Abstract：
We conducted a multicenter, prospective observational study of acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and chronic myelomonocytic leukemia (CMML) in Japan. From August 2011 to January 2016, we enrolled 6568 patients. Herein, we report the results for MDS (n = 2747) and CMML (n = 182). The percentage of patients aged 65 years or older was 79.5% for MDS and 79.7% for CMML. The estimated overall survival (OS) rate and cumulative incidence of AML evolution at 5 years were 32.3% (95% confidence interval: 30.2-34.5%) and 25.7% (23.9-27.6%) for MDS, and 15.0% (8.9-22.7%) and 39.4% (31.1-47.6%) for CMML. Both diseases were more common in men. The most common treatment for MDS was azacitidine, which was used in 45.4% of higher-risk and 12.7% of lower-risk MDS patients. The 5-year OS rate after treatment with azacitidine was 12.1% (9.5-15.1%) for of higher-risk MDS patients and 33.9% (25.6-42.4%) for lower-risk patients. The second most common treatment was erythropoiesis-stimulating agents, given to just 20% of lower-risk patients. This is the first paper presenting large-scale, Japanese data on survival and clinical characteristics in patients with MDS and CMML.
摘要：
我们进行了一个多中心，急性髓系白血病（AML）的前瞻性观察性研究，骨髓增生异常综合征（MDS），和日本的慢性粒单核细胞白血病（CMML）。从2011年8月至2016年1月，我们招募了6568名患者。在这里，我们报告了MDS(n=2747)和CMML(n=182)的结果。年龄在65岁以上的患者比例为MDS的79.5%和CMML的79.7%。MDS的估计总生存率（OS）和AML进展的累积发生率在5年分别为32.3％（95％置信区间：30.2-34.5％）和25.7％（23.9-27.6％），CMML为15.0%（8.9-22.7%）和39.4%（31.1-47.6%）。这两种疾病在男性中更为常见。MDS最常见的治疗方法是阿扎胞苷，用于45.4％的高危MDS患者和12.7％的低危MDS患者。高危MDS患者用阿扎胞苷治疗后的5年OS率为12.1%（9.5-15.1%），低危患者为33.9%（25.6-42.4%）。第二种最常见的治疗方法是红细胞生成刺激剂，只给20%的低风险患者。这是第一篇论文，日本关于MDS和CMML患者生存和临床特征的数据。
