Impact of Gender on Symptoms and Progression of IPF

这个K23奖项的目标是提供候选人所需的资源，以开发独立的 从事终末期肺部疾病临床研究的主要内容如下：(1)聚焦 在纵向数据分析、非参数统计、处理 缺少数据和以患者为中心的结果；(2)获得进一步实践经验的保护时间 作为IPF CRN的研究实习生进行临床试验；以及，(3)在两位博士的指导下密切指导。 费尔南多·J·马丁内斯和凯文·弗莱厄蒂，在IPF临床研究和 成功的指导。为了最大限度地实现这些目标，研究计划不仅将寻求前瞻性地 描述特发性肺疾病在生理进展、存活率和症状方面的性别差异 纤维化(IPF)，但也使用临床和生物学数据调查这些差异可能存在的原因 IPF CRN和肺组织研究联盟。回顾数据表明，患有IPF的男性进展 速度更快，存活率更差。初步数据表明，可测量的生物(肽) 激素松弛素)和生理(肺动脉收缩压)的差异对性别有影响 疾病表型的差异。具体目标包括：(1)确定男性和女性IPF表型 通过比较基线生物和生理参数(1b)，确定两者之间的关系 生物和生理参数以更好地了解IPF性别差异背后的机制 表型；(2a)确定性别是否影响肺功能的纵向变化，并探讨 已知与性别有关的其他因素的贡献改善(2b)比较急性 男性和女性之间的恶化(2c)前瞻性地决定了女性是否相关 改善了IPF中的存活率，独立于与 性别；(3a)确定基线健康状况和症状衡量，特别是焦虑和 抑郁，在患有IPF的女性中比男性更不正常(3b)决定了性别差异 存在于纵向行为的健康状况和症状措施。 相关性(请参阅说明)： 这项研究有可能对公共卫生产生重大影响。结果不仅会带来更好的结果 对IPF的预测和对性别在设计慢性阻塞性肺疾病未来研究中的作用的更深入的了解 肺部疾病，但也将提供关键数据，可能导致IPF生物标志物的开发 和治疗性的临床试验。

The goal of this K23 award is to provide the candidate resources needed to develop an independent research career in the clinical investigation of end stage lung disease including the following: (1) focused additional didactic training in the analysis of longitudinal data, nonparametric statistics, the handling of missing data, and patient centered outcomes; (2) protected time to gain further practical experience in clinical trial conduct as an investigative trainee of the IPF CRN; and, (3) close mentoring under both Drs. Fernando J. Martinez and Kevin Flaherty, individuals with extensive experience in IPF clinical research and successful mentoring. To maximize these goals the research plan will not only seek to prospectively describe gender differences in physiologic progression, survival, and symptoms in idiopathic pulmonary fibrosis (IPF) but also investigate why these differences might exist using clinical and biologic data from the IPF CRN and Lung Tissue Research Consortium. Retrospective data suggests that men with IPF progress more rapidly and experience worse survival. Preliminary data suggest that measurable biologic (peptide hormone relaxin) and physiologic (pulmonary artery systolic pressure) differences contribute to gender differences in disease phenotype. Specific aims include: (1a) characterize male and female IPF phenotypes through baseline comparison of biologic and physiologic parameters (1 b) determine the relationship between biologic and physiologic parameters to better understand mechanisms behind gender differences in IPF phenotype; (2a) determine if gender influences longitudinal change in pulmonary function and explore the contribution of other factors improves known to be associated with gender (2b) compare the rate of acute exacerbations between men and women (2c) determine prospectively whether female gender is associated with improved survival in IPF independent of other biologic and physiologic parameters associated with gender; (3a) determine whether baseline health status and symptom measures, particularly anxiety and depression, are more abnormal in women with IPF than men (3b) determine whether gender differences exist in the longitudinal behavior of health status and symptom measures. RELEVANCE (See instructions): This research has the potential for significant public health impact. The results could lead not only to better prognostication of IPF and improved insights into the role of gender in the design of future studies of chronic lung disease but will also provide key data that could lead to the both the development of a biomarker for IPF and a therapeutic clinical trial.