PT  - JOURNAL ARTICLE
AU  - Herrick, Ariane L
AU  - Pan, Xiaoyan
AU  - Peytrignet, Sébastien
AU  - Lunt, Mark
AU  - Hesselstrand, Roger
AU  - Mouthon, Luc
AU  - Silman, Alan
AU  - Brown, Edith
AU  - Czirják, László
AU  - Distler, Jörg H W
AU  - Distler, Oliver
AU  - Fligelstone, Kim
AU  - Gregory, William J
AU  - Ochiel, Rachel
AU  - Vonk, Madelon
AU  - Ancuţa, Codrina
AU  - Ong, Voon H
AU  - Farge, Dominique
AU  - Hudson, Marie
AU  - Matucci-Cerinic, Marco
AU  - Balbir-Gurman, Alexandra
AU  - Midtvedt, Øyvind
AU  - Jordan, Alison C
AU  - Jobanputra, Paresh
AU  - Stevens, Wendy
AU  - Moinzadeh, Pia
AU  - Hall, Frances C
AU  - Agard, Christian
AU  - Anderson, Marina E
AU  - Diot, Elisabeth
AU  - Madhok, Rajan
AU  - Akil, Mohammed
AU  - Buch, Maya H
AU  - Chung, Lorinda
AU  - Damjanov, Nemanja
AU  - Gunawardena, Harsha
AU  - Lanyon, Peter
AU  - Ahmad, Yasmeen
AU  - Chakravarty, Kuntal
AU  - Jacobsen, Søren
AU  - MacGregor, Alexander J
AU  - McHugh, Neil
AU  - Müller-Ladner, Ulf
AU  - Riemekasten, Gabriela
AU  - Becker, Michael
AU  - Roddy, Janet
AU  - Carreira, Patricia E
AU  - Fauchais, Anne Laure
AU  - Hachulla, Eric
AU  - Hamilton, Jennifer
AU  - İnanç, Murat
AU  - McLaren, John S
AU  - van Laar, Jacob M
AU  - Pathare, Sanjay
AU  - Proudman, Susannah
AU  - Rudin, Anna
AU  - Sahhar, Joanne
AU  - Coppere, Brigitte
AU  - Serratrice, Christine
AU  - Sheeran, Tom
AU  - Veale, Douglas J
AU  - Grange, Claire
AU  - Trad, Georges-Selim
AU  - Denton, Christopher P
TI  - Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS)
AID  - 10.1136/annrheumdis-2016-210503
DP  - 2017 Jul 01
TA  - Annals of the Rheumatic Diseases
PG  - 1207--1218
VI  - 76
IP  - 7
4099  - http://ard.bmj.com/content/76/7/1207.short
4100  - http://ard.bmj.com/content/76/7/1207.full
SO  - Ann Rheum Dis2017 Jul 01; 76
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.