Moseley Merrick J, Wallace Michael P, Stephens David A, Fielder Alistair R, Smith Laura C, Stewart Catherine E
Division of Optometry and Visual Science, City University, London, EC1V 0HB, UK.
Department of Epidemiology, Biostatistics and Occupational Health, McGill University, Montreal, Canada.
Trials. 2015 Apr 25;16:189. doi: 10.1186/s13063-015-0711-4.
Amblyopia is the commonest visual disorder of childhood in Western societies, affecting, predominantly, spatial visual function. Treatment typically requires a period of refractive correction ('optical treatment') followed by occlusion: covering the nonamblyopic eye with a fabric patch for varying daily durations. Recent studies have provided insight into the optimal amount of patching ('dose'), leading to the adoption of standardized dosing strategies, which, though an advance on previous ad-hoc regimens, take little account of individual patient characteristics. This trial compares the effectiveness of a standardized dosing strategy (that is, a fixed daily occlusion dose based on disease severity) with a personalized dosing strategy (derived from known treatment dose-response functions), in which an initially prescribed occlusion dose is modulated, in a systematic manner, dependent on treatment compliance.
METHODS/DESIGN: A total of 120 children aged between 3 and 8 years of age diagnosed with amblyopia in association with either anisometropia or strabismus, or both, will be randomized to receive either a standardized or a personalized occlusion dose regimen. To avoid confounding by the known benefits of refractive correction, participants will not be randomized until they have completed an optical treatment phase. The primary study objective is to determine whether, at trial endpoint, participants receiving a personalized dosing strategy require fewer hours of occlusion than those in receipt of a standardized dosing strategy. Secondary objectives are to quantify the relationship between observed changes in visual acuity (logMAR, logarithm of the Minimum Angle of Resolution) with age, amblyopia type, and severity of amblyopic visual acuity deficit.
This is the first randomized controlled trial of occlusion therapy for amblyopia to compare a treatment arm representative of current best practice with an arm representative of an entirely novel treatment regimen based on statistical modelling of previous trial outcome data. Should the personalized dosing strategy demonstrate superiority over the standardized dosing strategy, then its adoption into routine practice could bring practical benefits in reducing the duration of treatment needed to achieve an optimal outcome.
ISRCTN ISRCTN12292232.
在西方社会，弱视是儿童中最常见的视觉障碍，主要影响空间视觉功能。治疗通常需要一段时间的屈光矫正（“光学治疗”），随后进行遮盖：用织物贴片遮盖非弱视眼，每天遮盖不同时长。最近的研究深入了解了最佳遮盖量（“剂量”），从而采用了标准化给药策略，这虽然比以前的临时方案有所进步，但很少考虑个体患者特征。本试验比较标准化给药策略（即根据疾病严重程度确定固定的每日遮盖剂量）与个性化给药策略（源自已知的治疗剂量反应函数）的有效性，在个性化给药策略中，初始规定的遮盖剂量会根据治疗依从性进行系统调整。
方法/设计：总共120名年龄在3至8岁之间、被诊断为伴有屈光参差或斜视或两者皆有的弱视儿童，将被随机分配接受标准化或个性化遮盖剂量方案。为避免屈光矫正已知益处造成的混淆，参与者在完成光学治疗阶段之前不会被随机分组。主要研究目标是确定在试验终点时，接受个性化给药策略的参与者所需的遮盖小时数是否少于接受标准化给药策略的参与者。次要目标是量化观察到的视力变化（最小分辨角对数，logMAR）与年龄、弱视类型和弱视视力缺陷严重程度之间的关系。
这是第一项针对弱视遮盖疗法的随机对照试验，将代表当前最佳实践的治疗组与基于先前试验结果数据的统计模型的全新治疗方案代表组进行比较。如果个性化给药策略显示出优于标准化给药策略，那么将其应用于常规实践可能会在减少实现最佳结果所需的治疗持续时间方面带来实际益处。
ISRCTN ISRCTN12292232
