Objective: To compare the clinical efficacy of umbilical cord blood transplantation (UCBT) and hematopoietic stem cell transplantation from HLA-matched sibling donors (MSD-HSCT) in the treatment of myelodysplastic syndrome-EB (MDS-EB) or acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) . Methods: A cohort of 64 patients (including 38 cases of MDS-EB and 26 cases of AML-MRC) who received UCBT/MSD-HSCT from February 2011 to December 2017 were retrospectively analyzed. Results: ①Compared with MSD-HSCT group, UCBT group had a higher proportion of AML-MRC patients [52.8% (19/36) vs 25.0% (7/28) , P=0.025], and a lower median age [13 (1.5-52) years vs 32 (10-57) years, P=0.001]. ②The engraftment of neutrophils both in UCBT and MSD-HSCT groups on +42 d was 100%, and the median engraftment time was 17.5 (11-31) d and 11.5 (10-20) d, respectively. The engraftment of platelet at +100 d in UCBT group was 91.4%, the median engraftment time was 40 (15-96) d; The engraftment of platelet at +100 d in MSD-HSCT group was 100%, and the median engraftment time was 15 (11-43) d. ③There were no statistically significant differences in terms of the cumulative incidence of Ⅱ-Ⅳ and Ⅲ/Ⅳ aGVHD of 100 d and transplant related mortality (TRM) of 180 d, relapse rate, overall survival (OS) , disease-free survival (DFS) between UCBT and MSD-HSCT groups (P>0.05) . ④The 3-year cumulative incidence of chronic GVHD (cGVHD) and severe chronic GVHD in UCBT group were lower than of MSD-HSCT group [28.3% (95%CI 13.4%-45.3%) vs 67.9% (95%CI 46.1%-82.4%) , P=0.002; 10.3% (95%CI 2.5%-24.8%) vs 50.0% (95%CI 30.0%-67.1%) , respectively, P<0.001]. The cumulative 3-year incidence of GVHD-free and relapse-free survival (GRFS) of UCBT group was significantly higher than of MSD-HSCT group [55.0% (95%CI 36.0%-70.6%) vs 28.6% (95%CI 13.5%-45.6%) , P=0.038]. Conclusion: UCBT could obtain better quality of life after transplantation than MSD-HSCT in treatment of MDS-EB/AML-MRC.
目的： 比较非血缘脐血干细胞移植（UCBT）与同胞HLA全相合供者造血干细胞移植（MSD-HSCT）治疗骨髓增生异常综合征伴原始细胞增多（MDS-EB）和急性髓系白血病伴骨髓增生异常相关改变（AML-MRC）的临床疗效。 方法： 回顾性分析2011年2月至2017年12月接受UCBT/MSD-HSCT的MDS-EB/AML-MRC患者64例，其中MDS-EB 38例，AML-MRC 26例。 结果： ①与MSD-HSCT组比较，UCBT组AML-MRC患者比例较高[52.8%（19/36）对25.0%（7/28），P=0.025]，中位年龄较低[13（1.5~52）岁对32（10~57）岁，P=0.001]。②UCBT组与MSD-HSCT组+42 d粒细胞植入率均为100%，中位植入时间分别为17.5（11~31）d、11.5（10~20）d；UCBT组+100 d血小板植入率为91.4%，中位植入时间为40（15~96）d，MSD-HSCT组+100 d血小板植入率为100.0%，中位植入时间为15（11~43）d。③UCBT组和MSD-HSCT组比较，+100 dⅡ~Ⅳ度、Ⅲ/Ⅳ度急性GVHD累积发生率、180 d移植相关死亡率、3年累积复发率、3年总生存率和3年无病生存率差异均无统计学意义（P>0.05）。④UCBT组3年慢性GVHD、重度慢性GVHD的累积发生率均低于MSD-HSCT组[28.3%（95% CI 13.4%~45.3%）对67.9%（95%CI 46.1%~82.4%），P=0.002；10.3%（95%CI 2.5%~24.8%）对50.0%（95%CI 30.0%~67.1%），P<0.001]；UCBT组3年无严重急慢性GVHD及无复发生存（GRFS）率明显高于MSD-HSCT组[55.0%（95%CI 36.0%~70.6%）对28.6%（95%CI 13.5%~45.6%），P=0.038]。 结论： UCBT治疗MDS-EB/AML-MRC患者可获得比MSD-HSCT更好的移植后生存质量。.
Keywords: Cord blood stem cell transplantation; Hematopoietic stem cell transplantation; Leukemia, myeloid, acute; Myelodysplastic syndrome; Survival analysis.