在复发/复发的局部先进或转移的IDH1-突变性软骨瘤中使用olutasidenib:阶段1b/2试验
Robin L Jones1, Roman Groisberg2, Jean-Yves Blay3
1Royal Marsden Hospital/Institute of Cancer Research, London, UK. robin.jones4@nhs.net.
Nature communications
|January 29, 2026
概括
作为mIDH1抑制剂的olutasidenib在患有晚期冠状动脉瘤的患者中显示出疾病的控制. 药物耐受性很好,为这种罕见的癌症提供了新的选择,治疗方法有限.
科学领域:
- 在瘤学瘤学.
- 医学遗传学 医学遗传学
背景情况:
- 冠状腺瘤是一种罕见的骨癌,治疗选择很少.
- 异酸脱酶1/2 (mIDH1/2) 中的突变在65%的冠状腺癌中被发现,具有潜在的治疗标.
研究的目的:
- 为了评估olutasidenib的安全性和有效性,一个特定的mIDH1抑制剂.
- 评估olutasidenib对高级mIDH1软骨肉瘤患者的客观应答率,无进展生存率和整体生存率的影响.
主要方法:
- 一个I/II期临床试验 (NCT03684811) 招募了患有局部晚期或转移的mIDH1软骨瘤的患者.
- 奥卢塔西迪尼布每天两次服用150毫克的剂量.
- 瘤反应,不良事件,无进展生存率和整体生存率是初级和二级终点.
主要成果:
- 在21名可评估应答的患者中,52%的病情稳定,38%的病情进展.
- 中位数无进展生存期为2.0个月,中位数整体生存期为16.0个月.
- 在16名患有常规性肌肉瘤 (cCS) 患者的小组中,63%的患者患有稳定性疾病,中位数无进展生存时间为3.5个月,中位数整体生存时间为19.0个月.
结论:
- 在常规冠状腺癌患者中,olutasidenib耐受性良好,并证明了疾病的控制.
- 这项研究表明,olutasidenib可能是对mIDH1软骨肉瘤的有希望的治疗选择,尽管包括开放式设计和小样本大小在内的局限性.
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