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SAT0467 The Five Prospective Observational Trials of the International Systemic Sclerosis FP7-Health Research Project Desscipher: A Interim Report
  1. M. Frerix1,
  2. G. Abignano2,
  3. Y. Allanore3,
  4. J. Avouac3,
  5. L. Czirják4,
  6. F. Del Galdo5,
  7. C. Denton6,
  8. O. Distler7,
  9. I. Foeldvari8,
  10. B. Garay Toth9,
  11. S. Guiducci10,
  12. D. Huscher11,
  13. V. Lόránd4,
  14. V.K. Jaeger12,
  15. M. Matucci-Cerinic10,
  16. B. Maurer7,
  17. S. Nihtyanova6,
  18. G. Riemekasten13,
  19. E. Siegert13,
  20. I.H. Tarner1,
  21. G. Valentini14,
  22. S. Vettori14,
  23. U.A. Walker12,
  24. U. Müller-Ladner1
  25. on behalf of the DeSScipher Consortium und contributing EUSTAR centers
  1. 1Department of Rheumatology and Clinical Immunology, Justus-Liebig University Giessen, Kerckhoff Clinic Bad Nauheim, Bad Nauheim
  2. 2Leeds Musculoskeletal Biomedical Research Unit (LMBRU), University of Leeds, Leeds, Germany
  3. 3Department of Rheumatology, University of Paris Descartes, Paris, France
  4. 4Department of Rheumatology and Immunology, University of Pécs, Pécs, Hungary
  5. 5Leeds Musculoskeletal Biomedical Research Unit (LMBRU), University of Leeds, Leeds
  6. 6Department of Rheumatology, University College London, Royal Free Hospital, London, United Kingdom
  7. 7Department of Rheumatology, University Hospital Zurich, Zurich, Switzerland
  8. 8Centre for Pediatric Rheumatology, Hamburg, Germany
  9. 9Federation of European Scleroderma Associations aisbl, Budapest, Hungary
  10. 10Department of Rheumatology, University of Florence, Florence, Italy
  11. 11German Rheumatism Research Centre, Charité, Berlin, Germany
  12. 12Department of Rheumatology, University of Basel, Basel, Switzerland
  13. 13Department of Rheumatology and Immunology, University Hospital Charité, Berlin, Germany
  14. 14Department of Rheumatology, Second University of Naples, Naples, Italy


Background Due to lack of adequate clinical research data, drug treatment of the rare disease systemic sclerosis (SSc) is commonly off-label. The international EC-funded research project DeSScipher (acronym for “to decipher the optimal management of systemic sclerosis”) was designed to increase the evidence-based treatment strategies for SSc patients and subsequently to improve their long-term quality-of-life.

Objectives The primary objective is to compare the outcomes of different treatments with respect to efficacy and safety of currently used off-label drugs from the early to the advanced phases of the main SSc-associated organ dysfunctions in a routine rheumatology in- and outpatient setting.

Methods Five prospective observational trials (OTs), carried out within the EULAR Scleroderma Trials and Research (EUSTAR) group, have been designed to analyze current treatment approaches of early functionally relevant manifestations such as digital ulcers (OT1) and hand arthritis (OT2) to the morbidity and mortality-driving manifestations such as interstitial lung disease (OT3), pulmonary hypertension (OT4) and severe heart disease (OT5). The study protocols are accessible at Identifiers NCT01836263, NCT01834157, NCT01858259, NCT01840748, NCT01829126.

Results Between April 2013 and January 2015, 1781 SSc patients have been screened at 27 contributing EUSTAR centers. 1577 (89%) patients have been enrolled into at least one of the five OTs. In particular, 1179, 127, 981, 237 and 716 patients have been enrolled into OT1-5, respectively (3240 in total; a given patient could be enrolled into multiple OTs), which represents a baseline patient recruitment rate of 79% of the target number of 4098 patients (accordingly 226%, 79%, 59%, 25% and 91% of the required number of 522, 160, 1670, 960, 786 patients for OT1-5, respectively). The completion of 1-year (OT3, OT5) and 2-year follow-up visits (OT1, OT2, OT4) are pending.

Conclusions DeSScipher is currently the largest prospective observational research project ever for SSc. While patient recruitment is still ongoing, preliminary results of the five OTs are expected in late 2015 and the final results depending on the completion of follow-up visits are expected between 2017 and 2018.

Acknowledgements The DeSScipher project was funded by the European Community's Framework Programme 7 (FP7-HEALTH-2012.2.4.4-2 - Observational trials in rare diseases) under grant agreement N° 305495.

We acknowledge the contribution of the following EUSTAR centers: Wuppertal (member N°192), Lille (93), Bad Bramstedt (187), Moscow (78), Assiut (168), Moscow (190), Bucharest (100), Monserrato (142), Iasi (162), Cluj-Napoca (16), Frankfurt (124), Salford/Manchester (80), Tübingen (56), Ancona (34), Zagreb (51) and Roma (94).

Disclosure of Interest None declared

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