Polycythemia vera: historical oversights, diagnostic details, and therapeutic views.
Polycythemia vera: historical oversights, diagnostic details, and therapeutic views.
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DOI：
10.1038/s41375-021-01401-3
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发表时间：
2021-12
期刊：
影响因子：
11.4
通讯作者：
Barbui T
中科院分区：
文献类型：
--
作者：
Tefferi A;Vannucchi AM;Barbui T
真性红细胞增多症(PV)是一种相对迟缓的髓系肿瘤，年轻患者的中位生存期超过35年，但其自然病程可能被血栓形成、纤维化或白血病事件中断，其20年生存率分别为26%、16%和4%。目前PV的治疗策略并未被证明能延长生存期或降低白血病或纤维化进展的风险，而是针对预防血栓并发症。在后一方面，考虑了两种风险类别：高风险(年龄和60岁或血栓病史)和低风险(两种风险因素都不存在)。在没有禁忌症的情况下，所有患者都需要静脉采血将红细胞压积保持在45%以下，并每天服用一次小剂量阿司匹林。细胞减少性治疗被推荐用于高风险或有症状的低风险疾病；在这方面，我们选择的一线药物是羟基尿素，但我们认为聚乙二醇化干扰素在某些情况下是一种替代方案，包括在某些情况下，包括在育龄年轻女性中，在表现出对羟基尿素治疗不耐受或抵抗的患者中，以及在表明治疗是为了限制静脉采血要求而不是预防血栓形成的情况下。其他治疗方案包括白消安和鲁索利替尼；前者在老年患者中首选，后者在出现让人联想到PV后骨髓纤维化或长期瘙痒的症状时首选。我们的药物选择反映了我们对安全性的长期记录、降低血栓形成风险的证据以及更广泛的骨髓增殖抑制的赞赏。需要对照研究来阐明每天服用两次与一次阿司匹林和直接口服抗凝剂的附加值。在这篇受邀的综述中，我们讨论了我们目前诊断、预测和治疗PV的一般方法，以及特殊情况下的方法，包括妊娠和内脏静脉血栓形成。
Polycythemia vera (PV) is a relatively indolent myeloid neoplasm with median survival that exceeds 35 years in young patients, but its natural history might be interrupted by thrombotic, fibrotic, or leukemic events, with respective 20-year rates of 26%, 16%, and 4%. Current treatment strategies in PV have not been shown to prolong survival or lessen the risk of leukemic or fibrotic progression and instead are directed at preventing thrombotic complications. In the latter regard, two risk categories are considered: high (age >60 years or thrombosis history) and low (absence of both risk factors). All patients require phlebotomy to keep hematocrit below 45% and once-daily low-dose aspirin, in the absence of contraindications. Cytoreductive therapy is recommended for high-risk or symptomatic low-risk disease; our first-line drug of choice in this regard is hydroxyurea but we consider pegylated interferon as an alternative in certain situations, including in young women of reproductive age, in patients manifesting intolerance or resistance to hydroxyurea therapy, and in situations where treatment is indicated for curbing phlebotomy requirement rather than preventing thrombosis. Additional treatment options include busulfan and ruxolitinib; the former is preferred in older patients and the latter in the presence of symptoms reminiscent of post-PV myelofibrosis or protracted pruritus. Our drug choices reflect our appreciation for long-term track record of safety, evidence for reduction of thrombosis risk, and broader suppression of myeloproliferation. Controlled studies are needed to clarify the added value of twice- vs once-daily aspirin dosing and direct oral anticoagulants. In this invited review, we discuss our current approach to diagnosis, prognostication, and treatment of PV in general, as well as during specific situations, including pregnancy and splanchnic vein thrombosis.