Using ruxolitinib as salvage therapy in pediatric allogeneic hematopoietic stem cell transplant (HSCT) patients with steroid-refractory acute graft versus host disease (GVHD) resulted in fair overall response in a small study involving 13 children.

Participants received ruxolitinib orally at either 5 or 2.5 mg twice daily depending on their weight. Among the results:

• At 4 weeks, 1 patient had complete, 4 had partial, and 2 had no response.
• Treatment was unsuccessful due to adverse events in 4 patients.
• Overall response rate was 45%.
• 7 patients experienced grade 3-4 elevated alanine transaminase, 5 had grades 3-4 neutropenia, and 3 had grade 4 thrombocytopenia.
• The most common infectious complication was bacterial infection, occurring in 6 patients.
• 7 patients were alive at a median follow-up of 401 days post-HSCT.