Objective: To evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation (allo-HSCT) with decitabine (Dec)-conditioning regimen in the treatment of myelodysplastic syndrome (MDS) and MDS transformed acute myeloid leukemia (MDS-AML).
Methods: The characteristics and efficacy data of 93 patients with MDS and MDS-AML who received allo-HSCT in our center from April 2013 to November 2021 were retrospectively analyzed. All patients were administered by myeloablative conditioning regimen containing Dec (25 mg/m2 /d×3 d).
Results: Among the 93 patients, 63 males and 30 females, were diagnosed as MDS（n ＝77）, MDS-AML（n ＝16）. The incidence of I/II grade regimen-related toxicity (RRT) was 39.8%, and III grade RRT was only found in 1 patient (1%). Neutrophil engraftment was successful in 91 (97.8%) patients after a median neutrophil engraftment time of 14 (9-27) days; Successful platelet engraftment was achieved in 87 (93.5%) patients, with a median engraftment time of 18 (9-290) days. The incidence of acute graft versus host disease（aGVHD） and grade III-IV aGVHD was 44.2% and 16.2%, respectively. The incidence of chronic graft versus host disease（cGVHD） and moderate-to-severe cGVHD was 59.5% and 37.1%, respectively. Of the 93 patients, 54 (58%) developed posttransplant infections, among which lung infection (32.3%) and bloodstream infection (12.9%) were the most common. The median follow-up after transplantation was 45 (0.1-108) months. The 5-year overall survival (OS) rate, disease-free survival (DFS) rate, treatment-related mortality, and cumulative incidence of relapse were 72.7%, 68.4%, 25.1%, and 6.5%, respectively. And the 1-year graft-versus-host disease/relapse-free survival rate was 49.3%. The patients in different group of relative high-risk prognostic scoring or low-risk prognostic scoring, with or without poor-risk mutation(s), with mutations number ≥3 or <3 had similar 5-year OS rate (more than 70%). Multivariate analysis showed that the incidence of grade III-IV aGVHD was the independent risk factor affecting OS（P =0.008）and DFS (P =0.019).
Conclusion: Allo-HSCT with Dec-conditioning regimen is feasible and effective in the treatment of patients with MDS and MDS-AML, especially those in high prognostic risk and with poor-risk mutations.
题目: 含地西他滨预处理方案的allo-HSCT治疗MDS和MDS-AML的疗效及安全性.
目的: 评估含地西他滨（Dec）预处理方案的异基因造血干细胞移植（allo-HSCT）治疗骨髓增生异常综合征（MDS）和MDS转化的急性髓系白血病（MDS-AML）患者的疗效及安全性。.
方法: 回顾性分析2013年4月至2021年11月本中心收治的接受allo-HSCT治疗的93例MDS和MDS-AML患者的病例特征及疗效。所有患者均予以含Dec（25 mg/m2 /d× 3 d）的清髓预处理方案。.
结果: 93例患者中男63例、女30例；MDS 77例，MDS-AML 16例。I/II 级预处理相关毒性反应（RRT）发生率为39.8％，仅1例（1%）患者发生 III级 RRT。91例（97.8%）患者粒系植入成功，中位植入时间为 14（9-27）d；87例（93.5％）患者血小板植入成功，中位植入时间为18（9-290）d。急性移植物抗宿主病（aGVHD）、Ⅲ/Ⅳ度aGVHD发生率分别为44.2%和16.2%；慢性GVHD（cGVHD）、中重度cGVHD 发生率分别为59.5%和37.1%。93例患者中54例（58%）发生移植后感染，其中肺部感染（32.3%）和血流感染（12.9%）为最常见。移植后中位随访45（0.1-108）个月，5年总生存（OS）率、无病生存（DFS）率、治疗相关死亡（TRM）率、累计复发（CIR）率分别为 72.7%、68.4%、25.1%和6.5%，1年无移植物抗宿主病/无复发生存率为49.3%。预后评分相对高危组或相对低危组，伴或不伴不良预后基因突变组, 伴有突变数量≥3个或<3个组患者的5年OS率均相似（＞70%）。多因素分析结果显示，发生Ⅲ-Ⅳ度aGVHD为影响患者OS（P =0.008）、DFS（P =0.019）的独立危险因素。.
结论: 含Dec预处理方案的allo-HSCT治疗MDS和MDS-AML，特别对于高危患者和伴不良预后突变的患者是可行且有效的。.
Keywords: myelodysplastic syndrome; allogeneic hematopoietic stem cell transplantation; conditioning regimen; decitabine.