Patient-Reported Impact of Symptoms in Myotonic Dystrophy Type 2 (PRISM-2)
Patient-Reported Impact of Symptoms in Myotonic Dystrophy Type 2 (PRISM-2)
复制标题
DOI：
10.1212/wnl.0000000000002225
复制
发表时间：
2015-12-15
期刊：
影响因子：
9.9
通讯作者：
Moxley, Richard T., III
中科院分区：
文献类型：
--
作者：
Heatwole, Chad;Johnson, Nicholas;Moxley, Richard T., III
目的：确定强直性肌营养不良2型（DM2）的频率和相对重要性，最影响生活的症状，并确定与这些symptoms.Methods的因素，有最强的关联：我们进行了一项横断面研究，从国家登记处的DM2患者与DM2成人患者的患病率和相对重要性进行评估的310个症状和21个症状的主题。参与者的反应进行了比较，年龄类别，性别，教育程度，就业状况，和持续时间的symptoms.Results：症状的主题，在DM2的患病率最高的是无法做活动（94.4%），限制与流动性或行走（89.2%），髋关节，大腿，或膝关节无力（89.2%），疲劳（89.2%），肌强直（82.6%）。参与者将无法进行活动和疲劳确定为对他们的生活产生最大整体影响的症状主题。失业、症状持续时间较长和受教育程度较低与所有症状主题的平均患病率较高相关（p < 0.01）。失业、症状持续时间较长、性别和年龄增加与DM 2患者所有症状主题的平均效应较高相关（p < 0.01）。结论：DM 2患者的生活受到各种症状的影响。这些症状在该人群中具有不同的显著性和患病率水平，并且在不同人口统计学类别的DM 2亚组中有所不同。
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.