Sanjak Mohammed, Salachas Francois, Frija-Orvoen Elizabeth, Theys Paul, Hutchinson Duncan, Verheijde Joseph, Pianta Thomas, Stewart Heather, Brooks Benjamin Rix, Meininger Vincent, Douillet Patrice
Carolinas Neuromuscular/ALS-MDA Center, Neuroscience and Spine Institute, Carolinas Medical Center, Department of Neurology, Charlotte, North Carolina 28207-1885, USA.
Amyotroph Lateral Scler. 2010 Aug;11(4):383-8. doi: 10.3109/17482960903486083.
Currently, ALS clinical trials require large sample size and the participation of many clinical evaluators to perform the outcome measure. High variability due to testers, instruments, or patients performance errors may result in systematic bias or random error leading to erroneous or uninterpretable results. Consequently, a quality control system that aims to produce high quality data in terms of reproducibility and accuracy to ensure reliability of the primary outcome measure is essential. In this paper we report our experience in preparing and executing a prospective quality control system that was implemented in conjunction with a large multicenter, multinational randomized placebo-controlled phase III clinical trial in ALS. We have shown that a prospective quality control system is highly effective to ensure inter- and intra-rater reliability of vital capacity as a primary outcome measure during the entire trial.
目前，肌萎缩侧索硬化症（ALS）的临床试验需要大样本量以及众多临床评估人员的参与来进行结果测量。由于测试人员、仪器或患者操作误差导致的高变异性可能会产生系统偏差或随机误差，从而导致结果错误或无法解释。因此，一个旨在产生高质量数据（在可重复性和准确性方面）以确保主要结果测量可靠性的质量控制系统至关重要。在本文中，我们报告了我们在准备和执行前瞻性质量控制系统方面的经验，该系统是与一项大型多中心、跨国、随机、安慰剂对照的ALS III期临床试验同时实施的。我们已经表明，前瞻性质量控制系统对于确保肺活量作为整个试验期间主要结果测量的评分者间和评分者内可靠性非常有效。
