Objective: To evaluate the outcomes of human leukocyte antigen (HLA) matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) for adult acute myeloid leukemia (AML) in a single center. Methods: Consecutive adult AML who received MUD-HSCT in our center from January 2008 to April 2017 were studied retrospectively, comparing with patients undergoing matched sibling donor (MSD) -HSCT in the same period. The rates of overall survival (OS) , disease free survival (DFS) , relapse, non-relapse mortality (NRM) , engraftment, acute and chronic graft-versus-host disease (aGVHD and cGVHD) were analyzed. Results: A total of 247 consecutive cases were enrolled, including 46 patients with MUD-HSCT and 201 with MSD-HSCT. All the patients experienced neutrophil engraftment except for one patient who died early in the MSD group, but the median day of engraftment was longer in the MUD group (15.0 vs 14.0, P=0.017) . The accumulative engraftment rate of platelet was comparable between the two groups (93.5%vs 98.0%, P=0.128) . The accumulative incidences of aGVHD (50.0%vs 46.3%, P=0.421) and cGVHD (37.8%vs 43.0%, P=0.581) were not statistically different between the two groups. Compared with the MSD group, the accumulative NRM rate at+36 months after transplantation was significantly higher in the MUD group (22.0%vs 10.4%, P=0.049) , while the relapse rate was not statistical difference (20.5 vs 28.3%, P=0.189) . Both the 3-year OS (61.6%vs 63.3%, P=0.867) and DFS (57.5%vs 61.6%, P=0.760) were comparable between the two groups. Four independent risk factors were confirmed by the multivariate analysis: patient age ≥45 years old, CR2 or NR before transplantation, a history of extramedullary infiltration and the occurrence of grade Ⅲ-Ⅳ aGVHD. No statistical differences were demonstrated in the survival rate between MUD-and MSD-HSCT in different subgroups. Conclusions: The outcomes, such as GVHD, relapse, OS and DFS, were comparable between MUD-and MSD-HSCT for adult AML, but higher incidence of NRM and longer time to neutrophil engraftment in the MUD group. MUD-HSCT is practical and feasible for adult AML who are lack of MSD.
目的： 观察人类白细胞抗原（HLA）匹配的无关供者造血干细胞移植（MUD-HSCT）治疗成人急性髓系白血病（AML）的疗效。 方法： 回顾性分析2008年1月至2017年4月接受MUD-HSCT的成人AML病例资料，并以同期同胞全相合供者移植（MSD-HSCT）病例为对照，分析两组患者总生存（OS）率、无病生存（DFS）率、复发率、非复发死亡率（NRM）、植活率及急慢性移植物抗宿主病（aGVHD、cGVHD）发生率的差异。 结果： 共247例连续性病例入组，MUD组46例，MSD组201例。除1例MSD组患者早期死亡外，两组患者均获粒细胞植活，但MUD组中位植活时间长于MSD组（15 d对14 d，P=0.017），血小板累积植活率差异无统计学意义（93.5%对98.0%，P=0.128）。两组患者aGVHD、cGVHD累积发生率差异无统计学意义（50.0%对46.3%，P=0.421；37.8%对43.0%，P=0.581）。与MSD组相比，MUD组患者3年累积NRM显著升高（22.0%对10.4%，P=0.049），但累积复发率差异无统计学意义（20.5%对28.3%，P=0.189）。两组患者3年OS率和DFS率差异均无统计学意义（61.6%对63.3%，P=0.867；57.5%对61.6%，P=0.760）。经多因素分析发现，患者年龄≥45岁、移植前第2次完全缓解（CR(2)）及以上或未缓解状态、有髓外浸润病史及Ⅲ~Ⅳ度aGVHD是影响生存的独立危险因素。 结论： 与MSD-HSCT相比，MUD-HSCT治疗成人AML的NRM较高，粒细胞植活中位时间较长，但GVHD发生率、复发率、OS率及DFS率并无显著差异，是缺乏MSD的成人AML患者的理想替代移植方案。.
Keywords: Acute myeloid leukemia; Hematopoietic stem cell transplantation; Sibling donor; Unrelated donor.