Additional or replacement treatment with ruxolitinib for acute or chronic graft-vs-host disease (GVHD) resulted in a high overall response rate among a group of pediatric patients, according to the results of a study published in Drug Design, Development and Therapy.

Ruxolitinib is a selective nonreceptor Janus kinase (JAK) 1/2 inhibitor that has been shown to have activity in adults with steroid-refractory GVHD. This retrospective study analyzed the clinical efficacy of ruxolitinib in 53 pediatric patients with refractory GVHD that responded poorly to steroid and second-line treatment.

Drug dosing was 2.5 mg for patients with a bodyweight less than 25 kg and 5 mg for those with a body weight greater than 25 kg. The dose of ruxolitinib could be increased to a maximum of 10 mg twice daily.

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More than three-quarters of patients (75.5%) responded to treatment. For 17 patients with acute GVHD, the overall response rate was 64.7% with 5 complete responses, 6 partial responses, and 6 treatment failures. For the 36 patients with chronic GVHD, the overall response rate was greater at 80.6% with 10 complete responses and 19 partial responses.

Response rates did not significantly differ among patients with different primary diseases, the researchers noted. Only 1.9% of patients experienced a relapse of the primary tumor.

Treatment with steroids was able to be discontinued (39%) or reduced (22.2%) among the study participants.

Adverse events were more common in patients with acute GVHD compared with those with chronic GVHD (94.1% vs 30.6%). Additionally, patients with chronic GVHD had superior prognosis compared with those with acute GVHD after treatment with ruxolitinib. 

“Ruxolitinib may play a key role in treating refractory GVHD, especially chronic GVHD in children following HSCT treatment,” the researchers wrote. “The treatment can reduce the need to use steroids and other immunosuppressive agents for controllable adverse events.”


Yang W, Zhu G, Qin M, et al. The effectiveness of ruxolitinib for acute/chronic graft-versus-host disease in children: a retrospective study. Drug Design, Development and Therapy. 2021;15:743-752. doi:10.2147/DDDT.S287218