超小剂量地西他滨治疗中高危骨髓增生异常综合征患者的疗效及相关因素分析

Efficacy and related factors of ultra-low dose decitabine in patients with intermediate-to high-risk myelodysplastic syndrome

目的探讨超小剂量地西他滨治疗中高危骨髓增生异常综合征(MDS)患者的临床疗效及其相关因素。方法收集首都医科大学宣武医院血液科和北京航天总医院血液科于2014年7月1日~2018年12月31日收治的接受含超小剂量地西他滨方案治疗的中高危MDS患者19例,观察其疗效、不良反应及相关影响因素。结果19例患者共接受了81个周期含超小剂量地西他滨的方案治疗。总有效率为78.95%,其中获得改变自然病程反应和血液学改善(HI)的有效率分别为57.89%和78.95%。与年龄≥65岁患者比较,年龄<65岁患者更易获得改变疾病自然病程反应、完全缓解及血液学改善-红细胞反应(P值分别为0.024、0.045、0.024)。国际预后积分系统(IPSS)分层为相对高危的患者在治疗后更易获得血液学改善-血小板反应(P=0.018)。患者开始治疗后的中位无进展生存(PFS)时间为11个月、中位总生存(OS)时间为11个月。发病时外周血无原始细胞患者的PFS及OS时间明显延长(P值分别为0.013、0.005);治疗后发生自然病程反应或输血依赖改善的患者OS明显延长(P值分别为0.016、0.008);发病年龄≥65岁及伴有合并疾病对患者的PFS及OS均无影响(P>0.05)。不良反应以骨髓抑制所致感染及出血多见。结论应用含超小剂量地西他滨方案治疗中高危MDS患者的临床疗效较好、不良反应少,老年(年龄≥65岁)及有无合并疾病不影响患者的生存。

Objective To investigate the clinical efficacy and related factors of ultra-low dose decitabine in patients with intermediate-to high-risk myelodysplastic syndromes(MDS).Methods Nineteen patients with intermediate-to-high-risk MDS received ultra-low dose decitabine based therapy from July 1,2014 to December 31,2018 in hematology department of Xuanwu Hospital,Capital Medical University and Beijing Aerospace General Hospital were collected.The efficacy,adverse effects and related factors were analyzed.Results The 19 patients were treated with 81 cycles of ultra-low dose decitabine.The overall response rate was 78.95%,the rate of altering the natural history of the disease was 57.89%and the rate of hematologic improvement(HI)was 78.95%.Compared to patients older than 65,patients younger than 65 had significantly priority in altered disease natural cousre response rate,complete remission rate and HI-erythroid response rate(P values were 0.024,0.045,0.024,respectively).Relatively high-risk patients according to the International Prognostic Scoring System(IPSS)had significantly higher rate of HI-platelet response(P=0.018).The median progression-free survival(PFS)phase was 11 months and the median overall survival(OS)phase was also 11 months since the beginning of the treatment.The PFS and OS phase of patients without blasts in peripheral blood at the time of onset were significantly prolonged(P values were 0.013 and 0.005,respectively).In patients got natural pathological response or improved blood transfusion dependence after treatment,the OS phase were significantly prolonged(P values were 0.016 and 0.008,respectively).Older than 65 and combined diseases did not affect the PFS and OS.The common adverse events were infection and hemorrhage caused by myelosuppression.Conclusion Ultra-low dose decitabine is safe and effective in the treatment of intermediate-to high-risk MDS patients.Elderly(age≥65 years old)and combined diseases do not effect the survival of the patients.