Real-world outcomes of belumosudil in pediatric and young adult patients with cGvHD

Results from a multi-institutional real-world study, evaluating belumosudil for the treatment of pediatric, adolescent, and young adult patients with chronic graft-versus-host disease (cGvHD; N = 23) after multiple prior therapies, were published in Bone Marrow Transplantation by Ibrahimova et al. Response rates, durability, and safety were evaluated.

Key data: With a median treatment duration of 173 days (range, 38–1,131), 48% of patients had a partial response (PR) to belumosudil at any time. Organ-specific complete responses (CRs) included liver (50%), oral (36%), skin (31%), and joints/fascia (37%). All belumosudil responders who were receiving concurrent corticosteroids (n = 6) tapered their steroid doses. At a median last follow-up of 1,154 days (range, 173–4,737) from transplant, 83% of patients were alive and overall failure-free survival (FFS) was 74%. Infectious complications were experienced by 39% of patients.

Key learning: Belumosudil demonstrated efficacy and was well tolerated in pediatric and young adult patients with cGvHD. Larger prospective trials are needed to inform earlier use of belumosudil for the treatment of cGvHD.