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

1个CF跨膜电导调节剂(CFTR)调节剂，这类药物解决了 2囊性纤维化的缺陷，导致囊性纤维化患者的健康和预期寿命大幅增加。在……里面 现在，绝大多数人都在考虑为人父母。美国(US)CF基金会患者登记处 4报告(CFFPR)显示，CF患者的妊娠率几乎翻了一番，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指导生育决策和提供 6对那些决定成为父母的CF患者进行有效的干预。 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调节器时代，为人父母对患有CF的男性和女性的健康影响。至 9为父母对肺健康的影响以及家庭成员的影响提供了相对直接的证据。 10引入和使用所有可用的CFTR调节器，在目标1中，我们将评估育儿前和育儿内的变化 在一项回溯性纵向队列研究中，11%的人预测1秒用力呼气量(PpFEV1) 12将CFFPR数据与从249名参加美国CF的新父母收集的横断面调查相联系 2011-2020年间有13个中心。我们将使用747名非父母来确定肺功能丧失的预测因素和时间 14个参与中心作为对照组，检测CFTR调节子对亲本的影响 15健康。在目标2中，我们将前瞻性地收集身心健康数据，以全面评估 16在广泛使用高效CFTR调制器的情况下，为人父母在CF中的影响。我们将关注146位新父母 17名5岁及5岁以上儿童评估ppFEV1改变的主要结果。对潜力进行建模 18关于父母身份和结果之间的关联的混淆/影响修改，我们将结合目标 19身体健康数据和心理健康、父母责任/压力、生活质量以及 20坚持。我们将对一部分家长和他们的关键支持进行二元访谈，以探索 21为人父母的经历并确定未来的干预措施。这项关于CF种群的研究将是第一次大规模的- 22大规模流行病学数据收集如何成为父母与患有慢性病的相互作用。 23卡默斯基博士是首席研究员，是一名肺科医生和卫生服务研究员，患有- 24为患有儿科慢性病的人提供性和生殖保健。这样做的结果 25项长期的观察性研究将确定为人父母是否会对患有癌症的人的健康产生不利影响 26 cf和CFtr调制器缓解这种影响的能力。医疗保健提供者可以使用这些数据来支持- 27通过循证指导，帮助患有CF的人做出明智的育儿决定。未来的干预- 可以针对已确定的可修改风险因素和高风险时段制定和测试28项计划，以 29预防不良的父母健康结果。这项工作与国家心肺和 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。

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.