Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)

Journal article

Publication Details

Author(s): Herrick AL, Pan X, Peytrignet S, Lunt M, Hesselstrand R, Mouthon L, Silman A, Brown E, Czirjak L, Distler J, Distler O, Fligelstone K, Gregory WJ, Ochiel R, Vonk M, Ancuta C, Ong VH, Farge D, Hudson M, Matucci-Cerinic M, Balbir-Gurman A, Midtvedt O, Jordan AC, Jobanputra P, Stevens W, Moinzadeh P, Hall FC, Agard C, Anderson ME, Diot E, Madhok R, Akil M, Buch MH, Chung L, Damjanov N, Gunawardena H, Lanyon P, Ahmad Y, Chakravarty K, Jacobsen S, Macgregor AJ, Mchugh N, Mueller-Ladner U, Riemekasten G, Becker M, Roddy J, Carreira PE, Fauchais AL, Hachulla E, Hamilton J, Inanc M, Mclaren JS, Van Laar JM, Pathare S, Proudman S, Rudin A, Sahhar J, Coppere B, Serratrice C, Sheeran T, Veale DJ, Grange C, Trad GS, Denton CP
Journal: Annals of the Rheumatic Diseases
Publication year: 2017
ISSN: 0003-4967


The rarity of early diffuse cutaneous systemic sclerosis (dcSSc) makes randomised controlled trials very difficult. We aimed to use an observational approach to compare effectiveness of currently used treatment approaches.This was a prospective, observational cohort study of early dcSSc (within three years of onset of skin thickening). Clinicians selected one of four protocols for each patient: methotrexate, mycophenolate mofetil (MMF), cyclophosphamide or 'no immunosuppressant'. Patients were assessed three-monthly for up to 24 months. The primary outcome was the change in modified Rodnan skin score (mRSS). Confounding by indication at baseline was accounted for using inverse probability of treatment (IPT) weights. As a secondary outcome, an IPT-weighted Cox model was used to test for differences in survival.Of 326 patients recruited from 50 centres, 65 were prescribed methotrexate, 118 MMF, 87 cyclophosphamide and 56 no immunosuppressant. 276 (84.7%) patients completed 12 and 234 (71.7%) 24 months follow-up (or reached last visit date). There were statistically significant reductions in mRSS at 12 months in all groups: -4.0 (-5.2 to -2.7) units for methotrexate, -4.1 (-5.3 to -2.9) for MMF, -3.3 (-4.9 to -1.7) for cyclophosphamide and -2.2 (-4.0 to -0.3) for no immunosuppressant (p value for between-group differences=0.346). There were no statistically significant differences in survival between protocols before (p=0.389) or after weighting (p=0.440), but survival was poorest in the no immunosuppressant group (84.0%) at 24 months.These findings may support using immunosuppressants for early dcSSc but suggest that overall benefit is modest over 12 months and that better treatments are needed.NCT02339441.

FAU Authors / FAU Editors

Distler, Jörg PD Dr.
Heisenberg-Professur für Molekulare Mechanismen der Organfibrose

External institutions with authors

Assistance Publique-Hôpitaux de Paris (AP-HP)
Cambridge University Hospital
Cannock Chase Hospital
Centre Hospitalier Régional Universitaire de TOURS
Charité - Universitätsmedizin Berlin
Gateshead Health NHS Foundation Trust
Glasgow Royal Infirmary (GRI)
Grigore T. Popa University of Medicine and Pharmacy / Universitatea de Medicină și Farmacie "Grigore T. Popa" (UMF Iași)
Hôpital Edouard Herriot
Hôpital général juif - Jewish General Hospital
Hôpital Saint Joseph
Hospices Civils de Lyon (CHU)
Hospital Center University De Limoges Dupuytren
Hospital Universitario 12 de Octubre
Istanbul University / İstanbul Üniversitesi
James Cook University Hospital
Justus-Liebig-Universität Gießen
LILLE 1 University - Science and Technology
Lund University / Lunds universitet
Monash University
North Bristol NHS Trust
Nottingham University Hospitals
Oslo University Hospital / Oslo Universitetssykehus
Peter Maddison Rheumatology Centre
Queen's Hospital
Radboud University Nijmegen
Rappaport Faculty of Medicine
Royal National Hospital for Rheumatic Diseases
Royal Perth Hospital
Salford Royal NHS Foundation Trust
Sheffield Teaching Hospitals NHS Foundation Trust
Stanford University
St Vincent's Hospital
St Vincent's University Hospital
The University of Liverpool
UCL Centre for Rheumatology and Connective Tissue Diseases
Università degli Studi di Firenze / University of Florence
Universität Köln
Universität zu Lübeck
Universität Zürich (UZH)
Université de Nantes
University College London (UCL)
University Hospitals Birmingham NHS Foundation Trust
University Medical Centre Utrecht (UMC Utrecht)
University of Adelaide
University of Belgrade / Универзитет у Београду
University of Copenhagen
University of East Anglia
University of Gothenburg / Göteborgs universitet
University of Leeds
University of Manchester
University of Oxford
University of Paris 5 - René Descartes / Université Paris V René Descartes
University of Paris 7 - Denis Diderot / Université Paris VII Denis Diderot
University of Pécs
Whytemans Brae Hospital

How to cite

Herrick, A.L., Pan, X., Peytrignet, S., Lunt, M., Hesselstrand, R., Mouthon, L.,... Denton, C.P. (2017). Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). Annals of the Rheumatic Diseases.

Herrick, Ariane L., et al. "Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)." Annals of the Rheumatic Diseases (2017).


Last updated on 2018-06-10 at 02:47