Dataset Information

Patient perceptions of health-related quality of life in giant cell arteritis: international development of a disease-specific patient-reported outcome measure.

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 | 2021 Oct

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Patient perceptions of health-related quality of life in giant cell arteritis: international development of a disease-specific patient-reported outcome measure.

Robson Joanna C JC Almeida Celia C Dawson Jill J Bromhead Alison A Dures Emma E Guly Catherine C Hoon Elizabeth E Mackie Sarah S Ndosi Mwidimi M Pauling John J Hill Catherine C

Rheumatology (Oxford, England) 20211001 10

<h4>Objectives</h4>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.<h4>Methods</ ...[more]

PMID: 33528002

Similar Datasets

Project description:Objective or purposeTo develop and test a patient-reported outcome measure for assessing health-related quality of life (HRQOL) in surgically amenable epiphora.DesignQuestionnaire development and validation study.Participants201 patients with a cause of epiphora amenable to surgical intervention, recruited across three independent centres.Methods, intervention or testingThe watery eye quality of life (WEQOL) questionnaire was developed and refined according to defined psychometric standards. Both surgical and non-surgical participants completed WEQOL at baseline and follow-up (>3 months), along with the Lacrimal Symptom Questionnaire (Lac-Q), RAND Short Form Health Survey (SF-36) and Glasgow Benefit Inventory (GBI). Convergent validity of WEQOL was evaluated according to correlation (R > 0.40) with each of these additional tests. Responsiveness of WEQOL to intervention was evaluated according to patient-reported success. Test-retest reliability was assessed by the Bland-Altman method and intraclass correlation (ICC) in a subset of 64 participants at baseline.Main outcome measuresWEQOL construct validity, responsiveness and test-retest reliability.ResultsWEQOL was moderately correlated (R > 0.4) with the Lac-Q and several subscales of the SF-36 (physical role limitation, social, emotional role limitation and emotional well-being). A stronger correlation was found between the change in WEQOL at follow-up and GBI (R = 0.61). An appropriate graded response was found with a significant change in WEQOL score being observed in patients reporting successful (-28%, p < 0.0001) and partially successful surgery (-6%, p = 0.04), but not in those reporting unsuccessful surgery (+2%, p = 0.9). High test-retest reliability was observed (ICC = 0.93).ConclusionsThe WEQOL questionnaire has been developed systematically according to modern psychometric standards and has been designed to evaluate the quality of life in patients with epiphora that is of a surgically amenable cause. In this study, it has demonstrated appropriate test-retest reliability, responsiveness and construct validity.

Project description:Rationale & objectivesTo test a new patient-reported outcome measure that assesses end-stage kidney disease (ESKD) maintenance dialysis patients' experience with life goals discussions with their dialysis facility care team.Study designObservational cross-sectional study. Survey data collected via REDCap, paper form or telephone in a convenience sample of patients with ESKD receiving maintenance dialysis in the United States.Settings & participantsPeople aged 18 years or older with ESKD receiving maintenance hemodialysis or peritoneal dialysis in US dialysis facilities between the June and December 2020 study period.ExposuresTesting of 6 items providing the core quality assessment and 1 item measuring whether 1 or more members of the treatment team discussed life goals with the patient.OutcomesPreliminary reliability and validity of the Discussion of Patient Life Goals survey (D-PaLS).Analytic approachExploratory factor analysis (EFA), confirmatory factor analysis (CFA), and item response theory methods, including the graded response model (GRM) and differential item functioning (DIF).ResultsOf 517 participants, 479 completed the survey via REDCap; 38 completed the survey via paper or telephone. EFA and CFA supported the unidimensionality of the 6 core items. GRM overall and item fit analyses and DIF analyses supported retention of all core items. Preliminary reliability data indicated very good internal consistency (Cronbach's alpha = 0.84). Known-groups validity was supported whereby individuals receiving home dialysis had more positive responses, than those receiving in-center hemodialysis.LimitationsStudy participants were not fully representative of the US ESKD dialysis population.ConclusionsPreliminary analyses indicate the D-PaLS has excellent psychometric properties. The measure provides 2 important quality metrics: facilities' level of engagement talking with patients about their life goals and the percentage of patients reporting who on the treatment team talks with them about their life goals. Additional work is needed to establish comprehensive reliability and validity to support the clinical utility of this measure in patient care.

Project description:PurposeTo report the variability in outcome measures after meniscal surgery and to compare responsiveness between patient-reported outcome measures (PROMs).MethodsA systematic search of the PubMed/MEDLINE and Web of Science databases was performed according to the Preferred Reporting Items for Systematic Reviews and Meta-Analysis guidelines. A total of 257 studies met inclusion criteria. Patient and study attributes were extracted, including pre- and postoperative means for PROMs. Of the studies that met inclusion criteria for responsiveness analysis (2+ PROMs reported, 1-year minimum follow-up; n = 172), we compared the responsiveness between PROM instruments using effect size and relative efficiency (RE) if a PROM could be compared with another in at least 10 articles.ResultsIn total, 18,612 patients (18,690 menisci, mean age = 38.6 years, mean body mass index = 26.3) were included in this study. Radiographic measures were reported in 167 (65.0%) studies, range of motion was reported in 53 (20.6%) studies, and 35 different PROM instruments were identified. The mean number of PROMs in each article was 3.6 and 83.8% reported 2 or more PROMs. The most used PROMs were Lysholm (74.5%) and IKDC (51.0%). IKDC was found to be more responsive than other PROMs, which include Lysholm (RE = 1.03), Tegner (RE = 3.90), and Knee Injury and Osteoarthritis Outcome Score (KOOS) Activities of Daily Living (ADL) (RE = 1.12). KOOS Quality of Life (QoL) was also more responsive than other PROMs, such as IKDC (RE = 1.45) and KOOS ADL (RE = 1.48). Lysholm was more responsive compared with KOOS QoL (RE = 1.14), KOOS ADL (RE = 1.96), and Tegner (RE = 3.53).ConclusionsOur study found that IKDC, KOOS QoL, and Lysholm were the most responsive PROMs. However, because of the previously reported risks of either floor effects (KOOS QoL) or ceiling effects (Lysholm), the IKDC may offer a more complete psychometric profile when quantifying outcomes after meniscus procedures.Clinical relevanceTo improve clinical outcomes, surgical decision-making, and research methodology, it is important to determine which PROMs are the most responsive after meniscal surgery.

Project description:BackgroundHereditary spastic paraplegia (HSP) is a rare neurodegenerative disease that lacks specific and validated patient-centered outcome measures (PCOMs). We aimed to develop and validate a health-related quality of life (HRQoL) questionnaire specific to HSP ("TreatHSP-QoL") that could be used as a PCOM.ResultsThe pilot-items of the TreatHSP-QoL (45 five-level Likert scale items, with values per item between 0 and 4) were developed based on a qualitative data analysis of 54 semi-structured interviews, conducted in person with 36 HSP patients and 18 caregivers. It was then reduced and modified through the validation process to 25 items. The main validation was performed using the online questionnaire in 242 HSP patients and 56 caregivers. The exploratory factor analysis defined five subdomains. Cronbach's alpha ranged from 0.57 to 0.85 for the subdomains and reached 0.85 for the total score. The test-retest Pearson correlation reached 0.86 (95% Confidence Interval (CI) [0.79, 0.91]). Pearson correlations with the EuroQol-5 Dimension (5 levels) (EQ-5D-5L) and Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) questionnaires varied strongly among the subdomains, with the total scores reaching 0.53 (95% CI [0.42, 0.61]) and -0.45 (95% CI [- 0.55, - 0.35]), respectively. The caregiver-patient response Pearson correlation ranged between 0.64 and 0.82 for subdomains and reached 0.65 (95% CI [0.38, 0.81]) for the total score.ConclusionsTreatHSP-QoL can be used in high-quality clinical trials and clinical practice as a disease-specific PCOM (i.e., HRQoL measure) and is also applicable as a proxy questionnaire. Score values between 0 and 100 can be reached, where higher value represents better HRQoL. The Pearson correlations to the EQ-5D-5L and FARS-ADL support the additional value and need of HSP-specific PCOM, while non-specific QoL-assessment and specific clinical self-assessment tools already exist. All in all, the results demonstrate good validity and reliability for this new patient-centered questionnaire for HSP.

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.

Project description:ObjectiveLung cancer is the leading cause of cancer-related death globally and provides a major disease burden likely to substantially impact quality of life (QoL). Patient-reported outcome measures (PROMs) have been identified as effective methods of evaluating patient QoL. Existing lung cancer-specific PROMs however have uncertain utility and minimal patient involvement in their design and development. This qualitative study aimed to evaluate the patient perspective of existing PROMs and to explore their appropriateness for population-based descriptions of lung cancer-related QoL.MethodsA descriptive qualitative study was conducted consisting of semi-structured interviews with 14 patients recruited from the Victorian Lung Cancer Registry and Alfred Hospital using purposive sampling. Interviews first explored the factors most important to lung cancer patients QoL, and second, patient's perspectives on the appropriateness of existing PROMs. Thematic analysis was used to develop themes, and content analysis was conducted to determine PROM acceptability.ResultsFive novel themes were identified by patients as being important impacts on QoL: Personal attitude toward the disease is important for coping; independence is valued; relationships with family and friends are important; relationships with treating team are meaningful; personal and public awareness of lung cancer is limited. These patient-identified impacts are poorly covered in existing lung cancer-specific PROMs. Patients welcomed and appreciated the opportunity to complete PROMs; however, they identified problems with existing PROMs relevance, tone, and formatting.ConclusionExisting lung cancer PROMs poorly reflect the five themes identified in this study as most important to lung cancer patients QoL. This study reaffirms the need to review existing PROMs to ensure utility and construct validity. Future PROM development must engage key patient-generated themes and evolve to reflect the changing management and therapeutic landscape.

Dataset Information

Patient perceptions of health-related quality of life in giant cell arteritis: international development of a disease-specific patient-reported outcome measure.

Objectives

Methods

Results

Conclusion

Publications

Patient perceptions of health-related quality of life in giant cell arteritis: international development of a disease-specific patient-reported outcome measure.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets