Validation of a Questionnaire for Quality of Life Assessment in Patients With Transthyretin Amyloidosis in Argentina.
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 入组人数
- 70
- 试验地点
- 2
- 主要终点
- To develop and validate a structured and self-administered quality of life questionnaire in patients with hereditary amyloidosis
研究概览
简要总结
Prospective study of development and validation of a questionnaire on quality of life in patients with Hereditary Amyloidosis The questionnaire development process will include 4 medical specialists experts in Transthyretin Amyloidosis (2 neurologists, 1 cardiologist, 1 physician clinical/hepathologist), 1 psychologist; 1 social worker; and 3 patients (with confirmed Hereditary amyloidosis) members of ALAPA. (Local PAG)
Population and sample:
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •patients with confirmed ATTR (genetic test), older than 18 years old in stage I, II and III of the PND scale (polyneuropathy disability: I Sensory neuropathy ; II Motor symptoms, but with preserved ambulation; IIIa Requires 1 cane to ambulate; IIIb requires two canes/walker).
排除标准
- •patients who participated in the validation process of the questionnaire: patients who participated in the questionnaire construction process or adjustments to the questionnaire.
研究组 & 干预措施
Symptomatic patients with hereditary amyloidosis
结局指标
主要结局
To develop and validate a structured and self-administered quality of life questionnaire in patients with hereditary amyloidosis
时间窗: 6 months
To develop and validate a structured and self-administered questionnaire specific to covering physical, emotional and social aspects, to assess the impact of hereditary transthyretin amyloidosis on the quality of life of patients residing in Argentina.
次要结局
未报告次要终点
研究者
Fernando Torres
PhD
Hospital General de Niños Pedro de Elizalde