[Transplantation of haploidentical hematopoietic stem cells from family members without in vitro T-cell depletion for treatment of hematologic malignancies in pediatric patients].
OBJECTIVE
To evaluate the efficacy and safety of haploidentical (from family member donors) hematopoietic stem cell transplantation (HSCT) for children with hematologic malignancies.


METHODS
Fifty-eight children under fourteen years with hematological malignancies underwent haploidentical HSCT. All patients had poor-risk clinical or cytogenetic features and reclassified into high-risk and standard-risk groups. Transplantation outcomes were analyzed.


RESULTS
Of fifty-eight patient/donor pairs, seven (12.1%) were mismatched in two HLA loci, twenty (34.5%) in three loci, and thirty-one (53.4%) in four loci. Follow-ups were performed for a median of 1,663 (752-2,664) days after transplantation. All patients achieved stable engraftments. The cumulative incidence of acute graft-versus-host disease (GVHD) of grades 2-4 was (54.8+/-7.6)%, and that of grades 3-4 was (11.4+/-4.8)%. The cumulative incidence of chronic GVHD was (45.6+/-7.8)% for the total and (19.6+/-6.5)% for the extensive. Thirty-eight patients survived with a 2-year actual overall rate of survival 59.0% and 78.9% in the high-risk and standard-risk group, respectively. The 3-year probability of LFS was (58.6+/-8.0)% and (68.4+/-10.7)%, respectively.


CONCLUSIONS
The results encourage extending haploidentical HSCT without T-cell depletion treatments to children with an indication for transplantation.
Liu
Daihong
D
Peking University People's Hospital; Institute of Hematology, Peking University, Beijing 100044, China.
Liu
Kai-yan
KY
Xu
Lan-ping
LP
Chen
Huan
H
Chen
Yu-hong
YH
Han
Wei
W
Wang
Yu
Y
Zhang
Xiao-hui
XH
Jiang
Qian
Q
Huang
Xiao Jun
XJ
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005190:Family; D005260:Female; D006086:Graft vs Host Disease / Q000188:drug therapy; D006239:Haplotypes; D019337:Hematologic Neoplasms / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006648:Histocompatibility; D006801:Humans; D007223:Infant; D008297:Male; D016019:Survival Analysis; D013601:T-Lymphocytes / Q000276:immunology*; D016896:Treatment Outcome