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Ruxolitinib in the management of steroid-resistant/-dependent acute and chronic graft-versus-host disease: results of routine practice in an academic centre

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Abstract

Graft-versus-host disease (GVHD) is an important complication after allogeneic haematopoietic stem cell transplantation (HSCT). Corticosteroids are the standard first-line treatment. Steroid-resistant/-dependent (SR/D) acute and chronic GVHD (aGVHD, cGVHD) lead to significant morbidity/mortality. The JAK2 inhibitor ruxolitinib has recently been shown in clinical trials to be effective in SR/D aGVHD and cGVHD. We retrospectively analysed the efficacy and safety of ruxolitinib in a cohort of SR/D aGVHD and cGVHD patients treated in a non-trial setting. In the aGVHD cohort, there were 14 men and 12 women, median age at 38 (19–63) years. At day 28 post-ruxolitinib, the overall response rate (ORR) was 86% (complete response, CR, 36%; partial response, PR, 50%). Continued ruxolitinib beyond day 28 resulted in a final CR of 68%. However, 3/15 (20%) of CR patients developed cGVHD. In the cGVHD cohort, there were 16 men and 15 women, median age at 33 (21–64) years. The ORR, CR and PR rates changed with continued ruxolitinib treatment, being 86%, 17% and 69% at 1 month; 79%, 38% and 41% at 3 months; and 83%, 52% and 31% at 6 months. Five patients had overlap GVHD, four of whom achieved CR. Multivariate analysis showed that superior overall survival and failure-free survival were associated with CR at day 28 for aGVHD, and CR at 1 year for cGVHD. Ruxolitinib treatment was efficacious for SR/D aGVHD and cGVHD, and continued treatment for at least 6 months was needed to maximize benefit.

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Authors and Affiliations

  1. Department of Medicine, Queen Mary Hospital, Professorial Block, Pokfulam Road, Hong Kong, China

    Garret M. K. Leung, Joycelyn P. Y. Sim, Yu-Yan Hwang, Thomas S. Y. Chan, Albert K. W. Lie, Eric Tse & Yok-Lam Kwong

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  1. Garret M. K. Leung
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  2. Joycelyn P. Y. Sim
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Contributions

Garret M.K. Leung: clinical management, data analysis, manuscript writing and approval.

Joycelyn P.Y. Sim: clinical management, manuscript writing and approval.

Yu-Yan Hwang: clinical management, manuscript writing and approval.

Thomas S.Y. Chan: clinical management, manuscript writing and approval.

Albert K.W. Lie: clinical management, manuscript writing and approval.

Eric Tse: clinical management, manuscript writing and approval.

Yok-Lam Kwong: clinical management, manuscript writing and approval.

Corresponding author

Correspondence to Yok-Lam Kwong.

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The study was approved by the Institute Review Board.

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The patients gave informed consent.

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The authors declare that they have no conflict of interest.

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Garret M. K. Leung and Joycelyn P. Y. Sim contributed equally to this work.

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Leung, G.M.K., Sim, J.P.Y., Hwang, YY. et al. Ruxolitinib in the management of steroid-resistant/-dependent acute and chronic graft-versus-host disease: results of routine practice in an academic centre. Ann Hematol 101, 155–163 (2022). https://doi.org/10.1007/s00277-021-04658-x

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