Multinational retrospective study of tisagenlecleucel for R/R B-cell ALL in children younger than 3 years of age at screening

The ELIANA trial showed efficacy and safety of tisagenlecleucel for ALL in children and young adults. Children aged younger than 3 years were excluded from ELIANA. A retrospective study at 15 centers in ten European countries assessed efficacy and safety of tisagenlecleucel for relapsed or refractory (R/R) B-cell ALL in children younger than 3 years at screening. Complete response was seen in 86% of patients with measurable disease. The overall survival at 12 months was 84% and event-free survival was 69%. The authors conclude that tisagenlecleucel has antitumor activity and acceptable safety in young children with R/R B-cell ALL.