Disease burden affects outcomes of commercial tisagenlecleucel for B-ALL in CAYAs

After tisagenlecleucel commercialization, cross-institutional practices relating to tisagenlecleucel use are heterogeneous and data-capturing is limited. Real-world multicenter retrospective analysis of pediatric, adolescent and young adult R/R B-ALL patients found that commercial tisagelecleucel was efficacious and was tolerated. High disease burden (≥5% bone marrow blasts, CNS3, or non-CNS extramedullary disease) prior to infusion was associated with inferior event-free and overall survival, and with increased toxicity. Patients with high disease burden may benefit from further interventional strategies to consolidate CAR-mediated outcomes.