University of Maryland Greenebaum Cancer Center, Baltimore, MD, USA.
Expert Rev Hematol. 2014 Jun;7(3):325-32. doi: 10.1586/17474086.2014.905200. Epub 2014 Apr 21.
The recent discovery of the JAK2 mutation and its role in the pathogenesis of myeloproliferative neoplasms led to the development of a novel class of therapeutic agents, the oral JAK2 inhibitors. These agents are effective in decreasing organomegaly and ameliorating constitutional symptoms in patients with myelofibrosis, regardless of the mutational status. Among this new class of agents is pacritinib, a dual JAK2 and FLT3 inhibitor that showed evidence of clinical efficacy in early-phase trials of patients with myelofibrosis, with limited hematologic toxicity.
最近发现 JAK2 突变及其在骨髓增殖性肿瘤发病机制中的作用,导致了一类新型治疗药物的发展,即口服 JAK2 抑制剂。这些药物在减少骨髓纤维化患者的器官肿大和改善全身症状方面非常有效,无论突变状态如何。在这一新类药物中,有帕克里替尼,一种双重 JAK2 和 FLT3 抑制剂,在骨髓纤维化患者的早期临床试验中显示出了临床疗效的证据,且血液学毒性有限。