Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy 1, 2018, Okkersen et al

Lucibee · Jun 20, 2018

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, et al.
Lancet Neurology - 19th June 2018 - Online First

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 checklist-individual 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 web-based 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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[Sorry - I'm having terrible trouble with the formatting...
OK - think I have it sorted now!]

Lucibee · Jun 20, 2018

Details of the outcome measure used - DM1-ActivC - is here: https://www.ncbi.nlm.nih.gov/pubmed/26484877

(Oh look - another subjective measure that changes by tiny amounts and is called significant - aargh!)

Esther12 · Jun 20, 2018

Here's that tweet on the 'OPTIMISTIC' group:

I wonder if they had a prespecified criteria for clinical significance for their DM1-ActivC outcome?

They mention finding improvement for an objective measure of physical activity in the commentary excerpt.

Quite a big increase in the number of adverse events with CBT (244 vs 155).

I shouldn't use this to procrastinate PACE stuff!

strategist · Jun 20, 2018

They switched to another primary outcome measure. Unsure about details. They make it sound relatively harmless, and it could be legitimate, but could also be a way to inflate the efficacy of the CBT group. I'm not going to look into it because it doesn't interest me that much, but maybe someone with more interest will want to investigate.

Jonathan Edwards · Jun 20, 2018

So what is the point anyway if there was no improvement in quality of life?

large donner · Jun 20, 2018

Jonathan Edwards said: ↑

So what is the point anyway if there was no improvement in quality of life?
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The therapists quality of life will improve immensely sitting working from home nodding every few minutes and drinking coffee and having a fully booked client schedule at £50-£100 an hour. It pays much better than reiki and lets them put to use that little therapy room in the house they set up with all the crystals and dream catchers they hung up when they stopped working in Sainsburys cos that was just too boring.

Snow Leopard · Jun 20, 2018

Mean (24 h) physical activity (accelerometry [ENMO])† increased by 6.5%.

Not a big improvement, but acceptable for a adjunct therapy.

Sean · Jun 21, 2018

Snow Leopard said: ↑

Not a big improvement, but acceptable for a adjunct therapy.
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If sustained at follow-up.

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Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy 1, 2018, Okkersen et al

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