Novel targets in the treatment of chronic graft-versus-host disease

Abstract

Despite advances that have improved survival after allogeneic hematopoietic stem cell transplantation (HCT), chronic graft-versus-host disease (GVHD) remains a leading cause of late morbidity and mortality after transplant. Current treatment options show limited efficacy in steroid-refractory disease, and there exists a paucity of robust data to guide management decisions. Lack of United States Food and Drug Administration (FDA)- or European Medicines Agency (EMA)-approved agents in GVHD underscore the importance of developing novel therapies. Better understanding of the biology of chronic GVHD has provided novel targets for treatment, and structured guidelines in diagnosis and in clinical trial design have provided a common language and pathways for research in this area. These, combined with the surge of drug development in Oncology and Immunology, are factors that have contributed to the accelerating field of drug development and clinical research in chronic GVHD. In these exciting times, it is possible to foresee long awaited advances in the treatment of this devastating complication of HCT. This review will summarize the ongoing clinical development for novel therapies in chronic GVHD.

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Acknowledgements

This work was supported by the National Institutes of Health’s (NIH) National Cancer Institute (NCI), Center for Cancer Research, Intramural Research Program. The opinions expressed here are those of the authors and do not represent the official position of the NIH, NCI or the United States Government.

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Correspondence to S Z Pavletic.

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Im, A., Hakim, F. & Pavletic, S. Novel targets in the treatment of chronic graft-versus-host disease. Leukemia 31, 543–554 (2017). https://doi.org/10.1038/leu.2016.367

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