Rocha, V., Arcuri, L. J., Seber, A., Colturato, V., Zecchin, V. G., Kuwahara, C., Nichele, S., Gouveia, R., Fernandes, J. F., Macedo, A. V., Tavares, R., Daudt, L., De Souza, M. P., Darrigo-Jr, L. G., Villela, N. C., Mariano, L. C. B., Ginani, V. C., Zanette, A., Loth, G., Gomes, A. A., Hamerschlak, N., Flowers, M. E., and Bonfim, C.
Haploidentical hematopoietic-cell transplantation using post-transplant cyclophosphamide(Haplo-PTCy) is a feasible procedure in children with haematologic malignancies. However, data of a large series of children with acute leukaemia(AL) in this setting is missing. We analysed 144 AL Haplo-PTCy paediatric recipients; median age was 10 years. Patients had acute lymphoblastic(ALL; n= 86) or myeloblastic leukaemia(AML; n= 58) and were transplanted in remission(CR1: n= 40; CR2: n= 57; CR3+: n= 27) or relapse (n= 20). Bone marrow was the graft source in 57%; donors were father (54%), mother (35%), or sibling (11%). Myeloablative conditioning was used in 87%. Median follow-up was 31 months. At day +100, cumulative incidence (CI) of neutrophil recovery and acute GVHD (II–IV) were 94% and 40%, respectively. At 2-years, CI of chronic GVHD and relapse, were 31%, 40%, and estimated 2-year overall survival (OS), leukaemia-free survival (LFS) and graft-versus-host-relapse-free survival (GRFS) were 52%, 44% and 34% respectively. For patients transplanted in remission, positive measurable residual disease (MRD) prior to transplant was associated with decreased LFS (p= 0.05) and GRFS (p= 0.003) and increased risk of relapse (p= 0.02). Mother donor was associated with increased risk of chronic GVHD (p= 0.001), decreased OS (p= 0.03) and GRFS (p= 0.004). Use of PBSC was associated with increased risk of chronic GVHD (p= 0.04). In conclusion, achieving MRD negativity pre-transplant, avoiding use of mother donors and PBSC as graft source may improve outcomes of Haplo-PTCy in children with AL.