Browse by author
Lookup NU author(s): Professor Grainne Gorman,
Dr Cecilia Jimenez Moreno,
Professor Hanns Lochmuller,
Professor Michael Trenell,
Professor Mike Catt
Full text for this publication is not currently held within this repository. Alternative links are provided below where available.
© 2015 van Engelen and The OPTIMISTIC Consortium; licensee BioMed Central. 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. 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.
Author(s): van Engelen B, Abghari S, Aschrafi A, Bouman S, Cornelissen Y, Glennon J, Groot P, Heerschap A, Heskamp L, Heskes T, Kapusta K, Klerks E, Knoop H, Maas D, Okkersen K, Poelmans G, Rahmadi R, van Nimwegen M, Gorman G, Moreno CJ, Lochmuller H, Trenell M, van Laar S, Wood L, Schoser B, Wenninger S, Schuller A, Auguston R, Baptiste L, Barau C, Bassez G, Chevalier P, Couppey F, Delmas S, Deux J-F, Dogan C, Hamadouche A, Hankiewicz K, Lhermet L, Minier L, Rialland A, Schmitz D, Monckton DG, Cumming SA, Adam B, Donnan P, Hannah M, Hogarth F, Littleford R, McKenzie E, Rauchhaus P, Wilkie E, Williamson J, Catt M, Dittrich J, Schwalber A, Treweek S
Publication type: Article
Publication status: Published
Online publication date: 23/05/2015
Acceptance date: 01/05/2015
ISSN (electronic): 1745-6215
Publisher: BioMed Central Ltd.
Altmetrics provided by Altmetric