INSPPIRE TO STUDY ACUTE RECURRENT AND CHRONIC PANCREATITIS IN CHILDREN

启发研究儿童急性复发性和慢性胰腺炎

• 批准号: 8541852
• 负责人: Aliye Uc
• 金额: $ 22.67万
• 依托单位: UNIVERSITY OF IOWA
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-09-15 至 2016-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8541852
• 关键词: Acute; Admission activity; Advisory Committees; Algorithms; Biological; Child; Child Care; Childhood; Clinical; Clinical Data; Clinical Research; Collection; Constitution; DNA; Data; Data Collection; Databases; Diagnosis; Diagnostic; Disease; Economic Burden; Emotional; Epidemiology; Etiology; Family; Feces; Future; Goals; Hospitals; Inflammatory; Institution; Interest Group; International; Longitudinal Studies; Mental Health; Modality; Narcotics; Natural History; Nutritional; Operative Surgical Procedures; Outcome; Pancreatic Diseases; Pancreatitis; Pathogenesis; Pathology; Patients; Pediatrics; Pharmacologic Substance; Phenotype; Prevalence; Process; Prospective Studies; Recurrence; Registries; Research Infrastructure; Research Personnel; Sampling; Scientist; Serum; Site; Societies; Structure; Surveys; Testing; Therapeutic; Therapy Clinical Trials; Tissues; Treatment outcome; acute pancreatitis; addiction; burden of illness; chronic pain; chronic pancreatitis; cohort; control trial; design; effective therapy; information gathering; interstitial; meetings; member; novel; novel therapeutic intervention; patient registry; prevent; prospective; public health relevance; repository; social; tertiary care

DESCRIPTION (provided by applicant): Pancreatitis is an emerging problem in pediatrics with significant social and economic burden to society. Most children with acute pancreatitis recover uneventfully, but a fraction of patients develop recurrent episodes of acute pancreatitis (acute recurrent pancreatitis or ARP) or progress to chronic pancreatitis (CP). There are currently no effective treatments to stop the recurrent episodes of pancreatitis or prevent its progression to CP. A few pediatric studies were performed as retrospective chart reviews at tertiary care centers and on a small number of patients. Without prospective studies focusing on a well-phenotyped cohort of patients with pediatric pancreatitis, it is impossible to draw conclusions on the epidemiology, etiologies, pathogenesis, natural history and outcome of these disorders or to design diagnostic algorithms and therapeutic alternatives. To meet the need for the careful collection of data and a registry of well-phenotyped patients for clinical studies, we formed a pediatric pancreatitis interest group (INSPPIRE: International Study Group of Pediatric Pancreatitis: In search for a cure). Our immediate goal is to develop a database of children with ARP and CP and to understand the epidemiology, etiologies, natural history and outcome in a well-phenotyped cohort of children with ARP and CP. In this proposal, we propose to determine the feasibility of creating a collaborative, national network of pediatric centers to engage in prospective studies and analyses of children with ARP and CP. Our initial project is to define pediatric ARP and CP, understand its epidemiology and build a patient registry and sample repository. We have the following specific aims: (1) Develop a multi-center consortium to prospectively study pediatric ARP and CP and determine the feasibility of long-term studies; (2) Determine the epidemiology and potential etiologic factors of ARP and CP in children. In the first aim, we will solidify the administrative and scientific structure of the INSPPIRE Consortium and create an electronic database to accurately collect and share the clinical data and biological samples from children with ARP and CP from 14 participating sites. In the second aim, we will analyze the epidemiology, etiologies, diagnostic approaches, complications, outcome, and surgical therapies of children with ARP and CP. The objective of this application is to develop a database of children with ARP and CP, build a patient registry and sample repository and determine the feasibility of long-term studies. The constitution of our group and the proposal represents the first initiative to create a multi-center approach to systematically characterize pancreatitis in children. We expect that we will identify children with ARP and CP who have distinctive pathology and outcomes that will require unique, pathogenesis-directed therapies. These analyses will provide the rationale, feasibility and infrastructure for additional studies of patients with ARP and CP.

描述（由申请人提供）：胰腺炎是社会社会和经济负担重大的儿科中的一个新兴问题。大多数患有急性胰腺炎的儿童不明显地康复，但是一部分患者会出现急性胰腺炎（急性复发性胰腺炎或ARP）的复发性发作，或者进展为慢性胰腺炎（CP）。目前尚无有效的治疗方法可以阻止胰腺炎的复发发作或防止其发展为CP。在三级护理中心和少数患者的研究中，进行了一些小儿研究作为回顾性图表评论。没有前瞻性研究，重点是针对小儿胰腺炎的患者，就无法得出关于这些疾病的流行病学，病因，发病机理，自然病史和结果的结论，或者设计诊断算法和治疗疗法替代方案。为了满足仔细收集数据和临床研究良好的患者注册表的需求，我们成立了一个小儿胰腺炎兴趣组（Insppire：国际小儿胰腺炎国际研究小组：寻找治疗方法）。我们的直接目标是开发一个患有ARP和CP儿童的数据库，并了解具有ARP和CP的儿童的流行病学，病因，自然史和结果。在此提案中，我们建议确定建立一个协作，国家的儿科中心网络的可行性，以从事ARP和CP儿童的前瞻性研究和分析。我们最初的项目是定义小儿ARP和CP，了解其流行病学并建立患者注册表和样本存储库。我们具有以下具体目的：（1）开发一个多中心财团来前瞻性研究小儿ARP和CP，并确定长期研究的可行性； （2）确定儿童ARP和CP的流行病学和潜在病因。在第一个目标中，我们将巩固Insppire联盟的行政和科学结构，并创建一个电子数据库，以准确地收集和共享来自14个参与站点的ARP和CP儿童的临床数据和生物样品。在第二个目标中，我们将分析ARP和CP儿童的流行病学，病因，诊断方法，并发症，结局和手术疗法。该应用程序的目的是开发一个患有ARP和CP的儿童的数据库，建立患者注册表并进行样本存储库，并确定长期研究的可行性。我们小组的构成和提案代表了第一个创建多中心方法来系统地表征儿童胰腺炎的倡议。我们预计我们将确定患有ARP和CP的儿童，这些儿童具有独特的病理和结果，需要独特的发病机理指导的疗法。这些分析将提供基本原理，可行性和基础设施，以进行其他研究 ARP和CP的患者。