GVHD
Published on September 17, 2024
Phase I/II study of ruxolitinib in children with aGVHD
by Blood
Locatelli F, Kang HJ, Bruno B, et al. Ruxolitinib for Pediatric Patients With Treatment-Naïve and Steroid-Refractory Acute Graft-Versus-Host Disease (REACH4). Blood. 2024; (doi: 10.1182/blood.2023022565).
Findings from the REACH4 clinical trial demonstrate the safety and efficacy of ruxolitinib in pediatric cases of treatment-naïve or steroid-refractory acute graft-versus-host disease (aGVHD). The Phase I/II open-label study included 45 patients with aGVHD, all between the ages of 2-18 years, who received the study drug for a median 3.8 months. Dosing was set at 4 mg/m2 twice daily for children aged ≥2 to <6 years and at 5 mg twice daily for children aged ≥6 to <12 years, while youths aged ≥12 to <18 years received the same dosage as adults: 10 mg twice a day. At day 28 follow-up, the overall response rate (ORR) across the sample was 84.4%, with a durable ORR of 66.7% at day 56. Favorable response rates were observed across age groups and in both the treatment-naïve and steroid-refractory sub-populations. No new safety concerns were identified, as documented adverse events — including anemia and lower neutrophil and leukocyte counts — were in line with expectations.
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