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

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的一个重要目标是更早地识别患有胆道闭锁（BA）的婴儿，因为治疗前 30-45天的生命与避免需要肝移植的最佳机会相关。然而，早 识别很困难，因为婴儿在出生后的头几周通常没有症状。的一个解决方案 这个问题是用出生时升高的血清结合胆红素测量值来筛查新生儿 然而，在BA中，这种方法需要单独的抽血和各个出生医院来解释， 随访结果异常。另一个可能的解决方案是用干血斑（DBS）卡筛查新生儿 作为现有的国家新生儿筛查计划的一部分，已经从所有婴儿身上收集了这些信息。这 申请的长期，广泛的目标是评估是否有一种新的方法， DBS卡的胆红素水平可能与州新生儿筛查方案相结合。在目标1中， 我们将通过首先建立一个实验系统来改进新方法， 国家实验室的工作流程。利用该系统，可以对新方法的参数进行优化。在目标2中， 将使用先前收集并储存在 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 新方法的阳性率。此外，还将分析BA新生儿的DBS卡，以确定 该方法对BA的灵敏度与血清结合胆红素测试一样。这项研究的结果应该 确定新方法是否可用于将BA筛查纳入当前状态新生儿 筛选方案。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， 严重的儿科肝移植负担。