2020
Consistent improvement with eculizumab across muscle groups in myasthenia gravis
Mantegazza R, O'Brien F, Yountz M, Howard J, Mazia C, Wilken M, Barroso F, Saba J, Rugiero M, Bettini M, Chaves M, Vidal G, Garcia A, 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, de Siqueira Carvalho A, Brockhausen I, Feder D, Ambrosio D, César P, Melo A, Ribeiro R, Rocha R, Rosa B, Veiga T, da Silva L, Engel M, Geraldo J, da Penha Ananias Morita M, Coelho E, Paiva G, Pozo M, Prando N, Torres D, Butinhao C, Duran G, Fialho T, da Silva T, Gonçalves L, Pazetto L, Volpe L, Duca L, Friedrich M, Guerreiro A, Mohr H, Martins M, da Cruz Pacheco D, Ferreira L, Macagnan A, Pinto G, de Cassia Santos A, Oliveira A, de Andrade A, Annes M, Silva L, 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, Andersen H, Harbo T, Vinge L, Krogh S, Mogensen A, Vissing J, Højgaard J, Witting N, Autzen A, Pedersen J, Erälinna J, 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, Pasquale A, Evoli A, Alboini P, D’Amato V, Iorio R, Inghilleri M, Fionda L, Frasca V, Giacomelli E, Gori M, Lopergolo D, Onesti E, Frasca V, Gabriele M, Saccà F, Filla A, Costabile T, Marano E, Fasanaro A, Marsili A, Puorro G, Antozzi C, Bonanno S, Camera G, Locatelli A, Maggi L, Pasanisi M, Campanella A, 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, Okumura M, Funaka S, Kawamura T, Nakamori M, Takahashi M, Taichi N, Hasuike T, Higuchi E, Kobayashi H, Osakada K, Shiraishi H, Miyazaki 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, Imai T, Tsuda E, 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, van der Kooi A, de Visser M, Gibson T, Kim B, Lee C, Koo Y, Seok H, Kang H, Ra H, Kim B, Bin Cho E, Choi M, Lee H, Min J, Seok J, Lee J, Koh D, Kwon J, Park S, Choi E, Hong Y, Ahn S, Koo D, Lim J, Shin C, Hwang J, Kim M, Kim S, Jeong H, Jung J, Kim Y, Lee H, Shin H, Hwang E, Shin M, Casasnovas C, Aguilo M, Homedes‐Pedret C, Palacios N, Porras L, Santamaria V, Lazaro A, Carbonell J, Sune P, Figueras M, Gili G, Mazuela G, Illa I, Vicente E, Diaz‐Manera J, Gutiérrez L, Garcia R, Vidal N, Arribas‐Ibar E, Tejedor E, Salcedo P, Fernandez‐Fournier M, Ruiz P, de Rivera F, Fernandez‐Fournier M, Sastre M, Piehl F, Hietala A, Bjarbo L, Sengun I, Meherremova A, Ozcelik P, Balkan B, Tuga C, Ugur M, Erdem‐Ozdamar S, Bekircan‐Kurt C, Acar N, 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, Sen S, Cavdar T, Veske A, Norwood F, Dimitriou A, Gollogly J, Mahdi‐Rogers M, Seddigh A, Sokratous G, Maier G, Sohail F, Jacob S, 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, Kramer M, Nemeth J, Schommer C, Scott T, 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, 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, Kissel J, 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, Ardoin A, Darnell T, Golden L, Lokaitis B, Seelbach J, Muppidi S, Goyal N, Sakamuri S, So Y, Paulose S, Pol S, Welsh L, Bhavaraju‐Sanka R, 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, Rosenfeld J, Garcia R, Echevarria L, Garcia S, Pulley M, Aranke S, Berger A, 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, Sheldon D, Steele 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. Consistent improvement with eculizumab across muscle groups in myasthenia gravis. Annals Of Clinical And Translational Neurology 2020, 7: 1327-1339. PMID: 32700461, PMCID: PMC7448154, DOI: 10.1002/acn3.51121.Peer-Reviewed Original ResearchConceptsAnti-acetylcholine receptorOpen-label extensionMyasthenia Gravis ActivitiesMyasthenia gravisDaily Living ProfileMuscle groupsLiving ProfilesDomain scoresOpen-label extension trialQuantitative myasthenia gravis scalePhysician-reported outcomesTerminal complement inhibitorEculizumab treatmentExtension trialMuscle strengthSustained improvementPatientsGravisComplement inhibitorsREGAIN studyTotal scoreTreatment elicitsRapid improvementPlaceboEculizumab
2017
Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study
Howard JF, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Muppidi S, Nowak RJ, O'Brien F, Wang JJ, Mantegazza R, Group R, Mazia C, Wilken M, Ortea C, Saba J, Rugiero M, Bettini M, Vidal G, Garcia A, Lamont P, Leong W, Boterhoven H, Fyfe B, Roberts L, Jasinarachchi M, Willlems N, Wanschitz J, Löscher W, De Bleecker J, Van den Abeele G, de Koning K, De Mey K, Mercelis R, Wagemaekers L, Mahieu D, Van Damme P, Smetcoren C, Stevens O, Verjans S, D'Hondt A, Tilkin P, de Siqueira Carvalho A, Hasan R, Brockhausen I, Feder D, Ambrosio D, Melo A, Rocha R, Rosa B, Veiga T, da Silva L, Geraldo J, da Penha Morita Ananias M, Coelho E, Paiva G, Pozo M, Prando N, Torres D, Butinhao C, Coelho E, Volpe L, Duran G, da Silva T, Gonçalves L, Pazetto L, Duca L, Fialho T, Friedrich M, Guerreiro A, Mohr H, Martins M, da Cruz Pacheco D, Macagnan A, de Cassia Santos A, Oliveira A, de Andrade A, Annes M, Lino V, Pinto W, Miranda C, Carrara F, Souza I, Genge A, Massie R, Campbell N, Bril V, Katzberg H, Soltani M, Ng E, Siddiqi Z, Phan C, Blackmore D, Vohanka S, Bednarik J, Chmelikova M, Cierny M, Toncrova S, Junkerova J, Kurkova B, Reguliova K, Zapletalova O, Pitha J, Novakova I, Tyblova M, Wolfova M, Jurajdova I, Andersen H, Harbo T, Vinge L, Mogensen A, Højgaard J, Witting N, Autzen A, Pedersen J, Färkkilä M, Atula S, Nyrhinen A, Erälinna J, Laaksonen M, Oksaranta O, Eriksson J, Harrison T, Desnuelle C, Sacconi S, Soriani M, Decressac S, Moutarde J, Lahaut P, Solé G, Le Masson G, Wielanek-Bachelet A, Gaboreau M, Moreau C, Wilson A, Vial C, Bouhour F, Gervais-Bernard H, Merle H, Hourquin C, Lacour A, Outteryck O, Vermersch P, Zephir H, Millois E, Deneve M, Deruelle F, Schoser B, Wenninger S, Stangel M, Alvermann S, Gingele S, Skripuletz T, Suehs K, Trebst C, Fricke K, Papagiannopoulos S, Bostantzopoulou S, Vlaikidis N, Zampaki M, Papadopoulou N, Mitsikostas D, Kasioti E, Mitropoulou E, Charalambous D, Rozsa C, Horvath M, Lovas G, Matolcsi J, Szabo G, Szabadosne B, Vecsei L, Dezsi L, Varga E, Konyane M, Gross B, Azrilin O, Greenbereg N, Kuperman H, Antonini G, Garibaldi M, Morino S, Troili F, Di Pasquale A, Filla A, Costabile T, Marano E, Sacca F, Marsil A, Puorro G, Tassoni M, De Rosa A, Bonanno S, Antozzi C, Maggi L, Campanella A, Angelini C, Cudia P, Pegoraro V, Pinzan E, Bevilacqua F, Orrico D, Bonifati D, Evoli A, Alboini P, D'Amato V, Iorio R, Inghilleri M, Fionda L, Frasca V, Giacomelli E, Gori M, Lopergolo D, Onesti E, Gabriele M, Patti F, Caramma A, Messina S, Reggio E, Caserta C, Uzawa A, Kanai T, 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, Fujii Y, Futono K, Tsuda T, Saito Y, Suzuki H, Morikawa M, Samukawa M, Kamakura S, Shiraishi H, Mitazaki T, Motomura M, Mukaino A, Yoshimura S, Asada S, Kobashikawa T, Koga M, Maeda Y, Takada K, Takada M, Yamashita Y, Yoshida S, Suzuki Y, Akiyama T, Narikawa K, Tsukita K, Meguro F, Fukuda Y, Sato M, Matsuo H, Fukudome T, Gondo Y, Maeda Y, Nagaishi A, Nakane S, Okubo Y, Okumura M, Funaka S, Kawamura T, Makamori M, Takahashi M, Hasuike T, Higuchi E, Kobayashi H, Osakada K, Taichi N, Tsuda E, Hayashi T, Hisahara S, Imai T, Kawamata J, Murahara T, Saitoh M, Shimohama S, Suzuki S, Yamamoto D, Konno S, Imamura T, Inoue M, Murata M, Nakazora H, Nakayama R, Ikeda Y, Ogawa M, Shirane M, Kanda T, Kawai M, Koga M, Ogasawara J, Omoto M, Sano Y, Arima H, Fukui S, Shimose S, Shinozaki H, Watanabe M, Yoshikawa C, van der Kooi A, de Visser M, Gibson T, Maessen J, de Baets M, Faber C, Keijzers M, Miesen M, Kostera-Pruszczyk A, Kaminska A, Kim B, Lee C, Koo Y, Seok H, Kang H, Ra H, Kim B, Bin Cho E, Lee H, Min J, Seok J, Koh D, Kwon J, Lee J, Park S, Hong Y, Lim J, Kim M, Kim S, Kim Y, Lee H, Shin H, Hwang E, Shin M, Sazonov D, Yarmoschuk A, Babenko L, Malkova N, Melnikova A, Korobko D, Kosykh E, Pokhabov D, Nesterova Y, Abramov V, Balyazin V, Pons C, Aguilo M, Homedes-Pedret C, Palacios N, Lazaro A, Tejedor E, Fernandez-Fournier M, Ruiz P, de Rivera F, Figueras M, Gamez J, Salvado M, Vicente E, Diaz-Manera J, Gutierrez L, Garcia R, Vidal N, Arribas-Ibar E, Piehl F, Hietala A, Bjarbo L, Lindberg C, Jons D, Andersson B, Sengun I, Ozcelik P, Tuga C, Ugur M, Boz C, Altiparmak D, Gazioglu S, Aydin C, Erdem-Ozdamar S, Bekircan-Kurt C, Yilmaz E, Acar N, Caliskan Y, Efendi H, Aydinlik S, Cavus H, Semiz C, Tun O, Terzi M, Dogan B, Onar M, Sen S, Cavdar T, Norwood F, Dimitriou A, Gollogly J, Mahdi-Rogers M, Seddigh A, Maier G, Sohail F, Sathasivam S, Arndt H, Davies D, Watling D, Rivner M, Hartmann J, Quarles B, Smalley N, 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, Bravver E, Brooks B, Plevka S, Burdette M, Sanjak M, Kramer M, Nemeth J, Schommer C, Juel V, Guptill J, Hobson-Webb L, Beck K, Carnes D, Loor J, Anderson A, Lange D, Agopian E, Goldstein J, Manning E, Kaplan L, Holzberg S, Kassebaum N, Pascuzzi R, Bodkin C, Kincaid J, Snook R, Guinrich S, Micheels A, Chaudhry V, Corse A, Mosmiller B, Ho D, Srinivasan J, Vytopil M, Ventura N, Scala S, Carter C, Donahue C, Herbert C, Weiner E, McKinnon J, Haar L, McKinnon N, Alcon K, Daniels K, Sattar N, Jeffery D, McKenna K, Guidon A, David W, Dheel C, Levine-Weinberg M, Nigro C, Simpson E, Appel S, Lai E, Lay L, Pleitez M, Halton S, Faigle C, Thompson L, Sivak M, Shin S, Bratton J, Jacobs D, Brown G, Bandukwala I, Brown M, Kane J, Blount I, Freimer M, Hoyle J, Agriesti J, Khoury J, Marburger T, Kaur H, Dimitrova D, Mellion M, Sachs G, Crabtree B, Keo R, Perez E, Taber S, Gilchrist J, Andoin A, Darnell T, Goyal N, Sakamuri S, So Y, Welsh L, Bhavaraju-Sanka R, Gonzalez A, Jones F, Saklad A, Nations S, Trivedi J, Hopkins S, Kazamel M, Alsharabati M, Lu L, Mumfrey-Thomas S, Woodall A, Richman D, Butters J, Lindsay M, Mozaffar T, Cash T, Goyal N, Roy G, Mathew V, Maqsood F, Minton B, Jones H, Rosenfeld J, Garcia R, Garcia S, Echevarria L, Pulley M, Aranke S, Berger A, Shah J, Shabbir Y, Smith L, Varghese M, Gutmann L, Gutmann L, Swenson A, Olalde H, Hafer-Macko C, Kwan J, Zilliox L, Callison K, DiSanzo B, Naunton K, Bilsker M, Sharma K, Reyes E, Cooley A, Michon S, Steele J, Karam C, Chopra M, Bird S, Kaufman J, Gallatti N, Vu T, Katzin L, McClain T, Harvey B, Hart A, Huynh K, Beydoun S, Chilingaryan A, Droker B, Lin F, Shah A, Tran A, Akhter S, Malekniazi A, Tandan R, Hehir M, Waheed W, Lucy S, Weiss M, Distad J, Downing S, Strom S, Lisak R, Bernitsas E, Khan O, Sriwastava S, Tselis A, Jia K, Bertorini T, Arnold T, Henderson K, Pillai R, Liu Y, Wheeler L, Hewlett J, Vanderhook M, Dicapua D, Keung B, Kumar A, Patwa H, Robeson K, Nye J, Vu H. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. The Lancet Neurology 2017, 16: 976-986. PMID: 29066163, DOI: 10.1016/s1474-4422(17)30369-1.Peer-Reviewed Original ResearchConceptsAnti-acetylcholine receptor antibody-positive refractoryMyasthenia gravisEculizumab groupImmunosuppressive therapyIntravenous immunoglobulinPlacebo groupMulticentre studyPlasma exchangeUpper respiratory tract infectionEfficacy of eculizumabHistory of thymomaMyasthenia Gravis ActivitiesMyasthenia gravis exacerbationMyasthenia gravis therapySafety of eculizumabCommon adverse eventsPrimary efficacy endpointDaily living scorePhase 2 studyPhase 3 trialRespiratory tract infectionsWeb response systemMyasthenia Gravis FoundationPrimary endpoint resultsTerminal complement inhibitor