Liu X Z, Zhou S J, Huang J, Zhao C F, Jiang L X, Zhang Y D, Mei C, Ma L Y, Zhou X P, Shao Y P, Wu G Q, Xiao X B, Yao R X, Du X H, Hu T L, Qian S X, Li Y, Yan X F, Huang L, Wang M L, Fu J P, Shou L H, Jiang W H, Jin W M, Li L J, Le J, Luo W J, Zhang Y, Zhou X J, Zhang H, Lang X H, Zhou M, Jin J, Jiang H F, Zhang J, Ouyang G F, Tong H Y
Department of Hematology, the First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou 310003, China.
Department of Hematology, the First Affiliated Hospital of Wenzhou Medical University, Wenzhou 325015, China.
Zhonghua Xue Ye Xue Za Zhi. 2024 Aug 14;45(8):738-747. doi: 10.3760/cma.j.cn121090-20240405-00124.
To evaluate the efficacy and safety of hypomethylating agents (HMA) in patients with myelodysplastic syndromes (MDS) . A total of 409 MDS patients from 45 hospitals in Zhejiang province who received at least four consecutive cycles of HMA monotherapy as initial therapy were enrolled to evaluate the efficacy and safety of HMA. Mann-Whitney or Chi-square tests were used to compare the differences in the clinical data. Logistic regression and Cox regression were used to analyze the factors affecting efficacy and survival. Kaplan-Meier was used for survival analysis. Patients received HMA treatment for a median of 6 cycles (range, 4-25 cycles) . The complete remission (CR) rate was 33.98% and the overall response rate (ORR) was 77.02%. Multivariate analysis revealed that complex karyotype (=0.02, =0.39, 95% 0.18-0.84) was an independent favorable factor for CR rate. TP53 mutation (=0.02, =0.22, 95% 0.06-0.77) was a predictive factor for a higher ORR. The median OS for the HMA-treated patients was 25.67 (95% 21.14-30.19) months. HMA response (=0.036, =0.47, 95% 0.23-0.95) was an independent favorable prognostic factor, whereas complex karyotype (=0.024, =2.14, 95% 1.10-4.15) , leukemia transformation (<0.001, =2.839, 95% 1.64-4.92) , and TP53 mutation (=0.012, =2.19, 95% 1.19-4.07) were independent adverse prognostic factors. There was no significant difference in efficacy and survival between the reduced and standard doses of HMA. The CR rate and ORR of MDS patients treated with decitabine and azacitidine were not significantly different. The median OS of patients treated with decitabine was longer compared with that of patients treated with azacitidine (29.53 months 20.17 months, =0.007) . The incidence of bone marrow suppression and pneumonia in the decitabine group was higher compared with that in the azacitidine group. Continuous and regular use of appropriate doses of hypomethylating agents may benefit MDS patients to the greatest extent if it is tolerated.
评估去甲基化药物（HMA）治疗骨髓增生异常综合征（MDS）患者的疗效和安全性。纳入浙江省45家医院共409例接受至少四个连续周期HMA单药初始治疗的MDS患者，以评估HMA的疗效和安全性。采用Mann-Whitney检验或卡方检验比较临床数据差异。使用逻辑回归和Cox回归分析影响疗效和生存的因素。采用Kaplan-Meier法进行生存分析。患者接受HMA治疗的中位周期数为6个周期（范围4 - 25个周期）。完全缓解（CR）率为33.98%，总缓解率（ORR）为77.02%。多因素分析显示，复杂核型（=0.02，=0.39，95%置信区间0.18 - 0.84）是CR率的独立有利因素。TP53突变（=0.02，=0.22，95%置信区间0.06 - 0.77）是较高ORR的预测因素。接受HMA治疗患者的中位总生存期（OS）为25.67（95%置信区间21.14 - 30.19）个月。HMA反应（=0.036，=0.47，95%置信区间0.23 - 0.95）是独立的有利预后因素，而复杂核型（=0.024，=2.14，95%置信区间1.10 - 4.15）、白血病转化（<0.001，=2.839，95%置信区间1.64 - 4.92）和TP53突变（=0.012，=2.19，95%置信区间1.19 - 4.07）是独立的不良预后因素。HMA减量和标准剂量在疗效和生存方面无显著差异。地西他滨和阿扎胞苷治疗的MDS患者的CR率和ORR无显著差异。地西他滨治疗患者的中位OS较阿扎胞苷治疗患者更长（29.53个月对20.17个月，=0.007）。地西他滨组骨髓抑制和肺炎的发生率高于阿扎胞苷组。如果耐受，持续且规律地使用适当剂量的去甲基化药物可能使MDS患者最大程度获益。
