Eculizumab and aHUS: to stop or not

被引:18
作者
Brodsky, Robert A. [1 ]
机构
[1] Johns Hopkins Univ, Baltimore, MD 21218 USA
关键词
COMPLEMENT INHIBITOR ECULIZUMAB;
D O I
10.1182/blood.2020010234
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
In this issue of Blood, Fakhouri et al(1) provide evidence in a prospective phase 4, multicentric, noncontrolled study that discontinuing eculizumab is safe in most patients with atypical hemolytic uremic syndrome (aHUS) once they achieve complete remission. Risk of relapse was <25% overall, but as high as 50% in patients with a rare variant in at least 1 complement gene.
引用
收藏
页码:2419 / 2420
页数:2
相关论文
共 9 条
[1]   Discontinuation of Eculizumab Treatment in Atypical Hemolytic Uremic Syndrome: An Update [J].
Ardissino, Gianluigi ;
Possenti, Ilaria ;
Tel, Francesca ;
Testa, Sara ;
Salardi, Stefania ;
Ladisa, Vito .
AMERICAN JOURNAL OF KIDNEY DISEASES, 2015, 66 (01) :172-173
[2]   The long-acting C5 inhibitor, ravulizumab, is effective and safe in pediatric patients with atypical hemolytic uremic syndrome naive to complement inhibitor treatment [J].
Ariceta, Gema ;
Dixon, Bradley P. ;
Kim, Seong Heon ;
Kapur, Gaurav ;
Mauch, Teri ;
Ortiz, Stephan ;
Vallee, Marc ;
Denker, Andrew E. ;
Kang, Hee Gyung ;
Greenbaum, Larry A. .
KIDNEY INTERNATIONAL, 2021, 100 (01) :225-237
[3]   Eculizumab discontinuation in children and adults with atypical hemolytic-uremic syndrome: a prospective multicenter study [J].
Fakhouri, Fadi ;
Fila, Marc ;
Hummel, Aurelie ;
Ribes, David ;
Sellier-Leclerc, Anne-Laure ;
Ville, Simon ;
Pouteil-Noble, Claire ;
Coindre, Jean-Philippe ;
Le Quintrec, Moglie ;
Rondeau, Eric ;
Boyer, Olivia ;
Provot, Francois ;
Djeddi, Djamal ;
Hanf, William ;
Delmas, Yahsou ;
Louillet, Ferielle ;
Lahoche, Annie ;
Favre, Guillaume ;
Chatelet, Valerie ;
Launay, Emma Allain ;
Presne, Claire ;
Zaloszyc, Ariane ;
Caillard, Sophie ;
Bally, Stephane ;
Raimbourg, Quentin ;
Tricot, Leila ;
Mousson, Christiane ;
Le Thuaut, Aurelie ;
Loirat, Chantal ;
Fremeaux-Bacchi, Veronique .
BLOOD, 2021, 137 (18) :2438-2449
[4]   Haemolytic uraemic syndrome [J].
Fakhouri, Fadi ;
Zuber, Julien ;
Fremeaux-Bacchi, Veronique ;
Loirat, Chantal .
LANCET, 2017, 390 (10095) :681-696
[5]   Pathogenic Variants in Complement Genes and Risk of Atypical Hemolytic Uremic Syndrome Relapse after Eculizumab Discontinuation [J].
Fakhouri, Fadi ;
Fila, Marc ;
Provot, Francois ;
Delmas, Yahsou ;
Barbet, Christelle ;
Chatelet, Valerie ;
Rafat, Cedric ;
Cailliez, Mathilde ;
Hogan, Julien ;
Servais, Aude ;
Karras, Alexandre ;
Makdassi, Raifah ;
Louillet, Feriell ;
Coindre, Jean-Philippe ;
Rondeau, Eric ;
Loirat, Chantal ;
Fremeaux-Bacchi, Veronique .
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2017, 12 (01) :50-59
[6]   Complementopathies and precision medicine [J].
Gavriilaki, Eleni ;
Brodsky, Robert A. .
JOURNAL OF CLINICAL INVESTIGATION, 2020, 130 (05) :2152-2163
[7]   Terminal Complement Inhibitor Eculizumab in Atypical Hemolytic-Uremic Syndrome [J].
Legendre, C. M. ;
Licht, C. ;
Muus, P. ;
Greenbaum, L. A. ;
Babu, S. ;
Bedrosian, C. ;
Bingham, C. ;
Cohen, D. J. ;
Delmas, Y. ;
Douglas, K. ;
Eitner, F. ;
Feldkamp, T. ;
Fouque, D. ;
Furman, R. R. ;
Gaber, O. ;
Herthelius, M. ;
Hourmant, M. ;
Karpman, D. ;
Lebranchu, Y. ;
Mariat, C. ;
Menne, J. ;
Moulin, B. ;
Nuernberger, J. ;
Ogawa, M. ;
Remuzzi, G. ;
Richard, T. ;
Sberro-Soussan, R. ;
Severino, B. ;
Sheerin, N. S. ;
Trivelli, A. ;
Zimmerhackl, L. B. ;
Goodship, T. ;
Loirat, C. .
NEW ENGLAND JOURNAL OF MEDICINE, 2013, 368 (23) :2169-2181
[8]   Eculizumab cessation in atypical hemolytic uremic syndrome [J].
Merrill, Samuel A. ;
Brittingham, Zachary D. ;
Yuan, Xuan ;
Moliterno, Alison R. ;
Sperati, C. John ;
Brodsky, Robert A. .
BLOOD, 2017, 130 (03) :368-372
[9]   Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria [J].
Rother, Russell P. ;
Rollins, Scott A. ;
Mojcik, Christopher F. ;
Brodsky, Robert A. ;
Bell, Leonard .
NATURE BIOTECHNOLOGY, 2007, 25 (11) :1256-1264