Ruxolitinib as front-line therapy in graft versus host disease: Efficacy and safety in children

Abstract

Background: Ruxolinitib has been used as an effective salvage agent in steroid refractory acute and chronic graft versus host disease (GVHD). We aimed to analyze data on ruxolitinib as front-line therapy in children. Patients and methods: We included children up to 18 years of age who underwent hematopoietic stem cell transplantation (HSCT), from December 2017 to April 2020, where ruxolitinib was administered in children up to 18 years of age, at 48 hours of diagnosis with acute and within two weeks in chronic GVHD. The dose was 2.5 mg and 5 mg twice daily for children weighing 15 kg respectively. Results: A total of 80 out of 335 transplanted children were included. The indication was acute GVHD (aGVHD) in 41 and chronic GVHD (cGVHD) in 39 children. Response rates were 90% in aGVHD, with 92% in skin and 46% in acute gut GVHD. Overall response rate in cGVHD was 74.3%, with 80%, 67% and 100% in skin, lung and liver respectively. Thrombocytopenia was the predominant drug related side effect, noted in 65% children. Three children needed drug withdrawal, two due to uncontrolled hyponatremia in aGVHD, and one due to nephrotic syndrome in the cGVHD setting. The 100-day mortality in the historical cohort (January 2014 to October 2017) was 29/279 (10.4%) versus 17/335 (5%) in the study cohort (p = 0.029). Conclusion: Ruxolitinib used early in acute and chronic GVHD results in excellent response rates and is safe to use in children.

Keywords

• Ruxolitinib
• Children
• Graft versus host disease
• Mortality
• Infections
• Transplant-related mortality