【摘要】 目的 探讨异基因造血干细胞移植（allo-HSCT）治疗骨髓增生异常综合征（MDS）及MDS转化急性髓系白血病（MDS-AML）的疗效及安全性。方法回顾性分析2015年5月至2022年3月38例MDS和13例MDS-AML患者的临床资料，51例患者全部接受异基因造血干细胞移植，其中同胞全合移植17例，单倍体移植33例，无关供者移植1例。总结治疗过程及转归信息，分析患者移植前疾病状态、供者选择、干细胞来源、预处理方案、移植物抗宿主病（GVHD）等对移植疗效的影响。结果 49例（96.1%）患者移植后获得造血重建，中性粒细胞中位植活时间11(10, 12)d，血小板中位植活时间14(12, 19)d。Ⅱ～Ⅳ度急性移植物抗宿主病（aGVHD）及慢性移植物抗宿主病（cGVHD）发生率分别为14.3%和34.7%。截至随访日期，15例（29.4%）患者死亡，中位死亡时间为移植后21(7,44.5)个月，死亡原因包括肺部感染（6例）、复发（5例）、GVHD(3例)、脓毒血症（1例）。移植相关死亡（TRM）为19.6%，移植后100天内TRM为7.8%。中位随访时间7(2, 26)个月，3年预计总生存率（OS）为（64.2±7.7）%，预计无复发生存率（RFS）为（59.4±8.4）%，预计复发率为（13.7±3.9）%。MDS组与MDS-AML组比较，3年预计OS率分别为（69.9±8.9）%,(50.5±14.3)%(P=0.02)；预计RFS率分别为（67.7±9.1）%,(35.9±17.3)%(P=0.03)；预计复发率分别为（7.9±2.6）%,(30.8±7.7)%(P=0.01)。Kaplan-Meier单因素分析显示预处理方案中加用地西他滨和不加地西他滨患者3年预计OS率分别为（63.7±6.3）%和（34.9±13.1）%(P=0.007)。Cox比例风险回归多因素分析显示MDS进展为AML可能与OS有关（HR 0.326, 95%CI 0.107～0.993, P=0.033）。结论 Allo-HSCT是治疗MDS和MDS-AML有效方法，MDS转化为AML后移植疗效差，对于存在移植指征的MDS患者应尽早行allo-HSCT。更多还原

【Abstract】 Objective To investigate the efficacy and safety of allogeneic hematopoietic stem cell transplantation(allo-HSCT in the treatment of myelodysplastic syndrome(MDS) and MDS-transformed acute myeloid leukemia(MDS-AML).Methods The clinical data of 38 patients with MDS and 13 patients with MDS-AML from May 2015 to March 2022 were summarized. All 51 patients received allogeneic hematopoietic stem cell transplantation, including 17 matched sibling donor transplantation, 33 haploid transplantation and 1 unrelated donor transplantation. The treatment process and outcome were retrospectively analyzed. The effects of disease status, donor selection, source of stem cells, conditioning regimen and GVHD on the transplantation efficacy were also analyzed. Results 49 of 51 patients(96.1%) got hematopoietic reconstitution. The median time of granulocyte engraftment was 11(10, 12) days, and the median time of platelet engraftment was 14(12, 19) days. The incidences of grade Ⅱ-Ⅳ acute graft versus host disease(aGVHD) and chronic GVHD were 14.3% and 34.7%, respectively. By the follow-up date, 15 patients(29.4%) died with a median survival time of 21(7, 44.5) months after transplantation. The causes of death include pulmonary infection(6 cases), disease relapse(5 cases), GVHD(3 cases), and sepsis(1 cases). The 100 days and total transplant-related mortality(TRM) rates were 7.8% and 19.6%, respectively. The median follow-up was 7(2, 26) months.The 3-year estimated overall survival rate(OS), the relapse free survival rate(RFS) and relapse rate were(64.2±7.7)%,(59.4±8.4)% and(13.7±3.9)%, respectively. By comparison between MDS group and MDS-AML group, the 3-year estimated OS rate was(69.9±8.9)% and(50.5±14.3)%(P=0.02), the 3-year estimated RFS rate was(67.7±9.6)% and(35.9±17.3)%(P=0.03), and the relapse rate was(7.9±2.6)% and(30.8±7.7)%(P=0.01). Kaplan-Meier univariate analysis revealed the 3-year estimated OS rate in the conditioning regimen with and without dexcitabine was(63.7±6.3)% and(34.9±13.1)%(P=0.007), respectively. Cox multivariate analysis suggested transformation from MDS to AML might be associated with OS(HR 0.326, 95% CI 0.107-0.993, P=0.033). Conclusion Allo-HSCT is an effective method to treat MDS and MDS-AML. The efficacy of allo-HSTC is poor after MDS transforms into acute myeloid leukemia. For patients with allo-HSCT indications, transplantation should be carried out as early as possible.更多还原