Eculizumab discontinuation in children and adults with atypical hemolytic-uremic syndrome: a prospective multicenter study
Détails
ID Serval
serval:BIB_E11AC30A8988
Type
Article: article d'un périodique ou d'un magazine.
Collection
Publications
Institution
Titre
Eculizumab discontinuation in children and adults with atypical hemolytic-uremic syndrome: a prospective multicenter study
Périodique
Blood
ISSN
1528-0020 (Electronic)
ISSN-L
0006-4971
Statut éditorial
Publié
Date de publication
2021
Volume
137
Numéro
18
Pages
2438-2449
Langue
anglais
Notes
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
eng
Blood. 2021 May 6;137(18):2438-2449. doi: 10.1182/blood.2020009280.
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
eng
Blood. 2021 May 6;137(18):2438-2449. doi: 10.1182/blood.2020009280.
Résumé
The optimal duration of eculizumab treatment in patients with atypical hemolytic uremic syndrome (aHUS) remains poorly defined. We conducted a prospective national multicenter open-label study to assess eculizumab discontinuation in children and adults with aHUS. Fifty-five patients (including 19 children) discontinued eculizumab (mean treatment duration, 16.5 months). Twenty-eight patients (51%) had rare variants in complement genes, mostly in MCP (n = 12; 22%), CFH (n = 6; 11%), and CFI (n = 6; 10%). At eculizumab discontinuation, 17 (30%) and 4 patients (7%) had stage 3 and 4 chronic kidney disease, respectively. During follow-up, 13 patients (23%; 6 children and 7 adults) experienced aHUS relapse. In multivariable analysis, female sex and presence of a rare variant in a complement gene were associated with an increased risk of aHUS relapse, whereas requirement for dialysis during a previous episode of acute aHUS was not. In addition, increased sC5b-9 plasma level at eculizumab discontinuation was associated with a higher risk of aHUS relapse in all patients and in the subset of carriers with a complement gene rare variant, both by log-rank test and in multivariable analysis. Of the 13 relapsing patients, all of whom restarted eculizumab, 11 regained their baseline renal function and 2 had a worsening of their preexisting chronic kidney disease, including 1 patient who progressed to end-stage renal disease. A strategy of eculizumab discontinuation in aHUS patients based on complement genetics is reasonable and safe. It improves the management and quality of life of a sizeable proportion of aHUS patients while reducing the cost of treatment. This trial was registered at www.clinicaltrials.gov as #NCT02574403.
Pubmed
Création de la notice
01/03/2022 13:41
Dernière modification de la notice
02/03/2022 6:36