Shikhbabaeva D I, Shuvaev V A, Martynkevich I S, Zyuzgin I S, Abdulkadyrov K M
Vopr Onkol. 2016;62(4):386-93.
The discovery of the JAK2V617F mutation was the beginning of a new era in the study of myeloproliferative neoplasms (MPN). In addition to contributing to the understanding of the pathophysiology of Ph-negative MPN, JAK2 mutation has become a new therapeutic target in their treatment. In treatment of PV a new era began the era of targeted therapy, which gave a hope for better treatment outcomes and improved quality of life for patients who are resistant to standard therapy. This work presents literature data on molecular-genetic features of the pathogenesis of polycythemia vera (PV) and new possibilities in the treatment of this disease, literature review about JAKinhibitors, targeted therapy of PV. There are reviewed issues on resistance and intolerance of hydroxycarbamide and interferon (IFN-a) and the definition of the indications for administration of JAK-inhibitors. There are presented data on the efficacy and safety of ruxolitinib, which were proven within the clinical trial RESPONSE.
JAK2V617F 突变的发现开启了骨髓增殖性肿瘤（MPN）研究的新纪元。除了有助于理解 Ph 阴性 MPN 的病理生理学外，JAK2 突变已成为其治疗的新靶点。在真性红细胞增多症（PV）的治疗中，一个新的时代——靶向治疗时代开始了，这为那些对标准治疗耐药的患者带来了更好治疗效果和改善生活质量的希望。本文介绍了关于真性红细胞增多症（PV）发病机制的分子遗传学特征的文献数据以及该疾病治疗的新可能性，关于 JAK 抑制剂的文献综述，PV 的靶向治疗。文中回顾了羟基脲和干扰素（IFN-α）的耐药性和不耐受性问题以及 JAK 抑制剂给药指征的定义。还展示了在临床试验 RESPONSE 中得到证实的鲁索替尼的疗效和安全性数据。
