CART-Cell Therapy in Pediatric Acute Lymphoblastic Leukemia: A Review for General Pediatricians
Cet article passe en revue les connaissances sur les thérapies par lymphocytes CAR-T pour les leucémies lymphoblastiques aiguës chez l'enfant et le jeune adulte, résume les principes clés concernant leurs indications, leur production, leur efficacité et leurs complications puis examine les enjeux en matière de soins partagés et de suivi
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.
Pediatrics , résumé, 2026