Cognitive Behavioral Therapy and Exercise can help with Myotonic Dystrophy

There is no treatment for myotonic dystrophy…yet. In the interim period a new study shows that behaorial therapy and exercise can help to stem the huge impact this disease has on patients. Here is the conclusion : 

Interpretation Cognitive behavioural therapy increased the capacity for activity and social participation in patients with myotonic dystrophy type 1 at 10 months. With no curative treatment and few symptomatic treatments, cognitive behavioural therapy could be considered for use in severely fatigued patients with myotonic dystrophy type 1.

However, in reviewing the study this approach requires huge amount of medical resources that may not be available. Hours of analysis of the issues with patients and then tailoring the approach to each patient and tweaking it periodically. Working with a medical professionals for hours is very expensive and most health systems ahve no way to accomplish this.

Cognitive behavioural therapy with optional graded
exercise therapy in patients with severe fatigue with myotonic
dystrophy type 1: a multicentre, single-blind, randomised trial


Kees Okkersen, Cecilia Jimenez-Moreno, Stephan Wenninger, Ferroudja Daidj, Jeffrey Glennon, Sarah Cumming, Roberta Littleford,
Darren G Monckton, Hanns Lochmüller, Michael Catt, Catharina G Faber, Adrian Hapca, Peter T Donnan, Gráinne Gorman, Guillaume Bassez,
Benedikt Schoser, Hans Knoop, Shaun Treweek, Baziel G M van Engelen, for the OPTIMISTIC consortium†


Summary
Background Myotonic dystrophy type 1 is the most common form of muscular dystrophy in adults and leads to severe fatigue, substantial physical functional impairment, and restricted social participation. In this study, we aimed to
determine whether cognitive behavioural therapy optionally combined with graded exercise compared with standard care alone improved the health status of patients with myotonic dystrophy type 1.


Methods We did a multicentre, single-blind, randomised trial, at four neuromuscular referral centres with experience in treating patients with myotonic dystrophy type 1 located in Paris (France), Munich (Germany), Nijmegen (Netherlands), and Newcastle (UK). Eligible participants were patients aged 18 years and older with a confirmed
genetic diagnosis of myotonic dystrophy type 1, who were severely fatigued (ie, a score of ≥35 on the checklistindividual strength, subscale fatigue). We randomly assigned participants (1:1) to either cognitive behavioural therapy plus standard care and optional graded exercise or standard care alone. Randomisation was done via a central webbased system, stratified by study site. Cognitive behavioural therapy focused on addressing reduced patient initiative, increasing physical activity, optimising social interaction, regulating sleep–wake patterns, coping with pain, and
addressing beliefs about fatigue and myotonic dystrophy type 1. Cognitive behavioural therapy was delivered over a 10-month period in 10–14 sessions. A graded exercise module could be added to cognitive behavioural therapy in Nijmegen and Newcastle. The primary outcome was the 10-month change from baseline in scores on the DM1-Activ-c scale, a measure of capacity for activity and social participation (score range 0–100). Statistical analysis of the primary outcome included all participants for whom data were available, using mixed-effects linear regression models with
baseline scores as a covariate. Safety data were presented as descriptives

This trial is registered with ClinicalTrials. gov, number NCT02118779.


Findings Between April 2, 2014, and May 29, 2015, we randomly assigned 255 patients to treatment: 128 to cognitive behavioural therapy plus standard care and 127 to standard care alone. 33 (26%) of 128 assigned to cognitive behavioural therapy also received the graded exercise module. Follow-up continued until Oct 17, 2016. The DM1- Activ-c score increased from a mean (SD) of 61·22 (17·35) points at baseline to 63·92 (17·41) at month 10 in the cognitive behavioural therapy group (adjusted mean difference 1·53, 95% CI –0·14 to 3·20), and decreased from 63·00 (17·35) to 60·79 (18·49) in the standard care group (–2·02, –4·02 to –0·01), with a mean difference between groups of 3·27 points (95% CI 0·93 to 5·62, p=0·007). 244 adverse events occurred in 65 (51%) patients in the cognitive behavioural therapy group and 155 in 63 (50%) patients in the standard care alone group, the most common
of which were falls (155 events in 40 [31%] patients in the cognitive behavioural therapy group and 71 in 33 [26%] patients in the standard care alone group). 24 serious adverse events were recorded in 19 (15%) patients in the cognitive
behavioural therapy group and 23 in 15 (12%) patients in the standard care alone group, the most common of which were gastrointestinal and cardiac. Interpretation Cognitive behavioural therapy increased the capacity for activity and social participation in patients with myotonic dystrophy type 1 at 10 months. With no curative treatment and few symptomatic treatments, cognitive behavioural therapy could be considered for use in severely fatigued patients with myotonic dystrophy type 1.

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Myotonic Dysrophy Drugs Lead Candidates for Fast Track Autism Spectrum Disorder Testing

NIH officials announced yesterday that a new contract has been let with UCLA to form a network of researchers at various academic institutions to identify promising new and older drug compounds to treat Autism Spectrum Disorder (ASD)  and to see if they merit additional investments.

The program is part of a new initiative called  “Fast Fail” intends to vastly speed up the drug development process and reduce the costs of this drug development. Instead of taking years of work to see if a drug works this Fast Fail process could see results within weeks. As ASD is a huge issue for the USa and other countries this fast testing with myotonic dystrophy drugs could lead to treatments in a much faster time frame.

Dr. James McCraken who is leading the effort at UCLA states “The Whole idea is just getting much better in these early phases at identifying drugs that are going to be efficacious and safe and thereby speeding the development of effective new therapies and reducing the overall cost”

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Personality changes

In standard Myotonic Dystrophy one of the main characteristics of the disorder are “reduced initiative”, “inactivity”, and “apathetic temperament” and social deterioration of the families through generations has been stressed (Caughey 1963)

Because the mother is most often affected with myotonic dystrophy it generally becomes necessary for other assistance to maintain some standard for care for the Child with CMD. Here is one researchers global comments:

“While in the country in search of certain “myotonic’s” homes , it was often possible to identify a residence by its neglected appearance, the obvious need of repairs, the unkempt yard and garden choked with overgrown grass and weeds, which provided a vivid contrast with the surrounding well-kept homes” (Caughey 1963) ”

We have often found that affected individuals, when just mildly incapacitated, were often content to sit or lie idly for hours”

Studies by Brumback found depression to be an integral part of Myotonic Dystrophy using the Hamilton Depression Rating Scale. Bird found that 28% of myotonic dystrophy Patients had high scores on the Minnesota multiphasic personality inventory. Others have found that depression was secondary to the Chronic progression of the disease. Cuthill suggests that depression might occur but that it was seen as a reaction o the disease. In 1998 Bungener published the following information:

Patients with Myotonic Dystrophy were not severely depressed but did present symptoms of mild depression. He mentions that the literature suggests a close association between depression and progressive diseases such as Myotonic Dystrophy.

Patients with Myotonic Dystrophy did have high levels of emotional deficit. It manifests itself as anhedonia (Total loss of feeling of Pleasure in acts that normally give pleasure), lack of expressiveness as evidenced by monotonous mood, apathy, and an inability to anticipate pleasure. This emotional deficit occurs early and researchers that found apathy and lack of motivation are the primary manifestations. Four of 14 patients exhibited an avoidance personality disorder

He concludes that: Patients with Myotonic Dystrophy present a characteristic emotion profile that of emotional deficit, with the deficit appearing early in the disease and which could be interpreted as an adaptive psychological process or a direct consequence of the CNS lesions caused by the genetic mutation.

It is important to understand the personality changes of patients with Myotonic Dystrophy. As it is an autosomal dominant disorder at least one parent generally the mother will have the disease. As the the mother may be the primary caregiver to the nuclear family the personality information that has been elucidated here will have a significant impact on the family.

Full Text studies

Link to personality Study
Link to Study of 25 women with DM
Apathy and Sleepiness
Brain disorder
List of References citing the incidence of CMD Risks of Offspring of Women with Myotonic Dystrophy (Harper 1972)

Normal 50%
Neonatal Deaths & Still births 12%
Severely Affected Surviving 9%
Later Affected 29%

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