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: 8056171
• 负责人: HUICHUAN J LAI
• 金额: $ 8.04万
• 依托单位: UNIVERSITY OF WISCONSIN-MADISON
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-05-01 至 2011-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8056171
• 关键词: 20 year old; Accounting; Address; Adult; Age; Caring; Caucasians; Caucasoid Race; Centers for Disease Control and Prevention (U.S.); Cessation of life; Child; Childhood; Clinical; Cognitive; Complex; Cystic Fibrosis; Data; Databases; Diagnosis; Disease Association; Disease Progression; Early Diagnosis; Epidemiologic Studies; Epidemiologist; Foundations; Future; Grant; Health; Health Policy; Hereditary Disease; Incidence; Investigation; Knowledge; Life; Lung; Lung diseases; Malnutrition; Medical; Mentored Research Scientist Development Award; Methods; Modeling; Monitor; Neonatal Screening; Nutritional; Nutritional status; Obstructive Lung Diseases; Outcome; Outcome Study; Peer Review; Policy Maker; Principal Investigator; Program Effectiveness; Public Health; Randomized Controlled Trials; Recommendation; Recurrence; Registries; Reporting; Research; Research Personnel; Respiratory Failure; Respiratory Tract Infections; Risk; Screening procedure; Signs and Symptoms; Testing; Therapeutic Intervention; Uncertainty; Wisconsin; Work; clinical practice; cohort; cystic fibrosis patients; follow-up; improved; infancy; patient registry; predictive modeling; programs; tool; trial comparing

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）是最常见的危及生命的遗传性疾病，在白人中的发病率约为1：3400。虽然CF开始于婴儿早期，但延迟诊断是常见的，通常会导致严重的，可能不可逆转的营养不良。阻塞性肺病伴反复呼吸道感染也是不可避免的;大约80%的人死于心肺功能衰竭，三分之一的人在20岁之前死亡。本申请的总体目标是通过流行病学研究量化CF中营养不良和肺部疾病之间的关联，以改善CF的筛查和治疗。具体目标是：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基金会患者登记和威斯康星州CF新生儿筛查随机对照试验（RCT），开发预测营养不良和肺部疾病关联的风险模型，以检验营养不良与肺部疾病相关的假设，和2）检验实施常规全州新生儿筛查计划将提供先前在RCT中证明的类似医疗益处的假设，通过比较1994年开始实施的威斯康星州常规囊性纤维化新生儿筛查计划与威斯康星州随机对照试验队列中诊断的儿童队列的营养和肺部状况。没有其他调查有类似的研究人员和工具来进行拟议的工作。凭借K 01奖，主要研究者已发展成为全国公认的CF流行病学家，在营养和肺部方面具有特殊专长。她与Specifc Aim 1相关的工作已经导致了CF临床实践的变化。鉴于疾病预防控制中心2004年的新报告建议在全国范围内实施CF新生儿筛查计划，并进行随访结果研究以评估计划的有效性，在不久的将来完成特定目标2的临床影响也是可以预见的。实现该补助金的具体目标将促进我们对营养不良对CF中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，并解决公共卫生政策制定者实施常规CF新生儿筛查计划的益处的争议。