Titre :

Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia.

Auteur(s) :

Peffault De Latour, R. [Auteur]
Kulasekararaj, A. [Auteur]
Iacobelli, S. [Auteur]
Terwel, S. R. [Auteur]
Cook, R. [Auteur]
Griffin, M. [Auteur]
Halkes, C. J. M. [Auteur]
Recher, C. [Auteur]
Barraco, F. [Auteur]
Forcade, E. [Auteur]
Vallejo, J. C. [Auteur]
Drexler, B. [Auteur]
Mear, J. B. [Auteur]
Smith, A. E. [Auteur]
Angelucci, E. [Auteur]
Raymakers, R. A. P. [Auteur]
De Groot, M. R. [Auteur]
Daguindau, E. [Auteur]
Nur, E. [Auteur]
Barcellini, W. [Auteur]
Russell, N. H. [Auteur]
Terriou, Louis [Auteur]
Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) - U1286
Iori, A. P. [Auteur]
La Rocca, U. [Auteur]
Sureda, A. [Auteur]
Sánchez-Ortega, I. [Auteur]
Xicoy, B. [Auteur]
Jarque, I. [Auteur]
Cavenagh, J. [Auteur]
Sicre De Fontbrune, F. [Auteur]
Marotta, S. [Auteur]
Munir, T. [Auteur]
Tjon, J. M. L. [Auteur]
Tavitian, S. [Auteur]
Praire, A. [Auteur]
Clement, L. [Auteur]
Rabian, F. [Auteur]
Marano, L. [Auteur]
Hill, A. [Auteur]
Palmisani, E. [Auteur]
Muus, P. [Auteur]
Cacace, F. [Auteur]
Frieri, C. [Auteur]
Van Lint, M. T. [Auteur]
Passweg, J. R. [Auteur]
Marsh, J. C. W. [Auteur]
Socié, G. [Auteur]
Mufti, G. J. [Auteur]
Dufour, C. [Auteur]
Risitano, A. M. [Auteur]

Titre de la revue :

New England Journal of Medicine

Nom court de la revue :

N Engl J Med

Numéro :

386

Pagination :

11-23

Date de publication :

2022-01-08

ISSN :

1533-4406

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

Background A single-group, phase 1–2 study indicated that eltrombopag improved the efficacy of standard immunosuppressive therapy that entailed horse antithymocyte globulin (ATG) plus cyclosporine in patients with severe ...
Lire la suite >Background A single-group, phase 1–2 study indicated that eltrombopag improved the efficacy of standard immunosuppressive therapy that entailed horse antithymocyte globulin (ATG) plus cyclosporine in patients with severe aplastic anemia. Methods In this prospective, investigator-led, open-label, multicenter, randomized, phase 3 trial, we compared the efficacy and safety of horse ATG plus cyclosporine with or without eltrombopag as front-line therapy in previously untreated patients with severe aplastic anemia. The primary end point was a hematologic complete response at 3 months. Download a PDF of the Research Summary. Results Patients were assigned to receive immunosuppressive therapy (Group A, 101 patients) or immunosuppressive therapy plus eltrombopag (Group B, 96 patients). The percentage of patients who had a complete response at 3 months was 10% in Group A and 22% in Group B (odds ratio, 3.2; 95% confidence interval [CI], 1.3 to 7.8; P=0.01). At 6 months, the overall response rate (the percentage of patients who had a complete or partial response) was 41% in Group A and 68% in Group B. The median times to the first response were 8.8 months (Group A) and 3.0 months (Group B). The incidence of severe adverse events was similar in the two groups. With a median follow-up of 24 months, a karyotypic abnormality that was classified as myelodysplastic syndrome developed in 1 patient (Group A) and 2 patients (Group B); event-free survival was 34% and 46%, respectively. Somatic mutations were detected in 29% (Group A) and 31% (Group Β) of the patients at baseline; these percentages increased to 66% and 55%, respectively, at 6 months, without affecting the hematologic response and 2-year outcome. Conclusions The addition of eltrombopag to standard immunosuppressive therapy improved the rate, rapidity, and strength of hematologic response among previously untreated patients with severe aplastic anemia, without additional toxic effects. (Funded by Novartis and others; RACE ClinicalTrials.gov number, NCT02099747; EudraCT number, 2014-000363-40.)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 :

2024-01-12T05:17:31Z
2024-03-29T12:04:52Z