CoPE II: Individualizing patient-reported outcomes in patient care for vocal fold paralysis in the clinic and in research

CoPE II：在临床和研究中个性化患者报告的声带麻痹患者护理结果

基本信息

批准号：
10776074
负责人：
David O. Francis
金额：
$ 45.62万
依托单位：
UNIVERSITY OF WISCONSIN-MADISON
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-09-18 至 2028-08-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10776074
关键词：
Acute Address Adult Affect Aftercare Aspiration Pneumonia Biometry Breathing Characteristics Climacteric Clinic Clinical Clinical Trials Communication Communication impairment Decision Making Deglutition Distress Eating Effectiveness Employment Etiology Functional disorder Goals Health Health Services Research Healthcare Heterogeneity Impairment Injections Injury Intervention Measures Mental Health Methods Occupational Outcome Outcome Measure Paralysed Patient Care Patient Outcomes Assessments Patient Preferences Patients Persons Provider Quality of Care Quality of life Recurrent Laryngeal Nerve Reporting Research Risk Severities Site Speech Therapy Standardization Subgroup Surveys Symptoms Testing Thyroidectomy Treatment Effectiveness Universities Use Effectiveness Voice Wisconsin Work clinical decision-making clinically relevant design disability expectation experience forging functional disability improved individual patient innovation machine learning method novel observational cohort study patient population patient response personalized approach physical conditioning point of care psychosocial sociodemographics standard of care statistical and machine learning supervised learning teacher theories tool treatment effect treatment response vocal cord voice therapy

项目摘要

PROJECT SUMMARY Developing an individualized approach to interpreting patient-reported outcome measures (PROM) scores is essential to providing quality care. In this proposal, we plan to create an individualized minimally clinically important difference (MCID) that is specific to each patient in order to improve the ability of PROMs to assess individual patient preferences and measure responsiveness to treatment in heterogeneous patient populations. Specifically, we will evaluate how clinically relevant information (e.g., baseline severity, sociodemographic characteristics) affects the interpretation of PROMs and how this information should be incorporated when making clinical decisions for individual patients. Unilateral vocal fold paralysis (paralysis) is a life-changing condition caused by injury to one recurrent laryngeal nerve and results in temporary or permanent glottal incompetence. Effects on health and quality of life can include loss of voice, swallowing dysfunction, and psychosocial distress. Paralysis is suited for developing the individualized MCID because (1) treatments exist, but effectiveness is poorly characterized; (2) decision-making is based on individual patient preferences; and (3) patient symptoms are heterogeneous, affected by injury severity and unique patient characteristics. Our team has pioneered statistical theory to develop the individualized MCID and simultaneously developed and validated the Cord Paralysis Experience (CoPE) – a novel PROM designed to assess disability specific to paralysis. The proposed study will develop an individualized MCID that incorporates each patient's injury severity, vocal demands, and sociodemographic characteristics, which can be used to improve the interpretability of PROMs at the point-of-care and align patient-provider expectations. To do this, we will perform an observational cohort study that measures pre- and post-treatment CoPE score changes among adults treated for acute paralysis (<3 months from injury, when symptoms are most severe) with standard-of- care interventions across a 37-site national collaborative of high-volume voice centers. The specific aims for this project are: (1) create individualized minimal clinically important differences (iMCID) that account for each patient's baseline injury severity, vocal demands, and sociodemographic characteristics, (2) test the heterogeneity of treatment effect among patients with acute paralysis to identify factors associated with treatment effectiveness of speech therapy and/or injection augmentation, and (3) develop a method to contextualize patient preferences using the iMCID with expected treatment effects in order to align patient- provider expectations. This work will revolutionize how the MCID is applied in heterogeneous patient populations while enabling clinical trials to improve the care of patients with paralysis, and making CoPE a useful, standardized way to assess disability in patients with paralysis at the point-of-care.

项目概要开发一种个体化方法来解释患者报告的结果测量 (PROM) 分数是在本提案中，我们计划创建一个个性化的最低限度的临床护理。重要差异（MCID）针对每个患者，以提高 PROM 的评估能力个体患者偏好并测量异质患者群体对治疗的反应。具体来说，我们将评估临床相关信息（例如，基线严重程度、社会人口统计学特征）影响 PROM 的解释以及在何时应如何合并此信息为个别患者做出临床决定是改变生活的事情。由一根喉返神经损伤引起并导致暂时或永久性声门失灵的病症对健康和生活质量的影响包括失声、吞咽功能障碍和社会心理困扰适合发展个性化 MCID，因为 (1) 存在治疗方法，但有效性的描述很差；(2) 决策是基于患者的个人偏好； (3) 患者症状具有异质性，受损伤严重程度和患者独特特征的影响。我们的团队开创了统计理论来开发个性化MCID，并同步开发并验证了脊髓麻痹体验 (CoPE)——一种新颖的 PROM，旨在评估特定的残疾拟议的研究将开发一种个性化的 MCID，其中包含每个患者的损伤情况。严重程度、声音需求和社会人口特征，这些可用于改善为了做到这一点，我们将在护理点提高 PROM 的可解释性并调整患者提供者的期望。进行一项观察性队列研究，测量治疗前和治疗后 CoPE 评分的变化成人急性瘫痪（受伤后 3 个月以内，症状最严重时），符合以下标准：跨 37 个国家级高容量语音中心合作的护理干预措施的具体目标。该项目是：（1）创建个体最小化临床重要差异（iMCID）来解释每个患者的基线损伤严重程度、声音需求和社会人口学特征，(2) 测试急性瘫痪患者治疗效果的异质性，以确定与治疗相关的因素言语治疗和/或注射增强的治疗效果，以及（3）开发一种方法使用 iMCID 将患者偏好与预期治疗效果结合起来，以便根据患者的需求进行调整这项工作将彻底改变 MCID 在异质患者中的应用方式。人口，同时使临床试验能够改善瘫痪患者的护理，并使 CoPE 成为在护理点评估瘫痪患者残疾的有用、标准化的方法。