Multinational phase 3b trial of venetoclax for relapsed or refractory chronic lymphocytic leukemia

Most patients with chronic lymphocytic leukemia (CLL) progress after treatment with targeted therapy or chemoimmunotherapy and have limited subsequent treatment options. The multinational phase 3b VENICE-1 trial evaluated venetoclax monotherapy in patients with relapsed or refractory (R/R) CLL. Patients received 5-week ramp-up to 400 mg of oral venetoclax once daily and were treated for up to 108 weeks. At 48 weeks, objective responses were seen in the majority of patients, including patients who were previously treated with B-cell receptor-associated kinase inhibitor (BCRi). Grade 3 or worse treatment-emergent adverse events were reported in 79% and serious adverse events in 53% of patients. One patient died of autoimmune hemolytic anemia possibly related to venetoclax. The authors conclude that venetoclax monotherapy induced deep and durable responses in patients with R/R CLL, including BCRi-pretreated patients.