Usual Adult Dose for Mucopolysaccharidosis Type I

0.58 mg/kg IV once a week

Comments:

• Clinical studies did not include patients 65 years and older and it is therefore unknown if they respond differently than younger patients.

Uses: For the treatment of Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie forms who have moderate to severe symptoms; risk and benefits of treating mildly affected patients with the Scheie form have not been established

Usual Pediatric Dose for Mucopolysaccharidosis Type I

6 months or older: 0.58 mg/kg IV once a week

Uses: For the treatment of Hurler and Hurler-Scheie forms of MPS I and for patients with the Scheie forms who have moderate to severe symptoms; risk and benefits of treating mildly affected patients with the Scheie form have not been established

Renal Dose Adjustments

Data not available

Liver Dose Adjustments

Data not available

Precautions

US BOXED WARNING:

• RISK OF ANAPHYLAXIS: Life-threatening anaphylactic reactions have occurred in some patients during infusions. Therefore, appropriate medical support should be readily available when this drug is infused. Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring.

Safety and efficacy have not been established in patients younger than 6 months.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration Advice:

• For IV infusion only
• Pretreatment is recommended 60 minutes prior to start of infusion and may include antipyretics and/or antihistamines.
• If an infusion reaction occurs despite pretreatment, decreasing the infusion rate, temporarily stopping the infusion, or administering additional antipyretics and/or antihistamines may improve the symptoms.
• Have appropriate medical support and monitoring measures readily available during infusion. If used during sleep, have supplemental oxygen or continuous positive airway pressure readily available during infusion. Some patients may require prolonged observation, based on individual patient needs.
• Prepare using low protein binding containers and administer diluted solution using a low protein binding infusion set with an in-line, low-protein-binding 0.2 micrometer filter; do not administer with other products in infusion tubing.
• Infuse over about 3 to 4 hours; initial infusion rate of 10 mcg/kg/hr may be incrementally increased every 15 minutes during first hour as tolerated, to a maximum infusion rate of 200 mcg/kg/hr maintained for the remainder of the infusion (2 to 3 hours).

For patients weighing 20 kg or less (total infusion volume of 100 mL):

• Start infusion at 2 mL/hr for 15 minutes (10 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 4 mL/hr for 15 minutes (20 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 8 mL/hr for 15 minutes (50 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 16 mL/hr for 15 minutes (100 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 32 mL/hr for about 3 hours (200 mcg/kg/hr) for remainder of infusion.

For patients weighing greater than 20 kg (total infusion volume of 250 mL):

• Start infusion at 5 mL/hr for 15 minutes (10 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 10 mL/hr for 15 minutes (20 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 20 mL/hr for 15 minutes (50 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 40 mL/hr for 15 minutes (100 mcg/kg/hr).
• Obtain vital signs; if stable, increase rate to 80 mL/hr for about 3 hours (200 mcg/kg/hr) for remainder infusion.

For patients with underlying cardiac or respiratory compromise and weight up to 30 kg:

• Consider diluting in 100 mL and administering at a decreased infusion rate.

Storage Requirements:

• Store vials at 2C to 8C (36F to 46F); protect from light; do not freeze or shake.
• Use diluted solution immediately; if cannot use immediately, may store for up to 36 hours at 2C to 8C (36F to 46F).

Reconstitution/Preparation Techniques:

• The manufacturer product information should be consulted.
• Final volume for patients weighing 20 kg or less: 100 mL
• Final volume for patients weighing more than 20 kg: 250 mL

IV Compatibility:

• Compatible with 0.9% Sodium Chloride Injection, USP

General:

• This drug has been shown to improve walking capacity and pulmonary function; it has not been evaluated for effects on the CNS manifestations of the disorder.

Monitoring:

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

Patient advice:

• Seek medical attention if you experience any adverse reactions, including allergic reactions and anaphylaxis.
• You are encouraged to participate in the MPS I registry and should visit MPSIregistry.com for more information.