New England Gastropareis Consortium: Neurobiology of Gastroparesis

点击翻译按钮获取中文摘要

ABSTRACT Gastroparesis (Gp) in children and adults is characterized by delayed gastric emptying in the absence of mechanical obstruction. GP is associated with significant morbidity and mortality, yet little is known regarding its incidence, prevalence, and natural history in children. This knowledge gap in pediatric Gp is exacerbated by the overlap in symptoms and pathophysiology with functional dyspepsia (FD), a common disorder in adults and children. The limited data suggests significant differences between clinical symptoms and pathophysiology of Gp and FD in children vs adults. Thus, data regarding Gp and FD in adults is unlikely to provide insight and fill the knowledge gaps regarding Gp and FD in children. These issues (among others) underscore the need for childhood Gp- and FD-specific research strategies. Thus, the goals of this supplemental application are to build on and extend our Pediatric Gp Registry 2 (PGpR2) work as part of the NIDDK Gastroparesis Consortium (GpCRC) ultimately, to determine the factors contributing to disease severity measured by quality of life and symptoms. The Specific Aims of the current project are to: Aim 1: Create a national prospective registry of children and adolescents with gastroparesis Gp) and Gp-like syndrome (GLS; the latter group, using pediatric Rome IV criteria will be characterized as having FD (functional dyspepsia, including the two subtypes [FD-EPS, FD-PDS]), chronic nausea vomiting syndrome, cyclic vomiting syndrome, and/or chronic abdominal pain syndrome to include demographic, clinical, psychological, nutritional characteristics, physiological measures, and serial assessments of symptoms over 3 years during their clinical care. Aim 2: Follow this well-characterized cohort to further define the natural history, clinical course, and selected physiologic measures of children and adolescents with symptoms of Gp. Aim 3: Provide a reliable source for recruitment of well-characterized children and adolescents with Gp and FD for other studies including therapeutic clinical trials, pathophysiological, molecular, histopathologic, or other ancillary studies. These subsequent clinical trials or ancillary studies will be conducted under separate study protocols with separate consent processes. Supplement Aim: Expand the number of pediatric sites within the PGpR2 to facilitate recruitment and consequently, the goals of the PGpR2. This innovative multidisciplinary approach will prospectively begin to fill the vast knowledge void regarding Gp and FD in children. The current supplement proposal is responsive to RFA-DK-20-504 and PA-20-272 by achieving among other goals, to build on our previous gains and expand the number of pediatric sites to enhance recruitment.