Brentuximab vedotin for skin involvement in refractory diffuse cutaneous systemic sclerosis, an open-label trial.

Autor:	Fernández-Codina A; Division of Rheumatology, Western University, London, Ontario, Canada.; Division of General Internal Medicine-Windsor Campus, Western University, London, Ontario, Canada.; Division of Systemic Autoimmune Diseases and Critical Care, Hospital Clinic de Barcelona, Barcelona, Spain., Nevskaya T; Division of Rheumatology, Western University, London, Ontario, Canada., Baron M; Division of Rheumatology, Jewish General Hospital, McGill University, Montreal, Quebec, Canada., Thomas Appleton C; Division of Rheumatology, Western University, London, Ontario, Canada., Cecchini MJ; Division of Pathology, Western University, London, Ontario, Canada., Philip A; Division of Rheumatology, Western University, London, Ontario, Canada., El-Shimy M; Division of Rheumatology, Western University, London, Ontario, Canada., Vanderhoek L; Division of Rheumatology, Western University, London, Ontario, Canada., Pinal-Fernández I; Muscle Unit, National Institute of Arthritis and Musculoskeletal and Skin Diseases, Bethesda, Maryland, USA., Pope JE; Division of Rheumatology, Western University, London, Ontario, Canada.
Jazyk:	angličtina
Zdroj:	Rheumatology (Oxford, England) [Rheumatology (Oxford)] 2024 Apr 23. Date of Electronic Publication: 2024 Apr 23.
DOI:	10.1093/rheumatology/keae235
Abstrakt:	Objective: We explored the efficacy and safety of brentuximab vedotin, a chimeric anti-CD30 antibody drug conjugate, in patients with severe active diffuse cutaneous systemic sclerosis (dcSSc). Methods: This phase II proof-of-concept, single center, open-label, single arm, investigator-initiated trial included patients ≥18 years, with dcSSc, modified Rodnan skin score (mRSS) ≥15 with <5 years since the first non-Raynaud's symptom and/or skin worsening despite immunosuppression who were treated with intravenous brentuximab vedotin 0.6 mg/Kg q3 weeks for 45 weeks. The primary end point was a decrease in mRSS of ≥ 8 points at 48 weeks. Results: Eleven patients were treated with brentuximab vedotin, with 9 completing the study. The mean mRSS reduction at week 48 was 11.3 (95% CI 6.9, 15.8; p= 0.001), meeting the primary end point in the intention to treat analysis (7/11 had a decrease in mRSS ≥8). The % forced vital capacity increased by 7.8% (12.5). The Composite Response Index in dcSSc (CRISS) suggested a beneficial treatment effect (86% ≥0.6). Most adverse events were mild. No SAEs were attributed to brentuximab vedotin. Conclusion: In dcSSc, brentuximab vedotin improved skin and FVC; without safety concerns. A placebo-controlled trial is warranted to corroborate these initial findings. (© The Author(s) 2024. Published by Oxford University Press on behalf of the British Society for Rheumatology.)
Databáze:	MEDLINE
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