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Ruxolitinib: a steroid sparing agent in chronic graft-versus-host disease

Abstract

Inhibition of the Janus-associated kinases (JAK) with ruxolitinib (RUX) reduces graft-versus-host disease (GVHD) in preclinical and clinical models. In total 19 allograft recipients with moderate/severe steroid-dependent chronic GVHD received RUX as ≥2nd line salvage. RUX was well tolerated, and led to complete/partial resolution of oral (92/7%), cutaneous (82/0%), hepatic (71/28%), gastro-intestinal (75/17%), musculoskeletal (33/67%), pulmonary (0/80%), scleroderma (0/75%), vaginal (0/75%), and ocular (0/100%) chronic GVHD. Overall 18 achieved partial response and 1 complete response according to NIH Consensus Criteria. Responses occurred early and were sustained which enabled discontinuation (68%) or reduction of steroids to physiologic doses (21%). We conclude that RUX is an effective steroid-sparing agent in chronic GVHD.

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Acknowledgements

We thank Dr. Steve Pavletic for his critical review of this manuscript and constructive comments.

Author contributions

HJK and JFD: designed research, performed research, collected data, provided subjects, analyzed and interpreted data, performed statistical analysis, wrote the manuscript; VKK, AAL, IP, AJ, EW, DR, ZAK, IB, and MA performed research, collected data, provided subjects, analyzed and interpreted data, reviewed and approved the manuscript; JW, SB, and ASK: collected data, analyzed and interpreted data, reviewed, and approved the manuscript. JFD is supported by the National Cancer Institute (NIH/NCI: R35 CA210084 and P50 CA171963.

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Correspondence to Hanna Jean Khoury.

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Conflict of interest

HJK, VKK, EW, and JFD received research support from Incyte, and honoraria for attending advisory board meetings. The remaining authors declare that they have no conflict of interests.

Additional information

Part of this data was presented at the Annual Meeting of the American Society of Hematology in December 2015

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Khoury, H.J., Langston, A.A., Kota, V.K. et al. Ruxolitinib: a steroid sparing agent in chronic graft-versus-host disease. Bone Marrow Transplant 53, 826–831 (2018). https://doi.org/10.1038/s41409-017-0081-5

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