Many congenital or acquired nonmalignant diseases (NMDs) of the hematopoietic system can be potentially cured by allogeneic hematopoietic cell transplantation (HCT) with varying types of donor grafts, degrees of HLA matching, and intensity of conditioning regimens. Unique features that distinguish the use of allogeneic HCT in this population include higher rates of graft failure, immune-mediated cytopenias, and the potential to achieve long-term disease-free survival in a mixed chimerism state. Additionally, in contrast to patients with hematologic malignancies, a priority is to completely avoid graft-versus-host disease in patients with NMD because there is no theoretical beneficial graft-versus-leukemia effect that can accompany graft-versus-host responses. In this review, we discuss the current approach to each of these clinical issues and how emerging novel therapeutics hold promise to advance transplant care for patients with NMDs.
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Conflict-of-interest disclosure: Z.D. receives research support from Incyte Corp. and Regimmune Corp. and has received consulting fees from Syndax Pharmaceuticals Inc. and Omeros Corp. Y.-B.C. has received consulting fees from Incyte, Gamida Cell, Equilium, Celularity, Daiichi, Actinium, and Abbvie. B.R.B. serves on advisory boards for Magenta Therapeutics and BlueRock Theapeutics; receives research funding from BlueRock Therapeutics, Rheos Medicines, Equilibre Pharmaceuticals Corp., and Carisma Therapeutics, Inc; is a cofounder of Tmunity Therapeutics; and receives the following grant support: National Institutes of Health National Institute of Allergy and Infectious Diseases (R37 AI34495) and National Heart, Lung, and Blood Institute (R01 HL147324, R01 HL155114, R01 HL56067). M.H. declares no competing financial intererests.
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