Towards molecular-pathology informed clinical trials in childhood arthritis to achieve precision medicine in juvenile idiopathic arthritis.
Towards molecular-pathology informed clinical trials in childhood arthritis to achieve precision medicine in juvenile idiopathic arthritis.
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DOI：
10.1136/ard-2022-222553
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发表时间：
2023-04
影响因子：
27.4
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中科院分区：
文献类型：
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在儿童关节炎（统称为幼年特发性关节炎（JIA））中，近年来获得许可的生物和靶向小分子治疗方法的迅速兴起导致了结局的改善。然而，来自多个国家和登记处的真实世界数据显示，尽管有大量可用药物，但许多儿童和年轻人继续遭受发作，并多年来经历相当长的活动期疾病。超过50%的JIA年轻人需要持续的免疫抑制，直到成年，他们可能不得不在这段时间内尝试多种不同的治疗。目前没有经过验证的工具来选择特定的治疗方法，也没有反应的生物标志物来帮助这种选择，因此，目前的管理基本上使用试错法。最近取得的进展的另一个后果是，有资格接受新试验的儿童或年轻人人数减少。在这篇综述中，我们考虑了如何在JIA中采用基于分子的方法来定义治疗靶点并告知试验设计，结合临床试验的新方法，可以提供最大化发现和进展的策略，以便为关节炎儿童提供精准医学。
In childhood arthritis, collectively known as Juvenile idiopathic arthritis (JIA), the rapid rise of available licensed biological and targeted small molecule treatments in recent years has led to improved outcomes. However, real-world data from multiple countries and registries show that despite a large number of available drugs, many children and young people continue to suffer flares and experience significant periods of time with active disease for many years. More than 50% of young people with JIA require ongoing immune suppression well into adult life, and they may have to try multiple different treatments in that time. There are currently no validated tools with which to select specific treatments, nor biomarkers of response to assist in such choices, therefore, current management uses essentially a trial-and-error approach. A further consequence of recent progress is a reducing pool of available children or young people who are eligible for new trials. In this review we consider how progress towards a molecular based approach to defining treatment targets and informing trial design in JIA, combined with novel approaches to clinical trials, could provide strategies to maximise discovery and progress, in order to move towards precision medicine for children with arthritis.
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