Developing an Outcome Measuring Unilateral Vocal Fold Paralysis Disability

描述（由申请人提供）：单侧声带麻痹（UVFP）是一种严重影响健康和生活质量的衰弱性疾病。它是由喉返神经损伤引起的，50%的病例是医源性的，最常见的是甲状腺切除术或前颈椎间盘融合手术。在过去的几十年里，这些手术的比率增加了好几倍，当与其他手术、创伤和其他病因相结合时，相应的，增加了患者患这种疾病的风险比例。与uvfp相关的声音障碍患者的生活质量后果比哮喘、急性冠状动脉综合征和抑郁症患者更差。从健康角度看，60%的紫外线照射患者患有吞咽困难（其中23%有误吸），75%有呼吸困难。治疗的基本原则是重述喉部括约肌，使声带闭合，从而改善声音和吞咽，降低误吸风险，使呼吸正常化。治疗算法因时间和干预类型的不同而有很大差异。然而，存在着显著的实践差异，每个人都鼓吹自己的优势；然而，没有标准的测量方法来比较它们在缓解与喉功能所有领域（如声音、吞咽和呼吸）相关的症状方面的相对有效性。缺乏uvfp导致的残疾的标准复合测量是量化患者残疾、跟踪症状改善和比较治疗效果的障碍。迄今为止，客观测量（如声学）并不总是与症状改善相关。因此，评估生活质量和残疾的患者报告结果测量（PROM）可以说是最相关的，应该用于优化我们的治疗算法。目前还没有PROM评估喉功能的所有方面；相反，他们的范围仅限于声音或吞咽，没有评估喉相关的呼吸功能障碍。临床适用措施的这些缺陷限制了我们全面评估患者对旨在治疗UVFP的干预措施的反应的能力。这份提案描述了一个职业发展计划，使我能够在结果测量开发方法、系统回顾和荟萃分析以及外科比较有效性研究技术方面发展专业知识。本研究的具体目的是：a)系统回顾语音、吞咽和呼吸相关的PROMs文献，评价其结构，分析心理测量发展的充分性；b)采用经典测试理论原理，严格开发一种PROM来评估uvfp导致的残疾，并进行主成分分析以确定其潜在维度结构和评分；c)通过验证性因子分析，评估重测信度、替代给药/表格信度、并发效标相关效度，以及包括对时间和干预的纵向反应性在内的构念效度来验证PROM。职业发展规划包括：a)在结果测量开发方法、系统回顾/荟萃分析和手术比较有效性技术方面进行高级课程学习和个性化导师指导的指导；b)参加地方/国家研讨会和会议，除了进行系统回顾/荟萃分析和手术试验外，还可以提高prom开发中定性和定量方面的专业知识；c)多学科指导的研究经验；d)高度支持的研究环境。这一环境包括手术质量和结果研究中心、ahrq资助的循证实践中心、美国国立卫生研究院资助的临床翻译科学奖、一流的皮博迪教育学院、公共卫生硕士项目和最先进的比尔·威尔克森耳鼻喉科和通信科学中心。总的来说，这个职业发展奖将通过提供结果测量开发方法的教育，促进我在比较有效性研究方面的职业发展，并将产生一个严格开发的、心理计量学上有效的PROM，全面评估uvfp导致的残疾。重要的是，该奖项为未来独立资助的研究提供了关键支持，这些研究使用新创建的PROM来比较UVFP干预，跟踪恢复，预测和更好地定制管理算法，以最大限度地减少UVFP导致的残疾。

DESCRIPTION (provided by applicant): Unilateral vocal fold paralysis (UVFP) is a debilitating condition with major health and quality of life implications. It is caused by injury to the recurret laryngeal nerve and is iatrogenic in 50% of cases, most commonly from thyroidectomy or anterior cervical disc fusion surgeries. Rates of these procedures have increased several fold in the last few decades, which when combined with other surgical, traumatic, and other etiologies puts a correspondingly increased proportion of patients at risk for this condition. The quality of life consequences for patients with UVFP-associated voice disturbances are worse than those with asthma, acute coronary syndrome and depression. From a health perspective, 60% of UVFP patients suffer from dysphagia (23% among these have aspiration), and 75% have breathing difficulties. The basic tenet of treatment is to recapitulate the laryngeal sphincter to permit vocal fold closure thereby improving voice and swallowing, reducing aspiration risk, and normalizing respiration. Treatment algorithms vary widely based on timing and type of intervention. However, significant practice variation exists with advocates of each touting their advantages; however, no standard measure exists to compare their relative effectiveness at relieving symptoms related to all domains of laryngeal function (e.g. voice, swallowing, and breathing). Lack of a standard composite measure of UVFP-attributable disability is a barrier to quantifying patient disability, tracking symptom improvement, and comparing treatment effectiveness. To date, objective measures (e.g. acoustics) have not consistently correlated with symptomatic improvement. Therefore, patient reported outcome measures (PROM) assessing quality of life and disability are arguably most relevant and should be used to optimize our treatment algorithms. No PROM currently evaluates all aspects of laryngeal function; instead their scope is limited to voice or swallowing, and none assess laryngeal- associated respiratory dysfunction. Such deficiencies in clinically applicable measures have limited our ability to comprehensively assess patient response to interventions aimed at treating UVFP. This proposal describes a career development plan that will enable me to develop expertise in outcome measure development methodology, systematic reviews and meta-analysis, and surgical comparative effectiveness research techniques. The research specific aims are: a) to systematically review the literature on PROMs related to voice, swallowing, and breathing, evaluating their construct and analyzing adequacy of psychometric development; b) to rigorously develop a PROM to assess UVFP-attributable disability using classical test theory principles and to perform principal component analysis to determine its latent dimension structure and scoring; and c) validate the PROM by: performing confirmatory factor analysis and assessing test-retest reliability, alternate administration/form reliability, concurrent criterion-related validity, and construct validity including longitudinal responsiveness to time and intervention. The career development plan integrates: a) advanced coursework and individualized mentor-directed instruction in outcome measure development methodology, systematic review/meta-analysis, and surgical comparative effectiveness techniques, b) participation in local/national seminars and conferences to advance expertise in qualitative and quantitative aspects in development of PROMs in addition to conduct of systematic reviews/meta-analysis and surgical trials, c) a multidisciplinary mentored research experience, and d) a highly supportive research environment. This environment includes the Center for Surgical Quality and Outcomes Research, an AHRQ-funded Evidence-based Practice Center, a NIH-funded Clinical Translation Science Award, a top-ranked Peabody School of Education, a Masters in Public Health program, and the state-of-the- art Bill Wilkerson Center for Otolaryngology and Communication Sciences. Overall, this career development award will advance my career in comparative effectiveness research by providing education in outcome measure development methodology and will yield a rigorously developed and psychometrically valid PROM that comprehensively assesses UVFP-attributable disability. Importantly, this award provides critical support to seamlessly transition toward future independently funded studies that use the newly created PROM to compare UVFP interventions, track recovery, prognosticate, and better tailor management algorithms to minimize UVFP-attributable disability.