Science Highlights
Published on October 14, 2025
Belumosudil vs. best available therapy for chronic GVHD
by Blood Advances
Hall K, Lazaryan A, van Der Laan M, et al. Efficacy and Safety of Belumosudil as Compared With Best Available Therapy for the Treatment of cGVHD in the US. Blood Advances. 2025; (doi: 10.1182/bloodadvances.2025015832).
A real-world study affirmed the safety and efficacy of belumosudil in patients with chronic graft-versus-host disease (cGVHD) while also comparing its performance relative to best available therapy (BAT). While U.S. Food and Drug Administration approval hinged on a Phase II trial that evaluated two different doses of the drug, the new investigation examined outcomes with belumosudil versus BAT in patients with relapsed/refractory cGVHD after multiple previous lines of therapy. The research was informed by retrospective data on 196 patients who contributed 113 belumosudil and 245 BAT lines of therapy between March 2015 and 2024. The primary endpoint was the 6-month overall response rate, calculated at 38.7% with belumosudil and 26.8% with BAT. The 44.2% improvement with belumosudil, coupled with its lower incidence of adverse events, aligns with the medication's known safety and efficacy profile and underscores its clinical value versus BAT in cGVHD patients with 2-5 prior failed lines of therapy.
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