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Introduction/Aims: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti-acetylcholine receptor antibody-positive (AChR+) gMG previously treated with rituximab. Methods: This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open-label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE. Results: Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous-rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no-previous-rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous-rituximab and no-previous-rituximab groups (least-squares mean −4.4, standard error of the mean [SEM] 1.0 [n = 9] and least-squares mean −4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval −1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post-intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile. Discussion: Eculizumab is an effective therapy for patients with refractory AChR+ gMG, irrespective of whether they had received rituximab treatment previously.
Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study / Siddiqi, Z.A., Nowak, R.J., Mozaffar, T., O'Brien, F., Yountz, M., Patti, F., Mazia, C.G., Wilken, M., 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., et al.. - In: MUSCLE & NERVE. - ISSN 0148-639X. - 64:6(2021), pp. 662-669. [10.1002/mus.27422]
Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study
Siddiqi Z. A.;Nowak R. J.;Mozaffar T.;O'Brien F.;Yountz M.;Patti F.;Mazia C. G.;Wilken M.;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.;de Siqueira Carvalho A. A.;Brockhausen I. D.;Feder D.;Ambrosio D.;Cesar P.;Melo A. P.;Ribeiro R. M.;Rocha R.;Rosa B. B.;Veiga T.;da Silva L. A.;Engel M. S.;Geraldo J. G.;da Penha Ananias Morita M.;Coelho E. N.;Paiva G.;Pozo M.;Prando N.;Torres D. D. M.;Butinhao C. F.;Duran G.;Fialho T. A. S.;da Silva T. C. G.;Goncalves L. O. M.;Pazetto L. E.;Volpe L. R. C.;Duca L. S.;Friedrich M. A. G.;Guerreiro A.;Mohr H.;Martins M. P.;da Cruz Pacheco D.;Ferreira L.;Macagnan A. P.;Pinto G.;de Cassia Santos A.;Oliveira A. S. B.;de Andrade A. C. A.;Annes M.;Silva L. D.;Lino V. C.;Pinto W.;Assis N.;Carrara F.;Miranda C.;Souza I.;Fernandes P.;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.;Hojgaard J.;Witting N.;Autzen A. M. O.;Pedersen J.;Eralinna 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.;Di Pasquale A.;Garibaldi M.;Sacca F.;Filla A.;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.;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.;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.;Nakamori M.;Takahashi M.;Taichi N.;Hasuike T.;Higuchi E.;Kobayashi H.;Osakada K.;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.;Kim B. -J.;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.;Aguilo M. A. A.;Homedes-Pedret C.;Palacios N. J.;Porras L. D.;Santamaria V. V.;Lazaro A.;Tejedor E. D.;Salcedo P. G.;Fernandez-Fournier M.;Ruiz P. L.;de Rivera F. J. R.;Fernandez-Fournier M.;Sastre M.;Carbonell J. G.;Sune P.;Figueras M. S.;Gili G.;Mazuela G.;Illa I.;Vicente E. C.;Diaz-Manera J.;Gutierrez L. A. Q.;Garcia R. 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.;Cavdar T. K.;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.;Sanjak M.;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. C.;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. T.;Paulose S.;Pol S.;Welsh L.;Bhavaraju-Sanka R.;Gonzalez A. T.;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.;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.;Dicapua D.;Keung B.;Kumar A.;Patwa H.;Robeson K.;Yang I.;Nye J.;Vu H.
2021
Abstract
Introduction/Aims: Individuals with refractory generalized myasthenia gravis (gMG) who have a history of rituximab use and experience persistent symptoms represent a population with unmet treatment needs. The aim of this analysis was to evaluate the efficacy and safety of eculizumab in patients with refractory anti-acetylcholine receptor antibody-positive (AChR+) gMG previously treated with rituximab. Methods: This post hoc subgroup analysis of the phase 3 REGAIN study (NCT01997229) and its open-label extension (OLE; NCT02301624) compared baseline characteristics, safety, and response to eculizumab in participants who had previously received rituximab with those who had not. Rituximab use was not permitted within the 6 months before screening or during REGAIN/OLE. Results: Of 125 REGAIN participants, 14 had received rituximab previously (7 received placebo and 7 received eculizumab). In the previous-rituximab group, 57% had used at least four other immunosuppressants compared with 16% in the no-previous-rituximab group. Myasthenia Gravis Activities of Daily Living total scores from eculizumab baseline to week 130 of eculizumab treatment improved in both the previous-rituximab and no-previous-rituximab groups (least-squares mean −4.4, standard error of the mean [SEM] 1.0 [n = 9] and least-squares mean −4.6, SEM 0.3 [n = 67], respectively; difference = 0.2, 95% confidence interval −1.88 to 2.22). In addition, in both groups, most patients who were treated with eculizumab for 130 weeks achieved a Myasthenia Gravis Foundation of America post-intervention status of minimal manifestations (66.7% and 65.0%, respectively). The eculizumab safety profile was similar between groups and consistent with its established profile. Discussion: Eculizumab is an effective therapy for patients with refractory AChR+ gMG, irrespective of whether they had received rituximab treatment previously.
01 Pubblicazione su rivista::01a Articolo in rivista
Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study / Siddiqi, Z.A., Nowak, R.J., Mozaffar, T., O'Brien, F., Yountz, M., Patti, F., Mazia, C.G., Wilken, M., 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., et al.. - In: MUSCLE & NERVE. - ISSN 0148-639X. - 64:6(2021), pp. 662-669. [10.1002/mus.27422]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11573/1603296
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simulazione ASN
Il report seguente simula gli indicatori relativi alla propria produzione scientifica in relazione alle soglie ASN 2023-2025 del proprio SC/SSD. Si ricorda che il superamento dei valori soglia (almeno 2 su 3) è requisito necessario ma non sufficiente al conseguimento dell'abilitazione. La simulazione si basa sui dati IRIS e sugli indicatori bibliometrici alla data indicata e non tiene conto di eventuali periodi di congedo obbligatorio, che in sede di domanda ASN danno diritto a incrementi percentuali dei valori. La simulazione può differire dall'esito di un’eventuale domanda ASN sia per errori di catalogazione e/o dati mancanti in IRIS, sia per la variabilità dei dati bibliometrici nel tempo. Si consideri che Anvur calcola i valori degli indicatori all'ultima data utile per la presentazione delle domande.
La presente simulazione è stata realizzata sulla base delle specifiche raccolte sul tavolo ER del Focus Group IRIS coordinato dall’Università di Modena e Reggio Emilia e delle regole riportate nel DM 589/2018 e allegata Tabella A. Cineca, l’Università di Modena e Reggio Emilia e il Focus Group IRIS non si assumono alcuna responsabilità in merito all’uso che il diretto interessato o terzi faranno della simulazione. Si specifica inoltre che la simulazione contiene calcoli effettuati con dati e algoritmi di pubblico dominio e deve quindi essere considerata come un mero ausilio al calcolo svolgibile manualmente o con strumenti equivalenti.
