Recombinant human lysosomal acid lipase enzyme replacement
Sebelipase alfa
Brand names: Kanuma
Adult dose
Dose: 1 mg/kg IV every 2 weeks (rapidly progressing infants: 3 mg/kg/week)
Route: IV
Frequency: q2w
Clinical pearls
- Lysosomal acid lipase deficiency (LAL-D / Wolman / CESD)
- Specialist metabolic
Contraindications
- Life-threatening hypersensitivity (egg/component)
Side effects
- Infusion reactions
- Anaphylaxis (rare)
- GI upset
- Headache
Monitoring
- Lipids
- LFTs
- Infusion reactions
Reference: BNF; NICE HST4; SmPC; https://bnf.nice.org.uk/drugs/sebelipase-alfa/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Calculators
- Weight-Based Levothyroxine Dose Calculator · Thyroid
- Ranson Criteria (Pancreatitis) · Pancreatitis
- Tumor Lysis Syndrome Risk (Cairo-Bishop) · Oncological Emergency
- Cryoprecipitate Dose Calculator for Fibrinogen Replacement · Transfusion Medicine
- Free Water Deficit in Hypernatraemia · Fluid / Electrolytes
- Urine Anion Gap · Acid-Base
Pathways
- Sepsis Screening and Sepsis Six · UK Sepsis Trust; NICE NG51; Surviving Sepsis Campaign 2021
- Unintentional Weight Loss Workup · NICE NG12; BSG
- Chronic Fatigue Workup · NICE NG206; BMJ Best Practice
- Lymphadenopathy Workup · NICE NG12; BMJ Best Practice
- Pre-op Medical Clearance · NICE NG45; ESC 2022
- Secondary Hypertension Workup · NICE NG136; ESH 2023