Corticosteroids are used as first-line treatment for acute GVHD. However, they are effective in only about half of patients. This study prospectively evaluated Ruxolitinib combined with 1 mg/kg methylprednisolone in the initial treatment of acute GVHD. A total of 32 patients were enrolled. Acute GVHD involved skin (53.1%), gastrointestinal tract (68.8%) and liver (6.0%). The complete response rate at day
28 was 96.9%. The 1-year and 2-year cumulative incidence rates of chronic GVHD were 9.4% and 13.8%, respectively. The 1- year cumulative incidence of non-relapse mortality was 8.7%. The Kaplan-Meier curve estimated 1-year overall survival after transplantation at 73.4%. This prospective study suggested that patients with acute GVHD showed a high response rate to Ruxolitinib (5 mg daily) combined with 1 mg/kg/day methylprednisolone. The novel regimen spared steroid exposure, alleviated toxicity and resulted in long-term survival.