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ObjectiveThe objective was to evaluate the psychometric properties of the Rheumatoid Arthritis-Work Instability Scale (RA-WIS) in a clinical trial setting.MethodsSecondary analyses were conducted using data from a 56-week, randomized controlled trial of patients with early rheumatoid arthritis (RA). Patient-reported outcome measures included the RA-WIS, the Health Assessment Questionnaire (HAQ), the Rheumatoid Arthritis Quality of Life Questionnaire, and the Global Assessment of Disease Activity and Pain, data for which were collected at baseline and at weeks 12, 16, 24, and 56. Data were analyzed for reliability, validity, and responsiveness.ResultsAmong 148 patients whose data were analyzed, more than half were women (56.1%) with a mean age of 46.8 years. On average, patients experienced RA symptoms for 8.7 months; the mean 28-Joint Disease Activity Score (DAS28) was 5.9, and the mean HAQ – Disability Index was 1.3. The RA-WIS demonstrated excellent internal consistency and test-retest reliability (α = 0.89 and intraclass correlation coefficient = 0.91, respectively). At baseline and week 24, moderate to strong correlations were seen between RA-WIS total scores and the HAQ, the Global Assessment of Disease Activity, and the Pain Rheumatoid Arthritis Quality of Life Questionnaire, ranging from 0.47 to 0.81 (all P < 0.0001). Mean RA-WIS total scores and work disability risk levels discriminated between clinical severity scores on the DAS28, the HAQ – Disability Index, and the Physician Global Assessment of Disease Activity (all P < 0.05). Mean baseline to week 24 RA-WIS total change scores were significantly different among American College of Rheumatology responder groups (P ≤ 0.0001) and between DAS28 remission status groups (P < 0.001).ConclusionsThese findings provide evidence supporting the reliability, validity, and responsiveness of the RA-WIS for evaluating work disability in patients with RA in a clinical trial setting.

Background The purpose of this literature review was to examine the existing patient-reported outcome measurement literature to understand the empirical evidence supporting response scale selection in pain measurement for the adult population. Methods The search strategy involved a comprehensive, structured, literature review with multiple search objectives and search terms. Results The searched yielded 6918 abstracts which were reviewed against study criteria for eligibility across the adult pain objective. The review included 42 review articles, consensus guidelines, expert opinion pieces, and primary research articles providing insights into optimal response scale selection for pain assessment in the adult population. Based on the extensive and varied literature on pain assessments, the adult pain studies typically use simple response scales with single-item measures of pain—a numeric rating scale, visual analog scale, or verbal rating scale. Across 42 review articles, consensus guidelines, expert opinion pieces, and primary research articles, the NRS response scale was most often recommended in these guidance documents. When reviewing the empirical basis for these recommendations, we found that the NRS had slightly superior measurement properties (e.g., reliability, validity, responsiveness) across a wide variety of contexts of use as compared to other response scales. Conclusions Both empirical studies and review articles provide evidence that the 11-point NRS is likely the optimal response scale to evaluate pain among adult patients without cognitive impairment.

Background In the development of patient-reported outcome (PRO) instruments, little documentation is provided on the justification of response scale selection. The selection of response scales is often based on the developers’ preferences or therapeutic area conventions. The purpose of this literature review was to assemble evidence on the selection of response scale types, in PRO instruments. The literature search was conducted in EMBASE, MEDLINE, and PsycINFO databases. Secondary search was conducted on supplementary sources including reference lists of key articles, websites for major PRO-related working groups and consortia, and conference abstracts. Evidence on the selection of verbal rating scale (VRS), numeric rating scale (NRS), and visual analogue scale (VAS) was collated based on pre-determined categories pertinent to the development of PRO instruments: reliability, validity, and responsiveness of PRO instruments, select therapeutic areas, and optimal number of response scale options. Results A total of 6713 abstracts were reviewed; 186 full-text references included. There was a lack of consensus in the literature on the justification for response scale type based on the reliability, validity, and responsiveness of a PRO instrument. The type of response scale varied within the following therapeutic areas: asthma, cognition, depression, fatigue in rheumatoid arthritis, and oncology. The optimal number of response options depends on the construct, but quantitative evidence suggests that a 5-point or 6-point VRS was more informative and discriminative than fewer response options. Conclusions The VRS, NRS, and VAS are acceptable response scale types in the development of PRO instruments. The empirical evidence on selection of response scales was inconsistent and, therefore, more empirical evidence needs to be generated. In the development of PRO instruments, it is important to consider the measurement properties and therapeutic area and provide justification for the selection of response scale type.

Background Binge eating disorder (BED) is now a formal diagnosis in the Diagnostic and Statistical Manual of Mental Disorders, fifth edition (DSM-5). However, post-DSM-5 patient profiles and viewpoints on BED diagnosis and treatment remain unclear. This study used a focus group methodology to examine demographic and clinical characteristics, as well as perceptions of diagnosis and treatment from patients with BED symptoms who were either formally diagnosed with BED or undiagnosed. Methods Binge eating disorder-diagnosed individuals (n = 11) or those meeting the DSM-5 BED diagnostic criteria but were undiagnosed (n = 14) participated in 6 semistructured focus groups conducted by trained staff at 3 geographic locations in the United States. Patients completed a series of demographic and clinical measures and then engaged in a moderated discussion focused on identifying factors associated with their experiences with BED. Results Sixty percent of the patients were female, 48% were white and 40% were black, and 76% were employed. The diagnosed group had a slightly higher socioeconomic status; undiagnosed patients had a higher average body mass index. In the overall sample, comorbid anxiety (40%) and depression (40%) were the most common psychiatric comorbidities. Even in the diagnosed group, only half of the patients (54.5%) became aware of BED through their health care provider (HCP; n = 6). Patients perceived that HCPs were focused more on physical ailments, were judgmental about weight, and were unable to distinguish BED from obesity. They also expressed a desire for safe, nonjudgmental interactions with HCPs. Conclusions Education and income may be factors affecting access to care and BED diagnosis. Both patient groups reported considerable psychopathology and medical comorbidities. Moreover, the patient groups perceived HCPs as both having inadequate understanding of BED and providing insensitive and ineffective communication regarding eating behaviors. The study findings in diagnosed and undiagnosed patient groups underscore the need for greater BED disease state awareness and patient sensitivity among HCPs.

BACKGROUND: Combination therapy with ixabepilone and capecitabine (cape) is approved for use in patients with locally advanced/metastatic breast cancer that is resistant to treatment with anthracyclines or taxanes. The current study evaluated the trade-off between quality and quantity of life using quality-adjusted time without symptoms or toxicity (Q-TWiST) outcomes. METHODS: Within the trial, 752 women were randomly assigned to receive either the combination of ixabepilone and cape (once every 21 days) or cape alone (on days 1-14). The area under the survival curve was partitioned into 3 health states: toxicity (TOX), time without symptoms of disease progression or toxicity, and recurrence (relapse [REL]). The mean time in each health state was weighted by a range of utilities and summed to estimate quality-adjusted survival (QAS). Patient-reported outcomes were also evaluated using the Functional Assessment of Cancer Therapy (FACT)-Breast Symptom Index (FBSI). RESULTS: A statistically significant difference between groups with regard to change from baseline FBSI scores favoring the cape group was observed (P = .0002), but no differences were observed after adjusting for deaths in the analysis. All combinations of utilities for REL and TOX resulted in an observed difference in QAS favoring combination therapy. Differences were found to be statistically significant for comparisons, with higher tolerance for TOX. QAS was found to be greater for the combination therapy group (42.2 weeks vs 38.4 weeks), assuming the base case scenario of utility equal to 0.5 for both TOX and REL (P = .0227). CONCLUSIONS: The Q-TWiST analysis supports a positive benefit-risk ratio for the combination of ixabepilone plus cape in patients with advanced/metastatic breast cancer that is refractory to anthracyclines and taxanes versus cape alone, despite the potential for added toxicities with combination therapy. Cancer 2011;. © 2011 American Cancer Society.

Background In an international, randomized Phase III trial ipilimumab demonstrated a significant overall survival benefit in previously treated advanced melanoma patients. This report summarizes health-related quality of life (HRQL) outcomes for ipilimumab with/without gp100 vaccine compared to gp100 alone during the clinical trial’s 12 week treatment induction period. Methods The Phase III clinical trial (MDX010-20) was a double-blind, fixed dose study in 676 previously treated advanced unresectable stage III or IV melanoma patients. Patients were randomized 3:1:1 to receive either ipilimumab (3 mg/kg q3w x 4 doses) + gp100 (peptide vaccine; 1 mg q3w x 4 doses; ipilimumab plus gp100, n = 403); gp100 vaccine + placebo (gp100 alone, n = 136); or ipilimumab + placebo (ipilimumab alone, n = 137). The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) assessed HRQL. Baseline to Week 12 changes in EORTC QLQ-C30 function, global health status, and symptom scores were analyzed for ipilimumab with/without gp100 vaccine compared to gp100 alone. Mean change in scores were categorized “no change” (0–5), “a little” (5–10 points), “moderate” (10–20 points), and “very much” (>20). Results In the ipilimumab plus gp100 and ipilimumab alone groups, mean changes from baseline to Week 12 generally indicated “no change” or “a little” impairment across EORTC QLQ-C30 global health status, function, and symptom subscales. Significant differences in constipation, favoring ipilimumab, were observed (p Conclusions Ipilimumab with/without gp100 vaccine does not have a significant negative HRQL impact during the treatment induction phase relative to gp100 alone in stage III or IV melanoma patients. Trial registration Clinicaltrials.gov identification number NCT00094653

Combination therapy with ixabepilone and capecitabine (cape) is approved for use in patients with locally advanced/metastatic breast cancer that is resistant to treatment with anthracyclines or taxanes. The current study evaluated the trade-off between quality and quantity of life using quality-adjusted time without symptoms or toxicity (Q-TWiST) outcomes.Within the trial, 752 women were randomly assigned to receive either the combination of ixabepilone and cape (once every 21 days) or cape alone (on days 1-14). The area under the survival curve was partitioned into 3 health states: toxicity (TOX), time without symptoms of disease progression or toxicity, and recurrence (relapse [REL]). The mean time in each health state was weighted by a range of utilities and summed to estimate quality-adjusted survival (QAS). Patient-reported outcomes were also evaluated using the Functional Assessment of Cancer Therapy (FACT)-Breast Symptom Index (FBSI).A statistically significant difference between groups with regard to change from baseline FBSI scores favoring the cape group was observed (P = .0002), but no differences were observed after adjusting for deaths in the analysis. All combinations of utilities for REL and TOX resulted in an observed difference in QAS favoring combination therapy. Differences were found to be statistically significant for comparisons, with higher tolerance for TOX. QAS was found to be greater for the combination therapy group (42.2 weeks vs 38.4 weeks), assuming the base case scenario of utility equal to 0.5 for both TOX and REL (P = .0227).The Q-TWiST analysis supports a positive benefit-risk ratio for the combination of ixabepilone plus cape in patients with advanced/metastatic breast cancer that is refractory to anthracyclines and taxanes versus cape alone, despite the potential for added toxicities with combination therapy.

The patient-reported Dermatology Life Quality Index (DLQI) measures the impact of dermatologic diseases on patients' lives.To evaluate the content validity of the DLQI in patients with moderate to severe plaque psoriasis.In a two-part interview, participants were first asked open-ended questions about the impact of psoriasis-related complaints and symptoms on their lives and activities. The DLQI was then administered and cognitive debriefing interviews assessed participants' understanding of the instructions, items, and response scales, and relevance of the specific items to their experience with psoriasis.Twenty-one patients were interviewed at two US sites. Mean age was 48.8 years, 67% were white, and 43% reported Hispanic/Latino ethnicity. The majority reported living with a partner or spouse (81%) and working full time or part time (57%). Patients' spontaneous responses to open-ended questions were consistent with DLQI concepts and generally did not provide additional concepts. Most participants reported that the instructions, item content, and response scales were clear and easy to understand and relevant.The content of the DLQI included all important and relevant concepts from the perspective of patients with moderate to severe plaque psoriasis. This study provides further support for the content validity of the DLQI in this population.