School of Health & Population Sciences, University of Birmingham, Birmingham, UK.
BMC Pediatr. 2011 Jul 28;11:68. doi: 10.1186/1471-2431-11-68.
There is evidence that at least 5% of Multiple sclerosis (MS) cases manifest in childhood. Children with MS present with a demyelinating episode involving single or multiple symptoms prior to developing a second event (usually within two years) to then meet criteria for diagnosis. There is evidence from adult cohorts that the incidence and sex ratios of MS are changing and that children of immigrants have a higher risk for developing MS. A paediatric population should reflect the vanguard of such changes and may reflect trends yet to be observed in adult cohorts. Studying a paediatric population from the first demyelinating event will allow us to test these hypotheses, and may offer further valuable insights into the genetic and environmental interactions in the pathogenesis of MS.
METHODS/DESIGN: The Paediatric UK Demyelinating Disease Longitudinal Study (PUDDLS) is a prospective longitudinal observational study which aims to determine the natural history, predictors and outcomes of childhood CNS inflammatory demyelinating diseases. PUDDLS will involve centres in the UK, and will establish a cohort of children affected with a first CNS inflammatory demyelinating event for long-term follow up by recruiting for approximately 5 years. PUDDLS will also establish a biological sample archive (CSF, serum, and DNA), allowing future hypothesis driven research. For example, the future discovery of a biomarker will allow validation within this dataset for the evaluation of novel biomarkers. Patients will also be requested to consent to be contacted in the future. A secondary aim is to collaborate internationally with the International Paediatric Multiple Sclerosis Study Group when future collaborative studies are proposed, whilst sharing a minimal anonymised dataset. PUDDLS is the second of two jointly funded studies. The first (UCID-SS) is an epidemiological surveillance study that already received ethical approvals, and started on the 1st September 2009. There is no direct patient involvement, and UCID-SS aims to determine the UK and Ireland incidence of CNS inflammatory demyelinating disorders in children under 16 years.
A paediatric population should reflect the vanguard of MS epidemiological changes and may reflect trends yet to be observed in adult MS cohorts. The restricted window between clinical expression of disease and exposure to environmental factors in children offers a unique research opportunity. Studying a paediatric population from the first demyelinating event will allow us to investigate the changing epidemiology of MS, and may offer further valuable insights into the genetic and environmental interactions in the pathogenesis of MS.
有证据表明，至少有 5%的多发性硬化症 (MS) 病例发生在儿童期。儿童 MS 患者在出现第二个事件（通常在两年内）之前，会出现涉及单一或多个症状的脱髓鞘发作，然后符合诊断标准。有证据表明，成人队列中的 MS 发病率和性别比例正在发生变化，移民儿童患 MS 的风险更高。儿科人群应该反映这种变化的先锋，并且可能反映尚未在成人队列中观察到的趋势。从第一次脱髓鞘事件研究儿科人群，将使我们能够检验这些假设，并可能为 MS 发病机制中的遗传和环境相互作用提供进一步有价值的见解。
方法/设计：儿科英国脱髓鞘疾病纵向研究 (PUDDLS) 是一项前瞻性纵向观察性研究，旨在确定儿童中枢神经系统炎症性脱髓鞘疾病的自然史、预测因素和结局。PUDDLS 将涉及英国的中心，并通过大约 5 年的招募，为受第一次中枢神经系统炎症性脱髓鞘事件影响的儿童建立一个长期随访的队列。PUDDLS 还将建立一个生物样本档案（CSF、血清和 DNA），允许未来基于假设的研究。例如，未来发现生物标志物将允许在该数据集中验证，以评估新的生物标志物。还将要求患者同意在未来被联系。第二个目标是在未来提出合作研究时与国际儿科多发性硬化症研究组合作，同时共享最小的匿名数据集。PUDDLS 是两个联合资助研究中的第二个。第一个（UCID-SS）是一项已经获得伦理批准并于 2009 年 9 月 1 日开始的流行病学监测研究。没有直接的患者参与，UCID-SS 的目的是确定英国和爱尔兰 16 岁以下儿童中枢神经系统炎症性脱髓鞘疾病的发病率。
儿科人群应该反映 MS 流行病学变化的先锋，并且可能反映尚未在成人 MS 队列中观察到的趋势。儿童在疾病表现和暴露于环境因素之间的窗口期很窄，这为研究提供了一个独特的机会。从第一次脱髓鞘事件研究儿科人群，将使我们能够研究 MS 不断变化的流行病学，并可能为 MS 发病机制中的遗传和环境相互作用提供进一步有价值的见解。
