3 years ago

Belimumab for the Treatment of Early Diffuse Systemic Sclerosis: Results of a Randomized, Double-Blind, Placebo-Controlled, Pilot Trial

Jackie Szymonifka, Elana J. Bernstein, Jennifer M. Franks, Horatio F. Wildman, Robert F. Spiera, Tammara A. Wood, Cynthia Magro, Michael L. Whitfield, Jessica K. Gordon, Viktor Martyanov
Objective To assess the safety and efficacy of treatment with belimumab in patients with early diffuse cutaneous systemic sclerosis (dcSSc) treated with background mycophenolate mofetil (MMF). Methods In this 52-week, investigator-initiated, single-center, double-blind, placebo-controlled, pilot study, 20 patients recently started on MMF were randomized 1:1 to additionally receive belimumab 10 mg/kg intravenously or placebo. We assessed safety, efficacy and differential gene expression (NCT01670565). Results In the belimumab group, the median [interquartile range] modified Rodnan skin score (MRSS) decreased from 27 [26.5, 31] to 18 [11, 23], p=0.039. In the placebo group, the median MRSS decreased from 28 [22, 28] to 21 [14, 25], p=0.023. The median change in MRSS was -10 [-13,-9] in the belimumab group and -3.0 [-15,-1] in the placebo group, p=0.411. There were no significant differences in number of adverse events between the groups. Significant decrease in expression of B-cell signaling and pro-fibrotic genes and pathways was observed in patients with improved MRSS in the belimumab group but not in the placebo group. Conclusion Patients in both treatment groups experienced significant improvements in MRSS. The median difference was greater in the belimumab group but did not achieve statistical significance in this small pilot study. Adverse events were similar between the groups. Changes in gene expression were consistent with mechanism of action and show that clinical response to treatment with belimumab is associated with significant decrease in pro-fibrotic genes and pathways. Additional studies are needed to determine the role of belimumab in the treatment of dcSSc. This article is protected by copyright. All rights reserved.

Publisher URL: http://onlinelibrary.wiley.com/resolve/doi

DOI: 10.1002/art.40358

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