Alfonso Ana, Montalban-Bravo Guillermo, Garcia-Manero Guillermo
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Curr Opin Oncol. 2017 Jan;29(1):79-87. doi: 10.1097/CCO.0000000000000337.
The present review will focus on the current management of patients with chronic myelomonocytic leukemia (CMML) as well as in future therapeutic perspectives.
CMML is a clonal hematopoietic stem cell disorder characterized by peripheral blood monocytosis and myelodysplastic and myeloproliferative alterations in the bone marrow. Clinical behavior of the disease can be heterogeneous, with some patients having an indolent form of the disease, whereas others experience an aggressive course with decreased survival and eventual transformation to leukemia. Multiple studies have helped define the clinical, cytogenetic and mutational prognostic features of the disease. In addition, several prognostic scoring systems have been developed for patients with CMML. Incorporation of mutation data, particularly presence of frameshift and nonsense ASXL1 mutations, into these models seems to be allowing to further improve our ability to predict patient outcomes.
Prognosis of patients with CMML is heterogeneous. Incorporation of mutational data into current clinical prognostic models has allowed to improve our ability to predict patient outcomes. Allogeneic stem cell transplantation remains the only potentially curative treatment for patients with CMML but is only an option for a subset of patients. For this reason, hypomethylating agents such as 5-azacitidine and decitabine have become the backbone of current therapy for patients with CMML, but new therapeutic strategies are required to improve their outcomes.
本综述将聚焦于慢性粒-单核细胞白血病(CMML)患者的当前管理以及未来的治疗前景。
CMML是一种克隆性造血干细胞疾病,其特征为外周血单核细胞增多以及骨髓中的骨髓发育异常和骨髓增殖性改变。该疾病的临床行为具有异质性,一些患者病情进展缓慢,而另一些患者病程侵袭性强,生存率降低并最终转化为白血病。多项研究有助于明确该疾病的临床、细胞遗传学和突变预后特征。此外,已为CMML患者开发了多种预后评分系统。将突变数据,尤其是移码和无义ASXL1突变的存在,纳入这些模型似乎能够进一步提高我们预测患者预后的能力。
CMML患者的预后具有异质性。将突变数据纳入当前临床预后模型已提高了我们预测患者预后的能力。异基因干细胞移植仍然是CMML患者唯一可能治愈的治疗方法,但仅适用于一部分患者。因此,5-氮杂胞苷和地西他滨等去甲基化药物已成为CMML患者当前治疗的主要手段,但需要新的治疗策略来改善其治疗效果。