The role of ruxolitinib in the management of acute GVHD

Sinem Namdaroglu; Emine Hidayet; Muruvvet Seda Aydin; Mehmet Ali Erkurt; Ilhami Berber; Olgu Erkin Cinar; Gulsum Ozet; Seda Yilmaz; Merve Apaydin; Mehmet Sinan Dal; Serdal Korkmaz; Abdulkadir Basturk; Turgay Ulaş; Fevzi Altuntas

doi:10.1016/j.transci.2024.104055

The role of ruxolitinib in the management of acute GVHD

Transfus Apher Sci. 2024 Dec 16;64(1):104055. doi: 10.1016/j.transci.2024.104055. Online ahead of print.

Authors

Affiliations

¹ Dokuz Eylul University, Faculty of Medicine, Department of Hematology, Izmir, Turkey. Electronic address: drsinemnamdaroglu@gmail.com.
² University of Health Sciences, Ankara Oncology Training and Research Hospital, Department of Hematology & Apheresis Unit, Ankara, Turkey.
³ Ankara Bilkent City Hospital, Department of Hematology and Bone Marrow Transplantation Unit, Ankara, Turkey.
⁴ Inonu University, Faculty of Medicine, Department of Hematology, Malatya, Turkey.
⁵ University of Health Sciences, Konya Medical Faculty, Department of Hematology and Bone Marrow Transplantation Unit, Konya, Turkey.
⁶ University of Health Sciences, Kayseri Medical Faculty, Department of Hematology and Bone Marrow Transplantation Unit, Kayseri, Turkey.
⁷ University of Health Sciences, Ankara Oncology Training and Research Hospital, Department of Hematology & Apheresis Unit, Ankara, Turkey; Ankara Yildirim Beyazit University, School of Medicine, Department of Internal Medicine, Division of Hematology, Ankara, Turkey.

PMID: 39719750
DOI: 10.1016/j.transci.2024.104055

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.