CART-Cell Therapy in Pediatric Acute Lymphoblastic Leukemia: A Review for General Pediatricians

Chimeric antigen receptor (CAR) T cell therapy has transformed outcomes for children and young adults with relapsed/refractory CD19+ B-cell acute lymphoblastic leukemia (B-ALL), enabling deep remissions even in patients who are refractory to chemotherapy or have relapsed after hematopoietic stem cell transplantation (HSCT). Use of adoptive cellular immunotherapy requires a strict clinical pathway and is delivered at designated CART-cell centers. Initial complete remission rates are high and often MRD (minimal residual disease)-negative, but relapse remains frequent through loss of persistence or antigen escape. Key acute toxicities are cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), whereas longer-term issues include prolonged cytopenias, infection risk, B-cell aplasia, and hypogammaglobulinemia requiring immunoglobulin replacement. This narrative review for general pediatricians reviews the current state-of-the-art of CART-cell therapy for ALL and summarizes key principles about CART-cell indications, production, outcomes, complications, and relevant issues for shared-care centers on follow-up in the short and long term.