Clinical Audit of Managment of Gausher Disease in Children

Yasmeen Abd Elbaset Hamzaa

Status: Unknown

Conditions: Gaucher Disease

Treatments: Diagnostic Test: gausher disease

Study type: Observational

Funder types: Other

Identifiers

NCT03590548

egypt AssiutU

Details and patient eligibility

About

clinical audit on managment of gausher disease in children

Full 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.

Enrollment: 1 estimated patient

Sex: All

Ages: 1 month to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

1 participants in 1 patient group

gausher disease in children

Description:

• 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.

Treatment:

Diagnostic Test: gausher disease