Shannon L. Maude, MD, PhD, on Favorable Safety of Tisagenlecleucel in Pediatric B-ALL

This content originally appeared on our sister site, OncLive.

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

OncLive spoke with Shannon L. Maude, MD, PhD, attending physician, Division of Oncology, The Children’s Hospital of Philadelphia, and assistant professor, pediatrics, medical director, Center for Cellular Immunotherapies, Perelman School of Medicine, the University of Pennsylvania, to learn more about long-term safety data with tisagenlecleucel (Kymriah) in pediatric patients with B-ALL.

The study revealed common adverse effects (AEs) such as cytokine release syndrome (CRS) and neurotoxicity but no additional AEs of special interest were reported at the 5-year analysis. CRS incidence was 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 explained. 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.