Blinatumomab replacing part of chemotherapy in paediatric acute lymphoblastic leukaemia: 4-year event-free survival 83.0% vs 70.3%

In the randomised phase 3 AIEOP-BFM ALL 2017 trial, 709 children with high-risk B-cell acute lymphoblastic leukaemia had two chemotherapy blocks replaced by two cycles of blinatumomab, a CD19/CD3 bispecific antibody. At four years, event-free survival was 83.0% (95% CI 77.4–87.4) versus 70.3% (95% CI 63.8–75.9) with chemotherapy alone (HR 0.51; 95% CI 0.35–0.73; P = 0.0002). Toxicity fell at the same time: treatment-related infections in 23.9% versus 69.4% of children (P < 0.001) and life-threatening events in 2 (0.5%) versus 16 (4.7%) patients, at the cost of more neurological complications (12.0% vs 3.2%). This is a practice-changing result for this patient group; treatment of any individual child remains a decision for the treating paediatric haemato-oncology centre (New England Journal of Medicine, NCT03643276).