title: Five-year study of quality of life in myotonic dystrophy.
abstract: Background - Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy in adults. There is a complete lack of studies that assessed quality of life (QoL) trajectory during time in DM1 cohorts. Aim - To analyze changes of QoL in patients with DM1 during a 5-year follow-up period and to assess responsiveness of the SF-36 questionnaire. Patients and Method - At the baseline, this study comprised 84 DM1 patients, of whom 62 were retested after the mean period of 64.2 ± 3.9 months. Severity of muscular weakness was assessed using the Muscular Impairment Rating Scale (MIRS). Patients completed Serbian version of the SF-36 questionnaire as a measure of health-related QoL. Results - After 5 years, MIRS score of our DM1 patients showed significant progression of 0.5 grade (P < 0.01). All mental subdomains, role physical, and total SF-36 scores significantly improved after 5 years (P < 0.01). Unexpectedly, worsening of muscular weakness from mild to severe was in association with improvement of QoL. Conclusion - QoL improved in our cohort of DM1 patients during a 5-year period despite the progression of the disease. SF-36 should be used with caution as a patient-reported outcome measure in DM1 clinical trials.
authors.affiliation: Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia. stojanperic@gmail.com.
authors.forename: S
authors.initials: S
authors.lastname: Peric
authors.affiliation: Department of Pathophysiology, Faculty of Medicine, University of Banja Luka, Banja Luka, Republic of Srpska, Bosnia and Herzegovina.
authors.forename: M
authors.initials: M
authors.lastname: Vujnic
authors.affiliation: Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia.
authors.forename: V
authors.initials: V
authors.lastname: Dobricic
authors.affiliation: Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia.
authors.forename: A
authors.initials: A
authors.lastname: Marjanovic
authors.affiliation: Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia.
authors.forename: I
authors.initials: I
authors.lastname: Basta
authors.affiliation: Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia.|Institute of Human Genetics, School of Medicine, University of Belgrade, Belgrade, Serbia.
authors.forename: I
authors.initials: I
authors.lastname: Novakovic
authors.affiliation: Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia.
authors.forename: D
authors.initials: D
authors.lastname: Lavrnic
authors.affiliation: Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia.
authors.forename: V
authors.initials: V
authors.lastname: Rakocevic-Stojanovic
mesh_terms: D000328:Adult; D018450:Disease Progression; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D009223:Myotonic Dystrophy / Q000503:physiopathology*; D011788:Quality of Life
keywords: SF-36; follow-up; myotonic dystrophy type 1; quality of life; responsiveness