The role of ruxolitinib in the management of acute GVHD

NAMDAROĞLU S., Hidayet E., Aydin M. S., ERKURT M. A., BERBER İ., Cinar O. E., ...More

Transfusion and Apheresis Science, vol.64, no.1, 2025 (SCI-Expanded) identifier identifier

  • Publication Type: Article / Article
  • Volume: 64 Issue: 1
  • Publication Date: 2025
  • Doi Number: 10.1016/j.transci.2024.104055
  • Journal Name: Transfusion and Apheresis Science
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, BIOSIS, Biotechnology Research Abstracts, MEDLINE
  • Keywords: Graft versus host disease, Hematopoietic stem cell transplantation, JAK inhibitör, Ruxolitinib, Steroids
  • Ankara Yıldırım Beyazıt University Affiliated: Yes

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.