[Recent findings and advances in treatment of myeloproliferative neoplasms].

Yuka Sugimoto
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Abstract

Many novel agents have been developed for BCR::ABL1-negaive myeloproliferative neoplasms (MPN), namely, polycythemia vera (PV), essential thrombocythemia (ET), and primary myelofibrosis (PMF). Some of these agents not only achieve hematologic complete response, reduce spleen size, and alleviate constitutional symptoms, but also induce molecular response, which means that they reduce the allele burden of driver gene mutations. These agents also prevent and alleviate fibrosis in bone marrow, which reduces the incidence of thrombotic events and disease progression and might improve prognosis. This article discusses the latest findings and promising treatments, including ongoing clinical trials, in PV, ET, and PMF.

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[骨髓增生性肿瘤治疗的最新发现和进展]。
针对BCR::ABL1-negaive骨髓增殖性肿瘤(MPN),即真性红细胞增多症(PV)、原发性血小板增多症(ET)和原发性骨髓纤维化(PMF),已开发出许多新型药物。其中一些药物不仅能获得血液学完全应答、缩小脾脏体积和缓解体征,还能诱导分子应答,即减少驱动基因突变的等位基因负荷。这些药物还能预防和缓解骨髓纤维化,从而降低血栓事件和疾病进展的发生率,并可能改善预后。本文讨论了针对骨髓增生性白血病、骨髓移植和骨髓纤维化的最新发现和有前景的治疗方法,包括正在进行的临床试验。
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