Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study.

Lancet Neurology
David Adamspatisiran Global OLE study group

Abstract

Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. This multicentre, open-label extension (OLE) trial enrolled patients at 43 hospitals or clinical centres in 19 countries as of Sept 24, 2018. Patients were eligible if they had completed the phase 3 APOLLO or phase 2 OLE parent studies and tolerated the study drug. Eligible patients from APOLLO (patisiran and placebo groups) and the phase 2 OLE (patisiran group) studies enrolled in this global OLE trial and received patisiran 0·3 mg/kg by intravenous infusion every 3 weeks with plans to continue to do so for up to 5 years. Efficacy assessments included measures of polyneuropathy (modified Neuropathy Impairment Score +7 [mNIS+7]), quality of life, autonomic symptoms, nutritional status, disability, ambulation status, motor function, and cardiac stress, with analysis by study groups (APOLLO-placebo, APOLLO-patisiran, phase 2 OLE patisiran) based on allocation in the parent t...Continue Reading

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Citations

Apr 4, 2021·Biomolecules·Sanduni Wasana JayaweeraAnders Olofsson
Feb 26, 2021·The Canadian Journal of Neurological Sciences. Le Journal Canadien Des Sciences Neurologiques·Monica AlcantaraVera Bril
May 1, 2021·Brain Sciences·Luca GentileAnna Mazzeo
Jun 6, 2021·Cardiology and Therapy·Haruki KoikeMasahisa Katsuno
Jun 9, 2021·Kidney International·Ziad A Massy, Tilman B Drueke
Jul 3, 2021·The New England Journal of Medicine·Julian D GillmoreDavid Lebwohl
Aug 2, 2021·Molecular Aspects of Medicine·Manish PandeyGeetanjali Chawla
Aug 28, 2021·Molecules : a Journal of Synthetic Chemistry and Natural Product Chemistry·Haruki KoikeMasahisa Katsuno
Sep 23, 2021·Arquivos brasileiros de cardiologia·Marcus V SimõesEvandro Tinoco Mesquita
Nov 10, 2021·European Journal of Neurology : the Official Journal of the European Federation of Neurological Societies·José CastroIsabel Conceição
Jul 30, 2021·Journal of Cardiovascular Pharmacology·Zackary J TushakKeyur B Shah

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