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

Herrick, AL, Pan, X, Peytrignet, S, Lunt, M, Hesselstrand, R, Mouthon, L, Silman, A, Brown, E, Czirják, L, Distler, JHW
et al (show 54 more authors) (2017) Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). Annals of the rheumatic diseases, 76 (07). pp. 1207-1218.

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Objectives: The rarity of early diffuse cutaneoussystemic sclerosis (dcSSc) makes randomised controlledtrials very difficult. We aimed to use an observationalapproach to compare effectiveness of currently usedtreatment approaches. Methods: This was a prospective, observational cohortstudy of early dcSSc (within three years of onset of skinthickening). Clinicians selected one of four protocols foreach patient: methotrexate, mycophenolate mofetil(MMF), cyclophosphamide or‘no immunosuppressant’.Patients were assessed three-monthly for up to24 months. The primary outcome was the change inmodified Rodnan skin score (mRSS). Confounding byindication at baseline was accounted for using inverseprobability of treatment (IPT) weights. As a secondaryoutcome, an IPT-weighted Cox model was used to testfor differences in survival. Results: Of 326 patients recruited from 50 centres,65 were prescribed methotrexate, 118 MMF, 87cyclophosphamide and 56 no immunosuppressant.276 (84.7%) patients completed 12 and 234 (71.7%)24 months follow-up (or reached last visit date). Therewere statistically significant reductions in mRSS at12 months in all groups:−4.0 (−5.2 to−2.7) units formethotrexate,−4.1 (−5.3 to−2.9) for MMF,−3.3(−4.9 to−1.7) for cyclophosphamide and−2.2 (−4.0to−0.3) for no immunosuppressant (p value forbetween-group differences=0.346). There were nostatistically significant differences in survival betweenprotocols before (p=0.389) or after weighting(p=0.440), but survival was poorest in the noimmunosuppressant group (84.0%) at 24 months. Conclusions: Thesefindings may support usingimmunosuppressants for early dcSSc but suggest thatoverall benefit is modest over 12 months and that bettertreatments are needed.

Item Type: Article
Uncontrolled Keywords: Humans, Scleroderma, Diffuse, Mycophenolic Acid, Cyclophosphamide, Methotrexate, DNA Topoisomerases, Type I, RNA Polymerase III, Nuclear Proteins, Immunosuppressive Agents, Autoantibodies, Antibodies, Antinuclear, Treatment Outcome, Severity of Illness Index, Survival Rate, Cohort Studies, Prospective Studies, Adult, Middle Aged, Europe, Female, Male, Early Medical Intervention
Depositing User: Symplectic Admin
Date Deposited: 02 Jun 2017 10:21
Last Modified: 19 Jan 2023 07:03
DOI: 10.1136/annrheumdis-2016-210503
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