2020 Joint conference of the Italian Association of Miology (AIM) and the Italian Association for the study of the Perip
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ABSTRACTS
2020 Joint conference of the Italian Association of Miology (AIM) and the Italian Association for the study of the Peripheral Nervous System (ASNP), Abstracts
# Fondazione Società Italiana di Neurologia 2020
Long-Term Safety and Efficacy of Patisiran: Global Open-label Extension 24-month Data in Patients with H ereditary Transthyretin-mediated Amyloidosis D. Adams 1 , A.González-Duarte 2 , E.Mauricio 3 , T.Brannagan 4 , T.Coelho5, J.Wixner6, H.Schmidt7 , A. Mazzeo8, E.Berber 9, M.T Sweetser9, M.T White9, J.J Wang9, M.Polydefkis10 1
National Reference Center for FAP (NNERF)/ APHP/ INSERM U 1195/ CHU Bicêtre, Le Kremlin Bicêtre, France; 2 Instituto Nacional de Ciencias Médicas y Nutrición, Salvador Zubirán, Mexico City, Mexico; 3Mayo Clinic, Jacksonville, FL, USA; 4 Department of Neurology, Columbia University, New York City, NY, USA; 5 Centro Hospitalar Universitário do Porto, Portugal; 6 Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden; 7 University Hospital Muenster, Muenster, Germany; 8 Unit of Neurology and Neuromuscular Diseases, Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy (presenting on behalf of the authors); 9 Alnylam Pharmaceuticals, Cambridge, USA; 10 Johns Hopkins University, Baltimore, USA Introduction: Hereditary transthyretin-mediated (hATTR) amyloidosis is a progressive, life-threatening disease; most patients develop a mixed phenotype including polyneuropathy and cardiomyopathy. Patisiran’s safety/efficacy have been demonstrated in Phase 2 and 3 studies in patients with hATTR amyloidosis with polyneuropathy. Interim 24-month efficacy/safety analyses of the ongoing Global open-label extension (OLE) study are described. Methods: Multicentre, international, safety and efficacy study (NCT02510261) in eligible patients who completed parent studies, including patients in the Phase 3 APOLLO randomized to placebo (APOLLO/placebo, n=49) or patisiran (APOLLO/patisiran, n=137) over 18 months and patients in the Phase 2 OLE (n=25) receiving patisiran over 24 months. Results: 178/211 patients had ≥24 months of exposure as of 07/10/ 2019. Safety profile remained consistent with previous studies. After 24 months of additional patisiran treatment in the Global OLE, durable improvement was seen for modified Neuropathy Impairment Score+7 (mNIS+7) (mean change [SEM]) in APOLLO/patisiran (-4.9 [2.1]) and Phase 2 OLE (-5.9 [2.1]) groups compared to parent study baselines. Norfolk quality of life-diabetic neuropathy (QOL-DN) continued to show durable improvement in APOLLO/patisiran patients (-2.4 [2.4]) following additional 24-
month treatment. APOLLO/placebo patients experienced halting of disease progression and QOL improvement compared to Global OLE baseline after 24 months of patisiran in the OLE (mNIS+7: +0.1 [3.3], Norfolk QOL-DN: -4.1 [3.3]), although they had progressed relative to APOLLO baseline (mNIS+7: +26.3 [5.0], Norfolk QOL-DN: +15.8 [4.5]) given the progression while on placebo. Conclusion: Patients with long-term ex
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