PATISIRAN INJ,SOLN
Clinical Criteria Summary
Exclusion Criteria
- Sensorimotor or autonomic neuropathy (including diabetic neuropathy) without also having a diagnosis of hereditary transthyretin-mediated (hATTR) amyloidosis
- Primary amyloidosis
- Leptomeningeal amyloidosis
- Currently receiving another therapy for hATTR amyloidosis (e.g., diflunisal, tafamidis, vutrisiran)
- Prior liver transplant
- New York Heart Association (NYHA) Class III or IV heart failure
- Known pregnancy
Inclusion Criteria
- Provider is a VA or VA Community Care neurologist or locally designated hATTR amyloidosis provider
- Diagnosis of polyneuropathy of hATTR amyloidosis including clinical symptoms and genetic testing that confirms a variant in TTR
- Documented baseline Neuropathy Impairment Score (NIS) of 5 to 130, Polyneuropathy Disability Score (PND) I to IIIb or Familial Amyloid Polyneuropathy (FAP) stage 1 or 2
- Age 18-85 years
- Anticipated survival > 2 years
- Contraindication, intolerance, or lack of clinical response to at least a 9-month trial of vutrisiran
- Supplementation of the recommended daily allowance of vitamin A is planned to start upon approval
- Discussion with patient/caregiver/family member regarding realistic treatment expectations and discontinuation should be documented
Additional Inclusion Criteria
- For females who can become pregnant: Counseling provided on potential risks vs. benefits of treatment including the risk that patisiran can decrease serum vitamin A levels
Monitoring & Continuation Requirements
- Continuation of therapy assessed at 9 months and then periodically throughout treatment
- Therapy continued only if patient continues to demonstrate positive clinical response (e.g., improved motor function, quality of life, or ambulation or decreased neurological impairment)
- Refer to eye clinic if ocular symptoms suggestive of vitamin A deficiency develop (e.g., night blindness)