| Title: |
Long-term safety and efficacy of eculizumab in generalized myasthenia gravis |
| Authors: |
Tandan, Rup; Malekniazi, Ali; Akhter, Salma; Tran, Anh; Shah, Akshay; Pokala, Krishna; McClain, Terry; Lin, Frank; Karimi, Sanaz; Gong, Hui; Droker, Brian; Doan, Victor; Chilingaryan, Amaiak; Beydoun, Said; Huynh, Kristin; Hart, Adam; Harvey, Brittany; Katzin, Lara; Traub, Rebecca; Chopra, Manisha; Karam, Chafic; Steele, Julie; Sheldon, Danielle; Michon, Sara-Claude; Reyes, Eliana; Cooley, Anne; Sharma, Khema; Bilsker, Martin; Naunton, Kerry; DiSanzo, Beth; Young, Valerie; Callison, Karen; Zilliox, Lindsay; Kwan, Justin; Hafer-Macko, Charlene; Herbelin, Laura; Walsh, Maureen; Rico, Gabrielle; Johns, Kathryn; Fondaw, Alex; Locheke, Jerry; Jenci, Nicole; Emmons, Kelley; Liu, Tina; Wang, Yunxia; Statland, Jeffery; Pasnoor, Mamatha; McVey, April; Glenn, Melanie; Dimachkie, Mazen; Sieren, Jeri; Kressin, Nicole; Olalde, Heena; Swenson, Andrea; Nance, Christopher; Jerath, Nivedita; Gutmann, Ludwig; Gutmann, Laurie; Varghese, Mary; Smith, Lisa; Shabbir, Yasmeen; Shah, Jaimin; Berger, Alan Ross; Aranke, Shachie; Pulley, Michael; Garcia, Sonia; Echevarria, Laura; Garcia, Rebekah; Rosenfeld, Jeffrey; Jones, H. James; Minton, Brian; Maqsood, Fatima; Mathew, Veena; Roy, Gulmohor; Goyal, Namita; Cash, Tiyonnoh; Mozaffar, Tahseen; Woodall, Amy; Mumfrey-Thomas, Sandi; Nozaki, Kenkichi; Lu, Liang; Alsharabati, Mohammad; Kazamel, Mohamed; Hopkins, Steve; Trivedi, Jaya; Nations, Sharon; Silva, Marcela; Saklad, Amy; Padilla, Patricia; Gonzalez, Anna; Jones, Floyd; Dishman, Lorraine; Tobon Gonzales, Alejandro; Bhavaraju-Sanka, Ratna; Welsh, Lesly; Pol, Sabrina; Paulose, Shirley; So, Yuen T.; Sakamuri, Sarada; Goyal, Neelam; Seelback, Jenna; Lokaitis, Barbara; Golden, Laura; Darnell, Taylor; Andoin, Angela; Kohlrus, Stephanie; Elsayed, Mona; Agrawal, Brajesh; Gilchrist, James; Dimitrova, Diana; Kaur, Harpreet; Marburger, Tessa; Khoury, Julie; Muharrem, Filiz; Pineda, Colleen; Mezache, Louisa; Chelnick, Sharon; Agriesti, Julie; Hoyle, Joseph Chad; Freimer, Miriam; Jeffery, Dennis; Daniels, Kevin; Sattar, Nadia; McKenna, Kaitlyn; Alcon, Karan; McKinnon, Naya; Haar, Laura; McKinnon, Jonathan; Alam, Sharmeen; Weiner, Elaine; Herbert, Carol; Donahue, Craig; Carter, Cynthia; Scala, Stephanie; Ventura, Nicholas; Jara, Jordan; Vytopil, Michal; Srinivasan, Jayashri; Ho, Doreen; Kelley, Andrea; Mosmiller, Betsy; Corse, Andrea; Chaudhry, Vinay; Micheels, Angela; Guingrich, Sandra; Snook, Riley; Kincaid, John; Bodkin, Cynthia; Pascuzzi, Robert; Anderson, Amanda; Loor, John; Carnes, Donna; Sohail, Faisal; Maier, Gal; Sokratous, Giannis; Seddigh, Arshira; Mahdi-Rogers, Mohamed; Gollogly, Jakit; Dimitriou, Aikaterini; Norwood, Fiona; Veske, Adife; Kirbas Cavdar, Tugce; Sen, Sedat; Onar, Musa Kazim; Dogan, Baki; Terzi, Murat; Tun, Ozlem; Semiz, Cansu; Becerikli, Gulsar; Kutlu, Ayse; Cavus, Hakan; Aydinlik, Seda; Efendi, Husnu; Orsel, Gulsah; Caliskan, Yagmur; Yilmaz, Ezgi; Acar, Nazire Pinar; Erdem-Ozdamar, Sevim; Ugur, Muzeyyen; Tuga, Celal; Balkan, Bengu; Ozcelik, Pinar; Meherremova, Arzu; Sengun, Ihsan; Bjarbo, Lena; Hietala, Albert; Piehl, Fredrik; Arribas-Ibar, Elisabet; Vidal, Nuria; Rojas Garcia, Ricardo; Querol Gutierrez, Luis Antonio; Diaz-Manera, Jordi; Cortes Vicente, Elena; Mazuela, Gonzalo; Gili, Gisela; Salvado, Maria; Sune, Pilar; Gamez, Josep; Sastre, Maria; Rodriguez de Rivera, Francisco Javier; Lopez Ruiz, Pedro; Fernandez-Fournier, Mireya; Gomez Salcedo, Pilar; Diez Tejedor, Exuperio; Lazaro, Ana; Velez Santamaria, Valentina; Diez Porras, Laura; Julia Palacios, Natalia; Homedes-Pedret, Christian; Alberti Aguilo, Maria Antonia; Shin, Miju; Hwang, Eun Bi; Shin, Ha Young; Lee, Hyung Seok; Kim, Yool-hee; Jung, JinWoo; Jeong, Ha-Neul; Kim, Seung Min; Kim, Miri; Hwang, Ji Ye; Shin, Chae Won; Lim, Jae-Sung; Koo, Dae Lim; Ahn, So-Hyun; Hong, Yoon-Ho; Choi, Eun Hwa; Park, SangAe; Kwon, JuYoung; Koh, Da Yoon; Lee, JiEun; Seok, Jinmyoung; Min, Ju-Hong; Lee, HyeLim; Choi, MiSong; Cho, Eun Bin; Kim, Byoung Joon; Ra, HyeJin; Kang, Hoo Nam; Seok, Hung Youl; Koo, Yong Seo; Lee, Chang Nyoung; Kim, Byung-Jo; Gibson, Tamar; de Visser, Marianne; van der Kooi, Anneke; Yasuda, Ikuko; Uwasa, Kaori; Takeyama, Rumi; Noshiro, Mayuko; Ishiyama, Naoko; Ishiyama, Yoko; Yamamoto, Daisuke; Suzuki, Shuichiro; Saitoh, Masaki; Murahara, Takashi; Kawamata, Jun; Hisahara, Shin; Hayashi, Takashi; Shimohama, Shun; Tsuda, Emiko; Imai, Tomihiro; Osakada, Kaori; Kobayashi, Hisako; Higuchi, Eriko; Hasuike, Tomoya; Taichi, Namie; Takahashi, Masanori; Makamori, Masayuki; Kawamura, Tomohiro; Funaka, Soichiro; Okumura, Meinoshin; Sato, Miwako; Fukuda, Yusuke; Meguro, Fumie; Kurihara, Rikako; Tsukita, Kenichi; Tano, Ohito; Narikawa, Koichi; Akiyama, Tetsuya; Suzuki, Yasushi; Yamashita, Yumi; Tsurumaru, Masako; Takada, Mihoko; Takada, Kazumi; Maeda, Yasuko; Koga, Megumi; Bekircan-Kurt, CAN EBRU; Vu, Hong; Nye, Joan; Yang, Irene; Robeson, Kimberly; Patwa, Huned; Kumar, Aditya; Keung, Benison; Dicapua, Daniel; Vanderhook, Mollie; Hewlett, Jasmine; Wheeler, Lauren; Liu, Ye; Pillai, Rekha; Hendersen, Kendrick; Arnold, Thomas; Bertorini, Tulio; Kim, Bryan; Downing, Sharon; Strom, Susan; Distad, Jane; Weiss, Michael; Lucy, Shannon; Waheed, Waqar; Hehir, Michael; Kobashikawa, Tomomi; Amamoto, Shoko; Yoshida, Seiko; Asada, Shizuka; Yoshimura, Shunsuke; Mukaino, Akihiro; Motomura, Masakatsu; Mitazaki, Teiichiro; Shiraishi, Hirokazu; Miyawaki, Eriko; Kamakura, Sachiko; Samukawa, Makoto; Morikawa, Miyuki; Suzuki, Hidekazu; Saito, Yuka; Mizugami, Aya; Ozawa, Yukiko; Futono, Kazumi; Fujii, Yuko; Tsuda, Tomoko; Kamegamori, Ikuko; Nagane, Yuriko; Sano, Yoshiko; Nakamura, Yumiko; Kikutake, Keiko; Jingu, Maki; Shimpo, Misa; Uehara, Taira; Matsushita, Takuya; Matsuse, Dai; Masaki, Katsuhisa; Kobayashi, Eri; Kanzaki, Akiko; Kaneko, Yoko; Mori, Masahiro; Kawaguchi, Naoki; Kanai, Tetsuya; Uzawa, Akiyuki; Gabriele, Maria; Frasca, Vittorio; Onesti, Emanuela; Lopergolo, Diego; Gori, Maria; Giacomelli, Elena; Fionda, Laura; Inghilleri, Maurizio; Iorio, Raffaele; D'Amato, Valentina; Alboini, Paolo Emilio; Evoli, Amelia; Campanella, Angela; Pasanisi, Maria; Maggi, Lorenzo; Locatelli, Alberta; Camera, Giorgia; Bonanno, Silvia; Antozzi, Carlo; Puorro, Giorgia; Marsili, Angela; Fasanaro, Angiola; Saccà, Francesco; Marano, Enrico; Costabile, Teresa; Filla, Allessandro; Troili, Fernanda; Morino, Stefania; Garibaldi, Matteo; Di Pasquale, Antonella; Antonini, Giovanni; Konyane, Monika; Varga, Edina; Dezsi, Livia; Vecsei, Laszlo; Szabadosne, Brigitta; Jakab, Gedeonne; Szabo, Gyorgyi; Matolcsi, Judit; Lovas, Gabor; Horvath, Melinda; Rozsa, Csilla; Eriksson, Jaana; Harrison, Tuula; Oksaranta, Olli; Laaksonen, Mikko; Eralinna, Juha-Pekka; Pedersen, Jane; Ostergaard Autzen, Anne; Witting, Nanna; Højgaard, Joan; Mogensen, Anita; Krogh, Susanne; Vinge, Lotte; Harbo, Thomas; Wolfova, Marcela; Jurajdova, Ivana; Tyblova, Michaela; Novakova, Iveta; Pitha, Jiri; Zapletalova, Olga; Reguliova, Katarina; Kurkova, Barbora; Junkerova, Jana; Toncrova, Stanislava; Cierny, Marek; Chmelikova, Magda; Bednarik, Josef; Vohanka, Stanislav; Watt, Elizabeth; Roderus, Rikki; Mallon, Ashley; Blackmore, Derrick; Narayan, Jeffrey; Phan, Cecile; Siddiqi, Zaeem; Fernandes, Patricia; Souza, Iandra; Miranda, Carolina; Carrara, Fernanda; Assis, Natália; Pinto, Wladimir; Cavalcante Lino, Valeria; Duarte Silva, Liene; Annes, Marcelo; Amaral Andrade, Ana Carolina; Souza Bulle Oliveira, Acary; de Cassia Santos, Aline; Pinto, Graziela; Macagnan, Ana Paula; Ferreira, Luciana; da Cruz Pacheco, Daiane; Pereira Martins, Maurer; Mohr, Henrique; Guerreiro, Alexandre; Gheller Friedrich, Maurício André; Souza Duca, Luciana; Renata Cubas Volpe, Luciana; Fialho, Tomás Augusto Suriane; Pazetto, Lucas Eduardo; Otavio Maia Gonçalves, Luiz; Gomes da Silva, Tamires Cristina; Duran, Gustavo; Butinhao, Cristiani Fernanda; Martineli Torres, Debora Dada; Prando, Natalia; Pozo, Marina; Paiva, Gabriel; Nogueira Coelho, Erica; Ananias Morita, Maria da Penha; Gonçalves Geraldo, Jordana; Santos Engel, Murilo; da Silva, Luiz Augusto; Veiga, Thabata; Beck, Kate; Massey, Janice; Hobson-Webb, Lisa; Guptill, Jeffrey; Juel, Vern; Tierney, Scott; Schommer, Clara; Nemeth, Joanne; Kramer, Megan; Sanjak, Mohammad; Cunningham, Scott; Burdette, Maryanne; Plevka, Sarah; Brooks, Benjamin; Bravver, Elena; Forshew, Dallas; Engel, Marguerite; Miller, Robert; Katz, Jonathan; Zakalik, Karen; Patrick, Kara; Silvestri, Nicholas; Wolfe, Gil; Toska, Shirli; Amato, Katherine; Roe, Kristen; Salajegheh, Mohammed; Cochrane, Thomas; Amato, Anthony; Watling, Dave; Davies, Debbie; Arndt, Heike; Sathasivam, Sivakumar; Shah, Amna; Brown, Claire; Torane, Pravin; Sadalage, Girija; Bezerra Rosa, Bruno; Rocha, Rosana; Martins Ribeiro, Renata; Melo, Ana Paula; César, Pamela; Ambrosio, Daniel; Feder, David; Dias Brockhausen, Igor; Alves de Siqueira Carvalho, Alzira; Tilkin, Petra; D'Hondt, Ann; Vynckier, Jan; Verjans, Sarah; Vanhee, Annelies; Vandenbussche, Nicolas; Van Daele, Sien; Stevens, Olivier; Smetcoren, Charlotte; Schotte, Caroline; Depreitere, Annelies; Van Damme, Philip; Wagemaekers, Linda; Mahieu, Délphine; Mercelis, Rudy; De Mey, Katrien; de Koning, Kathy; Van den Abeele, Guy; Garcia, Alejandra Dalila; Vidal, Gonzalo; Chaves, Marcelo; Bettini, Mariela; Rugiero, Marcelo; Saba, Juliet; Barroso, Fabio; Wilken, Miguel; Mazia, Claudio Gabriel; Howard, James F.; Fujita, Kenji P.; Wang, Jing Jing; O'Brien, Fanny L.; Mantegazza, Renato; Vu, Tuan H.; de Bleecker, Jan L.; Casasnovas, Carlos; Andersen, Henning; Nowak, Richard J.; Kissel, John T.; Burns, Ted M.; Vissing, John; Jacob, Saiju; Illa, Isabel; Barohn, Richard J.; Murai, Hiroyuki; Benatar, Michael; Utsugisawa, Kimiaki; Muppidi, Srikanth |
| Publication Year: |
2019 |
| Description: |
© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.Introduction: Eculizumab is effective and well tolerated in patients with antiacetylcholine receptor antibody-positive refractory generalized myasthenia gravis (gMG; REGAIN; NCT01997229). We report an interim analysis of an open-label extension of REGAIN, evaluating eculizumab's long-term safety and efficacy. Methods: Eculizumab (1,200 mg every 2 weeks for 22.7 months [median]) was administered to 117 patients. Results: The safety profile of eculizumab was consistent with REGAIN; no cases of meningococcal infection were reported during the interim analysis period. Myasthenia gravis exacerbation rate was reduced by 75% from the year before REGAIN (P < 0.0001). Improvements with eculizumab in activities of daily living, muscle strength, functional ability, and quality of life in REGAIN were maintained through 3 years; 56% of patients achieved minimal manifestations or pharmacological remission. Patients who had received placebo during REGAIN experienced rapid and sustained improvements during open-label eculizumab (P < 0.0001). Discussion: These findings provide evidence for the long-term safety and sustained efficacy of eculizumab for refractory gMG. Muscle Nerve 2019. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| DOI: |
10.1002/mus.26447 |
| Availability: |
https://doi.org/10.1002/mus.26447; https://avesis.hacettepe.edu.tr/publication/details/fdb2384f-9e84-4ca3-9627-5565dcb089b0/oai |
| Rights: |
info:eu-repo/semantics/closedAccess |
| Accession Number: |
edsbas.CD78A4C2 |
| Database: |
BASE |