用DMT治疗多发性硬化症的III期临床试验的最佳实践:对已发表的试验进行系统分析和评估
Marta Mascarenas-Garcia1,2, Alejandro Rivero-de-Aguilar3,4, Mónica Pérez-Ríos1,5,6
1Preventive Medicine and Public Health, University of Santiago de Compostela, Santiago de Compostela, Spain.
Journal of neurology, neurosurgery, and psychiatry
|August 4, 2023
概括
对III期多发性硬化症 (MS) 临床试验的批判性评估揭示了方法和报告的经常性局限性. 解决这些问题对于提高MS疾病修饰疗法 (DMT) 研究的质量和有效性至关重要.
科学领域:
- 神经学 神经学
- 临床试验方法论 临床试验方法论
背景情况:
- 多发性硬化症 (MS) 治疗由于随机临床试验 (RCT) 而有显著的进展.
- 在MS中用于疾病修饰疗法 (DMT) 的III期RCT对于治疗开发至关重要.
研究的目的:
- 批判性地评估2010年以后发布的MS的DMT的III期RCT.
- 确定MS临床试验设计和报告的改进关键领域.
主要方法:
- 在2010年1月至2021年12月期间发表的MS RCT的系统搜索.
- 评估使用基于现有工具和MS特定指南的14项特设工具,涵盖方法质量,结果,不良事件,适用性和透明度.
主要成果:
- 评估了31个第三阶段RCT.
- 在样本大小,随机化,盲目化,参与者选择和不良事件报告方面高度遵守.
- 参与者描述,比较,消耗偏见,结果充分性,适用性,透明度和利益冲突的遵守程度较低. 没有一项试验符合结果分析和报告的要求.
- 常见的局限性包括缺乏并发症数据,不合理的安慰剂使用,不充分的结果测量,以及缺乏协议注册.
结论:
- 在MS中用于DMT的RCT显示出显著和反复出现的局限性.
- 需要提高质量,透明度和外部有效性.
- 解决发现的局限性将提高MS治疗研究的可靠性和适用性.
相关概念视频
Clinical Trials: Overview
3.0K
Clinical development focuses on how the drug will interact with the human body and encompasses four key phases of clinical trials, each serving a specific purpose in assessing the safety and effectiveness of new drugs. These phases overlap and build upon one another. Phase I involves a small group of healthy volunteers (typically 20-80 individuals) or, in cases where significant toxicity is expected, patients with the targeted disease, such as cancer or AIDS. The volunteers are tested for...
3.0K
Clinical Trials
6.9K
Clinical trials are prospective experimental studies conducted on humans to determine the safety and efficacy of treatments, drugs, diet methods, and medical devices. Using statistics in clinical trials enables researchers to derive reasonable and accurate conclusions from the collected data, allowing them to make wise decisions in uncertain situations. In medical research, statistical methods are crucial for preventing errors and bias.
There are four phases in a clinical trial. A phase one...
There are four phases in a clinical trial. A phase one...
6.9K


