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Background Many people with systemic lupus erythematosus (SLE) experience joint pain, swelling, and stiffness. These joint symptoms are associated with problems in physical functioning and work disability. We used survey data from adults with SLE to explore the burden and impact of joint symptoms. Methods SLE-UPDATE was a 2019 cross-sectional US survey of adults with SLE. We compared respondents with “currently active” joint symptoms’ and those “without currently active” joint symptoms. The active joint cohort comprised survey respondents who self-reported current “stiffness in joints” or “pain/swelling in joints” and who had moderate to severe joint pain (Worst Joint Pain Numeric Rating Scale [NRS] score ≥ 4). Respondents not fulfilling these criteria were included in the non-active joint cohort. Outcomes included frequency and severity of pain, patient-reported outcomes (LupusPRO™ and Work Productivity and Activity Impairment: Lupus [WPAI-Lupus]), satisfaction with current treatments, and importance of different treatment goals. Results More respondents in the active joint cohort (N = 285) than in the non-active joint cohort (N = 215) reported pain most or all the time over the preceding 7 days (77.5% vs. 32.1%, p < .0001), fibromyalgia (45% vs. 12%, p < .0001), and higher (worse) mean scores on the Worst Pain NRS (6.5 vs. 4.8, p < .0001) and Worst Joint Pain NRS (6.7 vs. 4.5, p < .0001). Mean Lupus PRO health-related quality of life (HRQoL) total score was lower (worse) in the active joint cohort (48.9 vs. 64.1, p < .0001). WPAI-Lupus scores indicated greater work productivity losses and activity impairment in the active joint cohort. More respondents in the active joint cohort than in the non-active joint cohort were neutral or not satisfied with current treatments and rated reducing pain as a “very important” treatment goal (26.7% vs. 18.1%). Conclusions Respondents with SLE and active joint manifestations in addition to having more pain report lower HRQoL and were less satisfied with their current treatments. Comorbid fibromyalgia may play a role in joint symptoms in patient with SLE joint manifestations. There is an unmet need for new therapeutic options to reduce joint symptom burden among patients with SLE.

Score reproducibility is an important measurement property of fit-for-purpose patient-reported outcome (PRO) measures. It is commonly assessed via test-retest reliability, and best evaluated with a stable participant sample, which can be challenging to identify in diseases with highly variable symptoms. To provide empirical evidence comparing the retrospective (patient global impression of change [PGIC]) and current state (patient global impression of severity [PGIS]) approaches to identifying a stable subgroup for test-retest analyses, 3 PRO Consortium working groups collected data using both items as anchor measures.The PGIS was completed on Day 1 and Day 8 + 3 for the depression and non-small cell lung cancer (NSCLC) studies, and daily for the asthma study and compared between Day 3 and 10. The PGIC was completed on the final day in each study. Scores were compared using an intraclass correlation coefficient (ICC) for participants who reported "no change" between timepoints for each anchor.ICCs using the PGIS "no change" group were higher for depression (0.84 vs. 0.74), nighttime asthma (0.95 vs. 0.53) and daytime asthma (0.86 vs. 0.68) compared to the PGIC "no change" group. ICCs were similar for NSCLC (PGIS: 0.87; PGIC: 0.85).When considering anchor measures to identify a stable subgroup for test-retest reliability analyses, current state anchors perform better than retrospective anchors. Researchers should carefully consider the type of anchor selected, the time period covered, and should ensure anchor content is consistent with the target measure concept, as well as inclusion of both current and retrospective anchor measures.

The Living with Idiopathic Pulmonary Fibrosis (L-IPF) questionnaire was developed with substantial input from patients with idiopathic pulmonary fibrosis (IPF) to assess symptoms and health-related quality of life (HRQoL). Because IPF is the prototypical chronic fibrosing interstitial lung disease (ILD) with a progressive phenotype, we expanded applicability of the L-IPF by deleting the word “idiopathic”, creating the L-PF (Living with Pulmonary Fibrosis) questionnaire, and then assessed its relevance among patients with progressive fibrosing ILDs in one-to-one interviews.Patients in the USA and Germany with any progressive fibrosing ILD other than IPF were asked about their disease and symptoms, completed the 44-item L-PF questionnaire (comprising two modules that assess symptoms and impacts of disease) and then answered a series of debriefing questions. Interviews were recorded, transcribed and coded for qualitative content analysis.20 patients were interviewed, but time constraints meant not all were asked about all items. The most frequent diagnoses were rheumatoid arthritis-associated ILD (25%) and mixed connective tissue disease-associated ILD (20%). Almost all patients endorsed the symptoms assessed by the L-PF: shortness of breath (19 out of 20 patients), cough (19 out of 20) and fatigue (18 out of 20). Most patients endorsed impacts of progressive fibrosing ILD on activities of daily living, physical well-being, sleep, emotional well-being, and social aspects of their lives. Most patients had an overall positive impression of the Symptoms module and understood items as intended. All seven patients asked understood the items of the Impacts module.The L-PF contains concepts relevant and important to patients with progressive fibrosing ILD, and items are understood as intended.

Objective The aim of this study was to perform psychometric testing of the Growth Hormone Deficiency-Child Impact Measure (GHD-CIM): a patient-reported outcome (PRO) for children with GHD aged 9 to 0.70), as was test–retest reliability for the SWB, EWB and overall (above 0.70). At least one convergent validity hypotheses for each domain and overall was proven (r > 0.40). Known-groups validity hypotheses for the EWB and SWB domains were significant (p

Rationale: Patients with chronic obstructive pulmonary disease (COPD) struggle with respiratory symptoms that impair their daily activities and quality of life. Understanding a treatment’s ability to relieve symptoms requires precise assessment. The Evaluating Respiratory Symptoms in COPD (E-RS(TM):COPD) was developed to quantify respiratory symptoms in clinical trials. This review study aimed to better understand how trials use this patient-reported outcome measure as an endpoint, as well as its responsiveness and performance relative to other outcome measures. Objectives: To summarize the use of the E-RS:COPD in pharmacological trials since its qualification by regulatory authorities. Methods: A rapid systematic literature review, using key biomedical databases to identify English language full-text publications of randomized controlled trials (RCTs) that included the E-RS:COPD as an endpoint (2010-2020), was conducted. Two investigators independently screened the publications and extracted data. Measurements and Main Results: Of 219 screened records, 28 full-text publications were included, and data from 17 reporting 20 unique double-blind RCTs were synthesized. The E-RS:COPD was positioned as a primary or secondary endpoint in 6 publications (35%), and served as an exploratory or additional endpoint in 11 (65%). Statistically significant E-RS:COPD treatment effects versus placebo/comparator were found in 13 of the 14 publications reporting symptom results. E-RS:COPD effects corresponded well with other outcome measures (e.g., St George's Respiratory Questionnaire [SGRQ] and forced expiratory volume in 1 second [FEV(1)]). Two publications reported the number of responders. Conclusions: E-RS:COPD is sensitive to treatment effects in clinical trials testing drug therapies. Presentation of trial results should include responder analyses to facilitate interpretation and application of results.

A survey was conducted in eight countries to examine conversations around, and experiences and treatments during, severe hypoglycemia among people with diabetes and caregivers of people with diabetes. This article reports a subgroup analysis from the United States involving 219 people with diabetes and 210 caregivers. Most respondents (79.7%) did not use professional health care services during their most recent severe hypoglycemic event, and 40.3% did not report the event to their health care providers at a subsequent follow-up visit. Hypoglycemic events left respondents feeling scared (70.9%), unprepared (42.7%), and helpless (46.9%). These clinically important psychosocial impacts on people with diabetes and caregivers underscore the need for conversations about hypoglycemia prevention and management.

BackgroundThere is a lack of fully validated patient-reported outcome measures for progressive fibrosing interstitial lung disease (ILD). We aimed to validate the King's Brief Interstitial Lung Disease (K-BILD) questionnaire for measuring health-related quality of life (HRQoL) in these patients. We also aimed to estimate the meaningful change threshold for interpreting stabilisation of HRQoL as a clinical end-point in progressive fibrosing ILD, where the current goal of treatment is disease stability and slowing progression.MethodsThis analysis evaluated data from 663 patients with progressive fibrosing ILD other than idiopathic pulmonary fibrosis from the INBUILD trial. Validation of the measurement properties was assessed for internal consistency, test–retest reliability, construct validity, known-groups validity and responsiveness. We calculated meaningful change thresholds for treatment response using anchor-based (within-patient) and distribution-based methods.ResultsK-BILD had strong internal consistency (Cronbach's α was 0.94 for total score, 0.88 for breathlessness and activities, 0.91 for psychological, and 0.79 for chest symptoms). The test–retest reliability intraclass correlation coefficient was 0.74 for K-BILD total score. K-BILD demonstrated weak correlations with forced vital capacity (FVC) percent predicted. Known-groups validity showed significant differences in K-BILD scores for patient groups with different disease severity based on use of supplemental oxygen or baseline FVC % pred (≤70% or >70%). We estimated a meaningful change threshold of ≥ –2 units for K-BILD total score for defining patients who remain stable/improved versus those with progressive deterioration.ConclusionsOur results validate K-BILD as a tool for assessing HRQoL in patients with progressive fibrosing ILD and set a meaningful change threshold of ≥ –2 units for K-BILD total score.

Background Niemann-Pick Disease, type C1 (NPC1) is a rare neurodegenerative genetic disorder characterized by impaired intracellular transport of cholesterol and other lipids. The Niemann-Pick Disease, type C1 Severity Scale (NPC-SS) was developed to quantify neurological progression of NPC; it is used to monitor the natural history of disease progression and assess response to treatment. Objective The objective of the study was to examine the inter-rater reliability of the NPC-SS in a Phase 2/3 clinical trial. Methods Study data were from a multicenter, prospective, randomized, double-blind, trial of adrabetadex in 56 subjects with NPC1. Clinical data recorded at each study site were distributed to two independent blinded central raters to generate a severity score. A composite four-item score was utilized as the primary clinical study endpoint, while a five-item focused score has been utilized in other NPC1 trials. Inter-rater reliability was assessed using two-way mixed models for instrument stability, Cohen's kappa, weighted kappa, and percent agreement for the four-item and five-item scores. Results The frequency distribution and mean (SD) of the NPC-SS domain assessments by the raters were almost identical. Evaluation at the patient visit level showed wide variability between visits, however, weighted kappa calculation provided a lower variability between visits. The average kappa coefficients ranged between 0.69 to 0.89, indicating good to very good agreement between raters. Conclusion These results support the NPC-SS, including derived 4- and 5-item composite scores, as reliable measures for use in a clinical trial setting.

BackgroundThe Living with Pulmonary Fibrosis (L-PF) questionnaire assesses symptoms and quality of life in patients with fibrosing interstitial lung diseases (ILDs). Its Dyspnoea and Cough domains, whose items’ responses are based on a 24-hour recall, have scores ranging from 0 to 100, with higher scores indicating greater symptom severity. We evaluated the ability of these domain scores to detect change and estimated their meaningful change thresholds in patients with progressive fibrosing ILDs.MethodsThe INBUILD trial enrolled subjects with progressive fibrosing ILDs other than idiopathic pulmonary fibrosis. The L-PF questionnaire was completed at baseline and week 52. The responsiveness of the Dyspnoea and Cough scores was evaluated by comparing changes in these scores with 52-week changes in three anchors: forced vital capacity % predicted and two self-reported items, one for global physical health and one for global quality of life. We used a triangulation approach including anchor-based and distribution-based methods to estimate meaningful change thresholds.ResultsThe analyses included 542 subjects with an L-PF Dyspnoea score at baseline and week 52, and 538 subjects with an L-PF Cough score at baseline and week 52. The L-PF Dyspnoea and Cough scores were responsive to change over 52 weeks. Triangulation of anchor-based and distribution-based estimates resulted in meaningful change thresholds of 6 to 7 points for the L-PF Dyspnoea score and 4 to 5 points for the L-PF Cough score to differentiate subjects who were stable or improved from those who deteriorated.ConclusionThese analyses support the responsiveness, one aspect of validity, of the L-PF Dyspnoea and Cough domains scores as measures of symptom severity in patients with progressive fibrosing ILDs. Estimates for meaningful change thresholds in these domain scores may be of value in interpreting the effects of interventions in these patients.Trial registration numberNCT02999178.

Background: Further validation of K-BILD questionnaire content is needed in PF-ILD. Aim: Assess relevance and comprehension of K-BILD questionnaire content in PF-ILD. Methods: 20 adult ILD patients (US [n=15]; Germany [n=5]) with prespecified PF-ILD criteria (eg, ≥10% relative decline in FVC%pred) were interviewed. After concept elicitation, participants completed the 15-item K-BILD questionnaire assessing 3 domains (breathlessness/activities, psychological and chest symptoms) within the past 2 weeks, followed by an interview. Interviews were audio-recorded, transcribed and coded for qualitative content analysis using ATLAS.ti. Results: Reporting values as median (range), participants were aged 73 (48–81) years with 67% (47–95) FVC%pred. Rheumatoid arthritis-associated (25%), hypersensitivity pneumonitis (20%) and other fibrosing (30%) ILD were amongst the underlying ILD diagnoses for this cohort. Breathlessness was one of the most frequent and impactful K-BILD items reported by patients. Comprehension of K-BILD time period was high, with most participants stating they were thinking of the past 2 weeks (n=14/20, 70%) and 100% (n=19/19) reporting ability to recall 2 weeks’ time. K-BILD items were well understood by participants, capturing relevant aspects of PF-ILD affecting most participants (Figure). Conclusion: For patients with PF-ILD, K-BILD is well understood and contains relevant concepts.

The CRASH online survey examined the experience and treatment of a severe hypoglycemic event (SHE) in people with T1DM or insulin-treated T2DM (PWD) or caregivers (CG). Eligible participants experienced ≥1 SHE in the last 3 years with insulin treatment at the time of event. Reported here are results from PWD aged ≥65 (T1DM 74, T2DM 104) and CG (T1DM 95, T2DM 231) from Canada, Germany, Spain, UK, and USA. During the last SHE, reported glucagon use was low for T1DM (9.5%) and T2DM (7.5%); primary reason reported was no prescription available or filled (T1DM 24.3%, T2DM 29.3%). Of those who ever discussed SHE at their healthcare provider (HCP) visit (T1DM 138, T2DM 243), less than half (T1DM 41.3%, T2DM 34.2%) reported discussion of SHE at every HCP visit. No discussion of the most recent SHE occurred for 34.3% (T1DM) and 31.0% (T2DM). During the most recent SHE many felt unprepared (T1DM 35.5%, T2DM 47.2%), scared (T1DM 60.9%, T2DM 64.2%), and helpless (T1DM 39.1%, T2DM 51.3%). After the last SHE, changes were reported to insulin regimens, meal plans, carrying sugar/sweets, checking blood glucose more often or using continuous glucose monitors, and increasing access to glucagon. Clinical guidelines recommending discussion of hypoglycemia at each HCP visit for PWD at risk for SHE are not being met and should occur, and CGs should be included in preparedness strategies. Disclosure F.J. Snoek: Advisory Panel; Self; Abbott, Lilly Diabetes, Novo Nordisk A/S, Roche Diabetes Care. Research Support; Self; Sanofi. E. Spaepen: None. D.M. Bushnell: None. C.J. Child: Employee; Self; Eli Lilly and Company. Stock/Shareholder; Self; Eli Lilly and Company. Z. Balantac: Other Relationship; Self; Eli Lilly and Company. B. Mitchell: Employee; Self; Eli Lilly and Company. Stock/Shareholder; Self; Eli Lilly and Company. M. Peyrot: Advisory Panel; Self; Eli Lilly and Company. Consultant; Self; CeQur Corporation, Eli Lilly and Company. Research Support; Spouse/Partner; Eli Lilly and Company. Funding Eli Lilly and Company

Objective: The Patient Assessment for Low Back Pain–Symptoms (PAL-S) and the Patient Assessment for Low Back Pain–Impacts (PAL-I) were developed to incorporate patient perspective of treatment bene...

Background This study’s purpose was to assess the minimal important difference (MID) for the Treatment-Related Impact Measure—Adult Growth Hormone Deficiency (TRIM–AGHD), a patient-reported outcome measure assessing growth hormone deficiency (GHD) impacts. The measure was demonstrated to have adequate psychometric measurement properties, and be reliable and valid. For scores to be interpretable, the TRIM-AGHD must be responsive to treatment benefit and the MID in scores quantified. Methods A prospective, non-interventional, observational, clinic-based survey study of naïve-to-treatment adult GHD patients (N = 98) was conducted. Key assessments were at baseline and follow-up (between 4 and approximately 8 weeks), with weekly telephone monitoring post-baseline (last n = 34 patients). Responsiveness was evaluated using the effect size of change scores from baseline to follow-up. MID estimates were derived from distribution-based (half standard deviation [0.5 SD], standard error of measurement [SEm]) and anchor-based methods (patient global rating of change [PGRC]) using change scores from baseline to initial report of minimal improvement in GHD severity. Findings from each method were converged to establish an acceptable MID. Results Patients were mean age 49.7 years, 65.6% female, and 76.0% Caucasian. The TRIM-AGHD was highly responsive to treatment with the total score effect size being 1.38. For the total score, the 0.5 SD was 8.09 and the SEm was 2.66. The difference found using the PGRC was 20.43. The converged MID value for the total score was 10 points. Conclusions The TRIM-AGHD is a highly responsive measure assessing AGHD treatment impacts. A 10-point change score is considered a clinically meaningful improvement.

The Patient Assessment for Low Back Pain–Symptoms is a newly developed patient-reported outcome instrument to assess specific symptoms of chronic low back pain for use in clinical trials and practice., We describe the mixed-methods (qualitative and quantitative) development and preliminary validation of the Patient Assessment for Low Back Pain–Symptoms (PAL-S), a patient-reported outcome measure for use in chronic low back pain (cLBP) clinical trials. Qualitative methods (concept elicitation and cognitive interviews) were used to identify and refine symptom concepts and quantitative methods (classical test theory and Rasch measurement theory) were used to evaluate item- and scale-level performance of the measure using an iterative approach. Patients with cLBP participated in concept elicitation interviews (N = 43), cognitive interviews (N = 38), and interview-based assessment of paper-to-electronic mode equivalence (N = 8). A web-based sample of patients with self-reported cLBP participated in quantitative studies to evaluate preliminary (N = 598) and revised (n = 401) drafts and a physician-diagnosed cohort of patients with cLBP (N = 45) participated in preliminary validation of the measure. The PAL-S contained 14 items describing symptoms (overall pain, sharp, prickling, sensitive, tender, radiating, shocking, shooting, burning, squeezing, muscle spasms, throbbing, aching, and stiffness). Item-level performance, scale structure, and scoring seemed to be appropriate. One-week test–retest reproducibility was acceptable (intraclass correlation coefficient 0.81 [95% confidence interval, 0.61-0.91]). Convergent validity was demonstrated with total score and MOS-36 Bodily Pain (Pearson correlation −0.79), Neuropathic Pain Symptom Inventory (0.73), Roland-Morris Disability Questionnaire (0.67), and MOS-36 Physical Functioning (−0.65). Individual item scores and total score discriminated between numeric rating scale tertile groups and painDETECT categories. Respondent interpretation of paper and electronic administration modes was equivalent. The PAL-S has demonstrated content validity and is potentially useful to assess treatment benefit in cLBP clinical trials.

Objective To identify patient-reported outcome (PRO) instruments that assess chronic low back pain (cLBP) symptoms (specifically pain qualities) and/or impacts for potential use in cLBP clinical trials to demonstrate treatment benefit and support labeling claims. Design Literature review of existing PRO measures. Methods Publications detailing existing PRO measures for cLBP were identified, reviewed, and summarized. As recommended by the US Food & Drug Administration (FDA) PRO development guidance, standard measurement characteristics were reviewed, including development history, psychometric properties (validity and reliability), ability to detect change, and interpretation of observed changes. Results Thirteen instruments were selected and reviewed: Low Back Pain Bothersomeness Scale, Neuropathic Pain Symptom Inventory, PainDETECT, Pain Quality Assessment Scale Revised, Revised Short Form McGill Pain Questionnaire, Low Back Pain Impact Questionnaire, Oswestry Disability Index, Pain Disability Index, Roland-Morris Disability Questionnaire, Brief Pain Inventory and Brief Pain Inventory Short Form, Musculoskeletal Outcomes Data Evaluation and Management System Spine Module, Orebro Musculoskeletal Pain Questionnaire, and the West Haven-Yale Multidimensional Pain Inventory Interference Scale. The instruments varied in the aspects of pain and/or impacts that they assessed, and none of the instruments fulfilled all criteria for use in clinical trials to support labeling claims based on recommendations outlined in the FDA PRO guidance. Conclusions There is an unmet need for a validated PRO instrument to evaluate cLBP-related symptoms and impacts for use in clinical trials.

The CRASH study examines the experience and treatment of a severe hypoglycemia event (SHE) among people with T1DM (ppl w/T1DM) or insulin-treated ppl w/T2DM and caregivers. The online survey was conducted in 8 countries. To be eligible in the global study, ppl had experienced or cared for a person who had ≥1 SHE in the last 3 years. SHE was defined as low blood glucose requiring assistance for recovery. Here, we report U.S. data for 220 ppl with diabetes (n=110 T1DM; n=110 T2DM). Sixty-five (59.1%) ppl w/T2DM used insulin for >5 years, while mean (SD) duration of diabetes was 30.6 (16.6) years for ppl w/T1DM and 16.0 (8.7) years for ppl w/T2DM. Forty (36.4%) ppl w/T1DM and 22 (20.0%) ppl w/T2DM reported impaired awareness (Gold score ≥4). During the last SHE, glucagon use was low (T1DM [8.2%]; T2DM [3.6%]); reasons for not using glucagon were not having a prescription (T1DM [40.0%]; T2DM [59%]) or it was unavailable (T1DM [20.0%]; T2DM [17.1%]). Also, ppl w/T1DM (75.5%) and ppl w/T2DM (83.6%) reported using oral glucose only for treatment; use of healthcare provider assistance was low. The main causes reported for a SHE were eating less than planned (T1DM [27.3%]; T2DM [47.3%]) or exercising more than planned (T1DM [21.8%]; T2DM [20.0%]). Results indicate that treatment of a SHE mainly occurred outside the healthcare system with fewer ppl having glucagon as an option for recovery. Disclosure F.J. Snoek: Advisory Panel; Self; AstraZeneca, Lilly Diabetes, Novo Nordisk A/S, Roche Diabetes Care. Research Support; Self; Sanofi. Speaker's Bureau; Self; Lilly Diabetes. A. Jiletcovici: Employee; Self; Eli Lilly and Company. D.M. Bushnell: None. C.J. Child: Employee; Self; Eli Lilly and Company. Stock/Shareholder; Self; Eli Lilly and Company. S.K. Bajpai: Employee; Self; Eli Lilly and Company. E. Spaepen: Consultant; Self; Eli Lilly and Company. J. Thomas: Employee; Self; Eli Lilly and Company. Z. Balantac: Other Relationship; Self; Eli Lilly and Company. M. Peyrot: Advisory Panel; Self; Eli Lilly and Company. Consultant; Self; Calibra Medical. Research Support; Spouse/Partner; Eli Lilly and Company. B. Mitchell: Employee; Self; Eli Lilly and Company. Stock/Shareholder; Self; Eli Lilly and Company.

It is important to understand patients' experiences of statin-associated adverse effects to potentially identify those at risk for stopping treatment.The goal of the STatin Adverse Treatment Experience survey was to describe patients' experiences after reporting ≥1 recent statin-associated adverse event and identify opportunities to improve adherence and outcomes.The survey was developed in 3 stages: qualitative item development, pilot evaluation of initial item performance, and quantitative evaluation using a large commercial sample. Respondents with self-reported high cholesterol who had taken a statin in the past 2 years and experienced ≥1 statin-associated symptom in the past 6 months were included (N = 1500).Mean age was 58 years, 40.3% were men, and 43.2% had tried ≥2 statins. Many had clinical comorbidities associated with increased risk for cardiovascular disease (atherosclerotic cardiovascular disease, 22.5%; diabetes, 25.8%; hypertension, 56.0%). The most important patient-reported reasons for continuing current statin therapy (n = 1168; 77.9%) were avoiding a heart attack or stroke, lowering cholesterol, and doctor recommendation. Being bothered by and not being able to tolerate side effects were the main reasons respondents discontinued statins (n = 332; 22.1%). Respondents who discontinued statins reported significantly higher mean Symptom Severity (10.6 vs 8.7, P .001) and Impact Severity scores (11.8 vs 9.8, P .001) compared with those who continued.The STatin Adverse Treatment Experience survey highlights the importance of patients' adverse experiences with statins and how symptom and impact scores affect decisions to continue or discontinue therapy. These data provide a foundation to increase providers' awareness of statin tolerability from the patient's perspective and encourage benefit-risk discussions.

Background The Symptoms of Major Depressive Disorder Scale (SMDDS) was expressly developed on the basis of qualitative data to directly incorporate patients' voices into evaluation of treatment benefit in major depressive disorder (MDD) clinical trials. Objectives To collect quantitative data necessary to refine/optimize the SMDDS and document its psychometric properties. Methods In this multicenter, observational study, participants with clinically diagnosed MDD completed questionnaires in 2 waves. Wave 1 was designed to refine the SMDDS using Rasch measurement evaluations and item reduction analyses. On a subset of wave 1 subjects, 7 to 12 months later, wave 2 further examined item performance and measurement properties. Exploratory factor analyses and assessments of construct validity and reliability (internal consistency and reproducibility) were completed. Results Using wave 1 data (N = 315; females = 71%, white = 81%, mean age = 44 years), the SMDDS was revised from 36 to 16 items. The Rasch item threshold map indicated that all but 1 item (suicidal ideation) were appropriately ordered. The 207 wave 2 participants were 74% females, 82% white, with a mean age of 45 years. The exploratory factor analyses resulted in a single component (all standardized factor loadings >0.46). Cronbach α was 0.93 and the 7-day test-retest intraclass correlation coefficient (n = 93) was 0.84 (95% confidence interval 0.77-0.89). SMDDS scores discriminated between MDD severity levels. Conclusions The 16-item SMDDS generated highly reliable scores with substantial evidence of construct validity. On the basis of the evidence of appropriate content validity and sound psychometric performance, the Food and Drug Administration qualified the SMDDS as an outcome measure to support exploratory efficacy endpoints in MDD clinical trials.

The Patient Assessment for Low Back Pain–Impacts is a newly developed patient-reported outcome instrument to assess specific impacts of chronic low back pain for use in clinical trials and practice., We describe qualitative and quantitative development and preliminary validation of the Patient Assessment for Low Back Pain–Impacts (PAL-I), a patient-reported outcome measure for use in chronic low back pain (cLBP) clinical trials. Concept elicitation and cognitive interviews (qualitative methods) were used to identify and refine symptom concepts. Classical test theory and Rasch measurement theory (quantitative methods) were used to evaluate item-level and scale-level performance of the PAL-I using an iterative approach between qualitative and quantitative methods. Patients with cLBP participated in concept elicitation interviews (N = 43), cognitive interviews (N = 38), and assessment of paper-to-electronic format equivalence (N = 8). A web-based sample of self-reported patients with cLBP participated in quantitative studies to evaluate preliminary (N = 598) and revised (n = 401) drafts and patients with physician-diagnosed cLBP (N = 45) participated in preliminary validation of the PAL-I. The instrument contained 9 items describing cLBP impacts (walking, sitting, standing, lifting, sleep, social activities, travelling, climbing, and body movements). Item-level performance, scale structure, and scoring seemed to be appropriate. One-week test–retest reproducibility was acceptable (intraclass correlation coefficient 0.88 [95% confidence interval, 0.78-0.94]). Convergent validity was demonstrated with PAL-I total score and Roland-Morris Disability Questionnaire (Pearson correlation 0.82), MOS-36 Physical Functioning (−0.71), and MOS-36 Bodily Pain (−0.71). Individual item scores and total score discriminated between numeric rating scale tertile groups and painDETECT categories. Interpretation of paper and electronic administration modes was equivalent. The PAL-I demonstrated content validity and is potentially useful to assess treatment benefit in clinical trials of cLBP therapies.

In psoriasis clinical trials, treatment success is often defined as achieving a static Physician Global Assessment (sPGA) score of 0 (clear) or 1 (almost clear). Patients with clear versus almost clear skin may experience psoriasis differently. This study assessed whether aggregating these patients underestimates subjective improvements associated with total skin clearance.Patients with plaque psoriasis with stable sPGA 0 or 1 currently treated with adalimumab, etanercept, infliximab, or ustekinumab reported Psoriasis Symptom Inventory (PSI) scores for seven days and Dermatology Life Quality Index (DLQI) scores on day 8. The PSI measures psoriasis signs and symptom severity; the DLQI measures the impact of skin disease on quality of life. This analysis compared PSI and DLQI outcomes between patients with sPGA 0 and 1.This study assessed 230 patients: 79 sPGA 0 and 151 sPGA 1. A greater percentage with sPGA 0 than sPGA 1 achieved a total PSI score of 0 ("best"; 61% vs. 5%, p 0.0001) and DLQI 0 ("no effect"; 79% vs. 24%, p 0.001). Patients with sPGA 0 reported better scores than sPGA 1 on all other PSI and DLQI assessments.Achieving total skin clearance, compared with almost clear skin, provides clinically meaningful improvements in psoriasis.

Non-severe hypoglycemic events (NSHEs) are commonly experienced by diabetes patients, particularly among insulin users, and can have serious impacts on daily functioning, emotional well-being, sleep, work productivity, and treatment adherence. Currently, no PRO measures are available to assess the impacts of non-severe hypoglycemia. To address this gap, the Treatment-Related Impact Measure-Non-severe Hypoglycemic Events (TRIM-HYPO) was developed. This paper describes the TRIM-HYPO development and validation. The creation of the TRIM-HYPO followed FDA’s guideline for PRO development. Concept elicitation data were gathered from literature review, clinical expert interviews, and focus groups of patients with Type 1 or 2 diabetes in four countries. Based on the qualitative analysis, draft items were generated and cognitively debriefed. Psychometric validation included factor analysis, item response theory analysis, and assessment of psychometric characteristics for the TRIM-HYPO. Eight clinical experts and 167 patients participated in concept elicitation. The validation study included 407 patients. Thirteen of the 46 items from the preliminary measure were dropped due to ceiling/floor effects and high correlations between conceptually similar items. Factor analysis confirmed five domains in the TRIM-HYPO: daily function, emotional well-being, diabetes management, sleep disruption, and work productivity. All scores were internally consistent (0.86–0.95) and reproducible with a test–retest range of 0.75–0.98. All but one a priori hypothesized associations for validity were confirmed. Study findings demonstrate that the final, 33-item TRIM-HYPO is reliable and valid and may be useful for assessing impacts related to NSHEs in research and clinical practice.

Introduction: The Psoriasis Symptom Inventory is a patient-reported outcome instrument that assesses severity of psoriasis signs and symptoms. In early qualitative research, patients reported pain related to psoriasis skin lesions and redness of affected areas of skin as key symptoms. Methods: Individual concept elicitation interviews and cognitive interviews were conducted in adults with moderate to severe plaque psoriasis. Interviews were audio-recorded and transcribed. Concepts were identified, coded and grouped by similar content using Atlas.ti software. Results were evaluated using qualitative research methods. Results: Of 30 patients recruited, 20 patients participated in concept elicitation interviews and 10 participated in cognitive interviews. Concept codes for skin pain and descriptions of color comprised 11% and 15%, respectively, of all symptom-related expressions. Of 90 pain-related expressions, 22 were about pain related to unconscious scratching and 68 were about pain from the psoriasis lesions. Of 199 color-related expressions, 72 were about red or reddish lesion color. Patients with darker skin tones were found to interpret redness consistently. Discussion: These results provide further support to content validity of pain and redness concepts in the Psoriasis Symptom Inventory. Conclusions: Symptoms of skin pain and redness are highly relevant to patients with psoriasis.

Background The Questionnaire on Pain caused by Spasticity (QPS) is a modular patient- and observer-reported outcome measure of spasticity-related pain (SRP) in children with cerebral palsy (CP). Originally developed for an English-speaking population, we conducted a psychometric validation of a recently developed Chinese language version of the QPS. Methods This was a prospective, observational study involving 137 children/adolescents with CP and upper and/or lower limb spasticity and their parents at three sites in China. Six QPS modules were used, three each for upper and lower limb SRP assessment: a patient self-report module; an interviewer-administered module used by site staff based on the cognitive, communicative, and motor abilities of a patient; and a parent/caregiver module administered for all children as an observer-reported outcome to complement the patient-reported outcome. If no assessment by the patient was possible because of age or cognitive impairments, only the parent/caregiver module was completed. Two visits with a 3-week interval provided data to evaluate and establish administrative ease of use, scoring of the QPS (factor analyses, Rasch analyses), reliability (Cronbach’s α, intraclass correlation coefficient), validity (correlations with quality of life [PedsQL™], motor impairment [Gross Motor Function Classification System, Gross Motor Function Measure-66, Manual Ability Classification System], and spasticity [Ashworth Scale, Modified Tardieu Scale]). Results For most children, clinic staff reported no difficulties associated with general QPS use or deciding which module to use. Children (and parents) who reported more demanding activities also reported higher levels of associated SRP (or observed SRP behavior). Activity-related SRP items were combined for a total QPS score. Cronbach’s α was low for child self-report, but was acceptable for interviewer-administered and parent reports on SRP. Test–retest reliability was high for all modules. Moderate–strong associations were frequently seen between QPS and quality of life, and were particularly strong in the child self-report group. Relatively weak associations were observed between QPS and motor impairment and spasticity. Conclusions This first study was successful in providing initial evidence for the psychometric properties. Clinic staff were able to administer the QPS modules easily, and both children and parents were able to complete the designated QPS appropriately. Electronic supplementary material The online version of this article (10.1186/s12955-017-0804-8) contains supplementary material, which is available to authorized users.

Objective: Nonsevere hypoglycemic events are common and may occur in one-third of persons with diabetes as often as several times a week. This study’s objective was to examine the economic burden of nonsevere nocturnal hypoglycemic events (NSNHEs). Methods: A 20minute Web-based survey, with items derived from the literature, expert input, and patient interviews, assessing the impact of NSNHEs was administered in nine countries to 18 years and older patients with self-reported diabetes having an NSNHE in the past month. Results: A total of 20,212 persons were screened, with 2,108 respondents meeting criteria and included in the analysis sample. The cost of lost work productivity per NSNHE was estimated to be between $10.21 (Germany) and $28.13 (the United Kingdom), representing 3.3 to 7.5 hours of lost work time per event. A reduction in work productivity (presenteeism) was also reported. Compared with respondents’ usual blood sugar monitoring practice, on average, 3.6 6.6 extra tests were conducted in the week following the event at a cost of approximately $87.1 per year. Additional costs were also incurred for doctor visits as well as medical care required because of falls or injuries incurred during the NSNHE for an annual cost of $2,111.3 per person per year. When taking into consideration the multiple impacts of NSNHEs for the total sample and the frequency that these events occur, the resulting total annual economic burden was $288,000 or $127 per person per event. Conclusions: NSNHEs have serious consequences for patients. Greater attention to treatments that reduce NSNHEs can have a major impact on reducing the economic burden of diabetes.

BACKGROUND & OBJECTIVE Objectives The Pain Assessment in Low Back Pain Symptoms (PAL-S) is a Patient Reported Outcome (PRO) instrument being developed to assess the key symptoms of chronic low back pain (cLBP). Qualitative development included both concept elicitation and cognitive interviews. As part of the ongoing development of the instrument, we further evaluated and refined the PAL-S using a mixed methods approach. Methods Adults self-reporting a clinical diagnosis of cLBP were recruited from an existing US-based commercial survey panel to participate in a pilot quantitative study. Qualifying participants completed a web-based survey consisting of the 14-item PAL-S and items assessing clinical, treatment, and demographic characteristics. Study data was analyzed to assess itemand scale-level performance of the PAL-S using Rasch Measurement Theory analyses. Following analysis and modification, two waves of cognitive interviews were conducted to evaluate respondent understanding of the revised PAL-S. Results The dataset included 598 respondents (mean age: 55.5±12.6; 67.9% female; 88.0% white; and 54.0% married) who had cLBP for mean of 15.2±11.5 years. The Rasch analyses item threshold maps showed only two items having ordered thresholds, suggesting that respondents experienced increased difficulty distinguishing between options at the lower levels of the 0-10 scale. Simulations collapsing the responses to a best-fit 4-point response scale resulted in improved ordering of thresholds, suggesting a more optimal response option structure. Based on these findings, the numeric response scale of the PAL-S items was replaced with a 4-point verbal rating scale incorporating response choices such as not at all, slight, moderate, and severe. Findings from eight cognitive interviews confirmed patient comprehension and relevance of the revised instrument. Conclusion The mixed-methods approach proved valuable to the ongoing development of the PAL-S, as Rasch analyses identified a need for refinement of the response scale. The measurement properties of the revised PAL-S will be evaluated in additional web-based and clinic-based quantitative studies. Although a number of patient-reported outcome (PRO) instruments are available in low back pain (LBP), none of them were developed based on the most recent FDA PRO Guidance (2009) and would not be acceptable to the FDA to support labeling claims. Consequently, Forest Research Institute and partner Grunenthal in collaboration with experts have developed the Pain Assessment for Lower Back-Symptoms (PAL-S) instrument. The PAL-S was developed using state of the science best practices and in accordance with the FDA PRO Guidance (2009) to ensure evidence of content validity and measurement appropriate to support a labeling claim using PRO data. The early development work to create the PALS has included: • Completion of systematic reviews of the LBP literature and existing PRO instruments • The formation of an expert panel of clinical and methodological experts to provide advice during the development process • The completion of forty-three qualitative concept elicitation interviews conducted to identify the LBP-related concepts that are most important and relevant to the patients’ experience • A formal item-generation process in which evidence from the concept elicitation interviews, systematic literature reviews, and expert input was used to develop the content of the PAL-S • Four waves of qualitative cognitive interviews with patients with LBP to evaluate and refine the draft instruments This process has resulted in a 14-item developmental version ready for further quantitative testing. The primary aim is to collect initial quantitative data to further evaluate the performance of the PAL-S as appropriate PRO measures for use as endpoints in clinical studies of low back pain. The information gathered in this study will be used in the process of instrument refinement (item response theory, item reduction and/or modification), assessment of measurement characteristics, and finalization of the scoring approach to support the use of the PAL-S in subsequent studies.

To describe daytime non-severe hypoglycemic events (NSHEs), assess their impact on patient functioning and diabetes self-management, and examine if these impacts differ by diabetes type or country.Internet survey to adults with diabetes in the US, UK, Germany, and France.Of 6756 screened respondents, 2439 reported a daytime NSHE in the past month. NSHEs occurred while active (e.g., running errands) (45.1%), 29.6% while not active (e.g., watching TV), and 23.8% at work. On average, it took half a day to respond and recover from NSHE. Respondents monitored their glucose 5.7 extra times on average over the following week. On the day of event, type 1 respondents tested significantly more often than type 2 (p0.05). Type 2 were less likely to confirm NSHE with glucose test (p0.001). Following NSHE, 12.6% of respondents reduced total insulin by an average of 7.6 units (SD=8.3). Total units and days with reduced dosing was significantly less, whilst number of additional glucose tests and time to recover was significantly longer if NSHE occurred at work (p0.001). Type 1 decreased insulin doses more often (p0.001); however, type 2 decreased a greater number of units (p0.01). Compared with other countries, US respondents were more likely to eat a light or full meal and respondents in France took significantly longer than all other countries to recognize (p0.05), respond to (p0.001), and recover from (p0.001) NSHE, used significantly more monitoring tests the day of (p0.05) and over the subsequent week (p0.001), and decreased their normal insulin dose more (p0.001). Limitations of the study include potential recall bias and selection bias.NSHEs are associated with a significant impact on patient functioning and diabetes management.

ObjectivesTo test the psychometric properties of the EUROHIS-QOL 8-item index, a shortened version of the World Health Organization Quality of Life Instrument-Abbreviated Version (WHOQOL-BREF).MethodsThe sample consisted of 2359 subjects identified from primary care settings, with 1193 having a confirmed diagnosis of depression. Data came from six countries (Australia, Brazil, Israel, Russia, Spain, and the United States) involved in a large international study, the Longitudinal Investigation of Depression Outcomes. The structure of the EUROHIS-QOL 8-item index follows that of the WHOQOL-BREF assessment. Internal consistency was measured by using Cronbach's alpha. Convergent validity was assessed by using correlations with different measures for mental health (Symptom Checklist 90), physical health (self-evaluation), and quality of life (WHOQOL-BREF and short form 36 health survey). Discriminant group validity was assessed between diagnosed depressed and nondepressed patients. Differential item functioning and unidimensionality were analyzed by using Rasch analysis. Factor structure was assessed with structural equation modeling analyses.ResultsInternal consistency was acceptable (ranged between 0.72 and 0.81 across countries), and the index discriminated well between depression (t = 6.31–20.33; P < 0.001) across all countries. Correlations between the EUROHIS-QOL 8-item index and different measures—Symptom Checklist 90 (r = −0.42), physical health (r = −0.42), WHOQOL-BREF domains (r = 0.61–0.77), and short form 36 health survey (r = 0.58)—were all significant (P < 0.001). The index is unidimensional with desired item fit statistics. Two items (“daily living activities” and “enough money to meet your needs”) had residuals exceeding 4. Differential item functioning was observed with general quality of life, general health, relationships, and home items for age. A common one-factor structure with acceptable fit was identified in three out of six countries (comparative fit index = 0.85, root mean square error of approximation = 0.11).ConclusionsThe EUROHIS-QOL 8-item index showed acceptable cross-cultural performance and a satisfactory discriminant validity and would be a useful measure to include in studies to assess treatment effectiveness.

Menorrhagia, or heavy menstrual bleeding (HMB), has a negative impact on women's quality of life (QOL). The objective was to develop, validate, and assess the performance of a disease-specific patient-reported outcome (PRO) measurement instrument for HMB (the Menorrhagia Impact Questionnaire [MIQ]).The MIQ was designed to measure the effect of HMB on a woman's self-assessment of menstrual blood loss (MBL), limitations in social/leisure activities, physical activities, and ability to work. Meaningfulness of these observed MBL changes were also measured. The development and psychometric validation of the MIQ was performed utilizing data from a long-term safety study of tranexamic acid (Lysteda * *Lysteda is a registered trade name of Ferring Pharmaceuticals Inc., Parsippany, NJ, USA. ), with comparison to an age-matched normal control group recruited from the general population. Performance of the MIQ was also evaluated using data from a six-cycle, randomized, double-blind, clinical study of tranexamic acid for the treatment of HMB. Correlations and sensitivity of each pertinent MIQ item to the treatment-induced changes in MBL were assessed, and the minimally important differences (MID) for the individual MIQ items were determined.The psychometric properties of the MIQ were fully validated. Correlations between individual MIQ items and changes in MBL were statistically significant (p 0.001). A clear differentiation between tranexamic acid and placebo groups confirmed sensitivity of the MIQ and its ability to detect treatment-induced changes in MBL. MIDs were estimated for the individual MIQ items, with sensitivities and specificities in the 64-79% and 63-82% ranges using receiver operating characteristic (ROC) curve analyses, respectively. MIDs were found to be equal to or greater than 0.5. Statistically significant treatment differences were also observed for the proportions of subjects achieving at least 1-point improvement in MIQ scores.The MIQ contains validated constructs important to women with HMB.NCT00113568 and NCT00386308 (ClinicalTrials.gov ID).

Background Previous studies found that depression is associated with a broad impairment in quality of life (QOL). This finding might be associated to a measurement overlap. Methods The objective of this study was to verify whether the items of the World Health Organization Quality of Life Instrument-Abbreviated version (WHOQOL-BREF), a measure of generic QOL, are invariant among patients having a current major depressive episode who come from primary care services. We investigated data from primary care services from the 6 countries (Australia, Brazil, Israel, Russia, Spain, and the United States) involved in the baseline sample of the Longitudinal Investigation of Depression Outcomes. The Rasch model was used to analyze items exhibiting differential item functioning (DIF) as a way of assessing invariance in relation to a depression factor defined by the diagnosis of depression using the Composite International Diagnostic Interview. In addition, the Center for Epidemiological Studies-Depression Scale (CES-D) score was correlated with the item and domain scores of the WHOQOL-BREF using the Pearson coefficient. Results The sample consisted of 2359 subjects, of which 1193 had a confirmed diagnosis of a current major depressive episode. Of the 26 items of the WHOQOL-BREF, 11 showed DIF due to the depression factor, and the physical domain presented more items displaying DIF. All Pearson coefficients between the WHOQOL-BREF item and domain scores and the CES-D score were weak and moderate ( r = −0.13 to r = 0.43). Conclusions Our findings indicate that most WHOQOL-BREF items do not exhibit DIF for a current major depressive episode and the variance associated with depression in this generic QOL measure is restricted to some facets of this construct. Thus, we recommend this restricted adjustment for depression in future analyses of this measure. Furthermore, our study indicates that researchers must measure QOL regardless of depression severity.

Variables associated with treatment satisfaction (TS) can be broadly categorized as precursors, ongoing treatment issues, and treatment outcomes. This study continues the validation process for the Insulin Treatment Satisfaction Questionnaire (ITSQ) while examining relationships between TS and these broader influences. Data (N = 299) were collected in a RCT comparing safety and efficacy of inhaled vs. injectable insulin. Validation examined item and factor characteristics, reliability and validity. Regression analyses examined relationships between overall and domain specific TS and the broader influences. Patients with less previous or current treatment burden had greater TS (p

Diabetic peripheral neuropathy (DPN) occurs in 26–47 % of diabetes patients and may have negative impacts on physical functioning, sleep, well-being, and quality of life. The Diabetic Peripheral Neuropathic Pain Impact measure (DPNPI) was developed to measure disease impacts and treatment effects. Presented are the DPNPI conceptual development and validation findings. The DPNPI was developed following the FDA Guidance for Industry on patient-reported outcome (PRO) measures. Concept elicitation (CE) included literature review, clinical expert interviews, and patient interviews/focus groups. Qualitative data were analyzed following grounded theory principles, and draft items were cognitively debriefed. The measure underwent psychometric validation, and an a priori statistical analysis plan assessed the measurement model, reliability, and validity. Simultaneous analyses of item functioning were conducted using Rasch measurement theory (RMT). All tests were performed for the total score and each domain. Twenty-five patients and three clinical experts participated in CE which resulted in a 27-item validation ready measure. In the validation study (N = 124), nine draft items were dropped due to high missing data and/or high correlations between items. Factor analysis revealed three domains: physical functioning/mobility, sleep, and daily activities. RMT confirmed adequate item fit and placement within domains. Internal consistency ranged from 0.91 to 0.96 and test–retest from 0.84 to 0.91. All prespecified hypotheses for convergent and discriminant validity were met. CE and psychometric results provide evidence that the final, 18-item DPNPI is a reliable and valid PRO measure of disease impacts and treatment for DPNP. Further validation work should include responsiveness assessment.

The literature supports the use of electronic diaries (e-diaries) in a variety of populations, including different disease and age groups. However, there has been little discussion of cross-cultural preferences. The aim of this study was to compare acceptability of paper and electronic diaries to collect symptom data from patients with benign prostatic hyperplasia (BPH) in the United States and Slovakia. Participants were 28 US and 20 Slovak men aged 49–83 years with moderate-to-severe BPH. All were randomly assigned to complete a paper or electronic urinary symptom diary for 7 consecutive days, then complete the alternate version for another 7 days. Participants’ experiences with the diaries were assessed via ease of use and preference questions and patient interview. Site issues were assessed via tracking of technical problems and investigator feedback. The US men preferred the electronic version, whereas men in Slovakia had equal preference between the formats. Most men in both countries felt they could continue using the e-diary for another 2 weeks and still provide accurate information. Technical issues at the Slovakian site included incompatibility with power supplies, loss of battery power, and problems downloading data. The use of e-diaries is feasible with BPH populations, although this may vary by country. When including in clinical trials patients who are less familiar with technology, careful consideration should be given to diary design, training, and technical issues in that country that may have an impact on the data collection process.

Objectives To assess the comparability, reliability, and subject acceptability of electronic data capture (EDC) versions of Irritable Bowel Syndrome—Quality of Life (IBS-QOL), EuroQoL (EQ-5D) and Work Productivity and Activity Impairment (WPAI:IBS) instruments. Methods Comparability of EDC and paper questionnaires was evaluated in 72 subjects with IBS who completed a baseline EDC or paper questionnaire, a crossover questionnaire 24 hours later, and a retest of the crossover version at 1 week. The EDC version was presented on a hand-held device. Comparability was assessed using paired t -test statistics, intraclass correlation coefficients (ICC) and tests for internal consistency (Cronbach's alpha). Results No significant differences were found between scores obtained by paper questionnaire and EDC at the baseline and crossover assessments. ICCs between baseline and crossover assessments ranged from 0.83 to 0.96 for the IBS-QOL scores, 0.82 to 0.96 for the WPAI:IBS scores, and 0.77 to 0.82 for the EQ-5D. Internal consistency was comparable for the two data collection methods for the IBS-QOL overall score (0.96) and subscales and the EQ-5D Index (0.70 vs. 0.74). Retest statistics (ICC) were generally comparable between the EDC and paper versions for all scores. Ease of use was comparable for the two modes of administration, but more patients preferred EDC (47.2%) than the paper questionnaire (23.6%). Conclusions EDC versions of the IBS-QOL, EQ-5D, and WPAI:IBS are comparable to paper questionnaires in internal consistency and test–retest reliability, and have greater patient acceptability.

Urinary incontinence (UI) has substantial and important impacts on health-related quality of life. The purpose of this research is to report the psychometric performance of 15 different language versions of the Incontinence-specific Quality of Life (I-QOL)measure, a patient-reported outcome measure specific to stress, urge and mixed urinary incontinence. The multi-national dataset consisted of data from four clinical trials for stress incontinent females and from two additional population studies, enrolling women with stress, urge and mixed UI. All enrolled patients completed the I-QOL and comparative measures at baseline. The clinical trial populations had multiple administrations up to 12 weeks, and the two population studies included a shorter retest. Country-specific psychometric testing for validity, reliability, and responsiveness followed standardized procedures. Confirmatory factor analyses were performed to assess the I-QOL subscales. The I-QOL measurement model was confirmed as three subscales. Summary and subscale scores for the 15 versions were internally consistent (alpha values = 0.91-0.96) and reproducible (ICC = 0.72-0.97). Using changes in the independent measures of incontinence episode frequency standardized response means were predominantly strong (ranged 0.71-1.05) across 13 versions (out of 15) in association with these measures and effect sizes. These additional language versions of the I-QOL instrument demonstrate psychometric properties similar to the original version. The I-QOL has shown good results in both community studies and clinical trials with varying types and severity of urinary incontinence. It is a reliable and valid measure of HRQOL, suitable for use in a variety of international settings.

BackgroundFew published studies address depression outcomes in primary care from a cross-cultural perspective.AimsTo define baseline factors associated with 9-month clinical outcome: across six countries.MethodAdults meeting criteria for current major depression were recruited from primary care clinics in Australia, Brazil, Israel, Spain, Russia and the USA; 968 patients were assessed at the 9-month follow-up. Predictors of complete remission were examined using logistic regression with a hierarchical model.ResultsRates of complete remission in the six sites ranged from 25% to 48%. Logistic regression using pooled data showed that education, key life events and the Quality of Life Depression Scale score at baseline were the final predictors of complete remission, adjusting for centres, socio-demographic data, severity of depression, comorbidity and general quality of life. Variation in predictors across sites was not statistically significant.ConclusionsThe two major findings of this study were the low proportion of people achieving complete remission at follow-up across the six sites, and that some baseline characteristics (education, Quality of Life Depression Scale score and key life events) are modest predictors of outcome in depression.

The purpose of the study was to evaluate the prevalence and predictors of depression treatment in a diverse cross-national sample of primary care patients.At primary care facilities in six countries (Spain, Israel, Australia, Brazil, Russia, and the United States), a two-stage screening process was used to identify 1,117 patients with current depressive disorder. At baseline, all patients completed a structured diagnostic interview as well as measures of anxiety symptoms, alcohol use, chronic comorbid physical conditions, and perceived barriers to treatment. Primary care physicians were advised if the research interview indicated a probable depressive disorder in their patients. Three and 9 months later, participants reported all health services (including specialty mental health care and antidepressant medication) used in the preceding 3 months.Across the six sites, the proportion of patients receiving any antidepressant pharmacotherapy ranged from a high of 38% in Seattle to a low of 0% in St. Petersburg; the proportion receiving any specialty mental health care varied from a high of 29% in Melbourne to a low of 3% in St. Petersburg. Patient characteristics were not consistently associated with receipt of either pharmacotherapy or specialty mental health care. Out-of-pocket cost was the most commonly reported barrier to treatment for depression; the percentage of patients who reported this barrier ranged from 24% in Barcelona to 75% in St. Petersburg.Depression screening and physician notification are not sufficient to prompt adequate treatment for depression. The probability of treatment may be more influenced by characteristics of health care systems than by the clinical characteristics of individual patients. Financial barriers may be more important than stigma as impediments to appropriate care.

The Longitudinal Investigation of Depression Outcomes (LIDO) Study examined the outcomes and economic correlates of previously untreated depression among primary care patients in Barcelona, Spain; Be'er Sheva, Israel; Melbourne, Australia; Porto Alegre, Brazil; St. Petersburg, Russia; and Seattle, USA. Across all sites, 968 patients with current depressive disorder completed assessments of depression severity (Composite International Diagnostic Interview and Center for Epidemiologic Studies Depression Scale) at baseline and 9 months, and assessments of health services utilization and work days missed at baseline, 9 months, and 12 months. Follow-up depression status was characterized as persistent depression (n=345), partial remission (n=283), or full remission (n=340). At each site, patients with more favorable depression outcomes had fewer days missed from work; however, this relationship did not reach the 5% level of statistical significance at any site, and reached the 10% significance level only at Porto Alegre. Patients with more favorable depression outcomes also had lower health services costs, but this relationship reached the 5% significance level only in St. Petersburg. While the lack of statistical precision does not permit definitive conclusions, our findings are consistent with recent studies showing that recovery from depression is associated with lower health services costs and less time missed from work due to illness.

In 1991, the World Health Organization initiated a project to simultaneously develop a quality of life (QOL) instrument in 15 countries: The World Health Organization Quality of Life (WHOQOL) instrument. This was intended as a generic QOL tool for use with patients across varying disease types, severities of illness, and cultural subgroups. The objective of the current study was to evaluate the WHOQOL-100 in the U.S., one of the original 15 participating countries. The WHOQOL is a 100-item self-report instrument consisting of 24 subscales within six domains: Physical, Psychological, Independence, Social, Environment, and Spiritual. Four additional items pertain to overall QOL/health. We tested the WHOQOL-100 (U.S. version) in a sample of 443 adults (n = 251 chronically ill, n = 128 healthy, and n = 64 childbearing) in the U.S. to test its reliability (internal consistency, test–retest), construct validity (convergent, discriminant), responsiveness, and factor structure. The WHOQOL-100 (U.S. version) has acceptable internal consistency (alpha range: 0.82–0.95 across domains) and reproducibility (ICC range: 0.83–0.96 at 2-week retest interval). It is responsive to change in clinical conditions, as evidenced by predicted score change (effect size) in women after childbirth. Construct validity was demonstrated by (1) its correlation with the Short Form-36 and Subjective Quality of Life Profile, and (2) its ability to discriminate between the diverse samples in this study. The conceptual structure was confirmed exactly with the exception of four facets that did not correlate most highly with the domains to which they were originally assigned, but these differences were minor. The WHOQOL measurement system is suitable for evaluating the QOL of adults in the U.S. The psychometric properties will be continually evaluated as more data become available in the U.S.

Parkinson's disease is a common progressive neurodegenerative disorder affecting an estimated 4 million people worldwide. A number of general health status measures exist but few fully capture the subjective evaluation of life quality associated with Parkinson's disease. We report here the results of: (1) translating the British PDQ-39 into a US version, (2) validity and reliability of the new US PDQ-39 questionnaire, and (3) parallel validation analyses following the method published in the development of the British version of the PDQ-39. Data were collected by postal survey on 150 patients recruited from neurology clinics in the Seattle area. A short, generic health status measure (SF-36) was used to test convergent validity, and a three-day test-retest assessed the reliability of the PDQ-39. The US version of the PDQ-39 demonstrated acceptable internal consistency (alpha = 0.51 to 0.96) and proved to be reproducible (0.86 to 0.96). Subscales of the PDQ-39 showed convergence with like scales of the SF-36 and was able to discriminate between levels of symptom severity.