Erschienen in:
25.01.2021 | Original Article
Real-world outcomes following venetoclax therapy in patients with chronic lymphocytic leukemia or Richter syndrome: a FILO study of the French compassionate use cohort
verfasst von:
Florian Bouclet, Anne Calleja, Marie-Sarah Dilhuydy, Lauren Véronèse, Bruno Pereira, Sandy Amorim, Florence Cymbalista, Charles Herbaux, Sophie de Guibert, Damien Roos-Weil, Bénédicte Hivert, Thérèse Aurran, Jehan Dupuis, Anaise Blouet, Emmanuelle Tchernonog, Kamel Laribi, Nataliya Dmytruck, Pierre Morel, Anne-Sophie Michallet, Caroline Dartigeas, Olivier Tournilhac, Florence Nguyen-Khac, Alain Delmer, Pierre Feugier, Loïc Ysebaert, Romain Guièze
Erschienen in:
Annals of Hematology
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Ausgabe 4/2021
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Abstract
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.