Usual Adult Dose for Gaucher Disease

Treatment-naive:

• Initial dose: 60 units/kg IV every other week

Conversion from imiglucerase:

• For patients on a stable dose of imiglucerase: Begin at same units/kg dose IV every other week

Dose adjustments should be based on individual's therapeutic goals

Comments:

• Administer as an IV infusion over 60 to 120 minutes every other week; in adult patients, an initial infusion rate of 1.2 mL/minute should be used.
• After tolerability to infusion is established, the infusion rate may be increased, but should not exceed the maximum recommended infusion rate of 2.2 mL/minute; infusion should be delivered over a minimum or 60 minutes.

Use: For long-term enzyme replacement therapy (ERT) for patients with confirmed diagnosis of type 1 Gaucher disease

Usual Pediatric Dose for Gaucher Disease

4 years or older:
Treatment-naive: Initial dose: 60 units/kg IV every other week

Conversion from imiglucerase:

• For patients on a stable dose of imiglucerase: Begin with same units/kg dose IV every other week

Dose adjustments should be based on individual's therapeutic goals

Comments:

• Administer as an IV infusion over 60 to 120 minutes every other week; in adult patients, an initial infusion rate of 1.2 mL/minute should be used.
• After tolerability to infusion is established, the infusion rate may be increased, but should not exceed the maximum recommended infusion rate of 2.2 mL/minute; infusion should be delivered over a minimum or 60 minutes.

Use: For the treatment of patients with a confirmed diagnosis of Type 1 Gaucher disease.

Renal Dose Adjustments

No adjustment recommended

Liver Dose Adjustments

No adjustment recommended

Precautions

CONTRAINDICATIONS:

• None

Safety and efficacy have not been established in patients younger than 4 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration Advice:

• For IV infusion over a minimum of 60 minutes every other week
• Due to the potential for anaphylaxis, appropriate medical support should be readily available during administration

Adults:

• Initial infusion rate: 1.2 mL/minute
• Once tolerability is established, may increase rate
• Maximum infusion rate: 2 mL/minute

Pediatrics:

• Initial infusion rate: 1 mL/minute
• Once tolerability is established, may increase

Maximum infusion rate: 2 mL/min

Reconstitution/preparation techniques:

• Should be prepared under the supervision of a healthcare professional; consult product labeling for additional instructions
• Reconstitute each vial with 5.1 mL of Sterile Water for Injection to yield a concentration of 40 units/mL and an extractable volume of 5 mL; upon reconstitution, mix vials gently, do not shake
• Further dilute with 0.9% sodium chloride to a final volume of 100 or 200 mL
• Following reconstitution and dilution, administer via IV infusion; filter through an in-line low-protein-binding 0.2 micrometer filter

Storage Requirements:

• Store in refrigerator at 2C to 8C (36F to 46F); protect from light
• Reconstituted product: May store for up to 24 hours when refrigerated (2C to 8C [36F to 46F]) and protected from light OR up to 4 hours at 20C to 25C (68F to 77F) without protection from light
• Diluted product: May store for up to 24 hours at 2C to 8C (36F to 46F) when protected from light
• Storage of reconstituted product and diluted products should not exceed a total of 24 hours
• Do not freeze

Monitoring:

• Hypersensitivity: Signs/symptoms of allergic reactions and/or infusion-related reactions (during and after infusion)

Patient Advice:

• Patients and caregivers should understand that infusion-related and hypersensitivity reactions including anaphylaxis may occur with therapy.
• Patients and caregivers should be informed of the signs and symptoms of anaphylaxis and hypersensitivity reactions and should be instructed to seek medical care should signs and symptoms occur.