Medical College of Wisconsin
CTSICores SearchResearch InformaticsREDCap

Real-world use of tisagenlecleucel in infant acute lymphoblastic leukemia. Blood Adv 2022 07 26;6(14):4251-4255



Pubmed ID


Pubmed Central ID




Scopus ID



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 a??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 a?Ygrade 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.

Author List

Moskop A, Pommert L, Baggott C, Prabhu S, Pacenta HL, Phillips CL, Rossoff J, Stefanski HE, Talano JA, Margossian SP, Verneris MR, Myers GD, Karras NA, Brown PA, Qayed M, Hermiston ML, Satwani P, Krupski C, Keating AK, Wilcox R, Rabik CA, Fabrizio VA, Chinnabhandar V, Goksenin AY, Curran KJ, Mackall CL, Laetsch TW, Guest EM, Breese EH, Schultz LM


Amy Moskop MD Assistant Professor in the Pediatrics department at Medical College of Wisconsin
Julie-An M. Talano MD Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Antigens, CD19
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Receptors, Antigen, T-Cell
Retrospective Studies
United States