Patient-Reported Outcome Measures for Severe Recurrent Bilateral Nasal Polyps: Psychometric Evaluation and Content Validity.

Objective: To date, no patient-reported outcome measures have been specifically developed to assess pharmacological treatment effect in participants with severe chronic rhinosinusitis (CRS) with recurrent bilateral nasal polyps (NP). These studies aimed to assess (1) the psychometric properties and (2) content validity of Visual Analogue Scales (VAS) assessing NP symptom severity. Study Design: (1) Retrospective psychometric validation study using clinical trial data and (2) cross-sectional qualitative patient interview study. Setting: (1) Multicentre trial; (2) real-world. Methods: (1) Psychometric validation was performed using data from a randomized, double-blind, placebo-controlled, Phase II study (NCT01362244) investigating the effect of mepolizumab in 105 participants with severe, recurrent bilateral NP currently needing polypectomy surgery. (2) Content validity was explored through cognitive debriefing interviews in 27 adults with severe CRS with recurrent bilateral NP who had received NP surgery in the past 10 years (NCT03221192). Results: (1) Acceptable reliability, validity, and responsiveness were shown for individual VAS items, although the loss of smell VAS item performed poorly in several analyses, suggesting further evaluation of this item is needed. (2) All individual VAS items were well understood, considered relevant and were consistently interpreted by most participants, providing evidence for their content validity. Conclusion: These findings support the use of symptom VAS measures to evaluate disease experience and treatment effect in clinical trials of participants with severe CRS with recurrent bilateral NP.

Understanding the Patient Experience of Severe, Recurrent, Bilateral Nasal Polyps: A Qualitative Interview Study in the United States and Germany.

OBJECTIVES: To qualitatively explore patient experiences of severe, recurrent, bilateral nasal polyps (NP). METHODS: A targeted literature review of published qualitative studies and online blogs describing patient experiences of NP was conducted. Semistructured concept elicitation interviews were conducted in the United States and Germany with participants ≥18 years with severe, recurrent, bilateral NP to explore their symptom experience and impacts on health-related quality of life (HRQoL; NCT03221192). A subset of 10 participants reported symptoms and impacts using a smartphone or tablet application (app) over a 10-day period. RESULTS: A paucity of qualitative evidence regarding patient experience of NP was identified from the literature or blog review. Twenty-seven participant interviews were conducted. Thirty-six symptoms were identified, including 7 primary symptoms (nasal congestion [n = 27 of 27], breathing difficulties [n = 27 of 27], postnasal drip [n = 25 of 27], runny nose [n = 24 of 27], head/facial pressure [n = 23 of 27], loss of smell [n = 23 of 27], loss of taste [n = 22 of 27]) and 29 secondary symptoms (the most common were mucus/catarrh and nose bleeds [both n = 20 of 27]). Most symptoms were reported to vary both within and between days. Sixty impacts of severe NP were reported, including impacts on sleep (n = 22 of 27), physical functioning (n = 21 of 27), activities of daily living (n = 21 of 27), emotional well-being (n = 27 of 27), treatment (n = 23 of 27), social life (n = 26 of 27), and work (n = 19 of 27). Symptoms/impacts reported using the app were consistent with interview findings, although new symptoms were identified (ear pain, throat pain, nasal scabs, and nasal burning). These results supported the development of a conceptual model outlining concepts related to symptoms, impacts, and treatment of NP. CONCLUSIONS: Severe, recurrent, bilateral NP are associated with a range of symptoms that have significant detrimental impact on HRQoL.

Healthcare costs and utilization associated with muscle weakness diagnosis codes in patients with chronic obstructive pulmonary disease: a United States claims analysis.

AIMS: Muscle weakness (MW)-attributable healthcare resource utilization (HCRU) and costs in patients with chronic obstructive pulmonary disease (COPD) have not been well-characterized in US insurance claims databases. The primary objective of this study was to estimate HCRU in patients with evidence of COPD with and without MW diagnosis codes. MATERIALS AND METHODS: This retrospective analysis used the MarketScan® Commercial Claims and Encounters and Medicare Supplemental and Coordination of Benefits databases. Between January 2007 and March 2016, we identified patients aged ≥40 years with diagnosis codes for COPD (≥1 emergency department or inpatient claim or ≥2 outpatient claims within 1 year). The cohort was divided into patients with and without ≥1 MW diagnosis code. Propensity score matching was used to generate pairs of patients with and without MW (1:1). Multivariable regression analyses were used to estimate adjusted incremental costs and utilization attributable to the presence of MW diagnosis codes among patients with COPD. RESULTS: Of 427,131 patients who met the study inclusion criteria, 14% had evidence of MW. After matching, 107,420 unique patients remained equally distributed across MW status. Patients with MW diagnosis codes had greater predicted annual HCRU, $2,465 greater total predicted annual COPD-related costs, and $15,179 greater total all-cause costs than those without MW diagnosis codes. Overall, <1% of patients received COPD-related pulmonary rehabilitation services. LIMITATIONS: Study limitations include the potential for undercoding of MW and lack of information on severity of MW in claims data. CONCLUSION: The presence of MW diagnosis codes yielded higher HCRU in this COPD population and suggests that the burden of MW affects both all-cause and COPD-related care. However, utilization of pulmonary rehabilitation, a known effective treatment for MW, remains low. Future research should expand on our results by assessing data sources that allow for clinical confirmation of MW among patients with COPD.

Real-world comparison of treatment patterns and effectiveness of albiglutide and liraglutide.

AIM: To compare medication adherence, discontinuation and glycemic control in patients receiving albiglutide versus liraglutide. PATIENTS & METHODS: Administrative claims data and glycated hemoglobin (HbA1c) results were analyzed from a sample of adult health plan members with Type 2 diabetes. RESULTS: Patients were matched 1:1 in the albiglutide (n = 2213) and liraglutide (n = 2213) overall cohorts and in 244 patients with HbA1c results from each treatment group. Mean HbA1c change from baseline was -1.0% for both groups. At 6 months, mean ± standard deviation adherence was 0.69 ± 0.29 versus 0.64 ± 0.29 (p < 0.001), and discontinuation was 33.2 versus 37.8% (p = 0.002) with albiglutide versus liraglutide, but these were not statistically or clinically different at 12 months. CONCLUSION: Similar treatment patterns and clinically meaningful reductions in HbA1c were observed for both treatments in this real-world comparison.

Patient Preferences for Attributes of Type 2 Diabetes Mellitus Medications in Germany and Spain: An Online Discrete-Choice Experiment Survey.

INTRODUCTION: Understanding patient preferences for attributes of type 2 diabetes mellitus (T2DM) medications may help explain how the attributes differentially affect patient perceptions and behaviors. In this survey, we quantified the relative preferences among patients in Germany and Spain in separate analyses. METHODS: A stated-preference, discrete-choice experiment (DCE) survey was designed to elicit preferences for T2DM treatment attributes among patients with self-reported T2DM and who reported being prescribed T2DM medication for > 2 years. Patients recruited from an online national consumer panel completed an online survey. The survey presented choices between eight pairs of hypothetical T2DM treatments defined by seven attributes: chance of reaching target hemoglobin A1c (HbA1c) level; reduced risk of serious heart attack or stroke; frequency of hypoglycemia; risk of gastrointestinal (GI) problems; weight change; mode of administration (oral or injectable); dosing frequency. Data were analyzed using random-parameters logit. Minimum acceptable benefit (MAB) was defined as the minimum increase in the probability of reaching target HbA1c for which respondents would accept less desirable levels of other attributes. RESULTS: In Germany and Spain, 474 and 401 respondents completed the survey, respectively. DCE analysis showed that risk of GI problems was most important to German respondents. MAB analysis found that respondents would require a 56 percentage point increase in the probability of reaching their HbA1c target to offset a change from 0% to 30% risk of GI problems. For Spanish respondents, mode of administration was the most important attribute. These respondents would require a 59 percentage point increase in the probability of reaching their HbA1c target to offset moving from oral to injectable medications. CONCLUSIONS: Respondents in Germany and Spain were willing to trade efficacy for improvements in side effects and mode of administration. Given the variety of T2DM medications currently available, the results suggest that careful discussion about patient preferences could help improve patient satisfaction with T2DM treatment.

Reasons for discontinuation of GLP1 receptor agonists: data from a real-world cross-sectional survey of physicians and their patients with type 2 diabetes.

AIM: Nonadherence to glucagon-like peptide-1 receptor agonists (GLP1 RAs) is relatively common among patients with type 2 diabetes mellitus (T2DM). This study sought to identify reasons why patients discontinue GLP1 RAs. MATERIALS AND METHODS: Retrospective data from the Adelphi Diabetes Disease Specific Programme were used. Physicians managing patients with T2DM were surveyed via face-to-face interviews, and patients treated for T2DM were surveyed via self-completed questionnaires. Patient data were stratified by current versus prior GLP1 RA use. RESULTS: Physicians (n=443) most frequently reported inadequate blood glucose control (45.6%), nausea/vomiting (43.8%), and gastrointestinal (GI) side effects (36.8%) as reasons for GLP1 RA discontinuation. Patients (n=194) reported the GI-related issues "Made me feel sick" (64.4%) and "Made me throw up" (45.4%) as their top reasons for discontinuation. The most common problems reported (excluding cost) for those currently using GLP1 RAs were "Prefer oral medication over injections" (patients 56%, physicians 32.6%), "Made me feel sick" (patients 38.1%, physicians 16.3%), and "Did not help lose weight" (patients 25.4%, physicians 18%). The most bothersome problems for patients globally (frequency reporting very/extremely bothersome) (excluding cost) were "Difficult to plan meals around" (55.6%), "Made me throw up" (51.6%), and "Caused weight gain" (50%). CONCLUSION: Both patients and physicians reported GI-related issues as a prominent factor, but disparities between patient experiences and physician perceptions were revealed, suggesting gaps in physician-patient communication. Understanding patients' expectations of GLP1 RAs and physicians' patient-management practices may help increase GLP1 RA adherence and thereby potentially enhance diabetes care.

Monitoring anticoagulation in atrial fibrillation.

BACKGROUND: Randomized control trials and observational studies show high-quality warfarin therapy leads to safe and effective stroke prophylaxis. In usual community practice, patient, physician and health care system factors are barriers to optimal anticoagulation. We examined the predictive relationship between inpatient and outpatient INR values in chronic non-valvular atrial fibrillation (AF) patients hospitalized for ischemic stroke (S), bleed (B) and control events (C) in usual community practice. METHODS: This nested case-control analysis identified AF patients hospitalized for S, B and C using medical and pharmacy claims spanning 4.5 years ('98-'03) and validating diagnosis with chart abstraction. AF was defined as 2 medical claims for AF >or= 42 days apart with a related prescription claim for warfarin. INRs from both outpatient and inpatient settings were used to yield a continuous history of coagulation status. Time-in-therapeutic-range (TTR) was calculated by Rosendaal's linear interpolation method. Correlation of inpatient and prognostic utility of last outpatient INRs was tested with S or B hospitalizations using univariate and multivariate logistic regression. RESULTS: Overall, 614 hospitalizations (means: age 73.9, CHADS(2) = 3.24; 52% male) included S (n = 98), B (n = 101) and C (n = 415) events. Average TTR was 28.6% (49.4% at INR <2.0, 21.9% at INR >3.0). First INR on admission (INR <2.0 or >3.0) was associated with S and B hospitalizations (OR-adjusted [95%CI], 1.68 [1.04-2.73] and 1.72 [1.02-2.90]), respectively. Last outpatient INR <2.0 was not associated with S (OR-adjusted [95%CI], 1.12 [0.77-1.81]), and INR >3.0 was not associated with B (OR-adjusted [95%CI], 1.25 [0.67-2.32]). Last outpatient INR measurement occurred at 28, 22 and 24 days (median; S, B & C, respectively) before hospitalization. CONCLUSION: Patients were observed within therapeutic range less than 30% of their time on warfarin. While inpatient INRs were clearly associated with both ischemic stroke and bleed events, last outpatient INR before event was not predictive.