Ai Hui-sheng, Huang Xiao-jun, Qiao Zhen-hua, Wang Jian-min, Chen Bao-an, Bai Hai, Shi Bao-fu, Liang Ying-min, Sun Wan-jun
Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing 100085, China.
Zhonghua Xue Ye Xue Za Zhi. 2009 Aug;30(8):505-8.
To observe the treatment effect and toxicity of nonmyeloablative allogeneic stem cell transplantation (NST) for hematologic diseases.
A total of 243 hematologic diseases patients received HLA-identical NST were enrolled in this study from 9 transplant centers of NST Cooperative Group in China. Nonmyeloablative conditioning regimen was based on fludarabine (Flud), rabbit anti-human thymocyte globulin (ATG), cyclophosphamide (CTX) (FAC), and plus cytarabine or busulfan (BU) etc. Graft-versus-host disease (GVHD) prophylaxis included cyclosporin A (CsA) and mycophenolate mofetil (MMF).
Among the 243 patients, 219 (90.1%) achieved full donor chimerism (FDC), 2(0.8%) engraftment failure. 78 (32.1%) had mixture chimerism (MC) at 4 weeks after NST, out of which 56 switched to FDC, 16 remained MC and 6 (2.5%) developed graft rejection. The incidence of acute GVHD was 34.2%, including 6.6% of grade III-IV acute GVHD. Chronic GVHD developed in 78 (32.1%) patients. The follow-up durations were 3 - 99 months, 162 (66.7%) were still alive and the overall survival rates were 76.5%, 73.9%, 70.7%, and 27.8% for MDS/SAA, chronic myeloid leukemia, acute leukemia at first remission, and refractory or relapsed leukemia, respectively.
The nonmyeloablative allogeneic stem cell transplantation based on FAC conditioning results in sustained engraftment and mild aGVHD, providing a new feasible curative therapy for hematology diseases.
观察非清髓性异基因干细胞移植（NST）治疗血液病的疗效及毒性。
本研究纳入了中国NST协作组9个移植中心共243例接受HLA全相合NST的血液病患者。非清髓性预处理方案基于氟达拉滨（Flud）、兔抗人胸腺细胞球蛋白（ATG）、环磷酰胺（CTX）（FAC），并加用阿糖胞苷或白消安（BU）等。移植物抗宿主病（GVHD）预防包括环孢素A（CsA）和霉酚酸酯（MMF）。
243例患者中，219例（90.1%）实现完全供者嵌合（FDC），2例（0.8%）植入失败。78例（32.1%）在NST后4周出现混合嵌合（MC），其中56例转为FDC，16例仍为MC，6例（2.5%）发生移植物排斥。急性GVHD发生率为34.2%，其中Ⅲ-Ⅳ级急性GVHD为6.6%。78例（32.1%）患者发生慢性GVHD。随访时间为3 - 99个月，162例（66.7%）仍存活，MDS/SAA、慢性髓性白血病、首次缓解期急性白血病、难治或复发白血病的总生存率分别为76.5%、73.9%、70.7%和27.8%。
基于FAC预处理的非清髓性异基因干细胞移植可实现持续植入且急性GVHD较轻，为血液病提供了一种新的可行的治愈性疗法。
