Department of Haematology, British Hospital, Av. Italia 2420, Montevideo, Uruguay,
Curr Treat Options Oncol. 2014 Jun;15(2):147-56. doi: 10.1007/s11864-014-0279-3.
Originally described by Dameshek in 1951, myeloproliferative disorders are today classified as myeloproliferative Neoplasms (MPNs) in WHO's Classification of Tumors of Hematopoietic and Lymphoid Tissues. The term includes a range of conditions, [ie, BCR-ABL-positive chronic myelogenous leukemia (CML), chronic neutrophilic leukemia (CNL), polycythemia vera (PV), primary myelofibrosis (PMF), essential thromobocythemia (ET), chronic eosinophilic leukemia not otherwise specified (CEL-NOS), mastocytosis, and unclassifiable myeloproliferative neoplasm]. In the specific case of CML, a better understanding of the pathogenesis and pathophysiology of the disease has led to a targeted therapy. The presence of chromosome Philadelphia, t(9;22)(q34;11) results in the oncogene BCR-ABL, which characterizes the disease; this molecular rearrangement gives rise to a tyrosine-kinase, which in turn triggers the proliferation of the myeloid line through the activation of the signaling pathways downstream. Tyrosine-kinase inhibitors (TKIs) have altered the therapy and monitoring of CML patients and improved both their prognosis and quality of life. In 2005, various groups of investigators described a new point mutation of the gene JAK2 associated to MPNs. Although the presence of this mutation has led to a modification in the diagnostic criteria of these conditions, the impact of the use of JAK2 inhibitors on the prognosis and course of the disease continues to be controversial.
最初由 Dameshek 在 1951 年描述的骨髓增生性疾病,如今在 WHO 造血和淋巴组织肿瘤分类中被归类为骨髓增生性肿瘤(MPN)。该术语包括一系列病症,例如 BCR-ABL 阳性慢性髓系白血病(CML)、慢性中性粒细胞白血病(CNL)、真性红细胞增多症(PV)、原发性骨髓纤维化(PMF)、特发性血小板增多症(ET)、慢性嗜酸粒细胞白血病未另分类(CEL-NOS)、肥大细胞病和未分类骨髓增生性肿瘤。在 CML 的具体情况下,对疾病发病机制和病理生理学的更好理解导致了靶向治疗。费城染色体的存在,t(9;22)(q34;11)导致癌基因 BCR-ABL 的出现,这是该疾病的特征;这种分子重排产生一种酪氨酸激酶,进而通过激活下游信号通路触发髓系细胞的增殖。酪氨酸激酶抑制剂(TKI)改变了 CML 患者的治疗和监测方法,改善了他们的预后和生活质量。2005 年,不同研究小组描述了与 MPN 相关的 JAK2 基因的一种新的点突变。尽管该突变的存在导致了这些病症的诊断标准的修改,但 JAK2 抑制剂的使用对疾病的预后和病程的影响仍然存在争议。