Bonnetain F
Unité de Biostatistiques et d'Epidémiologie, EA 4184, Plateforme de Recherche Clinique Qualité de vie et Cancer, Centre Georges-François-Leclerc, 1 rue du Pr-Marion, 21079 Dijon cedex, France.
Cancer Radiother. 2010 Oct;14(6-7):515-8. doi: 10.1016/j.canrad.2010.06.001. Epub 2010 Jul 31.
In order to perform a clinical trial in oncology, the choice of the primary endpoint is a major concern, not only for sample size calculation but also regarding the clinical meaning of the results which represents treatment utility for patients or public health system. Endpoints could be classified in two categories: "clinical endpoints" including overall survival and health related quality of life (QoL); "biological markers" including, for example, progression free survival (PFS). If conceptual and methodological limits of quality of life are well identified with dedicated researches, limits of progression free survival remain less targeted and known by clinicians. Numerous clinical trials having only demonstrated a progression free survival benefit could be discussed according to clinical benefit. In this context, quality of life constitutes an available and a useful alternative endpoint for trials to ensure treatment benefit in a patient or public health point of view. As quality of life, methodological researches should be pursued to improve scientific robustness for assessments and analyses of progression free survival.
为了开展肿瘤学临床试验，主要终点的选择是一个主要关注点，这不仅关乎样本量计算，还涉及结果的临床意义，而结果代表了对患者或公共卫生系统的治疗效用。终点可分为两类：“临床终点”，包括总生存期和健康相关生活质量（QoL）；“生物标志物”，例如无进展生存期（PFS）。如果通过专门研究能很好地确定生活质量的概念和方法学局限，那么无进展生存期的局限则较少受到临床医生的关注和了解。许多仅证明有无进展生存期获益的临床试验，从临床获益角度来看可能存在争议。在此背景下，从患者或公共卫生角度确保治疗获益而言，生活质量构成了一种可行且有用的替代终点。与生活质量一样，应开展方法学研究以提高无进展生存期评估和分析的科学稳健性。
