中文摘要：

目的 探讨白消安联合增加剂量氟达拉滨（Bu/ID-Flu）为主预处理方案进行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）的疗效与安全性.方法 回顾性分析Bu/ID-FIu预处理方案进行allo-HSCT治疗27例MDS和22例MDS-AML患者的临床资料.结果 全部49例患者均达到造血重建.白细胞植活中位时间为13（10～22）d,血小板植活中位时间为16（8～66）d.Ⅱ～Ⅳ度急性移植物抗宿主病（GVHD）、出血性膀胱炎及肝静脉阻塞病的发生率分别为28.6％、14.3％和2.0％.移植后100 d和总体移植相关死亡率（TRM）分别为4.1％（2/49）和8.2％（4/49）.中位随访14（1～92）个月,总生存（OS）率、无病生存（DFS）率分别为75.5％、73.5％.Kaplan-Meier分析示3年OS率、DFS率分别为（71.1±7.8）％、（66.7±8.3）％,复发率为16.3％.MDS、MDS-AML患者的OS率分别为81.5％ （22/27）、68.2％（15/22）,复发率分别为3.7％（1/27）、31.8％ （7/22）.allo-HSCT前达完全缓解（CR）与未达CR的MDS-AML患者的OS率分别为83.3％（10/12）、50.0％（5/10）,复发率分别为16.7％（2/12）、50.0％（5/10）.除化疗未达CR的MDS-AML患者（10例）外,其余39例患者的OS率及复发率分别为82.1％（32/39）和7.7％（3/39）.单因素分析显示,移植前疾病状态是影响OS的高危因素（P=0.031）,年龄、预处理中加入地西他滨、造血干细胞来源、HLA相合与否、供受者性别差异、输入CD34＋细胞数和GVHD均不是影响OS的危险因素.结论 应用Bu/ID-Flu为主的预处理方案对MDS和MDS-AML患者进行allo-HSCT,造血功能恢复迅速、植入稳定,并发症发生率和TRM较低.除MDS-AML化疗未达CR患者外,OS率较高且复发率较低.

英文摘要：

Objective To investigate the safety and efficacy of allogeneic hematopoietic stem cell transplantation （allo-HSCT） for myelodysplastic syndrome （MDS） and secondary acute myelogenous leukemia （MDS-AML） using conditioning regimen with busulfan （Bu） and increased-dose of fludarabine （ID-Flu）.Methods A total of 49 patients with MDS or MDS-AML were treated by allo-HSCT,the clinical data was analyzed retrospectively.Results All patients achieved hematopoietic reconstitution.Neutrophil engraftment was at 10-22 days （median 13 days）,and platelet engraftment was at 8-66 days （median 16 days）.The cumulative incidences of Ⅱ-Ⅳ degree acute graft-versus-host disease （GVHD）,hemorrhagic cystitis （HC）,and hepatic venous occlusive disease （VOD） were 28.6％,14.3％ and 2.0％,respectively.The transplant-related mortality （TRM） was only 4.1％ at 100d and 8.2％ at 1-92 months of followed-up （median 14 months） period.Overall survival （OS） and disease free survival （DFS） was 75.5％,73.5％,respectively.Kaplan-Meier curve showed that 3-year OS and 3-year DFS was （71.1 ±7.8） ％,（66.7±8.3） ％,respectively,with a relapse incidence （RI） 16.3％.OS for MDS and MDS-AML was 81.5％ and 68.2％,and RI in two settings was 3.7％,31.8％,respectively.OS for MDS-AML at complete remission （CR） and non-CR subgroup was 83.3％ and 50.0％,respectively,while cumulative RR was 16.7％ and 50.0％,respectively.OS and RI except for non-CR subgroup were 82.1％ and 7.7％.Univariate analysis showed that pre-HSCT disease status had correlation with OS （P=0.031）,but age,decitabine in conditioning regimen,stem cell source,HLA matching,patient-donor gender,dose of mononuclear cells and GVHD had no correlation with OS.Conclusions Bu/ID-Flu conditioning regimen for MDS and MDS-AML has high efficiency,fewer complications,lower toxicity and TRM.The OS and DFS were higher and RI was lower except for refractory MDS-AML patients.The regimen is valuable for clinical application.