Real-World Safety and Efficacy of Venetoclax in Chronic Lymphocytic Leukemia: A Single-Center Comparative Analysis With Randomized Clinical Trials.

Treating chronic lymphocytic leukemia (CLL) with the BCL-2 inhibitor venetoclax has shown favorable results in randomized clinical trials (RCTs). Regulatory authorities have recognized the need for also investigating the efficacy and safety of new antineoplastic therapies in real-world (RW) studies with patients often characterized by higher age and comorbidities than patients treated in RCTs. We present a RW single-center study of 112 patients with CLL or small lymphocytic lymphoma (SLL) treated with venetoclax at Zealand University Hospital. A total of 74 patients were treated according to the standard clinical practice and 38 were included in RCTs. No significant differences in efficacy profiles, or safety measures were observed between the two cohorts. Both groups presented overall acceptable tolerability and safety profiles to venetoclax. Moreover, our results suggest that tumor lysis syndrome (TLS) was not a clinical challenge in RW patients even when 6- and 12 h blood samples for TLS were omitted. RW CLL/SLL patients treated outside a clinical trial also had comparable safety and efficacy profiles as reported in the MURANO, CLL13, and CLL14 trials. In conclusion, patients with CLL treated with venetoclax in a RW clinical setting exhibit similar efficacy and safety outcomes to those observed in RCTs.