Gaucher Disease Clinical Trial
Official title:
Assiut University Children 's Hospital
clinical audit on managment of gausher disease in children
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.
;
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