文章：

Janus激酶抑制剂治疗骨髓纤维化的疗效与耐受性：一项系统评价与网状Meta分析

Efficacy and tolerability of Janus kinase inhibitors in myelofibrosis: a systematic review and network meta-analysis

原文发布日期：2021-07-27

DOI: 10.1038/s41408-021-00526-z

类型: Article

开放获取: 是

英文摘要：

Myelofibrosis is a myeloproliferative neoplasm associated with constitutional symptoms, increasing splenomegaly, and worsening cytopenias. Janus kinase (JAK) inhibitors have been used for the treatment of myelofibrosis for several years, but there is a lack of comparative information between those treatments. A systematic review and network meta-analysis was performed on randomized controlled trials in patients with myelofibrosis receiving JAK inhibitor or placebo or control. Primary outcomes were efficacy on spleen volume reduction and total symptom score reduction. Additional analyses were conducted on anemia and thrombopenia events. Seven studies were included in the network meta-analysis including 1953 patients randomly assigned to four JAK inhibitors—ruxolitinib, fedratinib, pacritinib, momelotinib—or control. In first-line therapy, momelotinib and fedratinib were associated with comparable efficacy to ruxolitinib, and with less toxicity on erythrocytes and platelets, respectively. Pacritinib was less effective on splenomegaly than ruxolitinib as a first-line treatment but seemed effective in second line, after ruxolitinib exposure. Fedratinib and ruxolitinib that are FDA approved in myelofibrosis have both confirmed being valuable option to treat splenomegaly and constitutional symptoms, and their slightly different tolerance-profiles can guide therapeutic choice for first-line treatment, according to patient profile. Momelotinib could be another option especially due to its positive effect on anemia.
 

摘要翻译： 

骨髓纤维化是一种骨髓增殖性肿瘤，常伴随全身症状、进行性脾肿大及加重的血细胞减少。Janus激酶（JAK）抑制剂用于治疗骨髓纤维化已有数年，但不同治疗方案之间缺乏比较性数据。本研究对接受JAK抑制剂、安慰剂或对照治疗的骨髓纤维化患者的随机对照试验进行了系统评价和网络荟萃分析，主要评估指标为脾体积缩小和症状总分改善的疗效，并对贫血和血小板减少事件进行了补充分析。

网络荟萃分析共纳入7项研究，涉及1953例随机分配至四种JAK抑制剂（鲁索替尼、菲卓替尼、帕克替尼、莫莫替尼）或对照组的患者。在一线治疗中，莫莫替尼和菲卓替尼的疗效与鲁索替尼相当，且分别对红细胞和血小板的毒性更低。帕克替尼在一线治疗中对脾肿大的疗效弱于鲁索替尼，但在鲁索替尼治疗后作为二线方案显示有效。经美国食品药品监督管理局批准的菲卓替尼和鲁索替尼均证实对治疗脾肿大及全身症状具有重要价值，二者略有差异的耐受性特征可根据患者个体情况指导一线治疗选择。莫莫替尼因其对贫血的积极作用，也可能成为另一种治疗选择。

原文链接：

Efficacy and tolerability of Janus kinase inhibitors in myelofibrosis: a systematic review and network meta-analysis