Developing an Outcome Measuring Unilateral Vocal Fold Paralysis Disability

描述（由申请人提供）：单侧声带麻痹（UVFP）是一种对健康和生活质量有重大影响的衰弱性疾病。它是由喉返神经损伤引起的，在50%的病例中是医源性的，最常见的是甲状腺切除术或颈椎前路椎间盘融合术。在过去的几十年里，这些手术的发生率增加了数倍，当与其他手术、创伤和其他病因相结合时，相应地增加了患这种疾病的患者比例。UVFP相关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。从健康的角度来看，60%的UVFP患者患有吞咽困难（其中23%患有误吸），75%患有呼吸困难。治疗的基本原则是重新概括喉括约肌，以允许声带关闭，从而改善声音和吞咽，减少误吸风险，并使呼吸正常化。治疗算法根据干预的时间和类型而变化很大。然而，每一种方法的倡导者都在宣传它们的优点，存在着显著的实践差异;然而，没有标准的测量方法来比较它们在缓解与喉功能的所有领域（例如声音，吞咽和呼吸）相关的症状方面的相对有效性。缺乏UVFP所致残疾的标准综合指标是量化患者残疾、跟踪症状改善和比较治疗有效性的障碍。迄今为止，客观测量（例如声学）与症状改善并不一致。因此，评估生活质量和残疾的患者报告结局指标（PROM）可以说是最相关的，应用于优化我们的治疗算法。目前没有PROM评估喉功能的所有方面;相反，他们的范围仅限于发声或吞咽，没有评估喉相关的呼吸功能障碍。临床适用措施中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。该提案描述了一个职业发展计划，将使我能够发展的专业知识，结果衡量发展方法，系统评价和荟萃分析，手术比较有效性研究技术。本研究的具体目标是：a）系统地回顾与声音、吞咽和呼吸相关的PROM文献，评估其结构并分析心理测量发展的充分性; B）使用经典测试理论原则严格开发PROM以评估UVFP归因的残疾，并进行主成分分析以确定其潜在维度结构和评分;以及c）通过以下方式验证PROM：进行验证性因素分析，并评估重测信度，交替管理/形式的信度，同时标准相关效度，结构效度，包括纵向反应时间和干预。职业发展计划包括：a）在结果测量开发方法、系统性综述/荟萃分析和手术比较有效性技术方面的高级课程作业和个性化导师指导教学，B）参加地方/国家研讨会和会议，以提高除了进行系统性综述/荟萃分析和手术试验之外，在开发PROM方面的定性和定量方面的专业知识，c）多学科指导的研究经验，以及d）高度支持的研究环境。该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，NIH资助的临床翻译科学奖，排名第一的皮博迪教育学院，公共卫生硕士课程，以及最先进的Bill Wilkerson耳鼻喉科和传播科学中心。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来推进我在比较有效性研究方面的职业生涯，并将产生一个严格开发和心理测量有效的PROM，全面评估UVFP归因的残疾。重要的是，该奖项提供了关键的支持，无缝过渡到未来的独立资助的研究，使用新创建的PROM比较UVFP干预，跟踪恢复，统计，并更好地定制管理算法，以尽量减少UVFP归因于残疾。

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.