β地中海贫血的异基因外周血干细胞移植

Allogeneic peripheral blood stem cell transplantation in beta-thalassemia.

作者信息

Fang Jianpei, Huang Shaoling, Chen Chun, Zhou Dunhua, Wu Yanfeng, Bao Rong

机构信息

Department of Pediatrics, Sun Yat-sen Memorial Hospital, Zhong Shan (Sun Yat-sen) University, Guangzhou, People's Republic of China.

出版信息

Pediatr Hematol Oncol. 2002 Oct-Nov;19(7):453-8. doi: 10.1080/08880010290097260.

DOI:10.1080/08880010290097260

PMID:12217190

Abstract

Six transfusion-dependent beta-thalassemia major patients were treated with allogeneic peripheral blood stem cell (PBSC) transplant. The donors were HLA identical siblings except one donor who was a father with one-antigen mismatch of HLA-B loci. The donors were mobilized with G-CSF and PBSC was infused without manipulation. Engraftment was documented in all patients. Acute graft versus host disease (GvHD) was present in 4 patients but could be controlled with steroid or/and ATG. One patient died of hepatic veno-occlusive disease (HVOD) and survivors were all transfusion independent (ex-thalassemia). Chronic GvHD occurred in one patient. Allogeneic PBSC transplantation could achieve disease-free survival in beta-thalassemia major patients.

摘要

六名依赖输血的重型β地中海贫血患者接受了异基因外周血干细胞（PBSC）移植治疗。供者均为人类白细胞抗原（HLA）相合的同胞，只有一名供者是父亲，其HLA - B位点有一个抗原不匹配。供者用粒细胞集落刺激因子（G - CSF）动员，外周血干细胞未经处理即进行输注。所有患者均证实有造血干细胞植入。4例患者出现急性移植物抗宿主病（GvHD），但可用类固醇或/和抗胸腺细胞球蛋白（ATG）控制。1例患者死于肝静脉闭塞病（HVOD），其余存活者均不再依赖输血（摆脱地中海贫血）。1例患者发生慢性移植物抗宿主病。异基因外周血干细胞移植可使重型β地中海贫血患者实现无病生存。