{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"omics_type":["Unknown"],"volume":["82(3)"],"submitter":["Adams D"],"pubmed_abstract":["<h4>Importance</h4>There is a lack of long-term efficacy and safety data on hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) and on RNA interference (RNAi) therapeutics in general. This study presents the longest-term data to date on patisiran for hATTR-PN.<h4>Objective</h4>To present the long-term efficacy and safety of patisiran in adults with hATTR-PN.<h4>Design, setting, and participants</h4>This global open-label extension (OLE) of the APOLLO randomized clinical trial and phase 2 OLE study enrolled patients from 43 hospitals or clinical centers across 19 countries between July 2015 and August 2017, with follow-up until November 2022. Of 212 eligible patients with hATTR who completed the phase 3 APOLLO or phase 2 OLE parent studies, 211 enrolled in and 138 completed "],"journal":["JAMA neurology"],"pagination":["228-236"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC11894486"],"repository":["biostudies-literature"],"pubmed_title":["Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension."],"pmcid":["PMC11894486"],"pubmed_authors":["Martins da Silva A","Cortese A","Kodaira M","Nygren A","Wixner J","Russo M","Kaku M","Lin Y","Florence J","Pinto MV","Takasone K","Taylor M","Coelho T","Cherneva Z","Yureneva E","Borrachero C","Vita G","Cioroiu C","Holtick U","Wajnsztajn F","LoRusso S","Lui AT","Megelin T","Polydefkis M","Hein S","Berk JL","Dyck P","Algalarrondo V","Bender S","Yang CC","Perez JM","Alves C","Miyahara T","Wiesman J","Pouget J","Durmus H","Shah SM","May J","Manos P","Rigo Oliver E","Cherninkova-Gopina S","Freitas M","Yamasaki M","Rezk T","Descals C","Croy D","Sommerville RB","Fridman V","Vista G","Dyck PJ","Khoshnoodi M","Cohen O","Wade NK","Schilling M","Dixon S","Not A","Goh KJ","Novais M","Rizzo V","Ajroud-Driss S","Zhelyazkova-Glaveeva S","Lopate G","Le Corvoisier P","Ramalho J","Green S","Sabatelli M","Tsai LK","Suhr O","Nagel C","Nomura T","Garcia Feijoo J","Neves C","Reis AH","Waddington-Cruz M","Kleopa K","Kuzume D","Berensztejn AC","Lavigne Moreira C","Hsieh ST","Campana-Salort E","Hwa L","Weimer L","Kishida D","Santos MO","Judge DP","Shin HJ","Aso Y","Rousseau A","Hernandez Reyes JP","Sarafov S","Gamez Martinez JM","Kleyman I","Fonder A","Kim BJ","Berthelot E","Romano A","Min JH","Elitok A","Al-Salameh A","Attarian S","Stephant M","Vorona E","Low SC","Choumert A","Gonzalez Moreno J","Yoshinaga T","Lopez IA","Labeyrie C","Gauvain C","Oda T","Gorevic P","Tateishi T","Dimberg E","Ayache SS","Lee GY","Nagai M","Gonzalez-Duarte Briseno MA","Chamova T","Theaudin-Saliou M","Ueda M","Lee YC","Sahin E","Conceicao IM","Zamba-Papanicolaou E","Karamursel S","Suan D","Obici L","Lee HL","Chacko L","Sufit R","Pedrosa-Domellof F","Daccach Marques V","Lautre A","Chaudhry V","Lin KP","Ando R","Valdrez K","Park JH","Cakar A","Sirin Inan NG","Rajkumar VS","Sekijima Y","Cardoso M","Chung TH","Gillmore JD","Leung N","Arrouasse R","Li JM","Misumi Y","Cardoso I","Hineno A","Tard C","Neshige S","DeLong D","Becquemont L","Mauricio EA","Jozefowicz E","Bressler N","Signate A","Hamaguchi H","Tsang S","Stewart G","Falcao de Campos C","Gentile L","Bernardo D","Jeon ES","Lebellec L","Overcash JS","Beaudonnet G","Tepasse PR","de Andrade Guedes M","Scheid C","Gonsalves W","Lin CW","patisiran Global OLE study group","Lachmann HJ","Di Paolantonio A","Serbest NG","Shije J","Kristen AV","Munoz Beamud F","Jay PY","Imai A","Mazzeo A","Coskun DJ","Monte JR","Lee MJ","Dalton J","Figuerola A","Allen AC","Darche P","Marques Junior W","Kabar I","Sanz M","Kim J","Galan Davila L","Bisogni G","Dias da Silva M","Rivera de la Parra D","Ferreira N","Gove L","Su JJ","Blasco Pelicano JM","Merlini G","Parman Y","Plante-Bordeneuve V","Hobbs M","Mezei MM","Eliahou L","Law S","Adams D","Kapoor P","Chao CC","Gendre T","Gervais de Santa Rosa R","Nienhuis H","Peltier A","Slama MS","Guimaraes N","Quan D","O'Riordan W","Correia N","Morimoto Y","Tournev I","Horga A","Lozza A","Guerrero Sola A","Dispenzieri A","Lecoq AL","Montala C","Deplanque D","Rosti V","Martinez-Naharro A","Singh M","Brannagan Iii TH","Seok JM","Grapperon AM","Guglielmino V","Shimomura M","Uson M","Migaud-Chervy MC","Tada S","Orellana LG","Barroso F","Yeh SJ","Jack K","Altinkurt E","Witzig TE","Dingli D","Cho EB","Holding T","Yamanishi Y","Pilebro B","Cantu Brito CG","Anan I","Oh J","Choi K","Delmont E","Aus dem Siepen F","Jarrett R","Cooper LT","Takahashi Y","Huesing-Kabar A","Gertz MA","Hachulla E","Vega FJ","Kato N","Schmidt HH","Cauquil-Michon C","Kumar SK","Freimer M","Chermenina M","Luigetti M","Cardenas-Soto K","Pytel Cordoba V","Capocelli K","Campistol JM","Souza de Abreu A","de Azevedo Coutinho C","Nordh E","Perez de Isla L","Ulane C","Kim HJ","Saniger Alba MDM","Salhi H","Lau V","Dalia S","Echaniz-Laguna A","Inamo J","Rodas Marin LM","Waters M","Ernande L","Charlin C","Grimaud J","Matsumoto K","Geisler S","Pinto LF","Verschueren A","Zepeda KA"],"additional_accession":[]},"is_claimable":false,"name":"Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.","description":"<h4>Importance</h4>There is a lack of long-term efficacy and safety data on hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) and on RNA interference (RNAi) therapeutics in general. This study presents the longest-term data to date on patisiran for hATTR-PN.<h4>Objective</h4>To present the long-term efficacy and safety of patisiran in adults with hATTR-PN.<h4>Design, setting, and participants</h4>This global open-label extension (OLE) of the APOLLO randomized clinical trial and phase 2 OLE study enrolled patients from 43 hospitals or clinical centers across 19 countries between July 2015 and August 2017, with follow-up until November 2022. Of 212 eligible patients with hATTR who completed the phase 3 APOLLO or phase 2 OLE parent studies, 211 enrolled in and 138 completed ","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Mar","modification":"2026-06-02T19:19:01.327Z","creation":"2025-04-04T02:50:52.91Z"},"accession":"S-EPMC11894486","cross_references":{"pubmed":["39804640"],"doi":["10.1001/jamaneurol.2024.4631"]}}