Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan 430022, China.
Bone Marrow Transplant. 2017 Sep;52(9):1253-1260. doi: 10.1038/bmt.2017.100. Epub 2017 Jun 5.
We designed a novel haploidentical hematopoietic stem cell transplantation (haplo-HSCT) system using idarubicin (IDA) intensified conditioning regimens and combination of antithymocyte globulin and basiliximab for GvHD prophylaxis. The outcomes of 110 high-risk acute leukemia patients undergoing haplo-HSCT were compared with 69 contemporaneous high-risk patients receiving HLA-matched sibling transplantation using uniform IDA-intensified regimens. The relapse incidence of haplo-HSCT was 23.4%, and 3-year overall survival (OS) and disease-free survival (DFS) achieved 62.9%, 59.1%, respectively. The cumulative incidences of II-IV and III-IV aGvHD were 28.6 and 14.3%, while limited and extensive cGvHD were 19.4, 13.8%. All these results were equivalent to those of concurrent identical sibling transplantation. Three-year OS and DFS for patients in advance stage reached 48.5, 47.3%. Furthermore, the relapse, 3-year OS of positive minimal residual disease (MRD) patients did not differ from negative MRD patients (18.9% vs 11.5%, 63.6% vs 69.6%), indicating our intensified haplo-HSCT technique could circumvent the dismal prognosis of MRD. These data provide reinforcing evidence that our haplo-HSCT system could dramatically improve the survival of high-risk acute leukemia with low relapse and acceptable transplantation-related mortality, and might be a promising therapeutic option for high-risk patients.
我们设计了一种新型的单倍体造血干细胞移植（haplo-HSCT）系统，使用伊达比星（IDA）强化预处理方案和抗胸腺细胞球蛋白与巴利昔单抗联合预防移植物抗宿主病（GVHD）。我们将 110 例高危急性白血病患者接受 haplo-HSCT 的结果与 69 例同期接受 HLA 匹配的同胞移植患者进行了比较，这些患者使用了相同的 IDA 强化方案。haplo-HSCT 的复发率为 23.4%，3 年总生存率（OS）和无病生存率（DFS）分别达到 62.9%和 59.1%。II-IV 和 III-IV 级急性 GVHD 的累积发生率分别为 28.6%和 14.3%，而局限性和广泛性慢性 GVHD 的累积发生率分别为 19.4%和 13.8%。所有这些结果都与同期相同的同胞移植相当。进展期患者的 3 年 OS 和 DFS 分别达到 48.5%和 47.3%。此外，阳性微小残留病（MRD）患者的复发和 3 年 OS 与阴性 MRD 患者无差异（18.9% vs 11.5%，63.6% vs 69.6%），表明我们的强化 haplo-HSCT 技术可以规避 MRD 不良预后。这些数据提供了有力的证据，表明我们的 haplo-HSCT 系统可以显著改善高危急性白血病患者的生存，复发率低，移植相关死亡率可接受，可能是高危患者的一种有前途的治疗选择。
