Dysphagia in Oculopharyngeal Muscular Dystrophy. Evaluation, Endoscopic Examination of Swallowing, Treatment and Long Term Follow up.
试验速览
- 阶段
- 1 期
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- Dysphagia, quality of life. Proper intervention timing in OPMD based on knowledge about the natural history of the feeding difficulty.
研究概览
简要总结
The investigators aimed to review the natural history of dysphagia and dysphonia in OPMD in order to identify the best candidates and the proper timing to perform dysphagia alleviating procedures in both heterozygote and homozygote patients from the large pool of cases with this disease in Israel.
详细描述
Clinical evaluation of patients suspected of having OPMD. Genetic confirmatory tests (diagnostic DNA test) at the genetic unit in Afula Hospital. Clinical follow-up with endoscopic fiber optic evaluation of swallowing. Pre-operative assessment. Crycopharyngeal myotomy intervention in selected patients. Nutrition follow-up.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •heterozygote and homozygote OPMD patients
排除标准
- 未提供
研究组 & 干预措施
Severe dysphagia
干预措施: Upper esophageal sphincter myotomy (Procedure)
Mild dysphagia
结局指标
主要结局
Dysphagia, quality of life. Proper intervention timing in OPMD based on knowledge about the natural history of the feeding difficulty.
时间窗: at least 3 years
Dysphagia, quality of life. Proper intervention timing in OPMD patients is very important and must be based on deep knowledge about the natural history of the feeding difficulty.
次要结局
未报告次要终点