HealthDay News — A single infusion of the anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, tisagenlecleucel, provides durable remission in pediatric and young adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL), according to a study published in the February 1 issue of the New England Journal of Medicine.

Shannon L. Maude, MD, PhD, from the Perelman School of Medicine in Philadelphia, and colleagues conducted a study of tisagenlecleucel in pediatric and young adult patients with CD19+ relapsed or refractory B-cell ALL. Seventy-five patients received a tisagenlecleucel infusion and could be assessed for efficacy. 

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The researchers found that within 3 months, the overall remission rate was 81%, with all patients who had a response to treatment found to be negative for minimal residual disease. At 6 and 12 months, the rates of event-free and overall survival were 73 and 90% and 50 and 76%, respectively. The median remission duration was not reached. For as long as 20 months, persistence of tisagenlecleucel in the blood was observed. Seventy-three percent of patients had grade 3 or 4 adverse events that were suspected to be related to tisagenlecleucel.

“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 effect,” the authors write.

The study was funded by Novartis, the manufacturer of tisagenlecleucel.

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