Inborn errors of metabolism:
Indications for KANUMA:
Treatment of lysosomal acid lipase (LAL) deficiency.
Adults and Children:
<1 month: not established. Give as IV infusion over ≥2hrs; consider further prolonging infusion time for the 3mg/kg dose or if a hypersensitivity reaction occurs. May consider a 1hr infusion for the 1mg/kg dose in those who tolerate the infusion. ≥1 month: 1mg/kg once every other week. If rapidly progressive within first 6 months of life: initially 1mg/kg once weekly; increase to 3mg/kg once weekly if optimal response not achieved. May pre-treat with antipyretics and/or antihistamines.
Monitor for hypersensitivity reactions during and after infusion; interrupt or lower infusion rate based on severity. Discontinue immediately and treat if severe hypersensitivity including anaphylaxis occurs. Eggs or egg products allergy. Pregnancy. Nursing mothers.
Hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme.
Diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, urticaria, headache, oropharyngeal pain, asthenia, constipation, nausea.