Treatment of lysosomal acid lipase (LAL) deficiency.
Being a recombinant form of LAL, it binds to cell surface receptors via glycans expressed on the protein and is subsequently internalized into lysosomes. It then catalyzes the lysosomal hydrolysis of cholesteryl esters and triglycerides to free cholesterol, glycerol, and free fatty acids.
Improved survival in infants; improved lipids in adults and children.
Absorption: IV administration results in complete bioavailability.
Distribution: Well distributed.
Metabolism and Excretion: Unknown.
Half-life: 5.4–6.6 min.
TIME/ACTION PROFILE (blood levels)
- Hypersensitivity to eggs.
Use Cautiously in:
- Geri: Older adults (safety and effectiveness not established);
- OB: Lactation: Pregnancy and lactation (safety not established);
- Pedi: Children <1 mo (safety and effectiveness not established).
Adverse Reactions/Side Effects
CNS: headache, anxiety
EENT: nasopharyngitis, rhinitis, sneezing
GI: diarrhea, oropharyngeal pain, vomiting, constipation, nausea
Metabolic: ↑ LDL cholesterol, ↑ triglycerides
Resp: cough, hypoxemia
Misc: HYPERSENSITIVITY REACTIONS (INCLUDING ANAPHYLAXIS, URTICARIA), fever
* CAPITALS indicate life-threatening.
Underline indicate most frequent.
Rapidly Progressive LAL Deficiency Presenting within First 6 Months of Age
IV Infants: 1 mg/kg once weekly; may ↑ to 3 mg/kg once weekly if suboptimal response achieved.
IV (Adults and Children): 1 mg/kg every other wk.
Solution for injection: 2 mg/mL
- Monitor for signs and symptoms of hypersensitivity reactions (chest discomfort, conjunctival injection, dyspnea, generalized and itchy rash, hyperemia, swelling of eyelids, rhinorrhea, severe respiratory distress, tachycardia, tachypnea, urticaria) during and after infusion. Anaphylaxis has occurred as early as sixth infusion and as late as 1 year after treatment initiation. May pretreat with antipyretics, corticosteroids, and antihistamines. If reaction occurs, decrease infusion rate. If anaphylaxis occurs, discontinue infusion and initiate symptomatic treatment.
Lab Test Considerations:
May cause ↑ circulating LDL-cholesterol (LDL-C) and triglycerides.
- Deficient knowledge, related to medication regimen (Patient/Family/Teaching)
- Intermittent Infusion: Remove solution vials from refrigerator and allow to reach room temperature. Diluent: Dilute with 0.9% NaCl. For 1 mg/kg dose, dilute each mL for 1–10.9 kg in 10 mL, 11–24 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 1–10.9 kg in 25 mL, 11–24 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. Concentration: 0.1 mg/mL to 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. Administer immediately after dilution; diluted solution may be stored up to 24 hrs in refrigerator. Discard unused portions of vials. Infuse via in-line low-protein binding 0.2 micron filter through a low-protein binding infusion set.
- Rate: Infuse over at least 2 hr. Patients receiving 3 mg/kg dose may require longer infusion. A 1 hr infusion may be used in patients who tolerate 1 mg/kg dose.
Instruct parent or patient to notify health care professional immediately if signs and symptoms of hypersensitivity reaction (chest tightness; shortness of breath; wheezing; dizziness or faintness; or swelling of face, eyelids, or lips) or if cough or shortness of breath occurs.
- Improved survival in infants.
- Improved lipids in adults and children.
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