Predicting Severity and Improving the Outcomes of Pediatric Pancreatitis

预测小儿胰腺炎的严重程度并改善预后

• 批准号: 10415235
• 负责人: Maisam Abu-El-Haija
• 金额: $ 18.93万
• 依托单位: CINCINNATI CHILDRENS HOSP MED CTR
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-07-17 至 2023-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10415235
• 关键词: Admission activity; Adult; Albumins; Biological Markers; Biometry; Caring; Categories; Characteristics; Child; Childhood; Clinical; Collaborations; Cystic Fibrosis Transmembrane Conductance Regulator; Data; Development; Diabetes Mellitus; Disease; Disease Progression; Elements; Endocrine; Environment; Evaluation; Foundations; Funding; Future; Gastroenterologist; Gastroenterology; Generations; Genes; Genetic; Genetic Markers; Genetic Models; Genetic study; Glucose Intolerance; Goals; Grant; Health; Hepatology; Heritability; High-Throughput Nucleotide Sequencing; Hospital Costs; IV Fluid; Incidence; Institution; Investigation; Knowledge; Laboratories; Lead; Length of Stay; Link; Lipase; Longitudinal prospective study; Matrix Metalloproteinases; Medical Genetics; Medical center; Mentors; Mentorship; Methods; Modeling; Morbidity - disease rate; National Institute of Diabetes and Digestive and Kidney Diseases; Natural History; Nature; Outcome; Outcomes Research; Pancreas; Pancreatitis; Patients; Pattern; Pediatric Hospitals; Pediatrics; Performance; Plasma; Population; Prediabetes syndrome; Prevention therapy; Prospective Studies; Public Health; Registries; Research; Research Design; Research Personnel; Resources; Retrospective cohort; Risk; Risk Management; Role; SPINK1 gene; Scientist; Severities; Severity of illness; Small Business Innovation Research Grant; System; Testing; Time; Tissues; Training; United States; Validation; Variant; Work; acute pancreatitis; adverse outcome; base; biobank; career; career development; clinical predictors; cohort; design; disease phenotype; epidemiology study; follow-up; gene panel; genetic analysis; genetic predictors; genetic testing; high risk; improved; improved outcome; indexing; inhibitor; innovation; mortality; novel; novel marker; novel therapeutics; nutrition; optimal treatments; patient subsets; pediatric department; predictive modeling; prevent; professor; prognostic; prognostic model; prognostic performance; prognostic tool; prospective; risk prediction; risk stratification; screening; skills; targeted treatment; tool

Project Summary/Abstract The incidence of pediatric Acute Pancreatitis (AP) has been rising to 1/10,000 cases, close to adult incidence. AP in pediatrics remains understudied with outcomes poorly defined. Previous studies in pediatric AP are mostly single-centered and retrospective in nature, and thereby insufficient for understanding the natural history. A high percentage (15-30%) of patients develop severe acute pancreatitis (SAP) with increased morbidity, increased length of hospital stay and cost. To date, there is no pediatric AP study that examines progression prospectively like we plan through our design, there is no validated pediatric prognostic severity system to improve outcomes of AP. Diabetes can result from AP in a subset of patients, and that leads to increased morbidity especially if underdiagnosed, given that there is no current method for screening post AP. The primary goal of my proposed career development is to acquire additional comprehensive training in biostatistics, study design, epidemiology and outcome research through risk modeling, to acquire the skills necessary to build multicenter collaborations to study AP risk stratification and management that lead to the improved outcomes and decreased morbidity and mortality. I am an Assistant Professor of Pediatrics and a board certified Gastroenterologist in the Division of Gastroenterology, Hepatology and Nutrition in the Department of Pediatrics at Cincinnati Children’s Hospital Medical Center (CCHMC). CCHMC is committed to improving the health outcomes of children through innovative research. The exceptional environment at CCHMC facilitates the development of young investigators by having essential elements through core resources, expert mentorship, as well as the potential for internal collaboration. The pancreas Care Center at CCHMC is one of only a few pediatric pancreas centers in the United States and has an established referral pattern, with 70-90 new patients referred per year. My mentors Dr. Lee A. Denson- a nationally recognized leader in pediatric gastroenterology, Dr. Sohail Husain- a national expert and scientist in pediatric pancreatology, Dr. Woo- an expert in epidemiology and outcome research are invested in my training and guiding my path to be an independent investigators. The primary objective of this project is to improve outcomes of AP by decreasing SAP and improve screening for prediabetes. This will be accomplished through our prospective longitudinal study design. Specific Aim 1 to validate and optimize our previously generated SAP model by adding more factors, applying the new SAP definition, incorporating therapy effect, and incorporating MMPs and TIMPs as novel biomarkers. Specific Aim 2 to build a model to predict prediabetes post AP and understand the role of expanded gene testing in disease progression to prediabetes. Our proposal will help us better understand SAP and progression to prediabetes to fill the knowledge gap. Successful completion of this study has the potential to lead to improved outcomes by building clinical tools that facilitate management, and potentially lead to targeted therapies for AP in pediatrics.

项目摘要/摘要 小儿急性胰腺炎（AP）的事件已升至1/10,000例，接近成人事件。 儿科中的AP仍然是对结果的理解。小儿AP的先前研究是 本质上大多以单一中心和回顾性，因此不足以理解自然 历史。高百分比（15-30％）患者患严重急性胰腺炎（SAP），并增加 发病率，住院时间增加和费用。迄今为止，还没有小儿AP研究 前瞻性地进展，就像我们通过设计计划一样，没有经过验证的小儿预后严重程度 改善AP结果的系统。糖尿病可能是由一部分患者中的AP引起的，这导致 鉴于当前没有筛选AP的方法，尤其是在诊断未诊断的情况下，发病率的增加。 我提议的职业发展的主要目标是获得其他综合培训 通过风险建模，生物统计学，研究设计，流行病学和结果研究，以获取技能 建立多中心合作以研究AP风险分层和管理所需的必要 改善结果，提高了发病率和死亡率。 我是儿科助理教授和董事会认证的胃肠病学家 辛辛那提儿童医院儿科部的胃肠病学，肝病学和营养 医疗中心（CCHMC）。 CCHMC致力于通过 创新研究。 CCHMC的特殊环境促进了年轻的发展 通过通过核心资源，专家精神和潜在的核心资源，专家精神制定基本要素的研究人员 用于内部协作。 CCHMC的胰腺护理中心是仅有的少数小儿胰腺中心之一 在美国，并具有既定的转诊模式，每年有70-90名新患者。我的 导师Lee A. Denson博士 - 儿科胃肠病学领导者，Sohail Husain-a博士 小儿胰腺学专家和科学家Woo博士 - 流行病学和结果专家 研究投入了我的培训，并指导我成为独立调查员的道路。主要 该项目的目的是通过减少SAP和改进筛选来改善AP的结果 糖尿病。这将通过我们的前瞻性纵向研究设计来实现。具体目标1 通过添加更多因素，应用新的SAP来验证和优化我们先前生成的SAP模型 定义，治疗效果的增加以及增加了MMP和TIMP作为新型生物标志物。具体目标 2建立一个模型以预测AP后的糖尿病前期并了解扩展基因测试在疾病中的作用 发展为糖尿病前期。我们的建议将有助于我们更好地了解SAP和糖尿病前期 填补知识空白。这项研究的成功完成有可能通过 建立促进管理的临床工具，并有可能导致儿科中AP的靶向疗法。

项目成果

Pancreatitis in the Complex Care Population: Presentation, Incidence, and Severity.

• DOI: 10.1097/mpg.0000000000003610
• 发表时间: 2022-12-01
• 期刊: JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION
• 影响因子: 2.9
• 作者: Hawa, Kathryn;Corker, Lisa;Hornung, Lindsey;Noritz, Garey;Gariepy, Cheryl;Shaikhkhalil, Ala;Abu-El-Haija, Maisam
• 通讯作者: Abu-El-Haija, Maisam

Menstruation-Associated Acute Pancreatitis in Patients with Hereditary Pancreatitis.

• DOI: 10.1016/j.jpeds.2020.11.024
• 发表时间: 2021-03
• 期刊: The Journal of pediatrics
• 作者: Khalaf R;Narkewicz M;El-Haija MA
• 通讯作者: El-Haija MA

Ultrasound findings of acute pancreatitis in children.

• DOI: 10.1007/s00247-022-05381-z
• 发表时间: 2022-11
• 期刊: Pediatric radiology
• 影响因子: 2.3

Continuous Glucose Monitoring in the Intensive Care Unit Following Total Pancreatectomy with Islet Autotransplantation in Children: Establishing Accuracy of the Dexcom G6 Model.

• DOI: 10.3390/jcm10091893
• 发表时间: 2021-04-27
• 期刊: Journal of clinical medicine
• 影响因子: 3.9
• 作者: Segev N;Hornung LN;Tellez SE;Courter JD;Lawson SA;Nathan JD;Abu-El-Haija M;Elder DA
• 通讯作者: Elder DA

A Practical Approach to Management of Acute Pancreatitis: Similarities and Dissimilarities of Disease in Children and Adults.

• DOI: 10.3390/jcm10122545
• 发表时间: 2021-06-08
• 期刊: Journal of clinical medicine
• 影响因子: 3.9
• 作者: Sellers ZM;Barakat MT;Abu-El-Haija M
• 通讯作者: Abu-El-Haija M