4CPS-216 Case series: real-world effectiveness and safety of tisagenlecleucel for the treatment of B-cell precursor acute lymphoblastic leukaemia in paediatrics
Year: 2025
Type: conference-paper
Abstract: Background and Importance Tisagenlecleucel is a CD19-directed genetically modified autologous T-cell immunotherapy indicated for the treatment of B-cell precursor acute lymphoblastic leukaemia (ALL) that is refractory or in relapse. Aim and Objectives To analyse the effectiveness and safety of tisagenlecleucel in paediatric patients with B-ALL. Material and Methods Retrospective observational study of all patients with B-ALL treated with tisagenlecleucel in our centre. Study period: August 2019 to September 2024. Variables collected: demographic (age, sex), clinical (diagnosis), therapeutic (previous treatments), effectiveness (overall remission rate (ORR): proportion of patients in complete remission (CR) or complete remission with incomplete blood count recovery (CRi) with minimal residual disease (MRD) <0.01% at 28 days, 3, 6 and 12 months, and median overall survival (mOS)), safety (cytokine release syndrome (CLS), immune effector cell-associated neurotoxicity syndrome (ICANS), cytopenias, infections, macrophage activation syndrome (MAS) and hypogammaglobulinaemia). Results Eight patients, 62.5% male, median age 6 years (3–15). 75% with previous allogeneic haematological transplant and one received blinatumomab. 100% were in relapse pre-CART receiving bridging therapy. Pre-CART tumour load: 49.40% (0.01–96.00). Median follow-up: 12.36 months (3.0–41.4). Effectiveness: ORR at 28 days 87.5%, at 3 months 62.5%, at 6 months 37.5%, at 12 months 14%. 83.3% relapses CD19-. mOS= 11.5 months (3.0–14.7). Safety CRS: 100% of patients at 1.5 days(0–6) (25% grade ≥3), 50% needed tocilizumab and 25% tocilizumab+corticosteroids. 62.5% admitted to ICU. ICANS: 37.5% at 10 days (9–11) (66.7% grade ≥3). Infections: 37.5%. Cytopenias: 100% (37.5% grade ≥3). MAS: 50%. Hypogammaglobulinaemia: 100%, all needed immunoglobulins. At the end of follow-up three patients were still alive, one after 42 months in CR. The rest died due to progression B-ALL. Conclusion and Relevance Tisagenlecleucel effectiveness in our cohort was lower than that observed in the pivotal clinical trial, while maintaining a similar safety level. The majority of relapses are CD19 negative. More detailed analysis of factors that could influence response is needed, as well as a larger sample size in order to identify optimisation strategies, ensure maximum benefit from this therapy and obtain conclusive results. References and/or Acknowledgements Maude SL, Laetsch TW, Buechner J, et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukaemia. N Engl J Med. 2018;378(5):439–448. doi:10.1056/NEJMoa1709866 Conflict of Interest No conflict of interest
Institution: Hospital Regional Universitario de Málaga
Language: English
Topic: CAR-T cell therapy research
Subfield: Oncology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
OpenAlex ID: W4408377057