Maude SL, et al. - Researchers attempted to scrutinize the efficacy of anti-CD19 chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel in children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). Durable remission with long-term persistence was yielded through a single infusion of tisagenlecleucel among the study cohort, displaying transient high-grade toxic effects.

Methods

• The scheme of this research was a phase 2, single-cohort, 25-center, global study of tisagenlecleucel.
• Researchers analyzed pediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL.
• The overall remission rate (the rate of complete remission or complete remission with incomplete hematologic recovery) within 3 months served as the primary end point.

Results

• Herein, 75 subjects received an infusion of tisagenlecleucel, which was assessed for its efficacy.
• As per the outcomes, the overall remission rate within 3 months was 81%, with all patients who had a response to treatment found to be negative for minimal residual disease, revealed via flow cytometry.
• It was determined that the rates of event-free survival and overall survival were 73% (95% confidence interval [CI], 60 to 82) and 90% (95% CI, 81 to 95), respectively, at 6 months and 50% (95% CI, 35 to 64) and 76% (95% CI, 63 to 86) at 12 months.
• Data indicated that the median duration of remission was not attained.
• Experts illustrated the persistence of tisagenlecleucel in the blood for as long as 20 months.
• In addition, the occurrence of grade 3 or 4 adverse events were discovered in 73% of the candidates, wherein the adverse events were suspected to be associated with tisagenlecleucel.
• It was illustrated that 77% of the patients presented with the cytokine release syndrome, 48% of whom received tocilizumab.
• Data displayed that 40% of the patients presented with neurologic events.
• These events were managed with supportive care, with no report of cerebral edema.