Advancing Clinical Science in Pediatric Gastroparesis

推进小儿胃轻瘫的临床科学

• 批准号: 10001460
• 负责人: Robert J Shulman
• 金额: $ 35.67万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-09-25 至 2022-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10001460
• 关键词: Adolescent; Adult; Affect; Age; Biopsy; Breath Tests; Characteristics; Child; Childhood; Clinical; Clinical Sciences; Collaborations; Communities; DNA; Data; Diabetes Mellitus; Diagnosis; Diagnostic; Disease; Dyspepsia; Epidemiology; Epigastrium; Equipment and supply inventories; Ethnic Origin; Etiology; Feces; Female Adolescents; Future; Gastric Emptying; Gastrointestinal Endoscopy; Gastroparesis; Gender; General Population; Goals; Hospitalization; Human Microbiome; Incidence; Intervention; Knowledge; Measures; Mechanics; Morbidity - disease rate; Mucous Membrane; Natural History; Nature; Nausea; Normal Range; Nutritional; Obstruction; Operative Surgical Procedures; Outcome Measure; Pain; Patient Outcomes Assessments; Patients; Pediatrics; Population; Prevalence; Quality of life; Race; Radioactive; Radionuclide Imaging; Registries; Research; Role; Serum; Solid; Spirulina preparation; Stomach; Symptoms; Therapeutic; Time; Translations; Vomiting; Woman; age related; base; biobank; boys; cognitive interview; common symptom; comorbidity; cytokine; design; early satiety; gastrointestinal; gastrointestinal symptom; girls; health related quality of life; improved outcome; infancy; innovation; insight; interdisciplinary approach; microbiome; mortality; novel; prospective; psychologic

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, particularly in children. This knowledge gap in pediatric GP is exacerbated by lack of both normative data for gastric emptying and a GP specific patient reported outcome measure. The limited data suggest significant differences between clinical symptoms of GP in children vs adults. Thus, even the limited data regarding GP in adults are unlikely to provide insight and fill the knowledge gaps regarding GP in children. These issues (among others) underscore the need for childhood GP specific research strategies rather than translation of adult based GP knowledge to this very different population. To begin to identify effective diagnostic and therapeutic strategies leading to improved outcomes for children with GP given the vast knowledge gaps in the field, we propose the following Specific Aims within a prospective, multiinstitutional collaboration: 1) Using the 13C-Spirulina breath test (BT) (which correlates well with gastric scintigraphy) define normal values for solid meal emptying in (n=260) healthy controls (HC) (5-18 yrs. of age). Sub-Aim: carry out the BT in children (n=420, 5-18 yrs. of age) with presumed GP who are undergoing gastric scintigraphy to identify children who truly have GP as defined by the normal values obtained in HC. 2) In children undergoing gastric scintigraphy in (Sub) Aim 1, determine the feasibility, reliability, and validity of generic and gastrointestinal (GI)-specific health-related quality of life using the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales and PedsQL GI Symptoms Scales and PedsQL GI Worry Scales. Sub- Aim 2A: Use the scales to differentiate patients with GP vs. age, gender, and race/ethnicity matched children with functional dyspepsia (whose symptoms often overlap with GP) and HC; Sub-Aim 2B: Use the GI scales and cognitive interviews to create a pediatric GP-specific health-related quality of life measure. 3) Create a national prospective: (a) Registry of children and adolescents with GP to include demographic, clinical, psychological, and nutritional characteristics and (b) Biorepository of serum, GI mucosal biopsies (in those undergoing upper GI endoscopy), and stool for future analyses such as DNA, cytokines, microbiome. This multidisciplinary approach is innovative as it will, for the first time, prospectively begin to fill the vast knowledge void regarding GP in children. These data will be an important step toward targeted and pediatric- friendly interventions for children with GP. This project also will be a first step to developing a national pediatric consortium to investigate GP in children. The current proposal fits RFA-DK-16-010 to, among other goals, better understand the epidemiology of GP, define the GP/FD spectrum, explore and design new patient reported outcomes, and the role of the human microbiome on disease expression.

儿童和成人中的胃轻瘫（GP）的特征是胃排空延迟在没有的情况下 机械阻塞。 GP与显着的发病率和死亡率有关，但知之甚少 发病率，患病率和自然历史，尤其是在儿童中。小儿GP中的这种知识差距是 由于缺乏胃排空的规范性数据和GP特定患者报告的结果而加剧 措施。有限的数据表明，儿童与成年人的GP临床症状之间存在显着差异。 因此，即使是成人中有关GP的有限数据，也不太可能提供洞察力并填补知识差距 关于儿童的GP。这些问题（除其他问题）强调了童年时期特定研究的需求 策略而不是将基于成人的GP知识转化为截然不同的人群。 开始确定有效的诊断和治疗策略，从而改善了儿童的结果 由于GP鉴于该领域的知识差距很大，我们提出了以下特定目标， 多机构合作： 1）使用13c-Spirulina呼气测试（BT）（与胃闪烁显像良好相关）定义了正常 （n = 260）健康对照（HC）（年龄5-18岁）中的固体餐空排空值。 Sub-aim：执行BT 儿童（n = 420，5-18岁的年龄）和假定的GP，他们正在经历胃闪烁显像识别儿童 谁确实具有在HC中获得的正常值所定义的GP。 2）在（sub）AIM 1中接受胃闪烁显像的儿童中，确定可行性，可靠性和有效性 使用儿科生活质量 库存（PEDSQL）通用核心尺度和PEDSQL GI症状量表和PEDSQL GI担心量表。亚 AIM 2A：使用量表来区分GP与年龄，性别和种族/种族相匹配的患者 功能性消化不良（其症状通常与GP重叠）和HC； Sub-aim 2b：使用GI量表和 认知访谈以创建儿科特异性健康相关的生活质量度量。 3）创建一个国家的前瞻性：（a）具有GP儿童和青少年的注册表，包括人口统计学， 临床，心理和营养特征以及（b）血清，胃粘膜活检的生物疗法（在 那些接受上胃肠道内窥镜检查）和粪便进行将来的分析，例如DNA，细胞因子，微生物组。 这种多学科的方法是创新的，因为它首次开始填补广阔的 关于儿童GP的知识空隙。这些数据将是朝着针对性和小儿迈出的重要一步 GP儿童的友好干预措施。该项目也将是开发国家儿科的第一步 财团调查儿童的全科医生。当前的提案适合RFA-DK-16-010，除其他目标外 了解GP的流行病学，定义GP/FD光谱，探索和设计新患者报告 结果，以及人类微生物组在疾病表达中的作用。