MALNUTRITION AND LUNG DISEASE IN CYSTIC FIBROSIS

囊性纤维化中的营养不良和肺部疾病

• 批准号: 7146230
• 负责人: HUICHUAN J LAI
• 金额: $ 26.66万
• 依托单位: UNIVERSITY OF WISCONSIN-MADISON
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-07-15 至 2011-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7146230
• 关键词: adolescence (12-20); child (0-11); chronic obstructive pulmonary disease; clinical research; cystic fibrosis; disease /disorder proneness /risk; human subject; lung disorder; malnutrition; mass screening; model design /development; nutrition related tag; outcomes research; patient oriented research; prognosis; respiratory infections

DESCRIPTION (provided by applicant): Cystic fibrosis (CF) is the most common life-threatening genetic disorder with an incidence of approximately 1:3400 in Caucasians. Although CF begins in early infancy, delayed diagnosis is common and often leads to severe, potentially irreversible malnutrition. Obstructive lung disease with recurrent respiratory infections is also inevitable; about 80 percent die from cardio respiratory failure and one-third die before age 20 years. The overall objective of this application is to quantify the associations between malnutrition and lung disease in CF by utilizing epidemiological studies in order to improve screening and treatment of CF. Specific aims are: 1) to test the hypothesis that malnutrition worsens lung disease, by developing risk models predictive of malnutrition and lung disease associations through the use of two ongoing, complimentary databases collected since 1985, namely, the CF Foundation Patient Registry and the Wisconsin Randomized Controlled Trial (RCT) of CF Neonatal Screening, and 2) to test the hypothesis that implementing a routine statewide neonatal screening program will provide similar medical benefits previously proven in RCT, by comparing the nutritional and pulmonary status of the cohort of children diagnosed through the Wisconsin Statewide Routine Cystic Fibrosis Newborn Screening Program since its inception in 1994 to the Wisconsin RCT cohort. No other investigation has comparable researchers and tools to conduct the proposed work. With a K01 award, the principal investigator has developed into a nationally recognized CF epidemiologist, with special expertise in both nutritional and pulmonary aspects. Her work related to Specifc Aim 1 has already led to changes in the clinical practices of CF. In view of the CDC's new 2004 report that recommends nationwide implementation of CF newborn screening programs with follow-up outcome studies to evaluate program effectiveness, the clinical impact as a result of completing Specific Aim 2 is also foreseeable in the near future. Accomplishing the specific aims of this grant will advance our understanding of the impact of malnutrition on lung disease progression iri CF, change the clinical practices of CF, improve the health outcomes of CF patients, and resolve the controversy on the benefits of implementing routine CF neonatal screening program for public health policy makers.

描述（由申请人提供）：囊性纤维化（CF）是最常见的威胁生命的遗传性疾病，在白种人中发病率约为1:3400。虽然CF始于婴儿期早期，但延迟诊断是常见的，往往导致严重的、可能不可逆转的营养不良。伴有反复呼吸道感染的阻塞性肺病也是不可避免的；大约80%的人死于心肺衰竭，三分之一的人在20岁之前死亡。本应用程序的总体目标是通过流行病学研究量化CF中营养不良与肺部疾病之间的关联，以改善CF的筛查和治疗。具体目标如下：1)通过使用自1985年以来收集的两个正在进行的免费数据库，即CF基金会患者登记处和CF新生儿筛查的威斯康星随机对照试验（RCT），通过建立预测营养不良和肺部疾病关联的风险模型，验证营养不良恶化肺部疾病的假设。2)通过比较自1994年开始实施的威斯康星州常规囊性纤维化新生儿筛查项目与威斯康星州随机对照试验队列诊断的儿童的营养和肺部状况，验证实施常规全州新生儿筛查项目将提供与之前在随机对照试验中证实的类似的医疗效益的假设。没有其他调查有可比的研究人员和工具来进行拟议的工作。凭借K01奖，首席研究员已经发展成为全国公认的CF流行病学家，在营养和肺部方面都有特殊的专业知识。她与Specific Aim 1相关的工作已经改变了CF的临床实践。鉴于CDC在2004年的新报告中建议在全国范围内实施CF新生儿筛查项目，并进行随访结果研究，以评估项目的有效性，完成Specific Aim 2的临床影响也可以预见在不久的将来。完成这项资助的具体目标将促进我们对营养不良对CF肺部疾病进展的影响的理解，改变CF的临床实践，改善CF患者的健康结果，并为公共卫生政策制定者解决关于实施常规CF新生儿筛查计划的益处的争议。