) visit compared with delaying treatment (31–180 days).
This retrospective, observational study (GSK: HO-15-15256) used healthcare claims from a commercial and Medicare claims database (January 1, 2008–December 31, 2015). Patients: ≥40 years of age, diagnosed with COPD and no history of TT 12 months pre-index. Patients experiencing a COPD-related hospitalization or ED visit (index) who initiated TT ≤ 6 months following index were included (January 1, 2009–December 31, 2014). Patients initiating TT ≤ 30 or 31–180 days following index were included in the Prompt or Delayed cohorts, respectively. All-cause and COPD-related costs (total, medical and prescription), and exacerbations (severe and moderate) per patient per year were determined for 12 months post index. Outcomes were adjusted by cohort, weighted for a balanced distribution of baseline covariates between cohorts using inverse probability weights.
Overall, 10,902 patients were identified (Prompt: n = 5701; Delayed: n = 5201). Total, medical and prescription all-cause costs were significantly higher in the Delayed versus Prompt cohorts (percent increase: 18.7%, 22.8% and 8.8%, respectively; all p < 0.0001). COPD-related total, medical and prescription costs were 49.3%, 66.3% and 10.3% higher in the Delayed versus Prompt cohorts, respectively (all p < 0.0001). Total and severe COPD-related exacerbation rates were 28.2% and 64.7% higher in the Delayed versus Prompt cohorts (p < 0.0001).
Prompt use of TT following a COPD-inpatient or ED visit may reduce future costs and subsequent exacerbations compared with delaying the initiation of TT.
•Prompt versus delayed triple therapy (TT) may reduce future costs in COPD.•Fewer exacerbations were reported in patients receiving prompt versus delayed TT.•Prompt TT following an exacerbation may reduce future COPD-related events.
Improved outcomes have been reported for patients with chronic obstructive pulmonary disease (COPD) receiving combination long-acting muscarinic antagonist/long-acting β2-agonist (LAMA/LABA) therapy ...compared with LAMA monotherapy. However, little is known about the relative characteristics of these patients and their rates of escalation to triple therapy (TT, combining a LAMA, LABA, and inhaled corticosteroid). This study aimed to characterize patients initiating treatment with the LAMA tiotropium (TIO) and the fixed-dose LAMA/LABA combination therapy umeclidinium/vilanterol (UMEC/VI), and to compare rates of escalation to TT between patients receiving these therapies.
Retrospective study of patients with COPD enrolled in a US health insurance plan during 2013–2015 and newly initiated on TIO or UMEC/VI. Patients were ≥40 years of age at index (date of therapy initiation) with continuous enrollment for 12 months pre-index and ≥30 days post-index. LAMA users were propensity score matched 1:1 to LAMA/LABA users, with TT initiation rates reported by cohort using pharmacy claims.
35,357 patients initiating on TIO and 2407 patients initiating on UMEC/VI were identified. After propensity score matching, the rate of TT initiation was significantly higher in new TIO users (n = 1320) than in new UMEC/VI users (n = 1320) (0.92 vs 0.49 per 100 months of exposure, respectively; p < 0.001). Relative to the UMEC/VI cohort, the TIO cohort had an 87% higher risk of TT initiation (hazard ratio: 1.87; 95% confidence interval: 1.4–2.5; p = 0.001).
Patients receiving UMEC/VI progressed to TT more slowly, and were at lower risk of progressing to TT, than patients receiving TIO.
•UMEC/VI therapy was associated with slower progression to triple therapy than TIO.•The risk of triple therapy initiation was 87% higher in TIO users than UMEC/VI users.•Results suggest better symptom control and slower disease progression with UMEC/VI.
Hereditary factor X deficiency (HFXD) is a rare bleeding disorder causing delayed haemostasis and potentially life-threatening bleeds. Patient/caregiver burden and diagnosis path have not been well ...characterized.
describe the diagnosis path, disease burden, and HFXD impact on quality of life (QoL) in patients and caregivers.This was a prospective, cross-sectional, web-based survey of patients with HFXD and caregivers addressing the patient/caregiver experience, QoL, humanistic and unmet needs.Thirty patients and 38 caregivers completed the survey with mean ages 24.7 and 44.6 years, respectively. Mean age at diagnosis was 4.1 years. The diagnostic process was somewhat/very difficult for 23% of patients and 26% of caregivers. Approximately half (53%) received single factor replacement (SFR) as prophylaxis or on-demand. Most patients (71%) reported regular prophylaxis treatment. Over one-fourth (27%) reported treatment with fresh frozen plasma. Bleeding episodes were less common in patients using SFR versus non-SFR: three bleeds or fewer were reported by 92% SFR and 75% non-SFR patients. HFXD patients reported low well being in work/school/social activities with mean HFXD-adapted Hemophilia Well being Index. Patient symptoms negatively impacted caregiver burden with a mean HFXD-adapted Hemophilia Caregiver Index (±SD) of 15.9 (4.6), but also unexpectedly had a positive impact on self-worth and inner strength.To our knowledge, this is the first study to assess patient and caregiver burden of HFXD and impact on QoL. Improvements in symptom recognition, prompt diagnosis, and adherence to expert recommendations for treatment could improve QoL and decrease burden on HFXD patients and caregivers.
Evidence suggests that real-world treatment patterns of chronic obstructive pulmonary disease (COPD) do not always follow evidence-based treatment recommendations such as those of the Global ...Initiative for Chronic Obstructive Lung Disease, which recommends treatment escalation based on disease progression. This U.S. database study evaluated treatment patterns in patients with COPD, focusing on time to initiation of triple therapy using multiple inhalers.
To (a) estimate time from diagnosis to initiation of long-acting muscarinic antagonist (LAMA) monotherapy, inhaled corticosteroid (ICS)/long-acting beta2-agonist (LABA) dual therapy, or LAMA/LABA dual therapy; (b) estimate time to initiation of triple therapy from LAMA monotherapy and ICS/LABA or LAMA/LABA dual therapies; and (c) estimate the likelihood of patient progression to triple therapy.
This study was a retrospective analysis of patients with COPD newly started on LAMA monotherapy, ICS/LABA, or LAMA/LABA therapy between July 1, 2010, and March 31, 2013, as identified in Humana's research database. Patients who were fully insured with commercial or Medicare Advantage insurance plans and were aged ≥ 40 years at index with at least 1 hospitalization, 1 emergency department, or 1 medical office visit claim with a COPD diagnosis in the pre-index year were included in the analysis. Time from diagnosis to initiation of index therapy and time to triple therapy after index therapy were assessed. Multivariable logistic regression models were used to estimate the likelihood of progression to triple therapy.
Of 13,541 patients with a confirmed diagnosis of COPD, 4,000 received LAMA monotherapy; 8,207 received ICS/LABA therapy; and 77 received LAMA/LABA therapy at index; mean time (± SD) from COPD diagnosis to initiation of triple therapy was 178 (± 134) days, 185 (± 130) days, and 252 (± 124) days, respectively. During the study, 28% (n = 1,130) of patients receiving LAMA monotherapy and 20% (n = 1,647) of patients receiving dual therapy (ICS/LABA, n = 1,615; LAMA/LABA, n = 32) progressed to triple therapy. Of the patients who progressed to triple therapy, 63% and 57% of patients receiving monotherapy and dual therapy, respectively, progressed in the 12 months after the index date. In the 12 months before initiation of triple therapy, approximately 50% of patients in the LAMA monotherapy, ICS/LABA, and LAMA/LABA therapy groups had an exacerbation. In the multivariable analysis, discontinuation of therapy, smoking history, and concomitant use of xanthenes and short-acting beta2-agonists were significant predictors of progression from index therapy to triple therapy.
Approximately 25% of patients with COPD progressed to triple therapy within 12 months of initiating treatment with monotherapy or dual therapy. Exacerbations were reported in only 50% of these patients, indicating that the other 50% may have escalated to triple therapy for other reasons. Treatment discontinuation, smoking history, the use of a LAMA, and concomitant medication use were significant predictors of progression to triple therapy.
This study was a GlaxoSmithKline-sponsored collaborative research study (HO-14-16145). GlaxoSmithKline funded this study and had a role in study design, data analysis, data interpretation, and writing of this report. Stemkowski is a paid employee of Comprehensive Health Insights, which is a wholly owned subsidiary of Humana and was contracted to conduct the study. No funding was provided to Comprehensive Health Insights for manuscript development. At the time of the study, Lane and Tao were paid employees of Comprehensive Health Insights. Stanford is an employee of and stockholder in GlaxoSmithKline.
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