Page 114 - Haematologica Vol. 109 - July 2024

ARTICLE - MUD versus haplo PT-CY stem cell transplantation in children with AML A. Ruggeri et al.
group in the adult setting.8,34 This platform is now the most widely adopted strategy because it is cost-effective and easily replicated. In recent years, emerging evidence also supports the use of this platform in the pediatric popula- tion with hematological malignancies.19,35
Saglio et al.15 retrospectively compared the outcome of 23 pediatric patients undergoing haplo-HCT with PT-CY for acute leukemia with patients undergoing HCT from MUD (N=41) and HLA-mismatched unrelated donor (MMUD) (N=26) from a single institution. Five-year OS, NRM and RI were not different for the three groups, confirming that haplo PT-CY is a suitable clinical option for pediatric patients. More recently, Srinivasan et al.16 compared outcomes of haplo-HCT PT-CY with peripheral blood (N=26) to matched sibling donor (MSD) (N=31) and MUD HCT (N=47), both with
BM as stem cell source. Results showed that haplo-HCT PT-CY with peripheral blood had comparable outcomes to BM MSD and MUD HCT. Hong et al.17 compared the outcomes of children and adolescents with high-risk acute leuke- mia who underwent haplo-HCT PT-CY (N=35) or MUD HCT (N=45) after a busulfan-based myeloablative conditioning. No differences were observed in the main outcomes. In a subgroup analysis of patients with AML (haplo, N=16; MUD, N=16), the 3-year GRFS, LFS, and OS rates in haplo-HCT and MUD groups were 80.8% versus 61.9%, 87.1% versus 73.9%, and 93.8% versus 85.6%, respectively.
Here, we report the largest series of children with AML treated with haplo PT-CY reported to the EBMT registry. Our results are in line with the previous single center reports and confirmed the overall feasibility of the PT-CY approach
Figure 1. Two-years survival outcomes after matched unrelated donor and haploidential hematopoietic cell transplantation after matched pair analysis. Two-year leukemia-free survival (LFS), overall survival (OS), relapse incidence (RI) and non-relapse mortal- ity (NRM) after matched unrelated donor (MUD) and haploidential hematopoietic cell transplantation in children with acute myleoid leukemia after matched pair analysis.
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