Efficient Assessment of Progression in Clinical Trials in Retinitis Pigmentosa.

IF 2.3 2区医学 Q2 OPHTHALMOLOGY Retina-The Journal of Retinal and Vitreous Diseases Pub Date : 2024-12-03 DOI:10.1097/IAE.0000000000004360

Ryo Asaoka, Manabu Miyata, Akio Oishi, Yuri Fujino, Hiroshi Murata, Shuichiro Aoki, Keiko Azuma, Ryo Obata, Tatsuya Inoue

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Abstract

Purpose: To conduct a simulation study to evaluate the effect of disease stage, frequency, and clustering of visual field (VF) tests, and inclusion of one or both eyes on sample size calculation in clinical trials on retinitis pigmentosa.

Methods: A series of VFs were simulated on the basis of test-retest VF data in the early, moderate, and advanced stages of retinitis pigmentosa. VF measurements were scheduled 8 times in approximately 2 years. The probability to detect significant difference between treatment and control groups was measured. The investigation was conducted for 50%, 40%, 30%, 20% and 10% treatment effects.

Results: When only one eye was used in each patient with a treatment effect of 50%, 80% probability of significance was observed in the moderate stage when sample size was 70 eyes in each arm (140 eyes in both arms). Early stage disease and inclusion of both eyes decreased this number to 30 eyes (60 eyes in both arms); these decreasing effects were larger than performing additional VFs at the beginning and end of the observation. A similar tendency was observed with 40%, 30%, 20% and 10% treatment effects.

Conclusions: When planning a clinical trial, it is important to consider disease stage, in addition to VF schedule and inclusion of one or both eyes.

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色素性视网膜炎临床试验进展的有效评估。

目的：开展一项模拟研究，评估视网膜色素变性临床试验中疾病分期、频次、视野（VF）测试聚类以及单眼或双眼纳入对样本量计算的影响。方法：在色素性视网膜炎早期、中期和晚期复测VF数据的基础上，模拟一系列VF。VF测量计划在大约2年内进行8次。测量治疗组和对照组之间发现显著差异的概率。分别对50%、40%、30%、20%、10%的处理效果进行调查。结果：当每例患者只使用一只眼，治疗效果为50%时，在每组样本量为70只眼（两组140只眼）的中度阶段，观察到显著性概率为80%。早期疾病和双眼包括使这一数字减少到30只（双臂60只）；这些减少效应比在观察开始和结束时进行额外的VFs更大。40%、30%、20%和10%的治疗效果也有类似的趋势。结论：在计划临床试验时，除了VF计划和包括一只或两只眼睛外，重要的是考虑疾病分期。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Retina-The Journal of Retinal and Vitreous Diseases 医学-眼科学

CiteScore

5.70

自引率

9.10%

发文量

554

审稿时长

3-6 weeks

期刊介绍： RETINA® focuses exclusively on the growing specialty of vitreoretinal disorders. The Journal provides current information on diagnostic and therapeutic techniques. Its highly specialized and informative, peer-reviewed articles are easily applicable to clinical practice. In addition to regular reports from clinical and basic science investigators, RETINA® publishes special features including periodic review articles on pertinent topics, special articles dealing with surgical and other therapeutic techniques, and abstract cards. Issues are abundantly illustrated in vivid full color. Published 12 times per year, RETINA® is truly a “must have” publication for anyone connected to this field.