The Institution-Based Prospective Inception Cohort Study: Design, Implementation, and Quality Assurance in Pediatric Thrombosis and Stroke Research
The Institution-Based Prospective Inception Cohort Study: Design, Implementation, and Quality Assurance in Pediatric Thrombosis and Stroke Research
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DOI：
10.1055/s-0032-1329551
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发表时间：
2013-02-01
影响因子：
5.7
通讯作者：
Goldenberg, Neil A.
中科院分区：
文献类型：
--
作者：
Bernard, Timothy J.;Armstrong-Wells, Jennifer;Goldenberg, Neil A.
在罕见病中开展精心设计的队列研究可以发现新的危险因素和预后标志物，增强对自然病史和结局的理解，并为治疗策略的随机对照试验提供初步数据。设计一个健壮的队列需要大量的前期设计和规划。理想情况下，对患病个体的队列研究从诊断时（即从疾病开始）前瞻性地跟踪患者。本文的目的是讨论一项基于机构的前瞻性初始队列研究的设计和实施，并以儿童中风和血栓形成为例。此外，我们将讨论优化此类研究所需的持续管理和质量保证机制。虽然实施前瞻性初始队列研究所需的资源很大，但这种方法可以提供关于自然史和预后因素的关键观察证据。在多中心验证后，其发现可以为急需的随机对照临床试验的设计和执行提供信息。
The development of well-designed cohort studies in rare diseases can lead to the discovery of new risk factors and prognostic markers, enhance understanding of natural history and outcomes, and provide preliminary data for randomized controlled trials of treatment strategies. Designing a robust cohort requires substantial upfront design and planning. Ideally, a cohort study of diseased individuals follows patients prospectively from the time of diagnosis (i.e., from the disease's inception). The objective of this article is to discuss the design and implementation of an institution-based prospective inception cohort study, with applied examples in pediatric stroke and thrombosis. Furthermore, we will discuss the ongoing management and quality assurance mechanisms necessary to optimize such a study. Although the resources necessary to implement a prospective inception cohort study are large, this approach can provide critical observational evidence on natural history and prognostic factors. Following multicenter validation, its findings can inform the design and execution of much-needed randomized controlled clinical trials.