Allogeneic Hematopoietic Stem Cell Transplantation with Decitabine-containing Regimen在骨髓增殖性疾病和骨髓增殖性疾病转化为急性髓细胞白血病中的功效和安全性。
[Efficacy and Safety of Allogeneic Hematopoietic Stem Cell Transplantation with Decitabine-containing Regimen in Myelodysplastic Syndromes and Myelodysplastic Syndromes Transformed Acute Myeloid Leukemia].
发表日期:2023 Apr
作者:
Yu-Xin Wang, Yao Sun, Jing Xie, Na Liu, Jiang-Wei Hu, Zhuo-Qing Qiao, San-Chun Lan, Long Zhao, Yang Yang, Yu-Hang Li, Bin Zhang, Liang-Ding Hu
来源:
Stem Cell Research & Therapy
摘要:
评估异基因造血干细胞移植(allo-HSCT)联合脱氧胞苷(Dec)预处理方案治疗骨髓增生异常综合症(MDS)和MDS转化为急性髓细胞白血病(MDS-AML)的疗效和安全性。回顾性分析了2013年4月至2021年11月在我们中心接受allo-HSCT治疗的93名MDS和MDS-AML患者的特征和疗效数据。所有患者均通过髓毒性预处理方案,包含Dec(25 mg/m2/d×3 d)。在93名患者中,有63名男性和30名女性,被诊断为MDS(n=77)和MDS-AML(n=16)。I/II级方案相关毒性(RRT)的发生率为39.8%,III级RRT仅在1名患者(1%)中发现。中位中性粒细胞植入时间为14(9-27)天,91名(97.8%)患者成功植入中性粒细胞;成功的血小板植入在87名(93.5%)患者中实现,中位植入时间为18(9-290)天。急性移植物抗宿主病(aGVHD)和III-IV级aGVHD的发生率分别为44.2%和16.2%。慢性移植物抗宿主病(cGVHD)和中度至重度cGVHD的发生率分别为59.5%和37.1%。在93名患者中,有54名(58%)发生移植后感染,其中肺部感染(32.3%)和血流感染(12.9%)最常见。移植后中位随访时间为45(0.1-108)个月。5年总生存率(OS)、无病生存率(DFS)、治疗相关死亡率和复发累积发生率分别为72.7%、68.4%、25.1%和6.5%。1年移植物抗宿主病/复发无病生存率为49.3%。不同相对高危预后评分组或低危预后评分组、带或不带劣势突变的患者、突变数≥3或<3的患者具有相似的5年OS率(超过70%)。多元分析显示,III-IV级aGVHD的发生率是影响OS(P=0.008)和DFS(P=0.019)的独立危险因素。allo-HSCT与Dec预处理方案在MDS和MDS-AML患者中的治疗可行性和疗效良好,特别是在高预后危险和带有劣势突变的患者中。
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).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).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).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.