AIEOP-BFM ALL 2017 phase III trial: Blinatumomab vs chemotherapy in pediatric high-risk Ph– B-ALL

Results from the randomized, international, phase III AIEOP-BFM ALL 2017 trial (NCT03643276), evaluating two cycles of blinatumomab (n = 358) vs two cycles of chemotherapy (control; n = 351) after consolidation in pediatric patients with newly diagnosed (ND) high-risk Philadelphia chromosome-negative (Ph–) B-cell acute lymphoblastic leukemia (B-ALL), were published in The New England Journal of Medicine by Schrappe et al. The primary endpoint was event-free survival (EFS). 

Key data: At a median follow-up of 2.9 years, the estimated 4-year EFS was 83.0% (95% confidence interval [CI], 77.4–87.4) in the blinatumomab group vs 70.3% (95% CI, 63.8–75.9) in the control group (p = 0.0002), with an estimated hazard ratio [HR] of 0.51 (95% CI, 0.35–0.73). Treatment-related infections occurred less frequently with blinatumomab vs control (23.9% vs 69.4%; p < 0.001), while treatment related neurotoxic events occurred more frequently with blinatumomab vs control (12.0% vs 3.2%; p < 0.001). Cytokine release syndrome (CRS) Grade ≥2 occurred in 1.1% of patients in the blinatumomab group. Life-threatening adverse events (AEs) were reported in 0.5% of patients receiving blinatumomab vs 4.7% in those receiving control.  

Key learning: In this study, two cycles of blinatumomab demonstrated a significantly higher EFS and vs two cycles of chemotherapy in pediatric patients with ND high-risk Ph– B-ALL, supporting the potential of immunotherapy-based strategies as a chemotherapy replacement for first-line treatment in this population