ERT |
Indications: |
Patients with chronic neuronopathic (type 3) GD*. |
Siblings of patients with chronic neuronopathic GD who are proven to have GD. |
Patients with the following high-risk genotypes: |
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Onset of severe systemic GD at ≤2 years of age. |
Dose: |
60 units/kg every 2 weeksΔ, initiated as soon as possible after diagnosis or identification. |
Duration: |
Treatment with 60 units/kg every 2 weeks should be continued until the patient attains adulthood and clearly has mild GD and stable neurologic involvement. At this point a dose reduction (to as low as 30 units/kg every 2 weeks) may be considered. |
Splenectomy: |
Total splenectomy should be avoided if at all possible. Partial, rather than total, splenectomy should be considered if splenectomy is required in an emergency situation. |
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