a Department of Neurology , Baylor College of Medicine , Houston , Texas , USA.
b Department of Radiation Oncology , The University of Texas MD Anderson Cancer Center , Houston , Texas , USA.
Expert Rev Anticancer Ther. 2018 Oct;18(10):1031-1040. doi: 10.1080/14737140.2018.1496824. Epub 2018 Jul 12.
Several recent phase III studies have attempted to improve the dismal survival seen in glioblastoma patients, with disappointing results despite prior promising phase II data. Areas covered: A literature review of prior phase II and phase III studied in glioblastoma was performed to help identify possible areas of concern. Numerous issues in previous phase II trials for glioblastoma were found that may have contributed to these discouraging outcomes and discordant results. Expert commentary: These concerns include the improper selection of therapeutics warranting investigation in a phase III trial, suboptimal design of phase II studies (often lacking a control arm), absence of molecular data, the use of imaging criteria as a surrogate endpoint, and a lack of pharmacodynamic testing. Hopefully, by recognizing prior phase II trial limitations that contributed to failed phase III trials, we can adapt quickly to improve our ability to accurately discover survival-prolonging treatments for glioblastoma patients.
几项最近的 III 期研究试图改善胶质母细胞瘤患者的悲惨生存状况，但尽管 II 期数据有希望，结果却令人失望。
对胶质母细胞瘤的 II 期和 III 期研究进行了文献回顾，以帮助确定可能关注的领域。在以前的胶质母细胞瘤 II 期试验中发现了许多问题，这些问题可能导致了这些令人沮丧的结果和不一致的结果。
这些问题包括在 III 期试验中需要研究的治疗方法选择不当、II 期研究设计不佳（通常缺乏对照组）、缺乏分子数据、将影像学标准作为替代终点以及缺乏药效学检测。希望通过认识到导致 III 期试验失败的 II 期试验的局限性，我们能够迅速适应，提高准确发现延长胶质母细胞瘤患者生存时间的治疗方法的能力。
