Performance of the EULAR Systemic Sclerosis Impact of Disease (ScleroID) Questionnaire As a Patient-reported Outcome Measure for Patients with Diffuse Systemic Sclerosis

Overview

Journal RMD Open

Publisher BMJ Publishing Group

Specialty Rheumatology

Date 2024 Nov 28

PMID 39608865

Authors

Rucsandra Dobrota

Alexandru Garaiman

Kim Fligelstone

Ann Tyrrell Kennedy

Annelise Roennow

Yannick Allanore

Patricia E Carreira

Laszlo Czirjak

Chris Denton

Roger Hesselstrand

Gunnel Sandqvist

Otylia Kowal-Bielecka

Cosimo Bruni

Marco Matucci-Cerinic

Carina Mihai

Ana Maria Gherghe

Ulf Mueller-Ladner

Tore Kvien

Turid Heiberg

Oliver Distler

Mike Oliver Becker

Affiliations

Soon will be listed here.

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.

References

Rannou F, Poiraudeau S, Berezne A, Baubet T, Le-Guern V, Cabane J . Assessing disability and quality of life in systemic sclerosis: construct validities of the Cochin Hand Function Scale, Health Assessment Questionnaire (HAQ), Systemic Sclerosis HAQ, and Medical Outcomes Study 36-Item Short Form Health Survey. Arthritis Rheum. 2007; 57(1):94-102. DOI: 10.1002/art.22468. View

Mouthon L, Poiraudeau S, Vernon M, Papadakis K, Perchenet L, Khanna D . Psychometric validation of the Hand Disability in Systemic Sclerosis-Digital Ulcers (HDISS-DU®) patient-reported outcome instrument. Arthritis Res Ther. 2020; 22(1):3. PMC: 6945532. DOI: 10.1186/s13075-019-2087-4. View

Gossec L, de Wit M, Kiltz U, Braun J, Kalyoncu U, Scrivo R . A patient-derived and patient-reported outcome measure for assessing psoriatic arthritis: elaboration and preliminary validation of the Psoriatic Arthritis Impact of Disease (PsAID) questionnaire, a 13-country EULAR initiative. Ann Rheum Dis. 2014; 73(6):1012-9. DOI: 10.1136/annrheumdis-2014-205207. View

Volkmann E, Andreasson K, Smith V . Systemic sclerosis. Lancet. 2022; 401(10373):304-318. PMC: 9892343. DOI: 10.1016/S0140-6736(22)01692-0. View

Pauling J, Caetano J, Campochiaro C, Luca G, Gheorghiu A, Lazzaroni M . Patient-reported outcome instruments in clinical trials of systemic sclerosis. J Scleroderma Relat Disord. 2022; 5(2):90-102. PMC: 8922614. DOI: 10.1177/2397198319886496. View

Tugwell P, Boers M, Brooks P, Simon L, Strand V, Idzerda L . OMERACT: an international initiative to improve outcome measurement in rheumatology. Trials. 2007; 8:38. PMC: 2169260. DOI: 10.1186/1745-6215-8-38. View

Gossec L, Dougados M, Rincheval N, Balanescu A, Boumpas D, Canadelo S . Elaboration of the preliminary Rheumatoid Arthritis Impact of Disease (RAID) score: a EULAR initiative. Ann Rheum Dis. 2008; 68(11):1680-5. DOI: 10.1136/ard.2008.100271. View

Cornett K, Menezes M, Bray P, Shy R, Moroni I, Pagliano E . Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS. Ann Clin Transl Neurol. 2020; 7(9):1713-1715. PMC: 7480902. DOI: 10.1002/acn3.51145. View

Gualtierotti R, Ingegnoli F, Scalone L, Cortesi P, Bruschi E, Gerosa M . Feasibility, acceptability and construct validity of EQ-5D in systemic sclerosis. Swiss Med Wkly. 2017; 146:w14394. DOI: 10.4414/smw.2016.14394. View

10.

Lafyatis R, Valenzi E . Assessment of disease outcome measures in systemic sclerosis. Nat Rev Rheumatol. 2022; 18(9):527-541. DOI: 10.1038/s41584-022-00803-6. View

11.

Crossnohere N, Brundage M, Calvert M, King M, Reeve B, Thorner E . International guidance on the selection of patient-reported outcome measures in clinical trials: a review. Qual Life Res. 2020; 30(1):21-40. DOI: 10.1007/s11136-020-02625-z. View

12.

Denton C . Challenges in systemic sclerosis trial design. Semin Arthritis Rheum. 2019; 49(3S):S3-S7. DOI: 10.1016/j.semarthrit.2019.09.019. View

13.

Gossec L, Paternotte S, Aanerud G, Balanescu A, Boumpas D, Carmona L . Finalisation and validation of the rheumatoid arthritis impact of disease score, a patient-derived composite measure of impact of rheumatoid arthritis: a EULAR initiative. Ann Rheum Dis. 2011; 70(6):935-42. DOI: 10.1136/ard.2010.142901. View

14.

Becker M, Dobrota R, Garaiman A, Debelak R, Fligelstone K, Tyrrell Kennedy A . Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire. Ann Rheum Dis. 2021; 81(4):507-515. DOI: 10.1136/annrheumdis-2021-220702. View

15.

Tugwell P, Boers M, DAgostino M, Beaton D, Boonen A, Bingham 3rd C . Updating the OMERACT filter: implications of filter 2.0 to select outcome instruments through assessment of "truth": content, face, and construct validity. J Rheumatol. 2014; 41(5):1000-4. PMC: 4212637. DOI: 10.3899/jrheum.131310. View

16.

Koo T, Li M . A Guideline of Selecting and Reporting Intraclass Correlation Coefficients for Reliability Research. J Chiropr Med. 2016; 15(2):155-63. PMC: 4913118. DOI: 10.1016/j.jcm.2016.02.012. View

17.

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. 2016; 46(5):609-614. DOI: 10.1016/j.semarthrit.2016.10.002. View

18.

Khanna D, Lovell D, Giannini E, Clements P, Merkel P, Seibold J . Development of a provisional core set of response measures for clinical trials of systemic sclerosis. Ann Rheum Dis. 2007; 67(5):703-9. PMC: 3887552. DOI: 10.1136/ard.2007.078923. View

19.

Nagy G, Dobrota R, Becker M, Minier T, Varju C, Kumanovics G . Characteristics of ScleroID highlighting musculoskeletal and internal organ implications in patients afflicted with systemic sclerosis. Arthritis Res Ther. 2023; 25(1):84. PMC: 10199621. DOI: 10.1186/s13075-023-03063-1. View

20.

Kluetz P, OConnor D, Soltys K . Incorporating the patient experience into regulatory decision making in the USA, Europe, and Canada. Lancet Oncol. 2018; 19(5):e267-e274. DOI: 10.1016/S1470-2045(18)30097-4. View