Patients who develop glucocorticoid-refractory acute graft-versus-host disease (aGVHD) after allogeneic stem cell transplantation tend to have poor outcomes. Now, a new study published in The New England Journal of Medicine has found that ruxolitinib (Jakafi®) therapy may be associated with significantly higher overall response rates (ORRs) than standard therapy. Researchers conducted a phase 3 study with this selective Janus kinase (JAK1 and JAK2) inhibitor in patients with aGVHD and found significantly greater ORRs with ruxolitinib (62%) compared with best available therapy (BAT; 39%). 


Ruxolitinib was approved by the US Food and Drug Administration (FDA) in 2019 for the treatment of steroid-refractory aGVHD in adult and pediatric patients aged 12 years or older. Researchers conducted a multicenter, randomized, open-label, phase 3 trial with 309 patients to compare the efficacy and safety of oral ruxolitinib (10 mg twice daily) with the investigator’s choice of therapy from a list of 9 commonly used options (control group). All the patients were age 12 or older and had developed glucocorticoid-refractory aGVHD after allogeneic stem cell transplantation (Safety and Efficacy of Ruxolitinib Versus Best Available Therapy in Patients With Corticosteroid-refractory Acute Graft vs Host Disease After Allogeneic Stem Cell Transplantation [REACH2]; Number: NCT02913261).

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For this investigation, 154 patients were assigned to ruxolitinib and 155 were assigned to the control group. The primary endpoint was overall response (OR), which was defined as a complete response or partial response at day 28. Secondary endpoints included durable ORR at day 14, ORR at day 56, duration of response, overall survival (OS), and event-free survival.

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All the patients were treated between April 12, 2017, and May 30, 2019, at 105 treatment centers in 22 countries. Median age was 54 years (range, 12 to 73), 59% were male, and 9 were adolescents. In this study, 34% of patients (104) had grade 2 aGVHD, 44% (136) had grade 3 disease, and 20% (62) had grade 4 disease.  The researchers evenly distributed patients according to aGVHD grade in the 2 treatment groups.