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AB0669 The eular systemic sclerosis impact of disease (SCLEROID) score – a new patient-reported outcome measure for patients with systemic sclerosis – preliminary results from the ongoing validation study
  1. R Dobrota1,
  2. M Becker1,
  3. K Fligelstone2,
  4. J Fransen3,
  5. A Kennedy4,
  6. Y Allanore5,
  7. P Carreira6,
  8. L Czirjak7,
  9. C Denton8,
  10. R Hesselstrand9,
  11. G Sandqvist9,
  12. O Kowal-Bielecka10,
  13. M Matucci Cerinic11,
  14. C Mihai12,
  15. A Gheorghiu12,
  16. U Müller-Ladner13,
  17. M Vonk3,
  18. T Heiberg14,
  19. O Distler1
  1. 1Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
  2. 2FESCA, London, United Kingdom
  3. 3Department of Rheumatology, Radboud University Medical Center, Nijmegen, Netherlands
  4. 4FESCA, Dublin, Ireland
  5. 5Department of Rheumatology, University Paris Descartes and Cochin Hospital, Paris, France
  6. 6Division of Rheumatology, Hospital 12 de Octubre, Madrid, Spain
  7. 7Department of Immunology and Rheumatology, Faculty of Medicine, University of Pécs, Pécs, Hungary
  8. 8Centre for Rheumatology, Royal Free and University College London Medical School, Royal Free Campus, London, United Kingdom
  9. 9Department of Rheumatology, Lund University, Lund, Sweden
  10. 10Department of Rheumatology and Internal Medicine, Medical University of Bialystok, Bialystok, Poland
  11. 11Department of Rheumatology, University of Florence, Florence, Italy
  12. 12Department of Internal Medicine and Rheumatology, Cantacuzino Hospital, Carol Davila University of Medicine and Pharmacy, Bucharest, Romania
  13. 13Department of Rheumatology and Clinical Immunology, Justus-Liebig University Giessen, Kerckhoff-Klinik Bad Nauheim, Bad Nauheim, Germany
  14. 14Department of Health and Social Sciences, Oestfold University College, Oslo, Norway

Abstract

Background Patient reported outcome measures (PROM) are required as key outcomes in therapeutic trials in systemic sclerosis (SSc). Given the unmet need of a validated, comprehensive PROM in SSc, the ScleroID questionnaire was developed by a team of patients with SSc and medical experts in the field. This is intended as a brief, disease-specific, patient-derived, disease impact score for scientific and clinical use in SSc.

Objectives To present a preliminary analysis from the ongoing ScleroID validation study.

Methods This EULAR-endorsed project involves 11 European centers specialized in SSc. Patients fullfilling the ACR/EULAR 2013 criteria were prospectively included since 05/16 in the ongoing observational cohort study. Patients filled in the ScleroID questionnaire (Figure 1), as well as selected comparators SHAQ, EQ5D, SF36. Additionally, they weighted the 10 dimensions of the ScleroID by distributing 100 points according to the perceived impact on their health. The final score calculation will be based on the ranking of the weights. The study includes a reliability arm (follow-up questionnaire 7–10 days from baseline), as well as a longitudinal arm, looking at sensitivity to change at follow-up visits after 6 and 12 months from baseline.

Results As of 01/2017 the study cohort included 224 patients with valid baseline data, 44 also had a reliability visit and 6 a 6-months follow-up visit. 84.4% of patients were female, 54.4% had limited SSc, median age 58, and median disease duration 8 years. The highest preliminary median weights for ScleroID domains were for Raynaud, impaired hand function, fatigue and pain (Table 1). Except for pain, these dimensions were also scored most highly in the ScleroID questionnaire at baseline.

Table 1

Conclusions The EULAR ScleroID score is a novel tool designed for use in clinical practice and clinical trials to display the disease impact of SSc. In this preliminary analysis, Raynaud syndrome, impaired hand function, and fatigue were the main patient reported drivers of disease impact, however, further recruitment and validation of this new instrument is ongoing.

Disclosure of Interest R. Dobrota: None declared, M. Becker: None declared, K. Fligelstone: None declared, J. Fransen: None declared, A. Kennedy: None declared, Y. Allanore Grant/research support from: Grant/research support from: Bristol-Myers Squibb, Roche/Genentech, Inventiva, Pfizer, Sanofi, and Servier, Consultant for: Actelion, Bayer, Roche/Genentech, Inventiva, Medac, Pfizer, Sanofi, Servier, and UCB, P. Carreira: None declared, L. Czirjak: None declared, C. Denton: None declared, R. Hesselstrand: None declared, G. Sandqvist: None declared, O. Kowal-Bielecka: None declared, M. Matucci Cerinic: None declared, C. Mihai: None declared, A. Gheorghiu: None declared, U. Müller-Ladner: None declared, M. Vonk: None declared, T. Heiberg: None declared, O. Distler Grant/research support from: 4 D Science, AbbVie, Actelion, Active Biotec, Bayer, BiogenIdec, BMS, Boehringer Ingelheim, ChemomAb, EpiPharm, espeRare foundation, Genentech/Roche, GSK, Inventiva, iQone Healthcare, Lilly, medac, Mepha, MedImmune, Mitsubishi Tanabe Pharma, Pharmacyclics, Pfizer, Sanofi, Serodapharm and Sinoxa, Consultant for: 4 D Science, AbbVie, Actelion, Active Biotec, Bayer, BiogenIdec, BMS, Boehringer Ingelheim, ChemomAb, EpiPharm, espeRare foundation, Genentech/Roche, GSK, Inventiva, iQone Healthcare, Lilly, medac, Mepha, MedImmune, Mitsubishi Tanabe Pharma, Pharmacyclics, Pfizer, Sanofi, Serodapharm and Sinoxa

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