2023
Chemotherapy-Associated Thrombotic Microangiopathy
Aklilu A, Shirali A. Chemotherapy-Associated Thrombotic Microangiopathy. Kidney360 2023, 4: 409-422. PMID: 36706238, PMCID: PMC10103319, DOI: 10.34067/kid.0000000000000061.Peer-Reviewed Reviews, Practice Guidelines, Standards, and Consensus StatementsConceptsDrug-induced thrombotic microangiopathyThrombotic microangiopathyPlasma exchangePrimary atypical hemolytic uremic syndromeAtypical hemolytic uremic syndromeEnd-organ dysfunctionMicroangiopathic hemolytic anemiaSecondary thrombotic microangiopathyTherapeutic plasma exchangeTerminal complement inhibitorLarge multicenter studyHemolytic uremic syndromeRole of complementPotential treatment strategyProteinuria reductionSupportive careBlood pressureMulticenter studyTreatment courseUremic syndromeComplement inhibitionTreatment strategiesHemolytic anemiaAntiangiogenesis agentsComplement inhibitors
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
1994
Expression of a functional human complement inhibitor in a transgenic pig as a model for the prevention of xenogeneic hyperacute organ rejection.
Fodor W, Williams B, Matis L, Madri J, Rollins S, Knight J, Velander W, Squinto S. Expression of a functional human complement inhibitor in a transgenic pig as a model for the prevention of xenogeneic hyperacute organ rejection. Proceedings Of The National Academy Of Sciences Of The United States Of America 1994, 91: 11153-11157. PMID: 7526391, PMCID: PMC45185, DOI: 10.1073/pnas.91.23.11153.Peer-Reviewed Original ResearchConceptsHuman complement inhibitorsHyperacute rejectionComplement inhibitorsAnti-porcine antibodiesTerminal complement inhibitorTransgenic pigsXenogeneic transplantation modelPorcine cell typesHigh-level cell surface expressionVariety of murineXenogeneic complementsXenogeneic organ transplantationGraft failureOrgan rejectionVessel occlusionCell surface expressionHyperacute organ rejectionTransplantation modelOrgan transplantationXenogeneic organsVascular endotheliumTransgenic miceSerum complementAutologous complementTransplantation
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