目的 比较阿扎胞苷和地西他滨治疗骨髓增生异常综合征的有效性和安全性，并探讨影响2种药物疗效的相关因素。方法 回顾性分析南京鼓楼医院2013年7月—2022年12月收治的63例骨髓增生异常综合征患者，根据不同治疗方案分为阿扎胞苷组（n＝37）和地西他滨组（n＝26）。阿扎胞苷组：sc注射用阿扎胞苷50 mg·m-2·d-1×10 d或75 mg·m-2·d-1×7 d，28 d为1个疗程。地西他滨组：sc注射用地西他滨20 mg·m-2·d-1×5 d或25 mg·m-2·d-1×4 d，每4周为1个疗程。对两组患者进行疗效和安全性评估，并对影响临床疗效的因素进行单因素及多因素Logistic回归分析。结果 阿扎胞苷组的总反应率（ORR）及骨髓完全缓解率高于地西他滨组（ORR：73.0% vs 42.3%，P=0.014；骨髓完全缓解率：40.5% vs 11.5%，P=0.012），但两组的完全缓解率和血液学改善率均无显著差异。单因素分析结果发现，患者年龄、疗程、基线血红蛋白与临床疗效相关（P< 0.05）；多因素分析结果显示，疗程≥4个［比值比（OR）＝5.439，P=0.007］和基线血红蛋白≥80 g·L-1 （OR＝4.788，P=0.027）是影响临床疗效的独立保护因素。地西他滨组骨髓抑制及发热的发生率高于阿扎胞苷组（骨髓抑制： 65.4% vs 32.4%，P= 0.012；发热：53.8% vs 24.3%，P=0.017）。结论 阿扎胞苷治疗骨髓增生异常综合征的临床疗效优于地西他滨，疗程及基线血红蛋白水平均可影响二者的疗效。阿扎胞苷在血液学方面的安全性高于地西他滨。
Objective To compare efficacy and safety of azacitidine or decitabine in treatment of myelodysplastic syndrome, and explore the factors affecting the efficacy of hypomethylating agents. Methods A total of 63 cases with myelodysplastic syndrome in Nanjing Drum Tower Hospital from July 2013 to December 2022 were selected as research objectives and divided into azacitidine group (n= 37) and decitabine group (n= 26) according to different treatment regimens. Azacitidine group: sc azacitidine for Injection 50 mg·m-2·d-1×10 d or 75 mg·m-2·d-1×7 d and 28 days are one course of treatment. Decitabine group: sc Decitabine for Injection 20 mg·m-2·d-1×5 d or 25 mg·m-2·d-1×4 d, one course of treatment every four weeks. Efficacy and safety were evaluated in both groups, and univariate and multivariate Logistic regression analysis was performed on the factors that affected the efficacy. Results Overall response rate (ORR) and marrow complete remission rate in the azacitidine group were higher than those in the decitabine group (ORR: 73.0% vs 42.3%, P= 0.014, marrow complete remission rate: 40.5% vs 11.5%, P= 0.012), but there was no significant difference in complete remission rate, hematologic improvement rate, median overall survival, and median progression-free survival. Univariate analysis showed that patient age, course of treatment, and baseline hemoglobin were associated with shortterm outcomes (P< 0.05). Multivariate analysis showed that, the number of courses ≥ 4 (OR = 5.439, P= 0.007) and baseline hemoglobin ≥ 80 g·L-1 (OR = 4.788, P= 0.027) were independent protective factors for efficacy. The incidence of myelosuppression and fever in the decitabine group was higher than the azacitidine group (myelosuppression: 65.4% vs 32.4%, P= 0.012, fever: 53.8% vs 24.3%, P= 0.017). Conclusion The efficacy of azacitidine intreatment of myelodysplastic syndrome is superior to decitabine, and the course of treatment and the baseline hemoglobin level can affect the short-term efficacy. Azacitidine has a higher hematological safety than decitabine.