含地西他滨方案桥接异基因造血干细胞移植治疗MDS／AML的效果评估

Therapeutic efficacies of decitabine application prior to hematopoietic cell transplantation in patientswith myelodysplastic syndrome and acute myeloid leukemia

目的观察骨髓增生异常综合征（MDS）及复发难治性急性髓系白血病（AML）患者造血干细胞移植前选择含地西他滨方案诱导或巩固作为过渡治疗的临床疗效，以期降低肿瘤负荷，控制疾病并为寻找供体赢取时间。方法回顾性分析苏州大学附属第一医院2009年9月至2013年2月期间在异基因造血干细胞移植前采用地西他滨单药或联合化疗进行治疗的14例MDS和32例AML患者的临床资料，评价治疗效果。结果经含地西他滨方案治疗后，14例MDS患者中10例达完全缓解（CR），2例为部分缓解（PR），1例稳定状态（SI）），1例未缓解（NR），1个疗程CR率达10／14。32例AML患者经地西他滨治疗后，17例获得CR，另有15例在未达缓解状态下进行移植，1个疗程CR率为53．1％（17／32），1个疗程的有效率（CR＋PR）为78．1％（25／32）。MDS患者移植物均植入成功，12例目前均无病生存，1例移植后8个月复发。AML患者中28例植入成功，随访至2013年2月，20例无病生存，10例死亡，2例带瘤生存。46例患者中急性及慢性移植物抗宿主病（GVHD）的发生率分别为4．3％（2／46）和23．9％（11／46）。中位随访8个月，治疗相关死亡率为23．9％（11／46）。30个月的元病生存率和总生存率分别为53．1％和61．9％。结论含地西他滨方案可安全有效桥接异基因造血干细胞移植治疗MDS和AML。

Objective To explore the therapeutic efficacies of decitabine application prior to hematopoietic cell transplantation （HSCY） in patients with myelodysplastic syndrome （MDS） and acute myeloid leukemia （AML）. Methods Retrospective reviews were conducted for 46 patients with MDS （ n = 14 ） and AML （ n = 32） on a therapy of decitabine prior to allo-HSCT between September 2009 and February 2013. Results In MDS patients, complete remission （CR, n = 10）, partial remission （PR, n =2） and stable disease （ SD, n = 1 ） were achieved prior to HSCT. And the remission rate of one course was 10/14. After deeitabine dosing, 17/32 patients achieved CR in 32 with AML and the remission rate of one course was 53.1% （17/32） and effective rate of one course （CR ＋ PR） achieves 78. 1% （25/32）. Successful engraftment was attained in all MDS patients and 12/14 patients survived disease-free and one died of pneumonia after relapse. And 28 patients with AML attained successful engraftment after using decitabine prior to allo-HSCT and there were 20 disease-free survivors. Ten patients died and another lived with tumor. The incidences of acute and chronic graft-versus-host disease （GVHD） among evaluable patients were 4. 3% （2/26） and 23.9% （11/46） respectively. After a median follow-up of 8 months for survivors, thetreatment-related mortality was 23.9% （11/46 ）. The 30-month disease-free survival （DFS） rate was 53.1% and 30-month overall survival rate after deeitabine dosing 61.9%. Conclusion Thus deeitabine is an effective therapy during bridge time to HSCT in patients with MDS and AML.