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.

儿童和成人胃轻瘫（GP）的特点是在缺乏胃排空的情况下胃排空延迟。 机械性阻塞。 GP 与显着的发病率和死亡率相关，但对其的了解甚少 发病率、患病率和自然史，尤其是儿童。儿科全科医生的知识差距是 由于缺乏胃排空的规范数据和全科医生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人 GP 的临床症状存在显着差异。 因此，即使有关成人全科医生的有限数据也不太可能提供见解并填补知识空白 关于儿童全科医生。这些问题（除其他外）强调了儿童全科医生特定研究的必要性 策略，而不是将基于成人的全科医生知识转化为这个非常不同的人群。 开始确定有效的诊断和治疗策略，以改善儿童的预后 鉴于该领域存在巨大的知识差距，我们与全科医生一起提出以下具体目标： 多机构合作： 1) 使用 13C-螺旋藻呼气试验 (BT)（与胃闪烁扫描密切相关）定义正常 (n=260) 健康对照 (HC)（5-18 岁）的固体膳食排空值。子目标：在 疑似全科医生的儿童（n=420，5-18 岁）正在接受胃闪烁扫描以识别儿童 真正具有 GP（由 HC 中获得的正常值定义）的人。 2) 在（子）目标 1 中接受胃闪烁扫描的儿童中，确定可行性、可靠性和有效性 使用儿科生活质量评估一般和胃肠道 (GI) 特定健康相关的生活质量 库存 (PedsQL) 通用核心量表和 PedsQL GI 症状量表和 PedsQL GI 忧虑量表。子 目标 2A：使用量表区分 GP 患者与年龄、性别和种族/民族匹配的儿童 患有功能性消化不良（其症状通常与 GP 重叠）和 HC；子目标 2B：使用 GI 量表并 认知访谈，以创建儿科全科医生特定的健康相关生活质量衡量标准。 3) 创建国家前景： (a) 儿童和青少年全科医生登记，包括人口统计、 临床、心理和营养特征以及 (b) 血清、胃肠道粘膜活检的生物储存库（在 那些接受上消化道内窥镜检查的人），以及用于未来分析（例如 DNA、细胞因子、微生物组）的粪便。 这种多学科方法是创新的，因为它将首次有望开始填补巨大的空白。 关于儿童全科医生的知识缺乏。这些数据将是迈向有针对性的儿科治疗的重要一步。 对患有全科医生的儿童进行友善的干预。该项目也将成为建立国家儿科中心的第一步。 联盟调查儿童全科医生。除其他目标外，当前提案更适合 RFA-DK-16-010 了解 GP 的流行病学，定义 GP/FD 谱系，探索和设计新的患者报告 结果以及人类微生物组对疾病表达的作用。