Long-Term Results with Patisiran in Familial Amyloidosis with Polyneuropathy

In a 5-year extension study, patisiran was associated with continued attenuation of disease progression, and with improved survival when initiated early.

Without treatment, polyneuropathy from hereditary transthyretin amyloidosis (hATTR) is relentlessly progressive, with median survival less than 5 years in some cohorts. In a landmark 2018 randomized, placebo-controlled trial (APOLLO), patients with polyneuropathy from hATTR who received patisiran, an RNA interference agent, had stability or improvement in their disease manifestations at 18 months. Participants who switched from placebo to patisiran in a 12-month, open-label extension trial (OLE) also had benefits. Now, a global, open-label, single-arm, industry-sponsored extension study of 212 patients from the APOLLO and OLE trials has evaluated the long-term efficacy and safety of 0.3 mg/kg of intravenous patisiran given every three weeks…

Reviewing Author

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

Long-Term Results with Patisiran in Familial Amyloidosis with Polyneuropathy

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Reviewing Author