背景:神经纤维瘤病2型相关神经鞘瘤病是一种以双侧前庭神经鞘瘤发展为特征的遗传性疾病,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。虽然已知耳鸣对一般人群个体的生活质量有显著的负面影响,对2型神经纤维瘤病患者的影响尚不清楚.关于选择合适的患者报告结果指标进行评估的共识可以促进对2型神经纤维瘤病患者耳鸣的进一步研究。这项工作的目的是通过神经纤维瘤病和神经鞘瘤病国际合作组织的反应评估达成共识建议,用于评估神经纤维瘤病2型临床试验中耳鸣领域的生活质量。
方法:神经纤维瘤病和神经鞘瘤病患者报告结果的反应评估通讯亚组使用先前发表的神经纤维瘤病和神经鞘瘤病评级程序中的反应评估,系统地评估了2型神经纤维瘤病患者耳鸣领域生活质量的患者报告结果指标。在19个确定的患者报告的结果指标中,排除了3项措施,因为它们未被验证为结果测量或不能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的结局指标进行了评分,并对其参与者特征进行了比较。项目内容,心理测量属性,以及用于临床试验的可行性。
结果:由于项目内容领域的优势,耳鸣功能指数被确定为评估2型神经纤维瘤病人群耳鸣的最高评价指标,心理测量属性,可行性,和可用的分数。
结论:神经纤维瘤病和神经鞘瘤病的反应评估目前推荐耳鸣功能指数在神经纤维瘤病2型临床试验中评估耳鸣。
Neurofibromatosis 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.
The 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.
The 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.
Response Evaluation in Neurofibromatosis and Schwannomatosis currently recommends the Tinnitus Functional Index for the assessment of tinnitus in neurofibromatosis type 2 clinical trials.