| Title: |
Impact of vutrisiran on quality of life and physical function in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy |
| Authors: |
Obici, Laura; Ajroud-Driss, Senda; Lin, Kon-Ping; Berk, John L.; Gillmore, Julian D.; Kale, Parag; Koike, Haruki; Danese, David; Aldinc, Emre; Chen, Chongshu; Vest, John; Adams, David; Wixner, Jonas; Backlund, Rolf; Pilebro, Björn; Anan, Intissar; Edbom, Fredrik; Ekman, Anna; Arvidsson, Sandra; Englund, Ulrika; Söderberg, Karin; Nordh, Erik; Uneus, Erica; Samuelsson, Kristin; Nilzen, Anna; Press, Rayomand; Bilecen, Mirjam; Coelho, Teresa; Novais, Marta; Rodrigues, Patricia; Martins da Silva, Ana; Cardoso, Inês; Rodrigues, Carla; Ramalho, Joana; Martins, Helder; Silva, Mónica; Guimaraes, Nádia; Perez, Javier; Reis, Antonio Hipólito; Monte, Julia; Ferreira, Natalia; Alves, Cristina; Cardoso, Marcio; Teixeira, Ricardo; Conceição, Isabel; Lamas, Filipa; Oliveira Santos, Miguel; Campos, Catarina; de Azevedo Coutinho, Conceiçao; Castro, José; Castro, Isabel; Silva, Daniela; Gonçalves, Susana; Di Buduo, Eleonora; Sforzini, Claudia; Mussinelli, Roberta; Rosti, Vittorio; Lozza, Alessandro; Racchi, Anna; Sabatelli, Mario; Luigetti, Marco; Bisogni, Giulia; Romano, Angela; Guglielmino, Valeria; Di Paolantonio, Andrea; Bernardo, Daniela; Vita, Giuseppe; Mazzeo, Anna; Russo, Massimo; Pareyson, Davide; Calabrese, Daniela; Fenu, Silvia; Saveri, Paola; Nienhuis, Hans; Bokhorst, Geert; Roos, Carlien; Couperus, Margriet; De Jong, Greetje; Brunger, Anne; Drost, Gea; Lange, Fiete; Colauto, Adinda; Waddington-Cruz, Márcia; Abreu, Aline; Pedrosa, Roberto Coury; Gervais de Santa Rosa, Renata; Dias, Moisés; Rakotondratafika, Fetra; Echaniz-Laguna, Andoni; Cauquil, Cecile; Labeyrie, Céline; Beaudonnet, Guillemette; Boubrit, Yasmine; Gaouar, Amina; Bourenane, Halima; Attarian, Shahram; Yahia, El Khansa; Verschueren, Annie; Grapperon, Aude-Marie; Delmont, Emilien; Planté-Bordeneuve, Violaine; Vervoitte, Laetitia; Ayache, Samar S.; Le Corvoisier, Philippe; Arrouasse, Raphaele; Gendre, Thierry; Abou Chakra, Laure; Focsénéanu, Cécile; Barau, Caroline; Sole, Guilhem; Belin, Laurie; Violleau, Marie Helene; Duval-Bontemps, Fanny; Massie, Rami; Dong, Xin; Muñoz-Beamud, Francisco; García Garrido, Sandra; Borrachero, Cristina; Gragera Martinez, Alvaro; Galán Dávila, Lucía; Palacios, Marta; Vicente, Laura M.; Perez de Isla, Leopoldo; Casasnovas, Carlos; Díez López, Carles; Fabra, Elena; González-Costello, José; Guerrero, Sonia; Yun Viladomat, Sergi; Martinez, Yurema; Velez-Santamaria, Valentina; Nedkova-Hristova, Velina; Garcia Pavia, Pablo; Gonzalez Segovia, Ariadna; De Frutos, Fernando; Gonzalez-Lopez, Esther; Dominguez, Fernando; Escobar-López, Luis E.; Cabrera-Romero, Eva; Sánchez Gismera, Paula; de la Iglesia, María; Martinez Valle, Fernando; Mazuela Aguila, Gonzalo; Lorite, Karen; Raguer, Núria; Suñé, Pilar; Piera, Pablo; Ortega, Carlos; Aguilar, Carla; Gili, Gisela; Schmidt, Hartmut; Langenstroer, Christel; Hüsing-Kabar, Anna; Kabar, Iyad; Schilling, Matthias; Friebel, Frauke; Tepasse, Phil-Robin; Birklein, Frank; Firros, Monika; Escolano-Lozano, Fabiola; Brueckner, Caitlin; Bahnam, Vanessa; Kaku, Michelle C.; Lau, K. H. Vincent; Wiesman, Janice; Grogan, Martha; Miller, Susanna; Frantz, Janell; Schmidt, Diane C.; AbouEzzeddine, Omar; Miller, Wayne; Lin, Grace; Gertz, Morie; Dispenzieri, Angela; Brannagan, Thomas; Fayerman, Raisy; DuVerger, Elizabeth; Cabrera, Jorge; Maurer, Mathew S.; Ulane, Christina M.; Weimer, Louis H.; Tsang, Stephen; Shije, Jeffrey; Carberry, Nathan; Thu, Sai Si; Quan, Dianna; Blume, Brianna; Overcash, J. Scott; Pu, He; Lee, Kia; Chu, Hanh; Zepeda, Karla; Waters, Michael; Vuong, Thao; Coskun, Derya; Quillin, Kimberly; Davis, Allison; Polydefkis, Michael; Ye, Jing; Li, Xiaoling; Khoshnoodi, Mohammad; Vista, Geno; Chung, Tae Hwan; Watt, Michele; Tsottles, Dan; Masri, Ahmad; Carlson, Dayna; Drachman, Brian; Divito, Patricia; Mathelier, Hansie; Shanks, Margaret; Maslowski, Karen; Khella, Sami; Pieretti, Janice; Joslin, Benjamin; Schmidt, Emma; Freimer, Miriam; Agriesti, Julie; Barroso, Fabio; Picone, Florencia; Lautre, Andrea; Orellana, Lucas; Du, Wenqin; Felius, Joost; González-Duarte, Alejandra; Cardenas Soto, Karla; Traub, Rebecca; Chopra, Manisha; Chao, Chi-Chao; Hsu, Chia-Hua; Tsai, Li-Kai; Lee, Ming-Jen; Su, Jen-Jen; Hsieh, Sung-Tsang; Hsueh, Hsueh-Wen; Chou, Hsi-Chieh; Kim, Byoung-Joon; Kang, Hyesun; Min, Ju-Hong; Jeon, Eun-Seok; Chung, Yeon Hak; Park, Jae Hong; Oh, Jeeyoung; Jeong, Hyun Joo; Tournev, Ivailo; Zhelyazkova, Sashka; Misumi, Yohei; Sakamoto, Yumiko; Hashimoto, Nami; Misumi, Yoshimi; Takahashi, Aya; Ueda, Mitsuharu; Masuda, Teruaki; Ueda, Akihiko; Katsuno, Masahisa; Tajima, Kazuki; Sumi, Momoko; Hasegawa, Fujiko; Okumura, Takahiro; Iijima, Masahiro; Fukami, Yuki; Ito, Daisuke; Kishimoto, Yoshiyuki; Kazuta, Tomoyuki; Kato, Katsuhiko; Mouri, Naohiro; Furukawa, Soma; Nishi, Ryoji; Sekijima, Yoshiki; Ito, Keiko; Kato, Nagaaki; Kishida, Dai; Mochizuki, Hideki; Okada, Kaori; Ohashi, Kurumi; Ikenaka, Kensuke; Nakamori, Masayuki; Kinoshita, Makoto; Mapalo, Bella Ruth; Law, Steven; Chacko, Liza; Lachmann, Helen; Cohen, Oliver; Razvi, Yousuf Siu Kay; Varughese, Sindhu; Martinez-Naharro, Ana; Orrell, Richard; Fontana, Marianna; Rannigan, Lisa; Louth, Sarah; Zamba-Papanicolaou, Eleni; Charalamnibous, Demetra; Manneh, Rana Abu; Kleopa, Kleopas; Christodoulides, Theodoros; Frangos, Savvas; Galganski-Cleanthous, Michele; Gaglia, Eftychia; Smoleski, Irene; Kkolou, Elena; Ploutarchou, Andry; Hanghiuc, Mariana; Chroidou, Galini; Stylianou, Olga; Krokou, Anastasia; Zannetou, Irene; Kastritis, Efstathios; Papadopoulou, Dimitra; Spinasas, Ilias; Bakalis, Panayiotis; Kanellias, Nikolaos; Fotiou, Despoina; Dialoupi, Ioanna; Migko, Magdalini; Gavriatopoulou, Maria; Low, Soon-Chai; Taylor, Mark; Stewart, Graeme; Knight, Helen; Vucic, Steve; Carroll, Antonia; Silsby, Matthew; Suan, Dan; Gibbs, Simon; Corfield, Carmela; Jakicic, Suzana; Jina, Hayden; Ting, Stephen; Wong, Shi Qin; Mollee, Peter; McKinley, Lynda; Abro, Emad; Korczyk, Dariusz; Remiche, Gauthier; Alaerts, Nick; De Veylder, Fabienne; Claeys, Kristl; Debien, Elisa; Cremers, Joyce; D'hondt, Ann; De Wel, Bram |
| Contributors: |
Obici, Laura; Ajroud-Driss, Senda; Lin, Kon-Ping; Berk, John L.; Gillmore, Julian D.; Kale, Parag; Koike, Haruki; Danese, David; Aldinc, Emre; Chen, Chongshu; Vest, John; Adams, David; Wixner, Jona; Backlund, Rolf; Pilebro, Björn; Anan, Intissar; Edbom, Fredrik; Ekman, Anna; Arvidsson, Sandra; Englund, Ulrika; Söderberg, Karin; Nordh, Erik; Uneus, Erica; Samuelsson, Kristin; Nilzen, Anna; Press, Rayomand; Bilecen, Mirjam; Coelho, Teresa; Novais, Marta; Rodrigues, Patricia; Martins da Silva, Ana; Cardoso, Inê; Rodrigues, Carla; Ramalho, Joana; Martins, Helder; Silva, Mónica; Guimaraes, Nádia; Perez, Javier; Reis, Antonio Hipólito; Monte, Julia; Ferreira, Natalia; Alves, Cristina; Cardoso, Marcio; Teixeira, Ricardo; Conceição, Isabel; Lamas, Filipa; Oliveira Santos, Miguel; Campos, Catarina; de Azevedo Coutinho, Conceiçao; Castro, José; Castro, Isabel; Silva, Daniela; Gonçalves, Susana; Di Buduo, Eleonora; Sforzini, Claudia; Mussinelli, Roberta; Rosti, Vittorio; Lozza, Alessandro; Racchi, Anna; Sabatelli, Mario; Luigetti, Marco; Bisogni, Giulia; Romano, Angela; Guglielmino, Valeria; Di Paolantonio, Andrea; Bernardo, Daniela; Vita, Giuseppe; Mazzeo, Anna; Russo, Massimo; Pareyson, Davide; Calabrese, Daniela; Fenu, Silvia; Saveri, Paola; Nienhuis, Han; Bokhorst, Geert; Roos, Carlien; Couperus, Margriet; De Jong, Greetje; Brunger, Anne; Drost, Gea; Lange, Fiete; Colauto, Adinda; Waddington-Cruz, Márcia; Abreu, Aline; Pedrosa, Roberto Coury; Gervais de Santa Rosa, Renata; Dias, Moisé; Rakotondratafika, Fetra; Echaniz-Laguna, Andoni; Cauquil, Cecile; Labeyrie, Céline; Beaudonnet, Guillemette; Boubrit, Yasmine; Gaouar, Amina; Bourenane, Halima; Attarian, Shahram; Yahia, El Khansa; Verschueren, Annie; Grapperon, Aude-Marie; Delmont, Emilien |
| Publisher Information: |
SPRINGER LONDON LTD |
| Publication Year: |
2023 |
| Collection: |
Università degli Studi di Messina: IRIS |
| Subject Terms: |
ATTRv amyloidosis; Nutritional status; Physical function; Polyneuropathy; Quality of life; RNA interference; Vutrisiran; hATTR amyloidosis |
| Description: |
IntroductionHereditary transthyretin (ATTRv; v for variant) amyloidosis, also known as hATTR amyloidosis, is a progressive and fatal disease associated with rapid deterioration of physical function and patients' quality of life (QOL). Vutrisiran, a subcutaneously administered RNA interference (RNAi) therapeutic that reduces hepatic production of transthyretin, was assessed in patients with ATTRv amyloidosis with polyneuropathy in the pivotal HELIOS-A study.MethodsThe phase 3 open-label HELIOS-A study investigated the efficacy and safety of vutrisiran in patients with ATTRv amyloidosis with polyneuropathy, compared with an external placebo group from the APOLLO study of the RNAi therapeutic patisiran. Measures of QOL and physical function were assessed.ResultsAt month 18, vutrisiran improved Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) total score (least squares mean difference [LSMD] in change from baseline [CFB]: -21.0; p = 1.84 x 10(-10)) and Norfolk QOL-DN domain scores, compared with external placebo. This benefit relative to external placebo was evident across all baseline polyneuropathy disability (PND) scores and most pronounced in patients with baseline PND scores I-II. Compared with external placebo, vutrisiran also demonstrated benefit in EuroQoL-Visual Analog Scale (EQ-VAS) score (LSMD in CFB: 13.7; nominal p = 2.21 x 10(-7)), 10-m walk test (LSMD in CFB: 0.239 m/s; p = 1.21 x 10(-7)), Rasch-built Overall Disability Score (LSMD in CFB: 8.4; p = 3.54 x 10(-15)), and modified body mass index (mBMI) (LSMD in CFB: 140.7; p = 4.16 x 10(-15)) at month 18. Overall, Norfolk QOL-DN, EQ-VAS, and mBMI improved from pretreatment baseline with vutrisiran, whereas all measures worsened from baseline in the external placebo group. At month 18, Karnofsky Performance Status was stable/improved from baseline in 58.2/13.1% with vutrisiran versus 34.7/8.1% with external placebo.ConclusionVutrisiran treatment provided significant clinical benefits in multiple measures of QOL and physical function in ... |
| Document Type: |
article in journal/newspaper |
| File Description: |
STAMPA |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/pmid/37523143; info:eu-repo/semantics/altIdentifier/wos/WOS:001040928300001; volume:12; issue:5; firstpage:1759; lastpage:1775; numberofpages:17; journal:NEUROLOGY AND THERAPY; https://hdl.handle.net/11570/3317114 |
| DOI: |
10.1007/s40120-023-00522-4 |
| Availability: |
https://hdl.handle.net/11570/3317114; https://doi.org/10.1007/s40120-023-00522-4; https://link.springer.com/article/10.1007/s40120-023-00522-4 |
| Rights: |
info:eu-repo/semantics/openAccess |
| Accession Number: |
edsbas.A40FC853 |
| Database: |
BASE |