title: Patient-Reported Impact of Symptoms in Myotonic Dystrophy Type 2 (PRISM-2).
abstract: 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.
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA. Chad_Heatwole@urmc.rochester.edu.
authors.forename: Chad
authors.initials: C
authors.lastname: Heatwole
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Nicholas
authors.initials: N
authors.lastname: Johnson
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Rita
authors.initials: R
authors.lastname: Bode
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Jeanne
authors.initials: J
authors.lastname: Dekdebrun
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Nuran
authors.initials: N
authors.lastname: Dilek
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: James E
authors.initials: JE
authors.lastname: Hilbert
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Elizabeth
authors.initials: E
authors.lastname: Luebbe
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: William
authors.initials: W
authors.lastname: Martens
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Michael P
authors.initials: MP
authors.lastname: McDermott
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Christine
authors.initials: C
authors.lastname: Quinn
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Nan
authors.initials: N
authors.lastname: Rothrock
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Charles
authors.initials: C
authors.lastname: Thornton
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Barbara G
authors.initials: BG
authors.lastname: Vickrey
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: David
authors.initials: D
authors.lastname: Victorson
authors.affiliation: From the Departments of Neurology (C.H., J.D., N.D., J.E.H., E.L., W.M., M.P.M., C.T., R.T.M.) and Biostatistics and Computational Biology (M.P.M.), and James P. Wilmot Cancer Institute (C.Q.), University of Rochester Medical Center, Rochester, NY; University of Utah (N.J.), Salt Lake City; Northwestern University Feinberg School of Medicine (R.B., N.R., D.V.), Chicago, IL; David Geffen School of Medicine (B.G.V.), UCLA Medical Center, Los Angeles; and Greater Los Angeles VA HealthCare System (B.G.V.), CA.
authors.forename: Richard T
authors.initials: RT
authors.lastname: Moxley
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D003430:Cross-Sectional Studies; D005221:Fatigue / Q000175:diagnosis / Q000209:etiology; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D051346:Mobility Limitation; D009223:Myotonic Dystrophy / Q000150:complications* / Q000175:diagnosis*; D012042:Registries; D057566:Self Report; D018434:Sickness Impact Profile; D012893:Sleep Wake Disorders / Q000175:diagnosis / Q000209:etiology