Patisiran

How it works

Delivered in a lipid nanoparticle, it silences hepatic transthyretin (TTR) messenger RNA via RNA interference, reducing production of both mutant and wild-type TTR protein and thereby amyloid deposition.

Prescribing in practice

• Infusion-related reactions can occur, so premedication (such as a corticosteroid, paracetamol and antihistamines) is given and the infusion administered under supervision with capacity to manage reactions.
• It reduces serum vitamin A, so vitamin A supplementation at the recommended daily allowance is advised and patients should be referred for ophthalmic review if visual symptoms suggesting deficiency develop.
• It is administered by intravenous infusion at regular intervals under specialist supervision in line with the SPC.

Monitoring

Monitor for infusion-related reactions during and after administration and review for symptoms of vitamin A deficiency over the course of treatment.

Counselling the patient

• Explain that treatment is given as a regular intravenous infusion and aims to slow nerve damage rather than reverse established disease.
• Advise taking the recommended vitamin A supplement and reporting any visual changes such as night-vision problems.

Evidence & guidelines

The APOLLO trial demonstrated improvement in neuropathy outcomes with patisiran in hereditary transthyretin amyloidosis.