Integrated Brain Health Clinical and Research Program, Psychiatry Department, Massachusetts General Hospital, One Bowdoin, Square, 1st floor Boston, 02114 Boston, MA, United States of America.
Department of Psychology, Fordham University, Dealy 336, 441 East Fordham Rd, Bronx, NY 10458, United States of America.
Contemp Clin Trials. 2020 Aug;95:106078. doi: 10.1016/j.cct.2020.106078. Epub 2020 Jul 4.
Neurofibromatoses (NF) are a group of genetically distinct disorders of the nervous system unified by the predisposition to nerve sheath tumors. Although adolescents with NF types 1 and 2 (NF1 and NF2) report poor quality of life and high psychosocial burden, there are no evidence-based interventions to address these needs. This paper presents the study design and protocol for the first randomized controlled trial (RCT) of a mind-body intervention for adolescents with NF, Resilient Youth with NF (RY-NF), versus an educational control group, Health Education for NF (HE-NF), both delivered in groups via secure live video.
This is an ongoing, single-blind efficacy RCT. Recruitment began in November 2019 and will continue until March 2022. We will enroll 200 English-speaking, geographically diverse adolescents (ages 12-17) with NF1 and NF2 who report significant distress or difficulty coping with their NF symptoms. We will use a shared-baseline, linear mixed model to compare the effect of RY-NF versus HE-NF on changes in quality of life (QoL) and psychosocial outcomes from baseline to post-intervention, and 6- and 12-month follow-ups. We will also develop NF-specific minimal clinically important difference (MCID) for QoL variables, and conduct mediation and moderation analyses to understand mechanisms of improvement.
This study has important clinical and public health implications for the psychosocial functioning of adolescents with NF. It provides a model for efficient delivery of virtual psychosocial care for adolescents with rare diseases. Plans for dissemination and implementation of the RY-NF should efficacy be ascertained are also discussed.
神经纤维瘤病（NF）是一组具有不同遗传特征的神经系统疾病，其特征为易发生神经鞘瘤。尽管 NF 1 型和 2 型（NF1 和 NF2）青少年报告生活质量差和心理社会负担高，但目前尚无针对这些需求的循证干预措施。本文介绍了首个针对 NF 青少年的身心干预研究的研究设计和方案，即具有韧性的 NF 青少年（RY-NF）与教育对照组（NF 健康教育（HE-NF））的比较，这两种干预均通过安全的实时视频进行团体干预。
这是一项正在进行的、单盲的疗效 RCT。招募工作于 2019 年 11 月开始，将持续到 2022 年 3 月。我们将招募 200 名讲英语、地理分布广泛的 NF1 和 NF2 青少年（12-17 岁），他们报告有明显的困扰或难以应对 NF 症状。我们将使用共享基线、线性混合模型来比较 RY-NF 与 HE-NF 对生活质量（QoL）和心理社会结果的影响，从基线到干预后、6 个月和 12 个月的随访。我们还将为 QoL 变量制定 NF 特异性的最小临床重要差异（MCID），并进行中介和调节分析以了解改善的机制。
这项研究对 NF 青少年的心理社会功能具有重要的临床和公共卫生意义。它为为青少年罕见病提供虚拟心理社会护理提供了一种有效的方法。还讨论了在确定 RY-NF 的疗效后，应如何计划传播和实施。
