Efficacy and safety of ruxolitinib in the treatment of chronic graft-versus-host disease: a retrospective analysis

Denk, Alexander and Mittermaier, Cornelia and Weber, Daniela and Fante, Matthias A. and Gunes, Sibel and Edinger, Matthias and Herr, Wolfgang and Wolff, Daniel (2024) Efficacy and safety of ruxolitinib in the treatment of chronic graft-versus-host disease: a retrospective analysis. ANNALS OF HEMATOLOGY, 103 (9). pp. 3755-3764. ISSN 0939-5555, 1432-0584

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Abstract

Steroid-refractory chronic graft-versus-host disease (cGvHD) is associated with significant morbidity and mortality, with ruxolitinib being the first drug approved for its treatment. We retrospectively analyzed the safety and efficacy of ruxolitinib for treatment of cGvHD at our center between 07/2015 and 12/2022 and identified 48 patients receiving ruxolitinib as second (18/48) or advanced (30/48) treatment line. Ruxolitinib was started on median day 340 (range 119-595) after cGvHD onset; median duration of administration was 176 (range, 79-294) days with 16/48 patients continuing treatment at last follow-up. National Institutes of Health organ grading and the intensity of immunosuppression were assessed at the start of ruxolitinib treatment and repeated after 1, 3, 6, and 12 months. Response assessment was terminated at the start of any additional new immunosuppressant treatment. The median time of follow-up was 582 (range, 104-1161) days. At the primary analysis after six months on ruxolitinib treatment, the overall response rate was 33%, and failure-free survival was 58%. Infectious adverse events >= CTCAE grade III were observed in 10/48 patients. The response rate was not associated with the severity of cGvHD, number of previous treatment lines, or number of additional agents combined with ruxolitinib applying a univariate regression model. At the time of the 12-month follow-up, four patients experienced recurrence of the underlying malignancy and two patients had experienced non-relapse-related mortality. Overall, ruxolitinib was relatively well-tolerated and showed outcomes comparable to the REACH3 trial in a heavily pretreated patient population.

Item Type: Article
Uncontrolled Keywords: ; Chronic graft versus host disease; Allogeneic hematopoietic stem cell transplantation; Ruxolitinib; Steroid-refractory chronic graft-versus-host disease; JAK 1/2 inhibitor
Subjects: 600 Technology > 610 Medical sciences Medicine
Divisions: Medicine > Lehrstuhl für Innere Medizin III (Hämatologie und Internistische Onkologie)
Depositing User: Dr. Gernot Deinzer
Date Deposited: 16 Jan 2026 11:34
Last Modified: 16 Jan 2026 11:34
URI: https://pred.uni-regensburg.de/id/eprint/64125

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