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

Autor: Sophie de Guibert, Lauren Veronese, Loic Ysebaert, Charles Herbaux, Olivier Tournilhac, Emmanuelle Tchernonog, Anne-Sophie Michallet, Florence Cymbalista, Damien Roos-Weil, Bruno Pereira, Jehan Dupuis, Pierre Feugier, Anne Calleja, Florian Bouclet, Caroline Dartigeas, Sandy Amorim, Anaise Blouet, Marie-Sarah Dilhuydy, Florence Nguyen-Khac, Pierre Morel, Nataliya Dmytruck, Kamel Laribi, Alain Delmer, Thérèse Aurran, Bénédicte Hivert, Romain Guieze
Přispěvatelé: Role of intra-Clonal Heterogeneity and Leukemic environment in ThErapy Resistance of chronic leukemias (CHELTER), Université Clermont Auvergne (UCA), FAYE, Fatimata
Jazyk: angličtina
Rok vydání: 2021
Předmět:
Oncology
[SDV.MHEP.HEM] Life Sciences [q-bio]/Human health and pathology/Hematology
Compassionate Use Trials
Male
medicine.medical_treatment
Chronic lymphocytic leukemia
Abnormal Karyotype
Kaplan-Meier Estimate
Targeted therapy
law.invention
chemistry.chemical_compound
0302 clinical medicine
Randomized controlled trial
law
Medicine
ComputingMilieux_MISCELLANEOUS
Aged
80 and over
Sulfonamides
Hematology
Hematopoietic Stem Cell Transplantation
[SDV.MHEP.HEM]Life Sciences [q-bio]/Human health and pathology/Hematology
General Medicine
Middle Aged
Progression-Free Survival
3. Good health
Tumor lysis syndrome
Treatment Outcome
030220 oncology & carcinogenesis
Cohort
Female
France
Lymphoma
Large B-Cell
Diffuse
Adult
medicine.medical_specialty
Antineoplastic Agents
Infections
03 medical and health sciences
Internal medicine
Complex Karyotype
Humans
Aged
Retrospective Studies
business.industry
Venetoclax
medicine.disease
Bridged Bicyclo Compounds
Heterocyclic
Genes
p53
Leukemia
Lymphocytic
Chronic
B-Cell
chemistry
Drug Evaluation
business
Tumor Lysis Syndrome
030215 immunology
Zdroj: Annals of Hematology
Annals of Hematology, Springer Verlag, 2021, 100 (4), pp.987-993. ⟨10.1007/s00277-021-04419-w⟩
ISSN: 0939-5555
1432-0584
Popis: 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.
Databáze: OpenAIRE
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