Peric S, Vujnic M, Dobricic V, Marjanovic A, Basta I, Novakovic I, Lavrnic D, Rakocevic-Stojanovic V
Neurology Clinic, Clinical Center of Serbia, School of Medicine, University of Belgrade, Belgrade, Serbia.
Department of Pathophysiology, Faculty of Medicine, University of Banja Luka, Banja Luka, Republic of Srpska, Bosnia and Herzegovina.
Acta Neurol Scand. 2016 Nov;134(5):346-351. doi: 10.1111/ane.12549. Epub 2015 Dec 21.
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.
背景 - 1型强直性肌营养不良症（DM1）是成人中最常见的肌肉营养不良症。目前完全缺乏对DM1队列中生活质量（QoL）轨迹随时间变化的研究。目的 - 分析DM1患者在5年随访期间QoL的变化，并评估SF-36问卷的反应性。患者与方法 - 在基线时，本研究纳入了84例DM1患者，其中62例在平均64.2±3.9个月后进行了重新测试。使用肌肉损伤评定量表（MIRS）评估肌肉无力的严重程度。患者完成塞尔维亚语版的SF-36问卷，作为健康相关QoL的一项指标。结果 - 5年后，我们的DM1患者的MIRS评分显示有0.5级的显著进展（P < 0.01）。5年后，所有精神亚领域、身体功能以及SF-36总分均有显著改善（P < 0.01）。出乎意料的是，肌肉无力从轻度到重度的恶化与QoL的改善相关。结论 - 在我们的DM1患者队列中，尽管疾病进展，但QoL在5年期间有所改善。在DM1临床试验中，作为患者报告的结局指标，使用SF-36时应谨慎。
