Department of Hematology, First Affiliated Hospital，Zhejiang University School of Medicine, Hangzhou, 310003, China.
Department of Hematology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, 450052, China.
Invest New Drugs. 2022 Oct;40(5):1117-1124. doi: 10.1007/s10637-022-01283-x. Epub 2022 Jul 14.
Chronic myelomonocytic leukemia (CMML) is a rare and aggressive myeloid malignancy with overlapped features of myelodysplastic syndromes/myeloproliferative neoplasms. Azacitidine (AZA), a hypomethylating agent, has been approved for the treatment of CMML in China, but real-world data are limited. Medical records of CMML patients who had received subcutaneously injected AZA were reviewed from January 2018 at five participating sites in China. Response was assessed according to the modified International Working Group (IWG 2006) criteria. Between January 2018 and November 2020, a total of 24 patients with CMML were included with a median age of 63 years. Patients received a median of 3 cycles of AZA treatment (range, 1-8). Overall response rate (ORR) was 37.5% (9 of 24); CR rate, PR rate, and mCR/HI rate were 8.3% (n = 2), 8.3% (n = 2), and 20.8% (n = 5), respectively. At a median duration of follow-up of 14.0 months (range 0.0-22.0 months), the median overall survival (OS) was 23.0 months. Univariate analysis revealed that ≥ 3 cycles of treatment was significantly associated with a higher 1-year OS rate compared with < 3 cycles of AZA treatment. Treatment was generally well-tolerated. The most common (> 10%) AEs were thrombocytopenia (n = 7, 29.2%), pneumonitis (n = 4, 16.7%) and fever (n = 3, 12.5%). This study provides valuable real-life data in China on the treatment schedules, efficacy and safety of AZA in the treatment of CMML.
慢性粒单核细胞白血病（CMML）是一种罕见且侵袭性的髓系恶性肿瘤，具有骨髓增生异常综合征/骨髓增殖性肿瘤的重叠特征。阿扎胞苷（AZA）是一种低甲基化药物，已在中国获批用于治疗 CMML，但真实世界的数据有限。本研究回顾了 2018 年 1 月至 2020 年 11 月期间在中国 5 个参与中心接受皮下注射 AZA 治疗的 CMML 患者的病历资料。根据改良的国际工作组（IWG 2006）标准评估反应。2018 年 1 月至 2020 年 11 月期间，共纳入 24 例 CMML 患者，中位年龄为 63 岁。患者接受 AZA 治疗的中位数为 3 个周期（范围，1-8）。总体缓解率（ORR）为 37.5%（24 例中有 9 例）；完全缓解率、部分缓解率和微小完全缓解/血液学完全缓解率分别为 8.3%（n=2）、8.3%（n=2）和 20.8%（n=5）。中位随访时间为 14.0 个月（范围 0.0-22.0 个月），中位总生存期（OS）为 23.0 个月。单因素分析显示，与接受＜3 个 AZA 周期治疗的患者相比，接受≥3 个 AZA 周期治疗的患者 1 年 OS 率显著更高。治疗总体上耐受性良好。最常见（>10%）的不良事件是血小板减少症（n=7，29.2%）、肺炎（n=4，16.7%）和发热（n=3，12.5%）。本研究提供了中国 AZA 治疗 CMML 的治疗方案、疗效和安全性的宝贵真实世界数据。
