Background: 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.
Methods: 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.
Discussion: 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.
Keywords: Adolescent; Mind-body program; Neurofibromatoses; Quality of life; Resiliency.
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