Recombinant N-acetylgalactosamine-6-sulfatase (enzyme replacement)
Elosulfase alfa
Brand names: Vimizim
Adult dose
Dose: 2mg/kg IV once weekly
Route: IV infusion
Frequency: Weekly
Clinical pearls
- MPS IVA (Morquio A syndrome) — NICE HST2
- UK Lysosomal Storage Disorder Service supervision
- Pre-medication with antihistamine ± corticosteroid often required
Contraindications
- Life-threatening hypersensitivity
Side effects
- Infusion reactions
- Anaphylaxis
- Headache
- Vomiting
- Pyrexia
- Antibody formation
Monitoring
- Disease progression (urinary keratan sulfate)
- Functional outcomes (6MWT)
- Infusion reactions
- ADAs
Reference: BNF; NICE HST2; UK LSD National Service; BIMDG; SmPC; https://bnf.nice.org.uk/drugs/elosulfase-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
- Cryoprecipitate Dose Calculator for Fibrinogen Replacement · Transfusion Medicine
- Free Water Deficit in Hypernatraemia · Fluid / Electrolytes
- Potassium Deficit Calculator · Electrolytes
- Indications for Renal Replacement Therapy (RRT) in AKI · Treatment Decision
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