FDA Approval Summary: Ruxolitinib for Treatment of Steroid-Refractory Acute Graft-Versus-Host Disease.

Abstract

On May 24, 2019, the Food and Drug Administration approved ruxolitinib for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in adult and pediatric patients 12 years and older. Approval was based on Study INCB 18424-271 (REACH-1; NCT02953678), an open-label, single-arm, multicenter trial that included 49 patients with grades 2-4 SR-aGVHD occurring after allogeneic hematopoietic stem cell transplantation. Ruxolitinib was administered at 5 mg twice daily, with dose increases to 10 mg twice daily permitted after 3 days in the absence of toxicity. The Day-28 overall response rate was 57.1% (95% confidence interval [CI]: 42.2-71.2). The median duration of response was 0.5 months (95% CI: 0.3-2.7), and the median time from Day-28 response to either death or need for new therapy for acute GVHD was 5.7 months (95% CI: 2.2 to not estimable). Common adverse reactions included anemia, thrombocytopenia, neutropenia, infections, edema, bleeding, and elevated transaminases. Ruxolitinib is the first drug approved for treatment of SR-aGVHD. IMPLICATIONS FOR PRACTICE: Ruxolitinib is the first Food and Drug Administration-approved treatment for steroid-refractory acute graft-versus-host disease in adult and pediatric patients 12 years and older. Its approval provides a treatment option for the 60% of those patients who do not respond to steroid therapy.

• Authors: Donna Przepiorka, Lola Luo, Sriram Subramaniam, Junshan Qiu, Ramadevi Gudi, Lea C Cunningham, Lei Nie, Ruby Leong, Lian Ma, Christopher Sheth, Albert Deisseroth, Kirsten B Goldberg, Gideon M Blumenthal, Richard Pazdur
• Journal: The oncologist (Oncologist) Vol. 25 Issue 2 Pg. e328-e334 (02 2020) ISSN: 1549-490X [Electronic] England
• PMID: 32043777 (Publication Type: Journal Article, Multicenter Study)
• Copyright: Published 2019. This article is a U.S. Government work and is in the public domain in the USA.

Chemical References

• Nitriles
• Pyrazoles
• Pyrimidines
• Steroids
• ruxolitinib

Topics

• Adult
• Child
• Graft vs Host Disease (drug therapy)
• Hematopoietic Stem Cell Transplantation (adverse effects)
• Humans
• Nitriles
• Pyrazoles (adverse effects)
• Pyrimidines
• Steroids (therapeutic use)