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

• 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
• 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' 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

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.

项目摘要/摘要 MD MHS Jennifer Dantzer是Johns Hopkins University的过敏和免疫学医师科学家， 流行病学硕士学位。这个K23奖将为她的长期提供宝贵的培训 成为专注于以患者为中心成果（PCO）食物过敏的独立研究者的职业目标 治疗。食物过敏是一种主要的公共卫生障碍，至少影响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的避免过敏原 以及对过敏反应的识别和治疗。但是，避免食物和正在进行的负担 对可能意外暴露的不确定性可能会对儿童产生重大的社会心理影响 父母。 FDA批准了对花生过敏的首次治疗，Palforzia，Peanut口服免疫疗法 （POIT），在2020年1月，迎来了一个新的食物过敏治疗时代。而这种范式的食物转变 过敏管理是巨大的，与患者的利益/风险相关的不确定性很大， 治疗偏好和以患者为中心的结果（PCO）。我们的理解差距导致了这一差距 项目的总体目标是评估患者对花生过敏疗法的感知益处的总体目标 了解有关中断和治疗偏好的决策。 AIM 1将检查 儿童，青少年和照顾者食物过敏与生活质量的纵向变化 人口开关poit。此外，POIT的长期利益取决于正在进行的治疗 由于停止治疗会导致大多数患者失去大部分或全部保护。 AIM 2将使用本地和 国家数据以及访谈和问卷，以确定利率，潜在预测因素和原因 用于停用的poit。最后，AIM 3将使用混合方法来了解患者和父母 PA治疗的偏好。该K23奖介绍了NIAID的使命，以更好地理解和治疗 通过使用针对PCO研究的新方法和方法，过敏性疾病。研究将 在约翰·霍普金斯（Johns Hopkins）的高度支持培训环境中完成，在团队的指导下 具有相关专业知识的专家导师（医学博士Robert Wood博士 MPH，Cynthia Rand，PhD和John F.P.桥梁，博士）。职业发展计划结合了动手 在统计分析，流行病学和 PCO研究中的高级技术。该计划将确保成功过渡到独立性。这 K23 Award将提供高度相关且适用于未来R01或U的初步数据和技能 水平赠款。调查结果有可能改变与POIT相关的临床实践，而且与未来有关 食物过敏治疗，通过增强的利益风险评估，最佳实践指南和患者 决策工具。