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Development and validation of a patient-reported outcome measure for systemic sclerosis: the EULAR Systemic Sclerosis Impact of Disease (ScleroID) questionnaire
  1. Mike O Becker1,
  2. Rucsandra Dobrota1,
  3. Alexandru Garaiman1,
  4. Rudolf Debelak2,3,
  5. Kim Fligelstone4,
  6. Ann Tyrrell Kennedy5,
  7. Annelise Roennow6,
  8. Yannick Allanore7,
  9. Patricia E Carreira8,
  10. László Czirják9,
  11. Christopher P Denton10,
  12. Roger Hesselstrand11,
  13. Gunnel Sandqvist11,
  14. Otylia Kowal-Bielecka12,
  15. Cosimo Bruni13,
  16. Marco Matucci-Cerinic13,14,
  17. Carina Mihai1,15,
  18. Ana Maria Gheorghiu15,
  19. Ulf Mueller-Ladner16,
  20. Joseph Sexton17,
  21. Tore K Kvien17,
  22. Turid Heiberg18,
  23. Oliver Distler1
  1. 1Department of Rheumatology, University Hospital of Zurich, Zurich, Switzerland
  2. 2Department of Psychology, Psychological Methods, Evaluation and Statistics, University of Zurich, Zurich, Switzerland
  3. 3Department of Psychology, Psychological Methodology, University of Leipzig, Leipzig, Germany
  4. 4Scleroderma and Raynaud's UK, London, UK
  5. 5Federation of the European Scleroderma Associations (FESCA) aisbl, Tournai, Belgium
  6. 6Federation of European Scleroderma Associations (FESCA), Saint Maur, Belgium
  7. 7Department of Rheumatology A, Descartes University, APHP, Cochin Hospital, Paris, France
  8. 8Department of Rheumatology, Hospital Universitario 12 de Octubre, Madrid, Spain
  9. 9Department of Rheumatology and Immunology, University of Pécs, Pécs, Hungary
  10. 10Centre for Rheumatology, University College London, Royal Free Campus, London, UK
  11. 11Department of Rheumatology, Lund University, Lund, Sweden
  12. 12Department of Rheumatology and Internal Medicine, Medical University of Bialystok, Bialystok, Poland
  13. 13Department of Experimental and Clinical Medicine, Division of Rheumatology AOUC, University of Florence, Florence, Italy
  14. 14IRCCS San Raffaele Hospital, Unit of Immunology, Rheumatology, Allergy and Rare diseases (UnIRAR), Milan, Italy
  15. 15Department of Internal Medicine and Rheumatology, Cantacuzino Hospital, Carol Davila University of Medicine and Pharmacy, Bucharest, Romania
  16. 16Department of Rheumatology and Clinical Immunology, Justus-Liebig University Giessen, Campus Kerckhoff, Bad Nauheim, Germany
  17. 17Division of Rheumatology and Research, Diakonhjemmet Hospital, Oslo, Norway
  18. 18Regional Research Support, Oslo University Hospital, Oslo, Norway
  1. Correspondence to Dr Oliver Distler, Department of Rheumatology, University of Zurich, University Hospital Zurich, 8091 Zurich, Switzerland; oliver.distler{at}usz.ch

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.

  • scleroderma
  • systemic
  • patient reported outcome measures
  • quality indicators
  • health care
  • immune system diseases

Data availability statement

Data are available on reasonable request. On request, and subject to review by the steering committee, access can be granted to the anonymised raw data and the R code. Deidentified data will be made available via secure data transfer. Data requests may be sent to the ScleroID steering committee, represented by OD, Department of Rheumatology, University Hospital Zurich, University of Zurich, Switzerland. Email oliver.distler@usz.ch.

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Data availability statement

Data are available on reasonable request. On request, and subject to review by the steering committee, access can be granted to the anonymised raw data and the R code. Deidentified data will be made available via secure data transfer. Data requests may be sent to the ScleroID steering committee, represented by OD, Department of Rheumatology, University Hospital Zurich, University of Zurich, Switzerland. Email oliver.distler@usz.ch.

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Footnotes

  • Handling editor Josef S Smolen

  • MOB and RD contributed equally.

  • TH and OD contributed equally.

  • Presented at Part of this work has previously been presented, as follows: Presentations: The EULAR Systemic Sclerosis Impact of Disease score – a new patient-reported outcome measure under development, 4th Scleroderma World Congress, Patient Congress, Lisbon, Feb 18th – 20th, 2016, Poster EULAR 2016, Poster ACR 2016, Abstract book EULAR 2017, Poster ACR 2017, Poster Patients’ Congress SWC 02/2018 Bordeaux, Abstract book EULAR 2018. Meetings: Working group patients and PIs inkl. teleconference – Rome, 06/2015, Task force meetings, EULAR 06/2016 London, ACR 11/2016 Washington, EULAR 06/2017 Madrid, EULAR 2020 Online E-Congress.

  • Contributors The authors as listed on the title page of the manuscript have all made substantial contributions which qualifies them as authors. All authors contributed to critical revisions and approved the final version of the manuscript. MB: design of the study, acquisition of data, analysis, interpretation of data, drafting and revising the article. RDo: design of the study, acquisition of data, analysis, interpretation of data, drafting and revising the article. AG: analysis, interpretation of data, drafting and revising the article. RDe: interpretation of data, revising the article. KF: design of the study, acquisition of data, interpretation of data, revising the article. ATK: design of the study, acquisition of data, interpretation of data, revising the article. AR: acquisition of data, interpretation of data, revising the article. YA: acquisition of data, analysis, interpretation of data, revising the article. PEC: acquisition of data, analysis, interpretation of data, revising the article. LC: acquisition of data, analysis, interpretation of data, revising the article. CPD: acquisition of data, analysis, interpretation of data, revising the article. RH: acquisition of data, analysis, interpretation of data, revising the article. GS: acquisition of data, analysis, interpretation of data, revising the article. OK-B: acquisition of data, analysis, interpretation of data, revising the article. CB: acquisition of data, analysis, interpretation of data, revising the article. MM-C: acquisition of data, analysis, interpretation of data, revising the article. CM: acquisition of data, analysis, interpretation of data, revising the article. AMG: acquisition of data, analysis, interpretation of data, revising the article. UM-L: acquisition of data, interpretation of data, revising the article. JS: design of the study, analysis, interpretation of data, revising the article. TKK: design of the study, analysis, interpretation of data, revising the article. TH: design of the study, acquisition of data, analysis, interpretation of data, drafting and revising the article. OD: design of the study, acquisition of data, analysis, interpretation of data, drafting and revising the article, guarantor.

  • Funding The development and validation of the ScleroID was in part supported by a grant from EULAR.

  • Competing interests MB personal fees from Amgen and Bayer, outside the submitted work. RDo reports grants from Articulum Fellowship, sponsored by Pfizer (2013-2014), grants from Actelion, personal fees from Actelion and Boehringer-Ingelheim, outside the submitted work. AG none with respect to the study. RDe none with respect to the study.KF none with respect to the study. ATK none with respect to the study. AR none with respect to the study. YA reports grants and personal fees from Bayer, Boehringer-Ingelheim, Bristol Myers Squibb, Curzion, Inventiva, Roche and Sanofi during the conduct of the study. PEC reports consultancy relationship and/or research grants from: Abbvie, Actelion, Bayer, Boehringer Ingelheim, Corbus Pharma, Emerald Health Pharmaceuticals, Galapagos, Gesynta Pharma, Inventiva, iQvia, Mitsubishi Tanabe Pharma, MSD, Roche and Sanofi Genzyme. LC none with respect to the study. CPD reports grants and personal fees from GSK, Mitsubishi Tanabe Pharma, Boehringer-Ingelheim, Servier, Arxx Therapeutics, Bayer, Inventiva, Galapagos, Horizon, Roche, CSL Behring, Sanofi during the conduct of the study.RH reports personal lecture fees from Actelion Pharmaceuticals Sweden AB, Boehringer-Ingelheim Sweden AB, and Roche Sweden AB, and has been co-investigator in clinical trials for United Therapeutics and Actelion Pharmaceuticals Sweden AB, and has been involved in research advisory boards for Actelion Pharmaceuticals Sweden AB and Boehringer-Ingelheim Sweden AB. GS none with respect to the study. OK-B reports personal fees and/or congress support from Bayer, Boehringer Ingelheim, CSL Behring, Gilead, Inventiva, Medac, MSD, Novartis, Pfizer, Roche, Sandoz, outside the submitted work. CB reports personal fees from Actelion, personal fees from Boehringer-Ingelheim, personal fees from Eli Lilly, grants from Gruppo Italiano Lotta alla Sclerodermia (GILS), grants from New Horizon Fellowship, grants from European Scleroderma Trials and Research (EUSTAR), grants from Foundation for Research in Rheumatology (FOREUM), grants from Fondazione Italiana per la Ricerca sull’Artrite (FIRA), outside the submitted work. MM-C reports grants and personal fees from Actelion, personal fees from Biogen, personal fees from Bayer, personal fees from Boehringer Ingelheim, personal fees from CSL Behring, personal fees from Eli-Lilly, outside the submitted work. CM reports personal fees from Boehringer Ingelheim, Eli-Lilly, Mepha, MEDTalks Switzerland and congress support from Actelion and Roche, outside the submitted work. AMG reports consultancy fees from Boehringer-Ingelheim and congress support from Abbvie and Novartis, outside the submitted work. UM-L none with respect to the study. JS none with respect to the study. TKK none with respect to the study. TH reports no conflicts of interest.OD has/had consultancy relationship and/or has received research funding in the area of potential treatments of scleroderma and its complications from (last three years): Abbvie, Acceleron Pharma, Alexion, Amgen, AnaMar, Arxx Therapeutics, Baecon Discovery, Bayer, Boehringer Ingelheim, Catenion, Drug Development International, CSL Behring, ChemomAb, GSK, Horizon (Curzion) Pharmaceuticals, Inventiva, Italfarmaco, iQvia, Lilly, Medac, Medscape, Mitsubishi Tanabe Pharma, MSD, Novartis, Pfizer, Roche, Sanofi, Serodapharm, Target Bio Science and UCB In addition, he has a patent mir-29 for the treatment of systemic sclerosis issued (US8247389, EP2331143).

  • Provenance and peer review Not commissioned; externally peer reviewed.

  • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.

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