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Muscle Nerve. 2019 Jul;60(1):14-24. doi: 10.1002/mus.26447. Epub 2019 Mar 8.

Long-term safety and efficacy of eculizumab in generalized myasthenia gravis.

Collaborators (540)

Mazia CG, Wilken M, Barroso F, Saba J, Rugiero M, Bettini M, Chaves M, Vidal G, Garcia AD, 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'Hondt A, Tilkin P, Alves de Siqueira Carvalho A, Dias Brockhausen I, Feder D, Ambrosio D, César P, Melo AP, Martins Ribeiro R, Rocha R, Bezerra Rosa B, Veiga T, da Silva LA, Santos Engel M, Gonçalves Geraldo J, Ananias Morita MDP, Nogueira Coelho E, Paiva G, Pozo M, Prando N, Martineli Torres DD, Butinhao CF, Duran G, Gomes da Silva TC, Otavio Maia Gonçalves L, Pazetto LE, Fialho TAS, Renata Cubas Volpe L, Souza Duca L, Gheller Friedrich MA, Guerreiro A, Mohr H, Pereira Martins M, da Cruz Pacheco D, Ferreira L, Macagnan AP, Pinto G, de Cassia Santos A, Souza Bulle Oliveira A, Amaral Andrade AC, 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 J, Kurkova B, Reguliova K, Zapletalova O, Pitha J, Novakova I, Tyblova M, Jurajdova I, Wolfova M, Harbo T, Vinge L, Krogh S, Mogensen A, Højgaard J, Witting N, Ostergaard Autzen A, Pedersen J, Eralinna JP, 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, Filla A, Costabile T, Marano E, Saccà F, Fasanaro A, Marsili A, Puorro G, Antozzi C, Bonanno S, Camera G, Locatelli A, Maggi L, Pasanisi M, Campanella A, Evoli A, Alboini PE, 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, Masaki K, Matsuse D, Matsushita T, Uehara T, Shimpo M, Jingu M, Kikutake K, Nakamura Y, Sano Y, Nagane Y, Kamegamori I, Tsuda T, Fujii Y, Futono K, Ozawa Y, Mizugami A, Saito Y, Suzuki H, Morikawa M, Samukawa 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, 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, Shimohama S, Hayashi T, Hisahara S, Kawamata J, Murahara T, Saitoh M, Suzuki S, Yamamoto D, Ishiyama Y, Ishiyama N, Noshiro M, Takeyama R, Uwasa K, Yasuda I, van der Kooi A, de Visser M, Gibson T, Kim BJ, Lee CN, Koo YS, Seok HY, Kang HN, Ra H, Kim BJ, Cho EB, Choi M, Lee H, Min JH, Seok J, Lee J, Koh DY, Kwon J, Park S, Choi EH, Hong YH, Ahn SH, Koo DL, Lim JS, Shin CW, Hwang JY, Kim M, Kim SM, Jeong HN, Jung J, Kim YH, Lee HS, Shin HY, Hwang EB, Shin M, Alberti Aguilo MA, Homedes-Pedret C, Julia Palacios N, Diez Porras L, Velez Santamaria V, Lazaro A, Diez Tejedor E, Gomez Salcedo P, Fernandez-Fournier M, Lopez Ruiz P, Rodriguez de Rivera FJ, Sastre M, Gamez J, Sune P, Salvado M, Gili G, Mazuela G, Cortes Vicente E, Diaz-Manera J, Querol Gutierrez LA, 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 CE, Acar NP, 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 MK, 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, Plevka S, Burdette M, Cunningham S, Sanjak M, Kramer M, Nemeth J, Schommer C, Tierney 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, Guingrich S, Micheels A, Chaudhry V, Corse A, Mosmiller B, Kelley A, Ho D, Srinivasan J, Vytopil M, Jara J, Ventura N, Scala S, Carter C, Donahue C, Herbert C, Weiner E, Alam S, McKinnon J, Haar L, McKinnon N, Alcon K, McKenna K, Sattar N, Daniels K, Jeffery D, Freimer M, Hoyle JC, Agriesti J, Chelnick S, Mezache L, Pineda C, Muharrem F, Karam C, Khoury J, Marburger T, Kaur H, Dimitrova D, Gilchrist J, Agrawal B, Elsayed M, Kohlrus S, Andoin A, Darnell T, Golden L, Lokaitis B, Seelback J, Goyal N, Sakamuri S, So YT, Paulose S, Pol S, Welsh L, Bhavaraju-Sanka R, Tobon Gonzales 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 HJ, Rosenfeld J, Garcia R, Echevarria L, Garcia S, Pulley M, Aranke S, Berger AR, Shah J, Shabbir Y, Smith L, Varghese M, Gutmann L, Gutmann L, Jerath N, Nance C, Swenson A, Olalde H, Kressin N, Sieren J, 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, Bilsker M, Sharma K, Cooley A, Reyes E, Michon SC, Sheldon D, Steele J, Karam C, Chopra M, Traub R, 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, Pokala 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, Hendersen K, Pillai R, Liu Y, Wheeler L, Hewlett J, Vanderhook M, Dicapua D, Keung B, Kumar A, Patwa H, Robeson K, Yang I, Nye J, Vu H.

Abstract

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.

KEYWORDS:

MG-ADL; MG-QOL15; MGC; QMG; eculizumab; myasthenia gravis

PMID:
30767274
DOI:
10.1002/mus.26447

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