• Media type: E-Article
  • Title: Real-world use of tisagenlecleucel in infant acute lymphoblastic leukemia
  • Contributor: Moskop, Amy; Pommert, Lauren; Baggott, Christina; Prabhu, Snehit; Pacenta, Holly L.; Phillips, Christine L.; Rossoff, Jenna; Stefanski, Heather E.; Talano, Julie-An; Margossian, Steve P.; Verneris, Michael R.; Myers, G. 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; [...]
  • imprint: American Society of Hematology, 2022
  • Published in: Blood Advances
  • Language: English
  • DOI: 10.1182/bloodadvances.2021006393
  • ISSN: 2473-9529; 2473-9537
  • Keywords: Hematology
  • Origination:
  • Footnote:
  • Description: <jats:title>Abstract</jats:title> <jats:p>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 &amp;lt;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 (&amp;gt;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.</jats:p>
  • Access State: Open Access