Joussen Antonia M, Lehmacher Walter, Hilgers Ralf-Dieter, Kirchhof Bernd
Department of Ophthalmology, University of Düsseldorf, Düsseldorf, Germany.
Surv Ophthalmol. 2007 May-Jun;52(3):266-78. doi: 10.1016/j.survophthal.2007.02.010.
A large variety of new treatment options for different forms of age-related macular degeneration (ARMD) are becoming available. Not all new therapies may meet the expectations of patients and ophthalmologists. Despite the given statistical significant priority of treatment investigations, the endpoints may not be relevant to the patient's requirements. Therefore, questions inevitably arise regarding patient's benefit and the validity of the randomized controlled trials. The randomized controlled trial is regarded as the "gold standard" in terms of evaluating the effectiveness of interventions. The external validity of randomized controlled trials may be compromised, if, for example, patients assigned to the study group are unrepresentative of the reference population. This review aims to analyze problems with external validity in the randomized controlled trials on ARMD and surveys the endpoints of clinical studies with respect to the patient benefit.
针对不同类型的年龄相关性黄斑变性（ARMD），各种各样的新治疗方案正逐渐问世。并非所有新疗法都能满足患者和眼科医生的期望。尽管治疗研究在统计学上具有显著的优先性，但研究终点可能与患者需求无关。因此，不可避免地会出现关于患者获益以及随机对照试验有效性的问题。随机对照试验在评估干预措施的有效性方面被视为“金标准”。例如，如果分配到研究组的患者不能代表参考人群，随机对照试验的外部有效性可能会受到影响。本综述旨在分析ARMD随机对照试验中外部有效性的问题，并就患者获益情况调查临床研究的终点。
