Cognitive behavioural therapy could be beneficial for people with myotonic dystrophy

Published Date
Jenny Sharpe

A landmark trial has shown that cognitive behavioural therapy (CBT) can benefit people with myotonic dystrophy type 1.

CBT is a type of psychological therapy, usually involving sessions with a trained therapist. It focuses on how your thoughts and attitudes affect your feelings and behaviour, and teaches you coping skills for dealing with different problems.

CBT can effectively treat a number of mental health conditions, and can also be helpful for people with physical health problems. For example, studies have shown that CBT can relieve fatigue in people with chronic fatigue syndrome and type 1 diabetes.

Fatigue is a major issue for people with myotonic dystrophy as it can impact greatly on everyday life. To see whether CBT might help to tackle fatigue, a group of European researchers – including Dr Baziel van Engelen, who is on our Medical Research Committee – conducted a clinical trial called OPTIMISTIC.

OPTMISTIC was the first-ever myotonic dystrophy trial involving multiple countries (UK, Netherlands, Germany and France). A total of 225 adults with myotonic dystrophy type 1 and severe fatigue took part. The UK site in Newcastle recruited 52 people, 40% of whom heard about the study through the myotonic dystrophy patient registry that MDUK co-funds.

All participants were randomly allocated to one of the following groups:

  • CBT group – received 10-14 sessions of CBT alongside standard care
  • Control group – received only standard care.

After ten months of follow-up, people in the CBT group felt less fatigued and more able to participate in daily activities than those in the control group. Social awareness also improved in the CBT group.

Although CBT was generally safe, more falls were reported in the CBT group than in the control group. This was probably because the CBT group were being more active, and so were more at risk of falling.

The researchers concluded that CBT is beneficial for some people with myotonic dystrophy type 1. However, it must be combined with appropriate measures to prevent falls.

The learnings from the OPTIMISTIC study go beyond the potential effectiveness of CBT. Researchers now know a lot more about outcome measures, genetic analysis, cardiac screening and biomarkers for myotonic dystrophy. This knowledge and experience will be valuable for designing and conducting future clinical trials.

In addition, OPTIMISTIC has shown that it is possible to conduct a large trial for myotonic dystrophy across multiple countries. This is an important message to other researchers and pharmaceutical companies, and could hopefully drive more research.

Dr Grainne Gorman, OPTIMISTIC Principal Investigator in Newcastle, and Dr Cecilia Jimenez Moreno, Research Physiotherapist in Newcastle, said:

The OPTIMISTIC trial has not only been a significant achievement in a scientific perspective but it has been a historic breakthrough for the myotonic dystrophy community. Everyone involved in OPTIMISTIC is and will always be immensely grateful to all those patients and their families who volunteered for the trial. We feel efforts to improve the standards of care of patients with myotonic dystrophy do not finish here but this is certainly a monumental step forward.

Further information

Read the full study in the Lancet Neurology

Find out more about the UK myotonic dystrophy registry

If you have any questions about CBT or myotonic dystrophy research, please contact our Research team at or phone 02078034813.

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