Ramdharry G.1,2, Wallace A.3,4, Pietrusz A.5, Dudziec M.6, Jones K.7, Sterr A.8, Skorupinska I.5, Skorupinska M.5, Baio G.9, Hennis P.10, Laura M.5, Machado P.5, Trenell M.11, Hanna M.5, Reilly M.M.5
1Kingston University, School of Allied Health, Midwifery and Social Work, London, United Kingdom, 2University College Hospitals NHS Foundation Trust, Queen Square Centre for Neuromuscular Diseases, London, United Kingdom, 3Brunel University, Department of Clinical Sciences, London, United Kingdom, 4UCL, Institute of Child Health, London, United Kingdom, 5UCL, Queen Square Centre for Neuromuscular Diseases, London, United Kingdom, 6Kingston University, Faculty of Health, Social Care and Education, London, United Kingdom, 7University College Hospitals NHS Foundation Trust, Queen Square Department of Neuromuscular Diseases, London, United Kingdom, 8University of Surrey, Brain & Behaviour Research group, Guildford, United Kingdom, 9UCL, Department of Statistical Science, London, United Kingdom, 10UCL, Institute of Sport & Exercise Science, London, United Kingdom, 11Newcastle University, Movelab Muscle Performance & Exercise Laboratory, Newcastle, United Kingdom
Background: The purpose of aerobic exercise to treat people with Charcot Marie Tooth disease type 1A (CMT) remains an important unresolved issue. Recent reviews of exercise in CMT highlight the need for high quality randomised controlled trials to establish the benefit of aerobic training in people with peripheral neuropathy.
People with CMT are less active and fit than the general population so are also at risk of morbidities common to the general population. They need to know if they can safely exercise to reduce these risks and improve function.
People with CMT are less active and fit than the general population so are also at risk of morbidities common to the general population. They need to know if they can safely exercise to reduce these risks and improve function.
Purpose: The proposed pilot/feasibility trial will be an important step in establishing whether community based aerobic training is an effective strategy to manage the secondary, long term problems experienced by people with CMT. The feasibility, safety and effect of aerobic training on fitness levels, muscle strength and function was explored. Motivation, confidence and barriers to exercise were also explored using in depth qualitative interviews.
Methods: A crossover design was used with training and control periods. Each subject was enrolled for a 34-week period, and randomly assigned to two groups: group A underwent 12 weeks training, 8-week washout period, then a 12-week control period. For group B, the training and control periods were reversed. For the training intervention, participants used bicycle ergometers in selected local gyms, with telephone support from the trial physiotherapist plus 8 sessions with a gym trainer.
Participants underwent maximal exercise testing on a bicycle ergometer to measure their peak oxygen uptake (VO2peak) at the four time points. Additional measures were taken of impairment (leg strength, disease severity, fatigue and body composition), function (walking speed, walking endurance, physical activity) and self-reported measures (self-efficacy, health status, motivation to exercise, barriers to exercise). Safety was monitored through measurement of blood plasma creatine kinase, pain and fatigue. On study completion, participants were invited to participate in an interview to explore their experience of exercise, motivation and involvement in the study.
Results: 285 people with CMT were invited to participate, 28 people were randomised and 8 withdrew. There was a good level of participation with 76% completion of training sessions. The training intervention was well tolerated with no increases in pain and blood serum creatine kinase levels.
An increase in VO2peak (ml/min/kg) was observed with training (mean difference VO2peak 2.14, 95% CI 0.33 to 3.94) that was not observed following the control period (mean difference VO2peak -0.23, 95% CI -1.68 to 1.23). There were no major changes in other measures of impairment, function and patient reported outcome measures. Large between subject variability was noted with many of the measures.
Qualitative interviews revealed that participants felt fitter, valued the support from trainers, reported greater wellbeing and they wished to continue exercising.
Conclusion(s): The study found that the exercise intervention was well tolerated, safe with good participation rates. The main outcome under exploration indicated some improvements in aerobic capacity with training.
Implications: Aerobic exercise shows some evidence of effect and is safe when delivered through a supportive community model. Larger trials of efficacy are required.
Funding acknowledgements: This work was funded by an NIHR Research for Patient Benefit grant PB-PG 0711-25151
Topic: Neurology
Ethics approval: Ethical approval was received from the Surrey Borders NRES Research Ethics Committee.
All authors, affiliations and abstracts have been published as submitted.
