Gowin Krisstina, Mesa Ruben
Department of Hematology and Medical Oncology, Mayo Clinic Arizona, Scottsdale, AZ, 85259, USA.
Division of Hematology and Medical Oncology, Mayo Clinic Cancer Center, Scottsdale, AZ, 85259, USA.
F1000Res. 2014 Sep 29;3:227. doi: 10.12688/f1000research.5361.1. eCollection 2014.
Essential thrombocythemia is a clonal myeloproliferative neoplasm characterized by an elevated platelet count, the potential for both microvascular and macrovascular sequelae, and a risk for transformation to myelofibrosis or acute myeloid leukemia. A systematic and detailed initial analysis is essential for accurate diagnosis of essential thrombocythemia, as many etiologies are reactive and benign. Once a diagnosis has been made, risk stratification and symptom assessment are vital to guide the subsequent therapy. Treatment may be required in high-risk disease, such as in cases of advanced age or prior thrombotic events. Systemic therapy is aimed at reducing the thrombotic risk and includes daily low dose aspirin and in some patients, cytoreductive therapy. Currently, the first line cytoreductive therapy includes hydroxyurea or pegylated interferon, with a phase III clinical trial underway comparing these two important agents. Anagrelide and clinical trials are reserved for refractory or intolerant patients. Looking to the future, new therapies including Janus kinase 2 (JAK2) and telomerase inhibitors are promising and may become valuable to the treatment armamentarium for those afflicted with essential thrombocythemia.
原发性血小板增多症是一种克隆性骨髓增殖性肿瘤，其特征为血小板计数升高、存在微血管和大血管后遗症的可能性以及转化为骨髓纤维化或急性髓系白血病的风险。由于许多病因是反应性的且为良性，因此系统而详细的初始分析对于准确诊断原发性血小板增多症至关重要。一旦做出诊断，风险分层和症状评估对于指导后续治疗至关重要。高危疾病可能需要治疗，例如高龄或既往有血栓形成事件的病例。全身治疗旨在降低血栓形成风险，包括每日低剂量阿司匹林，在一些患者中还包括细胞减灭治疗。目前，一线细胞减灭治疗包括羟基脲或聚乙二醇化干扰素，一项比较这两种重要药物的III期临床试验正在进行中。阿那格雷和临床试验仅用于难治性或不耐受的患者。展望未来，包括Janus激酶2（JAK2）和端粒酶抑制剂在内的新疗法很有前景，可能会成为原发性血小板增多症患者治疗手段中的重要补充。
