van Genderen P J, Troost M M
Department of Internal Medicine, Havenziekenhuis, Rotterdam, The Netherlands.
Drugs Aging. 2000 Aug;17(2):107-19. doi: 10.2165/00002512-200017020-00003.
Essential thrombocythaemia (ET) and polycythaemia vera (PV) both belong to the group of chronic myeloproliferative disorders which originate at the haematopoietic stem cell level with retention of their cellular differentiating capacity. The clinical course of both ET and PV is frequently complicated by potentially life-threatening thrombotic events and a variable rate of progression to myelofibrosis and leukaemic conversion (the latter in particular for PV). However, due to the relative paucity of randomised clinical trials in both ET and PV, a sound scientific basis for making therapeutic decisions is lacking. The management of patients with ET or PV should probably be addressed by categorising patient populations into groups with either a 'low risk' or 'high risk' for thrombosis after taking into account the risk factors for thrombosis (advanced age and/or previous thrombotic complications, and increased haematocrit). The goal of treatment in PV and ET is, however, not only to reduce the risk of thrombosis but also to reduce the risk of transformation into acute myeloid leukaemia or myelofibrosis. In this review we will discuss in detail the diagnosis, clinical manifestations and epidemiology, rationale for treatment, and the various treatment options for ET and PV with an emphasis on treatment efficacy and therapy-related leukaemic risk.
原发性血小板增多症（ET）和真性红细胞增多症（PV）均属于慢性骨髓增殖性疾病，起源于造血干细胞水平，且保留了细胞分化能力。ET和PV的临床病程常常因潜在的危及生命的血栓形成事件以及进展为骨髓纤维化和白血病转化的不同发生率（尤其是PV会发生白血病转化）而变得复杂。然而，由于ET和PV的随机临床试验相对较少，缺乏做出治疗决策的可靠科学依据。在考虑血栓形成的危险因素（高龄和/或既往血栓形成并发症以及血细胞比容升高）后，将ET或PV患者群体分为血栓形成“低风险”或“高风险”组，可能有助于解决对这些患者的管理问题。然而，PV和ET的治疗目标不仅是降低血栓形成风险，还包括降低转化为急性髓系白血病或骨髓纤维化的风险。在本综述中，我们将详细讨论ET和PV的诊断、临床表现和流行病学、治疗原理以及各种治疗选择，重点关注治疗效果和治疗相关的白血病风险。
