Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire

Mike O Becker; Rucsandra Dobrota; Alexandru Garaiman; Rudolf Debelak; Kim Fligelstone; Ann Tyrrell Kennedy; Annelise Roennow; Yannick Allanore; Patricia E Carreira; László Czirják; Christopher P Denton; Roger Hesselstrand; Gunnel Sandqvist; Otylia Kowal-Bielecka; Cosimo Bruni; Marco Matucci-Cerinic; Carina Mihai; Ana Maria Gheorghiu; Ulf Mueller-Ladner; Joseph Sexton; Tore K Kvien; Turid Heiberg; Oliver Distler

doi:10.1136/annrheumdis-2021-220702

Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire

Ann Rheum Dis. 2022 Apr;81(4):507-515. doi: 10.1136/annrheumdis-2021-220702. Epub 2021 Nov 25.

Authors

Mike O Becker^#¹, Rucsandra Dobrota^#¹, Alexandru Garaiman¹, Rudolf Debelak^{2

3}, Kim Fligelstone⁴, Ann Tyrrell Kennedy⁵, Annelise Roennow⁶, Yannick Allanore⁷, Patricia E Carreira⁸, László Czirják⁹, Christopher P Denton¹⁰, Roger Hesselstrand¹¹, Gunnel Sandqvist¹¹, Otylia Kowal-Bielecka¹², Cosimo Bruni¹³, Marco Matucci-Cerinic^{13

14}, Carina Mihai^{1

15}, Ana Maria Gheorghiu¹⁵, Ulf Mueller-Ladner¹⁶, Joseph Sexton¹⁷, Tore K Kvien¹⁷, Turid Heiberg^#¹⁸, Oliver Distler^#¹⁹

Affiliations

¹ Department of Rheumatology, University Hospital of Zurich, Zurich, Switzerland.
² Department of Psychology, Psychological Methods, Evaluation and Statistics, University of Zurich, Zurich, Switzerland.
³ Department of Psychology, Psychological Methodology, University of Leipzig, Leipzig, Germany.
⁴ Scleroderma and Raynaud's UK, London, UK.
⁵ Federation of the European Scleroderma Associations (FESCA) aisbl, Tournai, Belgium.
⁶ Federation of European Scleroderma Associations (FESCA), Saint Maur, Belgium.
⁷ Department of Rheumatology A, Descartes University, APHP, Cochin Hospital, Paris, France.
⁸ Department of Rheumatology, Hospital Universitario 12 de Octubre, Madrid, Spain.
⁹ Department of Rheumatology and Immunology, University of Pécs, Pécs, Hungary.
¹⁰ Centre for Rheumatology, University College London, Royal Free Campus, London, UK.
¹¹ Department of Rheumatology, Lund University, Lund, Sweden.
¹² Department of Rheumatology and Internal Medicine, Medical University of Bialystok, Bialystok, Poland.
¹³ Department of Experimental and Clinical Medicine, Division of Rheumatology AOUC, University of Florence, Florence, Italy.
¹⁴ IRCCS San Raffaele Hospital, Unit of Immunology, Rheumatology, Allergy and Rare diseases (UnIRAR), Milan, Italy.
¹⁵ Department of Internal Medicine and Rheumatology, Cantacuzino Hospital, Carol Davila University of Medicine and Pharmacy, Bucharest, Romania.
¹⁶ Department of Rheumatology and Clinical Immunology, Justus-Liebig University Giessen, Campus Kerckhoff, Bad Nauheim, Germany.
¹⁷ Division of Rheumatology and Research, Diakonhjemmet Hospital, Oslo, Norway.
¹⁸ Regional Research Support, Oslo University Hospital, Oslo, Norway.
¹⁹ Department of Rheumatology, University Hospital of Zurich, Zurich, Switzerland oliver.distler@usz.ch.

^# Contributed equally.

PMID: 34824049
DOI: 10.1136/annrheumdis-2021-220702

Abstract

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.

Keywords: health care; immune system diseases; patient reported outcome measures; quality indicators; scleroderma; systemic.

Publication types

Observational Study
Research Support, Non-U.S. Gov't

MeSH terms

Cohort Studies
Disability Evaluation
Female
Humans
Male
Middle Aged
Patient Reported Outcome Measures
Quality of Life
Reproducibility of Results
Rheumatology*
Scleroderma, Localized*
Scleroderma, Systemic* / complications
Severity of Illness Index
Surveys and Questionnaires