New England Gastropareis Consortium: Neurobiology of Gastroparesis

摘要 儿童和成人的胃轻瘫(GP)的特点是在缺乏胃排空障碍的情况下 机械障碍物。GP与严重的发病率和死亡率有关，但对其知之甚少。 儿童的发病率、患病率和自然病史。儿科全科医生的这种知识差距因 与功能性消化不良(FD)的症状和病理生理重叠，FD是成人和 孩子们。有限的数据表明，在临床症状和病理生理学之间存在显著差异。 儿童与成人的GP和FD之间的关系。因此，有关成人GP和FD的数据不太可能提供洞察力和补充性 儿童对GP和FD的认识差距。这些问题(以及其他问题)突出表明有必要 儿童GP和FD特定的研究策略。因此，此补充应用程序的目标是构建 作为NIDDK胃轻瘫联盟的一部分，对我们的儿科GP注册2(PGpR2)工作进行扩展 (GpCRC)最终确定影响疾病严重程度的因素，以生活质量和 症状。当前项目的具体目标是： 目标1：为患有胃轻瘫和类似胃轻瘫的儿童和青少年建立一个全国性的前瞻性登记 综合征(GLS；后一组，使用儿科罗马IV标准将被描述为FD(功能性 消化不良，包括两个亚型[FD-EPS，FD-PDS])，慢性恶心呕吐综合征，周期性呕吐 综合征和/或慢性腹痛综合征，包括人口统计学、临床、心理、营养 临床3年以上症状的特点、生理指标和系列评估 关心。 目标2：遵循这一特征良好的队列，进一步确定自然病史、临床病程和选定的 儿童和青少年GP症状的生理指标。 目标3：为招募特征良好的儿童和青少年提供可靠的来源 用于其他研究的FD，包括治疗临床试验、病理生理学、分子、组织病理学或其他 辅助研究。这些后续的临床试验或辅助研究将在单独的研究下进行。 具有单独同意程序的议定书。 补充目标：扩大PGpR2内儿科网站的数量，以促进招募和 因此，PGpR2的目标是。 这种创新的多学科方法将有望开始填补关于以下方面的巨大知识空白 儿童的GP和FD。目前的补充提案是对RFA-DK-20-504和PA-20-272的回应 实现其他目标之一，在我们以往成果的基础上，扩大儿科网站的数量，以加强 招聘。

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.