Real-world data confirmed the efficacy and safety profiles of tisagenlecleucel in patients with relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL) younger than 3 years old, according to a presentation at the 2022 American Society of Pediatric Hematology/Oncology Conference.

Previous data suggested that real-world outcomes are similar to those observed in clinical trials of tisagenlecleucel in patients with R/R ALL age 3 or older. However, children younger than 3 years were not enrolled in the clinical trials. The aim of the current study was to evaluate the real-world outcomes of this population after treatment with tisagenlecleucel.

The retrospective study included data for 47 patients with R/R ALL from the CIBMTR database who received tisagenlecleucel. The median age of the cohort was 20.4 months, 51% were female, 17% had primary refractory disease, and 57.4% had relapsed disease. The majority of patients were transplant-naïve.

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Leukapheresis was performed when patients were a median age of 18 months (range, 3-35 months). The chimeric antigen receptor (CAR) T-cell dose was a median of 2.4 x 106 /kg, with a viability of 89.8%. The median time from diagnosis to CAR-T infusion was 251 days, and the median time from apheresis to infusion was 46 days.

Among patients evaluable for efficacy, there were 76.3% who achieved a complete response within 100 days after infusion. At 3 months, the response rate was 79.9%, as was the rate of relapse-free survival. The median duration of response and relapse-free survival were not reached. The median event-free survival was 9.7 months, with a 3-month rate of 65.2%.

Cytokine release syndrome (CRS) occurred in 76.3% of patients. It was primarily mild to moderate, with 7.3% of patients experience grade 3 CRS. The median time to CRS onset was 6.5 days after infusion, and it lasted for a median of 6 days.

Neurotoxicity occurred in 12.2% of patients, including 7.3% with grade 3 neurotoxicity. The median time to onset was 9 days, and the median duration of neurotoxicity was 8 days. All cases of neurotoxicity resolved.

Progressive disease resulted in 2 deaths, and there were no deaths reported that were related to tisagenlecleucel treatment. The authors concluded that “registry data reveal high rates of durable response and a favorable safety profile in patients <3 years with R/R ALL treated with tisagenlecleucel.”

Disclosures: This research was supported by Novartis.


Guest E, Moskop A, Heim M, et al. Real-world outcomes for pediatric patients aged <3 years with R/R ALL treated with tisagenlecleucel. Presented at: 2022 American Society of Pediatric Hematology/Oncology (ASPHO) Conference; May 4-7, 2022. Abstract 2010.

This article originally appeared on Hematology Advisor