Clinical efficacy analysis for treatment of moderate to high risk myelodysplastic syndrome with decitabine
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摘要: 目的 评价地西他滨治疗中高危骨髓增生异常综合征(myelodysplastic syndromes,MDS)患者的有效性及安全性,探讨TET2、DNMT3A基因突变的临床意义。 方法 回顾性分析2013年10月—2018年7月浙江中医药大学附属第一医院血液科81例MDS患者,根据治疗方法不同分为单药组(32例,地西他滨)和联合化疗组(49例,地西他滨、高三尖杉酯碱、阿糖胞苷、阿克拉霉素)。 结果 ①疗效,单药组:完全缓解(CR)率为25.0%,总体有效(ORR)率为53.1%;联合化疗组:CR率为44.9%,ORR率为67.3%。②无进展生存时间(PFS):单药组为9个月,联合化疗组为13个月,2组比较差异无统计学意义(χ2=0.942,P=0.332)。③不良反应:单药组3~4级血细胞减少发生率为81.25%,联合化疗组为89.79%;单药组感染发生率为65.63%,联合化疗组感染发生率为77.55%。非血液学毒性均为1~2级。④TET2基因突变16例:单药组5例,联合化疗组11例;DNMT3A基因突变8例:单药组1例,联合化疗组7例;2组比较差异无统计学意义。 结论 地西他滨治疗中高危MDS有较好疗效,2种方案对比差异无统计学意义,TET2、DNMT3A基因突变对疗效无影响。Abstract: Objective To evaluate the efficacy and safety of decitabine in the treatment of patients with moderate to high risk myelodysplastic syndrome(MDS), and to explore the clinical significance of TET2 and DNMT3 A gene mutations. Methods Clinical data of 81 patients with MDS in the First Affiliated Hospital of Zhejiang Chinese Medical University were retrospectively analysed. According to the different treatment methods, they were divided into single drug group(32 cases, decitabine) and combined chemotherapy group(49 cases, decitabine, homoharringtonine, Ara-C and Aclacinomycin). Results ① The efficacy:the complete remission(CR) rate was 25.0% and the overall response(ORR) was 53.1% in the single drug group. The CR rate was 44.9% and the ORR rate was 67.3% in the combined chemotherapy group. ② The progression-free survival time(PFS):the single drug group was 9 months, and the combined chemotherapy group was 13 months, with no significant difference(χ2=0.942, P=0.332). ③ Adverse events:the incidence of grade 3-4 hemocytopenia was 81.25% in the single drug group and 89.79% in the combined chemotherapy group. The incidence of infection was 65.63% in the single drug group and 77.55% in the combined chemotherapy group. The non-hematological toxicity was grade 1-2 in two groups. ④ There were 16 cases of TET2 gene mutation, including 5 cases in single drug group and 11 cases in combined chemotherapy group. There were 8 mutations of DNMT3 A gene, including 1 in single drug group and 7 in combined chemotherapy group. There was no significant difference between two groups. Conclusion Decitabine have good efficacy in the treatment of moderate to high risk MDS. There was no significant difference between the two treatment methods. TET2 and DNMT3 A gene mutations have no significant effect on efficacy.
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Key words:
- Myelodysplastic syndromes /
- Decitabine /
- Gene mutation
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