Department of Hematology, AOU Careggi, University of Florence, Florence, Italy.
AOU Citta della Salute e della Scienza, Torino, Italy.
Leukemia. 2018 Feb;32(2):413-418. doi: 10.1038/leu.2017.186. Epub 2017 Jun 13.
Chronic myelomonocytic leukemia (CMML) is a complex clonal hematological disorder classified among myelodysplastic (MDS)/myeloproliferative neoplasms. Prognosis is poor and there is a lack of effective treatments. The hypomethylating agent decitabine has shown activity against MDS and elderly acute myeloid leukemia, but there is little data focusing specifically on its efficacy in CMML. In this prospective, phase 2 Italian study, CMML patients received intravenous decitabine 20 mg/m per day on Days 1-5 of a 28-day treatment cycle. Response was evaluated after four and six cycles; patients responding at the end of six cycles could continue treatment with decitabine. Forty-three patients were enrolled; >50% were high-risk according to four CMML-specific scoring systems. In the intent-to-treat population (n=42), the overall response rate after six cycles was 47.6%, with seven complete responses (16.6%), eight marrow responses (19%), one partial response (2.4%) and four hematological improvements (9.5%). After a median follow-up of 51.5 months (range: 44.4-57.2), median overall survival was 17 months, with responders having a significantly longer survival than non-responders (P=0.02). Grade 3/4 anemia, neutropenia and thrombocytopenia occurred in 28.6%, 50% and 38% of patients, respectively. Decitabine appears to be an effective and well-tolerated treatment for patients with high-risk CMML.
慢性粒单核细胞白血病（CMML）是一种复杂的克隆性血液系统疾病，归类于骨髓增生异常（MDS）/骨髓增殖性肿瘤。预后较差，且缺乏有效的治疗方法。低甲基化药物地西他滨已显示出对 MDS 和老年急性髓系白血病的活性，但针对 CMML 具体疗效的数据很少。在这项前瞻性、意大利的 2 期研究中，CMML 患者接受静脉滴注地西他滨，剂量为 20mg/m，每天一次，每 28 天为一个周期，连用 5 天。在第 4 和第 6 个周期后评估反应；在第 6 个周期结束时有反应的患者可以继续接受地西他滨治疗。共纳入 43 例患者；根据 4 种 CMML 特异性评分系统，超过 50%的患者为高危。在意向治疗人群（n=42）中，6 个周期后的总缓解率为 47.6%，其中完全缓解 7 例（16.6%），骨髓缓解 8 例（19%），部分缓解 1 例（2.4%），血液学改善 4 例（9.5%）。中位随访时间为 51.5 个月（范围：44.4-57.2），中位总生存期为 17 个月，缓解者的生存时间明显长于无缓解者（P=0.02）。3/4 级贫血、中性粒细胞减少和血小板减少的发生率分别为 28.6%、50%和 38%。地西他滨似乎是一种有效且耐受良好的治疗高危 CMML 的药物。
