[Haploidentical hematopoietic stem cell transplantation for beta-thalassemia major in children].

Abstract

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.

• Authors: Wen-Ge Hao, Xin Sun, Sha Liu, Zhe Zhao, Zhan-Xi Chen
• Journal: Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics (Zhongguo Dang Dai Er Ke Za Zhi) Vol. 11 Issue 7 Pg. 546-8 (Jul 2009) ISSN: 1008-8830 [Print] China
• PMID: 19650987 (Publication Type: English Abstract, Journal Article)

Topics

• Child
• Child, Preschool
• Female
• Follow-Up Studies
• Graft vs Host Disease (etiology)
• Haplotypes
• Hematopoietic Stem Cell Transplantation (adverse effects)
• Histocompatibility Testing
• Humans
• Infant
• Male
• beta-Thalassemia (therapy)