文章：

年轻患者的慢性粒单核细胞白血病：生存及治疗结局的分子与细胞遗传学预测因素

Chronic myelomonocytic leukemia in younger patients: molecular and cytogenetic predictors of survival and treatment outcome

原文发布日期：2015-01-02

DOI: 10.1038/bcj.2014.90

类型: Original Article

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英文摘要：

In patients with chronic myelomonocytic leukemia (CMML), age>65 years is an adverse prognostic factor. Our objective in the current study was to examine risk factors for survival and treatment outcome in 261 ‘young’ adults with CMML, as defined by age ⩽65 years. In multivariable analysis, lower HB (P=0.01), higher circulating blast % (P=0.002), ASXL1 (P=0.0007) and SRSF2 mutations (P=0.008) and Mayo-French cytogenetic stratification (P=0.04) negatively impacted survival. Similarly, leukemia-free survival was independently affected by higher circulating blast % (P<0.0001), higher bone marrow blast % (P=0.0007) and the presence of circulating immature myeloid cells (P=0.0002). Seventy-five (29%) patients received hypomethylating agents (HMA), with the median number of cycles being 5, and the median duration of therapy being 5 months. The over-all response rate was 40% for azacitidine and 30% for decitabine. Fifty-three (24%) patients underwent an allogeneic hematopoietic stem cell transplant (AHSCT), with a response rate of 56% and a non-relapse mortality of 19%. Survival in young adults with CMML, although higher than in older patients, is poor and even worse in the presence of ASXL1 and SRSF2 mutations. Treatment outcome was more impressive with AHSCT than with HMA and neither was influenced by ASXL1/SRSF2 mutations or karyotype.

摘要翻译： 

在慢性粒-单核细胞白血病（CMML）患者中，年龄＞65岁是不良预后因素。本研究旨在探讨261例年龄≤65岁的“年轻”成人CMML患者生存及治疗结局的风险因素。多变量分析显示，较低血红蛋白（P=0.01）、较高外周血原始细胞百分比（P=0.002）、ASXL1（P=0.0007）与SRSF2基因突变（P=0.008）及梅奥-法国细胞遗传学分层（P=0.04）对生存率产生负面影响。无白血病生存期同样独立受到较高外周血原始细胞百分比（P<0.0001）、较高骨髓原始细胞百分比（P=0.0007）及外周血未成熟髓系细胞存在（P=0.0002）的影响。75例（29%）患者接受低甲基化药物治疗，中位治疗周期数为5周期，中位治疗持续时间为5个月。阿扎胞苷总体缓解率为40%，地西他滨为30%。53例（24%）患者接受异基因造血干细胞移植，缓解率达56%，非复发死亡率为19%。年轻CMML患者虽生存率高于老年患者，但预后仍较差，若存在ASXL1和SRSF2突变则预后更差。异基因造血干细胞移植的治疗效果优于低甲基化药物，且两者均不受ASXL1/SRSF2突变或核型影响。

原文链接：

Chronic myelomonocytic leukemia in younger patients: molecular and cytogenetic predictors of survival and treatment outcome