Multinational phase 2 trial of ruxolitinib for treatment-naïve or corticosteroid-refractory chronic graft-versus-host disease in pediatric patients

The phase 2 REACH5 study, at 21 centers across 14 countries, evaluated ruxolitinib for patients aged 2 tot 18 years with treatment-naïve or corticosteroid-refractory moderate-to-severe chronic graft-versus-host disease (cGVHD). Interim analysis of the study found that objective response rate at day 1 of cycle 7 was 40% (90% CI 27.7 to 53.3), with responses seen in 41% of treatment-naïve patients and 39% of corticosteroid-refractory patients. The most common grade 3 or worse treatment-related adverse events were neutropenia (18% of patients) and thrombocytopenia (13%). Grade 3 or worse serious treatment-related adverse events were seen in 16%. Seven percent of patients died while on-treatment, none of these deaths were considered to be related to ruxolitinib. The authors conclude that this interim analysis suggests that ruxolitinib is active and well tolerated in pediatric patients with treatment-naïve or corticosteroid-refractory cGVHD.