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

项目总结 开发一种个性化的方法来解释患者报告的结果测量(PROM)分数是 对于提供高质量的护理至关重要。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 针对每个患者的重要差异(MCID)，以提高PROM评估的能力 在不同的患者群体中，个体患者偏好和衡量对治疗的反应性。 具体地说，我们将评估临床相关信息(例如，基线严重性、社会人口统计 特征)会影响对PROM的解释，以及在以下情况下应如何结合这些信息 为个别患者做出临床决策。单侧声带麻痹是一种改变人生的疾病 由一条喉返神经损伤引起的情况，导致暂时性或永久性声门 无能。对健康和生活质量的影响可能包括失声、吞咽功能障碍和 心理社会苦恼。瘫痪适合于发展个性化的MCID，因为(1)存在治疗方法， 但有效性的特点很差；(2)决策是基于患者的个人偏好；以及 (3)患者的症状具有异质性，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的先河，开发了个性化的MCID，同时开发了 并验证了脊髓瘫痪体验(COPE)-一种新的PROM，旨在评估特定于 瘫痪。这项拟议的研究将开发一种个性化的MCID，将每个患者的伤害合并在一起 严重性、直言不讳的要求和社会人口学特征，可以用来改善 PROM在护理点的可解释性，并符合患者-提供者的期望。要做到这一点，我们将 进行观察性队列研究，测量治疗前和治疗后COPE评分的变化 接受急性麻痹治疗的成人(从受伤起3个月，症状最严重) 护理干预覆盖37个站点的高音量语音中心的全国协作。具体目标是 这个项目是：(1)创建个体化的最小临床重要差异(IMCID)，它解释了每个 患者的基线损伤严重程度、发声需求和社会人口学特征，(2)测试 急性麻痹患者治疗效果的异质性及其相关因素 语言治疗和/或注射增强的治疗效果，以及(3)开发一种方法来 使用iMCID将患者的偏好与预期的治疗效果联系起来，以使患者- 提供商期望。这项工作将彻底改变MCID在异种患者中的应用方式 同时使临床试验能够改善瘫痪患者的护理，并使应对措施成为 在护理点评估瘫痪患者残疾的有用的、标准化的方法。

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.