Advancing Clinical Science in Pediatric Gastroparesis

儿童和成人的胃轻瘫（GP）的特征是在缺乏胃排空的情况下胃排空延迟。 机械性梗阻GP与显著的发病率和死亡率相关，但对其 儿童的发病率、患病率和自然史。儿科全科医生的这种知识差距因以下因素而加剧： 症状和病理生理学与功能性消化不良（FD）重叠，FD是成人常见疾病， 孩子有限的数据表明，临床症状和病理生理学之间的显着差异， 儿童与成人的GP和FD。因此，关于成人GP和FD的数据不太可能提供洞察力和填补 关于儿童GP和FD的知识差距。这些问题（除其他外）强调需要 儿童GP-和FD-具体的研究策略。因此，此更新应用程序的目标是建立在 并将我们之前的儿科登记工作作为NIDDK胃轻瘫联盟（GpCRC）的一部分进行扩展 (e.g.,招募入登记研究，开发儿科生活质量和症状GP模块; PedsQL/症状GP模块），最终确定导致测量的疾病严重程度的因素 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/Symptom GP模块的验证，并开发 儿科FD的类似模块，使用定性测量，然后进行现场测试。 目的2-确定以下潜在因素对儿科GP疾病严重程度的作用 FD： 2a -病理生理学：a）内脏超敏反应（通过水负荷饱腹感试验）; B）胃 调节（通过胃内膳食分布）; c）胃排空率（消化道造影）; d）十二指肠/胃 炎症（肥大细胞和嗜酸性粒细胞）; e）胃/近端肠屏障功能（肠渗透性）。 2b -社会心理困扰：与心理学家合作评估a）躯体化（儿童躯体化） 问卷）; B）抑郁（儿童抑郁问卷）; c）焦虑（状态-特质焦虑问卷）; d） 残疾（功能性残疾量表）; e）灾难性（疼痛灾难性量表）。 2c -饮食不耐受：与营养师、患者和家长一起确定自我认定的食物 GP和FD儿童的不耐受和饮食行为。 目标3 -为了实现上述目标，通过纳入更多儿科中心来扩大登记研究招募， 链接到成人GpCRC站点，以便开始探索从儿科到成人护理过渡的障碍。 这一创新的多学科方法将有望开始填补关于 儿童的GP和FD。当前提案响应RFA-DK-20-504，除其他目标外， 以我们以前的成果为基础，争取心理学家和营养师的专业知识，扩大招聘网站， 研究儿童到成人护理的转变。

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.