Provider Summary
Primary Uses
hATTR amyloidosis with polyneuropathy in adults per label/payer criteria
Mechanism of Action
siRNA that reduces hepatic production of transthyretin (TTR).
Pre-treatment / Baseline Requirement
Premedication is required prior to each infusion to reduce infusion-related reactions (corticosteroid, acetaminophen, H1 and H2 blockers per PI); vitamin A supplementation at recommended daily allowance is advised; assess for infusion center readiness and monitor during infusion.
Common side effects
Infusion-related reactions, flushing, nausea, back pain, dyspnea, peripheral edema.
Serious adverse effects / key risks
Severe infusion reactions/hypersensitivity; vitamin A deficiency effects if not supplemented.
Referral requirements
Standard infusion referral form + drug-specific checklist
Patient & Caregiver Education
What it treats
hATTR amyloidosis with polyneuropathy in adults
How it works
Uses RNA-interference to reduce a disease-driving protein made by the liver.
Before treatment
Tell your clinician if you have an active infection/fever, are pregnant/planning pregnancy, or have major heart/nerve problems. Depending on the medication, you may need labs or screening tests (e.g., TB/hepatitis) and a vaccine review.
Common side effects
Infusion-related reactions, flushing, nausea, back pain, dyspnea, peripheral edema.
Get urgent help for:
Severe infusion reactions/hypersensitivity; vitamin A deficiency effects if not supplemented.
On treatment Day:
Plan to stay for monitoring. If you feel dizzy, drowsy, or unwell afterward, do not drive and follow your clinician’s instructions.