Titre :

Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria.

Auteur(s) :

Peffault De Latour, R. [Auteur]
Röth, A. [Auteur]
Kulasekararaj, A. G. [Auteur]
Han, B. [Auteur]
Scheinberg, P. [Auteur]
Maciejewski, J. P. [Auteur]
Ueda, Y. [Auteur]
De Castro, C. M. [Auteur]
Di Bona, E. [Auteur]
Fu, R. [Auteur]
Zhang, L. [Auteur]
Griffin, M. [Auteur]
Langemeijer, S. M. C. [Auteur]
Panse, J. [Auteur]
Schrezenmeier, H. [Auteur]
Barcellini, W. [Auteur]
Mauad, V. A. Q. [Auteur]
Schafhausen, P. [Auteur]
Tavitian, S. [Auteur]
Beggiato, E. [Auteur]
Chew, L. P. [Auteur]
Gaya, A. [Auteur]
Huang, W. H. [Auteur]
Jang, J. H. [Auteur]
Kitawaki, T. [Auteur]
Kutlar, A. [Auteur]
Notaro, R. [Auteur]
Pullarkat, V. [Auteur]
Schubert, J. [Auteur]
Terriou, Louis [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Uchiyama, M. [Auteur]
Wong Lee Lee, L. [Auteur]
Yap, E. S. [Auteur]
Sicre De Fontbrune, F. [Auteur]
Marano, L. [Auteur]
Alashkar, F. [Auteur]
Gandhi, S. [Auteur]
Trikha, R. [Auteur]
Yang, C. [Auteur]
Liu, H. [Auteur]
Kelly, R. J. [Auteur]
Höchsmann, B. [Auteur]
Kerloeguen, C. [Auteur]
Banerjee, P. [Auteur]
Levitch, R. [Auteur]
Kumar, R. [Auteur]
Wang, Z. [Auteur]
Thorburn, C. [Auteur]
Maitra, S. [Auteur]
Li, S. [Auteur]
Verles, A. [Auteur]
Dahlke, M. [Auteur]
Risitano, A. M. [Auteur]

Titre de la revue :

New England Journal of Medicine

Nom court de la revue :

N Engl J Med

Numéro :

390

Pagination :

994-1008

Date de publication :

2024-03-14

ISSN :

1533-4406

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

Background Persistent hemolytic anemia and a lack of oral treatments are challenges for patients with paroxysmal nocturnal hemoglobinuria who have received anti-C5 therapy or have not received complement inhibitors. ...
Lire la suite >Background Persistent hemolytic anemia and a lack of oral treatments are challenges for patients with paroxysmal nocturnal hemoglobinuria who have received anti-C5 therapy or have not received complement inhibitors. Iptacopan, a first-in-class oral factor B inhibitor, has been shown to improve hemoglobin levels in these patients. Methods In two phase 3 trials, we assessed iptacopan monotherapy over a 24-week period in patients with hemoglobin levels of less than 10 g per deciliter. In the first, anti-C5–treated patients were randomly assigned to switch to iptacopan or to continue anti-C5 therapy. In the second, single-group trial, patients who had not received complement inhibitors and who had lactate dehydrogenase (LDH) levels more than 1.5 times the upper limit of the normal range received iptacopan. The two primary end points in the first trial were an increase in the hemoglobin level of at least 2 g per deciliter from baseline and a hemoglobin level of at least 12 g per deciliter, each without red-cell transfusion; the primary end point for the second trial was an increase in hemoglobin level of at least 2 g per deciliter from baseline without red-cell transfusion. Results In the first trial, 51 of the 60 patients who received iptacopan had an increase in the hemoglobin level of at least 2 g per deciliter from baseline, and 42 had a hemoglobin level of at least 12 g per deciliter, each without transfusion; none of the 35 anti-C5–treated patients attained the end-point levels. In the second trial, 31 of 33 patients had an increase in the hemoglobin level of at least 2 g per deciliter from baseline without red-cell transfusion. In the first trial, 59 of the 62 patients who received iptacopan and 14 of the 35 anti-C5–treated patients did not require or receive transfusion; in the second trial, no patients required or received transfusion. Treatment with iptacopan increased hemoglobin levels, reduced fatigue, reduced reticulocyte and bilirubin levels, and resulted in mean LDH levels that were less than 1.5 times the upper limit of the normal range. Headache was the most frequent adverse event with iptacopan. Conclusions Iptacopan treatment improved hematologic and clinical outcomes in anti-C5–treated patients with persistent anemia — in whom iptacopan showed superiority to anti-C5 therapy — and in patients who had not received complement inhibitors.Lire moins >

Langue :

Anglais

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286

Date de dépôt :

2024-05-06T21:37:04Z
2024-09-11T08:55:22Z