Title :

Real-world outcomes following venetoclax therapy in patients with chronic lymphocytic leukemia or Richter syndrome: a FILO study of the French compassionate use cohort

Author(s) :

Bouclet, F. [Auteur]
Calleja, A. [Auteur]
Dilhuydy, M. S. [Auteur]
Véronèse, L. [Auteur]
Pereira, B. [Auteur]
Amorim, S. [Auteur]
Cymbalista, F. [Auteur]
Herbaux, Charles [Auteur]
De Guibert, S. [Auteur]
Roos-Weil, D. [Auteur]
Hivert, B. [Auteur]
Aurran, T. [Auteur]
Dupuis, J. [Auteur]
Blouet, A. [Auteur]
Tchernonog, E. [Auteur]
Laribi, K. [Auteur]
Dmytruck, N. [Auteur]
Morel, Pierre [Auteur]
METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694
Michallet, A. S. [Auteur]
Dartigeas, C. [Auteur]
Tournilhac, O. [Auteur]
Nguyen-Khac, F. [Auteur]
Delmer, A. [Auteur]
Feugier, P. [Auteur]
Ysebaert, L. [Auteur]
Guièze, R. [Auteur]

Journal title :

Annals of Hematology

Abbreviated title :

Ann Hematol

Volume number :

100

Pages :

p. 987–993

Publication date :

2021

ISSN :

1432-0584

English keyword(s) :

Chronic lymphocytic leukemia
Richter syndrome
B cell
Targeted therapy
Bcl-2 inhibitor
Venetoclax

HAL domain(s) :

Sciences du Vivant [q-bio]

English abstract : [en]

The BCL2 inhibitor venetoclax is transforming the management of patients with chronic lymphocytic leukemia (CLL), given its high efficacy in relapsed/refractory CLL as observed in both early-phase and randomized clinical ...
Show more >The BCL2 inhibitor venetoclax is transforming the management of patients with chronic lymphocytic leukemia (CLL), given its high efficacy in relapsed/refractory CLL as observed in both early-phase and randomized clinical trials. The present study aimed to determine whether venetoclax is effective and well tolerated in patients with CLL or Richter’s syndrome (RS) in a real-world setting and to highlight factors impacting survival. Data from a venetoclax French compassionate use program were collected for 67 patients (60 with CLL and 7 with RS). Most patients presented adverse genetic features, such as TP53 disruption (74%) or complex karyotype (58%). Tumor lysis syndrome was observed in 14 (22%) patients, and 16 (24%) patients were hospitalized for grade III/IV infection. In the CLL cohort, ORR was 75 %, 1-year PFS was 61% (95% CI = 47–72%) and 1-year OS 70% (95% CI = 56–80%). No impact of TP53 disruption was noted while complex karyotype was identified as a predictor of both inferior PFS (HR = 3.46; 95% CI = 1–12; log-rank p = 0.03) and OS (HR = 3.2; 95% CI = 0.9–11.4, log-rank p = 0.047). Among the seven patients with RS, two achieved an objective response to venetoclax; however, the median OS was only 1.1 month. The well-balanced safety/efficacy profile of venetoclax is confirmed in this real-world setting. Complex karyotype should be evaluated as a predictive factor of survival for patients treated by venetoclax.Show less >

Language :

Anglais

Audience :

Internationale

Popular science :

Non

Administrative institution(s) :

Université de Lille
CHU Lille

Collections :

• METRICS : Evaluation des technologies de santé et des pratiques médicales - ULR 2694

Submission date :

2023-11-15T07:18:03Z
2024-02-20T13:48:30Z