INSPPIRE TO STUDY ACUTE RECURRENT AND CHRONIC PANCREATITIS IN CHILDREN

描述(申请人提供)：胰腺炎是儿科的一个新出现的问题，给社会带来了巨大的社会和经济负担。大多数儿童急性胰腺炎恢复顺利，但一小部分患者出现急性胰腺炎(急性复发性胰腺炎或ARP)复发或进展为慢性胰腺炎(CP)。目前还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。一些儿科研究在三级护理中心和少数患者身上进行了回顾性图表回顾。如果没有前瞻性研究集中在儿童胰腺炎患者的良好表型队列上，就不可能就这些疾病的流行病学、病因、发病机制、自然病史和结果得出结论，也不可能设计诊断算法和治疗替代方案。为了满足仔细收集数据和登记表型良好的患者进行临床研究的需要，我们成立了一个儿科胰腺炎兴趣小组(INSPPIRE：国际儿科胰腺炎研究小组：寻找治疗方法)。我们的近期目标是开发ARP和CP儿童的数据库，并了解ARP和CP儿童的流行病学、病因、自然病史和预后。在这项提案中，我们建议确定建立一个合作的、全国性的儿科中心网络的可行性，以参与对患有ARP和CP的儿童的前瞻性研究和分析。我们最初的项目是定义儿科ARP和CP，了解其流行病学，并建立患者登记和样本库。我们有以下具体目标：(1)建立一个多中心联盟，对儿童ARP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ARP和CP的流行病学和潜在致病因素。在第一个目标中，我们将巩固INSPPIRE联合会的行政和科学结构，并创建一个电子数据库，以准确收集和共享来自14个参与地点的ARP和CP儿童的临床数据和生物样本。在第二个目标中，我们将分析儿童ARP和CP的流行病学、病因、诊断方法、并发症、结局和手术治疗。该应用程序的目标是开发ARP和CP儿童的数据库，建立患者登记和样本库，并确定长期研究的可行性。我们小组的组成和该提案代表了创建一个多中心方法来系统地描述儿童胰腺炎的第一个倡议。我们预计我们将发现患有ARP和CP的儿童，他们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基础、可行性和基础设施 ARP和CP患者。 公共卫生相关性：胰腺炎是一种新出现的儿童疾病，但人们对此知之甚少，给社会带来了重大的社会和经济负担。该应用程序的目标是开发一个复发性急性和慢性胰腺炎儿童数据库，建立患者登记和样本库，并确定长期研究的可行性。我们的长期目标是为反复发作的急性和慢性胰腺炎儿童开发新的有效的治疗方法。

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.