Non-invasive Biomarkers of Symptom Severity and Treatment Response in Pediatric Feeding Disorders

儿科喂养障碍症状严重程度和治疗反应的非侵入性生物标志物

• 批准号: 10723599
• 负责人: Suzanna Hirsch
• 金额: $ 19.38万
• 依托单位: BOSTON CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-01 至 2028-05-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10723599
• 关键词: Address; Affect; Age; Agonist; Animals; Antral; Biological Markers; Boston; Caring; Child; Childhood; Chronic; Clinical; Clinical Management; Clinical Research; Clinical Trials Design; Complex; Consumption; Cross-Sectional Studies; Cyproheptadine; Data; Data Analyses; Development; Development Plans; Diet; Discipline of Nuclear Medicine; Disease; Enrollment; Enteral Feeding; Environment; Erythromycin; Feeds; Functional disorder; Future; Gastric Emptying; Gastroparesis; Gastrostomy; Goals; Growth; Health; High Prevalence; Histamine Antagonists; Impairment; Institution; K-Series Research Career Programs; Knowledge; Literature; Longitudinal cohort study; Manometry; Measurable; Measures; Medical; Mentors; Mentorship; Morbidity - disease rate; Motor; Muscarinic Antagonists; Nutritional; Observational Study; Oral; Outcome; Parents; Patients; Pattern; Pediatric Hospitals; Pharmaceutical Preparations; Physiological; Physiology; Population; Prospective Studies; Prospective cohort; Public Health; Quality of life; Radiation; Radiation exposure; Radionuclide Imaging; Randomized, Controlled Trials; Research Activity; Research Design; Research Methodology; Research Personnel; Resources; Retrospective Studies; Role; Scientific Advances and Accomplishments; Serotonin Antagonists; Severities; Stomach; Symptoms; Testing; Time; Training; Tube; Ultrasonography; Vomiting; career; career development; cell motility; clinical biomarkers; clinical decision-making; cost; design; evidence base; experience; feeding; gastrointestinal; gastrointestinal symptom; health care service utilization; improved; innovation; insight; instructor; medical schools; motilin; motility disorder; neonatal period; patient oriented research; personalized medicine; preterm newborn; prospective; skills; success; treatment response; ultrasound

PROJECT SUMMARY Pediatric feeding disorders are extremely common, affecting 5-20% of children, and they cause significant patient morbidity, decreased quality of life for children and parents, and increased healthcare utilization and cost. Many of these patients require feeding tube placement, but even this does not ensure successful feeding and growth. Up to 50% of children with gastrostomy tubes suffer from feeding intolerance, defined as vomiting or inability to tolerate adequate feed volumes to sustain growth. Despite the high prevalence and large health impact of this problem, no prospective studies exist to help inform management. Furthermore, the gastrointestinal mechanisms underlying feeding intolerance remain largely unknown, hindering our ability to effectively target treatments toward patient pathophysiology. Two proposed mechanisms of feeding intolerance are (1) delayed gastric emptying and (2) impaired gastric accommodation. We have shown that 49% of children with feeding intolerance have delayed gastric emptying; however, no studies have correlated delayed gastric emptying with feeding intolerance symptoms or assessed the role of other abnormalities of gastric physiology such as impaired accommodation. In Aim 1 of this proposal, we will use a cross-sectional study design to fully characterize (using both ultrasonography and scintigraphy) gastric emptying and accommodation patterns in gastrostomy-fed patients with and without feeding intolerance. In Aim 2, we will use a prospective cohort design to assess changes in gastric physiology and feeding intolerance symptoms after initiating therapy with erythromycin (which improves gastric emptying) or cyproheptadine (which improves gastric accommodation). Dr. Suzanna Hirsch, MD is an Instructor at Harvard Medical School (HMS) and a subspecialist within the Aerodigestive Center at Boston Children’s Hospital (BCH). She has gained substantial clinical research experience during her medical training and has demonstrated commitment to an academic career in patient- oriented research. Her career goal is to fundamentally advance scientific understanding of gastric dysfunction in pediatric feeding disorders and to use this mechanistic knowledge to drive evidence-based care and fuel treatment innovation. The mentorship and training afforded by this career development award will be critical for Dr. Hirsch’s academic development. Her primary mentor, Dr. Rachel Rosen, is an expert in pediatric motility disorders, and her co-mentor, Dr. Odd Helge Gilja, is an expert in gastrointestinal ultrasonography – both are exceptional researchers with longstanding commitments to mentorship. Dr. Hirsch has carefully crafted a career development plan with opportunities to gain skills in gastric ultrasonography and clinical research methods. She will pursue a Master’s in Public Health including coursework in repeated measures, longitudinal data analyses, and clinical trial design. Her research activities will be conducted in the unparalleled academic environments of BCH and HMS, which are firmly committed to her successful transition to independence.

项目摘要 小儿喂养障碍非常普遍，影响5-20％的儿童，并且会导致 大量患者发病率，改善儿童和父母的生活质量，并增加医疗保健 利用和成本。这些患者中有许多需要进食管的放置，但即使这也不能确保 成功的喂养和增长。多达50％的胃造口管儿童患有摄入剂， 定义为呕吐或无法耐受足够的饲料量以维持增长。尽管很高 该问题的患病率和巨大的健康影响，没有前瞻性研究来帮助管理层。 此外，喂养摄入剂的胃肠道机制仍然很大未知， 阻碍我们有效靶向患者病理生理学的能力。 提出的两种喂养intlerance的机制是（1）延迟的胃排空和（2）受损 胃住宿。我们已经表明，有49％的喂养肠儿童胃延迟 排空;但是，没有研究将延迟胃排空与喂养intlerance症状或 评估了胃生理学其他异常的作用，例如受损的住宿。在目标1中 该建议，我们将使用横截面研究设计来充分表征（使用超声检查和 壁画的胃排空和住宿模式，有或没有 喂食intlerance。在AIM 2中，我们将使用前瞻性队列设计来评估胃生理的变化 并在用红霉素开始治疗后喂养耐摄取症状（改善胃排空） 或Cyproheptadine（可改善胃容纳）。 医学博士Suzanna Hirsch博士是哈佛医学院（HMS）的讲师，也是专业人士 波士顿儿童医院（BCH）的气化中心。她获得了大量的临床研究 在她的医学培训期间的经验，并表现出对患者学术生涯的承诺 - 定向研究。她的职业目标是从根本上提高对胃功能障碍的科学理解 在小儿喂养障碍中，并使用这些机械知识来推动基于证据的护理和燃料 治疗创新。该职业发展奖提供的心态和培训对于 赫希博士的学术发展。她的主要导师瑞秋·罗森（Rachel Rosen）博士是儿科运动的专家 疾病和她的同事Odd Helge Gilja博士是胃肠道超声检查的专家 - 都 杰出的研究人员长期以来对精神训练做出了长期承诺。赫希博士精心制作了 职业发展计划有机会获得胃超声和临床研究技能 方法。她将攻读公共卫生硕士学位，包括重复措施，纵向的课程 数据分析和临床试验设计。她的研究活动将在无与伦比的学术中进行 BCH和HMS的环境最初致力于她成功过渡到独立性。