Mira Reichman1, Eric Riklin2, Eric Macklin3, Ana-Maria Vranceanu4. 1. 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. Electronic address: mreichman@mgh.harvard.edu. 2. Department of Psychology, Fordham University, Dealy 336, 441 East Fordham Rd, Bronx, NY 10458, United States of America. Electronic address: eriklin@fordham.edu. 3. Biostatistics Center, Massachusetts General Hospital, 50 Staniford Street, St 560, Boston, MA 02114, United States of America. Electronic address: emacklin@mgh.harvard.edu. 4. 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; Harvard Medical School, One Bowdoin Square, 1st floor Boston, 02114 Boston, MA, United States of America. Electronic address: avranceanu@mgh.harvard.edu.
Abstract
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.
RCT Entities:
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.
Authors: Keenae Tiersma; Mira Reichman; Paula J Popok; Zoe Nelson; Maura Barry; A Rani Elwy; Efrén J Flores; Kelly E Irwin; Ana-Maria Vranceanu Journal: JMIR Form Res Date: 2022-04-08
Authors: Jonathan Greenberg; Tanya Singh; Grant L Iverson; Noah D Silverberg; Eric A Macklin; Robert A Parker; Joseph T Giacino; Gloria Y Yeh; Ana-Maria Vranceanu Journal: JMIR Res Protoc Date: 2021-01-14