Haploidentical Transplants: Nonmalignant Diseases in Adults

Abstract

Haploidentical transplantation is becoming a common practice in recent times particularly after the introduction of post-transplant cyclophosphamide by the Johns Hopkins group (Luznik et al. Biol Blood Marrow Transplant 14(6):641–650, 2008; Bacigalupo et al. Adv Hematol 2016:7802967, 2016). In the past, the excessive morbidity and mortality caused by the HLA-mismatched transplant made this intervention very high risk for treatment-related mortality, but nowadays it appears that the outcomes are similar to those of HLA-matched transplants, i.e., HLA-matched, related, or unrelated (Ciurea et al. Blood 126(8):1033–1040, 2015; Kanate et al. Blood 127(7):938–947, 2016; Ghosh et al. J Clin Oncol 34(26):3141–3149, 2016). In fact, they appear to compare well with other alternative donors such as cord transplants (Brunstein et al. Blood 118(2):282–288, 2011). There are many potential advantages in using haploidentical donors as alternative donors when needed. Given that the toxicity seen in haploidentical transplants is not different compared with other types of allogeneic transplant approaches, its use in hereditary nonmalignant disorders seems logical for exploration. In this chapter, the results of such approach in patients with hereditary nonmalignant disorders will be reviewed.