Improved outcomes of allogeneic hematopoietic stem cell transplantation including haploidentical transplantation for childhood myelodysplastic syndrome.

This retrospective study aimed to investigate the outcomes of allogeneic hematopoietic stem cell transplantation (HSCT) for childhood myelodysplastic syndrome (MDS). Thirty-six patients (low-grade MDS, 24; advanced MDS, 12) received HSCT at the Asan Medical Center over two decades (early period, 1997-2007; recent period, 2008-2017). The transplantation outcomes were analyzed according to disease status, conditioning regimen, various donor types, and period of HSCT. During a median follow-up of 5.6 (range, 1.4-21.1) years, the probability of overall survival (OS) and failure-free survival was 77% and 69%, respectively. The cumulative incidence of transplantation-related mortality (TRM) was 12%. Significantly reduced TRM and improved OS were observed in patients who received HSCT during the recent period vs. the early period (TRM, 4% vs. 30%, P = 0.021; OS, 87% vs. 50%, P = 0.006). Comparable outcomes were observed for HSCT from haploidentical family donors vs. HLA-identical donors (TRM, 10% vs. 14%, P= 0.837; OS, 86% vs. 79%, P = 0.625). This study identified the improved outcomes of allogeneic HSCT for childhood MDS over time, in addition, the feasible outcomes of haploidentical HSCT suggested its use as an attractive alternative in the future procedures.