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.

项目摘要 单方面声带瘫痪（UVFP）是一种令人衰弱的状况，具有重大健康和生活质量 含义。它是由对复发性喉神经的损伤造成的，在50％的病例中是医源性的，大多数 通常来自甲状腺切除术或颈前椎间盘融合手术。这些程序的比率 在过去的几十年中，增加了几倍，与其他外科手术，创伤和其他 病因使患者的比例相应增加，患有这种疾病的风险。生活质量 与UVFP相关的语音障碍患者的后果比患有哮喘的患者更糟糕 冠状动脉综合征和抑郁症。从健康的角度来看，有60％的UVFP患者患有吞咽困难 （其中有23％的攻击性），有75％的人呼吸困难。 治疗的基本原则是概括喉括约肌以允许声带闭合 改善声音和吞咽，降低抽吸风险以及使呼吸正常化。处理算法 根据时间和干预类型的差异很大。但是，存在重大的实践差异 提倡每个吹捧他们的优势的拥护者；但是，没有标准措施比较其亲戚 可缓解与喉功能所有领域有关的症状的有效性（例如，声音，吞咽， 呼吸）。缺乏UVFP-ATRIBUTABITION的标准复合度量是障碍 量化患者残疾，跟踪症状的改善并比较治疗效果。 迄今为止，客观措施（例如声学）尚未与症状改善持续相关。 因此，可以说是评估生活质量和残疾的患者报告的结果指标（PROM）是 最相关的，应用于优化我们的治疗算法。目前没有舞会评估所有 喉功能的各个方面；相反，它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。临床适用措施中的这种缺陷限制了我们的能力 全面评估患者对旨在治疗UVFP的干预措施的反应。 该建议描述了一项职业发展计划，该计划将使我能够发展成果的专业知识 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。研究具体的目的是：a）系统地回顾有关文献 与声音，吞咽和呼吸有关的舞会，评估其结构和分析的充分性 心理测量发展； b）严格建立舞会，以评估使用UVFP侵蚀性残疾 经典测试理论原理并执行主成分分析以确定其潜在维度 结构和评分； c）通过：执行确认性因素分析和评估来验证舞会 测试可靠性，替代管理/形式可靠性，与标准相关的有效性以及构建 有效性，包括对时间和干预的纵向响应。 职业发展计划集成：a）高级课程和个性化导师指导 结果度量的指导开发方法，系统审查/荟萃分析和手术 比较有效性技术，b）参加本地/国家研讨会和会议以促进 除了进行系统的舞会发展方面的定性和定量方面的专业知识 评论/荟萃分析和外科试验，c）多学科的指导研究经验，d）高度 支持性研究环境。该环境包括手术质量和结果中心 研究，一个由AHRQ资助的循证实践中心，NIH资助的临床翻译科学 奖项，排名最高的皮博迪教育学院，公共卫生硕士课程以及最先进的 艺术比尔·威尔克森（Art Bill Wilkerson）耳鼻喉科和传播科学中心。 总体而言，该职业发展奖将推动我在比较有效性研究中的职业 在结果衡量发展方法中提供教育方法，并将产生严格的发展和 精神法法上有效的舞会，可全面评估UVFP侵蚀性残疾。重要的是，这个 奖项为无缝过渡向未来的独立资助研究提供了关键的支持，该研究使用 新创建的舞会以比较UVFP干预措施，跟踪恢复，预后和更好的裁缝 管理算法以最大程度地减少UVFP-ATRIBUTABITABIES。