Title: ACCEPTANCE & COMMITMENT THERAPY FOR MUSCLE DISEASE (ACTMUS)
Funder: Department of Health and Social Care

Abstract: Background Muscle diseases (MD) are characterised by progressive muscle weakness usually of the limb muscles but occasionally muscles of swallowing and breathing, and cardiac muscle can be affected. MD can be acquired or genetic and can have congenital to adult age of onset. The wasting and weakness of the muscles causes increasing difficulties with mobility and with arm and hand functions and not surprisingly reduces quality of life (QoL). In most cases there is no direct cure for the MD and efforts are directed at supportive care and avoidance of complications with occasional modest benefit on QoL . Our previous research has shown that while QoL is determined by the severity of the MD this does not explain all aspects of the reduced QoL of those with MD and that psychosocial factors including illness perceptions explains some aspects of the reduction in QoL better than does the severity of the MD. We have therefore been motivated to explore the potential for psychosocial intervention to improve QoL of those with MD. Further research by us which included cluster analysis of our QoL survey data and qualitative interviews with those with MD suggested that a cognitive behavioural approach using Acceptance and Commitment Therapy (ACT) would best suit those with MD. Our pilot study of this approach in 7 subjects with MD was promising. We therefore wish to conduct a definitive trial of ACT psychological intervention to see if it does improve the QoL of those with MD. This trial will be the first of its kind for muscle disease. Aims & Objectives, the primary objective is to conduct a randomised controlled trial to determine the efficacy of psychological intervention for improving the QoL of those with muscle disease. Secondary objectives are to; 1) Assess the processes that may be responsible for mediating the treatment effect, 2) obtain data on the performance of the chosen outcome measures for future trials, 3) obtain information that will facilitate the roll out of the psychological intervention as a self-help intervention needing minimal support for all adults with muscle disease. Design; this will be an unblinded randomised controlled trial with 154 subjects with one of four specific muscle diseases recruited from the muscle clinics of Kings College Hospital and The Royal London Hospital as well as from the Muscular Dystrophy Campaign (MDC), a patient support group, and from UK registries of muscle disease patients. Participants will be randomised to either active intervention with 5 self help modules supported by weekly telephone calls over a 6 week period or to enhanced standard care. The primary outcome will be the change in overall QoL score at 9 weeks as assessed by a validated and well used muscle disease specific QoL scale. Secondary outcomes will include assessments of mood, functional ability, and muscle strength. Dissemination The trial protocol will be published in open access journal and the final full report will be published in a peer-reviewed journal. We will also publicise the opening of the trial to the MDC membership and the patient registries used for recruitment. We will give summary results to those organisations. If the ACT intervention is successful in improving QoL we envisage that it will be generalizable to all those with MD of any description. We have purposely designed the intervention as a self-help treatment requiring minimal support so as to avoid the requirement for specialist psychology services which are already over-stretched in the NHS. Patient benefit; MD are long term chronic conditions that have no specific treatment and so the prospect of improving the QoL of such patients by psychosocial intervention is a valuable opportunity.

Lay abstract: Muscle diseases (MD) are a group of primary disorders of the muscle itself which can be acquired or genetic and that mostly result in progressive muscle wasting and weakness causing difficulties with mobility and arm and hand functions. There are a number of specific muscle diseases which can be diagnosed on basis of several criteria including; the pattern of the muscle weakness, the pathology appearances of the muscle on a muscle biopsy and in some cases genetic testing. In adults muscle disease are usually chronic long-term conditions that do not have a definitive cure. Supportive care has reduced complications from muscle disease and improved survival in some cases but we would like to improve QoL also. We know that the QoL of those with MD is not just affected by the severity of their MD but that a variety of psychological variables also worsen QoL. Based upon our knowledge of these psychological variables we feel that a particular type of psychological intervention known as &ldquo;acceptance and commitment therapy&rdquo; (ACT) could potentially improve QoL in those with MD. We therefore propose to test whether ACT does in fact improve QoL in those with MD by randomising 154 patients to receiving either the ACT therapy or standard medical care and seeing if the QoL of those receiving the ACT treatment improves more that those not receiving this treatment. If this trial shows this psychological treatment to be beneficial we will then expect that it can be applied to all adults with muscle disease nationally. For this reason we have purposely designed the ACT treatment to be one that can be applied to the general MD population in the UK without the need for specialist psychological support which is scarce in the NHS.

Research activity codes: 6.6 PSYCHOLOGICAL AND BEHAVIOURAL
Health categories: MUSCULOSKELETAL