Assiut University Children 's Hospital
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 1
- 主要终点
- evaluate the management of Gausher disease patient under enzyme replacement therapy in Assuit University Children's Hospital and compare our policy for management with International guideline of management of Gausher disease in children (8)
研究概览
简要总结
clinical audit on managment of gausher disease in children
详细描述
The main aim of study is to evaluate the management of Gausher disease patient under enzyme replacement therapy in Assuit University Children's Hospital and compare our policy for management with International guideline of management of Gausher disease in children (8)
- Type of study : clinical audit
- Inclusion criteria: All cases with confirmed diagnosis of Gaucher disease type 1 and type 3 receiving enzyme replacement therapy at Assuit University Children's Hospital.
- Site of study :Assuit University children's Hospital Methodiological
- Exclusion criteria 3-New diagnosed cases not on ERT. 2-other lipid storage disease.
- Patient& method The study will include 42 diagnosed Gaucher disease patient under enzyme replacement therapy attend the hematology unit in Assuit University Children's Hospital in one year Reviewing the proposal will be carried out before starting via the ethical committee of Assuit Faculty of Medicine.
- Ethical consideration The aim of the study will be explained to each patient before beginning of the process.
We will compare our policy in management of Gausher disease with International guideline of management of gausher disease
1-cases of Gaucher type 2.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Other
入排标准
- 年龄范围
- 1 Month 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All cases with confirmed diagnosis of Gaucher disease type 1 and type 3 receiving enzyme replacement therapy at Assuit University Children's Hospital.
排除标准
- •1-cases of Gaucher type
- •2-other lipid storage disease. 3-New diagnosed cases not on ERT.
结局指标
主要结局
evaluate the management of Gausher disease patient under enzyme replacement therapy in Assuit University Children's Hospital and compare our policy for management with International guideline of management of Gausher disease in children (8)
时间窗: one year
• Type of study : clinical audit
次要结局
未报告次要终点
研究者
Yasmeen Abd Elbaset Hamzaa
Assiut University
Assiut University