1. Cognitive behaviour therapy plus aerobic exercise training to increase activity in patients with myotonic dystrophy type 1 (DM1) compared to usual care (OPTIMISTIC): study protocol for randomised controlled trial
- Author
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Engelen, B.G.M. van, Groot, P.C., Abghari, S., Aschrafi, A., Bouman, S.F., Cornelissen, Y., Glennon, J.C., Heerschap, A., Heskamp, L., Heskes, T., Kapusta, K.A., Klerks, E., Knoop, H., Maas, D, Okkersen, C.P., Poelmans, G.J.V., Rahmadi, R., Nimwegen, M. van, and Other departments
- Subjects
Gerontology ,Male ,Time Factors ,medicine.medical_treatment ,Psychological intervention ,Medicine (miscellaneous) ,Cognitive behavioural therapy ,Severity of Illness Index ,law.invention ,Study Protocol ,0302 clinical medicine ,Randomized controlled trial ,Quality of life ,Clinical Protocols ,law ,Myotonic Dystrophy ,Pharmacology (medical) ,Fatigue ,0303 health sciences ,Exercise Tolerance ,Myotonic dystrophy type 1 ,Disorders of movement Donders Center for Medical Neuroscience [Radboudumc 3] ,Combined Modality Therapy ,Aerobic exercise training ,Checklist ,3. Good health ,Graded exercise therapy ,Exercise Therapy ,Rare diseases ,Europe ,Treatment Outcome ,Female ,DM1 ,Neuroinformatics ,musculoskeletal diseases ,medicine.medical_specialty ,Biophysics ,Neurophysiology ,Myotonic dystrophy ,03 medical and health sciences ,Predictive Value of Tests ,Urological cancers Radboud Institute for Molecular Life Sciences [Radboudumc 15] ,medicine ,Aerobic exercise ,Humans ,Physical Examination ,030304 developmental biology ,Neurodevelopmental disorders Donders Center for Medical Neuroscience [Radboudumc 7] ,Cognitive Behavioral Therapy ,business.industry ,Surrogate endpoint ,Patient Selection ,Data Science ,medicine.disease ,Sample Size ,Physical therapy ,Quality of Life ,business ,030217 neurology & neurosurgery - Abstract
Contains fulltext : 144005.pdf (Publisher’s version ) (Open Access) BACKGROUND: Myotonic dystrophy type 1 (DM1) is a rare, inherited chronic progressive disease as well as an autosomal dominant multi-systemic disorder. It is probably one of the most common adult forms of muscular dystrophy, with a prevalence of approximately 10 per 100,000 people affected. With 733 million people in Europe, we estimate that 75,000 people in Europe are affected with DM1. METHODS/DESIGN: OPTIMISTIC is a multi-centre, randomised trial designed to compare an intervention comprising cognitive behavioural therapy (CBT) plus graded exercise therapy against standard care. Participants will be recruited from myotonic dystrophy clinics and neuromuscular centres in France, Germany, the Netherlands and the United Kingdom. A sample size of 208 individuals is needed. To allow for some potential loss to follow-up, a total of 296 male and female patients aged 18 years and older with genetically proven classical or adult DM1 and suffering from severe fatigue (only DM1 patients with a Checklist Individual Strength (CIS) subscale fatigue severity score >/=35 are likely to benefit from the intervention), able to walk independently and able to complete the trial interventions will be included. The primary outcome of the study is the score on the DM1-Activ scale, which is a measure of activity and participation for patients with DM1. Secondary outcomes include the 6-minute walk test, objective physical activity measured with an accelerometer, quality of life and cognitive measures. The trial will also collect data on potential effect modifiers of the short- and long-term clinical response, including pain, muscular impairment and cognitive-behavioural variables. In addition, OPTIMISTIC will identify genetic factors that predict outcome and potential biomarkers as surrogate outcome measures that best explain the observed clinical variation. DISCUSSION: OPTIMISTIC will not only provide effectiveness data on an intervention that could fill a treatment-gap for DM1 patients but will also improve our understanding of the relevant determinants of the prognosis of DM1. TRIAL REGISTRATION: REGISTRATION NUMBER: Cinicaltrials.gov NCT02118779 ; registered 11 April 2014. 19 p.
- Published
- 2015