Laronidase

How it works

It is a recombinant form of human alpha-L-iduronidase that replaces the deficient enzyme, enabling lysosomal breakdown of accumulated glycosaminoglycans (dermatan and heparan sulphate).

Prescribing in practice

• Infusion-associated and hypersensitivity reactions can occur, so it is given as a controlled intravenous infusion with appropriate premedication and facilities to manage reactions.
• Slow the infusion rate or interrupt treatment if reactions develop, and observe the patient during and after administration.
• It does not cross the blood-brain barrier and so does not treat the central nervous system manifestations of the disease.

Monitoring

Monitor for infusion reactions and assess clinical response, including respiratory and functional outcomes, over the course of treatment.

Counselling the patient

• Treatment is given as a regular infusion in a supervised setting and continues long term.
• Report any reaction during infusion such as flushing, rash, breathlessness or fever.
• This therapy controls many systemic features but does not address effects on the brain.

Evidence & guidelines

Enzyme replacement with laronidase is the established disease-specific treatment for mucopolysaccharidosis type I.