■JAK抑制剂(JAKis),用于治疗骨髓纤维化,已经进入标准治疗,提供显著改善脾脏大小和症状负担。尽管脾肿大可以减少和改善血细胞减少,还有一段路要走。正在研究新型JAKIS,以克服获得治疗的障碍,比如治疗挑战,不容忍,和反应迟钝。
■这篇综述包括JAKi治疗骨髓纤维化的现状,主要集中在研究JAKIS;jaktinib,列妥替尼,伊塔替尼,gandotinib,BMS-911543,伊吉纳替尼,TQ05105和马来酸氟诺替尼。对MEDLINE和clinicaltrials.gov进行了筛选,以确定有关该主题的所有已完成或活跃的研究。介绍并讨论了每种药物的临床前研究和临床试验的结果。
■在骨髓纤维化患者中,莫美替尼治疗贫血有效,而jaktinib对贫血和总症状评分(TSS)均有效.需要更多的3期研究来提供更精确的证据。越来越多的JAKis将在未来为骨髓纤维化提供更个性化的治疗选择。对疾病进展的潜在影响,分子反应,这种反应的持续时间将成为未来评估这些药物的重要参数。
UNASSIGNED: JAK inhibitors (JAKis), used in the treatment of myelofibrosis, have entered standard treatment, providing significant improvements in spleen size and symptom burden. Although splenomegaly provides a reduction and some improvement in cytopenia, there is still a way to go. Novel JAKis are being investigated to overcome barriers to treatment access, such as therapeutic challenges, intolerance, and unresponsiveness.
UNASSIGNED: This review includes the current status of JAKi treatment for myelofibrosis, mainly focusing on investigational JAKis; jaktinib,
lestaurtinib, itacitinib, gandotinib, BMS-911543, ilginatinib, TQ05105, and flonoltinib maleate. MEDLINE and clinicaltrials.gov were screened to identify all completed or active studies on this topic. The outcomes of the preclinical studies and clinical trials are presented and discussed for each drug.
UNASSIGNED: In patients with myelofibrosis, momelotinib was effective in treating anemia, whereas jaktinib was effective in both anemia and Total Symptom Score (TSS). More phase 3 studies are needed to provide more precise evidence. The increasing variety of JAKis will allow for more personalized treatment options for myelofibrosis in the future. The potential impact on disease progression, molecular responses, and the duration of this response will become important parameters for future evaluations of these drugs.