Real-world Canadian data on belumosudil therapy in heavily pretreated patients with steroid-refractory chronic graft-versus-host disease: treatment outcomes and risk factor analysis for failure-free survival.

This multicenter real-world study evaluated belumosudil (BEL), a selective ROCK2 inhibitor, in 46 heavily pretreated patients with refractory chronic graft-versus-host disease (cGvHD) under a Canadian compassionate use program.

With a median follow-up of 11.4 months:

• The overall response rate was 52%.
• 12-month failure-free survival (FFS) was 64.3%.
• 12-month overall survival (OS) was 91.1%.
• 73% of patients discontinued corticosteroids by 12 months.

Combination therapy of BEL with ruxolitinib showed similar efficacy to BEL monotherapy, even though it was used in patients with more advanced disease.

A prognostic model using prior acute GvHD and involvement of ≥4 organs effectively stratified FFS outcomes.

BEL demonstrated durable efficacy, an acceptable safety profile, and a high likelihood of steroid discontinuation. Risk stratification may aid personalized treatment decisions.