BIOMARKERS OF DISEASE PROGRESSION IN CHILDREN W/CF IDENTIFIED BY NEWBORN SCREEN

通过新生儿筛查识别患有 CF 的儿童疾病进展的生物标志物

• 批准号: 7605056
• 负责人: Frank J. Accurso
• 金额: $ 10.89万
• 依托单位: UNIVERSITY OF COLORADO DENVER
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-03-01 至 2008-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7605056
• 关键词: Age; Biological Markers; Bone Density; Carotene; Chest; Child; Childhood; Chronic; Complex; Computer Retrieval of Information on Scientific Projects Database; Cystic Fibrosis; DEXA; Development; Disease; Disease Progression; Early Intervention; Elastases; Fat-Soluble Vitamin; Funding; Goals; Grant; Growth; Hepatic; Hereditary Disease; Infant; Inflammation; Institution; Interleukin-6; Lipase; Liver; Lung; Lung diseases; Measurement; Neonatal Screening; Nose; Outcome; Pancreatic Elastase; Pancreatic Injury; Patients; Peroxidase; Population; Protease Inhibitor; Proteins; Pulmonary function tests; Research; Research Personnel; Resources; Serum; Source; System; Testing; Trypsinogen; United States National Institutes of Health; X-Ray Computed Tomography; base; improved; nutrition

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Cystic fibrosis (CF) is a multi-system hereditary disease characterized by pancreatic injury, poor growth, under-nutrition, chronic pulmonary disease, and the potential for hepatic disease. It is known that each of these features can be present in infants. The progression of lung disease determines survival; however how the other abnormalities relate to this progression is incompletely understood. In addition, given the difficulty with performing pulmonary function tests at a young age improved markers for lung disease are needed. The early recognition of liver involvement may allow improved treatment approaches for those patients who have hepatic disease. Within this study we aim to develop a panel of biomarkers of disease progression in CF that can predict outcome in later childhood. We also test the usefulness of this panel of biomarkers in predicting outcome most importantly pulmonary outcome. The biomarkers that we are testing are: Serum immunoreactive trypsinogen and lipase, fecal elastase, anthropometrics, DEXA to assess bone density, fat soluble vitamin and carotene levels, putative markers of inflammation: (c-reactive protein, elastase alpha one antiprotease Complexes, IL-6, ECP Myeloperoxidase), CT scan of the chest, Systemic markers of hepatic disease, and Nasal Potential Difference Measurements. Our long term objective is to use biomarkers of disease progression as a basis for early intervention. Newborn screening for CF is likely to become more widespread and hence the development of biomarkers for disease progression in a population identified through newborn screening is an important goal.

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 囊性纤维化是一种以胰腺损伤、生长不良、营养不良、慢性肺部疾病和肝脏疾病为特征的多系统遗传性疾病。众所周知，这些特征中的每一个都可能出现在婴儿身上。肺部疾病的进展决定生存；然而，其他异常如何与这种进展相关尚不完全清楚。此外，考虑到在年轻时进行肺功能测试的困难，需要改进肺部疾病的标志物。及早认识到肝脏受累可能会改善那些患有肝病的患者的治疗方法。在这项研究中，我们的目标是开发一组可以预测儿童后期预后的CF疾病进展的生物标记物。我们还测试了这组生物标记物在预测结果方面的有用性，最重要的是肺结果。我们正在测试的生物标记物包括：血清免疫反应性胰蛋白酶原和脂肪酶、粪便弹性蛋白酶、人体测量学、用于评估骨密度的DEXA、脂溶维生素和胡萝卜素水平、假定的炎症标记物：(C反应蛋白、弹力酶α1抗蛋白复合体、IL-6、ECP髓过氧化物酶)、胸部CT扫描、肝病系统标记物和鼻部电位差测量。我们的长期目标是使用疾病进展的生物标志物作为早期干预的基础。新生儿CF筛查可能会变得更加普遍，因此，在通过新生儿筛查确定的人群中开发疾病进展的生物标记物是一个重要目标。