RT Journal Article
SR Electronic
T1 Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)
JF Annals of the Rheumatic Diseases
JO Ann Rheum Dis
FD BMJ Publishing Group Ltd and European League Against Rheumatism
SP 1207
OP 1218
DO 10.1136/annrheumdis-2016-210503
VO 76
IS 7
A1 Herrick, Ariane L
A1 Pan, Xiaoyan
A1 Peytrignet, Sébastien
A1 Lunt, Mark
A1 Hesselstrand, Roger
A1 Mouthon, Luc
A1 Silman, Alan
A1 Brown, Edith
A1 Czirják, László
A1 Distler, Jörg H W
A1 Distler, Oliver
A1 Fligelstone, Kim
A1 Gregory, William J
A1 Ochiel, Rachel
A1 Vonk, Madelon
A1 Ancuţa, Codrina
A1 Ong, Voon H
A1 Farge, Dominique
A1 Hudson, Marie
A1 Matucci-Cerinic, Marco
A1 Balbir-Gurman, Alexandra
A1 Midtvedt, Øyvind
A1 Jordan, Alison C
A1 Jobanputra, Paresh
A1 Stevens, Wendy
A1 Moinzadeh, Pia
A1 Hall, Frances C
A1 Agard, Christian
A1 Anderson, Marina E
A1 Diot, Elisabeth
A1 Madhok, Rajan
A1 Akil, Mohammed
A1 Buch, Maya H
A1 Chung, Lorinda
A1 Damjanov, Nemanja
A1 Gunawardena, Harsha
A1 Lanyon, Peter
A1 Ahmad, Yasmeen
A1 Chakravarty, Kuntal
A1 Jacobsen, Søren
A1 MacGregor, Alexander J
A1 McHugh, Neil
A1 Müller-Ladner, Ulf
A1 Riemekasten, Gabriela
A1 Becker, Michael
A1 Roddy, Janet
A1 Carreira, Patricia E
A1 Fauchais, Anne Laure
A1 Hachulla, Eric
A1 Hamilton, Jennifer
A1 İnanç, Murat
A1 McLaren, John S
A1 van Laar, Jacob M
A1 Pathare, Sanjay
A1 Proudman, Susannah
A1 Rudin, Anna
A1 Sahhar, Joanne
A1 Coppere, Brigitte
A1 Serratrice, Christine
A1 Sheeran, Tom
A1 Veale, Douglas J
A1 Grange, Claire
A1 Trad, Georges-Selim
A1 Denton, Christopher P
YR 2017
UL http://ard.bmj.com/content/76/7/1207.abstract
AB Objectives 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.Methods 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.Results 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.Conclusions 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.Trial registration number NCT02339441.