Mentored patient-oriented research in preschool wheezing disorders

指导以患者为导向的学前喘息障碍研究

• 批准号: 10055039
• 负责人: Anne Mentro Fitzpatrick
• 金额: $ 12.6万
• 依托单位: EMORY UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-09-01 至 2025-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10055039
• 关键词: 6 year old; Address; Adrenal Cortex Hormones; Affect; Age; Anxiety; Area; Asthma; Award; Biological; Caregivers; Child; Chronic Disease; Clinical; Clinical Research; Cohort Studies; Collaborations; Complex; Disease; Economic Burden; Education; Emergency department visit; Ensure; Environment; Esthesia; Expression Profiling; Family; Fatigue; Fostering; Future; Gene Expression; Goals; Health; Impairment; Incidence; Inflammatory; Infrastructure; Intervention Studies; Malignant Neoplasms; Mental Depression; Mental Health; Mentors; Molecular Biology; Morbidity - disease rate; Nursery Schools; Outcome; Pain; Pathway interactions; Patient Outcomes Assessments; Phenotype; Play; Population; Preschool Child; Prevalence; Principal Investigator; Productivity; Psyche structure; Public Health; Quality of life; Recurrence; Research; Research Activity; Research Design; Resources; Respiratory Signs and Symptoms; Role; Severities; Sleep disturbances; Statistical Methods; Statistical Study; Strategic Planning; Symptoms; Testing; Time; Training; United States; Universities; Virus Diseases; Wheezing; Work; affective disturbance; associated symptom; career development; cohort; cost; experience; functional status; hospitalization rates; impaired functional status; improved; innovation; mRNA sequencing; next generation sequencing; novel marker; patient oriented; patient oriented research; personalized intervention; physical conditioning; physical symptom; precision medicine; predict clinical outcome; primary outcome; programs; secondary outcome; social; symptom cluster; symptom science; therapy design

PROJECT SUMMARY/ABSTRACT Wheezing is a highly prevalent symptom among preschool children that is notoriously difficult to treat. While the factors associated with wheezing are complex, it is also recognized that preschool children are a heterogeneous group, with differing symptom profiles that may contribute to differing clinical outcomes and disease trajectories. However, symptom science is quite limited in preschool children and has focused primarily on respiratory symptoms in isolation, ignoring other physical symptoms and symptoms of mental and social health in caregivers that are important to overall functional status and quality of life. To date, there has been no attempt to identify or study symptom clusters (defined as two or more concurrent symptoms independent of other clusters) in preschool children with recurrent wheezing. This K24 application entitled “Mentored Patient-Oriented Research in Preschool Wheezing Disorders” seeks five years of support for protected time to expand the Principal Investigator’s mentoring activities, promote her career development and provide momentum to advance her research on symptom clustering in preschool children with recurrent wheezing. The Principal Investigator has built an extensive research program on preschool wheezing in an environment with unparalleled infrastructure, resources and collaborators. Career Development goals are to ensure that the Principal Investigator will be provided sufficient time for mentoring and patient-oriented clinical research activities. The award will also allow the Principal Investigator to further her education in statistical methods and study design, molecular biology, and precision medicine to help her expand her patient-oriented research program. This training will also facilitate the design of interventions to reduce the severity and incidence of wheezing in young children. The overarching research hypothesis is that symptom clusters and their associated inflammatory pathways predict clinical outcomes in preschool children age 12-59 months with recurrent wheezing. Specific aims are to determine whether symptom cluster predicts exacerbation (primary outcome) and quality of life (secondary outcome) and to utilize next-generation sequencing to identify pathways underlying symptom clusters and their trajectories. This project addresses innovative questions in symptom science and may result in identification of novel biomarkers for personalized intervention. Greater understanding of symptoms underlying morbidity will also help tackle a critical public health problem (wheezing) that affects a growing proportion of children in the United States.

项目总结/摘要 喘息是学龄前儿童中非常普遍的症状，众所周知，难以治疗。 与喘鸣相关的因素是复杂的，也认识到学龄前儿童是一个 异质性组，具有不同的症状特征，可能导致不同的临床结局， 疾病轨迹然而，症状科学在学龄前儿童中相当有限， 主要是孤立的呼吸道症状，忽视其他身体症状和精神症状， 照顾者的社会健康对整体功能状态和生活质量很重要。迄今为止， 没有尝试识别或研究症状群（定义为两个或多个并发症状 独立于其他集群）在学龄前儿童反复喘息。该K24应用程序的标题为 “学龄前喘息性疾病的指导性患者导向研究”寻求五年的支持， 保护时间，以扩大主要研究者的指导活动，促进其职业发展， 提供动力，以推进她对学龄前儿童反复发作的症状聚集的研究 气喘首席研究员已经建立了一个广泛的研究计划，对学龄前喘息， 环境，拥有无与伦比的基础设施、资源和协作者。职业发展目标是 确保为主要研究者提供足够的时间进行指导和以患者为导向的临床研究 研究活动。该奖项还将使首席研究员能够进一步接受统计学方面的教育 方法和研究设计，分子生物学，和精准医学，以帮助她扩大她的病人为导向， 研究计划。这一培训还将有助于设计干预措施，以减少 幼儿喘息的发生率。总体研究假设是，症状集群和 它们相关的炎症通路可预测年龄在12-59个月的学龄前儿童的临床结果， 反复喘息。具体目的是确定症状群是否预测急性加重（原发性 结果）和生活质量（次要结果），并利用下一代测序来识别 症状群的潜在途径及其轨迹。该项目解决了以下创新问题： 症状科学，并可能导致识别用于个性化干预的新生物标志物。更大 了解发病的潜在症状也将有助于解决一个关键的公共卫生问题 在美国，影响越来越多的儿童的哮喘。