亲缘单倍体相合外周血造血干细胞移植治疗重症再生障碍性贫血

Haploidentical peripheral blood hematopoietic stem cell transplantation for treatment of severe aplastic anemia

目的探讨改良白消安/环磷酰胺+抗胸腺细胞球蛋白(Bu/Cy+ATG)预处理方案在亲缘单倍体造血干细胞移植治疗重症再生障碍性贫血(SAA)临床应用中的有效性和安全性。方法回顾分析河北唐山钢铁集团有限责任公司医院血液肿瘤科自2009年10月至2011年5月间采用亲缘单倍体外周血干细胞移植治疗的3例SAA患者资料。供者均为母亲,1例HLA3/6位点相合,2例HLA4/6位点相合。预处理方案均为改良Bu/Cy+ATG,具体为白消安0.8mg/kg,每天4次,连用2d;环磷酰胺50mg.kg-1.d-1,连用4d;抗胸腺细胞球蛋白2.5mg.kg-1.d-1,连用4d。环孢素+短程甲氨蝶呤+吗替麦考酚酯预防排斥反应。结果 3例患者均达完全供者植入,2例合并Ⅱ～Ⅲ度急性移植物抗宿主病(GVHD),1例患者合并局限型慢性GVHD。3例患者均发生血CMV感染,经抗病毒治疗均得以控制。随访5～25个月,3例患者至今均无病存活。结论初步经验Bu/Cy+ATG预处理方案经改良用于亲缘单倍体外周血造血干细胞移植治疗SAA安全、有效。

Objective To investigate the efficacy and safety of modified busulfan/ cyclophosphamide ＋ antithymocyte globulin （ Bu/Cy ＋ ATG） conditioning regimen to treat severe aplastic anemia （SAA）. Methods Retrospective analysis was performed on 3 patients with SAA treated by modified Bu/Cy ＋ ATG conditioning regimen before related haploidentical hematopoietic stem cell transplantation. Donors were their mothers, with 3/6 HLA matches in 1 patient and 4/6 matches in the other 2 patients. Bu/Cy ＋ ATG conditioning regimen contained busulfan 0.8 mg/kg, 4 times per day for 2 d, cyclophosphamide 50 mg · kg ·d- 1 for 4 d, and antithymocyte globulin 2.5 mg · kg ·d- 1 for 4 d. Cyclosporine, methotrexate, and mycophenolate were used to prevent graft-versus-host disease （GVHD）. Results All 3 patients achieved full donor type engraftment. Two suffered from grade 11 - III acute GVHD. One suffered from limited chronic GVHD. Three patients were infected with CMV, but the condition was effectively controlled by antiviral treatments. With 5-25 month follow-up, all 3 patients survived. Conclusion Preliminary results showed that the modified Bu/Cy ＋ ATG conditioning regimen used to treat SAA with haploidentical hematopoietic stem cell transplantation was safe and effective.