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: 8359486
• 负责人: Aliye Uc
• 金额: $ 19.72万
• 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 2014-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8359486
• 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; 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. PUBLIC HEALTH RELEVANCE: Pancreatitis is an emerging, but poorly understood condition in children that carries significant social and economic burden to society. The objective of this application is to develop a database of children with recurrent acute and chronic pancreatitis, build a patient registry and sample repository and determine the feasibility of long-term studies. Our long-term goal is to develop novel and effective therapies for children with recurrent acute and chronic pancreatitis.

描述（由申请人提供）：胰腺炎是儿科的一个新问题，对社会造成重大的社会和经济负担。大多数儿童急性胰腺炎恢复顺利，但一小部分患者发生急性胰腺炎复发（急性复发性胰腺炎或阿普）或进展为慢性胰腺炎（CP）。目前没有有效的治疗方法来阻止胰腺炎的复发或防止其进展为CP。在三级护理中心和少数患者中进行了一些儿科研究，作为回顾性病历审查。如果没有针对儿童胰腺炎患者的良好表型队列的前瞻性研究，就不可能对这些疾病的流行病学、病因学、发病机制、自然史和结局得出结论，也不可能设计诊断算法和治疗方案。为了满足仔细收集数据和登记临床研究的良好表型患者的需要，我们成立了一个儿科胰腺炎兴趣小组（INSPPIRE：儿科胰腺炎国际研究小组：寻找治愈方法）。我们的近期目标是建立一个儿童阿普和CP的数据库，并了解流行病学，病因，自然史和结果在一个良好的表型队列的儿童阿普和CP。在本提案中，我们建议确定建立一个合作的全国儿科中心网络的可行性，以参与对阿普和CP儿童的前瞻性研究和分析。我们最初的项目是定义小儿阿普和CP，了解其流行病学，并建立一个患者登记和样本库。我们有以下具体目标：（1）建立一个多中心联盟，对儿童阿普和CP进行前瞻性研究，并确定长期研究的可行性;（2）确定儿童阿普和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，以准确收集和共享来自14个参与站点的阿普和CP儿童的临床数据和生物样本。在第二个目标，我们将分析流行病学，病因学，诊断方法，并发症，结果，和手术治疗的儿童与阿普和CP。本申请的目的是开发一个儿童阿普和CP的数据库，建立一个患者登记和样本库，并确定长期研究的可行性。我们小组的组成和提案代表了创建多中心方法以系统地描述儿童胰腺炎的第一个倡议。我们希望我们将识别出患有阿普和CP的儿童，这些儿童具有独特的病理和结果，需要独特的、以发病机制为导向的治疗。这些分析将为进一步的研究提供理论依据、可行性和基础设施， 阿普和CP患者。 公共卫生相关性：胰腺炎是一种新出现的疾病，但对儿童的了解甚少，给社会带来了重大的社会和经济负担。本申请的目的是开发一个复发性急性和慢性胰腺炎儿童的数据库，建立一个患者登记和样本库，并确定长期研究的可行性。我们的长期目标是为患有复发性急性和慢性胰腺炎的儿童开发新的有效疗法。