文章：

2018年原发性血小板增多症治疗流程图

Essential thrombocythemia treatment algorithm 2018

原文发布日期：2018-01-10

DOI: 10.1038/s41408-017-0041-8

类型: Current Treatment Algorithm

开放获取: 是

英文摘要：

Current drug therapy for myeloproliferative neoplasms, including essential thrombocythemia (ET) and polycythemia vera (PV), is neither curative nor has it been shown to prolong survival. Fortunately, prognosis in ET and PV is relatively good, with median survivals in younger patients estimated at 33 and 24 years, respectively. Therefore, when it comes to treatment in ET or PV, less is more and one should avoid exposing patients to new drugs that have not been shown to be disease-modifying, and whose long-term consequences are suspect (e.g., ruxolitinib). Furthermore, the main indication for treatment in ET and PV is to prevent thrombosis and, in that regard, none of the newer drugs have been shown to be superior to the time-tested older drugs (e.g., hydroxyurea). We currently consider three major risk factors for thrombosis (history of thrombosis, JAK2/MPL mutations, and advanced age), in order to group ET patients into four risk categories: “very low risk” (absence of all three risk factors); “low risk” (presence of JAK2/MPL mutations); “intermediate-risk” (presence of advanced age); and “high-risk” (presence of thrombosis history or presence of both JAK2/MPL mutations and advanced age). Herein, we provide a point-of-care treatment algorithm that is risk-adapted and based on evidence and decades of experience.

摘要翻译： 

目前针对骨髓增殖性肿瘤（包括原发性血小板增多症和真性红细胞增多症）的药物治疗既无法治愈，也尚未被证实能延长生存期。所幸的是，ET和PV患者的预后相对良好，年轻患者的中位生存期预计分别为33年和24年。因此，在ET和PV的治疗中，少即是多，应避免让患者使用尚未被证实能改变疾病进程、且长期效果存疑的新型药物（如鲁索替尼）。此外，ET和PV治疗的主要适应症是预防血栓形成，就此而言，尚无任何新型药物被证明优于经时间验证的传统药物（如羟基脲）。我们目前依据三大血栓形成危险因素（血栓病史、JAK2/MPL基因突变、高龄）将ET患者分为四个风险类别：极低危（无任何危险因素）、低危（存在JAK2/MPL突变）、中危（存在高龄因素）以及高危（有血栓病史或同时存在JAK2/MPL突变与高龄）。本文提出一套基于风险评估、循证医学证据及数十年临床经验打造的床旁治疗决策方案。

原文链接：

Essential thrombocythemia treatment algorithm 2018