Therapeutic Classification: none assigned
Pharmacologic Classification: enzymes
Absorption: IV administration results in complete bioavailability.
Distribution: Well distributed to tissues.
Metabolism/Excretion: Unknown.
Half-Life: 5.4–6.6 min.
Rapidly Progressive Lysosomal Acid Lipase Deficiency Presenting within First 6 Mo of Age
- IV (Infants ): 1 mg/kg once weekly; may ↑ to 3 mg/kg once weekly if suboptimal response (poor growth, deteriorating biochemical markers, or persistent or worsening organomegaly) achieved; may ↑ to 5 mg/kg once weekly if suboptimal response continues to be achieved.
Lysosomal Acid Lipase Deficiency in Children and Adults
- IV (Adults and Children ): 1 mg/kg every other wk; may ↑ to 3 mg/kg every other wk if suboptimal response (poor growth, deteriorating biochemical markers [e.g., ALT, AST], and/or parameters of lipid metabolism [e.g., LDL cholesterol, triglycerides].
Rate: Infuse over 2 hr. Patients receiving >1 mg/kg or having experienced hypersensitivity reactions may require longer infusion. A 1-hr infusion may be used in patients who tolerate 1 mg/kg dose.
- Administer antihistamine, antipyretics, and/or corticosteroids prior to infusion, if required, to prevent hypersensitivity reactions.
- Administer in a health care setting with appropriate medical monitoring and supportive measures (cardiopulmonary resuscitation equipment) available during administration.
- If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue immediately and initiate appropriate medical treatment (epinephrine). Consider the risks and benefits of readministering following severe hypersensitivity reactions.
- Intermittent Infusion: Remove solution vials from refrigerator and allow to reach room temperature. Follow recommended calculations per manufacture guidelines.Diluent: Dilute with 0.9% NaCl. For 1 mg/kg dose, dilute each mL for 1–2.9 kg in 4 mL, 3–5.9 kg in 6 mL, 6–10.9 kg in 10 mL, 11–24.9 kg in 25 mL, 25–49.9 kg in 50 mL, 50–99.9 kg in 100 mL, and 100–120.9 kg in 250 mL. For 3 mg/kg dose, dilute each mL for 1–2.9 kg in 8 mL, 3–5.9 kg in 12 mL, 6–10.9 kg in 25 mL, 11–24.9 kg in 50 mL, 25–49.9 kg in 100 mL, 50–99.9 kg in 250 mL, and 100–120.9 kg in 500 mL. For 5 mg/kg dose,dilute each mL for 1–2.9 kg in 12 mL, 3–5.9 kg in 20 mL, 6–10.9 kg in 50 mL, 11–24.9 kg in 150 mL, 25–49.9 kg in 250 mL, 50–99.9 kg in 500 mL, and 100–120.9 kg in 600 mL. Concentration: 0.1–1.5 mg/mL. Mix gently by inversion; do not shake. Solution is clear to slightly opalescent, colorless to slightly colored, and may contain thin, translucent particles or fibers. Do not use solutions that are cloudy or contain other particulate matter. Discard unused portions of vials. Administer immediately after dilution; diluted solution may be stored up to 24 hr in refrigerator. Infuse via in-line low-protein-binding 0.2 micron filter through a low-protein-binding infusion set.