siRNA targeting transthyretin (TTR)
Patisiran
Brand names: Onpattro
Adult dose
Dose: 300 micrograms/kg IV every 3 weeks (max 30mg)
Route: IV
Frequency: q3w
Clinical pearls
- Hereditary transthyretin amyloidosis with polyneuropathy; specialist amyloid centre
- Vitamin A 2500 IU/day required
Contraindications
- Hypersensitivity
Side effects
- Infusion reactions
- Vitamin A deficiency
- Headache
- Atrial fibrillation
Interactions
- Pre-medicate with steroid + antihistamine + paracetamol
Monitoring
- Cardiac function
- Vitamin A levels
Reference: BNF; NICE HST10; SmPC; https://bnf.nice.org.uk/drugs/patisiran/. Verify against your local formulary and the latest BNF before prescribing.
Related
Curated clinical cross-links plus same-class fallbacks.
Calculators
Pathways
- Major Haemorrhage / Massive Transfusion · BCSH; RCOA; RCEM; RCS — BCSH Guidelines
- Anaemia Investigation · BSH / NICE
- Splenomegaly Workup · BSH; BMJ Best Practice
- Deep Vein Thrombosis Diagnosis and Treatment · NICE CG144 / NICE NG158
- Sickle Cell Crisis · BSH 2021 / BCSH
- Neutropenic Sepsis · NICE CG151 2012 / ESMO