Department of Hematology, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China.
Department of Pediatrics, The First Affiliated Hospital of Guangxi Medical University, Nanning, Guangxi, China.
Biol Blood Marrow Transplant. 2019 Oct;25(10):2040-2044. doi: 10.1016/j.bbmt.2019.06.009. Epub 2019 Jun 15.
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.
作为一种遗传性贫血，重型地中海贫血（TM）目前只能通过异基因造血干细胞移植（allo-HSCT）治愈。在这里，我们报告了一种 allo-HSCT 方案，用于接受来自匹配同胞供体（MSD）的粒细胞集落刺激因子预处理的骨髓和外周血干细胞（G-BM 和 PBSCs）的 TM 患者。预处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。如果怀疑排斥反应，应终止免疫抑制治疗，如果观察到无反应，则给予供者淋巴细胞输注。2007 年 7 月至 2018 年 7 月期间，共有 184 例 TM 患者入组该研究。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 6%。在总队列中，3 年总生存率、无地中海贫血生存率和无 GVHD、无复发生存率分别为 97.8%、97.3%和 89.5%。总之，我们的结果表明，来自 MSD 的 G-BM 和 PBSCs 是接受 allo-HSCT 的 TM 患者的良好干细胞来源。
