Treatment with cognitive behavioral therapy helps patients with myotonic dystrophy type 1 in improving their physical abilities and social participation. This is the result of a survey of more than 250 patients in four European countries. Never before has a behavioral intervention for a rare genetic disorder been investigated on such a large scale. Results published in The Lancet Neurology indicate for the first time that patients with myotonic dystrophy are now receiving a new treatment option. The research was published by a group of researchers and doctors, led by Radboud university medical center’s Spierziektecentrum (center for neuromuscular diseases).Myotonic dystrophy type 1, abbreviated as MD1, is a rare inherited disease that affects almost all organs, including the brain, and patients run the risk of premature death. The disease is chronic and progressive. In addition to physical inactivity and loss of initiative, patients are particularly affected by muscle weakness and severe fatigue. This leads to considerable physical and social limitations. There is no cure for the disease, and only a few treatments can alleviate the symptoms.
Behavioral interventionBecause MD1 is a multisystem disease, the disease also has a strong behavioral component. Patients could, therefore, benefit from a behavioral intervention that teaches them to look at their symptoms differently and deal with them differently. Patients with type 1 diabetes, for example, appear to be able to overcome their fatigue symptoms through such an intervention. Some patients with chronic fatigue syndrome also recover from cognitive behavioral therapy.
Based on the symptoms of patients with MD1, Kees Okkersen and Baziel van Engelen of Radboud university medical center, together with European colleagues, developed the so-called OPTIMISTIC study, studying the effect of cognitive behavioral therapy in more than 250 patients. This is the largest research ever into a treatment for MD1.
Personal programThe patients were recruited in specialized centers for the treatment of muscle diseases in Paris, Munich, Newcastle, and Nijmegen. All patients in the study received the standard treatment, as is customary in the participating countries. Also, some patients were assigned ten sessions of cognitive behavioral therapy. Together with their physician, test subjects could choose from seven different program components, based on, for example, sleeping patterns, own initiative and existing thoughts about fatigue and illness. The entire study period lasted ten months, after which the effect of the treatment was determined. Also, there was a follow-up measurement after sixteen months.
More activity, less fatiguePatients undergoing cognitive behavioral therapy showed significantly more activity and social participation than patients who followed standard treatment alone, despite differences between individual programs. They were also fitter, less tired and less sleepy. Baziel van Engelen: “Hopefully we can keep the muscle strength stable for as long as possible. MRI examination of the muscles showed that muscle size had even increased through training. "
Saskia Baas (43) from Groningen (the Netherlands) participated in the study and was part of the group that received cognitive behavioral therapy: “I was very happy with that. The beauty of the cognitive behavioral therapy was that it was tailor-made. I was already quite active, so for me, it was important to learn how to pace myself, and to improve my day-night rhythm. The treatment was a real breakthrough for me. By dividing tasks between my husband and myself and by paying more attention to my activity level, I can now pick up my son every day and help with the cooking. It has certainly improved my quality of life a lot.”
More fallsThe increased activity of the patients as a result of cognitive behavioral therapy did seem to have a disadvantage: patients would often fall. Incidentally, this also happened to patients who did not receive behavioral therapy. Kees Okkersen: “It is very well possible that cognitive behavioral therapy leads to more falls, but the number of falls can also be higher because people are more visible and are normally not so aware that they often fall due to their illness. Deciding whether the advantages outweigh the disadvantages of cognitive behavioral therapy should, therefore, be left to patients, healthcare providers, and family.”
Refresher sessionsThe results of the follow-up, six months after the study, showed that the effect of cognitive behavioral therapy had slightly subsided. The gap between the results of both groups seemed to be closing. Baziel van Engelen: “That is an argument for regular refresher sessions. For example, we can apply e-health options to help people keep up with their behavioral change. On the other hand, some of the participants have really been able to make the switch. It is wonderful to see that people have started to live their lives differently thanks to the study."
OptimisticThe results of this research stem from the OPTIMISTIC study, which started in 2012 with a European subsidy grant of three million euros. Baziel van Engelen: “With this research, we wanted to set a different tone. MD1 causes a lot of problems for patients and their environment. It is a difficult disease, and simple successes aren't available. These people need care, and while there is no cure for the disease, you can certainly ensure progress for these patients. We are now showing that this is indeed possible. The results of this study facilitate the search for a drug for MD1."
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