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Leo I. Gordon, MD, FACP


Treatment With Tisagenlecleucel for Infants and Toddlers With B-Cell ALL

By: Joseph Fanelli
Posted: Friday, December 2, 2022

For younger children and infants with B-cell precursor acute lymphoblastic leukemia (ALL), tisagenlecleucel may produce antitumor activity and seems to have an acceptable safety profile, according to international study findings presented in The Lancet Haematology. Treatment with with autologous cellular immunotherapy was consistent with results in older children and adults enrolled in the phase II ELIANA trial, concluded André Baruchel, MD, of Hôpital Universitaire Robert Debré, Paris, and colleagues.

“If confirmed with longer follow-up, [the findings] support the development of prospective studies to compare the efficacy of tisagenlecleucel against [hematopoietic stem cell transplantation] earlier in the course of therapy in this population,” the authors concluded.

In this multicenter, retrospective cohort study, the authors focused on 38 patients younger than age 3 who had relapsed or refractory B-cell precursor ALL. Of the patients, 35 received a tisagenlecleucel infusion. Among the group, 29 patients had KMT2A-rearranged ALL, and 25 had relapsed following previous allogeneic hematopoietic stem cell transplantation. A total of 7 patients had received inotuzumab and 14 received blinatumomab.

After a median of 14 months of follow-up, the 12-month overall survival rate following tisagenlecleucel infusion was 84%, the event-free survival rate was 69%, and the stringent event-free survival was 41%. The authors found that the probability of ongoing B-cell aplasia was 70% after 12 months of treatment. The most common adverse event was cytokine-release syndrome, which was reported at any grade in 21 patients (60%) and at grade 3 or higher in 5 patients (14%).

Disclosure: For full disclosures of the study authors, visit

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