Dr. Wang on the Potential Utility of Ruxolitinib in Pediatric Patients With cGVHD

YunZu Michele Wang, MD, member, Division of Bone Marrow Transplantation and Immune Deficiency, Cincinnati Children’s Hospital Medical Center, instructor, University of Cincinnati Department of Pediatrics, discusses the potential utility of ruxolitinib (Jakafi) in children and young adult patients with chronic graft-versus-host disease (cGVHD).

Findings from a retrospective study, which were presented in a virtual poster during the 2021 Transplantation and Cellular Therapy Meetings, showed that ruxolitinib led to an overall response rate of 60% in 20 patients with chronic GVHD. Patients had a median age of 14.6 years. 

Two patients with moderate cGVHD achieved a complete response (CR), and 10 patients achieved a partial response. Of the 2 patients who achieved a CR, 1 has weaned off ruxolitinib and maintained their response; the other patient is weaning off the drug and appears to be maintaining their response, Wang explains.

Additionally, patients’ responses occurred in a range of organs affected by cGVHD, including the skin, joints, gastrointestinal tract, liver, and muscles. Notably, all 7 patients with sclerotic skin cGVHD responded to ruxolitinib. Finally, 2 of 3 patients with lung involvement achieved stabilization of their pulmonary function tests, concludes Wang.