Ruxolitinib for pediatric acute and chronic graft-versus-host disease: a single-center retrospective study of efficacy and safety

Graft-versus-host disease (GVHD) is a major concern for patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Ruxolitinib has been proven effective in treating adult steroid-refractory GVHD; however, studies on pediatric patients are relatively scarce. Thus, this single-center study evaluated the efficacy and safety of ruxolitinib in pediatric patients with steroid-refractory GVHD. We retrospectively reviewed the data of patients aged < 18 years who underwent allogeneic HSCT and received ruxolitinib treatment for either acute GVHD (aGVHD) or chronic GVHD (cGVHD) between 2018 and 2023. Data on the clinical response, concomitant and subsequent medications, adverse events, and outcomes were obtained through medical chart review. Sixteen patients were analyzed in this study: seven with aGVHD and nine with cGVHD. The overall response rate for the 16 patients was 81% (aGVHD, 86%; cGVHD, 77%). The overall survival rate was 56%: (aGVHD, 57%; cGVHD, 55%). For 11 patients with at least stable disease, steroid dosage could be reduced by at least 75%; however, corticosteroids were successfully tapered off in only six patients at the last follow-up. Among four patients with documented lung cGVHD, none experienced lung cGVHD progression at 1-year follow-up. Further, 50% of the patients experienced grade 3 or 4 neutropenia and/or thrombocytopenia, and 56% had viral reactivation. Two patients discontinued ruxolitinib owing to adverse events. Ruxolitinib treatment for pediatric patients with aGVHD and cGVHD is associated with a high overall response rate, significant steroid-sparing effect, acceptable toxicity, and manageable adverse events. However, blood count and viral reactivation should be closely monitored during ruxolitinib use.