Fang J, Huang S, Chen C, Wu Y, Bao R, Huang K
Department of Pediatrics, Sun Yat-sen Memorial Hospital, Sun Yat-sen University of Medical Sciences, Guangzhou, Guangdong, 510120 P. R. China.
Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2001 Dec;18(6):452-5.
To examine the feasibility of allogeneic peripheral blood stem cell transplantation (Allo-PBSCT) for the hereditary haemolytic anemia (HHA).
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).
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.
Allo-PBSCT is an effective and a new way to treat beta-thalassemia major and congenital idiopathic haemolytic anemia.