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

Rucsandra Dobrota^{1

2}, Alexandru Garaiman¹, Kim Fligelstone³, Ann Tyrrell Kennedy⁴, Annelise Roennow⁵, Yannick Allanore⁶, Patricia E Carreira⁷, László Czirják⁸, Chris Denton^{9

10}, Roger Hesselstrand¹¹, Gunnel Sandqvist¹¹, Otylia Kowal-Bielecka¹², Cosimo Bruni^{1

13}, Marco Matucci-Cerinic^{13

14}, Carina Mihai¹, Ana Maria Gherghe¹⁵, Ulf Mueller-Ladner¹⁶, Tore Kvien^{17

18}, Turid Heiberg¹⁹, Oliver Distler^{1

2}, Mike Oliver Becker^{20

2}

Affiliations

¹ Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland.
² Center of Experimental Rheumatology, University of Zurich, Zurich, Switzerland.
³ Federation of European Scleroderma Associations (FESCA), London, UK.
⁴ Federation of European Scleroderma Associations (FESCA), Tournai, Belgium.
⁵ Federation of European Scleroderma Associations (FESCA), Aalborg, Denmark.
⁶ Department of Rheumatology A, Universite Paris Descartes, APHP and Cochin Hospital, Paris, France.
⁷ Department of Rheumatology, Hospital Universitario Doce de Octubre, Madrid, Spain.
⁸ Department of Rheumatology and Immunology, Medical School, University of Pécs, Pécs, Hungary.
⁹ Centre for Rheumatology, University College London, London, UK.
¹⁰ Royal Free Hospital, London, UK.
¹¹ Department of Rheumatology, Lund University, Lund, Sweden.
¹² 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, Carol Davila University of Medicine and Pharmacy, Cantacuzino Hospital, Bucharest, Romania.
¹⁶ Department of Rheumatology and Clinical Immunology, Justus Liebig University Giessen, Campus Kerckhoff, Bad Nauheim, Germany.
¹⁷ Center for Treatment of Rheumatic and Musculoskeletal Diseases (REMEDY), Diakonhjemmet Hospital, Oslo, Norway.
¹⁸ Faculty of Medicine, University of Oslo, Oslo, Norway.
¹⁹ Regional Research Support, Oslo University Hospital, Oslo, Norway.
²⁰ Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland MikeOliver.Becker@usz.ch.

PMID: 39608865
PMCID: PMC11603699
DOI: 10.1136/rmdopen-2024-004653

Multicenter Study

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

Rucsandra Dobrota et al. RMD Open. 2024.

. 2024 Nov 27;10(4):e004653.

doi: 10.1136/rmdopen-2024-004653.

Authors

Affiliations

¹ Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland.
² Center of Experimental Rheumatology, University of Zurich, Zurich, Switzerland.
³ Federation of European Scleroderma Associations (FESCA), London, UK.
⁴ Federation of European Scleroderma Associations (FESCA), Tournai, Belgium.
⁵ Federation of European Scleroderma Associations (FESCA), Aalborg, Denmark.
⁶ Department of Rheumatology A, Universite Paris Descartes, APHP and Cochin Hospital, Paris, France.
⁷ Department of Rheumatology, Hospital Universitario Doce de Octubre, Madrid, Spain.
⁸ Department of Rheumatology and Immunology, Medical School, University of Pécs, Pécs, Hungary.
⁹ Centre for Rheumatology, University College London, London, UK.
¹⁰ Royal Free Hospital, London, UK.
¹¹ Department of Rheumatology, Lund University, Lund, Sweden.
¹² 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, Carol Davila University of Medicine and Pharmacy, Cantacuzino Hospital, Bucharest, Romania.
¹⁶ Department of Rheumatology and Clinical Immunology, Justus Liebig University Giessen, Campus Kerckhoff, Bad Nauheim, Germany.
¹⁷ Center for Treatment of Rheumatic and Musculoskeletal Diseases (REMEDY), Diakonhjemmet Hospital, Oslo, Norway.
¹⁸ Faculty of Medicine, University of Oslo, Oslo, Norway.
¹⁹ Regional Research Support, Oslo University Hospital, Oslo, Norway.
²⁰ Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland MikeOliver.Becker@usz.ch.

PMID: 39608865
PMCID: PMC11603699
DOI: 10.1136/rmdopen-2024-004653

Abstract

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.

Keywords: Connective Tissue Diseases; Health-Related Quality Of Life; Patient Reported Outcome Measures; Scleroderma, Systemic.

PubMed Disclaimer

Conflict of interest statement

Competing interests: RD: speakers bureau for Actelion, advisory board for Boehringer-Ingelheim, grants/research support from Pfizer, Actelion, Iten-Kohaut foundation, Walter und Gertrud Siegenthaler Fellowship, congress participation support from Amgen, Otsuka. AG, KF, ATK, AR, YA, PEC and LC: none reported. CD: consultancy fees from: Janssen, GlaxoSmithKline, Bayer, Sanofi, Boehringer Ingelheim, Roche, CSL Behring, Corbus, Acceleron, Horizon, Arxx, Lilly, Novartis, Certa and research grants to institution from: Abbvie, Servier, Horizon, GlaxoSmithKline. RH, GS, OK-B: none reported.CB: consulting for Boehringer Ingelheim. Research grants from Foundation for Research in Rheumatology (FOREUM), Gruppo Italiano Lotta alla Sclerodermia (GILS), European Scleroderma Trials and Research Group (EUSTAR), Scleroderma Clinical Trials Consortium (SCTC), Scleroderma Research Foundation (SRF), Novartis Foundation for biological-medical research, EMDO Foundation. Educational grants from AbbVie and Wellcome Trust. Congress participation support from Boehringer Ingelheim and Müller-Hartmann foundation. MM-C: none reported. CM received speaker and/or consultancy fees from Janssen-Cilag AG, Boehringer Ingelheim, Deutsche Rheumaakademie, MED Talks Switzerland, Mepha, Novartis and PlayToKnow AG, and travel support for congress from Boehringer Ingelheim. AMG: consulting and congress participation support from Boehringer Ingelheim. Research grant from Foundation for Research in Rheumatology (FOREUM). UM-L, TK and TH: none reported. OD: has/had consultancy relationship with and/or has received research funding from and/or has served as a speaker for the following companies in the area of potential treatments for systemic sclerosis and its complications in the last three calendar years: 4P-Pharma, Abbvie, Acceleron, Alcimed, Altavant, Amgen, AnaMar, Argenx, Arxx, AstraZeneca, Blade, Bayer, Boehringer Ingelheim, Cantargia AB, Corbus, CSL Behring, Galderma, Galapagos, Glenmark, Gossamer, Horizon, Janssen, Kymera, Lupin, Medscape, Merck, Miltenyi Biotec, Mitsubishi Tanabe, Nkarta Inc., Novartis, Orion, Prometheus, Redxpharma, Roivant, EMD Serono, Topadur and UCB. Patent issued 'mir-29 for the treatment of systemic sclerosis' (US8247389, EP2331143). Cofounder of CITUS AG. OD is a member of the editorial board of RMD open. MB has consultancy relationship with and/or received research funding from and/or served as speaker for the following companies in the area of potential treatments for systemic sclerosis and its complications in the last three calendar years: GSK, Amgen, Novartis and outside of this research area: Vifor.

References

1. Lafyatis R, Valenzi E. Assessment of disease outcome measures in systemic sclerosis. Nat Rev Rheumatol. 2022;18:527–41. doi: 10.1038/s41584-022-00803-6. - DOI - PubMed
1. Pauling JD, Caetano J, Campochiaro C, et al. Patient-reported outcome instruments in clinical trials of systemic sclerosis. J Scleroderma Relat Disord. 2020;5:90–102. doi: 10.1177/2397198319886496. - DOI - PMC - PubMed
1. Ingegnoli F, Carmona L, Castrejon I. Systematic review of systemic sclerosis-specific instruments for the EULAR Outcome Measures Library: An evolutional database model of validated patient-reported outcomes. Semin Arthritis Rheum. 2017;46:609–14. doi: 10.1016/j.semarthrit.2016.10.002. - DOI - PubMed
1. Crossnohere NL, Brundage M, Calvert MJ, et al. International guidance on the selection of patient-reported outcome measures in clinical trials: a review. Qual Life Res. 2021;30:21–40. doi: 10.1007/s11136-020-02625-z. - DOI - PubMed
1. Kluetz PG, O’Connor DJ, Soltys K. Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada. Lancet Oncol. 2018;19:e267–74. doi: 10.1016/S1470-2045(18)30097-4. - DOI - PubMed

Publication types

Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

LinkOut - more resources

Full Text Sources
- HighWire
- PubMed Central

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

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

Affiliations

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

Authors

Affiliations

Abstract

Conflict of interest statement

References

Publication types

MeSH terms

LinkOut - more resources

Full Text Sources