地西他滨联合依托泊苷为主方案与单独地西他滨治疗老年骨髓增生异常综合征疗效分析

Efficacy analysis of decitabine combined with etoposide as the main regimen and decitabine alone in the treatment of elderly patients with myelodysplastic syndrome

目的 基于多中心数据比较地西他滨联合依托泊苷为主方案和单独地西他滨治疗老年骨髓增生异常综合征(MDS)及转化的急性髓系白血病(AML)患者疗效及预后。方法 收集2016年1月至2021年10月复旦大学附属华东医院等6家医院确诊的61例老年MDS/tAML患者资料。对患者临床特征、疗效、不良事件及治疗相关死亡进行比较,计算生存率并绘制生存曲线。结果 41例患者接受联合方案治疗,平均年龄(68.5±6.35)岁;20例患者接受单药治疗,平均年龄(67.3±6.14)岁。联合治疗患者在完全缓解率(51.2%vs 20.0%,p<0.05)、一年内转白率(5.9%vs 35.0%,p<0.01)上均优于单药治疗。亚组分析中,TP53突变患者接受联合治疗后总体缓解率(94.7%vs 50.0%,p<0.05)、中位OS (31.0月vs 12.0月,p<0.05)、中位PFS(24.0月vs 8.0月,p=0.012)均优于单药治疗。结论 地西他滨联合依托泊苷为主方案治疗MDS/tAML患者较单用地西他滨在疗效上有优势。在TP53突变患者中,联合方案较单药治疗能够改善患者疗效及生存预后。

Objective To compare the efficacy and prognosis of decitabine combined with etoposide as the main regimen and decitabine alone in the treatment of elderly patients with myelodysplastic syndrome(MDS) and transformed acute myeloid leukemia(tAML) based on multicenter data.Method The data of 61 elderly patients with MDS/tAML diagnosed in 6 hospitals including Huadong Hospital Affiliated to Fudan University from January 2016 to October 2021 were collected.The clinical characteristics,curative effects,adverse events,and treatment-related deaths of the patients were compared,the survival rate was calculated,and the survival curve was drawn.Results 41 patients were treated with combined regimen,with an average age of(68.5±6.35) years;20 ones received monotherapy with an average age of(67.3±6.14) years.The complete remission rate(51.2% vs 20.0%,P<0.05) and the conversion rate to leukemia within one year(5.9% vs 35.0%,P<0.01) of the patients treated with combination therapy were better than those treated with monotherapy.In subgroup analysis,among patients with TP53 mutaion,the overall response rate(94.7% vs 50.0%,P<0.05),median OS(31.0 months vs 12.0 months,P<0.05),and median PFS(24.0 months vs 8.0 months,P=0.012) of patients who received combination therapy were better than those who received monotherapy.Conclusion Decitabine combined with etoposide as the main regimen is superior to decitabine alone in the treatment of MDS/tAML patients.In patients with TP53 mutation,the combined regimen can improve the curative effect and survival prognosis of patients compared with monotherapy.