Haploidentical hematopoietic stem cell transplantation for beta-thalassemia major in children / 中国当代儿科杂志
Chinese Journal of Contemporary Pediatrics
;
(12): 546-548, 2009.
Article
in Chinese
| WPRIM
| ID: wpr-304655
ABSTRACT
<p><b>OBJECTIVE</b>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.</p><p><b>METHODS</b>Between November 2001 and November 2007, 10 patients with beta-thalassemia major at median ages of 4.4 years (range1.6-9.4 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.</p><p><b>RESULTS</b>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).</p><p><b>CONCLUSIONS</b>Haploidentical stem cell transplantation is an alternative option for children with beta-thalassemia major, particularly when a matched sibling donor is not available.</p>
Full text:
Available
Index:
WPRIM (Western Pacific)
Main subject:
Therapeutics
/
Haplotypes
/
Histocompatibility Testing
/
Follow-Up Studies
/
Beta-Thalassemia
/
Hematopoietic Stem Cell Transplantation
/
Graft vs Host Disease
Type of study:
Observational study
/
Prognostic study
Limits:
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
Language:
Chinese
Journal:
Chinese Journal of Contemporary Pediatrics
Year:
2009
Type:
Article
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