Belumosudil for ocular SR-cGvHD: A retrospective case series

Results from a retrospective case series evaluating belumosudil (200 mg daily) in 28 patients with ocular steroid-refractory chronic graft-versus-host disease (SR-cGvHD) were published in the American Journal of Ophthalmology by Wu et al. Ocular assessments at baseline and 6 months included the International Chronic Ocular GvHD Consensus Group severity score, National Institutes of Health (NIH) eye score (0–3), corneal fluorescein staining (CFS, 0–4), tear break-up time (TBUT), Schirmer's I test, and the Ocular Surface Disease Index (OSDI). 

Key data: At 6 months, 53.6% of patients showed improvement in the ocular GvHD severity score. The NIH eye score remained unchanged in most patients (baseline, 2 [1–2]; 6 months, 2 [1–3]). Objective ocular surface parameters also improved: CFS grade decreased from 4 to 3, TBUT increased from 1.0 s to 2.0 s, and Schirmer's test result improved from 1.0 mm to 2.0 mm. OSDI improved in 64.3% of patients. Greater ocular benefit was observed among the 53.6% of patients who achieved a systemic overall response rate (ORR) at 6 months, with 66.7% showing improved ocular GvHD severity scores and 80.0% reporting improvement in OSDI. Corticosteroid dose reduction was observed in 42.9% of patients. Treatment was well tolerated, with no drug-related discontinuations or severe hematologic toxicities. 

Key learning: Systemic belumosudil demonstrated encouraging efficacy in patients with ocular SR-cGvHD, with improvements in ocular GvHD severity scores and ocular surface parameters in over half of patients, alongside favorable tolerability.