Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 发起方
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- Evaluate Pulmonary Hypertension (PH) in Egyptian GD Type 3 Patients
研究概览
简要总结
all cases confirmed diagnosis of Gaucher disease type 3 recieving enzyme replacement therapy at Assiut university hospital
详细描述
evaluate cardiac involvement in a group of GD type 3 patients, evaluate pulmonary hypertension and valvular involvement, compare the findings of basic echo before the start of replacement therapy as well as with healthy controls, to assess the effects of enzyme replacement therpay on the cardiac function.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •No age limit
- •Gaucher disease type 3
- •Receiving enzyme replacement therapy
排除标准
- •Age18 years old
- •Cases of gaucher type 1 and 2
- •Other lipid storage disease
结局指标
主要结局
Evaluate Pulmonary Hypertension (PH) in Egyptian GD Type 3 Patients
时间窗: 6 months
To assess the presence and severity of pulmonary hypertension using echocardiography. Unit of Measure: mmHg (for systolic pulmonary artery pressure)
Evaluate Valvular Involvement in Egyptian GD Type 3 Patients
时间窗: 6 months
To assess the degree of valvular involvement, including stenosis or regurgitation, using echocardiography. Unit of Measure: Severity grading (mild, moderate, severe)
次要结局
未报告次要终点
研究者
Noha Osman
Lecturer
Assiut University