Myeloproliferative neoplasms: recent progresses in therapy
Myeloproliferative neoplasms: recent progresses in therapy
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骨髓增生性肿瘤：治疗的最新进展
DOI：
10.11406/rinketsu.59.741
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发表时间：
2018
期刊：
影响因子：
--
通讯作者：
下田 和哉
中科院分区：
文献类型：
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作者：
上運天 綾子;幣 光太郎;下田 和哉
真性红细胞增多症（PV）和原发性血小板增多症（ET）患者的预期生存时间并不明显低于普通人群。目前的治疗目标是预防与PV和ET相关的血栓出血性事件。目前PV的一线治疗是静脉切开术、羟基脲（HU）和阿司匹林，而ET的治疗是HU或阿纳格雷德。随访的3期随机试验评估了接受ropeg干扰素α-2b（下一代单聚干扰素α-2b或HU）治疗的PV患者的血液学反应，结果显示，接受ropeg干扰素治疗的患者具有优越的血液学效果和较低的不良事件发生率。原发性骨髓纤维化（PMF）的预后比PV或ET差。PMF患者唯一可治愈的治疗选择是异体造血干细胞移植（HSCT）。除了HSCT选择，ruxolitinib改善脾肿大和mf相关症状，并提供中2或高风险PMF患者的总体生存获益。几种不同的JAK抑制剂已经被开发出来；然而，由于毒性问题，其中许多已经停产。近年来，不同的JAK抑制剂以及直接靶向贫血和骨髓纤维化的药物的效果已经得到了可喜的结果。
The expected survival duration of polycythemia vera (PV) and essential thrombocythemia (ET) patients is not substantially lower than that of the general population. The current goal of therapy is to prevent thrombohemorrhagic events associated with PV and ET. The current first line therapy for PV is phlebotomy, hydroxyurea (HU), and aspirin, while that for ET was HU or anagrelide. The follow-up phase 3 randomized trial wherein the hematological response was evaluated in PV patients treated with ropeginterferon alfa-2b, a next-generation monopegylated IFN-α-2b, or HU, demonstrated a superior hematological effect and a lower incidence of adverse events in patients who were treated with ropeginterferon. The prognosis of primary myelofibrosis (PMF) is poorer than that of PV or ET. The only curative therapeutic option for PMF patients is allogeneic hematopoietic stem cell transplantation (HSCT). Other than HSCT options, ruxolitinib ameliorates splenomegaly and MF-associated symptoms and provides an overall survival benefit in PMF patients with intermediate-2 or high risk. Several different JAK inhibitors have been developed; however, many of them were discontinued because of toxicity concerns. Recently, promising results have been demonstrated for the effect of different JAK inhibitors as well as the drugs that directly target anemia and bone marrow fibrosis.