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

Bone Marrow Transplantation (2024)Cite this article

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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.

Author information

Author notes

  1. These authors contributed equally: Alistair Murray, Swe Mar Linn.

  2. These authors jointly supervised this work: Kareem Jamani, Dennis Dong Hwan Kim.

Authors and Affiliations

  1. Division of Hematology and Hematologic Malignancies, University of Calgary, Calgary, AB, Canada

    Alistair Murray & Kareem Jamani

  2. Hans Messner Allogeneic Blood and Marrow Transplant Unit, Princess Margaret Cancer Centre, Toronto, ON, Canada

    Swe Mar Linn, Igor Novitzky-Basso, Jonas Mattsson & Dennis Dong Hwan Kim

  3. Laval University, Quebec City, QC, Canada

    Benoit Yu & Christopher Lemieux

  4. Department of Hematology, Faculty of Medicine, University of Toronto, Toronto, ON, Canada

    Igor Novitzky-Basso, Jonas Mattsson & Dennis Dong Hwan Kim

  5. Department of Hematology, The Ottawa Hospital, Ottawa, ON, Canada

    Michael Kennah

  6. University of Saskatchewan, Saskatoon, SK, Canada

    Mohamed Elemary

  7. Saskatchewan Cancer Agency, Saskatoon, SK, Canada

    Mohamed Elemary

  8. The Leukemia/Bone Marrow Transplant Program, Vancouver General Hospital, British Columbia Cancer Agency, Vancouver, BC, Canada

    Jennifer White

  9. University of British Columbia, Columbia, BC, Canada

    Jennifer White

  10. Division of Hematology and Medical Oncology, CHU de Québec -Université Laval, Quebec City, QC, Canada

    Christopher Lemieux

  11. Alberta Blood & Marrow Transplant Program, Tom-Baker Cancer Centre, Calgary, AB, Canada

    Kareem Jamani

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  1. Alistair Murray
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Contributions

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.

Corresponding author

Correspondence to Dennis Dong Hwan Kim.

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Competing interests

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 (2024). https://doi.org/10.1038/s41409-024-02249-8

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