Patient-Centered Outcomes of the First Approved Peanut Allergy Treatment

项目总结/摘要 Jennifer Dantzer，医学博士，MHS是约翰霍普金斯大学的过敏和免疫学医生兼科学家， 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独立的研究者，专注于以患者为中心的食物过敏结局（PCO） 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， 越来越普遍从历史上看，食物过敏管理一直是基于严格的过敏原避免 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 对可能的意外接触的不确定性可能对儿童产生重大的心理社会影响， 父母FDA批准了第一个治疗花生过敏的药物，Palforzia，花生口服免疫疗法 （POIT）于2020年1月推出，开创了食物过敏治疗的新时代。虽然食物的这种范式转变 过敏管理是巨大的，存在与对患者的益处/风险相关的显著不确定性， 治疗偏好和以患者为中心的结局（PCO）。我们理解上的差距导致了 项目的总体目标是评估患者对花生过敏治疗的感知益处， 了解关于停药和治疗偏好的决策。目标1将检查 在一个多样化的现实世界中，儿童、青少年和照顾者食物过敏相关生活质量的纵向变化 人口上和下POIT。此外，POIT的长期获益取决于持续治疗 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调查，以确定比率，潜在的预测因素和原因 POIT停药。最后，目标3将使用混合方法来了解患者和父母 对PA治疗的偏好。这个K23奖项解决了NIAID的使命，以更好地了解和治疗 通过使用针对PCO研究的新途径和方法来治疗过敏性疾病。这项研究将 在约翰霍普金斯高度支持的培训环境中完成，并在团队的指导下 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 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实践 培训，正规教学，并在统计分析，流行病学， PCO研究的先进技术。该计划将确保向独立的成功过渡。这 K23奖将提供与未来R 01或U高度相关且适用的初步数据和技能 级赠款。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，通过加强效益-风险评估，最佳实践指南， 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.