Non-invasive Assessment of Liver Fibrosis in a French Cohort of Pediatric Patients With Type III Glycogen Storage Disease: Current State and Perspectives
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Liver elastography values
研究概览
简要总结
Patients with type III glycogen storage disease (GSDIII) can develop liver fibrosis, which can be complicated by liver failure or even hepatocellular carcinoma. Since the beginning of the 21st century, non-invasive techniques for assessing fibrosis, such as liver elastography, have been developed. These techniques often make it possible to avoid liver biopsies during patient follow-up and have already been validated in the management of several diseases in adults. These techniques are also beginning to be recommended for monitoring certain chronic liver diseases in children.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 1 Month 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Minors: from birth to 17 years
- •Adults: 18 to 21 years
- •Subjects being monitored for type III glycogen storage disease and having had at least one liver elastography measurement during their follow-up.
排除标准
- •- Patients monitored for type III glycogen storage disease but who had never undergone liver elastography during their follow-up.
结局指标
主要结局
Liver elastography values
时间窗: 1 hour after consultation
Liver fibrosis is classified into four stages, from 0 to 4: * F0 corresponds to the absence of fibrosis * F4 corresponds to established cirrhosis.
次要结局
未报告次要终点