Developing an Outcome Measuring Unilateral Vocal Fold Paralysis Disability

开发测量单侧声带麻痹障碍的结果

• 批准号: 8618574
• 负责人: David O. Francis
• 金额: $ 19.8万
• 依托单位: VANDERBILT UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-12-01 至 2018-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8618574
• 关键词: Acoustics; Advocate; Algorithms; Anterior; Area; Asthma; Award; Breathing; Cervical; Climacteric; Clinical; Clinical Trials; Communication; Coughing; Deglutition; Deglutition Disorders; Development; Development Plans; Dimensions; Disease; Education; Effectiveness; Environment; Etiology; Evidence based practice; Exercise Tolerance; Factor Analysis; Functional disorder; Funding; Future; Goals; Health; Hoarseness; Incidence; Injection of therapeutic agent; Injury; Instruction; Intervention; K-Series Research Career Programs; Laryngeal Diseases; Laryngeal Nerves; Larynx; Malignant - descriptor; Malignant neoplasm of thyroid; Measurement; Measures; Mental Depression; Mentors; Meta-Analysis; Methodology; Modality; Observational Study; Operative Surgical Procedures; Otolaryngology; Outcome; Outcome Assessment; Outcome Measure; Outcomes Research; Paralysed; Patient Outcomes Assessments; Patient-Focused Outcomes; Patients; Phonation; Physicians; Principal Component Analysis; Procedures; Process; Psychometrics; Public Health; Quality of life; Randomized Controlled Trials; Recovery; Recurrent Laryngeal Nerve; Relative (related person); Research; Research Technics; Respiration; Review Literature; Risk; Role; Schools; Science; Shortness of Breath; Speech Therapy; Sphincter; Structure; Surgeon; Symptoms; Techniques; Testing; Thyroidectomy; Time; Translations; Treatment Effectiveness; Treatment outcome; United States Agency for Healthcare Research and Quality; United States National Institutes of Health; Validation; Validity and Reliability; Variant; Voice; Voice Disturbances; acute coronary syndrome; base; career; career development; clinical application; comparative effectiveness; comparative trial; compare effectiveness; disability; effectiveness research; experience; improved; innovation; multidisciplinary; nerve supply; neurological recovery; novel; patient population; preference; programs; public health relevance; respiratory; response; standard measure; symposium; symptomatic improvement; systematic review; theories; tool; trial comparing; vocal cord

PROJECT SUMMARY Unilateral vocal fold paralysis (UVFP) is a debilitating condition with major health and quality of life implications. It is caused by injury to the recurrent 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, 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.

项目概要 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 影响。它是由喉返神经损伤引起的，50% 的病例是医源性的，大多数是医源性的。 通常来自甲状腺切除术或前路颈椎间盘融合手术。这些程序的费率 在过去的几十年里增加了几倍，当与其他手术、创伤和其他治疗相结合时 病因学使处于这种疾病风险的患者比例相应增加。生活质量 UVFP 相关的声音障碍患者的后果比哮喘患者更严重，急性 冠状动脉综合征和抑郁症。从健康角度来看，60%的UVFP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演喉部括约肌，从而使声带闭合 改善声音和吞咽，降低误吸风险，并使呼吸正常化。治疗算法 根据干预的时间和类型，差异很大。然而，实践中存在显着差异 双方的拥护者都宣扬自己的优势；然而，不存在标准措施来比较它们的相对 有效缓解与喉部功能所有领域相关的症状（例如发声、吞咽、 呼吸）。缺乏 UVFP 归因残疾的标准综合衡量标准是 量化患者残疾、跟踪症状改善并比较治疗效果。 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果测量（PROM）可以说是 最相关，应该用于优化我们的治疗算法。目前没有 PROM 评估所有 喉功能方面；相反，它们的范围仅限于声音或吞咽，并且没有评估喉部 相关的呼吸功能障碍。临床适用措施的此类缺陷限制了我们的能力 全面评估患者对治疗 UVFP 的干预措施的反应。 该提案描述了一个职业发展计划，该计划将使我能够发展成果方面的专业知识 测量开发方法、系统评价和荟萃分析以及手术比较 有效性研究技术。研究的具体目标是： a) 系统地回顾相关文献 PROM 与声音、吞咽和呼吸相关，评估其结构并分析其充分性 心理测量发展； b) 严格开发 PROM 来评估 UVFP 归因的残疾，使用 经典测试理论原理并进行主成分分析以确定其潜在维度 结构和评分； c) 通过以下方式验证 PROM： 进行验证性因素分析和评估 重测信度、替代管理/表格信度、并发标准相关效度以及构建 有效性包括对时间和干预的纵向反应。 职业发展计划包括：a) 高级课程和个性化导师指导 结果测量开发方法、系统评价/荟萃分析和外科手术方面的指导 比较有效性技术，b) 参加地方/国家研讨会和会议以推进 除了进行系统的研究之外，还具备 PROM 开发中定性和定量方面的专业知识 评论/荟萃分析和手术试验，c) 多学科指导的研究经验，以及 d) 高度的 支持性的研究环境。该环境包括手术质量和结果中心 研究、AHRQ 资助的循证实践中心、NIH 资助的临床转化科学 奖、一流的皮博迪教育学院、公共卫生硕士项目以及最先进的- 艺术比尔威尔克森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以下方式推动我在比较有效性研究方面的职业生涯： 提供成果衡量开发方法方面的教育，并将产生严格开发和 心理测量上有效的 PROM，可全面评估 UVFP 造成的残疾。重要的是，这 该奖项为无缝过渡到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 用于比较 UVFP 干预措施、跟踪恢复、预测和更好的定制 管理算法，以最大限度地减少 UVFP 造成的残疾。