Evaluation of a New Method of Bilirubin Screening for Biliary Atresia

胆红素筛查胆道闭锁新方法的评价

• 批准号: 10379379
• 负责人: SANJIV HARPAVAT
• 金额: $ 12万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-04-01 至 2025-03-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10379379
• 关键词: 1 year old; Accounting; Address; Age; Alagille Syndrome; Bile fluid; Biliary Atresia; Bilirubin; Biological Assay; Birth; Blood; Child; Childhood; Data; Detection; Diagnosis; Disease; Drainage procedure; Early identification; Eligibility Determination; Ensure; Evaluation; Foundations; Funding; Future; Goals; Growth; Health Services; Heel; Hospitals; Individual; Infant; Infrastructure; Intestines; K-Series Research Career Programs; Laboratories; Life; Liver; Liver diseases; Measurement; Measures; Mentored Patient-Oriented Research Career Development Award; Methods; National Institute of Diabetes and Digestive and Kidney Diseases; Neonatal Screening; Newborn Infant; Outcome; Peer Review Grants; Problem Solving; Process; Prospective Studies; Publications; Publishing; Research Personnel; Sampling; Screening Result; Serum; Signal Transduction; Specificity; Spottings; System; Testing; Texas; Transplantation; United States; United States National Institutes of Health; alpha 1-Antitrypsin Deficiency; base; bile duct; experimental study; follow-up; implementation study; improved; improved outcome; liver injury; liver transplantation; novel; programs; prospective; screening; screening program; trend; 1 year old; Accounting; Address; Age; Alagille Syndrome; Bile fluid; Biliary Atresia; Bilirubin; Biological Assay; Birth; Blood; Child; Childhood; Data; Detection; Diagnosis; Disease; Drainage procedure; Early identification; Eligibility Determination; Ensure; Evaluation; Foundations; Funding; Future; Goals; Growth; Health Services; Heel; Hospitals; Individual; Infant; Infrastructure; Intestines; K-Series Research Career Programs; Laboratories; Life; Liver; Liver diseases; Measurement; Measures; Mentored Patient-Oriented Research Career Development Award; Methods; National Institute of Diabetes and Digestive and Kidney Diseases; Neonatal Screening; Newborn Infant; Outcome; Peer Review Grants; Problem Solving; Process; Prospective Studies; Publications; Publishing; Research Personnel; Sampling; Screening Result; Serum; Signal Transduction; Specificity; Spottings; System; Testing; Texas; Transplantation; United States; United States National Institutes of Health; alpha 1-Antitrypsin Deficiency; base; bile duct; experimental study; follow-up; implementation study; improved; improved outcome; liver injury; liver transplantation; novel; programs; prospective; screening; screening program; trend

PROJECT SUMMARY An important goal of the NIDDK is to identify infants with biliary atresia (BA) earlier, because treatment before 30-45 days of life correlates with the best chances of avoiding need for liver transplantation. However, early identification is difficult, because infants often appear asymptomatic in the first weeks of life. One solution to this problem is screening newborns with serum conjugated bilirubin measurements, which are elevated at birth in BA; however, this method requires a separate blood draw and individual birth hospitals to interpret and follow-up abnormal results. Another possible solution is to screen newborns with dried blood spot (DBS) cards which are already collected from all infants as part of existing state newborn screening programs. This application’s long, broad-term objective is to assess whether a new method which approximates conjugated bilirubin levels from DBS cards can potentially be integrated with state newborn screening protocols. In Aim 1, we will improve the new method by first establishing an experimental system that replicates the analytical workflow of state laboratories. With this system, the new method’s parameters will be optimized. In Aim 2, we will test the new method using newborn DBS cards which were previously collected and are stored at the Texas State Department of Health Services. DBS cards from healthy newborns will be analyzed, to determine the new method’s positive rate. In addition, DBS cards from newborns with BA will be analyzed, to determine if the method is as sensitive for BA as serum conjugated bilirubin testing. Results from this study should determine whether the new method can be used to incorporate BA screening into current state newborn screening protocols. The findings will help guide the best approach for future prospective screening implementation studies, with the ultimate goal of identifying BA in the first weeks of life and reducing BA’s significant pediatric liver transplantation burden.

项目摘要 NIDDK的一个重要目标是较早地识别患有胆道闭锁（BA）的婴儿，因为 30-45天的生命与避免需要肝移植的最佳机会相关。但是，早 识别很困难，因为婴儿在生命的头几周通常会出现无症状。一种解决方案 这个问题是用血清共轭胆红素测量的新生儿筛查，它们在出生时升高 在ba;但是，这种方法需要单独抽血和个别出生医院来解释和 随访异常结果。另一个可能的解决方案是用干血（DBS）卡筛选新生儿 作为现有州新生儿筛查计划的一部分，已经从所有婴儿那里收集。这 应用程序漫长的广泛目标是评估一种近似结合的新方法 DBS卡中的胆红素水平可能与州新生儿筛查方案相结合。在AIM 1中， 我们将首先建立复制分析的实验系统来改善新方法 国家实验室的工作流程。使用此系统，将优化新方法的参数。在AIM 2中，我们 将使用以前收集的新生儿DBS卡测试新方法，并存储在 德克萨斯州卫生服务部。将分析来自健康新生儿的DBS卡，以确定 新方法的正率。此外，还将分析来自新生儿的DBS卡，以确定是否 该方法对BA就像血清共轭胆红素测试一样敏感。这项研究的结果应 确定是否可以使用新方法将BA筛选纳入当前状态新生儿 筛选协议。这些发现将有助于指导未来预期筛查的最佳方法 实施研究，其最终目的是在生命的头几周识别BA并减少BA 明显的小儿肝移植伯恩。