Yamauchi Takuji
Medicine and Biosystemic Science, Kyushu University Graduate School of Medical Sciences.
Rinsho Ketsueki. 2025;66(7):597-610. doi: 10.11406/rinketsu.66.597.
The treatment of myeloproliferative neoplasms (MPNs) has advanced significantly in recent years, leading to a reassessment of therapeutic strategies for polycythemia vera (PV), essential thrombocythemia (ET), and myelofibrosis (MF). In PV, ropeginterferon alfa-2b has shown potential disease-modifying effects by reducing the JAK2 V617F allele burden. For ET, treatment remains focused on thrombosis prevention, with hydroxyurea as the mainstay, and novel agents such as pegylated interferon and bomedemstat (LSD1 inhibitor) are also under development. In MF, the evolution of JAK inhibitors is particularly noteworthy, with momelotinib and pacritinib showing potential benefits in alleviating anemia. Additionally, now that HemeSight is covered by Japanese national health insurance, genetic mutation analysis has become more accessible, paving the way for risk classification based on genetic profiling. This review provides a comprehensive update on the latest risk stratification and therapeutic strategies for MPNs, highlighting emerging treatments and their potential impact on disease management.
近年来，骨髓增殖性肿瘤（MPN）的治疗取得了显著进展，这导致了对真性红细胞增多症（PV）、原发性血小板增多症（ET）和骨髓纤维化（MF）治疗策略的重新评估。在PV中，聚乙二醇化干扰素α-2b通过降低JAK2 V617F等位基因负担显示出潜在的疾病修饰作用。对于ET，治疗仍集中在预防血栓形成上，羟基脲是主要药物，聚乙二醇化干扰素和博美司他（LSD1抑制剂）等新型药物也在研发中。在MF中，JAK抑制剂的发展尤其值得注意，莫洛替尼和帕西替尼在缓解贫血方面显示出潜在益处。此外，由于HemeSight已被纳入日本国民健康保险，基因突变分析变得更加容易获得，为基于基因谱的风险分类铺平了道路。本综述全面更新了MPN的最新风险分层和治疗策略，重点介绍了新兴治疗方法及其对疾病管理的潜在影响。
