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

## TL;DR

This study examines the real-world effectiveness and safety of ruxolitinib for treating steroid-resistant acute graft-versus-host disease in transplant patients.

## Contribution

The study provides real-world data on ruxolitinib's use for aGvHD and identifies potential biomarkers predicting treatment response.

## Key findings

- Ruxolitinib showed a 65% overall response rate and 78% failure-free survival in treating aGvHD.
- Patients responding to ruxolitinib required fewer steroids and had lower levels of specific serum biomarkers.
- Steroid-dependent aGvHD was a significant predictor of better response to ruxolitinib.

## 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.

The online version contains supplementary material available at 10.1007/s00277-024-05696-x.