外周血造血干细胞移植治疗3例重型β地中海贫血和1例特发性溶血性贫血

目的探讨异基因外周血造血干细胞移植(allogeneicperipheral blood stem cell transplantation,Allo-PBSCT)治疗遗传性溶血性贫血(hereditaryhaemolytic

Allogeneic peripheral blood stem cell transplantation for hereditary 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. CONCLUSION: Allo-PBSCT is an effective and a new way to treat beta-thalassemia major and congenital idiopathic haemolytic anemia.