在复发的H3 K27M突变扩散中线质瘤中,ONC201 (多达维)
Isabel Arrillaga-Romany1, Sharon L Gardner2, Yazmin Odia3
1Massachusetts General Hospital, Boston, MA.
概括
在复发的H3 K27M突变扩散中线质瘤 (DMG) 中,ONC201单一疗法表现出持久的疗效. 这种耐受良好的治疗方法在患有这种侵袭性脑瘤的患者中显示出临床上有意义的反应.
科学领域:
- 神经瘤学神经瘤学
- 分子瘤学分子瘤学
- 临床试验中的临床试验.
背景情况:
- 有H3 K27M突变的扩散中线质瘤 (DMG) 的预后不好.
- 对复发性DMG的有效治疗方法有限,放射治疗是主要的治疗方法.
研究的目的:
- 评估单剂ONC201 (多达维) 在患有复发性H3 K27M突变DMG的患者的疗效和安全性.
- 评估总体响应率 (ORR),响应持续时间 (DOR) 和响应时间 (TTR) 作为主要和次要终点.
主要方法:
- 一项综合分析包括50名患有复发H3 K27M突变DMG的患者,这些患者接受了口服ONC201单疗法.
- 患者符合神经瘤学响应评估 (RANO) 高度质瘤 (HGG) 标准,并具有足够的绩效评分.
- 放射性终点通过盲目独立的中央审查来评估.
主要成果:
- 根据RANO-HGG标准的ORR为20.0%,中位数DOR为11.2个月,中位数TTR为8.3个月.
- 合并的RANO-HGG/LGG ORR为30.0%. 这是一个非常好的结果.
- 观察到显著的皮质类固醇剂量减少 (46.7%) 和性能得分改善 (20.6%);治疗耐受性良好,没有4级不良事件.
结论:
- 在单一治疗中,ONC201证明了在复发的H3 K27M突变DMG中具有持久性和临床意义的疗效.
- 该疗法耐受性良好,这表明它对这一具有挑战性的患者群体具有治疗选择的潜力.
相关概念视频
02:57Targeted Cancer Therapies
7.6K
The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
There are several types of targeted therapies against...
7.6K
02:49Cancer Therapies
7.7K
Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...
7.7K