含10天地西他滨预处理方案异基因造血干细胞移植治疗31例急性髓系白血病/骨髓增生异常综合征患者的疗效及安全性

Effect and safety of 10-day decitabine-containing conditioning regimen for allogeneic hematopoietic stem cell transplantation in 31 patients with acute myeloid leukemia/myelodysplastic syndrome

目的探讨含10天地西他滨预处理方案异基因造血干细胞移植(allo-HSCT)治疗急性髓系白血病(AML)/骨髓增生异常综合征(MDS)的早期疗效及安全性。方法选择2021年4月至2022年5月间在中国医学科学院血液病医院接受含10天地西他滨(20 mg·m^(-2)·d^(-1),-13 d~-4 d)预处理方案allo-HSCT的31例AML/MDS患者,对其临床资料进行分析。结果①31例患者中男16例,女15例,中位年龄41(20~55)岁;AML 10例,MDS转变AML(MDS-AML)6例,慢性粒-单核细胞白血病转变AML(CMML-AML)1例,MDS 14例。②31例(100%)患者均获得粒细胞植入和血小板植入,中位植入时间分别为12(9~30)d、14(9~42)d。③预处理期间16例(51.6%)患者发生口腔黏膜炎,其中Ⅰ/Ⅱ级15例(48.4%),Ⅲ级1例(3.2%);移植后13例(41.9%)患者发生巨细胞病毒血症,6例(19.4%)患者发生出血性膀胱炎,4例(12.9%)患者发生局部感染。④急性移植物抗宿主病(GVHD)中位发生时间为移植后33(12~111)d,移植后100 d急性GVHD累积发生率为41.9%(95%CI 26.9%~61.0%),Ⅲ/Ⅳ级急性GVHD累积发生率为22.9%(95%CI 13.5%~47.7%);轻、中度慢性GVHD累积发生率为23.5%(95%CI 12.1%~43.6%),未发生重度慢性GVHD。⑤1例患者死于复发,未发生非复发死亡;移植后1年累积复发率为3.2%(95%CI 0.5%~20.7%);移植后1年总生存率、无病生存率分别为92.9%(95%CI 80.3%~100.0%)、96.8%(95%CI 90.8%~100.0%)。结论含10天地西他滨预处理方案allo-HSCT治疗AML/MDS可获得较好的早期疗效且安全性良好。

Objective To investigate the early effect and safety of allogeneic hematopoietic stem cell transplantation(allo-HSCT)with a 10-day decitabine-containing conditioning regimen in the treatment of acute myeloid leukemia(AML)/myelodysplastic syndrome(MDS).MethodsFrom April 2021 to May 2022,31 AML/MDS patients who received allo-HSCT with a 10-day decitabine-containing conditioning regimen were analyzed.ResultsAML(n=10),MDS-AML(n=6),CMML-AML(n=1),and MDS(n=14)were identified in 31 patients,16 males,and 15 females,with a median age of 41(20-55)yr.Neutrophils and platelets were successfully implanted in 31 patients(100%),with a median implantation duration of 12(9-30)and 14(9-42)days,respectively.During the preconditioning period,16 patients(51.6%)developed oral mucositis,with 15 cases ofⅠ/Ⅱgrade(48.4%)and one case ofⅢgrade(3.2%).After transplantation,13 patients(41.9%)developed CMV viremia,six patients(19.4%)developed hemorrhagic cystitis,and four patients(12.9%)developed a local infection.The median time of acute graft versus host disease(aGVHD)following transplantation was 33(12-111)days.The cumulative incidence of aGVHD andⅢ/Ⅳgrade aGVHD was 41.9%(95%CI 26.9%-61.0%)and 22.9%(95%CI 13.5%-47.5%),respectively.There was no severe cGVHD,and mild and moderate chronic GVHD(cGVHD)incidence was 23.5%(95%CI 12.1%-43.6%).As of November 30,2022,only one of the 31 patients had relapsed,with a 1-yr cumulative relapse rate(CIR)of 3.2%(95%CI 0.5%-20.7%).There was only one relapse patient death and no non-relapse deaths.The 1-yr overall survival(OS)and disease-free survival(DFS)rates were 92.9%(95%CI 80.3%-100%)and 96.8%(95%CI 90.8%-100%),respectively.ConclusionsA 10-day decitabine-containing conditioning regimen for allo-HSCT reduced relapse and was safe and feasible in treating AML/MDS.