Pathophysiological Evidence Driven Management of GERD in Neonatal ICU Infants: Randomized Controlled Trial

新生儿 ICU 婴儿 GERD 的病理生理学证据驱动管理：随机对照试验

• 批准号: 10717324
• 负责人: Sudarshan R Jadcherla
• 金额: $ 66.36万
• 依托单位: RESEARCH INST NATIONWIDE CHILDREN'S HOSP
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-09-15 至 2028-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10717324
• 关键词: Acids; Adverse effects; Age; Algorithms; Benchmarking; Biological Markers; Caring; Categories; Cereals; Characteristics; Childhood; Chronology; Clinic Visits; Clinical; Clinical Trials; Coughing; Data; Diagnosis; Diagnostic; Diagnostic tests; Distal; Eating; Economic Burden; Economics; Effectiveness; Enteral Feeding; Equilibrium; Equipoise; Esophagus; Evaluation; Event; Evidence based practice; Evidence based treatment; Failure; Feeding Methods; Feeds; Future; Gastroesophageal reflux disease; Goals; Growth; Health; Hospitalization; Hour; Infant; Intake; Intervention; Knowledge; Length of Stay; Measurement; Mission; Modification; National Institute of Diabetes and Digestive and Kidney Diseases; Neonatal; Neonatal Intensive Care Units; Neurologic; Nutrient; Oral; Outcome; Parents; Pathologic; Peripheral; Pharmaceutical Preparations; Physiological; Population; Pregnancy; Prevalence; Probability; Proton Pump Inhibitors; Provider; Public Health; Quality of life; Randomized; Randomized, Controlled Trials; Relative Risks; Research; Research Personnel; Rice; Safety; Severities; Severity of illness; Stratification; Symptoms; Testing; Therapeutic; Therapeutic Effect; Time; Treatment Efficacy; Tube; United States National Institutes of Health; Variant; Vomiting; Work; arm; associated symptom; clinical efficacy; comparative efficacy; cost; decision making algorithm; design; diagnostic algorithm; diagnostic criteria; disease diagnosis; effective therapy; efficacy testing; electric impedance; evidence base; feeding; follow-up; high risk; high risk infant; hospital readmission; improved; indexing; novel; novel diagnostics; nutrition; primary endpoint; primary outcome; programs; response; secondary outcome; skills; socioeconomics; stem; success; treatment group; treatment response; unnecessary treatment

PROJECT SUMMARY/ABSTRACT The prevalence of symptom-based diagnosis of gastroesophageal reflux disease (GERD), its empiric treatment, and practice variation in high-risk hospitalized infants remain high. Prior clinical trials data lack physiological- based, objective diagnostic evidence and treatment. GERD is frequently diagnosed by inadequate criteria, and the relative risks, benefits, and indications of GERD therapies are unclear. Thus, gaps in knowledge persist, and a GERD diagnosis can cost an extra $70k and 30 days of hospitalization per infant. Objective, effective, evidence-based therapies are critically needed. As nearly all infants in the neonatal intensive care unit (NICU) are symptomatic, twenty-four-hour pH-Impedance (pH-Imp) testing can diagnose objectively and detect mechanisms of pathophysiologic GERD. Therefore, this study’s overall objective is to conduct a single-center randomized control trial (RCT) to test the central hypothesis that the effects of common therapies [natural maturation, acid suppression-proton pump inhibitor (PPI), or thickened feeds using added rice (AR) formula] are distinct in high-risk infants, under six months corrected maturational age with pH-Imp defined criteria for GERD. The rationale is to refine novel diagnostic criteria and rigorously investigate the impact of randomized therapies to achieve the long-term goal of creating effective, simplified, evidence-based, and scalable treatment algorithms. We will test this hypothesis in the following Specific Aims: Aim 1: Conduct the RCT to evaluate and compare the efficacy and safety of interventions on the primary clinical endpoint (oral feeding improvement and absence of troublesome symptoms) to test the working hypothesis that AR is superior to natural maturation and PPI. Aim 2: Evaluate pH-Imp characteristics and test the working hypotheses that pH-Imp mechanisms are distinct between a) assigned therapies, and b) clinical success and failure. This project challenges current empiric approaches to diagnosing and managing GERD in NICU infants by using both a novel combination of pH-Imp metrics and randomized allocations with targeted stratification (GERD severity and feeding skills) to determine true therapeutic effects on clinical and mechanistic characteristics. The anticipated outcomes will identify diagnostic algorithms for determining GERD and effective treatment approaches. In addition, the proposed research is significant because the knowledge gained will provide the diagnostic basis, framework, and scientific and economic rationale for future studies to further refine therapies across the pediatric age spectrum. We expect new benchmarks to develop preventative and therapeutic strategies for GERD, which will positively impact the quality of life for infants and parents by improving digestive health, overall nutrition, and growth, all relevant to the mission of NIH (NIDDK).

项目总结/文摘