The US national registry for childhood interstitial and diffuse lung disease: Report of study design and initial enrollment cohort.
The US national registry for childhood interstitial and diffuse lung disease: Report of study design and initial enrollment cohort.
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美国国家儿童间质性和弥漫性肺疾病登记处：研究设计和初始入组队列报告。
DOI：
10.1002/ppul.26568
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发表时间：
2023
影响因子：
3.1
通讯作者：
Conrad
中科院分区：
文献类型：
--
作者：
Nevel,RebekahJ;Deutsch,GailH;Craven,Daniel;Deterding,Robin;Fishman,MarthaP;Wambach,JenniferA;Casey,Alicia;Krone,Katie;Liptzin,DeborahR;O'Connor,MichaelG;Kurland,Geoffrey;Taylor,JaneB;Gower,WilliamA;Hagood,JamesS;Conrad
儿童间质性和弥漫性肺疾病（chILD）包括一系列罕见疾病。儿童间质性和弥漫性肺疾病研究网络（chILDRN）建立了一个前瞻性登记，以促进有关病因学，表型，自然史和这些疾病的管理知识。来自美国25个chILDRN中心的参与。使用研究电子数据采集（REDCap）收集和管理临床数据结果我们报告了研究设计和初始登记注册组的选定要素，该队列包括683名具有广泛儿童ILD诊断的受试者。报告的最常见诊断为婴儿期神经内分泌细胞增生，有155例（23%）受试者。通过入组研究中心确定基础疾病生物学的组成部分，最常报告的是间质性纤维化、免疫失调和气道疾病队列。影响登记儿童的突出发病率包括家庭辅助供氧使用（63%）和未能茁壮成长（46%）。ConclusionThis Registry是迄今为止美国最大的纵向儿童队列研究，为致力于提高对这些罕见疾病的理解和治疗的合作中心提供了一个强大的框架。
IntroductionChildhood interstitial and diffuse lung disease (chILD) encompasses a broad spectrum of rare disorders. The Children's Interstitial and Diffuse Lung Disease Research Network (chILDRN) established a prospective registry to advance knowledge regarding etiology, phenotype, natural history, and management of these disorders.MethodsThis longitudinal, observational, multicenter registry utilizes single‐IRB reliance agreements, with participation from 25 chILDRN centers across the U.S. Clinical data are collected and managed using the Research Electronic Data Capture (REDCap) electronic data platform.ResultsWe report the study design and selected elements of the initial Registry enrollment cohort, which includes 683 subjects with a broad range of chILD diagnoses. The most common diagnosis reported was neuroendocrine cell hyperplasia of infancy, with 155 (23%) subjects. Components of underlying disease biology were identified by enrolling sites, with cohorts of interstitial fibrosis, immune dysregulation, and airway disease being most commonly reported. Prominent morbidities affecting enrolled children included home supplemental oxygen use (63%) and failure to thrive (46%).ConclusionThis Registry is the largest longitudinal chILD cohort in the United States to date, providing a powerful framework for collaborating centers committed to improving the understanding and treatment of these rare disorders.