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

• 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
• 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

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 开发个体化的方法来解释患者报告的结局指标（PROM）评分， 这是提供优质护理的关键。在这个提案中，我们计划创建一个个性化的最低限度的临床 重要差异（MCID），具体针对每个患者，以提高PROM评估 个体患者偏好和测量异质患者群体对治疗的反应性。 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 特性）影响到PROM的解释，以及在下列情况下应如何纳入这些信息： 为个别患者做出临床决策。单侧声带麻痹（麻痹）是一种改变生活的 一条喉返神经损伤导致暂时或永久性声门闭合的情况 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， 社会心理困扰瘫痪适合于开发个性化的MCID，因为（1）存在治疗方法， 但有效性特征不佳;（2）决策基于患者个人偏好;以及 (3)患者的症状是异质的，受到损伤严重性和独特患者特征的影响。 我们的团队开创了统计理论，开发了个性化的MCID，同时开发了 并验证了脊髓麻痹经验（科普）-一种新的PROM，旨在评估特定于 瘫痪拟议的研究将开发一个个性化的MCID，包括每个病人的伤害 严重程度，声音需求和社会人口特征，可用于改善 PROMs在护理点的可解释性，并与患者-提供者的期望保持一致。为此，我们将 进行一项观察性队列研究，测量治疗前后科普评分的变化， 成人治疗急性瘫痪（受伤后<3个月，症状最严重时）， 在全国37个大容量语音中心的合作中进行护理干预。具体目标为 该项目是：（1）创建个体化的最小临床重要差异（iMCID）， 患者的基线损伤严重程度、声音需求和社会人口统计学特征，（2）测试 急性瘫痪患者治疗效果的异质性，以确定与 言语治疗和/或注射增强的治疗效果，以及（3）开发一种方法， 将使用iMCID的患者偏好与预期治疗效果联系起来，以使患者- 供应商的期望这项工作将彻底改变MCID在异质性患者中的应用 同时使临床试验能够改善瘫痪患者的护理，并使科普成为 有用的，标准化的方式来评估残疾瘫痪患者在护理点。