BackgroundNeurofibromatosis type 2–related schwannomatosis is a genetic disease characterized by the development of bilateral vestibular schwannomas, ependymomas, meningiomas, and cataracts. Mild to profound hearing loss and tinnitus are common symptoms reported by individuals with neurofibromatosis type 2. While tinnitus is known to have a significant and negative impact on the quality of life of individuals from the general population, the impact on individuals with neurofibromatosis type 2 is unknown. Consensus regarding the selection of suitable patient-reported outcome measures for assessment could advance further research into tinnitus in neurofibromatosis type 2 patients. The purpose of this work is to achieve a consensus recommendation by the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration for patient-reported outcome measures used to evaluate quality of life in the domain of tinnitus for neurofibromatosis type 2 clinical trials.MethodsThe Response Evaluation in Neurofibromatosis and Schwannomatosis Patient-Reported Outcomes Communication Subgroup systematically evaluated patient-reported outcome measures of quality of life in the domain of tinnitus for individuals with neurofibromatosis type 2 using previously published Response Evaluation in Neurofibromatosis and Schwannomatosis rating procedures. Of the 19 identified patient-reported outcome measures, 3 measures were excluded because they were not validated as an outcome measure or could not have been used as a single outcome measure for a clinical trial. Sixteen published patient-reported outcome measures for the domain of tinnitus were scored and compared on their participant characteristics, item content, psychometric properties, and feasibility for use in clinical trials.ResultsThe Tinnitus Functional Index was identified as the most highly rated measure for the assessment of tinnitus in populations with neurofibromatosis type 2, due to strengths in the areas of item content, psychometric properties, feasibility, and available scores.DiscussionResponse Evaluation in Neurofibromatosis and Schwannomatosis currently recommends the Tinnitus Functional Index for the assessment of tinnitus in neurofibromatosis type 2 clinical trials.

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患者报告的 2 型神经纤维瘤病相关神经鞘瘤病患者的耳鸣测量：临床试验建议

背景神经纤维瘤病2型相关神经鞘瘤病是一种遗传性疾病，其特征是出现双侧前庭神经鞘瘤、室管膜瘤、脑膜瘤和白内障。轻度至重度听力损失和耳鸣是 2 型神经纤维瘤病患者报告的常见症状。虽然已知耳鸣对普通人群的生活质量有显着的负面影响，但对 2 型神经纤维瘤病患者的影响是未知。关于选择合适的患者报告结果评估指标的共识可以推动对 2 型神经纤维瘤病患者耳鸣的进一步研究。这项工作的目的是获得神经纤维瘤病和神经鞘瘤病反应评估国际合作组织对用于评估神经纤维瘤病 2 型临床试验耳鸣领域生活质量的患者报告结果测量的共识建议。方法神经纤维瘤病反应评估神经纤维瘤病患者报告的结果沟通小组使用先前发表的神经纤维瘤病和神经鞘瘤病评级程序的反应评估，系统地评估了患者报告的 2 型神经纤维瘤病患者耳鸣领域生活质量的结果测量。在 19 项已确定的患者报告的结果测量中，有 3 项被排除，因为它们未作为结果测量进行验证或无法用作临床试验的单一结果测量。对 16 个已发表的患者报告的耳鸣领域结果测量进行了评分，并根据其参与者特征、项目内容、心理测量特性以及在临床试验中使用的可行性进行了比较。结果耳鸣功能指数被确定为评价最高的评估测量由于项目内容、心理测量特性、可行性和可用分数等方面的优势，神经纤维瘤病和神经鞘瘤病的反应评估目前推荐使用耳鸣功能指数来评估 2 型神经纤维瘤病临床中的耳鸣试验。