Ruxolitinib for the treatment of acute graft-versus-host disease: a retrospective analysis

Denk, Alexander and Edinger, Matthias and Weber, Daniela and Holler, Ernst and Fante, Matthias A. and Meedt, Elisabeth and Gunes, Sibel and Poeck, Hendrik and Mittermaier, Cornelia and Herr, Wolfgang and Wolff, Daniel (2024) Ruxolitinib for the treatment of acute graft-versus-host disease: a retrospective analysis. ANNALS OF HEMATOLOGY, 103 (8). pp. 3071-3081. ISSN 0939-5555, 1432-0584

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Abstract

Steroid-refractory acute graft-versus-host disease (aGvHD) is a serious complication after allogeneic hematopoietic stem cell transplantation, associated with significant mortality. Ruxolitinib was the first drug approved for aGvHD, based on results of the REACH2 trial; however, real-world data are limited. We retrospectively analyzed the safety and efficacy of ruxolitinib for treatment of aGvHD at our center from March 2016 to August 2022 and assessed biomarkers of risk. We identified 49 patients receiving ruxolitinib as second- (33/49), third- (11/49), fourth- (3/49), or fifth-line (2/49) treatment. Ruxolitinib was started on median day 11 (range, 7-21) after aGvHD onset; median duration of administration was 37 days (range, 20-86), with 10 patients continuing treatment at last follow-up. Median follow-up period was 501 days (range, 95-905). In the primary analysis at the 1-month assessment, overall response rate was 65%, and failure-free survival was 78%. Infectious complications >= CTCAE Grade III were observed in 10/49 patients within 1-month followup. Patients responding to ruxolitinib therapy required fewer steroids and exhibited lower levels of the serum biomarkers regenerating islet-derived protein 3-alpha, suppression of tumorigenicity 2, and the Mount Sinai Acute GVHD International Consortium algorithm probability. A univariate regression model revealed steroid-dependent aGvHD as a significant predictor of better response to ruxolitinib. Within 6-months follow-up, four patients experienced recurrence of underlying malignancy, and eight died due to treatment-related mortality. Overall, ruxolitinib was welltolerated and showed response in heavily pretreated patients, with results comparable to those of the REACH2 trial. Biomarkers may be useful predictors of response to ruxolitinib.

Item Type: Article
Uncontrolled Keywords: MARROW; THERAPY; GVHD; BIOMARKER; RISK; Acute graft-versus-host disease; aGvHD; Allogeneic hematopoietic stem cell transplantation; Ruxolitinib; Steroidrefractory acute graft-versus-host disease; JAK1/2 kinase 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: 24 Jul 2025 10:19
Last Modified: 24 Jul 2025 10:19
URI: https://pred.uni-regensburg.de/id/eprint/63667

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