TY - JOUR
T1 - Patient-Reported Impact of Symptoms in Myotonic Dystrophy Type 2 (PRISM-2)
AU - Heatwole, Chad
AU - Johnson, Nicholas
AU - Bode, Rita
AU - Dekdebrun, Jeanne
AU - Dilek, Nuran
AU - Hilbert, James E.
AU - Luebbe, Elizabeth
AU - Martens, William
AU - McDermott, Michael P.
AU - Quinn, Christine
AU - Rothrock, Nan
AU - Thornton, Charles
AU - Vickrey, Barbara G.
AU - Victorson, David
AU - Moxley, Richard T.
N1 - Publisher Copyright:
© 2015 American Academy of Neurology.
PY - 2015/12/15
Y1 - 2015/12/15
N2 - Objective: To determine the frequency and relative importance of the most life-affecting symptoms in myotonic dystrophy type 2 (DM2) and to identify the factors that have the strongest association with these symptoms. Methods: We conducted a cross-sectional study of adult patients with DM2 from a National Registry of DM2 Patients to assess the prevalence and relative importance of 310 symptoms and 21 symptomatic themes. Participant responses were compared by age categories, sex, educational attainment, employment status, and duration of symptoms. Results: The symptomatic themes with the highest prevalence in DM2 were the inability to do activities (94.4%), limitations with mobility or walking (89.2%), hip, thigh, or knee weakness (89.2%), fatigue (89.2%), and myotonia (82.6%). Participants identified the inability to do activities and fatigue as the symptomatic themes that have the greatest overall effect on their lives. Unemployment, a longer duration of symptoms, and less education were associated with a higher average prevalence of all symptomatic themes (p < 0.01). Unemployment, a longer duration of symptoms, sex, and increased age were associated with a higher average effect of all symptomatic themes among patients with DM2 (p < 0.01). Conclusions: The lives of patients with DM2 are affected by a variety of symptoms. These symptoms have different levels of significance and prevalence in this population and vary across DM2 subgroups in different demographic categories.
AB - Objective: To determine the frequency and relative importance of the most life-affecting symptoms in myotonic dystrophy type 2 (DM2) and to identify the factors that have the strongest association with these symptoms. Methods: We conducted a cross-sectional study of adult patients with DM2 from a National Registry of DM2 Patients to assess the prevalence and relative importance of 310 symptoms and 21 symptomatic themes. Participant responses were compared by age categories, sex, educational attainment, employment status, and duration of symptoms. Results: The symptomatic themes with the highest prevalence in DM2 were the inability to do activities (94.4%), limitations with mobility or walking (89.2%), hip, thigh, or knee weakness (89.2%), fatigue (89.2%), and myotonia (82.6%). Participants identified the inability to do activities and fatigue as the symptomatic themes that have the greatest overall effect on their lives. Unemployment, a longer duration of symptoms, and less education were associated with a higher average prevalence of all symptomatic themes (p < 0.01). Unemployment, a longer duration of symptoms, sex, and increased age were associated with a higher average effect of all symptomatic themes among patients with DM2 (p < 0.01). Conclusions: The lives of patients with DM2 are affected by a variety of symptoms. These symptoms have different levels of significance and prevalence in this population and vary across DM2 subgroups in different demographic categories.
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U2 - 10.1212/WNL.0000000000002225
DO - 10.1212/WNL.0000000000002225
M3 - Article
C2 - 26581301
AN - SCOPUS:84951264075
SN - 0028-3878
VL - 85
SP - 2136
EP - 2146
JO - Neurology
JF - Neurology
IS - 24
ER -