一个基因组定制的多剂精密医学临床试验,用于患有复发性质母细胞瘤的成年人
Jiaying Chen1, Nancy Ann Oberheim Bush2, Jennifer A Grabowsky3
1University of California, San Francisco United States.
概括
一项1期临床试验探索了针对复发性质母细胞瘤 (rGBM) 的基因组定制多剂疗法. 虽然可行,但由于毒性限制,个性化方法并没有显著改善生存结果.
科学领域:
- 神经瘤学神经瘤学
- 基因组学就是基因组学.
- 临床试验 临床试验
背景情况:
- 复发性质母细胞瘤 (rGBM) 是一个重大的临床挑战,救援疗法选择有限.
- 现有的rGBM治疗方法提供了大约9个月的中位生存期.
- 质母细胞瘤 (GBM) 的分子异质性使单一向治疗变得无效.
研究的目的:
- 评估针对手术治疗复发性质母细胞瘤的个性化多剂治疗方案的可行性,安全性和有效性.
- 为了评估一个基因组定制的方法,在成年患者的rGBM.
主要方法:
- 一项1期临床试验招募了30名患有复发性质母细胞瘤 (IDH野生型或IDH突变型) 的成年人.
- 复发性瘤的全面基因组分析指导了个性化治疗方案的选择.
- 疗法结合了多达4种FDA批准的药物,包括细胞毒性脊柱,由分子瘤委员会确定.
主要成果:
- 在18种组合中使用了12种药物;罗穆斯丁,阿法替尼和阿贝马西克利布是最常见的治疗方案.
- 常见的毒性包括细胞衰竭和皮疹,需要频繁降低剂量.
- 6个月的无进展生存率为40%,9个月的总生存率为73%,平均总生存时间为12.7个月.
结论:
- 个性化,基因组定制的治疗方案是可行的手术切除的rGBM.
- 与历史对照相比,该研究没有显示出整体疗效的显著改善.
- 治疗毒性限制了组合疗法的全剂量,影响了潜在的疗效.
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