成人炎症性肠病临床试验的结果:评估青少年发病者与成年发病者的相似性。

Joel R Rosh, Dan Turner, Jeffrey S Hyams, Marla Dubinsky, Anne M Griffiths, Stanley A Cohen, Kim Hung Lo, Lilianne Kim, Sheri Volger, Renping Zhang, Richard Strauss, Laurie S Conklin
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引用次数: 0

摘要

背景和目的:大多数儿科 IBD 研究都是在成人用药获得批准后进行的,这些研究的参与者大多是青少年。我们假设青少年发病的 IBD 与成人发病的 IBD 并无本质区别。如果这一假设是正确的,那么推迟青少年使用新型药物的价值就会受到质疑:方法: 分析了 4 种生物制剂的 11 项随机、双盲、安慰剂对照成人 2 期和 3 期试验的数据。参与者被分为青少年或成人发病者(诊断为 12 岁至成年):对 6283 名研究参与者(2575 名克罗恩病 [CD]患者,3708 名溃疡性结肠炎 [UC]患者)的数据进行了评估。在 2,575 名克罗恩病研究参与者中,325 人患有溃疡性结肠炎:本文提供的数据支持将生物制剂的疗效从成人推断到患有 IBD 的青少年,这将有助于更早地为患者贴上标签并使其获得治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Outcomes in Adult Inflammatory Bowel Disease Clinical Trials: Assessment of Similarity Among Participants with Adolescent-onset and Adult-onset Disease.

Background and aims: Most paediatric inflammatory bowel disease [IBD] studies are performed after medications are approved in adults, and the majority of participants in these studies are adolescents. We hypothesised that adolescent-onset IBD is not fundamentally different from adult-onset IBD. If this is correct, the value of delaying access to novel drugs in adolescents becomes questioned.

Methods: Data from 11 randomised, double-blind, placebo-controlled, adult Phases 2 and 3 trials of four biologics were analysed. Participants were categorised as having adolescent- or adult-onset disease [diagnosed 12 to <18, or ≥18 years]. Multivariable modelling explored the association between age at diagnosis and response to treatment, after adjustment for disease duration, extent, and severity at baseline. Data from dose arms were pooled to evaluate similarity of therapeutic response between adolescent- and adult-onset IBD within the same trial [not between doses or across trials]. Ratios of odds ratios [ORs] between the two groups were evaluated.

Results: Data from 6283 study participants (2575 with Crohn's disease [CD], 3708 with ulcerative colitis [UC]) were evaluated. Of 2575 study participants with CD, 325 were 12-<18 years old at diagnosis; 836 participants [32.4%] received placebo. Of 3708 participants with UC, 221 were 12-<18 years old at diagnosis; 1212 [33%] were receiving placebo. The majority of the ratios of ORs were within 2-fold, suggesting that responses in adolescent- and adult-onset participants are generally similar.

Conclusion: Data presented lend support for extrapolating efficacy of biologics from adults to adolescents with IBD, which would facilitate earlier labelling and patient access.

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