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

• Published in: European review for medical and pharmacological sciences Vol. 19; no. 22; p. 4379
• Main Authors: Sun, Y, Xiao, J, Li, Z H, Fan, S F, Shen, Y
• Format: Journal Article
• Language: English
• Published: Italy 01.11.2015
• Subjects: Adolescent; Antilymphocyte Serum - therapeutic use; Busulfan - therapeutic use; Child; Child, Preschool; Cyclophosphamide - therapeutic use; Female; Graft vs Host Disease - diagnosis; Graft vs Host Disease - prevention & control; Hematopoietic Stem Cell Transplantation - adverse effects; Hematopoietic Stem Cell Transplantation - methods; Humans; Leukemia - diagnosis; Leukemia - therapy; Male; Parents; Tissue Donors; Transplantation Conditioning - methods; Treatment Outcome; Whole-Body Irradiation - methods
• ISSN: 2284-0729

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. 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). 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). 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.