The role of ruxolitinib in the management of acute GVHD

Abstract

Background and objectives: Following an allogeneic hematopoietic stem cell transplant (allo-HSCT), a primary cause of morbidity and mortality is still steroid-refractory acute graft-versus-host disease (SR-aGVHD). Recently, ruxolitinib, an oral inhibitor of JAK1 and JAK2, was approved for use in individuals suffering from SR-aGVHD. This study aimed to analyze the efficacy and toxicity of ruxolitinib in the real world.

Material and methods: In the present study, we investigated the effectiveness and toxicity of ruxolitinib in patients with SR-aGVHD using a multicenter retrospective analysis. We enrolled 23 patients between 2018 and 2024 who received ruxolitinib treatment for SR-aGVHD.

Results: The first response was acheived in a median of 28 days (range, 12-150). The overall response rate (ORR) for ruxolitinib therapy was 43.5 % (10/23) after one month and 61 % (14/23) after two months, respectively. The median overall survival was 69 months. Reactivation of cytomegalovirus (26.1 %) and grade 3-4 anemia (30.4 %) were the two main side effects of ruxolitinib therapy. Seven patients (30.4 %) passed away following a follow-up of a median of six months (range 1-70). The reasons for death included sepsis (n = 2, 28.6 %), progression of aGVHD (n = 3, 42.8 %), and other reasons.

Conclusion: Ruxolitinib has an ORR of 61 % for SR-aGVHD, making it a safe and effective therapy choice in real-world settings.

Keywords: Graft versus host disease; Hematopoietic stem cell transplantation; JAK inhibitör; Ruxolitinib; Steroids.