6 research outputs found
Graft-versus-host disease induced by tisagenlecleucel in patients after allogeneic stem cell transplantation
S111: TREATMENT OF POST-TRANSPLANT RELAPSE IN CHILDREN, ADOLESCENTS AND YOUNG ADULTS WITH BCP ALL USING CD19-CAR-T: A EUROPEAN RETROSPECTIVE ANALYSIS OF REAL-WORLD DATA
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Intention-to-treat outcomes utilising a stringent event definition in children and young people treated with tisagenlecleucel for r/r ALL through a national access scheme
Acknowledgements: Although this project received no specific funding, MOE is funded by Children with Cancer UK (CPTSTSFA\100003) and would like to acknowledge their support. We would like to thank all staff who have contributed to patients’ care and especially acknowledge the late Amit Patel for his commitment to haematology and cell therapy. In addition, we would like to acknowledge all patients and their families.AbstractCAR T-cell therapy has transformed relapsed/refractory (r/r) B-cell precursor acute lymphoblastic leukaemia (B-ALL) management and outcomes, but following CAR T infusion, interventions are often needed. In a UK multicentre study, we retrospectively evaluated tisagenlecleucel outcomes in all eligible patients, analysing overall survival (OS) and event-free survival (EFS) with standard and stringent definitions, the latter including measurable residual disease (MRD) emergence and further anti-leukaemic therapy. Both intention-to-treat and infused cohorts were considered. We collected data on feasibility of delivery, manufacture, toxicity, cause of therapy failure and followed patients until death from any cause. Of 142 eligible patients, 125 received tisagenlecleucel, 115/125 (92%) achieved complete remission (CR/CRi). Severe cytokine release syndrome and neurotoxicity occurred in 16/123 (13%) and 10/123 (8.1%), procedural mortality was 3/126 (2.4%). The 2-year intent to treat OS and EFS were 65.2% (95%CI 57.2–74.2%) and 46.5% (95%CI 37.6–57.6%), 2-year intent to treat stringent EFS was 35.6% (95%CI 28.1–44.9%). Median OS was not reached. Sixty-two responding patients experienced CAR T failure by the stringent event definition. Post failure, 1-year OS and standard EFS were 61.2% (95%CI 49.3–75.8) and 55.3% (95%CI 43.6–70.2). Investigation of CAR T-cell therapy for B-ALL delivered on a country-wide basis, including following patients beyond therapy failure, provides clinicians with robust outcome measures. Previously, outcomes post CAR T-cell therapy failure were under-reported. Our data show that patients can be successfully salvaged in this context with good short-term survival.</jats:p