Disease Burden and Living Situation of Patients With Facioscapulohumeral Muscular Dystrophy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 300
- 试验地点
- 1
- 主要终点
- EQ-5D
研究概览
简要总结
The goal of this observational study is to learn the patient journey, disease burden, living situation, quality of life and etc. in patients with Facioscapulohumeral Muscular Dystrophy in all sex/gender and age groups. The main questions it aims to answer are:
- What's the patient journey of patients with Facioscapulohumeral Muscular Dystrophy?
- How does Facioscapulohumeral Muscular Dystrophy burden the patients?
- How's the quality of life of patients with Facioscapulohumeral Muscular Dystrophy?
Patients with Facioscapulohumeral Muscular Dystrophy will be asked to:
- fill a questionnaire.
- be interviewed and answer questions about their illness experience, economic burden, quality of life and etc.
Medical experts on Facioscapulohumeral Muscular Dystrophy will be asked to be interviewed and answer questions about understanding on the disease and the patients.
详细描述
Study Objective This observational study aims to comprehensively understand the patient journey, disease burden, living situation, and quality of life in individuals diagnosed with Facioscapulohumeral Muscular Dystrophy (FSHD). The study encompasses all sex/gender and age groups to provide a holistic view of the disease's impact.
Research Questions
The study focuses on answering the following key questions:
- What is the patient journey of individuals with Facioscapulohumeral Muscular Dystrophy?
- How does Facioscapulohumeral Muscular Dystrophy burden the patients?
- What is the quality of life of patients with Facioscapulohumeral Muscular Dystrophy?
Methodology
研究设计
- 研究类型
- Observational
- 观察模型
- Ecologic Or Community
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Having been diagnosed as Facioscapulohumeral Muscular Dystrophy
排除标准
- 未提供
结局指标
主要结局
EQ-5D
时间窗: 1 year
EQ-5D questionnaires will be used to measure the quality of life of the patients. The questionnaires consist of two main parts: Descriptive System and Visual Analog Scale. Descriptive System includes Mobility, Self-Care, Usual Activities, Pain/Discomfort and Anxiety/Depression, each with 5 levels for adults and 3 levels for children. Visual Analog Scalerequires patients to rate their overall health on a scale from 0 to 100, where 0 represents "the worst health you can imagine" and 100 represents "the best health you can imagine." In the end, an index value will be calculated that reflects the patient's overall health status.
Anxiety Level
时间窗: 1 year
The Self-Rating Anxiety Scale will be used to measure the anxiety level of the patients. It consists of 20 items, each describing a common symptom of anxiety. Each item is rated on a 4-point Likert scale. The total score ranges from 20 to 80. In doing so, the anxiety level of the patients can be screened.
Economic Burden
时间窗: 1 year
Questions will designed to explore the economic burden of the patients. Questions include direct and indirect medical costs over the past year, annual incomes, accumulated treatment costs and debts. Each questions include 7 levels. Besides, whether they have public health insurance and commercial insurance will be asked to discover how much the costs can be covered.
Disease Burden
时间窗: 1 year
Questions will designed to explore the disease burden of the patients. Questions include the influence of the disease on respiratory system, dry eyes, whistle, deglutition, speaking and walking. Each question contains 5 levels.
次要结局
未报告次要终点
研究者
Wenjing Ji
Associate Professor
Health Science Center of Xi'an Jiaotong University