Patient-Centered Outcomes of the First Approved Peanut Allergy Treatment

项目摘要/摘要 Jennifer Dantzer，医学硕士，约翰霍普金斯大学过敏和免疫内科科学家， 流行病学硕士学位。这项K23奖项将为她的长期职业生涯提供宝贵的培训 职业目标是成为一名关注以患者为中心的食物过敏结果(PCO)的独立调查者 治疗。食物过敏是一种主要的公共卫生疾病，影响到至少590万美国儿童和 患病率正在增加。从历史上看，食物过敏管理建立在严格避免过敏原的基础上 以及对过敏反应的识别和治疗。然而，避免食物和持续的负担 对可能的意外接触的不确定性会对儿童产生重大的心理社会影响 父母。FDA批准了第一种治疗花生过敏的方法，Palforzia，花生口服免疫疗法 (POIT)，2020年1月，开启了食物过敏治疗的新纪元。虽然食品领域的这一范式转变 过敏管理是非常重要的，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)。我们在理解上的这种差距导致了这一点 该项目的总体目标是评估花生过敏疗法对患者的感知益处，并更好地 了解有关停药和治疗偏好的决策。Aim 1将检查 多样化现实世界中儿童、青少年和照顾者食物过敏相关生活质量的纵向变化 人口断断续续。此外，POIT的长期益处取决于持续的治疗。 因为停止治疗会导致大多数患者失去大部分或全部保护。AIM 2将使用本地和 国家数据以及访谈和问卷调查，以确定比率、潜在预测因素和原因 因为毒药停药。最后，目标3将使用混合方法来理解患者和父母 对PA治疗的偏好。这个K23奖项表明NIAID的使命是更好地理解和治疗 利用一种新的途径和方法进行过敏性疾病的研究。这项研究将 在约翰霍普金斯大学高度支持的培训环境中完成，在团队的指导下完成 具有相关专业知识的专家导师(Robert Wood博士，医学博士，Corinne Keet博士，医学博士，Albert Wu，医学博士 公共卫生硕士、辛西娅·兰德博士和约翰·F·P·布里奇斯博士)职业发展计划结合了亲身实践 在统计分析、流行病学和其他关键领域的培训、正规教学和杰出的指导 PCO研究中的先进技术。这项计划将确保向独立的成功过渡。这 K23奖项将提供与未来的R01或U高度相关和适用的初步数据和技能 级别助学金。这些发现有可能改变临床实践，不仅与POIT有关，而且对未来也是如此 食物过敏治疗，通过增强的收益-风险评估、最佳实践指南和患者 决策工具。

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.