First‐line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy.

Abstract: We retrospectively compared the outcomes of children with severe aplastic anemia (SAA) who received immunosuppressive therapy (IST) or who underwent hematopoietic stem cell transplantation (HSCT) from a haploidentical donor (HID), between 2007 and 2016. A total of 52 children with SAA under the age of 17 years were initially treated with IST (n = 24) or haploidentical HSCT (n = 28) as first‐line treatment. The estimated 10‐year overall survival was 73.4 ± 12.6% and 89.3 ± 5.8% in patients treated with IST or HID‐HSCT (<italic>P </italic>=<italic> </italic>.806). The failure‐free survival was significantly inferior in patients receiving IST than in those undergoing transplantation from an HID (52.6 ± 10.5% vs 89.3 ± 5.8, <italic>P </italic>=<italic> </italic>.008). In univariate and multivariate analysis, the choice of first‐line immunosuppressive therapy was the only adverse predictor for failure‐free survival. At the last follow‐up, completely normal blood count was observed in 11 of 20 (55.0%) and 24 of 25 (96.0%) live cases in IST and HID‐HSCT cohort (<italic>P </italic>=<italic> </italic>.003). These suggest that HSCT from a haploidentical donor could be considered as first‐line treatment in children who lack a matched related donor, especially in experienced transplantation centers. [ABSTRACT FROM AUTHOR]