Tisa-cel in R/R B-ALL: Real-world data from CIBMTR

A real-world study of data from the Center for International Blood and Marrow Transplant Research (CIBMTR) assessing the safety and efficacy of tisagenlecleucel (tisa-cel) in pediatric and young adult patients with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL) (N = 768) was published by John et al. in Blood Advances. The efficacy endpoint was best overall response (BOR) of complete remission (CR) / CR with incomplete blood count recovery (CRi). The safety endpoints included cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). The efficacy set included 578 patients, while the safety set included 703 patients.

Key data: The BOR of CR/CRi was 86% in patients with ≥12 months of post-infusion follow-up. The 12-month relapse-free survival (RFS) was 61.8%, and overall survival (OS) was 79.4%, while the 24-month RFS and OS were 50.3% and 63.8%, respectively. Age <18 years and lower disease burden were associated with improved outcomes, while prior inotuzumab exposure and KMT2A rearrangement were associated with worse outcomes. Grade ≥3 CRS and ICANS were lower in patients aged <3 years vs the overall population. Rates of any grade of CRS and ICANS were lower in the <18 years age group.

Key learning: The findings represent the largest data set for any chimeric antigen receptor (CAR) T-cell therapy in pediatric and young adult patients with B-ALL and are consistent with previous studies demonstrating a favorable safety profile of tisa-cel in this population. Ongoing follow-up will continue to provide real-world insights into the impact of tisa-cel on outcomes in pediatric and young adults with R/R B-ALL.