Blended cognitive behaviour therapy for children and adolescents with mitochondrial disease targeting fatigue (PowerMe): study protocol for a multiple baseline single case experiment

I L Klein,T J Hoogeboom,M C H Janssen,J A M Smeitink,E Van Der Veer,C M Verhaak,K F E Van De Loo,J A E Custers

doi:10.1186/s13063-021-05126-7

Abstract

BackgroundMitochondrial disease is a rare, hereditary disease with a heterogeneous clinical presentation. However, fatigue is a common and burdensome complaint in children and adolescents with mitochondrial disease. No psychological intervention targeting fatigue exists for paediatric patients with a mitochondrial disease. We designed the PowerMe intervention, a blended cognitive behaviour therapy targeting fatigue in children and adolescents with mitochondrial disease. The aim of the intervention is to reduce perceived fatigue by targeting fatigue-related cognitions and behaviours.MethodsA multiple baseline single case experiment will be conducted in five children (8–12 years old) and 5 adolescents (12–18 years old) with mitochondrial disease and severe fatigue. Patients will be included in the study for 33 weeks, answering weekly questions about the fatigue. Patients will be randomly assigned a baseline period of 5 to 9 weeks before starting the PowerMe intervention. The intervention consists of face-to-face and online sessions with a therapist and a website with information and assignments. The treatment will be tailored to the individual. Each patient will work on their personalized treatment plan focusing on personally relevant goals. The primary outcome is perceived fatigue. Secondary outcomes are quality of life, school presence and physical functioning.DiscussionThe results of the PowerMe study will provide information on the efficacy of a blended cognitive behaviour therapy on reducing perceived fatigue and its impact on daily life in children and adolescents with mitochondrial disease. Strengths and limitations of the study design are discussed.Trial registrationDutch Trial Register NTR 7675. Registered on 17 December 2018. Identifier https://www.trialregister.nl/trial/7433

Highlights

Mitochondrial disease is a rare, hereditary disease with a heterogeneous clinical presentation
We propose a blended cognitive behaviour therapy (CBT) aimed at reducing perceived fatigue: the PowerMe intervention
We hypothesize that the intervention significantly reduces perceived fatigue in severely fatigued children and adolescents with Mitochondrial diseases (MD)

Summary

Introduction

Mitochondrial disease is a rare, hereditary disease with a heterogeneous clinical presentation. Fatigue is a common and burdensome complaint in children and adolescents with mitochondrial disease. No psychological intervention targeting fatigue exists for paediatric patients with a mitochondrial disease. We designed the PowerMe intervention, a blended cognitive behaviour therapy targeting fatigue in children and adolescents with mitochondrial disease. The aim of the intervention is to reduce perceived fatigue by targeting fatigue-related cognitions and behaviours. Cells that make up organs and tissues that require a lot of energy to function properly contain a higher number of mitochondria. These high-energy organs and tissues are most commonly affected in a MD; examples are the brain, heart, skeletal muscles and kidneys [2]. The course of the disease is often progressive [5]

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