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

Fiche publication

Date publication

janvier 2021

Journal

Annals of hematology

Auteurs

Membres identifiés du Cancéropôle Est :
Pr FEUGIER Pierre , Pr DELMER Alain

Tous les auteurs :
Bouclet F, Calleja A, Dilhuydy MS, Véronèse L, Pereira B, Amorim S, Cymbalista F, Herbaux C, de Guibert S, Roos-Weil D, Hivert B, Aurran T, Dupuis J, Blouet A, Tchernonog E, Laribi K, Dmytruck N, Morel P, Michallet AS, Dartigeas C, Tournilhac O, Nguyen-Khac F, Delmer A, Feugier P, Ysebaert L, Guièze R

Lien Pubmed

https://www.ncbi.nlm.nih.gov/pubmed/33495922

Résumé

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.