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Abstract：
The prevalence and impact of symptoms affecting individuals with pediatric forms of myotonic dystrophy type-1 (DM1) are not well understood.
Patients from the United States, Canada, and Sweden completed a survey that investigated 20 themes associated with pediatric-onset DM1. Participants reported the prevalence and importance of each theme affecting their lives. Surveys from participants were matched with surveys from their caregivers for additional analysis.
The most prevalent symptomatic themes included problems with hands or fingers (79%) and gastrointestinal issues (75%). Problems with urinary/bowel control and gastrointestinal issues were reported to have the greatest impact on patients\' lives. Responses from participants and their caregivers had varying levels of agreement among symptomatic themes.
Many symptoms have meaningful impact on disease burden. The highest levels of agreement between caregivers and individuals with pediatric forms of myotonic dystrophy were found for physical activity themes.
Patients from the United States, Canada, and Sweden completed a survey that investigated 20 themes associated with pediatric-onset DM1. Participants reported the prevalence and importance of each theme affecting their lives. Surveys from participants were matched with surveys from their caregivers for additional analysis.
The most prevalent symptomatic themes included problems with hands or fingers (79%) and gastrointestinal issues (75%). Problems with urinary/bowel control and gastrointestinal issues were reported to have the greatest impact on patients\' lives. Responses from participants and their caregivers had varying levels of agreement among symptomatic themes.
Many symptoms have meaningful impact on disease burden. The highest levels of agreement between caregivers and individuals with pediatric forms of myotonic dystrophy were found for physical activity themes.
摘要：
影响儿童强直性肌营养不良1型（DM1）患者的症状的患病率和影响尚不清楚。
来自美国的患者，加拿大,瑞典完成了一项调查，调查了20个与儿童发病DM1相关的主题。参与者报告了影响他们生活的每个主题的普遍性和重要性。参与者的调查与他们的照顾者的调查相匹配，以进行额外的分析。
最普遍的症状主题包括手或手指问题（79％）和胃肠道问题（75％）。据报道，泌尿/肠道控制问题和胃肠道问题对患者生活影响最大。参与者及其护理人员的反应在症状主题之间有不同程度的共识。
许多症状对疾病负担具有有意义的影响。对于身体活动主题，护理人员与患有小儿强直性肌营养不良症的个体之间的最高共识。
来自美国的患者，加拿大,瑞典完成了一项调查，调查了20个与儿童发病DM1相关的主题。参与者报告了影响他们生活的每个主题的普遍性和重要性。参与者的调查与他们的照顾者的调查相匹配，以进行额外的分析。
最普遍的症状主题包括手或手指问题（79％）和胃肠道问题（75％）。据报道，泌尿/肠道控制问题和胃肠道问题对患者生活影响最大。参与者及其护理人员的反应在症状主题之间有不同程度的共识。
许多症状对疾病负担具有有意义的影响。对于身体活动主题，护理人员与患有小儿强直性肌营养不良症的个体之间的最高共识。
