INSPPIRE to Study Pediatric Chronic Pancreatitis

INSPPIRE 研究小儿慢性胰腺炎

基本信息

批准号：
10012208
负责人：
Aliye Uc
金额：
$ 27.9万
依托单位：
UNIVERSITY OF IOWA
依托单位国家：
美国
项目类别：
财政年份：
2015
资助国家：
美国
起止时间：
2015-09-28 至 2020-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10012208
关键词：
Abdominal Pain Acute Address Adult Anxiety Calcium-Sensing Receptors Carboxypeptidase Cations Characteristics Child Childhood Clinical Cognitive Therapy Control Groups Cystic Fibrosis DNA Sequence Alteration Data Development Diabetes Mellitus Diagnostic Disease Disease Progression Emotional Endocrine Enrollment Environmental Risk Factor Epidemiology Exocrine pancreatic insufficiency Foundations Frequencies Functional disorder Gene Frequency Gene Mutation Genes Genetic Genotype Goals Growth High Prevalence Hospitalization Hybrids Impaired health Incidence Interdisciplinary Study International Internet Knowledge Laboratories Manuscripts Measures Mental Depression Modality Natural History Nutritional Onset of illness Outcome Pain Pain management Pancreas Pancreatitis Pediatric cohort Phenotype Population Problem behavior Procedures Prognostic Factor Radiology Specialty Randomized Controlled Trials Recurrence Registries Research Personnel Risk Factors Role Serine Protease Serine Proteinase Inhibitors Severity of illness Site Testing Trypsinogen United States National Institutes of Health Work acute pancreatitis base behavioral health burden of illness chronic pain chronic pancreatitis chymotrypsin C clinical practice clinically relevant cohort data registry early onset genetic risk factor health care service utilization health related quality of life improved innovation pediatric patients programs prospective public health relevance repository socioeconomics

项目摘要

DESCRIPTION (provided by applicant): Acute pancreatitis (AP) is increasingly recognized in children, with an incidence approaching that of adults. Most children with pancreatitis have a single, mild, acute episode that resolves without complications. However, a subset of children with AP develops recurrent episodes (defined as acute recurrent pancreatitis or ARP) and some progress to chronic pancreatitis (CP). Few studies have been performed to characterize ARP and CP in children. We recognized that the lack of a well-phenotyped pediatric cohort presented a major obstacle in understanding the pathophysiology of CP and its sequelae. To address this knowledge gap, we have formed INSPPIRE (INternational Study Group of Pediatric Pancreatitis: In search for a cuRE), composed of 18 participating sites. Results from 301 pediatric patients enrolled into our study revealed a high frequency of pancreatitis-associated gene mutations and substantial disease burden in children with CP. The objective of this application is to determine the natural history of pediatric CP, identify risk factors and genetic modifiers for its onset and sequelae and develop approaches to improve clinical outcomes. The overall hypothesis of this proposal is that pediatric CP has unique characteristics that determine disease onset and sequelae. Our specific aims are: 1) Characterize pediatric CP, determine predictors of disease severity and progression from ARP to CP; 2) Define the role of genetic mutations in early onset CP and identify disease modifiers; 3) Determine the efficacy of early pain management using cognitive-behavioral intervention on pediatric CP. Our long-term goal is to develop diagnostic modalities, prognostic factors and innovative treatment approaches for pediatric ARP and CP.

描述（由申请人提供）：急性胰腺炎（AP）在儿童中的发病率接近成人，大多数患有胰腺炎的儿童有单一的、轻微的、急性发作，并且不会出现并发症。 AP 会反复发作（定义为急性复发性胰腺炎或 ARP），并会进展为慢性胰腺炎 (CP)。我们认识到，缺乏对儿童 ARP 和 CP 特征的研究。一个表型良好的儿科队列对了解 CP 的病理生理学及其后遗症构成了主要障碍。为了解决这一知识差距，我们成立了 INSPPIRE（国际小儿胰腺炎研究组：寻找 cuRE），由 18 个参与中心组成。参与我们研究的 301 名儿科患者的结果显示，胰腺炎相关基因突变的频率很高，并且 CP 儿童的疾病负担很大。该应用的目的是确定胰腺炎的自然史。儿科 CP，确定其发病和后遗症的危险因素和遗传修饰因素，并制定改善临床结果的方法。该提案的总体假设是，儿科 CP 具有决定疾病发病和后遗症的独特特征，我们的具体目标是：1）表征。儿科 CP，确定疾病严重程度和从 ARP 到 CP 进展的预测因素；2) 定义基因突变在早期 CP 中的作用并确定疾病调节因素；3) 确定使用认知行为的早期疼痛管理的功效我们的长期目标是开发儿科 ARP 和 CP 的诊断模式、预后因素和创新治疗方法。