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‘Minimal symptom expression’ in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab
- Authors
- Vissing, John; Jacob, Saiju; Fujita, Kenji P.; O’Brien, Fanny; Howard, James F.; Mazia, C.G.; Wilken, M.; Barroso, F.; Saba, J.; Rugiero, M.; Bettini, M.; Chaves, M.; Vidal, G.; Garcia, A.D.; De Bleecker, J.; Van den, Abeele G.; de Koning, K.; De Mey, K.; Mercelis, R.; Mahieu, D.; Wagemaekers, L.; Van Damme, P.; Depreitere, A.; Schotte, C.; Smetcoren, C.; Stevens, O.; Van Daele, S.; Vandenbussche, N.; Vanhee, A.; Verjans, S.; Vynckier, J.; D’Hont, A.; Tilkin, P.; Alves, de Siqueira Carvalho A.; Dias Brockhausen, I.; Feder, D.; Ambrosio, D.; César, P.; Melo, A.P.; Martins Ribeiro, R.; Rocha, R.; Rosa, B.B.; Veiga, T.; da Silva, L.A.; Santos Engel, M.; Gonçalves Geraldo, J.; da Penha, Ananias Morita M.; Nogueira Coelho, E.; Paiva, G.; Pozo, M.; Prando, N.; Martineli Torres, D.D.; Butinhao, C.F.; Duran, G.; Suriane Fialho, T.A.; Gomes, da Silva T.C.; Maia Gonçalves, L.O.; Pazetto, L.E.; Cubas Volpe, L.R.; Souza Duca, L.; Gheller Friedrich, M.A.; Guerreiro, A.; Mohr, H.; Pereira Martins, M.; da Cruz, Pacheco D.; Ferreira, L.; Macagnan, A.P.; Pinto, G.; de Cassia, Santos A.; Souza, Bulle Oliveira A.; Amaral, de Andrade A.C.; Annes, M.; Duarte Silva, L.; Cavalcante Lino, V.; Pinto, W.; Assis, N.; Carrara, F.; Miranda, C.; Souza, I.; Fernandes, P.; Siddiqi, Z.; Phan, C.; Narayan, J.; Blackmore, D.; Mallon, A.; Roderus, R.; Watt, E.; Vohanka, S.; Bednarik, J.; Chmelikova, M.; Cierny, M.; Toncrova, S.; Junkerova, Barbora Kurkova J.; Reguliova, K.; Zapletalova, O.; Pitha, J.; Novakova, I.; Tyblova, M.; Jurajdova, I.; Wolfova, M.; Andersen, H.; Harbo, T.; Vinge, L.; Krogh, S.; Mogensen, A.; Vissing, J.; Højgaard, J.; Witting, N.; Mette, Ostergaard Autzen A.; Pedersen, J.; Erälinna, J.-P.; Laaksonen, M.; Oksaranta, O.; Harrison, T.; Eriksson, J.; Rozsa, C.; Horvath, M.; Lovas, G.; Matolcsi, J.; Szabo, G.; Jakab, G.; Szabadosne, B.; Vecsei, L.; Dezsi, L.; Varga, E.; Konyane, M.; Antonini, G.; Di Pasquale, A.; Garibaldi, M.; Morino, S.; Troili, F.; Fionda, L.; Saccà, F.; previous,; Filla, A.; sub-investigators,; Costabile, T.; Marano, E.; Fasanaro, A.; Marsili, A.; Puorro, G.; Mantegazza, R.; Antozzi, C.; Bonanno, S.; Camera, G.; Locatelli, A.; Maggi, L.; Pasanisi, M.; Campanella, A.; Evoli, A.; Alboini, P.E.; D’Amato, V.; Iorio, R.; Inghilleri, M.; Fionda, L.; Frasca, V.; Giacomelli, E.; Gori, M.; Lopergolo, D.; Onesti, E.; Frasca, V.; Gabriele, M.; Uzawa, A.; Kanai, T.; Kawaguchi, N.; Mori, M.; Kaneko, Y.; Kanzaki, A.; Kobayashi, E.; Murai, H.; Masaki, K.; Matsuse, D.; Matsushita, T.; Uehara, T.; Shimpo, M.; Jingu, M.; Kikutake, K.; Nakamura, Y.; Sano, Y.; Utsugisawa, K.; Nagane, Y.; Kamegamori, I.; Tsuda, T.; Fujii, Y.; Futono, K.; Ozawa, Y.; Mizugami, A.; Saito, Y.; Samukawa, M.; Suzuki, H.; Morikawa, M.; Kamakura, S.; Miyawaki, E.; Shiraishi, H.; Mitazaki, T.; Motomura, M.; Mukaino, A.; Yoshimura, S.; Asada, S.; Yoshida, S.; Amamoto, S.; Kobashikawa, T.; Koga, M.; Maeda, Y.; Takada, K.; Takada, M.; Tsurumaru, M.; Yamashita, Y.; Yoshida, S.; Suzuki, Y.; Akiyama, T.; Narikawa, K.; Tano, O.; Tsukita, K.; Kurihara, R.; Meguro, F.; Fukuda, Y.; Sato, M.; Okumura, M.; Funaka, S.; Kawamura, T.; Makamori, M.; Takahashi, M.; Taichi, N.; Hasuike, T.; Higuchi, E.; Kobayashi, H.; Osakada, K.; Imai, T.; Tsuda, E.; previous,; Shimohama, S.; Hayashi, T.; Hisahara, S.; Imai, T.; Kawamata, J.; Murahara, T.; Saitoh, M.; Shimohama, S.; Suzuki, S.; Yamamoto, D.; Ishiyama, Y.; Ishiyama, N.; Noshiro, M.; Takeyama, R.; Uwasa, K.; Yasuda, I.; Kim, Byung-Jo; Lee, C.N.; Koo, Y.S.; Seok, H.Y.; Kang, H.N.; Ra, H.J.; Kim, B.J.; Cho, E.B.; Choi, M.S.; Lee, H.L.; Min, J.-H.; Seok, J.; Lee, J.E.; Koh, D.Y.; Kwon, J.Y.; Park, S.A.; Choi, E.H.; Hong, Y.-H.; Ahn, S.-H.; Koo, D.L.; Lim, J.-S.; Shin, C.W.; Hwang, J.Y.; Kim, M.; Kim, S.M.; Jeong, H.-N.; Jung, J.W.; Kim, Y.-H.; Lee, H.S.; Shin, H.Y.; Hwang, E.B.; Shin, M.; van, der Kooi A.; de Visser, M.; Gibson, T.; Casasnovas, C.; Alberti Aguilo, M.A.; Homedes-Pedret, C.; Palacios, N.J.; Diez Porras, L.; Velez Santamaria, V.; Lazaro, A.; Diez Tejedor, E.; Gomez Salcedo, P.; Fernandez-Fournier, M.; Lopez Ruiz, P.; Rodriguez, de Rivera F.J.; Fernandez-Fournier, M.; Sastre, M.; Gamez Carbonell, J.; Sune, P.; Salvado Figueras, M.; Gili, G.; Mazuela, G.; Illa, I.; Cortes Vicente, E.; Diaz-Manera, J.; Querol Gutiérrez, L.A.; Rojas Garcia, R.; Vidal, N.; Arribas-Ibar, E.; Piehl, F.; Hietala, A.; Bjarbo, L.; Sengun, I.; Meherremova, A.; Ozcelik, P.; Balkan, B.; Tuga, C.; Ugur, M.; Erdem-Ozdamar, S.; Bekircan-Kurt, C.E.; Acar, N.P.; Yilmaz, E.; Caliskan, Y.; Orsel, G.; Efendi, H.; Aydinlik, S.; Cavus, H.; Kutlu, A.; Becerikli, G.; Semiz, C.; Tun, O.; Terzi, M.; Dogan, B.; Onar, M.K.; Sen, S.; Kirbas Cavdar, T.; Veske, A.; Norwood, F.; Dimitriou, A.; Gollogly, J.; Mahdi-Rogers, M.; Seddigh, A.; Sokratous, G.; Maier, G.; Sohail, F.; Sadalage, G.; Torane, P.; Brown, C.; Shah, A.; Sathasivam, S.; Arndt, H.; Davies, D.; Watling, D.; Amato, A.; Cochrane, T.; Salajegheh, M.; Roe, K.; Amato, K.; Toska, S.; Wolfe, G.; Silvestri, N.; Patrick, K.; Zakalik, K.; Katz, J.; Miller, R.; Engel, M.; Forshew, D.; Bravver, E.; Brooks, B.; Sanjak, M.; Plevka, S.; Burdette, M.; Cunningham, S.; Sanjak, M.; Kramer, M.; Nemeth, J.; Schommer, C.; Tinerney, S.; Juel, V.; Guptill, J.; Hobson-Webb, L.; Massey, J.; Beck, K.; Carnes, D.; Loor, J.; Anderson, A.; Pascuzzi, R.; Bodkin, C.; Kincaid, J.; Snook, R.; Guinrich, S.; Micheels, A.; Chaudhry, V.; Corse, A.; Mosmiller, B.; Kelley, A.; Ho, D.; Srinivasan, J.; Vytopil, M.; Jara, J.; Ventura, N.; Carter, C.; Donahue, C.; Herbert, C.; Scala, S.; Weiner, E.; Alam, S.; McKinnon, J.; Haar, L.; McKinnon, N.; Alcon, K.; McKenna, K.; Sattar, N.; Daniels, K.; Jeffery, D.; Freimer, M.; Hoyle, J.C.; Kissel, J.; Agriesti, J.; Chelnick, S.; Mezache, L.; Pineda, C.; Muharrem, F.; Karam, C.; Khoury, J.; previous,; Marburger, T.; Kaur, H.; Dimitrova, D.; Gilchrist, J.; Agrawal, B.; Elsayed, M.; Kohlrus, S.; Andoin, A.; Darnell, T.; Golden, L.; Lokaitis, B.; Seelbach, J.; Muppidi, S.; Goyal, N.; Sakamuri, S.; So, Y.T.; Paulose, S.; Pol, S.; Welsh, L.; Bhavaraju-Sanka, R.; Tobon Gonzalez, A.; Dishman, L.; Jones, F.; Gonzalez, A.; Padilla, P.; Saklad, A.; Silva, M.; Nations, S.; Trivedi, J.; Hopkins, S.; Kazamel, M.; Alsharabati, M.; Lu, L.; Nozaki, K.; Mumfrey-Thomas, S.; Woodall, A.; Mozaffar, T.; Cash, T.; Goyal, N.; Roy, G.; Mathew, V.; Maqsood, F.; Minton, B.; Jones, H.J.; Rosenfeld, J.; Garcia, R.; Echevarria, L.; Garcia, S.; Pulley, M.; Aranke, S.; Berger, A.R.; Shah, J.; Shabbir, Y.; Smith, L.; Varghese, M.; Shabbir, Y.; Gutmann, L.; Gutmann, L.; Jerath, N.; Nance, C.; Swenson, A.; Olalde, H.; Kressin, N.; Sieren, J.; Barohn, R.; Dimachkie, M.; Glenn, M.; McVey, A.; Pasnoor, M.; Statland, J.; Wang, Y.; Liu, T.; Emmons, K.; Jenci, N.; Locheke, J.; Fondaw, A.; Johns, K.; Rico, G.; Walsh, M.; Herbelin, L.; Hafer-Macko, C.; Kwan, J.; Zilliox, L.; Callison, K.; Young, V.; DiSanzo, B.; Naunton, K.; Benatar, M.; Bilsker, M.; Sharma, K.; Cooley, A.; Reyes, E.; Michon, S.-C.; Sheldon, D.; Steele, J.; Howard, J.; Karam, C.; Traub, R.; Chopra, M.; Vu, T.; Katzin, L.; McClain, T.; Harvey, B.; Hart, A.; Huynh, K.; Beydoun, S.; Chilingaryan, A.; Doan, V.; Droker, B.; Gong, H.; Karimi, S.; Lin, F.; McClain, T.; Polaka, K.; Shah, A.; Tran, A.; Akhter, S.; Malekniazi, A.; Tandan, R.; Hehir, M.; Waheed, W.; Lucy, S.; Weiss, M.; Distad, J.; Strom, S.; Downing, S.; Kim, B.; Bertorini, T.; Arnold, T.; Henderson, K.; Pillai, R.; Liu, Y.; Wheeler, L.; Hewlett, J.; Vanderhook, M.; Nowak, R.; Dicapua, D.; Keung, B.; Kumar, A.; Patwa, H.; Robeson, K.; Yang, I.; Nye, J.; Vu, H.; The, REGAIN study group
- Issue Date
- Jul-2020
- Publisher
- Dr. Dietrich Steinkopff Verlag
- Keywords
- Eculizumab; Refractory; Myasthenia gravis; Minimal symptom expression; Acetylcholine receptor
- Citation
- Journal of Neurology, v.267, no.7, pp 1991 - 2001
- Pages
- 11
- Indexed
- SCIE
SCOPUS
- Journal Title
- Journal of Neurology
- Volume
- 267
- Number
- 7
- Start Page
- 1991
- End Page
- 2001
- ISSN
- 0340-5354
1432-1459
- Abstract
- Background The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension. Methods Attainment of ‘minimal symptom expression’ was evaluated using patient-reported outcome measures of gMG symptoms [MG activities of daily living scale (MG-ADL), 15-item MG quality of life questionnaire (MG-QOL15)] at the completion of REGAIN and during the open-label extension. ‘Minimal symptom expression’ was defined as MG-ADL total score of 0–1 or MG-QOL15 total score of 0–3. Results At REGAIN week 26, more eculizumab-treated patients achieved ‘minimal symptom expression’ versus placebo [MG-ADL: 21.4% vs 1.7%; difference 19.8%; 95% confidence interval (CI) 8.5, 31.0; p = 0.0007; MG-QOL15: 16.1% vs 1.7%; difference 14.4%; 95% CI 4.3, 24.6; p = 0.0069]. During the open-label extension, the proportion of patients in the placebo/eculizumab group who achieved ‘minimal symptom expression’ increased after initiating eculizumab treatment and was sustained through 130 weeks of open-label eculizumab (MG-ADL: 1.7 to 27.8%; MG-QOL15: 1.7 to 19.4%). At extension study week 130, similar proportions of patients in the eculizumab/eculizumab and placebo/eculizumab groups achieved ‘minimal symptom expression’ (MG-ADL: 22.9% and 27.8%, respectively, p = 0.7861; MG-QOL15: 14.3% and 19.4%, respectively, p = 0.7531). The long-term tolerability of eculizumab was consistent with previous reports. Conclusions Patients with AChR+ refractory gMG who receive eculizumab can achieve sustained ‘minimal symptom expression’ based on patient-reported outcomes. ‘Minimal symptom expression’ may be a useful tool in measuring therapy effectiveness in gMG.
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Related Researcher
- Kim, Byung-Jo
- Anam Hospital (Department of Neurology, Anam Hospital)