Home Monitoring of Metabolic Disorders

项目摘要 本提案是对NOT-HD-22-009特别关注通知（NOSI）的直接响应：创新 技术，以改善评估，干预，和个人与智力和 发育障碍[IDD]（R43/R44）。患有某些代谢紊乱的所有年龄段的人都有 当某种代谢物积累时，代谢危机，而这种代谢物可以在临床上测量。 在实验室中，目前没有基于家庭的设备来监测导致代谢危机的代谢物。 我们团队的长期目标是降低与代谢相关的代谢危机的发病率和死亡率。 一定的代谢积累。通过以前和正在进行的国家科学基金会和国家 卫生研究院小企业创新研究基金，我们已经开发了一种家用设备， 准确地测量这种代谢物从一滴血，并正在努力争取FDA的批准。整体 本提案的目的是研究每日在家监测目标代谢物的影响 使用我们开发的设备。这将是第一次在家里测量代谢物， 在家中的目标人群中测量。 我们的中心假设是，当参与者在家中，空腹，早晨测量代谢物水平时， 代谢物结果升高与患者进一步服用 采取行动联系其医疗团队，以进一步监测或采取措施降低代谢物水平。 我们的具体目标是：1）在10名患有疾病的参与者中进行临床试验， 代谢物水平升高，2）分析收集的大量数据，3）准备 结果为了完成我们的方法，我们将从俄勒冈州的现有患者人群中招募患者 健康与科学大学我们将从受试者的病历、观察结果中收集大量数据， 以及装置每日代谢物测量。 这项研究具有创新性，因为它首次提供了一种简单的家庭设备 用于目标代谢物的测量。在这个项目结束时，我们希望了解更多的 家庭代谢物监测对患者护理的影响。

PROJECT SUMMARY This proposal is submitted in direct response to NOT-HD-22-009 Notice of Special Interest (NOSI): Innovative Technologies to Improve Assessments, Interventions, and Outcomes for Individuals with Intellectual and Developmental Disabilities [IDD] (R43/R44). Persons of all ages with certain metabolic disorders are at-risk for metabolic crises when a certain metabolite accumulates, while this metabolite can be measured in a clinical laboratory, currently there is no home-based device to monitor the metabolie that causes the metabolic crises. Our team’s long-term goal is to reduce the morbidity and mortality of metabolic crises associated with certain metabolic accumulation. Through previous and ongoing National Science Foundation and National Institutes of Health Small Business Innovation Research funding, we have developed an at-home device to accurately measure this metabolite from a drop of blood and are working towards FDA clearance. The overall objective of this proposal is to investigate the impact of daily home-based monitoring of the target metabolite using the device we developed. This will be the first time that at-home measurement of the metabolite will be measured in the target population at home. Our central hypothesis is that when participants measure at-home, fasting, morning metabolite levels there will be an association between getting an elevated metabolite result and then the patient taking further action to contacting their medical team to further monitor or take steps to reduce the metabolite level. Our specific aims are 1) to perform a clinical trial in 10 participants with disorders resulting in risk of elevated metabolite levels, 2) to analyze the extensive data collected, and 3) to prepare a manuscript of the results. To complete our approach we will recruit patients from the available patient population at Oregon Health & Science University. We will collect extensive data from the participant medical record, observations, and the device daily metabolite measurements. This research is innovative because, for the first time, it provides a straightforward, home-based device for measurement of the target metabolite. At the conclusion of this project, we expect to understand more the impact of home-based metabolite monitoring on patient care.