Title: Evaluating the benefits of community based aerobic training on the physical health and well-being of people with neuromuscular diseases: a pilot study
Funder: Department of Health (England)

Abstract: Background: Historically people with NMDs were told to avoid exercise for fear of exacerbation of the disease process. In recent years, small studies of aerobic exercise have indicated there may be benefit without deterioration in muscle strength and function. Cochrane reviews of exercise in nerve and muscle disease highlighted the small size of these studies and recommended further work with inclusion of a control condition4,5. Exercise to reduce the impact of disease is currently not supported by the evidence to date. The potential beneficial impact on risk factors for diabetes and cardiovascular diseases cannot be under-estimated in a population that has been seen to be under active9,10. Aims: The specific objective of this pilot study is to investigate the effect of aerobic training in two common disabling neuromuscular diseases (NMD): Charcot-Marie-Tooth disease (CMT) and Inclusion Body Myositis (IBM) for which there is no standard effective treatment. The key difference to previous work in these conditions is the larger sample size, the inclusion of a control group and consideration of the wider effects of exercise in a community setting. The primary aim is to assess if aerobic training gives an improvement in fitness levels compared to a control condition. The data from this study will demonstrate if there is any effect of training on aerobic capacity and can be used to power larger studies of training efficacy. Secondary aims are to ascertain if there are measureable improvement in muscle strength, body composition and functional abilities of participants. In addition, the effects on fatigue, quality of life, activity levels, self efficacy and perceived motivations/barriers to exercise will be explored. The feasibility and acceptability of training people in the community environment will also be ascertained by this study. Safety will be monitored using physiological and patient reported measures. Plan of investigation: Sixty subjects, (30 from each disease group, aged 18 to 80), will be recruited from the established large neuromuscular clinics and patient cohorts at Queen Square and the established Queen Square neuromuscular database. Both disease groups will be investigated concurrently with the same methods but will be viewed and analysed as separate studies. A crossover design will be used with training and control periods. The trial will span three years with each subject participating for a 34 week period. For the training intervention, participants will train in selected local gyms using a bicycle ergometer. Primary outcome measurement for this study is maximum aerobic capacity during exercise testing. Secondary outcome measurement will be muscle strength, lean body mass composition, and activity levels. Importantly, this study will investigate non-motoric effects of exercise such as mood, motivation, sleep and fatigue using questionnaires and interviews. Benefits to patients and the NHS: This study will provide information regarding possible physiological benefits and safety of aerobic training. It will also shed light on whether there any improvements in fitness alter the impact of the disease. A positive outcome will also add to the evidence for the benefits of exercise. It has been our experience that some of our patients have been refused support to exercise by primary care trusts due to lack of evidence of efficacy. It will provide support for people with NMDs to access facilities such as NHS exercise prescription schemes. It is also our experience that people with NMD traditionally access exercise through physiotherapy and rehabilitation services. If this study demonstrates feasibility and acceptability of exercise in community gym facilities, this will remove some of the burden from NHS services. People will be encouraged to self manage and take up exercise on a longer term basis.


Research activity codes: 6.7 PHYSICAL
Health categories: MUSCULOSKELETAL;NEUROLOGICAL