Division of Neuro- Oncology, Department of Neurological Surgery, University of California San Francisco, San Francisco, California 94143-0350, USA.
Curr Opin Neurol. 2012 Dec;25(6):780-5. doi: 10.1097/WCO.0b013e328359b45e.
Recent advances in survival for patients with newly diagnosed and recurrent brain tumors, combined with the development of an ever-increasing number of potential treatments, has led to significant growth in the number of clinical trials for patients with brain tumors. Suitable clinical trial design and endpoints are vital for successfully evaluating these new treatments that may continue to improve outcome. However, inadequacies of clinical trial endpoints have challenged how best to evaluate promising new therapeutics.
Pseudoprogression and pseudoresponse confound imaging-based endpoints, including overall radiographic response and progression-free survival. Overall survival is still regarded as the definitive endpoint, but recently identified active salvage agents such as bevacizumab may diminish the association between presalvage therapy and overall survival, making interpretation of clinical trial results difficult. Novel imaging and the assessment of patient function, quality of life (QOL), and cognition are more frequently employed as endpoints.
An awareness of the benefits and imperfections of clinical trial endpoints will lead to improved clinical trial design and results. Validated endpoints of patient function, QOL, and cognition are available and increasingly valued as secondary endpoints.
新诊断和复发性脑肿瘤患者的生存率最近有所提高，加上越来越多潜在治疗方法的发展，导致脑肿瘤患者的临床试验数量显著增加。合适的临床试验设计和终点对于成功评估这些可能继续改善疗效的新治疗方法至关重要。然而，临床试验终点的不足一直困扰着如何最好地评估有前途的新疗法。
假性进展和假性缓解混淆了基于影像学的终点，包括总体放射学反应和无进展生存期。总生存期仍然被认为是明确的终点，但最近确定的活性挽救剂，如贝伐单抗，可能会降低挽救治疗前与总生存期之间的关联，从而使临床试验结果的解释变得困难。新的影像学检查以及对患者功能、生活质量（QOL）和认知的评估更频繁地被用作终点。
了解临床试验终点的优点和缺陷将导致临床试验设计和结果的改进。患者功能、QOL 和认知的验证终点可用，并且越来越被视为次要终点而受到重视。
