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儿科临床试验中的人类测量排除:对营养不良儿童药物剂量的影响
Susan M Abdel-Rahman1, Sherbet Samuels1, Janie Cole2
1Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, USA.
Journal of clinical pharmacology
|October 13, 2025
概括
儿科临床试验经常排除营养不良的儿童,特别是营养不良的儿童. 这种排除限制了脆弱人群的数据,可能不反映真实世界的患者人口统计数据.
科学领域:
- 儿科临床试验儿童临床试验
- 药物开发 药物开发
- 营养科学 营养科学
背景情况:
- 营养不良在患有复杂疾病的儿童中很普遍,影响药物的疗效.
- 目前FDA批准的药物标签很少考虑儿童用药剂量中的营养不良.
- 临床试验中的排除标准可能会限制研究结果的概括性.
研究的目的:
- 调查儿科临床试验中与营养不良相关的排除标准的频率和性质.
- 确定特定的研究阶段,干预类型或适应症是否与更高的排除率有关.
- 评估这些标准对试验人群的影响及其对现实患者的代表性.
主要方法:
- 从ClinicalTrials.Gov.中对行业赞助的儿科I-III期临床试验协议进行系统审查.
- 对营养不良的人类指标和临床指标的纳入和排除 (I/E) 标准的分析.
- 根据研究阶段,干预类型和治疗指示对I/E标准的表征.
主要成果:
- 在1759个审查的协议中,616个 (35%) 包含了777个与营养不良相关的I/E标准.
- 71%的协议不包括营养不良的儿童,9%不包括营养不良的儿童,20%不包括两者.
- 排除在呼吸道,精神/行为和产科/产周症状中最常见;在皮肤学和瘤学研究中最不常见.
- 非特定的标准可能会导致低估基于营养不良的排斥.
结论:
- 儿科临床试验不成比例地排除营养不良的儿童,尽管人们越来越关注肥胖问题.
- 这些排除创造了可能不反映复杂疾病患者临床现实的试验人群.
- 应考虑在涉及高营养不良率条件的研究中修改I/E标准,以提高试验相关性.
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