Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis

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...

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Main Authors: Ulf Mueller-Ladner, Marco Matucci-Cerinic, Yannick Allanore, 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
Format: Article
Language:English
Published: BMJ Publishing Group 2024-11-01
Series:RMD Open
Online Access:https://rmdopen.bmj.com/content/10/4/e004653.full
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author Ulf Mueller-Ladner
Marco Matucci-Cerinic
Yannick Allanore
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
author_facet Ulf Mueller-Ladner
Marco Matucci-Cerinic
Yannick Allanore
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
author_sort Ulf Mueller-Ladner
collection DOAJ
description 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.
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spelling doaj-art-7b4b4609e7d54da5a3ff297304e83bed2025-08-20T02:32:20ZengBMJ Publishing GroupRMD Open2056-59332024-11-0110410.1136/rmdopen-2024-004653Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosisUlf Mueller-Ladner0Marco Matucci-Cerinic1Yannick Allanore2Oliver Distler3Cosimo Bruni4Otylia Kowal-Bielecka5Kim Fligelstone6Roger Hesselstrand7László Czirják8Carina Mihai9Gunnel Sandqvist10Rucsandra Dobrota11Patricia E Carreira12Ana Maria Gherghe13Tore Kvien14Turid Heiberg15Alexandru Garaiman16Mike Oliver Becker17Annelise Roennow18Ann Tyrrell Kennedy19Chris Denton20Department of Rheumatology and Clinical Immunology, Kerckhoff Klinik, Justus-Liebig University of Giessen, Bad Nauheim, GermanyUnit of Immunology, Rheumatology, Allergy and Rare diseases (UnIRAR), and Inflammation, fibrosis and ageing initiative (INFLAGE), IRCCS San Raffaele Hospital, Milano, ItalyDepartment of Rheumatology A, Universite Paris Descartes, APHP and Cochin Hospital, Paris, FranceDepartment of Rheumatology, University Hospital Zurich, Zurich, SwitzerlandDepartment of Rheumatology, University Hospital Zurich, Zurich, SwitzerlandDepartment of Rheumatology and Internal Medicine, Medical University of Bialystok, Bialystok, PolandFederation of European Scleroderma Associations (FESCA), London, UKDepartment of Rheumatology, Lund University, Lund, SwedenDepartment of Rheumatology and Immunology, Medical School, University of Pécs, Pécs, HungaryDepartment of Rheumatology, University Hospital Zurich, Zurich, SwitzerlandDepartment of Rheumatology, Lund University, Lund, SwedenDepartment of Rheumatology, University Hospital Zurich, Zurich, SwitzerlandDepartment of Rheumatology, Hospital Universitario Doce de Octubre, Madrid, SpainDepartment of Internal Medicine and Rheumatology, Carol Davila University of Medicine and Pharmacy, Cantacuzino Hospital, Bucharest, RomaniaCenter for Treatment of Rheumatic and Musculoskeletal Diseases (REMEDY), Diakonhjemmet Hospital, Oslo, NorwayRegional Research Support, Oslo University Hospital, Oslo, NorwayDepartment of Rheumatology, University Hospital Zurich, Zurich, SwitzerlandDepartment of Rheumatology, University Hospital Zurich, Zurich, SwitzerlandFederation of European Scleroderma Associations (FESCA), Aalborg, DenmarkFederation of European Scleroderma Associations (FESCA), Tournai, BelgiumCentre for Rheumatology, University College London, London, UKObjective 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.https://rmdopen.bmj.com/content/10/4/e004653.full
spellingShingle Ulf Mueller-Ladner
Marco Matucci-Cerinic
Yannick Allanore
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
Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis
RMD Open
title Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis
title_full Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis
title_fullStr Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis
title_full_unstemmed Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis
title_short Performance of the EULAR Systemic sclerosis Impact of Disease (ScleroID) questionnaire as a patient-reported outcome measure for patients with diffuse systemic sclerosis
title_sort performance of the eular systemic sclerosis impact of disease scleroid questionnaire as a patient reported outcome measure for patients with diffuse systemic sclerosis
url https://rmdopen.bmj.com/content/10/4/e004653.full
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