Development and validation of the Ulcerative Colitis patient-reported outcomes signs and symptoms (UC-pro/SS) diary

Abstract Background The clinical course of ulcerative colitis (UC) and the effects of treatment are assessed through patient-reported signs and symptoms (S&S), and endoscopic evidence of inflammation. The Ulcerative Colitis Patient-Reported Outcomes Signs and Symptoms (UC-PRO/SS) measure was developed to standardize the quantification of gastrointestinal S&S of UC in clinical trials through direct report from patient ratings. Design The UC-PRO/SS was developed by collecting data from concept elicitation (focus groups, and individual interviews), then refined through a process of cognitive interviews of 57 UC patients. Measurement properties, including item-level statistics, scaling structure, reliability, and validity, were evaluated in an observational, four-week study of adults with mild to severe UC (N = 200). Results Findings from qualitative focus groups and interviews identified nine symptom items covering bowel and abdominal symptoms. The final UC-PRO/SS daily diary includes two scales: Bowel S&S (six items) and Abdominal Symptoms (three items), each scored separately. Each scale showed evidence of adequate reliability (α = 80 and 0.66, respectively); reproducibility (intraclass correlation coefficient = 0.81, 0.71) and validity, including moderate-to-high correlations with the Partial Mayo Score (0.79; 0.45) and Inflammatory Bowel Disease Questionnaire (IBDQ) total score (− 0.70; − 0.61). Scores discriminated by level of disease severity, as defined by the Partial Mayo Score, Patient Global Rating, and Clinician Global Rating (p < 0.0001). Conclusions Results suggest that the UC-PRO/SS is a reliable and valid measure of gastrointestinal symptom severity in UC patients. Additional longitudinal data are needed to evaluate the ability of the UC-PRO/SS scores to detect responsiveness and inform the selection of responder definitions.https://deepblue.lib.umich.edu/bitstream/2027.42/143869/1/41687_2018_Article_49.pd

Effects of omecamtiv mecarbil on symptoms and health-related quality of life in patients with chronic heart failure: results from the COSMIC-HF study

Background: Chronic HF with reduced ejection fraction (HFrEF) impairs health related quality of life (HRQL). Omecamtiv mecarbil, a novel activator of cardiac myosin, improves left ventricular systolic function and remodeling and reduces natriuretic peptides. We sought to evaluate the effect of omecamtiv mecarbil on symptoms and HRQL in patients with chronic HFrEF and elevated natriuretic peptides enrolled in the COSMIC-HF trial. Methods: Patients (n = 448) were randomized 1:1:1 to placebo, 25 mg of omecamtiv mecarbil twice daily (OM 25 mg), or to pharmacokinetically-guided dose titration (OM-PK) for 20 weeks. The Kansas City Cardiomyopathy Questionnaire (KCCQ) was administered to assess HRQL at baseline, 16 weeks, and 20 weeks. The primary scores of interest were the Total Symptom Score (TSS), Physical Limitation Scale (PLS), and Clinical Summary Score (CSS). Results: Mean change in score from baseline to 20 weeks for the TSS was 5.0 (95%CI: 1.8-8.1) for placebo, 6.6(95%CI: 3.4-9.8) for OM 25 mg (p = 0.32 vs placebo), and 9.9 (95%CI: 6.7-13.0) for OM-PK (p = 0.03 vs placebo); for the PLS, it was 3.1 for placebo (95%CI: -0.3-6.6), 6.0 (95%CI: 3.1-8.9) for OM 25 mg (p=0.12), and 4.3 (95%CI: 0.7-7.9) for OM-PK (p=0.42); for the CSS, it was 4.1 (95%CI: 1.4-6.9) for placebo, 6.3 (95%CI: 3.6-9.0) for OM 25 mg (p=0.19), and 7.0 (95%CI: 4.1-10.0) for OM-PK (p=0.14). Differences between omecamtiv mecarbil and placebo were greater in patients who were more symptomatic at baseline. Conclusions: HRQL as measured by the TSS improved in patients with HFrEF assigned to omecamtiv mecarbil (OM-PK group) relative to placebo. Ongoing trials are prospectively testing whether omecamtiv mecarbil improves symptoms and HRQL in HFrEF

Development and validation of the Crohn’s disease patient-reported outcomes signs and symptoms (CD-PRO/SS) diary

Abstract Background The clinical course of Crohn’s disease (CD) and the effect of its treatment are monitored through patient-reported signs and symptoms (S&S), and endoscopic evidence of inflammation. The Crohn’s Disease Patient-reported Outcomes Signs and Symptoms (CD-PRO/SS) measure was developed to standardize the quantification of gastrointestinal S&S of CD through direct report from patient ratings. Methods The CD-PRO/SS was developed based on data from concept elicitation (focus groups, interviews; n = 29), then refined through cognitive interviews of CD patients (n = 20). Measurement properties, including item-level statistics, scaling structure, reliability, and validity, were examined using secondary analyses of baseline and two-week clinical trial data of adults with moderate-to-severe CD (n = 238). Results Findings from qualitative interviews identified nine S&S items covering bowel and abdominal symptoms. The final CD-PRO/SS daily diary includes two scales: Bowel S&S (three items) and Abdominal Symptoms (three items), each scored separately. Each scale showed evidence of adequate reliability (α = 0.74 and 0.67, respectively); reproducibility (intraclass correlation coefficient > 0.80), and validity, with the last including moderate correlations with the Inflammatory Bowel Disease Questionnaire bowel symptom score and select items (ranging from r = 0.43–0.54). Scores distinguished patients categorized by patient global ratings of disease severity (p < 0.0001). Conclusions Results suggest the CD-PRO/SS is a reliable and valid measure of gastrointestinal symptom severity in CD patients. Additional longitudinal data are needed to evaluate the ability of the CD-PRO/SS scores to detect responsiveness and inform the selection of responder definitions.https://deepblue.lib.umich.edu/bitstream/2027.42/143542/1/41687_2018_Article_44.pd

Is the EQ-5D fit for purpose in asthma? Acceptability and content validity from the patient perspective

Background The increasing emphasis on patient-reported outcomes in health care decision making has prompted greater rigor in the evidence to support the instruments used. Acceptability and content validity are important properties of any measure to ensure it assesses the relevant aspects of the target concept. The purpose of this study was to evaluate the acceptability and content validity of the EQ-5D 5-Level (EQ-5D-5L) to assess the impact of asthma on patients’ lives. Methods Qualitative interviews were conducted with 40 adults with asthma in the United Kingdom. The first 25 interviews used cognitive-debriefing methods to assess the relevance and acceptability of the EQ-5D-5L and two asthma-specific measures for comparison: an asthma-specific, preference-based measure (the Asthma Quality of Life Utility Index–5 Dimensions) and an Asthma Symptom Diary. The final 15 interviews combined concept elicitation to identify patient-perceived asthma impact, and cognitive debriefing to assess relevance and acceptability of the EQ-5D-5L and the Asthma Symptom Diary. Cognitive-debriefing feedback on the content of the measures was collated and summarized descriptively. The concept-elicitation data were analyzed thematically. Results Participants were aged 20 to 57 years and 62.5% were female. Although some participants expressed positive opinions on aspects of the EQ-5D-5L, only the usual activities dimension was consistently considered relevant to participants’ asthma experiences. The mobility and self-care dimensions prompted strong negative reactions from some participants. Variations in interpretation of the mobility dimension and difficulties with multiple concepts in the pain/discomfort and anxiety/depression dimensions also were noted. Concepts reported by participants as missing included environmental triggers, asthma symptoms, emotions, and sleep. The EQ-5D-5L was the least preferred measure to describe the impact of asthma on participants’ lives. Participants reported shortness of breath and impact on activities as especially salient issues. Conclusions The content of the EQ-5D-5L was poorly aligned with the patient-perceived impact of asthma, and the measure failed to meet basic standards for acceptability and content validity as a measure to assess the impact of asthma from the patient perspective. The shortcomings identified raise concerns regarding the appropriateness of the EQ-5D in asthma and further evaluation is warranted

Rethinking non-traditional resistance at work : the case of the Indian Diaspora in Mauritius

Resistance at work can take many forms and this is reflected in the multiple ways it has been theorised in research. In this paper, I use postcolonial theory to analyse employee resistance in Mauritius. To do this, I deploy Homi Bhabha’s concepts of mimicry, ambivalence and hybridity to explore non-traditional forms of resistance among the Indian Diaspora working in the hotel industry. Using ethnographic research, I firstly look at its ‘home’-making practices as it is within the home that visions of community emerge (Bhabha, 1994) which could later influence behaviours at work. I argue that the Diaspora maintains connections with its ancestral roots via routine religious practices and language use while concomitantly resisting assimilation in the local context. The analysis is then extended to their workplace where further forms of non-traditional resistance are found to be enacted which are strongly influenced by the same values emerging at home