文章：

真性红细胞增多症治疗流程图（2018年版）

Polycythemia vera treatment algorithm 2018

原文发布日期：2018-01-10

DOI: 10.1038/s41408-017-0042-7

类型: Current Treatment Algorithm

开放获取: 是

英文摘要：

Recently reported mature survival data have confirmed the favorable prognosis in polycythemia vera (PV), with an estimated median survival of 24 years, in patients younger than age 60 years old. Currently available drugs for PV have not been shown to prolong survival or alter the natural history of the disease and are instead indicated primarily for prevention of thrombosis. Unfortunately, study endpoints that are being utilized in currently ongoing clinical trials in PV do not necessarily target clinically or biologically relevant outcomes, such as thrombosis, survival, or morphologic remission, and are instead focused on components of disease palliation. Even more discouraging has been the lack of critical appraisal from “opinion leaders”, on the added value of newly approved drugs. Keeping these issues in mind, at present, we continue to advocate conservative management in low-risk PV (phlebotomy combined with once- or twice-daily aspirin therapy) and include cytoreductive therapy in “high-risk” patients; in the latter regard, our first, second, and third line drugs of choice are hydroxyurea, pegylated interferon-α and busulfan, respectively. In addition, it is reasonable to consider JAK2 inhibitor therapy, in the presence of protracted pruritus or markedly enlarged splenomegaly shown to be refractory to the aforementioned drugs.

摘要翻译： 

近期公布的成熟生存数据证实了真性红细胞增多症（PV）的良好预后，60岁以下患者的预估中位生存期可达24年。当前可用的PV药物尚未被证实能延长生存期或改变疾病自然病程，其主要适应症在于血栓预防。遗憾的是，目前正在进行的PV临床试验所采用的研究终点并未必然针对具有临床或生物学相关性的结局指标（如血栓形成、生存率或形态学缓解），而是聚焦于疾病姑息治疗的组成部分。更令人沮丧的是，"权威专家"对新获批药物附加价值的批判性评估始终缺位。鉴于这些问题，我们目前继续主张对低危PV采取保守治疗（放血术联合每日一次或两次的阿司匹林治疗），对"高危"患者则纳入细胞减灭疗法；就后者而言，我们首选的一线、二线和三线药物分别为羟基脲、聚乙二醇干扰素-α和白消安。此外，若患者出现持续性瘙痒或明显脾大且对上述药物耐药，考虑采用JAK2抑制剂治疗是合理的。

原文链接：

Polycythemia vera treatment algorithm 2018