[Allogeneic peripheral blood stem cell transplantation for 3 patients with beta-thalassemia major and 1 patient with congenital idiopathic haemolytic anemia].
OBJECTIVE
To examine the feasibility of allogeneic peripheral blood stem cell transplantation (Allo-PBSCT) for the hereditary haemolytic anemia (HHA).


METHODS
Allo-PBSCTs were performed on 3 patients with beta-thalassemia major and 1 patient with congenital idiopathic haemolytic anemia by the HLA-identical siblings' donors or 1 locus-mismatch parent. The harvested stem cell contained nucleated cells (4.7-14.4) X 10(8)/kg recipient weight, CD(34) (+) CD(38)(-) cells(4.8-32.9) X 10(6)/kg and colony-forming-unit-granulocyte-macrophages (CFU-GM)(3.22-9.8) X 10(5)/kg. The conditioning regimen consisted of busulfan, cyclophosphamide, melphalan, thiotepa, fludarabine and antithymocyte globulin (ATG).


RESULTS
All patients were engrafted and accomplished with the acute graft-versus-host disease (aGVHD). A patient had the controlled chronic GVHD. Three patients are survivals with ex-anemia; their Hb levels have been maintaining normality without transfusion. One died of the hepatic veno-occlusive disease.


CONCLUSIONS
Allo-PBSCT is an effective and a new way to treat beta-thalassemia major and congenital idiopathic haemolytic anemia.
Fang
J
J
Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University of Medical Sciences, Guangzhou, Guangdong, 510120 P. R. China. infor@gdtc.net
Huang
S
S
Chen
C
C
Wu
Y
Y
Bao
R
R
Huang
K
K
D000745:Anemia, Hemolytic, Congenital / Q000473:pathology / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006504:Hepatic Veno-Occlusive Disease / Q000209:etiology; D006801:Humans; D008297:Male; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000473:pathology / Q000628:therapy*