Advancing Clinical Science in Pediatric Gastroparesis

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)强调了 儿童GP和FD特定的研究策略。因此，此续订申请的目标是在 并将我们之前的儿科注册工作扩展为NIDDK胃轻瘫联盟(GpCRC)的一部分 (例如，招募到登记处，开发儿科生活质量和症状综合治疗模块； PedsQL/症状GP模块)，最终确定影响测量的疾病严重程度的因素 根据生活质量和症状。我们的具体目标是： 目标1-通过现场测试完成PedsQL/症状GP模块的验证，并开发 儿童功能性消化不良的模拟模块，采用定性测量，然后进行现场测试。 目标2-确定以下潜在因素在儿科全科医生疾病严重程度中的作用 和fd： 2a-病理生理学：a)内脏过敏(通过水负荷饱足试验)；b)胃 调节(通过胃内配餐)；c)胃排空率(核素扫描)；d)十二指肠/胃 炎症(肥大细胞和嗜酸性粒细胞)；e)胃/近端肠道屏障功能(肠道通透性)。 2B-心理社会苦恼：与心理学家联系评估a)躯体化(儿童的躯体化 (B)抑郁(儿童抑郁问卷)；c)焦虑(状态-特质焦虑问卷)；d) 残疾(功能残疾调查表)；e)灾害化(疼痛灾害化量表)。 2C-饮食不耐受：与营养师、患者和父母接触，以确定自我认同的食物 GP和FD患儿的耐受性和饮食行为。 目标3--为了实现上述目标，扩大注册中心的招聘范围，增加儿科中心，增加一些 与成人GpCRC网站链接，以便开始探索从儿童护理过渡到成人护理的障碍。 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。目前的提案是对RFA-DK-20-504的响应，除其他目标外， TO：在以往成果的基础上，招募心理学家和营养师的专业知识，扩大招聘网站，以及 研究从儿科到成人护理的过渡。

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 need for childhood GP- and FD-specific research strategies. Thus, the goals of this renewal application are to build on and extend our previous Pediatric Registry work as part of the NIDDK Gastroparesis Consortium (GpCRC) (e.g., recruitment into the Registry, development of a pediatric Quality of Life and Symptom GP Module; PedsQL/Symptom GP Module), ultimately, to determine the factors contributing to disease severity measured by quality of life and symptoms. Our Specific Aims are: Aim 1 – Complete validation of the PedsQL/Symptom GP Module via field testing and develop an analogous module for pediatric FD using qualitative measures followed by field testing. Aim 2- Determine the role of the following potential contributors to disease severity in pediatric GP and FD: 2a - Pathophysiologic: a) Visceral hypersensitivity (via water load satiety testing); b) Gastric accommodation (via intragastric meal distribution); c) Gastric emptying rate (scintigraphy); d) Duodenal/gastric inflammation (mast cells and eosinophils); e) Gastric/proximal gut barrier function (gut permeability). 2b - Psychosocial distress: Engage with psychologists to assess a) Somatization (Children’s Somatization Inventory); b) Depression (Children’s Depression Inventory) c) Anxiety (State-Trait Anxiety Inventory); d) Disability (Functional Disabilities Inventory); e) Catastrophizing (Pain Catastrophizing Scale). 2c - Dietary intolerances: Engage with dietitians, patients, and parents to determine self-identified food intolerances and eating behaviors in children with GP and FD. Aim 3 - To accomplish the above, expand Registry recruitment by including more pediatric centers, with some linked to adult GpCRC sites in order to begin to explore barriers to transition from pediatric to adult care. This innovative multidisciplinary approach will prospectively begin to fill the vast knowledge void regarding GP and FD in children. The current proposal is responsive to RFA-DK-20-504 by achieving among other goals, to: build on our previous gains, enlist expertise of psychologists and dietitians, expand recruitment sites, and study pediatric to adult care transition.