Real-world outcomes of belumosudil for treatment-refractory cGvHD: Italian experience

Results from a retrospective, multicenter study, evaluating real-world outcomes of belumosudil within an Italian compassionate use program, were published in Haematologica by Bonifazi et al.  The study included patients with steroid-refractory or steroid-dependent moderate/severe chronic graft-versus-host disease (cGvHD) who had received ≥2 prior lines of systemic therapy (N = 80). Clinical response was assessed by the treating physician at 3, 6, and 12 months following belumosudil initiation. 

Key data: The best overall response (BOR) rate was 62.5% (partial response [PR], 61.2%; complete response [CR], 1.3%), with overall response rates (ORRs) of 52.6%, 57.6%, and 55.0% at 3, 6, and 12 months, respectively. The 12‑month duration of response (DoR) was 83.4% (95% confidence interval [CI], 72.8–95.5) and median time to response (TTR) was 3 months (95% CI, 2.69–3.09). Median failure-free survival (FFS) was 18.07 months (95% CI, 12.71–not available [NA]). Improvements in patient-reported outcomes (PROs) (≥2‑point reduction on the National Institutes of Health [NIH] Severity Index Score [SIS]) were reported in 33%, 36%, and 29% of patients at 3, 6, and 12 months, respectively. Belumosudil was well tolerated, with adverse event (AE)-related discontinuation in 11% of patients.

Key learning: This real-world Italian experience confirms the durable efficacy and favorable safety profile of belumosudil in patients with moderate/severe treatment-refractory cGvHD.