目的 比較阿扎胞苷和地西他濱治療骨髓增生異常綜合征的有效性和安全性，并探討影響2種藥物療效的相關(guān)因素。方法 回顧性分析南京鼓樓醫(yī)院2013年7月—2022年12月收治的63例骨髓增生異常綜合征患者，根據(jù)不同治療方案分為阿扎胞苷組（n＝37）和地西他濱組（n＝26）。阿扎胞苷組：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為1個療程。地西他濱組：sc注射用地西他濱20 mg·m^-2·d^-1×5 d或25 mg·m^-2·d^-1×4 d，每4周為1個療程。對兩組患者進(jìn)行療效和安全性評估，并對影響臨床療效的因素進(jìn)行單因素及多因素Logistic回歸分析。結(jié)果 阿扎胞苷組的總反應(yīng)率（ORR）及骨髓完全緩解率高于地西他濱組（ORR：73.0% vs 42.3%，P=0.014；骨髓完全緩解率：40.5% vs 11.5%，P=0.012），但兩組的完全緩解率和血液學(xué)改善率均無顯著差異。單因素分析結(jié)果發(fā)現(xiàn)，患者年齡、療程、基線血紅蛋白與臨床療效相關(guān)（P< 0.05）；多因素分析結(jié)果顯示，療程≥4個［比值比（OR）＝5.439，P=0.007］和基線血紅蛋白≥80 g·L^-1 （OR＝4.788，P=0.027）是影響臨床療效的獨(dú)立保護(hù)因素。地西他濱組骨髓抑制及發(fā)熱的發(fā)生率高于阿扎胞苷組（骨髓抑制： 65.4% vs 32.4%，P= 0.012；發(fā)熱：53.8% vs 24.3%，P=0.017）。結(jié)論 阿扎胞苷治療骨髓增生異常綜合征的臨床療效優(yōu)于地西他濱，療程及基線血紅蛋白水平均可影響二者的療效。阿扎胞苷在血液學(xué)方面的安全性高于地西他濱。

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.

R979.1

江蘇省研究型醫(yī)院學(xué)會精益化用藥科研基金項目（JY202113）；南京鼓樓醫(yī)院臨床研究專項資金（2022-LCYJ-PY-48）；南京藥學(xué)會-常州四藥醫(yī)院藥學(xué)科研基金資助課題（2020YX010）