大多数患有功能性便秘的儿童是否符合常用的临床试验终点?
Samantha Arrizabalo1, Carlos Alberto Velasco-Benitez2, Daniela Alejandra Velasco-Suarez2
1Department of Pediatrics Gastroenterology, Hepatology, University of Miami, Miami, FL 33136, USA.
功能性便秘 (FC) 在儿童中很常见,但临床试验往往忽略了关键症状,如疼痛的肠道排便. 这项研究发现,试验终点可能不反映儿科FC患者的现实症状分布.
科学领域:
- 儿科胃肠病学 儿科胃肠病学
- 肠-大脑相互作用障碍 肠-大脑相互作用障碍
- 临床试验方法论 临床试验方法论
背景情况:
- 功能性便秘 (FC) 诊断依赖于罗马四号标准,要求至少有七种症状中的两种.
- 对FC的常见临床试验终点包括肠道排便频率,便一致性和便失禁.
- 关于这些终点如何代表FC儿童的症状分布的数据有限.
研究的目的:
- 在一个大规模的,基于社区的儿童样本中,评估每个"罗马四号"标准的频率.
- 确定常用的临床试验终点是否准确地反映了儿科FC的症状分布.
- 确定临床试验实践与现实世界症状患病率之间的潜在差异.
主要方法:
- 一项涉及哥伦比亚七个城市6611名8至18岁的学龄儿童的横截面研究.
- 参与者填写了儿科胃肠道症状罗马四号问卷 (QPGS-IV).
- 分析包括FC的患病率以及诊断标准的分布和组合.
主要成果:
- 功能性便秘 (FC) 在12.8%的儿童中被诊断为最常见的肠-大脑相互作用障碍.
- 最常见的罗马IV标准是不频繁的便 (<2周,66.1%) 和疼痛的肠道排便 (65%).
- 便失禁是不常见的 (6.9%),而60.5%的人只符合两个标准,通常是不常见的便和疼痛的肠道排便.
结论:
- 在社区环境中,儿科FC的罗马IV标准流行率存在显著的变化.
- 疼痛性肠道排便是关键的诊断组成部分,但作为临床试验的终点未得到充分利用.
- 建议在儿科FC临床试验中重新评估终点选择,以更好地与现实世界症状分布保持一致.
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