Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis
Ulf Mueller-Ladner,
Marco Matucci-Cerinic,
Oliver Distler,
Cosimo Bruni,
Otylia Kowal-Bielecka,
Kim Fligelstone,
Roger Hesselstrand,
László Czirják,
Carina Mihai,
Gunnel Sandqvist,
Rucsandra Dobrota,
Patricia E Carreira,
Ana Maria Gherghe,
Tore Kvien,
Turid Heiberg,
Alexandru Garaiman,
Mike Oliver Becker,
Annelise Roennow,
Ann Tyrrell Kennedy,
Chris Denton
Affiliations
Ulf Mueller-Ladner
Department of Rheumatology and Clinical Immunology, Justus Liebig University Giessen, Campus Kerckhoff, Bad Nauheim, Germany
Marco Matucci-Cerinic
Department of Experimental and Clinical Medicine, Division of Rheumatology AOUC, University of Florence, Florence, Italy
Oliver Distler
Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
Cosimo Bruni
Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
Otylia Kowal-Bielecka
Rheumatology and Internal Medicine, Medical University of Bialystok, Bialystok, Poland
Kim Fligelstone
Federation of European Scleroderma Associations (FESCA), London, UK
Roger Hesselstrand
Department of Rheumatology, Lund University, Lund, Sweden
László Czirják
Department of Rheumatology and Immunology, Medical School, University of Pécs, Pécs, Hungary
Carina Mihai
Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
Gunnel Sandqvist
Department of Clinical Sciences Lund, Section of Rheumatology, Lund University, Skåne University Hospital, Lund, Sweden
Rucsandra Dobrota
Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
Patricia E Carreira
Department of Rheumatology, Hospital Universitario Doce de Octubre, Madrid, Spain
Ana Maria Gherghe
Department of Internal Medicine and Rheumatology, Carol Davila University of Medicine and Pharmacy, Cantacuzino Hospital, Bucharest, Romania
Tore Kvien
Center for Treatment of Rheumatic and Musculoskeletal Diseases (REMEDY), Diakonhjemmet Hospital, Oslo, Norway
Turid Heiberg
Regional Research Support, Oslo University Hospital, Oslo, Norway
Alexandru Garaiman
Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
Mike Oliver Becker
Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
Annelise Roennow
Federation of European Scleroderma Associations (FESCA), Aalborg, Denmark
Ann Tyrrell Kennedy
Federation of European Scleroderma Associations (FESCA), Tournai, Belgium
Chris Denton
Centre for Rheumatology, University College London, London, UK
Objective Systemic sclerosis Impact of Disease (ScleroID) is the first comprehensive patient-reported outcome measure (PROM) specifically developed for systemic sclerosis (SSc). We investigated the performance of ScleroID in patients with diffuse cutaneous SSc (dcSSc), as a prerequisite for its use in randomised controlled trials (RCTs) testing potentially disease-modifying drugs.Methods All patients with dcSSc from the large, multicentric, ScleroID cohort were included. SSc-Health Assessment Questionnaire (HAQ), EuroQol-5 Dimensions and 36-item Short Form Health Survey (SF-36) were used as comparators. The study includes a longitudinal arm with a reliability visit at 7±3 days and a 12 months follow-up visit. The performance of ScleroID in dcSSc was assessed according to the Outcome Measures in Rheumatology filter.Results In total, 152 dcSSc patients were analysed (29% male, median age 54 years). ScleroID reflected well the disease impact of dcSSc, showing a good construct validity with high Spearman’s correlation coefficients with comparators (SSc-HAQ, 0.79, 95% CI (0.69, 0.86); HAQ-Disability Index, 0.72 95% CI (0.60, 0.80); SF-36 physical score, −0.69 95% CI (−0.77, –0.60)). The internal consistency was strong (Cronbach’s alpha 0.87, split-half reliability coefficient 0.88).In the longitudinal arm, 44 patients had a reliability visit and 113 had a follow-up visit, of whom 19/113 (17%) reported a significant change (11 improved, 8 worsened). ScleroID showed a good consistency and discriminative ability with excellent test–retest reliability (intraclass correlation coefficient 0.89, 95% CI (0.84, 0.92)) and moderate sensitivity to change (standardised response mean −0.63 in the improved subgroup and 0.48 in the worsened subgroup), but superior to the comparators.Conclusion The European Alliance of Associations for Rheumatology (EULAR) ScleroID performs well for patients with dcSSc. This supports its inclusion and regular assessment as PROM in RCTs.
