Talati Chetasi, Padron Eric
H. Lee Moffitt Cancer Center and Research Institute, Malignant Hematology, Tampa, FL, USA.
Curr Hematol Malig Rep. 2016 Dec;11(6):425-433. doi: 10.1007/s11899-016-0350-1.
According to the recently published 2016 World Health Organization (WHO) classification of myeloid malignancies, myelodysplastic/myeloproliferative neoplasms (MDS/MPN) include atypical chronic myeloid leukemia (aCML), MDS/MPN-unclassifiable (MDS/MPN-U), chronic myelomonocytic leukemia (CMML), juvenile myelomonocytic leukemia (JMML), and MDS/MPN ring sideroblasts with thrombocytosis (MDS/MPN-RS-T). MDS/MPN-RS-T was previously a provisional category known as refractory anemia with ring sideroblasts with thrombocytosis (RARS-T) which has now attained a distinct designation in the 2016 WHO classification. In this review, we focus on biology and management of aCML, MDS/MPN-U, and MDS/MPN-RS-T. There is considerable overlap between these entities which we attempt to further elucidate in this review. We also discuss recent advances in the field of molecular landscape that further defines and characterizes this heterogeneous group of disorders. The paucity of clinical trials available secondary to unclear pathogenesis and rarity of these diseases makes the management of these entities clinically challenging. This review summarizes some of the current knowledge of the molecular pathogenesis and suggested treatment guidelines based on the available data.
根据世界卫生组织（WHO）近期发布的2016年髓系恶性肿瘤分类，骨髓增生异常/骨髓增殖性肿瘤（MDS/MPN）包括非典型慢性髓性白血病（aCML）、无法分类的MDS/MPN（MDS/MPN-U）、慢性粒单核细胞白血病（CMML）、青少年粒单核细胞白血病（JMML）以及伴有血小板增多的MDS/MPN环形铁粒幼细胞（MDS/MPN-RS-T）。MDS/MPN-RS-T以前是一个临时类别，称为伴有血小板增多的环形铁粒幼细胞难治性贫血（RARS-T），现在在2016年WHO分类中已有明确的命名。在本综述中，我们重点关注aCML、MDS/MPN-U和MDS/MPN-RS-T的生物学特性及管理。这些实体之间存在相当多的重叠，我们试图在本综述中进一步阐明。我们还讨论了分子图谱领域的最新进展，这些进展进一步明确并描述了这一异质性疾病组。由于发病机制不明确以及这些疾病罕见，可用的临床试验很少，这使得这些实体的管理在临床上具有挑战性。本综述总结了一些关于分子发病机制的当前知识，并根据现有数据提出了治疗指南。
