2020
Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension
Mantegazza R, Wolfe G, Muppidi S, Wiendl H, Fujita K, O'Brien F, Booth H, 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, Alves de Siqueira Carvalho A, Brockhausen I, Feder D, Ambrosio D, César G, Melo A, Ribeiro R, Rocha R, Rosa B, Veiga T, Augusto da Silva L, Engel M, Geraldo J, da Penha Ananias Morita M, Coelho E, Paiva G, Pozo M, Prando N, Martineli Torres D, Butinhao C, Duran G, Suriane Fialho T, Gomes da Silva T, Maia Gonçalves L, Pazetto L, Cubas Volpe L, Duca L, AndréGheller Friedrich M, Guerreiro A, Guerreiro A, Mohr H, Martins M, da Cruz Pacheco D, Ferreira L, Macagnan A, Pinto G, de Cassia Santos A, Bulle Oliveira A, Amaral 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, Ostergaard Autzen A, Pedersen J, Erälinna J, Laaksonen M, Oksaranta O, Harrison T, Eriksson J, Rozsa C, Horvath M, Lovas G, Matolcsi J, Jakab G, 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, Di 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, Yasuko M, 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, 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, Alberti 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, Querol Gutiérrez L, Garcia R, Vidal N, Arribas-Ibar E, Tejedor E, Salcedo P, Fernandez-Fournier M, Ruiz P, Rodriguez 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, 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, 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, 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. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Neurology 2020, 96: e610-e618. PMID: 33229455, PMCID: PMC7905790, DOI: 10.1212/wnl.0000000000011207.Peer-Reviewed Original ResearchConceptsOpen-label extensionPost-intervention statusOpen-label studyMinimal manifestationsEculizumab treatmentMyasthenia gravisAmerica Post-Intervention StatusMGFA post-intervention statusEculizumab-treated patientsPlacebo-treated patientsRefractory myasthenia gravisClass II evidenceNew safety signalsUse of eculizumabMyasthenia Gravis FoundationPharmacologic remissionWeek 130Safety profileWeek 26Patient populationSafety signalsEculizumabPatient statusPatientsMM status
2019
Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study
Murai H, Uzawa A, Suzuki Y, Imai T, Shiraishi H, Suzuki H, Okumura M, O’Brien F, Wang J, Fujita K, Utsugisawa K, Group I, 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'Hondt 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, da Silva T, Gonçalves L, Pazetto L, Fialho T, Volpe L, Duca L, Oliveira A, 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, 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, Eralinna J, Laaksonen M, Oksaranta O, Harrison T, Eriksson J, Rozsa C, Horvath M, Lovas G, Matolcsi J, Szabo G, Jakab G, Szabadosne B, 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, Mantegazza R, Antozzi C, Bonanno S, Camera G, Locatelli A, Maggi L, Pasanisi M, Campanella A, Evoli A, Alboini P, D'Amato V, Iorio R, 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, Morikawa M, Samukawa M, Kamakura S, Miyawaki E, 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, Akiyama T, Narikawa K, Tano O, Tsukita K, Kurihara R, Meguro F, Fukuda Y, Sato M, Funaka S, Kawamura T, Makamori M, Takahashi M, Taichi N, Hasuike T, Higuchi E, Kobayashi H, Osakada K, 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, Casasnovas C, Aguilo M, Homedes-Pedret C, Palacios N, Porras L, Santamaria V, Lazaro A, Tejedor E, Salcedo P, Fernandez-Fournier M, Ruiz P, de Rivera F, Sastre M, Gamez J, Sune P, Salvado M, Gili G, Mazuela G, Illa I, Vicente E, Diaz-Manera J, Gutierrez L, 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, 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, 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 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, Andoin A, Darnell T, Golden L, Lokaitis B, Seelback J, Muppidi S, Goyal N, Sakamuri S, So Y, Paulose S, Pol S, Welsh L, Bhavaraju-Sanka R, Gonzales A, Dishman L, Jones F, Gonzalez A, Padilla P, Saklad A, Silva M, 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, 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, Howard J, Karam C, Chopra M, Traub R, 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, 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, Nowak R, Dicapua D, Keung B, Kumar A, Patwa H, Robeson K, Yang I, Nye J, Vu H. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study. Journal Of The Neurological Sciences 2019, 407: 116419. PMID: 31698177, DOI: 10.1016/j.jns.2019.08.004.Peer-Reviewed Original ResearchConceptsSafety of eculizumabMyasthenia gravisCaucasian patientsAnti-acetylcholine receptor antibody-positive refractoryTerminal complement inhibitor eculizumabOpen-label extension studyMyasthenia Gravis ActivitiesQuantitative Myasthenia GravisGeneralized myasthenia gravisOpen-label extensionComplement inhibitor eculizumabLong-term efficacyMyasthenia Gravis QualityMyasthenia Gravis CompositeExtension baselineWeek 52Safety profileSubgroup analysisJapanese patientsDaily livingEculizumabGravisExtension studyPatientsREGAIN studyLong‐term safety and efficacy of eculizumab in generalized myasthenia gravis
Muppidi S, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Nowak RJ, Andersen H, Casasnovas C, de Bleecker J, Vu TH, Mantegazza R, O'Brien FL, Wang JJ, Fujita KP, Howard JF, Mazia C, Wilken M, Barroso F, Saba J, Rugiero M, Bettini M, Chaves M, Vidal G, Garcia A, 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, 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, da Silva T, Gonçalves L, Pazetto L, Fialho T, 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, 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, Harbo T, Vinge L, Krogh S, Mogensen A, Højgaard J, Witting N, Autzen A, Pedersen J, Eralinna 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, 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 P, 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 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, Aguilo M, Homedes-Pedret C, Palacios N, Porras L, Santamaria V, Lazaro A, Tejedor E, Salcedo P, Fernandez-Fournier M, Ruiz P, de Rivera F, Sastre M, Gamez J, Sune P, Salvado M, Gili G, Mazuela G, Vicente E, Diaz-Manera J, Gutierrez L, 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, 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, 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 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, Andoin A, Darnell T, Golden L, Lokaitis B, Seelback J, Goyal N, Sakamuri S, So Y, Paulose S, Pol S, Welsh L, Bhavaraju-Sanka R, 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 H, Rosenfeld J, Garcia R, Echevarria L, Garcia S, Pulley M, Aranke S, Berger A, 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 S, 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. Long‐term safety and efficacy of eculizumab in generalized myasthenia gravis. Muscle & Nerve 2019, 60: 14-24. PMID: 30767274, PMCID: PMC6619057, DOI: 10.1002/mus.26447.Peer-Reviewed Original ResearchMeSH KeywordsActivities of Daily LivingAdultAngioedemaAntibodies, Monoclonal, HumanizedAspergillosisComplement Inactivating AgentsDisease ProgressionFemaleHeart DiseasesHumansInjection Site ReactionLongitudinal StudiesMaleMeningococcal InfectionsMeningococcal VaccinesMiddle AgedMuscle StrengthMyasthenia GravisQuality of LifeTreatment OutcomeConceptsLong-term safetyMyasthenia gravisEfficacy of eculizumabGeneralized myasthenia gravisOpen-label extensionQuality of lifeExacerbation rateMeningococcal infectionPharmacological remissionSafety profileSustained efficacyMuscle strengthEculizumabDaily livingInterim analysisMinimal manifestationsSustained improvementFunctional abilityPatientsGravisEfficacyRegainRefractorySafetyPlacebo
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 inhibitorDurability of the Rituximab Response in Acetylcholine Receptor Autoantibody–Positive Myasthenia Gravis
Robeson KR, Kumar A, Keung B, DiCapua DB, Grodinsky E, Patwa HS, Stathopoulos PA, Goldstein JM, O’Connor K, Nowak RJ. Durability of the Rituximab Response in Acetylcholine Receptor Autoantibody–Positive Myasthenia Gravis. JAMA Neurology 2017, 74: 60-66. PMID: 27893014, DOI: 10.1001/jamaneurol.2016.4190.Peer-Reviewed Original ResearchConceptsDurability of responseMyasthenia gravisRituximab treatmentAnti-AChR antibody levelsB cell-targeted therapiesLong-term clinical responseRetrospective case series studyTreatment of MGEvidence-based practice parametersLast rituximab treatmentRefractory myasthenia gravisSerum cytokine levelsComplete stable remissionSubset of patientsCase series studyMyasthenia Gravis FoundationMG clinicPharmacologic remissionRituximab cyclesSustained remissionAutoantibody levelsInflammatory markersRituximab therapyClinical improvementClinical response