Multinational randomized trial of blinatumomab versus chemotherapy for pediatric acute lymphoblastic leukemia

Blinatumomab is a bispecific T-cell engager targeting the CD19 antigen on B cells. The multinational randomized AIEOP-BFM ALL 2017 trial evaluated blinatumomab as an option to replace cycles of traditional chemotherapy in pediatric patients with newly diagnosed high-risk B-cell acute lymphoblastic leukemia (ALL). After consolidation, children with high-risk B-cell ALL were randomized 1:1 to receive two cycles of blinatumomab (blinatumomab group; n=358) or two cycles of chemotherapy (control group; n=351). The primary endpoint was event-free survival (EFS). A planned interim analysis at a median follow-up of 2.9 years showed an estimated 4-year EFS rate of 83.0% (95% CI 77.4-87.4) in the blinatumomab group and 70.3% (63.8-75.9) in the control group (p=0.0002). The estimated hazard ratio for a primary endpoint event was 0.51 (95% CI 0.35-0.73). Treatment-related infection occurred in 23.9% of patients in the blinatumomab group and 69.4% of patients in the control group. Life-threatening adverse events occurred in two patients (0.5%) in the blinatumomab group, including one that was fatal, and in 16 patients (4.7%) in the control group; neurotoxic events were reported in 12.0% and 3.2%, respectively. Cytokine release syndrome of grade 2 or higher occurred in 1.1% of patients in the blinatumomab group. The authors conclude that among children with newly diagnosed high-risk B-cell ALL, replacement ot two cycles conventional chemotherapy with blinatumomab resulted in significantly greater percentage of patients with EFS at 4 years (quick take).