biostudies-literatureUnknown82(3)Adams D<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 JAMA neurology228-236https://www.ebi.ac.uk/biostudies/studies/S-EPMC11894486biostudies-literatureFive-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.PMC11894486Martins da Silva ACortese AKodaira MNygren AWixner JRusso MKaku MLin YFlorence JPinto MVTakasone KTaylor MCoelho TCherneva ZYureneva EBorrachero CVita GCioroiu CHoltick UWajnsztajn FLoRusso SLui ATMegelin TPolydefkis MHein SBerk JLDyck PAlgalarrondo VBender SYang CCPerez JMAlves CMiyahara TWiesman JPouget JDurmus HShah SMMay JManos PRigo Oliver ECherninkova-Gopina SFreitas MYamasaki MRezk TDescals CCroy DSommerville RBFridman VVista GDyck PJKhoshnoodi MCohen OWade NKSchilling MDixon SNot AGoh KJNovais MRizzo VAjroud-Driss SZhelyazkova-Glaveeva SLopate GLe Corvoisier PRamalho JGreen SSabatelli MTsai LKSuhr ONagel CNomura TGarcia Feijoo JNeves CReis AHWaddington-Cruz MKleopa KKuzume DBerensztejn ACLavigne Moreira CHsieh STCampana-Salort EHwa LWeimer LKishida DSantos MOJudge DPShin HJAso YRousseau AHernandez Reyes JPSarafov SGamez Martinez JMKleyman IFonder AKim BJBerthelot ERomano AMin JHElitok AAl-Salameh AAttarian SStephant MVorona ELow SCChoumert AGonzalez Moreno JYoshinaga TLopez IALabeyrie CGauvain COda TGorevic PTateishi TDimberg EAyache SSLee GYNagai MGonzalez-Duarte Briseno MAChamova TTheaudin-Saliou MUeda MLee YCSahin EConceicao IMZamba-Papanicolaou EKaramursel SSuan DObici LLee HLChacko LSufit RPedrosa-Domellof FDaccach Marques VLautre AChaudhry VLin KPAndo RValdrez KPark JHCakar ASirin Inan NGRajkumar VSSekijima YCardoso MChung THGillmore JDLeung NArrouasse RLi JMMisumi YCardoso IHineno ATard CNeshige SDeLong DBecquemont LMauricio EAJozefowicz EBressler NSignate AHamaguchi HTsang SStewart GFalcao de Campos CGentile LBernardo DJeon ESLebellec LOvercash JSBeaudonnet GTepasse PRde Andrade Guedes MScheid CGonsalves WLin CWpatisiran Global OLE study groupLachmann HJDi Paolantonio ASerbest NGShije JKristen AVMunoz Beamud FJay PYImai AMazzeo ACoskun DJMonte JRLee MJDalton JFiguerola AAllen ACDarche PMarques Junior WKabar ISanz MKim JGalan Davila LBisogni GDias da Silva MRivera de la Parra DFerreira NGove LSu JJBlasco Pelicano JMMerlini GParman YPlante-Bordeneuve VHobbs MMezei MMEliahou LLaw SAdams DKapoor PChao CCGendre TGervais de Santa Rosa RNienhuis HPeltier ASlama MSGuimaraes NQuan DO'Riordan WCorreia NMorimoto YTournev IHorga ALozza AGuerrero Sola ADispenzieri ALecoq ALMontala CDeplanque DRosti VMartinez-Naharro ASingh MBrannagan Iii THSeok JMGrapperon AMGuglielmino VShimomura MUson MMigaud-Chervy MCTada SOrellana LGBarroso FYeh SJJack KAltinkurt EWitzig TEDingli DCho EBHolding TYamanishi YPilebro BCantu Brito CGAnan IOh JChoi KDelmont EAus dem Siepen FJarrett RCooper LTTakahashi YHuesing-Kabar AGertz MAHachulla EVega FJKato NSchmidt HHCauquil-Michon CKumar SKFreimer MChermenina MLuigetti MCardenas-Soto KPytel Cordoba VCapocelli KCampistol JMSouza de Abreu Ade Azevedo Coutinho CNordh EPerez de Isla LUlane CKim HJSaniger Alba MDMSalhi HLau VDalia SEchaniz-Laguna AInamo JRodas Marin LMWaters MErnande LCharlin CGrimaud JMatsumoto KGeisler SPinto LFVerschueren AZepeda KAfalseFive-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.<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 2025-01-01T00:00:00Z2025 Mar2026-06-02T19:19:01.327Z2025-04-04T02:50:52.91ZS-EPMC118944863980464010.1001/jamaneurol.2024.4631