G-CSF-Mobilized Blood and Bone Marrow Grafts as the Source of Stem Cells for HLA-Identical Sibling Transplantation in Patients with Thalassemia Major
G-CSF-Mobilized Blood and Bone Marrow Grafts as the Source of Stem Cells for HLA-Identical Sibling Transplantation in Patients with Thalassemia Major
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G-CSF 动员的血液和骨髓移植物作为重型地中海贫血患者 HLA 相同同胞移植的干细胞来源
DOI：
10.1016/j.bbmt.2019.06.009
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发表时间：
2019-10-01
影响因子：
4.3
通讯作者：
Lai, Yongrong
中科院分区：
文献类型：
--
作者：
Li, Qiaochuan;Luo, Jianming;Lai, Yongrong
作为一种遗传性贫血，重型地中海贫血（TM）目前只能通过同种异体造血干细胞移植（allo-HSCT）来治愈。在这里，我们报告了一种针对 TM 患者的异基因 HSCT 方案，这些患者接受了来自匹配的同胞供体 (MSD) 的粒细胞集落刺激因子引发的骨髓和外周血干细胞 (G-BM 和 PBSC) 的组合。调理方案包括静脉注射。白消安、环磷酰胺、氟达拉滨和抗胸腺细胞球蛋白。对所有患者进行嵌合分析。如果怀疑有排斥反应，则终止免疫抑制治疗，一旦没有观察到反应，则进行供体淋巴细胞输注。 2007年7月至2018年7月期间共有184名TM患者入组。II-IV级急性移植物抗宿主病（GVHD）的累积发生率为13.1%，中重度慢性GVHD的累积发生率为5.7%。移植物排斥的累积发生率为0.6%。在整个队列中，3年总生存率、无地中海贫血生存率、无GVHD、无复发生存率分别为97.8%、97.3%和89.5%。总的来说，我们的结果表明，来自 MSD 的 G-BM 和 PBSC 是接受异基因造血干细胞移植的 TM 患者的良好干细胞来源。 (C) 2019 年美国移植和细胞治疗学会。由爱思唯尔公司出版
As an inherited anemia, thalassemia major (TM) is currently only curable with allogeneic hematopoietic stem cell transplantation (allo-HSCT). Here we report an allo-HSCT protocol for patients with TM who received a combination of granulocyte colony-stimulating factor-primed bone marrow and peripheral blood stem cells (G-BM & PBSCs) from a matched sibling donor (MSD). The conditioning regimen consisted of i.v. busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin. Chimerism analysis was performed for all patients. Immunosuppressive treatment was terminated if rejection was suspected, and donor lymphocyte infusion was administered once no response was observed. A total of 184 patients with TM were enrolled in the study between July 2007 and July 2018. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 13.1%, and that of moderate or severe chronic GVHD was 5.7%. The cumulative incidence of graft rejection was .6%. In the total cohort, the 3-year overall survival, thalassemia-free survival, and GVHD-free, relapse-free survival were 97.8%, 97.3%, and 89.5%, respectively. Collectively, our results indicate that G-BM & PBSCs from an MSD is be a good stem cell source for patients with TM undergoing allo-HSCT. (C) 2019 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.