Donor type/graft source selection strategies based on disease status for paediatric acute myeloid leukemia transplantation

In the absence of a human leukocyte antigen-matched sibling donor for paediatric patients with acute myeloid leukemia (AML) who underwent the first allogeneic hematopoietic cell transplantation (allo-HCT), the optimal donor type/graft source remain undetermined among donor types—haploidentical related donor (HRD) and matched unrelated donor (MUD)—as well as graft source—umbilical cord blood (UCB). This study aimed to determine the optimal donor type/graft source. The Japanese database on 441 recipients (median age, 6.0 years, range [0.0–15.0]) for HRD (n = 71), MUD (n = 111), and UCB (n = 259) was analyzed retrospectively. Regarding recipients in remission [complete remission (CR) 1 or CR2 at allo-HCT, n = 271], the rate of a composite endpoint consisting of extensive chronic graft-versus-host disease (cGVHD)- and leukemia-free survival (LFS)—extensive cGVHD-LFS—was not statistically significant among UCB, MUD, and HRD recipients. Regarding recipients not in remission (n =170), extensive cGVHD-LFS was significantly higher for MUD recipients than for UCB ( p = 0.007) and HRD ( p = 0.015) recipients. HRD, MUD, and UCB were optimal for recipients in remission, while MUD was optimal for recipients not in remission. Namely, the donor type/graft source needs to be selected based on disease status at HCT.