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.

项目摘要开发一种个性化的方法来解释患者报告的结果指标（PROM）分数是提供优质护理至关重要。在此提案中，我们计划在临床上创建一个个性化的最小化重要的差异（MCID），该差异是针对每个患者的特定差异，以提高舞会评估的能力个体患者的偏好并衡量异质患者人群中对治疗的反应能力。具体而言，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）如何特征）影响舞会的解释以及如何在为个别患者做出临床决定。单方面声带瘫痪（瘫痪）是一种改变生活的由一种复发性喉神经损伤引起的状况，并导致暂时或永久性的震颤无能。对健康和生活质量的影响可能包括声音丧失，吞咽功能障碍以及社会心理困扰。瘫痪适合开发个性化的MCID，因为（1）存在治疗，但是有效性的特征很差。（2）决策是基于个别患者的偏好；和（3）患者症状是异质的，受损伤严重程度和独特的患者特征的影响。我们的团队已经开创了统计理论来开发个性化的MCID，并简单地发展并验证了脐带麻痹经验（COPE） - 一个新颖的舞会，旨在评估特定于麻痹。拟议的研究将开发一个个性化的MCID，该MCID纳入每个患者的受伤严重性，声音需求和社会人口统计学特征，可用于改善舞会在护理点上的可解释性和对准患者预期的期望。为此，我们将进行观察队列研究，以测量治疗前和治疗后的应对评分变化接受急性瘫痪治疗的成年人（受伤<3个月，症状最严重时）在37个地点的大批配音中心的国家合作中进行的护理干预措施。具体目标该项目是：（1）创建个性化的最小临床重要差异（IMCID）患者的基线伤害严重程度，人声需求和社会人口统计学特征，（2）测试急性麻痹患者的治疗效应的异质性，以鉴定与语音疗法和/或注射增强的治疗效果，以及（3）开发一种方法使用IMCID具有预期治疗效果的情况将患者的偏好与情境化，以使患者对齐提供者期望。这项工作将彻底改变MCID如何应用于异质患者种群在使临床试验的同时改善了麻痹患者的护理，并使Cope成为评估治疗点麻痹患者残疾的有用，标准化的方法。