DM1-associated declines in quality of life, including changes in daily activity and social participation, have become an important focus for both clinical research and patient care. Recognizing this burden of disease for both patients and caregivers, the MDF-led Consensus-Based Care Recommendations for Adults with Myotonic Dystrophy Type I includes a section on psychosocial management. Moreover, the recently published OPTIMISTIC study concluded that cognitive behavior therapy could improve patient socialization and capacity for participation in daily activities (Okkersen et al., 2018).

A Descriptive Longitudinal Study in DM1

An understanding of the progression of participation restrictions in relationship to disease course is essential to help maintain the quality of life of DM1 patients and their caregivers. Doctoral candidate, Kateri Raymond, Dr. Cynthia Gagnon (Université de Sherbrooke), and their colleagues have utilized their natural history database to publish a report on daily and social activities in patients living with DM1 (Raymond et al., 2019). 200 adults meeting eligibility criteria were enrolled and assessed at baseline and 9 years later; 115 completed both assessments. The primary instrument used to assess level of and changes in participation was a 77-item Assessment of Life Habits questionnaire within the Human Development Model – Disability Creation Process (HDM-DCP) framework—both daily activities (nutrition, fitness, personal care, communication, housing, and mobility) and social activities (responsibilities, interpersonal relationships, community life, education, employment, and recreation) were assessed.

Over the course of the study, a decline in participation was observed for the overall instrument score, as well as for nutrition, fitness, personal care, mobility, community life, and recreation domains. Increases in disease duration were associated with increased disruption of daily personal and social activities. 10% of the study population was severely affected, with declines in almost all of the participation scores.

Although the study revealed similar progression of restrictions in both genders, there was a trend toward greater decline in each domain for men. Comparison of the study cohort with an age-matched typical control group, showed similar instrument scores at baseline, but lower values in the DM1 cohort at follow-up. One limitation identified by the study authors was ‘survival bias,’ in that many older patients, typically those with greater disease severity, died between the baseline assessment and 9-year follow-up.

Data Utilization and Next Steps

The authors suggest that these data may support improved targeting by health care professionals and family members of DM1-linked declines in participation. Data from this study should also inform the design and evaluation of interventions to address this aspect of the burden of disease. Finally, the authors note the importance for future studies of taking into account both baseline participation status and use of shorter intervals between assessments.

References:

Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial.
Okkersen K, Jimenez-Moreno C, Wenninger S, Daidj F, Glennon J, Cumming S, Littleford R, Monckton DG, Lochmüller H, Catt M, Faber CG, Hapca A, Donnan PT, Gorman G, Bassez G, Schoser B, Knoop H, Treweek S, van Engelen BGM; OPTIMISTIC consortium.
Lancet Neurol. 2018 Aug;17(8):671-680. doi: 10.1016/S1474-4422(18)30203-5. Epub 2018 Jun 19.

Progressive decline in daily and social activities: A 9-year longitudinal study of participation in myotonic dystrophy type 1.
Raymond K, Levasseur M, Mathieu J, Gagnon C.
Arch Phys Med Rehabil. 2019 Mar 1. pii: S0003-9993(19)30148-0. doi: 10.1016/j.apmr.2019.01.022. [Epub ahead of print]