Specificities of Quality of Life and Influence of Participation on the Quality of Life of the Adult with Spinal Muscular Atrophy in France: a Cross-sectional Study
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 149
- 试验地点
- 2
- 主要终点
- Quality of life
研究概览
简要总结
Spinal muscular atrophy is a hereditary motorneuron disease caused by a mutation of the SMN1 gene, which is at the origin of a progressive limb and axial motor deficiency. It concerns 1200 individuals in France, including 700 adults in 2018. The main objective of this study is to assess the quality of life of SMA patients in France.
The secondary objectives are, in one hand, to compare the quality of life of SMA patients to a population of neuromuscular diseases patients. And on the other hand to evaluate the determinants of participation and the impact of participation on quality of life in adult SMA patients.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient with SMA type 1, 2, 3 or 4
- •≥ 18 years old
- •giving informed consent to participate to the study
- •patients from the study of Dany et al "Construction of a Quality of Life Questionnaire for slowly progressive neuromuscular disease" (2015)
排除标准
- •patients who do not complete ≥ 80% of the questionnaire)-
结局指标
主要结局
Quality of life
时间窗: At inclusion
Quality of life of adult patients with spinal muscular atrophy in France, assessed using the QOLNMD score
次要结局
未报告次要终点