Taylor Jennie W, Molinaro Annette M, Butowski Nicholas, Prados Michael
Department of Neurological Surgery, University of California San Francisco, San Francisco, California (J.W.T., A.M.M., N.B., M.P.).
Department of Neurology, University of California San Francisco, San Francisco, California (J.W.T.).
Neurooncol Pract. 2017 Dec;4(4):201-208. doi: 10.1093/nop/npw034. Epub 2017 Feb 14.
Malignant glioma represents a diverse set of molecularly heterogeneous diseases. Few therapeutic agents have been approved despite decades of clinical trials research and pre-clinical investigation. Attempts to refine neuroimaging criteria and recent discovery of the genomic profiles linking tumor subsets to survival outcomes have spurred discussion on a variety of new approaches in clinical trial design and relevant endpoints. Here we focus on those endpoints in clinical trial design for patients with primary glioma and related issues still to be resolved.
恶性胶质瘤代表了一系列分子异质性疾病。尽管经过了数十年的临床试验研究和临床前研究,但获批的治疗药物寥寥无几。完善神经影像学标准的尝试以及最近发现的将肿瘤亚群与生存结果相关联的基因组图谱,引发了关于临床试验设计中各种新方法及相关终点的讨论。在此,我们聚焦于原发性胶质瘤患者临床试验设计中的那些终点以及仍有待解决的相关问题。