Haploidentical hematopoietic stem cell transplantation as first-line therapy for aplastic anemia in children: A single-center experience

Abstract

Abstract HID-HSCT is considered a viable treatment option for children with AA who do not have a suitable identical donor or an unrelated donor, but there are not enough studies on how it fares in comparison to with stem cells from MSD-HSCT and UD-HSCT. Therefore, this study compared the outcomes of three ways as the first-line treatment for children with AA. We retrospectively compared the outcomes of 85 pediatric patients who underwent HID-HSCT (n = 41), MSD-HSCT (n = 10), and UD-HSCT (n = 34)from August 2016 toJune 2023. Cumulative rates of neutrophil and platelet implantation, incidence of aGVHD, cGVHD, and 6-year OS were not statistically significant in the three groups. In the univariate and multivariate analyses, the survival outcome for the entire population was adversely associated with TMA (P = 0.040), but was not related to other factors. In summary, there were no statistically significant differences in terms of hematopoietic reconstruction and prognosis between the three transplantation methodologies. Thus, for individuals without HLA-matched siblings or unrelated donors, HID-HSCT might be a practical and promising primary treatment option.