City St George's, University of London, School of Health and Medical Sciences, Neurosciences Clinical Academic Group, Cranmer Terrace, London SW17 0RE, UK.
Epilepsy Behav. 2024 Oct;159:110030. doi: 10.1016/j.yebeh.2024.110030. Epub 2024 Sep 1.
Despite burgeoning interest in trials in status epilepticus over the last 20 years, outcomes have yet to improve and a number of high profile studies have failed to deliver for a range of reasons. The range of reasons a trial may fail to meet the intended outcomes are discussed. Recent well designed, adequately powered studies in established status epilepticus failed to meet primary endpoints, but are nonetheless influencing practice, reflecting the importance of interpreting results in the context of broader literature, safety and practical considerations. Studies in refractory and super-refractory status epilepticus have yet to do so, frequently failing to deliver as hoped despite huge financial and human cost. The importance of reviewing regulatory frameworks, and our approach to trial design to address important clinical questions is reviewed, reflecting on lessons from the COVID-19 RECOVERY trials, and other disease areas, together with the potential associated with the use artificial intelligence tools. This paper is based on a presentation made at the 9th London-Innsbruck Colloquium on Status Epilepticus and Acute Seizures in April 2024.
尽管在过去的 20 年中，人们对癫痫持续状态的试验产生了浓厚的兴趣，但结果仍未得到改善，并且由于多种原因，一些备受瞩目的研究未能取得成功。讨论了试验可能无法达到预期结果的一系列原因。最近在已建立的癫痫持续状态中进行的设计良好、充分有力的研究未能达到主要终点，但仍在影响实践，反映了在更广泛的文献、安全性和实际考虑的背景下解释结果的重要性。难治性和超难治性癫痫持续状态的研究尚未如此，尽管花费了巨大的财力和人力，但仍未能如愿以偿。审查监管框架以及我们解决重要临床问题的试验设计方法的重要性，反映了从 COVID-19 RECOVERY 试验和其他疾病领域吸取的经验教训，以及使用人工智能工具的潜在可能性。本文基于 2024 年 4 月在第九届伦敦-因斯布鲁克癫痫持续状态和急性发作研讨会上的演讲。
