Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia.
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Maude Shannon L, Laetsch Theodore W, Buechner Jochen, Rives Susana, Boyer Michael, Bittencourt Henrique, Bader Peter, Verneris Michael R, Stefanski Heather E, Myers Gary D, Qayed Muna, De Moerloose Barbara, Hiramatsu Hidefumi, Schlis Krysta, Davis Kara L, Martin Paul L, Nemecek Eneida R, Yanik Gregory A, Peters Christina, Baruchel Andre, Boissel Nicolas, Mechinaud Francoise, Balduzzi Adriana, Krueger Joerg, June Carl H, Levine Bruce L, Wood Patricia, Taran Tetiana, Leung Mimi, Mueller Karen T, Zhang Yiyun, Sen Kapildeb, Lebwohl David, Pulsipher Michael A, Grupp Stephan A
Аннотация
METHODS: We conducted a phase 2, single-cohort, 25-center, global study of tisagenlecleucel in pediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL. The primary end point was the overall remission rate (the rate of complete remission or complete remission with incomplete hematologic recovery) within 3 months.
RESULTS: For this planned analysis, 75 patients received an infusion of tisagenlecleucel and could be evaluated for efficacy. 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, as assessed by means of flow cytometry. 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. The median duration of remission was not reached. Persistence of tisagenlecleucel in the blood was observed for as long as 20 months. Grade 3 or 4 adverse events that were suspected to be related to tisagenlecleucel occurred in 73% of patients. The cytokine release syndrome occurred in 77% of patients, 48% of whom received tocilizumab. Neurologic events occurred in 40% of patients and were managed with supportive care, and no cerebral edema was reported.
CONCLUSIONS: In this global study of CAR T-cell therapy, a single infusion of tisagenlecleucel provided durable remission with long-term persistence in pediatric and young adult patients with relapsed or refractory B-cell ALL, with transient high-grade toxic effects. (Funded by Novartis Pharmaceuticals; ClinicalTrials.gov number, NCT02435849 .).