학술논문
Real-World Use of Tisagenlecleucel in Infant Acute Lymphoblastic Leukemia
Document Type
article
Author
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
Source
Blood Advances. 6(14)
Subject
Language
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 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.