Journal article
Authors list: Herrick, Ariane L.; Pan, Xiaoyan; Peytrignet, Sebastien; Lunt, Mark; Hesselstrand, Roger; Mouthon, Luc; Silman, Alan; Brown, Edith; Czirjak, Laszlo; Distler, Joerg H. W.; Distler, Oliver; Fligelstone, Kim; Gregory, William J.; Ochiel, Rachel; Vonk, Madelon; Ancuta, Codrina; Ong, Voon H.; Farge, Dominique; Hudson, Marie; Matucci-Cerinic, Marco; Balbir-Gurman, Alexandra; Midtvedt, Oyvind; Jordan, Alison C.; Jobanputra, Paresh; Stevens, Wendy; Moinzadeh, Pia; Hall, Frances C.; Agard, Christian; Anderson, Marina E.; Diot, Elisabeth; Madhok, Rajan; Akil, Mohammed; Buch, Maya H.; Chung, Lorinda; Damjanov, Nemanja; Gunawardena, Harsha; Lanyon, Peter; Ahmad, Yasmeen; Chakravarty, Kuntal; Jacobsen, Soren; MacGregor, Alexander J.; McHugh, Neil; Mueller-Ladner, Ulf; Riemekasten, Gabriela; Becker, Michael; Roddy, Janet; Carreira, Patricia E.; Fauchais, Anne Laure; Hachulla, Eric; Hamilton, Jennifer; Inanc, Murat; McLaren, John S.; van Laar, Jacob M.; Pathare, Sanjay; Proudman, Susannah; Rudin, Anna; Sahhar, Joanne; Coppere, Brigitte; Serratrice, Christine; Sheeran, Tom; Veale, Douglas J.; Grange, Claire; Trad, Georges-Selim; Denton, Christopher P.
Publication year: 2017
Pages: 1207-1218
Journal: Annals of the Rheumatic Diseases
Volume number: 76
Issue number: 7
ISSN: 0003-4967
eISSN: 1468-2060
DOI Link: https://doi.org/10.1136/annrheumdis-2016-210503
Publisher: Elsevier
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.
Abstract:
Citation Styles
Harvard Citation style: Herrick, A., Pan, X., Peytrignet, S., Lunt, M., Hesselstrand, R., Mouthon, L., et al. (2017) Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS), Annals of the Rheumatic Diseases, 76(7), pp. 1207-1218. https://doi.org/10.1136/annrheumdis-2016-210503
APA Citation style: Herrick, A., Pan, X., Peytrignet, S., Lunt, M., Hesselstrand, R., Mouthon, L., Silman, A., Brown, E., Czirjak, L., Distler, J., Distler, O., Fligelstone, K., Gregory, W., Ochiel, R., Vonk, M., Ancuta, C., Ong, V., Farge, D., Hudson, M., ...Denton, C. (2017). Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS). Annals of the Rheumatic Diseases. 76(7), 1207-1218. https://doi.org/10.1136/annrheumdis-2016-210503
Keywords
CYCLOPHOSPHAMIDE; HEALTH-ASSESSMENT QUESTIONNAIRE; PLACEBO; PROPENSITY SCORE; SKIN THICKNESS SCORE; TRIALS
