Department of Medical Oncology, Bellaria-Maggiore Hospitals, Azienda USL, Bologna, Italy.
Eur J Cancer. 2012 Apr;48(6):896-903. doi: 10.1016/j.ejca.2011.10.027. Epub 2011 Nov 24.
The progression-free survival rate at 6 months (PFS-6) has long been considered the best end-point for assessing the efficacy of new agents in phase II trials in patients with recurrent glioblastoma. However, due to the introduction of antiangiogenic agents in this setting, and their intrinsic propensity to alter neuroradiological disease assessment by producing pseudoregression, any end-point based on neuroradiological modifications should be reconsidered. Further, statistically significant effects on progression-free survival (PFS) only should not automatically be considered reliable evidence of meaningful clinical benefit. In this context, because of its direct and unquestionable clinical relevance, overall survival (OS) represents the gold standard end-point for measuring clinical efficacy, despite the disadvantage that it is influenced by subsequent therapies and usually takes longer time to be evaluated. Therefore, while awaiting novel imaging criteria for response evaluation and/or new imaging tools to distinguish between 'true' and 'pseudo'-responses to antiangiogenic agents, the measurement of OS or OS rates should be considered primary end-points, also in phase II trials with these agents.
无进展生存期 6 个月（PFS-6）一直被认为是评估复发胶质母细胞瘤患者二线治疗新药物疗效的最佳终点。然而，由于在该环境中引入了抗血管生成药物，以及它们通过产生假性缓解而改变神经影像学疾病评估的固有倾向，任何基于神经影像学改变的终点都应重新考虑。此外，仅对无进展生存期（PFS）有统计学意义的影响不应被自动视为有意义的临床获益的可靠证据。在这种情况下，由于其直接和无可置疑的临床相关性，总生存期（OS）代表了衡量临床疗效的金标准终点，尽管它受到后续治疗的影响，并且通常需要更长的时间进行评估。因此，在等待新的影像反应评估标准和/或新的影像工具来区分抗血管生成药物的“真实”和“假性”反应的同时，OS 或 OS 率的测量应被视为主要终点，即使在这些药物的二期临床试验中也是如此。
