Upfront Ruxolitinib Treatment for Chronic Graft-vs-host Disease in Children and Young Adults: A Corticosteroid-sparing Pilot Study
While hematopoietic stem cell transplant (HSCT) is an effective therapy, graft versus host disease (GVHD) is the most significant complication after HSCT. Corticosteroids (or steroids) have been the mainstay of treatment for chronic GVHD for many decades now. Increasingly, newer immunosuppressive and immunomodulating agents are being studied in adults and children affected by cGVHD. Ruxolitinib is one of these promising newer agents, which has been shown to be effective in the treatment of cGVHD in both children and adults. Currently, ruxolitinib is generally added to a patient's treatment regimen after (or with) a course of high dose steroids. The purpose of this study is to examine the effectiveness of upfront single agent ruxolitinib for cGVHD.
• Newly diagnosed moderate to severe chronic graft versus host disease (as defined by NIH cGVHD consensus criteria), requiring systemic treatment
• Patient aged ≥12 year-old and ≤30 year-old
• Patient able to take oral or enteral medication
• No active, clinically significant uncontrolled infections
• ALT ≤ 5x upper limit of normal (ULN) and total bilirubin ≤ 5xULN (unless presumed liver GVHD)
• Platelet ≥ 20k and ANC ≥ 500. The use of transfusions or growth factors is permitted to maintain counts at these thresholds
• No prior systemic treatment for chronic GVHD. Patients previously treated for acute GVHD are eligible, including those who received ruxolitinib for treatment of their aGVHD
• Patients must be on ≤ physiologic dosing (i.e. hydrocortisone 8-12mg/m2/day) at enrollment
• Patients with prior acute GVHD on \< 1 mg/kg steroids with new onset moderate-severe chronic GVHD may be considered for enrollment if they can taper steroids to reach physiological hydrocortisone in 1 month. If unable to do so these patients will come off study and be replaced