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| 减低剂量阿扎胞苷治疗中高危MDS及MDS/MPN患者的临床疗效 |
| The clinical efficacy of reduced-dose azacitidine in patients with intermediate and high risk myelodysplastic syndrome and myelodysplastic syndrome/myeloproliferative neoplasms |
| 投稿时间：2021-02-02 |
| DOI：10.3969/j.issn.1000-0399.2021.09.001 |
| 中文关键词: 减低剂量 阿扎胞苷 骨髓增生异常综合征 骨髓增生异常综合征/骨髓增殖性肿瘤 临床疗效 不良反应 |
| 英文关键词: Reduceddose Azacitidine Myelodysplastic syndrome Myelodysplastic syndrome/myeloproliferative neoplasms Clinical efficacy Adverse effects |
| 基金项目:国家自然科学基金（项目编号：81900118） |
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| 中文摘要: |
| 目的 观察减低剂量阿扎胞苷治疗中高危骨髓增生异常综合征（MDS）及骨髓增生异常综合征/骨髓增殖性肿瘤（MDS/MPN）患者的临床疗效。方法 选取2018年12月至2021年1月安徽医科大学第一附属医院收治中高危MDS（24例）及MDS/MPN（13例）患者，采取减低剂量阿扎胞苷[60 mg/（m2·d），连续7 d，28 d为1个疗程]治疗，分析临床疗效与不良反应。结果 中位疗程为2个疗程。32例接受二代基因测序的患者中，27例（84.38%）存在基因突变。治疗总反应率为54.05%，阿扎胞苷治疗≥ 3个疗程的患者总反应率高于<3个疗程者（80.00% vs 36.36%，P=0.018）。中位生存期为15.00个月。不良反应包括骨髓抑制、感染及胃肠道反应。结论 减低剂量阿扎胞苷治疗中高危MDS及MDS/MPN患者具有良好的有效性及安全性，多疗程治疗可提高临床疗效。 |
| 英文摘要: |
| Objective To observe the clinical efficacy of reduced-dose azacitidine on patients with intermediate and high risk myelodysplastic syndrome (MDS) and myelodysplastic syndrome/myeloproliferative neoplasms(MDS/MPN). Methods Twenty-four patients with MDS and thirteen patients with MDS/MPN from the First Affiliated Hospital of Anhui Medical University between December 2018 and January 2021 were treated with reduced-dose azacitidine[60 mg/(m2·d) for 7 days per 28-day cycle], then the clinical efficacy and adverse effects were analyzed. Results The median number of cycles was two. Next-generation gene sequence was performed among 32 patients, and mutations in genes were identified in 27 patients (84.38%). 54.05% of patients responded to the reduced-dose azacitidine. Patients who received at least three cycles had a better response to azacitidine than those who received fewer than three cycles (80.00% vs 36.36%, P=0.018). The median survival was 15.00 months in the follow-up period. Adverse effects included myelosuppression, infection, and gastrointestinal reaction. Conclusions Reduced-dose azacitidine is effective and safe for patients with intermediate risk or high risk MDS and MDS/MPN, and the clinical efficacy is better in patients who have received multiple cycles. |
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