PDF(Pubmed)
Abstract：
Individuals with neurofibromatosis, including neurofibromatosis 1 (NF1), neurofibromatosis 2 (NF2)-related schwannomatosis (SWN), and other forms of SWN, often experience disease manifestations and mental health difficulties for which psychosocial interventions may help. An anonymous online survey of adults with neurofibromatosis assessed their physical, social, and emotional well-being and preferences about psychosocial interventions to inform clinical trial design.
Neurofibromatosis clinical researchers and patient representatives from the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration developed the survey. Eligibility criteria included age ≥ 18 years, self-reported diagnosis of NF1, NF2, or SWN, and ability to read and understand English. The online survey was distributed internationally by the Neurofibromatosis Registry and other neurofibromatosis foundations from June to August 2020.
Surveys were completed by 630 adults (18-81 years of age; M = 45.5) with NF1 (78%), NF2 (14%), and SWN (8%) who were mostly White, not Hispanic/Latino, female, and from the United States. The majority (91%) reported that their neurofibromatosis symptoms had at least some impact on daily life. In the total sample, 51% endorsed a mental health diagnosis, and 27% without a diagnosis believed they had an undiagnosed mental health condition. Participants indicated that neurofibromatosis affected their emotional (44%), physical (38%), and social (35%) functioning to a high degree. Few reported ever having participated in a drug (6%) or psychosocial (7%) clinical trial, yet 68% reported they \"probably\" or \"definitely\" would want to participate in a psychosocial trial if it targeted a relevant concern. Top treatment targets were anxiety, healthier lifestyle, and daily stress. Top barriers to participating in psychosocial trials were distance to clinic, costs, and time commitment. Respondents preferred interventions delivered by clinicians via individual sessions or a combination of group and individual sessions, with limited in-person and mostly remote participation. There were no significant group differences by neurofibromatosis type in willingness to participate in psychosocial trials (p = 0.27). Regarding interest in intervention targets, adults with SWN were more likely to prefer psychosocial trials for pain support compared to those with NF1 (p < 0.001) and NF2 (p < 0.001).
This study conducted the largest survey assessing physical symptoms, mental health needs, and preferences for psychosocial trials in adults with neurofibromatosis. Results indicate a high prevalence of disease manifestations, psychosocial difficulties, and untreated mental health problems in adults with neurofibromatosis and a high degree of willingness to participate in psychosocial clinical trials. Patient preferences should be considered when designing and implementing psychosocial interventions to develop the most feasible and meaningful studies.
Neurofibromatosis clinical researchers and patient representatives from the Response Evaluation in Neurofibromatosis and Schwannomatosis International Collaboration developed the survey. Eligibility criteria included age ≥ 18 years, self-reported diagnosis of NF1, NF2, or SWN, and ability to read and understand English. The online survey was distributed internationally by the Neurofibromatosis Registry and other neurofibromatosis foundations from June to August 2020.
Surveys were completed by 630 adults (18-81 years of age; M = 45.5) with NF1 (78%), NF2 (14%), and SWN (8%) who were mostly White, not Hispanic/Latino, female, and from the United States. The majority (91%) reported that their neurofibromatosis symptoms had at least some impact on daily life. In the total sample, 51% endorsed a mental health diagnosis, and 27% without a diagnosis believed they had an undiagnosed mental health condition. Participants indicated that neurofibromatosis affected their emotional (44%), physical (38%), and social (35%) functioning to a high degree. Few reported ever having participated in a drug (6%) or psychosocial (7%) clinical trial, yet 68% reported they \"probably\" or \"definitely\" would want to participate in a psychosocial trial if it targeted a relevant concern. Top treatment targets were anxiety, healthier lifestyle, and daily stress. Top barriers to participating in psychosocial trials were distance to clinic, costs, and time commitment. Respondents preferred interventions delivered by clinicians via individual sessions or a combination of group and individual sessions, with limited in-person and mostly remote participation. There were no significant group differences by neurofibromatosis type in willingness to participate in psychosocial trials (p = 0.27). Regarding interest in intervention targets, adults with SWN were more likely to prefer psychosocial trials for pain support compared to those with NF1 (p < 0.001) and NF2 (p < 0.001).
This study conducted the largest survey assessing physical symptoms, mental health needs, and preferences for psychosocial trials in adults with neurofibromatosis. Results indicate a high prevalence of disease manifestations, psychosocial difficulties, and untreated mental health problems in adults with neurofibromatosis and a high degree of willingness to participate in psychosocial clinical trials. Patient preferences should be considered when designing and implementing psychosocial interventions to develop the most feasible and meaningful studies.
摘要：
目的：神经纤维瘤病患者，包括神经纤维瘤病1（NF1），神经纤维瘤病2（NF2）相关神经鞘瘤病（SWN），和其他形式的SWN，经常经历疾病表现和心理健康困难，心理社会干预可能会有所帮助。一项针对神经纤维瘤病患者的匿名在线调查评估了他们的身体状况，社会,以及情绪健康和对心理社会干预的偏好，为临床试验设计提供信息。
方法：神经纤维瘤病的临床研究人员和患者代表来自神经纤维瘤病和神经鞘瘤病的反应评估国际合作组织开展了这项调查。资格标准包括年龄≥18岁，NF1、NF2或SWN的自我报告诊断，以及阅读和理解英语的能力。在线调查由神经纤维瘤病注册中心和其他神经纤维瘤病基金会于2020年6月至8月在国际上分发。
结果：调查由630名成年人（18-81岁；M=45.5）完成，NF1（78％），NF2（14%），SWN(8%)主要是白人，不是西班牙裔/拉丁裔,女性,来自美国。大多数（91％）报告说，他们的神经纤维瘤病症状至少对日常生活有一定影响。在总样本中，51%的人支持心理健康诊断，没有诊断的27%的人认为他们有未诊断的精神健康状况。参与者表示，神经纤维瘤病影响他们的情绪（44％），物理（38%），和社会功能(35%)在很大程度上。很少有报道曾参与药物（6％）或社会心理（7％）临床试验，然而，68%的人报告说，如果针对相关问题，他们“可能”或“肯定”会想要参加心理社会试验。最主要的治疗目标是焦虑，更健康的生活方式,每天的压力。参与心理社会试验的最大障碍是距离诊所，成本,时间承诺。受访者更喜欢由临床医生通过个人会议或小组和个人会议的组合提供的干预措施。有限的人，大多是远程参与。参与社会心理试验的意愿按神经纤维瘤病类型没有显着差异（p=0.27）。关于对干预目标的兴趣，与NF1(p<0.001)和NF2(p<0.001)患者相比,SWN患者更有可能选择心理社会试验进行疼痛支持.
结论：这项研究进行了最大的评估身体症状的调查，心理健康需求，以及对神经纤维瘤病成年人的社会心理试验的偏好。结果表明，疾病表现的患病率很高，心理社会困难,和未经治疗的精神健康问题的成人神经纤维瘤病和参与心理社会临床试验的高度意愿。在设计和实施心理社会干预措施以开发最可行和有意义的研究时，应考虑患者的偏好。
方法：神经纤维瘤病的临床研究人员和患者代表来自神经纤维瘤病和神经鞘瘤病的反应评估国际合作组织开展了这项调查。资格标准包括年龄≥18岁，NF1、NF2或SWN的自我报告诊断，以及阅读和理解英语的能力。在线调查由神经纤维瘤病注册中心和其他神经纤维瘤病基金会于2020年6月至8月在国际上分发。
结果：调查由630名成年人（18-81岁；M=45.5）完成，NF1（78％），NF2（14%），SWN(8%)主要是白人，不是西班牙裔/拉丁裔,女性,来自美国。大多数（91％）报告说，他们的神经纤维瘤病症状至少对日常生活有一定影响。在总样本中，51%的人支持心理健康诊断，没有诊断的27%的人认为他们有未诊断的精神健康状况。参与者表示，神经纤维瘤病影响他们的情绪（44％），物理（38%），和社会功能(35%)在很大程度上。很少有报道曾参与药物（6％）或社会心理（7％）临床试验，然而，68%的人报告说，如果针对相关问题，他们“可能”或“肯定”会想要参加心理社会试验。最主要的治疗目标是焦虑，更健康的生活方式,每天的压力。参与心理社会试验的最大障碍是距离诊所，成本,时间承诺。受访者更喜欢由临床医生通过个人会议或小组和个人会议的组合提供的干预措施。有限的人，大多是远程参与。参与社会心理试验的意愿按神经纤维瘤病类型没有显着差异（p=0.27）。关于对干预目标的兴趣，与NF1(p<0.001)和NF2(p<0.001)患者相比,SWN患者更有可能选择心理社会试验进行疼痛支持.
结论：这项研究进行了最大的评估身体症状的调查，心理健康需求，以及对神经纤维瘤病成年人的社会心理试验的偏好。结果表明，疾病表现的患病率很高，心理社会困难,和未经治疗的精神健康问题的成人神经纤维瘤病和参与心理社会临床试验的高度意愿。在设计和实施心理社会干预措施以开发最可行和有意义的研究时，应考虑患者的偏好。
