Patient perceptions of health-related quality of life in giant cell arteritis: international development of a disease-specific patient-reported outcome measure.
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ABSTRACT: Objectives
GCA is a large vessel vasculitis (LVV) presenting with headache, jaw claudication, musculoskeletal and visual involvement. Current treatment is glucocorticoids and anti-IL-6 tocilizumab in refractory disease. The objective of this study was to explore the impact of GCA and its treatment on people's health-related quality of life (HRQoL), to inform the development of a disease-specific patient-reported outcome measure (PROM) for use in clinical trials and practice.Methods
Participants from the UK and Australia, with biopsy- or imaging-confirmed GCA, were interviewed to identify salient aspects of HRQoL in relation to GCA and its treatment. Purposive sampling included a range of demographic and disease features (cranial, LVV-GCA and visual involvement). Inductive analysis identified individual themes of importance, then domains. Candidate questionnaire items were developed from the individual themes, refined by piloting, cognitive interviews and a linguistic translatability assessment.Results
Thirty-six interviews were conducted to saturation with participants with GCA from the UK (25) and Australia (11). Mean age was 74 years, 23 (63.9%) were female, 13 (36.1%) had visual loss and 5 (13.9%) had LVV-GCA. Thirty-nine individual themes within five domains were identified: physical symptoms; activity of daily living and function; participation; psychological impact; and impact on sense of self and perception of health. Sixty-nine candidate items were developed from individual themes; piloting and refinement resulted in a 40-item draft questionnaire.Conclusion
This international qualitative study underpins the development of candidate items for a disease-specific PROM for GCA. The draft questionnaire is now ready for psychometric testing.
SUBMITTER: Robson JC
PROVIDER: S-EPMC8487303 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:BackgroundICU survivorship includes a diverse burden of disease. Current questionnaires used for collecting information about health-related problems and their relation to quality of life lack detailed questions in several areas relevant to ICU survivors. Our aim was to construct a provisional questionnaire on health-related issues based on interviews with ICU survivors and to test if this questionnaire was able to show differences between ICU survivors and a control group.MethodsThirty-two ICU survivors were identified at a post-ICU clinic and interviewed at least six months after ICU discharge. Using an established qualitative methodology from oncology, all dysfunctions and disabilities were extracted, rephrased as questions and compiled into a provisional questionnaire. In a second part, this questionnaire was tested on ICU survivors and controls. Inclusion criteria for the ICU survivors were ICU stay at least 72 h with ICU discharge six months to three years prior to the study. A non-ICU-treated control group was obtained from the Swedish Population Register, matched for age and sex. Eligible participants received an invitation letter and were contacted by phone. If willing to participate, they were sent the questionnaire. Descriptive statistics were applied.ResultsAnalysis of the interviews yielded 238 questions in 13 domains: cognition, fatigue, physical health, pain, psychological health, activities of daily living, sleep, appetite and alcohol, sexual health, sensory functions, gastrointestinal functions, urinary functions and work life. In the second part, 395 of 518 ICU survivors and 197 of 231 controls returned a completed questionnaire, the response rates being 76.2% and 85.3%, respectively. The two groups differed significantly in 13 of 22 comorbidities. ICU survivors differed in a majority of questions (p ≤ 0.05) distributed over all 13 domains compared with controls.ConclusionsThis study describes the development of a provisional questionnaire to identify health-related quality of life issues and long-term burden of disease after intensive care. The questionnaire was answered by 395 ICU survivors. The questionnaire could identify that they experience severe difficulties in a wide range of domains compared with a control group. Trial registry ClinicalTrials.gov Ref# NCT02767180.
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Project description:IntroductionWe conducted a systematic review of patient-reported outcome measures (PROMs) regarding quality of life, disability, mood abnormalities (anxiety, depression), fatigue, illness perceptions and fibromyalgia in Takayasu arteritis (TAK). Wherever available, comparisons with healthy controls, disease controls or longitudinal changes in PROMs were noted.MethodsMEDLINE, EMBASE, Scopus, Web of Science and Pubmed Central databases, major recent international rheumatology conference abstracts, clinical trial databases and the Cochrane library were searched for relevant articles. Wherever possible, outcome measures across studies were pooled using the restricted maximum likelihood model. Inter-group differences were pooled and compared using standardized mean differences (SMD) with 95% confidence intervals (95% CI). Heterogeneity was assessed using the I2 statistic. Quality of randomized controlled trials was assessed using the Cochrane risk of bias tool. For cross-sectional and cohort studies, the Joana Briggs Institute checklist and Newcastle-Ottawa scale were used, respectively. GRADE methodology was used to determine the certainty of evidence for outcomes.ResultsTwenty-one studies (all but one observational) involving 1311 patients with TAK and 308 healthy controls were identified. Ten studies (559 TAK patients, 182 healthy controls were synthesized in a meta-analysis. Patients with TAK had worse quality of life (pooled SMD - 6.66, 95% CI - 10.08 to - 3.23 for individual domains; - 0.64, 95% CI - 1.19 to - 0.09 for pooled physical and mental component scores of 36-item Short Form Survey), depression (SMD 0.26, 95% 0.05-0.47) and anxiety (SMD 0.34, 95% CI - 0.06 to 0.75) scores and higher disability (SMD 0.64, 95% CI 0.43-0.84) than healthy controls. Patients with active TAK had worse quality of life, depression and work impairment when compared with those with inactive disease. Included studies were of moderate to high quality. Certainty of evidence for individual outcomes was low to very low.ConclusionLiterature on PROMs in TAK, albeit sparse, appears to indicate worse scores in patients with TAK compared to healthy individuals. These results, however, require cautious interpretation. Development of a TAK-specific PROM is an important focus of the research agenda.
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Project description:IntroductionTreatments for type 2 diabetes vary widely in their complexity. The simplicity or complexity of a treatment regimen may have an impact on patient preference, treatment adherence, and health outcomes. The purpose of this qualitative study was to develop two draft patient-reported outcome instruments focusing on patients' experience with simplicity and complexity of treatment for type 2 diabetes.MethodsThe instruments were developed in a series of steps: gather information to support development of a concept elicitation interview guide (literature review and expert interviews), concept elicitation interviews with patients (N = 30), cognitive interviews with patients (N = 20), and a translatability assessment.ResultsIn concept elicitation interviews, patients with type 2 diabetes reported a range of treatment attributes that influence their perceptions of treatment simplicity and complexity, such as injection devices, medication preparation, dose timing, dose frequency, ease of taking the correct dose, flexibility of dose schedule, remembering to take medication, and food requirements. Two draft questionnaires were developed based on the literature review, expert interviews, and concept elicitation interviews with patients. Revisions were made to these draft instruments based on qualitative interviews with patients and translatability assessment.DiscussionThe qualitative research conducted in this study supports the content validity of two newly developed instruments, the Simplicity of Diabetes Treatment Questionnaire (Sim-Q) and the Simplicity of Diabetes Treatment Questionnaire-Comparison (Sim-Q-Comp), designed to assess the simplicity and complexity of diabetes treatment from the patient's perspective.
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Project description:PurposeTo assess and compare the subjective improvement in symptoms and quality of life in adult patients who underwent commonly performed oculoplastic surgical interventions to treat epiphora.Materials and methodsA prospective study was undertaken involving all adult patients undergoing dacryocystorhinostomy (DCR), lid tightening (lateral tarsal strip or lateral wedge resection), and punctoplasty surgery at our institution. We assessed severity of epiphora preoperatively using the Munk score. At 3 months postoperatively, all patients were sent postal questionnaires comprising of Munk score, 'social impact score' from validated Lac-Q questionnaire ranging from 0 (no impact) to 5 (maximal negative impact) and Glasgow Benefit Inventory (GBI) score, ranging from -100 (maximal detriment) to +100 (maximal benefit).ResultsA total of 134 questionnaires were sent with an overall response rate of 74.6%. For the purpose of data analysis, patients were divided into four groups: DCR, lid tightening, punctoplasty, and combined group (lid tightening plus punctoplasty). There was statistically significant improvement in subjective epiphora postoperatively, as assessed by Munk score (P<0.001) in all groups. The total GBI scores were +42.67 (95% CI: 33.42-51.91) for DCR, +19.65 (95% CI: 10.33-28.97) for lid tightening, +16.06 (95% CI: 2.65-29.48) for punctoplasty, and +26.53 (95% CI: 13.15-39.90) for the combined group, demonstrating a positive change in health status for all groups. There was negative correlation between total GBI and post-operative Munk scores (r=-0.58, P<0.001), and positive correlation between Lac-Q and Munk scores (r=0.65, P<0.001).ConclusionPatients derived significant improvement in symptoms and health-related quality of life benefit following all surgical interventions for epiphora.
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