Cognitive Behavioral Therapy May Help Patients with Myotonic Dystrophy Type 1, Trial Shows

Cognitive Behavioral Therapy May Help Patients with Myotonic Dystrophy Type 1, Trial Shows

Cognitive behavioral therapy improved physical activity and social participation in patients with myotonic dystrophy type 1, indicating its potential as a treatment method for these patients, according to results from a clinical trial.

Findings were reported in the study, “Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial,” published in the journal The Lancet Neurology.

Myotonic dystrophy type 1 is the most common form of muscular dystrophy in adults. Patients with this disease have severe fatigue, extensive impairment in physical functioning, and reduced social participation.

There is limited evidence for the effectiveness of various rehabilitative approaches in this patient population, leading to a significant unmet need for treatments.

Past studies have suggested that cognitive behavioral therapy has the potential to positively influence the health outcomes of patients with myotonic dystrophy type 1. However, this had not been tested in a clinical trial setting.

To address this, researchers designed the OPTIMISTIC study (NCT02118779) to determine whether cognitive behavioral therapy combined with graded exercise improved the health status of patients with myotonic dystrophy type 1, compared with standard care alone.

Researchers conducted the multicenter, randomized trial across four neuromuscular referral centers in Paris; Munich; Nijmegen, the Netherlands; and Newcastle, England, all which had prior experience in treating patients with myotonic dystrophy type 1.

From a total of 255 participants, 128 were randomly assigned to receive cognitive behavioral therapy and optional graded exercise plus standard care, and 127 were assigned to receive standard care alone.

For this study’s purposes, cognitive behavioral therapy was focused on addressing “reduced patient initiative, increasing physical activity, optimizing social interaction, regulating sleep-wake patterns, coping with pain, and addressing beliefs about fatigue and myotonic dystrophy type 1,” according to the researchers.

Effectiveness of the therapy was evaluated using the DM1-Activ-c Scale, which measures the capacity for activity and social participation. The primary objective of the study was a positive change in the DM1-Activ-c Scale score from the beginning of the study to 10 months.

Results showed that the DM1-Activ-c score increased from an initial mean of 61.22 points to 63.92 after 10 months of cognitive behavioral therapy. For patients in the standard care group, the score decreased from 63 to 60.79 after 10 months. The mean difference in the DM1-Activ-c score between the two groups was 3.27 points.

For secondary outcome measures, such as fatigue and exercise capacity, patients in the cognitive behavioral therapy group experienced significant improvements, compared with patients in the standard care group.

On the other hand, improvements in quality of life or disease burden were not significant between the groups.

“It should be noted that the trial was not powered for any of the secondary outcome measures except the 6-min walk test,” the researchers wrote.

In the cognitive behavioral therapy group, 65 patients experienced 244 adverse events, and in the standard care group, 63 patients reported 155 adverse events.

The most common adverse event experienced by both groups was falls — recorded 155 times in 40 patients in the cognitive behavioral therapy group, and 71 times in 33 patients in the standard care group.

“Nevertheless, it seems reasonable to conclude that increasing activity levels in people with myotonic dystrophy type 1 will lead to more falls, though most are minor. Balancing this potential harm against the potential benefit of increased activity levels needs to be a shared decision between patients, carers, and health professionals,” the researchers said.

Additionally, the cognitive behavioral therapy group experienced 24 serious adverse events in 19 patients, while the standard care alone group experienced 23 serious adverse events in 15 patients. The most common serious adverse events were gastrointestinal or cardiac.

“Cognitive behavioural therapy increased the capacity for activity and social participation in patients with myotonic dystrophy type 1 at 10 months,” they said. “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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