Treatment of childhood leukemia with haploidentical hematopoietic stem cell transplantation using parent as donor: a single-center study of 111 case

Y. Sun, J. Xiao, Z.-H. Li, S.-F. Fan, Y. Shen

Department of Paediatric Hematology, Beijing Daopei Hospital, Bei Jing, China. zhuhuamail@yeah.net

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• Hematology

OBJECTIVE: In the present study, the outcomes of childhood leukemia treated with haplo-HSCT using parent as donor were evaluated and the risk factors for survival were identified.

PATIENTS AND METHODS: 111 consecutive cases from March 2002 to March 2012 in our center were analyzed. The median age of patients was 10 (3-14) years old. All patients received unmanipulated combined marrow and peripheral blood stem cells for transplant after conditioning with busulfan and cyclophosphamide (Cy)/Cy and total body irradiation (TBI) plus antithymocyte globulin (ATG).

RESULTS: Durable hematopoietic reconstitution was seen in 98% of recipients. Engraftment failure occurred in 3 cases including 2 cases of father to daughter transplants. One-hundred-day transplant-related mortality (TRM) was only 4.5%. The cumulative incidences of grade II to IV acute graft-versus-host disease (aGvHD) and chronic GvHD (cGvHD) were 47.6% and 28.3%, respectively. With the median follow-up of 32 (12-134) months, 2-year and 5-year overall survival (OS) rates for all patients were 82.1% and 79.2%, respectively. Five-year OS rates for patients in early, intermediate and advanced disease were 84.0%, 81.0%, and 57.1%, respectively (p = 0.08). Five-year OS of transplants in father to son, father to daughter, mother to son, and mother to daughter were was 88.1%, 57.1%, 70.6%, and 82.6%, respectively (p = 0.08).

CONCLUSIONS: Under current protocol, children with leukemia tolerate haplo-HSCT from their parent very well with lower TRM, less cGvHD, and better OS compared with our published data. Pre-transplant disease status and donor- recipient relationship and the recipient age have significant impact on survival.

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