Health Outcomes of Parents with Cystic Fibrosis (HOPe:CF)

患有囊性纤维化的父母的健康结果 (HOPe:CF)

基本信息

批准号：
10581321
负责人：
Traci Kazmerski
金额：
$ 76.26万
依托单位：
UNIVERSITY OF PITTSBURGH AT PITTSBURGH
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-02-01 至 2028-01-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10581321
关键词：
5 year old Address Adherence Adoption Affect Behavior Therapy Blood Body mass index Child Child Rearing Childhood Chronic Disease Clinic Visits Communication Complex Comprehensive Health Care Cross-Sectional Studies Cystic Fibrosis Cystic Fibrosis Transmembrane Conductance Regulator Data Data Collection Data Reporting Decision Making Defect Disease Fathers Fostering Foundations Funding Future Health Health Personnel Health Services Health Surveys Healthcare Heart Hormonal Intervention Interview Life Life Expectancy Link Longitudinal cohort study Lung Membrane Mental Depression Mental Health Mission Modeling Modification Mothers Multicenter Studies Observational Study Outcome Parents Patients Persons Pharmaceutical Preparations Population Pregnancy Principal Investigator Prospective Studies Psychosocial Assessment and Care Pulmonary Function Test/Forced Expiratory Volume 1 Quality of life Registries Reporting Reproductive Health Research Research Personnel Retrospective Studies Risk Factors Role Self Management Sexual Health Stress Structure Surveys Testing Time United Kingdom United States United States National Institutes of Health Woman Work comparison group effective intervention epidemiologic data evidence base experience health data high risk improved loss of function lung health men modifiable risk non-genetic patient registry physical conditioning prepregnancy prevent primary outcome prospective pulmonary function reproductive respiratory trend young woman

项目摘要

1 CF transmembrane conductance regulator (CFTR) modulators, a class of drugs that addresses the basic 2 defect in cystic fibrosis (CF), have led to large increases in the health and life expectancy of people with CF. In 3 turn, a large majority are now considering parenthood. The United States (US) CF Foundation Patient Registry 4 report (CFFPR) demonstrated a near-doubling of pregnancies in people with CF from 310 in 2019 to 619 in 2020. 5 Greater understanding of the health impacts of parenthood is critical to inform reproductive decisions and provide 6 effective interventions for those with CF who decide to become parents. 7 This co-funded application with the National Institutes of Health and the CF Foundation (CFF) will seek 8 to determine the health impact of parenthood on men and women with CF in the era of CFTR modulators. To 9 provide relatively immediate evidence on parenthood’s effect on pulmonary health and the influence of the intro- 10 duction and use of all available CFTR modulators, in Aim 1 we will assess changes in pre- vs. intra-parenthood 11 percent predicted forced expiratory volume in 1 second (ppFEV1) in a retrospective longitudinal cohort study 12 linking CFFPR data with cross-sectional surveys collected from 249 new parents attending participating US CF 13 centers between 2011-2020. We will identify predictors and timing of lung function loss using 747 non-parents 14 from participating centers as a comparison group and examine the impact of CFTR modulators on parental 15 health. In Aim 2, we will prospectively collect physical and mental health data to comprehensively evaluate the 16 impact of parenthood in CF with widespread highly effective CFTR modulator use. We will follow 146 new parents 17 of children <5 years of age over 5 years to assess the primary outcome of ppFEV1 change. To model potential 18 confounding/effect modification on the association between parenthood and outcomes, we will combine objective 19 physical health data and survey assessments of mental health, parental responsibility/stress, quality of life, and 20 adherence. We will conduct dyadic interviews with a subset of parents and their key supports to explore the 21 parenthood experience and identify future interventions. This study of the CF population would be the first, large- 22 scale epidemiologic data collection of how becoming a parent interplays with having a chronic disease. 23 Dr. Kazmerski, the principal investigator, is a pulmonologist and health services researcher with exper- 24 tise in sexual and reproductive healthcare for people with pediatric-onset chronic disease. The results of this 25 long-term, observational study will determine whether parenthood adversely impacts the health of people with 26 CF and the ability of CFTR modulators to mitigate such effects. Healthcare providers can use the data to pro- 27 vide evidence-based guidance to help those with CF make informed parenthood decisions. Future interven- 28 tions targeted to identified modifiable risk factors and high-risk time periods can be developed and tested to 29 prevent poor parental health outcomes. This work is aligned with the missions of the National Heart, Lung, and 30 Blood Institute and CFF as it improves the comprehensive care and quality of life for people with CF.

1 CF 跨膜电导调节剂 (CFTR) 调节剂，一类解决基本问题的药物 2 囊性纤维化 (CF) 缺陷导致 CF 患者的健康状况和预期寿命大幅增加。第三轮，绝大多数人现在正在考虑成为父母美国 (US) CF 基金会患者登记处。 2019 年 4 号报告 (CFFPR) 显示，囊性纤维化患者的怀孕率几乎翻了一番，从 2019 年的 310 例增加到 2020 年的 619 例。 5 更好地了解为人父母对健康的影响对于为生殖决策提供信息并提供针对决定成为父母的 CF 患者的 6 种有效干预措施。 7 这项与美国国立卫生研究院和 CF 基金会 (CFF) 共同资助的申请将寻求 8 确定在 CFTR 调节剂时代为人父母对患有 CF 的男性和女性的健康影响。 9 提供了相对直接的证据，证明为人父母对肺部健康的影响以及介绍性的影响 10 所有可用 CFTR 调节剂的归纳和使用，在目标 1 中，我们将评估亲子关系前与亲子关系中的变化在一项回顾性队列纵向研究中，11% 的人预测 1 秒用力呼气量 (ppFEV1) 12 将 CFFPR 数据与从参加参与 US CF 的 249 名新父母收集的横断面调查联系起来我们将在 2011 年至 2020 年期间对 13 个中心进行研究，利用 747 名非父母患者来确定肺功能丧失的预测因子和时间。来自参与中心的 14 个作为对照组，检查 CFTR 调节剂对父母的影响 15 健康在目标2中，我们将前瞻性地收集身心健康数据，以综合评估 16 广泛使用有效的 CFTR 调节剂对父母身份的影响我们将跟踪 146 位新父母。 17 名<5 岁的儿童评估了 5 岁以上 ppFEV1 变化的主要结果，以建立模型潜力。 18 混杂/效应修正对父母身份和结果之间的关联，我们将结合客观 19 项身体健康数据和心理健康、父母责任/压力、生活质量和调查评估 20 遵守情况。我们将与部分家长及其主要支持者进行双向访谈，以探索遵守情况。 21 为人父母的经历并确定未来的干预措施这项针对 CF 人群的研究将是第一个大规模的研究。收集 22 个规模的流行病学数据，了解为人父母与患有慢性病之间的相互作用。 23 主要研究者 Kazmerski 博士是一位肺科医生和卫生服务研究员，拥有丰富的经验 24 儿童慢性疾病患者的性和生殖保健研究结果。 25 长期观察性研究将确定为人父母是否会对患有此类疾病的人的健康产生不利影响 26 CF 和 CFTR 调节剂减轻此类影响的能力医疗保健提供者可以使用这些数据来支持。 27 提供基于证据的指导，帮助患有 CF 的人做出明智的育儿决定。可以开发和测试针对已识别的可改变风险因素和高风险时间段的 28 项措施 29 防止父母健康状况不佳这项工作与国家心肺科的使命相一致。 30 血液研究所和 CFF，因为它改善了 CF 患者的综合护理和生活质量。