INSPPIRE to Study Pediatric Chronic Pancreatitis

INSPPIRE 研究小儿慢性胰腺炎

• 批准号: 9352341
• 负责人: Aliye Uc
• 金额: $ 42.01万
• 依托单位: UNIVERSITY OF IOWA
• 依托单位国家: 美国
• 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/9352341
• 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; 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

 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）在儿童中越来越多地被认识到，其发病率接近成人。大多数儿童胰腺炎有一个单一的，轻微的，急性发作，解决没有并发症。然而，一部分AP患儿会反复发作（定义为急性复发性胰腺炎或阿普），并在一定程度上进展为慢性胰腺炎（CP）。很少有研究被用来描述儿童的阿普和CP。我们认识到，缺乏一个良好的表型儿科队列提出了一个主要的障碍，在了解CP的病理生理及其后遗症。为了解决这一知识缺口，我们成立了INSPPIRE（国际儿童胰腺炎研究小组：寻找治愈方法），由18个参与研究中心组成。来自301名儿童患者的研究结果显示，胰腺炎相关基因突变的频率很高，CP儿童的疾病负担很大。本申请的目的是确定小儿CP的自然史，确定其发病和后遗症的风险因素和遗传修饰因子，并开发改善临床结局的方法。该建议的总体假设是，小儿CP具有决定疾病发作和后遗症的独特特征。我们的具体目标是：1）表征小儿CP，确定疾病严重程度和从阿普进展到CP的预测因素; 2）定义基因突变在早发CP中的作用，并确定疾病修饰因子; 3）确定使用认知行为干预对小儿CP进行早期疼痛管理的疗效。我们的长期目标是为小儿阿普和CP开发诊断模式、预后因素和创新治疗方法。