Real-world use of tisagenlecleucel in infant acute lymphoblastic leukemia.

Abstract

Infants with B-cell acute lymphoblastic leukemia (B-ALL) have poor outcomes because of chemotherapy resistance leading to high relapse rates. Tisagenlecleucel, a CD19-directed chimeric antigen receptor T-cell (CART) therapy, is US Food and Drug Administration approved for relapsed or refractory B-ALL in patients ≤25 years; however, the safety and efficacy of this therapy in young patients is largely unknown because children <3 years of age were excluded from licensing studies. We retrospectively evaluated data from the Pediatric Real-World CAR Consortium to examine outcomes of patients with infant B-ALL who received tisagenlecleucel between 2017 and 2020 (n = 14). Sixty-four percent of patients (n = 9) achieved minimal residual disease-negative remission after CART and 50% of patients remain in remission at last follow-up. All patients with high disease burden at time of CART infusion (>M1 marrow) were refractory to this therapy (n = 5). Overall, tisagenlecleucel was tolerable in this population, with only 3 patients experiencing ≥grade 3 cytokine release syndrome. No neurotoxicity was reported. This is the largest report of tisagenlecleucel use in infant B-ALL and shows that this therapy is safe and can be effective in this population. Incorporating this novel immunotherapy into the treatment of infant B-ALL offers a promising therapy for a highly aggressive leukemia.

• Authors: Amy Moskop, Lauren Pommert, Christina Baggott, Snehit Prabhu, Holly L Pacenta, Christine L Phillips, Jenna Rossoff, Heather E Stefanski, Julie-An Talano, Steve P Margossian, Michael R Verneris, G Doug Myers, Nicole A Karras, Patrick A Brown, Muna Qayed, Michelle L Hermiston, Prakash Satwani, Christa Krupski, Amy K Keating, Rachel Wilcox, Cara A Rabik, Vanessa A Fabrizio, Vasant Chinnabhandar, A Yasemin Goksenin, Kevin J Curran, Crystal L Mackall, Theodore W Laetsch, Erin M Guest, Erin H Breese, Liora M Schultz
• Journal: Blood advances (Blood Adv) Vol. 6 Issue 14 Pg. 4251-4255 (07 26 2022) ISSN: 2473-9537 [Electronic] United States
• PMID: 35580324 (Publication Type: Journal Article)
• Copyright: Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.

Chemical References

• Antigens, CD19
• Receptors, Antigen, T-Cell
• Receptors, Chimeric Antigen
• tisagenlecleucel

Topics

• Antigens, CD19 (immunology, therapeutic use)
• Child
• Humans
• Precursor Cell Lymphoblastic Leukemia-Lymphoma (therapy)
• Receptors, Antigen, T-Cell (therapeutic use)
• Receptors, Chimeric Antigen (therapeutic use)
• Retrospective Studies
• United States