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)

• 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
• 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

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，一类针对基本的药物 2囊性纤维化（CF）缺陷导致CF患者的健康和预期寿命大大提高。在 3转，绝大多数正在考虑育儿。美国（美国）CF基金会患者注册表 4报告（CFFPR）显示，CF患者的怀孕几乎增加了2019年的310人，到2020年的619人。 5对父母身份的健康影响的更多了解对于告知生殖决策并提供 6对于决定成为父母的患者的有效干预措施。 7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在CFTR调节剂时代，确定父母对患有CF的男性和女性的健康影响。到 9提供相对直接的证据，证明父母对肺部健康的影响以及介绍的影响 10所有可用CFTR调制器的销量和使用，在AIM 1中，我们将评估Pre-pre-Inter-of-ostrenthood的变化 11％的预测在回顾性纵向队列研究中1秒（PPFEV1）的强迫呼气量（PPFEV1） 12将CFFPR数据与249位新父母的横断面调查联系起来 2011 - 2020年之间的13个中心。我们将使用747个非父母确定肺功能损失的预测因素和时间安排 14从参与中心作为比较组，并检查CFTR调制器对父母的影响 15健康。在AIM 2中，我们可能会收集身心健康数据，以全面评估 16在CF中使用宽度高效的CFTR调制器的影响。我们将关注146个新父母 5岁以上5岁的儿童中有17个评估PPFEV1变化的主要结果。建模潜力 18对父母与结果之间关联的混淆/效果修改，我们将结合目标 19身体健康数据和调查评估，父母责任/压力，生活质量以及 20依从性。我们将向父母的一部分及其主要支持进行二元采访，以探索 21父母的经验并确定未来的干预措施。这项对CF人群的研究将是第一个大型的 22量表流行病学数据收集如何成为父母与患有慢性疾病的相互作用。 23首席研究员Kazmerski博士是专家的肺科医生和卫生服务研究员 24针对儿科患者慢性病患者的性和生殖医疗保健。结果的结果 25长期观察性研究将确定父母身份是否会对患者的健康产生不利影响 26 CF以及CFTR调节器减轻此类效果的能力。医疗保健提供者可以使用数据来支持 27基于视频证据的指导，以帮助患有CF的人做出明智的父母决定。未来介入 - 28针对识别可修改风险因素和高风险时间段的目标可以开发和测试 29防止父母健康状况不佳。这项工作与民族心脏，肺部和 30血液研究所和CFF可以改善CF患者的综合护理和生活质量。