Fabry disease
Conditions
Interventions
Case series:No
Sponsors
Beijing Children's Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
8 Years to 18 Years
Inclusion criteria
Inclusion criteria: Children aged 8 to 18 years with Fabry's disease who were diagnosed in Beijing Children's Hospital and agreed to be treated with agatase ß. The diagnostic criteria of Fabry disease were based on the Consensus of Chinese Fabry Disease diagnosis and Treatment experts (2021 edition) : a -gal A activity was seriously decreased or lost in male patients; Female patients need to be diagnosed simultaneously with genetic testing, substrate and derivative levels.
Exclusion criteria
Exclusion criteria: Children diagnosed with Fabry's disease but without symptoms, requiring regular follow-up but temporarily without further drug intervention; Children who refused to use agatase ß after diagnosis of Fabry's disease
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| survival rate in 1 year after intervention;Hospitalization rate of renal complications in 1 year after intervention; | — |
Secondary
| Measure | Time frame |
|---|---|
| new organ injury rate in 1 year after intervention;improvement rate of existing organ damage in 1 year after intervention; | — |
Countries
China
Contacts
Public ContactQian Suyun
Beijing Children's Hospital, Capital Medical Unive
Outcome results
None listed