Clinical observation of haploid hematopoietic stem cell transplantation and intensive immunosuppressive therapy for the treatment of severe aplastic anemia in pediatric patients / 中华实用儿科临床杂志

ABSTRACT

Objective:To compare the efficacy of haploid hematopoietic stem cell transplantation (haplo-HSCT) and intensive immunosuppressive therapy (IST) in children with severe aplastic anemia (SAA).Methods:The medical records of children newly diagnosed as SAA in the First Affiliated Hospital of Zhengzhou University from January 2013 to June 2018 were retrospectively analyzed.Among them, 33 patients received haplo-HSCT and 24 patients received IST that combined anti-thymocyte globulin(ATG) with Cyclosporine (CsA). The effective rate, overall survival (OS) rate, and failure free survival(FFS) rate of children in the haplo-HSCT group and the IST group were compared.Results:The median follow-up period was 25 months (9-60 months). There were 5 cases of early death in the haplo-HSCT group and 4 cases in the IST group, and the differences were not statistically significant ( P=0.822). Leaving out the early death cases, the effective rate in the haplo-HSCT group [100%(28/28 cases)] was higher than that in the IST group [30%(6/20 cases)] after 3 months of treatment, the difference was statistically significant ( χ2=27.671, P<0.01). After 6 months of treatment, the effective rate in the haplo-HSCT group [92.9%(26/28 cases)] was higher than that in the IST group [65.0%(13/20 cases)], and the difference was statistically significant ( χ2=5.943, P=0.015). After 12 months of treatment, the effective rate in the haplo-HSCT group [89.3%(25/28 cases)] was higher than that in the IST group [70.0%(14/20 cases)], but the difference was not statistically significant( P>0.05). The 3-year expected OS rate of children in the haplo-HSCT group and the IST group were 75.0% and 70.3%, respectively, with no statistically significant difference ( χ2=0.133, P=0.716). The 3-year expected FFS rate of children in the haplo-HSCT group (74.2%) was significantly higher than that in the IST group (48.7%), and the difference was statistically significant ( χ2=4.036, P=0.045). Conclusion:For children with SAA, haplo-HSCT is also an effective treatment if there is no sibling donor of hematopoietic stem cell transplantation.