- Long-term enzyme replacement therapy for pediatric and adult patients with confirmed diagnosis of Type1 Gaucher disease resulting in one or more of the following conditions:
3. Bone disease
- Refrigerate vials. Warm vials to room temperature before reconstitution.
- Reconstituted product stable 12 hours at room temperature or refrigerated.
- Diluted in normal saline, stable 24 hours refrigerated.
- 200 units Vial: Reconstitute with 5.1 mL sterile water for injection for a 40 units/mL solution.
- 400 units Vial: Reconstitute with 10.2 mL sterile water for injection for a 40 units/mL solution.
- Do not shake vial. Gently swirl each vial to mix solution.
- Do not use any vials exhibiting opaque particles or discoloration.
- Solutions compatible: 0.9% NaCl
(For approved routes of administration by nursing personnel, refer to Policy for the Administration of Intravenous Medications.)
|IV Intermittent Infusion||
Yes DO NOT SHAKE VIAL OR BAG
Usual dilution: to a total volume of 100-200 mL with normal saline
Infusion time: 1-2 hours (or 0.5-1 unit/kg/minute)
|IV Continuous Infusion||No|
(For neonatal dosages, refer to Neonatal IV Drug Manual.)
- Initial dose: Range 2.5 units/kg 3x/week to 60 units/kg once every 2 weeks IV
- Initial dose and frequency depend on disease severity.
- Dosage adjustments based on individual patient response/ therapeutic goals.
- Dosage administered in individual infusions may be adjusted to utilize full vials as long as the monthly administered dosage remains substantially unaltered. Low toxicity and extended time course of response allows for small dosage adjustments to be made to avoid discarding partially used vial.
- Experience with doses up to 240 units/kg every 2 weeks reported with no obvious toxicity.
- Adverse effects associated with administration and increase in frequency; discomfort, pruritus, burning, swelling or sterile abscess at site of venipuncture
- Hypersensitivity; pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis, hypotension.
- Other; nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, tachycardia, transient peripheral edema
- Slight flocculation (translucent fibers) occurs occasionally after dilution.
- Agitation of the reconstituted or diluted bag may increase the chance of precipitation.
- Filter diluted solution through an in-line low protein binding 0.2 or 0.22 micron filter
- Infusion should be discontinued if formation of precipitate is observed.