[Haploidentical hematopoietic stem cell transplantation for beta-thalassemia major in children].
OBJECTIVE
Hematopoietic stem cell transplantation is currently a unique curative therapy for beta-thalassemia major. However, only 30% of patients have HLA-identical siblings to serve as donors. This study investigated the feasibility of hematopoietic stem cell transplantation from HLA mismatched related donors for beta-thalassemia major in children.


METHODS
Between November 2001 and November 2007, 10 patients with beta-thalassemia major at median ages of 4.4 years (range:1.6-9.4 years) received 11 transplantations from their haploidentical donors, either HLA mismatched sibling umbilical cord bloods (n=6) or parents marrows (n=4) or sibling marrow (n=1). The conditioning regiment included fludarabine (100 mg/m2), busulfan (16 mg/kg), cyclophosphamide (200 mg/kg) and antithymocyte globulin.


RESULTS
Of the 10 patients, 6 (60%) had sustained engraftment and red blood cell transfusion independence; 2 patients showed transient engraftment but rejected the graft quickly; 1 patients had no evidence of engraftment and developed aplastic anemia; 1 patient who received two transplantations had no evidence of engraftment and developed persistent aplastic anemia. All eight engrafted patients showed grade I to III acute graft-versus-host disease (GVHD), and only one developed limited skin chronic GVHD. The probability of overall and disease-free survival was 90% and 60%, respectively, with a median follow-up duration of 57.1 months (range: 2.5 to 85.1 months).


CONCLUSIONS
Haploidentical stem cell transplantation is an alternative option for children with beta-thalassemia major, particularly when a matched sibling donor is not available.
Hao
Wen-Ge
WG
Department of Pediatrics, Guangzhou Women and Children's Medical Center, Guangzhou 510120, China. haowg1016@163.com
Sun
Xin
X
Liu
Sha
S
Zhao
Zhe
Z
Chen
Zhan-Xi
ZX
D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006086:Graft vs Host Disease / Q000209:etiology; D006239:Haplotypes; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D017086:beta-Thalassemia / Q000628:therapy*