Belumosudil in adult cGvHD: A systematic review and meta-analysis

A systematic review and meta-analysis evaluating the efficacy and safety of belumosudil in adults with chronic graft-versus-host disease (cGvHD; N = 651) was published in Transplantation and Cellular Therapy by Rathje et al. The analysis included 16 prospective and retrospective studies. The primary endpoint was overall response rate (ORR), including in prespecified organ categories; secondary outcomes included partial response (PR), complete response (CR), time to response (TTR), failure-free survival (FFS), overall survival (OS), and adverse events (AEs).

Key data: The pooled ORR was 60% (95% confidence interval [CI], 53–67; I² = 62%). Organ-specific responses were highest in gastrointestinal (52%; 95% CI, 40–65) and joint/fascia cGvHD (52%; 95% CI, 37–66), and lowest in lung cGvHD (25%; 95% CI, 19–33). Pooled PR and CR rates were 54% (95% CI, 46–61) and 7% (95% CI, 4–10), respectively; pooled TTR was 8.94 weeks (95% CI, 5.75–13.88). Pooled FFS and OS rates were 57% (95% CI, 46–67) and 87% (95% CI, 84–90), respectively. In meta-regression analyses, prior ruxolitinib exposure was associated with lower ORR (p = 0.0033) and accounted for 65% of the between-study heterogeneity in ORR estimates. Grade ≥3 AEs occurred in 29–61% of patients. 

Key learning: Belumosudil demonstrates promising efficacy and a manageable safety profile in cGvHD, supporting further evaluation in earlier treatment lines and combination strategies.