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

基本信息

批准号：
10194633
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
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-04-01 至 2023-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10194633
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

项目摘要

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.

项目概要 NIDDK 的一个重要目标是尽早识别患有胆道闭锁 (BA) 的婴儿，因为在治疗之前生命中 30-45 天是避免肝移植的最佳时机。识别很困难，因为婴儿在出生后的最初几周通常不会出现任何症状。这个问题是通过血清结合胆红素测量来筛查新生儿，血清结合胆红素在出生时会升高然而，在 BA 中，这种方法需要单独抽血并需要个别分娩医院来解释和随访异常结果另一种可能的解决方案是用干血斑（DBS）卡筛查新生儿。作为现有州新生儿筛查计划的一部分，已经从所有婴儿中收集了这些信息。应用程序的长期、广泛的目标是评估是否有一种新方法可以近似共轭 DBS 卡中的胆红素水平有可能与州新生儿筛查方案相结合。我们将通过首先建立一个复制分析的实验系统来改进新方法通过该系统，我们将优化国家实验室的工作流程，实现目标 2。将使用先前收集并存储在的新生儿 DBS 卡来测试新方法德克萨斯州卫生服务部将对健康新生儿的 DBS 卡进行分析，以确定此外，还将对患有 BA 的新生儿的 DBS 卡进行分析，以确定是否存在这种情况。该方法对 BA 的敏感性与本研究的血清结合胆红素检测结果一样。确定新方法是否可用于将 BA 筛查纳入当前状态的新生儿研究结果将有助于指导未来前瞻性筛查的最佳方法。实施研究，最终目标是在生命的最初几周内识别 BA 并减少 BA 显着的小儿肝移植负担。