Virtual mind-body treatment for adolescents with neurofibromatosis: Study protocol for a single-blind randomized controlled trial.


Journal

Contemporary clinical trials
ISSN: 1559-2030
Titre abrégé: Contemp Clin Trials
Pays: United States
ID NLM: 101242342

Informations de publication

Date de publication:
08 2020
Historique:
received: 30 03 2020
revised: 24 06 2020
accepted: 29 06 2020
pubmed: 8 7 2020
medline: 25 9 2021
entrez: 8 7 2020
Statut: ppublish

Résumé

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.

Sections du résumé

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.

Identifiants

pubmed: 32634485
pii: S1551-7144(20)30156-7
doi: 10.1016/j.cct.2020.106078
pii:
doi:

Types de publication

Clinical Trial Protocol Journal Article Research Support, U.S. Gov't, Non-P.H.S.

Langues

eng

Sous-ensembles de citation

IM

Pagination

106078

Informations de copyright

Copyright © 2020 Elsevier Inc. All rights reserved.

Auteurs

Mira Reichman (M)

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.

Eric Riklin (E)

Department of Psychology, Fordham University, Dealy 336, 441 East Fordham Rd, Bronx, NY 10458, United States of America. Electronic address: eriklin@fordham.edu.

Eric Macklin (E)

Biostatistics Center, Massachusetts General Hospital, 50 Staniford Street, St 560, Boston, MA 02114, United States of America. Electronic address: emacklin@mgh.harvard.edu.

Ana-Maria Vranceanu (AM)

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.

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Classifications MeSH