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Real-world experience with ruxolitinib therapy for steroid-refractory acute graft versus host disease

Abstract

Acute graft versus host disease (aGVHD) is a complication of allogeneic hematopoietic stem cell transplant (HCT) and is associated with significant morbidity and mortality. Steroid refractory aGVHD (SR-aGVHD) carries a particularly grim prognosis. Ruxolitinib has shown promise for treatment of SR-aGVHD in a phase 3 trial; however, safety and efficacy data outside of the clinical trial setting is lacking. We performed a multicenter retrospective study to examine the response to ruxolitinib and its efficacy in patients with SR-aGVHD. We included 59 patients treated with ruxolitinib for SR-aGVHD between 2015 and 2022. Of these 59 patients, 36 patients (61.0%) achieved a complete (CR) or partial response (PR) at 28 days, while 31 patients (52.5%) obtained a CR/PR at day 56. Patients that achieved a CR or PR at day 28 had a higher rate of overall survival (OS; 69.2%), compared with patients that did not (31.6%; p = 0.037). OS at 12 months was 41.5%, with a median OS duration of 5.3 months. Failure free survival (FFS) at 12 months was 29.1%, with a median FFS of 2.6 months. Overall, this real-world experience data support ruxolitinib as the standard of care for SR-aGVHD in a non-controlled trial population.

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Fig. 1: Response and clinical benefit of ruxolitinib therapy.
Fig. 2: Overall and failure-free survival, including reasons for failure.
Fig. 3: Change in steroid dose starting at day 0 of ruxolitinib therapy.

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Data availability

The anonymized datasets generated and analyzed during the current study are available from the corresponding author on reasonable request.

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Acknowledgements

The study was supported by Novartis and from Princess Margaret Cancer Foundation.

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AM and SML performed data collection, analysis, and manuscript writing. KJ and DK designed the study, provided all administrative support, supervised the project and manuscript revision. All the other authors contributed to data collection, reviewed the manuscript and approved it.

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Correspondence to Dennis Dong Hwan Kim.

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DK received research grant and honoraria from Novartis, Pfizer and Paladin.

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Murray, A., Linn, S.M., Yu, B. et al. Real-world experience with ruxolitinib therapy for steroid-refractory acute graft versus host disease. Bone Marrow Transplant 59, 759–764 (2024). https://doi.org/10.1038/s41409-024-02249-8

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