Patient-Centered Outcomes of the First Approved Peanut Allergy Treatment

首个获批的花生过敏治疗以患者为中心的结果

基本信息

批准号：
10370510
负责人：
Jennifer Anne Dantzer
金额：
$ 19.87万
依托单位：
JOHNS HOPKINS UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-12-08 至 2026-11-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10370510
关键词：
Address Adolescent Affect Age Allergens Allergic Disease Allergic Reaction Allergy to peanuts American Anaphylaxis Anxiety Area Benefits and Risks Caregivers Caring Child Childhood Clinical Clinical Investigator Clinical Trials Complex Data Data Analyses Databases Decision Making Development Development Plans Disease Doctor of Philosophy Economics Emergency Situation Ensure Environment Epidemiology Equilibrium Exposure to FDA approved Food Hypersensitivity Future Goals Grant Health Benefit Heterogeneity Hypersensitivity Immunology Impairment Individual Ingestion Institutes Interview Knowledge Label Lead Life Maintenance Master&apos s Degree Mentored Patient-Oriented Research Career Development Award Mentors Mentorship Methods Mission National Institute of Allergy and Infectious Disease Outcome Outcomes Research Parents Patient-Focused Outcomes Patients Pharmaceutical Preparations Physicians Population Practice Guidelines Prevalence Prospective cohort Public Health Quality of life Questionnaires Reaction Reporting Research Research Personnel Risk Risk-Benefit Assessment Scientist Statistical Data Interpretation Stratification Surveys Techniques Teenagers Therapeutic Time Training Uncertainty Universities Wood material Work age group base career career development clinical practice design food avoidance immunotherapy trials insight low socioeconomic status novel strategies novel therapeutics oral immunotherapy preference prevent psychologic psychosocial skills targeted treatment tool translational scientist treatment center treatment choice treatment effect

项目摘要

PROJECT SUMMARY/ABSTRACT Jennifer Dantzer, MD MHS is an allergy and immunology physician-scientist at Johns Hopkins University, with a Master's Degree in Epidemiology. This K23 award will provide her valuable training towards her long-term career goal of being an independent investigator focused on patient centered outcomes (PCO) of food allergy treatments. Food allergy is a major public health disorder that affects at least 5.9 million American children and is increasing in prevalence. Historically, food allergy management has been based on strict allergen avoidance and recognition and treatment of allergic reactions. However, the burden of food avoidance and on-going uncertainty over possible accidental exposures can have a significant psychosocial impact on children and parents. The FDA approved the first treatment for peanut allergy, Palforzia, peanut oral immunotherapy (POIT), in January 2020, ushering in a new era for food allergy treatment. While this paradigm shift in food allergy management is monumental, there is significant uncertainty related to benefits/risks to the patient, treatment preferences, and patient-centered outcomes (PCO). This gap in our understanding lead to this project's overall objective to evaluate the patient perceived benefit of peanut allergy therapy and to better understand decision-making with regard to discontinuation and treatment preferences. Aim 1 will examine longitudinal change in child, adolescent, and caregiver food allergy related quality of life in a diverse, real-world population on and off POIT. In addition, the long-term benefit of POIT is dependent upon on-going treatment since stopping treatment causes the majority of patients to lose most or all protection. Aim 2 will use local and national data as well as interviews and questionnaires to determine the rates, potential predictors, and reasons for POIT discontinuation. Finally, Aim 3 will use a mixed methods approach to understand patient and parent preferences for PA treatment. This K23 award addresses the mission of NIAID to better understand and treat allergic diseases by using a novel approach and methods directed towards PCO research. The research will be completed within the highly supportive training environment at Johns Hopkins, with the guidance of a team of expert mentors with relevant expertise (Drs. Robert Wood, MD, Corinne Keet, MD PhD, Albert Wu, MD MPH, Cynthia Rand, PhD and John F.P. Bridges, PhD). The career development plan combines hands-on training, formal didactics, and outstanding mentorship in key areas of statistical analysis, epidemiology, and advanced techniques in PCO research. This plan will ensure a successful transition to independence. This K23 award will provide preliminary data and skills that are highly relevant and applicable to future R01 or U level grants. Findings have the potential to change clinical practice not only related to POIT, but also for future food allergy treatments, through enhanced benefit-risk assessment, best practice guidelines, and patient decision-making tools.

项目概要/摘要 Jennifer Dantzer，MD MHS 是约翰霍普金斯大学的过敏和免疫学医师兼科学家，流行病学硕士学位。这个 K23 奖项将为她的长期发展提供宝贵的培训职业目标是成为一名专注于以患者为中心的食物过敏结果 (PCO) 的独立研究者治疗。食物过敏是一种主要的公共卫生疾病，影响至少 590 万美国儿童和患病率正在增加。从历史上看，食物过敏管理一直基于严格避免过敏原以及过敏反应的识别和治疗。然而，避免食物的负担和持续的对可能发生的意外接触的不确定性可能会对儿童和儿童产生重大的社会心理影响父母。 FDA批准首个治疗花生过敏的药物Palforzia，花生口服免疫疗法（POIT），2020 年 1 月，开创了食物过敏治疗的新时代。虽然食品的这种范式转变过敏管理至关重要，对患者的益处/风险存在很大的不确定性，治疗偏好和以患者为中心的结果（PCO）。我们理解上的差距导致了这一点项目的总体目标是评估患者对花生过敏治疗的感知益处并更好地了解有关停药和治疗偏好的决策。目标 1 将检查在多样化的现实世界中，儿童、青少年和护理人员与食物过敏相关的生活质量的纵向变化 POIT 内外的人口。此外，POIT 的长期益处取决于持续治疗因为停止治疗会导致大多数患者失去大部分或全部保护。目标 2 将使用本地和国家数据以及访谈和问卷，以确定比率、潜在预测因素和原因 POIT 终止。最后，目标 3 将使用混合方法来了解患者和家长 PA 治疗的偏好。该 K23 奖项体现了 NIAID 的使命，即更好地理解和治疗通过使用针对 PCO 研究的新方法和方法来治疗过敏性疾病。该研究将在团队的指导下，在约翰霍普金斯大学高度支持的培训环境中完成具有相关专业知识的专家导师（Robert Wood 博士、Corinne Keet 博士、Albert Wu 博士 MPH、Cynthia Rand 博士和 John F.P.布里奇斯博士）。职业发展计划结合实践统计分析、流行病学和统计等关键领域的培训、正式教学和出色的指导 PCO 研究中的先进技术。该计划将确保成功过渡到独立。这 K23奖项将提供与未来R01或U高度相关且适用的初步数据和技能级补助金。研究结果有可能不仅改变 POIT 相关的临床实践，而且有可能改变未来的临床实践食物过敏治疗，通过加强效益风险评估、最佳实践指南和患者决策工具。