Chronic graft-versus-host disease (cGVHD) remains a major barrier to a successful hematopoietic stem cell transplantation (HSCT); in cases refractory to first-line therapy with steroids, there is no standard of care for second-line therapy. As such, ruxolitinib is a promising drug in this scenario. We retrospectively analyzed its efficacy and safety as treatment of steroid-refractory cGVHD in 35 patients from two transplant centers, with the longest follow-up described to date. Evaluated patients had a median of 3 organs affected (1-7), with most (64%) having moderate cGVHD. The median number of previous therapy lines was two (1-6). Overall response rate was 89% (complete response, 26%) after a median of 4 weeks of therapy. Median follow-up was 43 months (range 11-59). At follow-up, of the 27 patients still alive, 18 patients (67%) are free of any immunosuppression, and 6 (22%) are using ruxolitinib as the only immunosuppressive drug. Failure-free survival was 77.1%, 68.6%, 54% and 51,4% at 6, 12, 24 and 36 months, respectively. Median overall survival (OS) was not reached. Toxicities were mostly hematological and resolved after dose reduction in most cases. In conclusion, our data, which represent the cohort of patients with cGVHD treated with ruxolitinib with the longest follow-up to date, support the use of this drug as a safe and effective option for refractory cGVHD.
Copyright © 2021. Published by Elsevier Inc.

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