Usual Adult Dose for Gaucher Disease

Initial dose: May range from 2.5 units/kg 3 times a week IV infusion to 60 units/kg IV infusion once every 2 weeks

Comments:

• The majority of data available is for the 60 units/kg every 2 weeks dose.
• Disease severity may dictate that treatment be initiated at a relatively high dose or relatively frequent administration.
• Dose adjustments should be made on an individual basis and may increase or decrease based on achievement of therapeutic goals as assessed by routine evaluations of the patient's clinical manifestations.

Use: For long-term enzyme replacement therapy in patients with a confirmed diagnosis of Type 1 Gaucher disease that results in 1 or more of the following conditions: anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly

Usual Pediatric Dose for Gaucher Disease

2 years or older:

• Initial dose: May range from 2.5 units/kg 3 times a week IV infusion to 60 units/kg IV infusion once every 2 weeks

Comments:

• The majority of data available is for the 60 units/kg every 2 weeks dose.
• Disease severity may dictate that treatment be initiated at a relatively high dose or relatively frequent administration.
• Dose adjustments should be made on an individual basis and may increase or decrease based on achievement of therapeutic goals as assessed by routine evaluations of the patient's clinical manifestations.

Use: For long-term enzyme replacement therapy in patients with a confirmed diagnosis of Type 1 Gaucher disease that results in 1 or more of the following conditions: anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

Safety and efficacy have not been established in patients younger than 2 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Administer via IV infusion over 1 to 2 hours.
• Diluted solution may be filtered through an in-line low protein-binding 0.2 micrometer filter during administration.

Storage requirements:

• Store vials at 2C to 8C (36F to 46F).
• Following reconstitution, this drug is stable for up to 12 hours when stored at room temperature at 25C (77F) or at 2C to 8C (36F to 46F)
• Use diluted solution immediately; if cannot use immediately, may be stored for up to 24 hours at 2C to 8C (36F to 46F).

Reconstitution/preparation techniques:

• The manufacturer product information should be consulted.

IV compatibility:

• Compatible with 0.9% sodium chloride injection, USP

Monitoring:

• Immunologic: Monitor patients periodically for IgG antibody formation during the first year of treatment.