目的：评价异基因造血干细胞移植（allo-HSCT）治疗骨髓增生异常综合征伴骨髓增生异常（MDS-EB）的疗效及预后因素，并比较世界卫生组织（WHO）分类的不同亚型患者的预后。 ）2022。方法：纳入2006年10月至2022年12月在中国医学科学院血液病医院接受allo-HSCT的282例MDS-EB患者。 WHO 2022年诊断标准将MDS重新分为三组：伴1/2型原始细胞增殖的骨髓增生异常肿瘤（MDS-IB1/IB2，222例）、伴纤维化的MDS（MDS-f，41例）和伴双等位基因TP53的MDS突变（MDS-biTP53，19例）。对他们的临床数据进行回顾性分析。结果：①282例患者中位年龄46（15~66）岁，其中男性191例，女性91例。其中，MDS-EB1 和 MDS-EB2 分别为 118 例（42%）和 164 例（58%）。 ②282例患者中，256例（90.8%）移植后实现造血重建，其中11例（3.9%）和15例（5.3%）分别出现原发性和继发性着床功能障碍。移植后100 d急性移植物抗宿主病(GVHD)累计发生率为(42.6±3.0)%，其中Ⅱ～Ⅳ级急性GVHD累计发生率为(33.0±2.8)%。移植后1年慢性GVHD累积发生率为(31.0±2.9)%。移植后，128例（45.4%）、63例（22.3%）、35例（12.4%）和17例（6.0%）患者出现巨细胞病毒感染、菌血症、肺部真菌感染和EB病毒感染。 ③移植后中位随访时间为22.1（19.2-24.7）个月，3年总生存（OS）和无病生存（DFS）率为71.9%（95% CI 65.7%-78.6%）和 63.6%（95% CI 57.2% -70.7%）。 3年非复发死亡率（NRM）为17.9%（95% CI 13.9% -22.9%），3年累积复发率（CIR）为9.8%（95% CI 6.7% -13.7%） 。影响移植后OS的独立危险因素包括单核细胞核型（P=0.004，HR=3.26，95% CI 1.46-7.29）、造血干细胞移植并发症指数（HCI-CI）≥3分（P<0.001，HR） =2.86, 95% CI 1.72-4.75), 发生Ⅱ-Ⅳ级急性胃肠道GVHD(P<0.001, HR=5.94, 95% CI 3.50-10.10)。 ④MDS-IB1/IB2组移植后3年OS和DFS率优于MDS-biTP53组[OS：72.0%（95% CI 63.4% -80.7%）vs 46.4%（95% CI） CI 26.9%-80.1%), P=0.020; DFS：67.4%（95% CI 60.3% -75.3%）对比 39.7%（95% CI 22.3% -70.8%），P=0.015]。 3年CIR低于MDS-biTP53组[7.3% (95% CI 4.3% -11.4% ) vs 26.9% (95% CI 9.2% -48.5% )，P=0.004]。 MDS-IB1/IB2、MDS-f 和 MDS-biTP53 组移植后 3 年的 NRM 分别为 16.7% (95% CI 12.1% -22.1%)、20.5% (95% CI 9.4% -34.6%) ）和 26.3%（95% CI 9.1% -47.5%）（P=0.690）。结论：Allo-HSCT是治疗MDS-EB的有效方法，单体核型、HCI-CI、Ⅱ~Ⅳ级急性胃肠道GVHD是影响患者OS的独立危险因素。 WHO 2022分类有助于区分allo-HSCT在不同亚组患者中的疗效。 Allo-HSCT可以改善MDS-f患者的不良预后，但MDS-biTP53患者移植后复发的风险较高。

Objective: To evaluate the efficacy and prognostic factors of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with myelodysplastic syndrome accompanied by myelodysplasia (MDS-EB) and to compare the prognosis of different subtypes of patients classified by World Health Organization (WHO) 2022. Methods: A total of 282 patients with MDS-EB who underwent allo-HSCT at the Hematology Hospital of the Chinese Academy of Medical Sciences from October 2006 to December 2022 were included in the study. The WHO 2022 diagnostic criteria reclassified MDS into three groups: myelodysplastic tumors with type 1/2 of primitive cell proliferation (MDS-IB1/IB2, 222 cases), MDS with fibrosis (MDS-f, 41 cases), and MDS with biallelic TP53 mutation (MDS-biTP53, 19 cases). Their clinical data were retrospectively analyzed. Results: ① The median age of 282 patients was 46 (15-66) years, with 191 males and 91 females. Among them, 118 (42% ) and 164 (58% ) had MDS-EB1 and MDS-EB2, respectively. ②Among the 282 patients, 256 (90.8% ) achieved hematopoietic reconstruction after transplantation, with 11 (3.9% ) and 15 (5.3% ) having primary and secondary implantation dysfunctions, respectively. The cumulative incidence of acute graft-versus-host disease (GVHD) 100 days post-transplantation was (42.6±3.0) %, and the cumulative incidence of grade Ⅱ-Ⅳ acute GVHD was (33.0±2.8) %. The cumulative incidence of chronic GVHD 1 year post-transplantation was (31.0±2.9) %. Post-transplantation, 128 (45.4% ), 63 (22.3% ), 35 (12.4% ), and 17 patients (6.0% ) developed cytomegalovirus infection, bacteremia, pulmonary fungal infection, and Epstein-Barr virus infection. ③The median follow-up time post-transplantation was 22.1 (19.2-24.7) months, and the 3-year overall survival (OS) and disease-free survival (DFS) rates were 71.9% (95% CI 65.7% -78.6% ) and 63.6% (95% CI 57.2% -70.7% ), respectively. The 3-year non-recurrent mortality rate (NRM) is 17.9% (95% CI 13.9% -22.9% ), and the 3-year cumulative recurrence rate (CIR) is 9.8% (95% CI 6.7% -13.7% ). The independent risk factors affecting OS post-transplantation include monocyte karyotype (P=0.004, HR=3.26, 95% CI 1.46-7.29), hematopoietic stem cell transplantation complication index (HCI-CI) of ≥3 points (P<0.001, HR=2.86, 95% CI 1.72-4.75), and the occurrence of acute gastrointestinal GVHD of grade Ⅱ-Ⅳ (P<0.001, HR=5.94, 95% CI 3.50-10.10). ④The 3-year OS and DFS rates in the MDS-IB1/IB2 group post-transplantation were better than those in the MDS-biTP53 group [OS: 72.0% (95% CI 63.4% -80.7% ) vs 46.4% (95% CI 26.9% -80.1% ), P=0.020; DFS: 67.4% (95% CI 60.3% -75.3% ) vs 39.7% (95% CI 22.3% -70.8% ), P=0.015]. The 3-year CIR was lower than that of the MDS-biTP53 group [7.3% (95% CI 4.3% -11.4% ) vs 26.9% (95% CI 9.2% -48.5% ), P=0.004]. The NRM at 3 years post-transplantation in the MDS-IB1/IB2, MDS-f, and MDS-biTP53 groups were 16.7% (95% CI 12.1% -22.1% ), 20.5% (95% CI 9.4% -34.6% ), and 26.3% (95% CI 9.1% -47.5% ), respectively (P=0.690) . Conclusion: Allo-HSCT is an effective treatment for MDS-EB, with monomeric karyotype, HCI-CI, and grade Ⅱ-Ⅳ acute gastrointestinal GVHD as independent risk factors affecting the patient's OS. The WHO 2022 classification helps distinguish the efficacy of allo-HSCT in different subgroups of patients. Allo-HSCT can improve the poor prognosis of patients with MDS-f, but those with MDS-biTP53 have a higher risk of recurrence post-transplantation.