- Moskop, Amy;
- Pommert, Lauren;
- Baggott, Christina;
- Prabhu, Snehit;
- Pacenta, Holly L;
- Phillips, Christine L;
- Rossoff, Jenna;
- Stefanski, Heather;
- Talano, Julie-An;
- Margossian, Steven P;
- Verneris, Michael R;
- Myers, Gary Doug;
- Karras, Nicole A;
- Brown, Patrick A;
- Qayed, Muna;
- Hermiston, Michelle L;
- Satwani, Prakash;
- Krupski, Christa;
- Keating, Amy K;
- Wilcox, Rachel;
- Rabik, Cara A;
- Fabrizio, Vanessa A;
- Chinnabhandar, Vasant;
- Goksenin, A Yasemin;
- Curran, Kevin J;
- Mackall, Crystal L;
- Laetsch, Theodore W;
- Guest, Erin M;
- Breese, Erin H;
- Schultz, Liora M
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.