Shannon L. Maude, MD, PhD, attending physician, Division of Oncology, The Children’s Hospital of Philadelphia; assistant professor, pediatrics, medical director, Center for Cellular Immunotherapies, Perelman School of Medicine, the University of Pennsylvania, discusses notable long-term safety data with tisagenlecleucel (Kymriah) in pediatric patients with acute lymphoblastic leukemia (ALL).

The phase 2 ELIANA study (NCT02435849) investigated the efficacy and safety of tisagenlecleucel in pediatric patients with relapsed/refractory B-cell ALL (B-ALL). A 5-year follow-up of these data was presented at the 2022 EHA Congress.

Although tisagenlecleucel has known, common adverse effects (AEs) such as cytokine release syndrome (CRS) and neurotoxicity, no additional AEs of special interest were reported at the 5-year analysis of the study, Maude says. Initial study results showed the incidence of CRS to be significant, occurring in 77% of patients, including grade 3/4 in 48%, though many patients had a substantial disease burden at the time of treatment, Maude explains. Neurotoxicity was also seen in 40% of patients, Maude adds.

This longer-term follow-up of the ELIANA data provided the opportunity to look for late AEs, primarily infection and prolonged cytopenia, Maude says. Among patients in remission, any-grade infection occurred in 33% of patients, including grade 3 or higher in 20%, and hematological disorders of any grade, including cytopenia, occurred in 10% of patients, including grade 3 or higher in 6%. No other serious or unexpected late AEs have been reported, Maude concludes.

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Shannon L. Maude, MD, PhD, discusses notable long-term safety data with tisagenlecleucel in pediatric patients with acute lymphoblastic leukemia. 

Shannon L. Maude, MD, PhD

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Dr. Maude on Safety of Tisagenlecleucel in Pediatric B-ALL