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Clinical Audit of Managment of Gausher Disease in Children

Conditions
Gaucher Disease
Interventions
Diagnostic Test: gausher disease
Registration Number
NCT03590548
Lead Sponsor
Yasmeen Abd Elbaset Hamzaa
Brief Summary

clinical audit on managment of gausher disease in children

Detailed Description

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.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
1
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.
Exclusion Criteria
  • 1-cases of Gaucher type 2. 2-other lipid storage disease. 3-New diagnosed cases not on ERT.

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
gausher disease in childrengausher disease• 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.
Primary Outcome Measures
NameTimeMethod
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)one year

• Type of study : clinical audit

Secondary Outcome Measures
NameTimeMethod
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