دورية أكاديمية

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

التفاصيل البيبلوغرافية
العنوان: Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
المؤلفون: Adams, David, Polydefkis, Michael, González-Duarte, Alejandra, Wixner, Jonas, Kristen, Arnt V, Schmidt, Hartmut H, Berk, John L, Losada López, Inés Asunción, Dispenzieri, Angela, Quan, Dianna, Conceição, Isabel M, Slama, Michel S, Gillmore, Julian D, Kyriakides, Theodoros, Ajroud-Driss, Senda, Waddington-Cruz, Márcia, Mezei, Michelle M, Planté-Bordeneuve, Violaine, Attarian, Shahram, Mauricio, Elizabeth, Brannagan, Thomas H, Ueda, Mitsuharu, Aldinc, Emre, Wang, Jing Jing, White, Matthew T, Vest, John, Berber, Erhan, Sweetser, Marianne T, Coelho, Teresa, Giuseppe Vita, Vincenzo Rizzo, Massimo Russo, Anna Mazzeo, Luca Gentile, John L Berk, Caitlin Brueckner, Victoria Lazzari, Janice Wiesman, Douglas DeLong, Jennifer Victory, James Dalton, John May, Catherine Gilmore, Shahram Attarian, Saran Diallo, Emilien Delmont, Jean Pouget, Annie Verschueren, Aude-Marie Grapperon, Emmanuelle Campana-Salort, Isabel M Conceição, Ana Lopes, Filipa Lamas, Carlos Neves, Jose Castro, Pedro Pereira, Isabel Castro, Ana Franco, Miguel Oliveira Santos, Conceição de Azevedo Coutinho, Catarina Falcao de Campos, Teresa Coelho, Antonio Hipólito Reis, Nuno Correia, Javier M Perez, Ana Martins da Silva, Cristina Alves, Marcio Cardoso, Katia Valdrez, Julia R Monte, Bernardete Pessoa, Nadia Guimaraes, Monica Freitas, Joana Ramalho, Natalia Ferreira, Daisuke Kuzume, Celine Tard, Nawal Waucquier, Isabelle Rougeaux, Sylvie Brice, Emmanuelle Kasprzyk, Elise Elrezzi, Sayah Meguig, Eric Hachulla, Clement Gauvain, Maria-Claire Migaud-Chervy, Dominique Deplanque, Elsa Jozefowicz, Loic Lebellec, David Adams, Line Balaya-Gouraya, Nathalie Jehan Lacour, Halima Bournane, Nathalie Martin, Mongia Elabed, Niamey Sacko, Yasmine Boubrit, Amina Gaouar, Fetra Rakotondratafika, Marie Théaudin-Saliou, Cécile Cauquil-Michon, Celine Labeyrie, Adeline Not, Abdallah Al-Salameh, Anne-Lise Lecoq, Maeva Stephant, Andoni Echaniz-Laguna, Laurent Becquemont, Guillemette Beaudonnet, Vincent Algalarrondo, Ludivine Eliahou, Michel S Slama, Aissatou Signate, Emeline Berthelot, Jocelyn Inamo, Violaine Planté-Bordeneuve, Laetitia Vervoitte, Cecile Focseneanu, Thierry Gendre, Raphaele Arrouasse, Samar S Ayache, Laura Ernande, Philippe Le Corvoisier, Hayet Salhi, Ariane Choumert, Vincent Ehinger, Julie Ruiz, Cyril Charlin, Thomas Megelin, Thomas H Brannagan Iii, Raisy Fayerman, Arreum Kim, Allan Paras, Leidy J Gonzalez, Steven Tsang, Fernanda Wajnsztajn, Jeffrey Shije, Christina Ulane, Inna Kleyman, Louis Weimer, Comana Cioroiu, Sakis Lambrianides, Rana Abu-Manneh, Eleni Zamba-Papanicolaou, Petros Agathangelou, Eleni Leonidou, Theodoros Kyriakides, Satoshi Tada, Akemi Fujita, Masahiro Nagai, Rina Ando, Yuko Hosokawa, Yuki Yamanishi, J Scott Overcash, Elena Giardino, Leslie Boyer, Lien Dang, An Le, Tyler Nguyen, Lien Giang, Peter Sellers, Leyla Tran, Nghi Truong, Maita Vinas, Nicole Hrkman, Sarah Miller, David Nguyen, Ashley Smith, Helen Pu, Steve Li, Thao Vuong, Holly Dioso, Sinikka Green, Kia Lee, Hanh Chu, Michael Waters, Derya J Coskun, Karla A Zepeda, William O'Riordan, Laura Obici, Andrea Cortese, Alessandro Lozza, Giampaolo Merlini, Vittorio Rosti, Mario Sabatelli, Giulia Bisogni, Daniela Bernardo, Marco Luigetti, Andrea Di Paolantonio, Valeria Guglielmino, Angela Romano, Hans Nienhuis, Janita Bulthuis-Kuiper, Arnt V Kristen, Olga Gerk, Hannah Ulbricht, Lenka Taylor, Eva Meyle, Natalia Kleinschmidt, David Meyrath, Simone Noe-Schwenn, Ulrike Meng, Ralf Bauer, Fabian Aus dem Siepen, Selina Hein, Tetsuya Takahashi, Tomohiko Oshita, Yoko Koujin, Shuichiro Neshige, Tomohisa Nezu, Akiko Segawa, Hiroki Ueno, Hiroyuki Morino, Josep M Campistol, Lida Maria Rodas Marin, Josep Miquel Blasco Pelicano, Lucía Galán Dávila, Marta Palacios, Vanesa Pytel Cordoba, Antonio Guerrero Sola, Alejandro Horga, Julián García Feijoo, Leopoldo Perez de Isla, Wilson Marques Júnior, Mariana Moscardini, Debora Cristina Litcanov, Ana Flavia Viera Lima, Leonardo Rodrigues, Barbara Marques Coutinho, Carolina Lavigne Moreira, Vanessa Daccach Marques, Francisco Munoz Beamud, Álvaro Gragera Martínez, Cristina Borrachero, Inés Asunción Losada López, Eugenia Cisneros Barroso, Adrián Rodríguez Rodríguez, Monica Sanz, Elena Rigo Oliver, Juan González Moreno, Jose M Gamez Martinez, Cristina Descals, Mercedes Uson, Francisco Jose Vega, Antoni Figuerola, Carles Montala, Márcia Waddington-Cruz, Moises Dias da Silva, Renata Gervais de Santa Rosa, Luiz Felipe Pinto, Marcus Vinicius Pinto, Amanda Cardoso Berensztejn, Fabio Barroso, Andrea Lautre, Lucas G Orellana, Maria Alejandra González-Duarte Briseño, Karla Cárdenas-Soto, Brenda Poled Jiménez López, Sandra Lorena Pérez-Castañeda, Carlos Gerardo Cantú Brito, David Rivera de la Parra, Jose Pablo Hernandez Reyes, Maria Del Mar Saniger Alba, Elia Criollo Mora, Yesim Parman, Kus Jülide Rezzan, Erdi Sahin, Nail G Serbest, Hacer Durmus, Arman Cakar, Nuriye Ilknur Tugal Tutkun, Sacit Karamursel, Ali Elitok, Nermin G Sirin Inan, Emre Altinkurt, Michael Polydefkis, Jing Ye, Adriane C Allen, Vinay Chaudhry, Raquel Jarrett, Neil Bressler, Kathleen L Burks, Qingfeng Liu, Mohammad Khoshnoodi, Daniel P Judge, Geno Vista, Syed Mahmood Shah, Hirotoshi Hamaguchi, Junko Oda, Emi Fukase, Ikuko Taniguchi, Tetsuya Oda, Hironobu Endo, Masahiro Shimomura, Kimitaka Katanazaka, Shusuke Koto, Takahiro Nakano, Christof Scheid, Andreas Zueiter, Lars Pester, Doreen Walter, Betül Özdemir, Lukas F Frenzel, Udo Holtick, Jeeyoung Oh, Hee Jin Kim, Hyun Jin Shin, Kyomin Choi, Taro Yamashita, Mitsuharu Ueda, Teruaki Masuda, Yohei Misumi, Akihiko Ueda, Keiichi Nakahara, Akiko Yorita, Seiko Tsuruhisa, Takayuki Taniwaki, Masaya Harada, Taiga Moritaka, Naonori Sakurada, Elizabeth A Mauricio, Amber Baskin, Elliot Dimberg, Angela Dispenzieri, Amie Fonder, Miriam Hobbs, Stephen J Russell, Peter Dyck, Wilson Gonsalves, Nelson Leung, Thomas E Witzig, Steven R Zeldenrust, Lisa Hwa, Prashant Kapoor, Shaji K Kumar, Yi Lin, John A Lust, Vincent S Rajkumar, David Dingli, Morie A Gertz, Ronald Go, Suzanne R Hayman, Samir Dalia, Esmeralda Carrillo, Peter Gorevic, Garnette Mason, Chi-Chao Chao, Ming-Jen Lee, Jen-Jen Su, Sung-Tsang Hsieh, Li-Kai Tsai, Shin-Joe Yeh, Chih-Chao Yang, Senda Ajroud-Driss Ajroud-Driss, Patricia Casey, Benjamin C Joslin, Miriam Freimer, Alison Sankey, Amanda Kenepp, Sarah Heintzman, Samantha LoRusso, Youichi Hokezu, Byoung-Joon Kim, JuHyeon Kim, Ga Yeon Lee, Eun Bin Cho, Eun-Seok Jeon, Ju-Hong Min, Jin Myoung Seok, Hye Lim Lee, Jae Hong Park, Yoshiki Sekijima, Chinatsu Miyazawa, Nagaaki Kato, Dai Kishida, Akiyo Hineno, Minori Kodaira, Tsuneaki Yoshinaga, Teruyoshi Miyahara, Akira Imai, Kazuhiko Matsumoto, Kon-Ping Lin, Yi-Chung Lee, Jonas Wixner, Malin Falk, Bjorn Pilebro, Ole Suhr, Per Lindqvist, Karin Soderberg, Fatima Pedrosa-Domellöf, Intissar Anan, Erik Nordh, Ivaylo Tournev, Sashka Zhelyazkova-Glaveeva, Zheyna Cherneva, Staiko Sarafov, Teodora Chamova, Sylvia Cherninkova-Gopina, Hartmut H Schmidt, Frauke Friebel, Andree Zibert, Natasa Mihailovic, Friederike Schubert, Elena Vorona, Larissa Lahme, Anna Huesing-Kabar, Matthias Schilling, Iyad Kabar, Julian D Gillmore, Ana Martinez-Naharro, Liza Chacko, Oliver Cohen, Steven Law, Tamer Rezk, Helen J Lachmann, Dianna Quan, Brianna Blume, Stacy Dixon, Soon Chai Low, Soo Looi Chan, He Eng Li Lim, Khean Jin Goh, Michelle M Mezei, Deborah Kraus, Kristin Jack, Glenn Lopate, Brittany Zwijack, Julaine Florence, R Brian Sommerville, Graeme Stewart, Julie Ryder, Linda Mekhael, Mark Taylor, Daniel Suan, Karen Wells, Paula Stone, Amenze Itoya, Mercy Owusu-Sekyere, Desmond Thai, Ilonah Chahine, Salve Pedrosa, Thi Hoa Therese Do
المساهمون: David, Adam, Michael, Polydefki, Alejandra, González-Duarte, Jonas, Wixner, Arnt V, Kristen, Hartmut H, Schmidt, John L, Berk, Inés Asunción, Losada López, Angela, Dispenzieri, Dianna, Quan, Isabel M, Conceição, Michel S, Slama, Julian D, Gillmore, Theodoros, Kyriakide, Senda, Ajroud-Dri, Márcia, Waddington-Cruz, Michelle M, Mezei, Violaine, Planté-Bordeneuve, Shahram, Attarian, Elizabeth, Mauricio, Thomas H, Brannagan, Mitsuharu, Ueda, Emre, Aldinc, Jing Jing, Wang, Matthew T, White, John, Vest, Erhan, Berber, Marianne T, Sweetser, Teresa, Coelho, Vita, Giuseppe, Rizzo, Vincenzo, Russo, Massimo, Mazzeo, Anna, Gentile, Luca, L Berk, John, Brueckner, Caitlin, Lazzari, Victoria, Wiesman, Janice, DeLong, Dougla, Victory, Jennifer, Dalton, Jame, May, John, Gilmore, Catherine, Attarian, Shahram, Diallo, Saran, Delmont, Emilien, Pouget, Jean, Verschueren, Annie, Grapperon, Aude-Marie, Campana-Salort, Emmanuelle, M Conceição, Isabel, Lopes, Ana, Lamas, Filipa, Neves, Carlo, Castro, Jose, Pereira, Pedro, Castro, Isabel, Franco, Ana, Oliveira Santos, Miguel, de Azevedo Coutinho, Conceição, Falcao de Campos, Catarina, Coelho, Teresa, Hipólito Reis, Antonio, Correia, Nuno, M Perez, Javier, Martins da Silva, Ana, Alves, Cristina, Cardoso, Marcio, Valdrez, Katia, R Monte, Julia, Pessoa, Bernardete, Guimaraes, Nadia, Freitas, Monica, Ramalho, Joana, Ferreira, Natalia, Kuzume, Daisuke, Tard, Celine, Waucquier, Nawal, Rougeaux, Isabelle, Brice, Sylvie, Kasprzyk, Emmanuelle, Elrezzi, Elise, Meguig, Sayah, Hachulla, Eric, Gauvain, Clement, Migaud-Chervy, Maria-Claire, Deplanque, Dominique, Jozefowicz, Elsa, Lebellec, Loic, Adams, David, Balaya-Gouraya, Line, Jehan Lacour, Nathalie, Bournane, Halima, Martin, Nathalie, Elabed, Mongia, Sacko, Niamey, Boubrit, Yasmine, Gaouar, Amina, Rakotondratafika, Fetra, Théaudin-Saliou, Marie
بيانات النشر: Elsevier
سنة النشر: 2021
المجموعة: Università degli Studi di Messina: IRIS
مصطلحات موضوعية: amyloidosis, transthyretin, polyneuropathy, patisiran, OLE study
الوصف: Background: 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. Methods: 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 trial. The global OLE is ongoing with no new enrolment, and current findings are based on the interim analysis of the patients who had completed 12-month efficacy assessments as of the data cutoff. Safety analyses included all patients who received one or more dose of patisiran up to the data cutoff. This study is registered with ClinicalTrials.gov, NCT02510261. Findings: Between July 13, 2015, and Aug 21, 2017, of 212 eligible patients, 211 were enrolled: 137 patients from the APOLLO-patisiran group, 49 from the APOLLO-placebo group, and 25 from the phase 2 OLE patisiran group. At the data cutoff on Sept 24, 2018, 126 (92%) of 137 patients from the APOLLO-patisiran group, 38 (78%) of 49 from the APOLLO-placebo group, and 25 (100%) of 25 from the phase 2 OLE patisiran ...
نوع الوثيقة: article in journal/newspaper
وصف الملف: STAMPA
اللغة: English
العلاقة: info:eu-repo/semantics/altIdentifier/pmid/33212063; info:eu-repo/semantics/altIdentifier/wos/WOS:000600831400023; volume:20; issue:1; firstpage:49; lastpage:59; numberofpages:11; journal:LANCET NEUROLOGY; http://hdl.handle.net/11570/3181060Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85097405151; https://www.sciencedirect.com/science/article/pii/S1474442220303689?via=ihubTest
DOI: 10.1016/S1474-4422(20)30368-9
الإتاحة: https://doi.org/10.1016/S1474-4422Test(20)30368-9
http://hdl.handle.net/11570/3181060Test
https://www.sciencedirect.com/science/article/pii/S1474442220303689?via=ihubTest
رقم الانضمام: edsbas.88526C1C
قاعدة البيانات: BASE