Systemic treatment with the ROCK2 inhibitor belumosudil in ocular chronic graft versus host disease

To evaluate the efficacy of oral belumosudil on ocular surface disease in patients with steroid-refractory chronic graft-versus-host disease (SR-cGVHD).

Retrospective case series.

In this retrospective study, we analyzed patients with SR-cGVHD and ocular involvement (oGVHD) treated with belumosudil (200 mg daily). Ocular assessments at baseline and 6 months included the International Chronic Ocular GVHD Consensus Group severity score, 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). Treatment response rates were assessed per NIH consensus criteria.

Among 78 treated patients, 40 had oGVHD; 28 completed both assessments. The cohort (median baseline NIH eye score: 2; oGVHD severity score: 9) had high-risk cGVHD features: 50.0% had severe systemic cGVHD and 82.1% had conjunctival fibrosis. At 6 months, 53.6% of patients showed improvement in the oGVHD severity score and 64.8% reported symptomatic improvement (OSDI). Objective signs significantly improved: median CFS decreased from 4 to 3, TBUT increased from 1.00s to 2.00s, and Schirmer's test improved from 1.0 mm to 2.0 mm. A greater ocular benefit was observed in the 43.6% of patients who achieved systemic overall response, with 66.7% showing improved oGVHD severity scores and 80.0% reporting better OSDI. Visual acuity remained stable, and 42.9% of patients achieved corticosteroid dose reduction. Treatment was well-tolerated.

Systemic belumosudil was associated with improvements in ocular surface parameters and symptoms in steroid-refractory oGVHD, with favorable tolerability. Ocular improvement was closely linked to systemic response, warranting prospective validation.