Combined methotrexate and short-term superpotent topical corticosteroids compared with prolonged superpotent topical corticosteroids alone in bullous pemphigoid: a multicentre, noninferiority randomised clinical trial

Dereure, Olivier; Bernard, Philippe; Chosidow, Olivier; Ingen-Housz-Oro, Saskia; Machet, Laurent; Duvert-Lehembre, Sophie; Maillard, Hervé; Abasq, Claire; Sassolas, Bruno; Acquitter, Marie; Konstantinou, Maria Polina;

Combined methotrexate and short-term superpotent topical corticosteroids compared with prolonged superpotent topical corticosteroids alone in bullous pemphigoid: a multicentre, noninferiority randomised clinical trial

Abstract

Abstract Background Superpotent topical corticosteroids (STS) are highly effective in bullous pemphigoid (BP), but prolonged use is associated with practical limitations and a risk of relapse. Objective To evaluate whether combining short-term STS with methotrexate (MTX) could maintain efficacy, reduce relapses, and preserve safety compared with prolonged STS alone. Methods In this multicentre, randomised, open-label, non-inferiority trial, adults with BP were assigned to receive either STS (clobetasol propionate) for one month plus low-dose MTX as maintenance (MTX + STS arm), or prolonged STS alone (STS arm). The primary endpoint was 9-month overall survival (OS), with a predefined non-inferiority margin of 15%. Secondary outcomes included relapse-free survival (RFS) and severe adverse events (SAE). Results Among 266 randomised patients (mean age 80.8 years), 9-month OS was 86.5% in the MTX + STS arm and 83.5% in the STS arm (p=0.498; hazard ratio [HR] 0.80, 95% CI 0.40–1.50). The lower limit of the 90% confidence interval for the difference in survival rates (−4.6%) remained within the predefined non-inferiority margin, meeting the primary endpoint. Among the 87.4% of patients who achieved disease control within 28 days, relapse occurred in 31 patients in the MTX + STS arm and 52 in the STS arm, corresponding to a 9-month RFS of 68.4% (95% CI 59.3–78.8) versus 47.4% (95% CI 37.7–59.5), respectively (p=0.042). SAE were more frequent in the MTX + STS arm than in the STS arm (mean number per patient per month 0.40 [95% CI 0.23–0.57] vs 0.19 [95% CI 0.01–0.36], p=0.005). Limitations The study was open-label, did not include a validated disease activity score and was conducted in a selected BP population managed under clinical trial conditions. Conclusion Maintenance therapy with low-dose MTX is non-inferior to prolonged STS in terms of survival and is associated with a reduced risk of relapse, at the cost of increased toxicity

Meet DeepDyve

Get unlimited, instant access to over 150 million full-text scientific articles for less than the price of buying a single PDF.

150M+Research Papers

35M+Full-Text Streaming Papers

25M+Open Access Papers

$2/dayStarting Price

DeepDyve Collection

Access top-tier journals without the top-tier price. We partner directly with the world’s leading academic publishers to bring you legally compliant, instant access. Read the latest findings from Springer, Wiley, Oxford University Press, and more—all in one subscription.

Browse our collection of titles from leading publishers

AI Research Assistant

Accelerate your research with our advanced AI tools. Chat directly with your uploaded papers, and DeepDyve’s 50M+ LitStream and OA collections of full-text papers. AI responses come directly from the literature, footnoted and sourced, to provide the most authoritative and verifiable responses. AI outputs powered by OpenAI and Anthropic Claude. AI Smart Folders (coming soon)

Stop wasting time on paywalls, pirated sites and scattered journal portals

Streamline your research workflow with DeepDyve's comprehensive literature management solution for less than the price of buying a single PDF.

Try 1 month for $0