Waggoner Matthew
From Arizona School of Health Sciences, Mesa, Arizona.
J Adv Pract Oncol. 2023 Jul;14(5):405-413. doi: 10.6004/jadpro.2023.14.5.5. Epub 2023 Jul 1.
Polycythemia vera is a Philadelphia chromosome-negative myeloproliferative neoplasm that results in increased myeloproliferation. It is a debilitating disease characterized by the overproduction of red blood cells, but it also can result in increased white blood cells and platelets. Patients experience a shortened overall survival due to an increased risk of thrombotic events, including stroke, myocardial infarction, pulmonary embolism, and deep vein thrombosis. Current treatment strategies in clinical practice are driven by mitigating the risk of these thrombotic events by reducing patients' hematocrit. In addition to thrombosis risk, polycythemia vera patients have constitutional symptoms such as fatigue, itching, bone pain, erythromelalgia, and splenomegaly. An increased risk of transformation of their disease to acute myeloid leukemia and/or myelofibrosis can also affect long-term survival in polycythemia vera. Additional research has identified other risk factors, such as increased white blood cells, increased platelet count, and cytokine levels, which can alter the prognosis of the disease. In this review, we will discuss the current treatment strategies in polycythemia vera and determine if incorporating additional biomarkers as endpoints is feasible in clinical practice.
真性红细胞增多症是一种费城染色体阴性的骨髓增殖性肿瘤，会导致骨髓增殖增加。它是一种使人衰弱的疾病，其特征是红细胞过度生成，但也可导致白细胞和血小板增多。由于包括中风、心肌梗死、肺栓塞和深静脉血栓形成在内的血栓形成事件风险增加，患者的总生存期缩短。临床实践中的当前治疗策略是通过降低患者的血细胞比容来降低这些血栓形成事件的风险。除了血栓形成风险外，真性红细胞增多症患者还有诸如疲劳、瘙痒、骨痛、红斑性肢痛症和脾肿大等全身性症状。其疾病转化为急性髓细胞白血病和/或骨髓纤维化的风险增加也会影响真性红细胞增多症患者的长期生存。更多研究已确定了其他风险因素，如白细胞增多、血小板计数增加和细胞因子水平，这些因素可改变疾病的预后。在本综述中，我们将讨论真性红细胞增多症的当前治疗策略，并确定在临床实践中纳入额外的生物标志物作为终点是否可行。
