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Tisagenlecleucel Shows Long-Term Safety, Efficacy in Pediatric ALL


By  Cecilia Brown - Last Updated: April 9, 2023

Tisagenlecleucel may be a “curative treatment option” for children and young adults with heavily pretreated relapsed or refractory acute lymphoblastic leukemia (ALL), according to results from the phase II ELIANA trial.

Theodore Laetsch, MD, of the Children’s Hospital of Philadelphia and the University of Pennsylvania and colleagues conducted the research and published their findings in the Journal of Clinical Oncology.

The primary analysis of the global phase II trial previously showed an overall remission rate of 81% at a median follow-up of 13.1 months in 75 children and young adults with relapsed or refractory B-cell ALL who received tisagenlecleucel.

Tisagenlecleucel is a CD19-directed chimeric antigen receptor (CAR) T-cell therapy approved by the US Food and Drug Administration for children, adolescents, and young adults who have relapsed or refractory B-cell ALL.

In the updated analysis, which had a median follow-up of 38.8 months, Dr. Laetsch and colleagues evaluated efficacy, safety, and patient-reported quality of life in 79 children and young adults with relapsed or refractory B-cell ALL.

The overall remission rate was 82%, with a median event-free survival (EFS) of 24 months. The EFS rate was 44%, with an overall survival (OS) rate of 63% at three years. The median OS was not reached. The estimated three-year relapse-free survival (RFS) rate was 52% with censoring for subsequent therapy, while it was 48% without censoring for subsequent therapy. In the 32 patients who responded to tisagenlecleucel and received no subsequent therapy while in remission, the estimated two-year RFS rate was 81%, while the estimated three-year RFS rate was 76%.

“Patients reported improvements in quality of life up to 36 months after infusion,” Dr. Laetsch and colleagues wrote.

The study’s authors did not report any new or unexpected long-term adverse events. Around one-third (29%) of patients had a grade 3 or grade 4 adverse event more than one year after CAR-T infusion. However, the rate of grade 3 to grade 4 infections did not increase more than one year following the infusion.

“These findings demonstrate favorable long-term safety and suggest tisagenlecleucel as a curative treatment option for heavily pretreated pediatric and young adult patients with [relapsed or refractory B-cell ALL].”


Laetsch TW, Maude SL, Rives S, et al. Three-year update of tisagenlecleucel in pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia in the ELIANA trial. J Clin Oncol. 2023;41(9):1664-1669. doi:10.1200/JCO.22.00642


Original Source: Tisagenlecleucel Shows Long-Term Safety, Efficacy in Pediatric ALL | Blood Cancers Today

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