RT Journal Article
SR Electronic
T1 Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire
JF Annals of the Rheumatic Diseases
JO Ann Rheum Dis
FD BMJ Publishing Group Ltd and European League Against Rheumatism
SP 507
OP 515
DO 10.1136/annrheumdis-2021-220702
VO 81
IS 4
A1 Mike O Becker
A1 Rucsandra Dobrota
A1 Alexandru Garaiman
A1 Rudolf Debelak
A1 Kim Fligelstone
A1 Ann Tyrrell Kennedy
A1 Annelise Roennow
A1 Yannick Allanore
A1 Patricia E Carreira
A1 László Czirják
A1 Christopher P Denton
A1 Roger Hesselstrand
A1 Gunnel Sandqvist
A1 Otylia Kowal-Bielecka
A1 Cosimo Bruni
A1 Marco Matucci-Cerinic
A1 Carina Mihai
A1 Ana Maria Gheorghiu
A1 Ulf Mueller-Ladner
A1 Joseph Sexton
A1 Tore K Kvien
A1 Turid Heiberg
A1 Oliver Distler
YR 2022
UL http://ard.bmj.com/content/81/4/507.abstract
AB Objectives Patient-reported outcome measures (PROMs) are important for clinical practice and research. Given the high unmet need, our aim was to develop a comprehensive PROM for systemic sclerosis (SSc), jointly with patient experts.Methods This European Alliance of Associations for Rheumatology (EULAR)-endorsed project involved 11 European SSc centres. Relevant health dimensions were chosen and prioritised by patients. The resulting Systemic Sclerosis Impact of Disease (ScleroID) questionnaire was subsequently weighted and validated by Outcome Measures in Rheumatology criteria in an observational cohort study, cross-sectionally and longitudinally. As comparators, SSc-Health Assessment Questionnaire (HAQ), EuroQol Five Dimensional (EQ-5D), Short Form-36 (SF-36) were included.Results Initially, 17 health dimensions were selected and prioritised. The top 10 health dimensions were selected for the ScleroID questionnaire. Importantly, Raynaud’s phenomenon, impaired hand function, pain and fatigue had the highest patient-reported disease impact. The validation cohort study included 472 patients with a baseline visit, from which 109 had a test–retest reliability visit and 113 had a follow-up visit (85% female, 38% diffuse SSc, mean age 58 years, mean disease duration 9 years). The total ScleroID score showed strong Pearson correlation coefficients with comparators (SSc-HAQ, 0.73; Patient’s global assessment, Visual Analogue Scale 0.77; HAQ-Disability Index, 0.62; SF-36 physical score, −0.62; each p<0.001). The internal consistency was strong: Cronbach’s alpha was 0.87, similar to SSc-HAQ (0.88) and higher than EQ-5D (0.77). The ScleroID had excellent reliability and good sensitivity to change, superior to all comparators (intraclass correlation coefficient 0.84; standardised response mean 0.57).Conclusions We have developed and validated the EULAR ScleroID, which is a novel, brief, disease-specific, patient-derived, disease impact PROM, suitable for research and clinical use in SSc.Data are available on reasonable request. On request, and subject to review by the steering committee, access can be granted to the anonymised raw data and the R code. Deidentified data will be made available via secure data transfer. Data requests may be sent to the ScleroID steering committee, represented by OD, Department of Rheumatology, University Hospital Zurich, University of Zurich, Switzerland. Email oliver.distler@usz.ch.