Pediatrics 2027

Introduction: Relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) remains a challenge in pediatric oncology. CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy has produced deep remissions, but relapse and treatment-related toxicity remain important concerns.

Objective: To evaluate the efficacy and safety of CAR-T therapy in children and adolescents with relapsed or refractory B-ALL.

Methods: An updated systematic search of PubMed was performed to identify clinical studies evaluating CAR-T therapy in pediatric patients with relapsed/refractory B-ALL. Outcomes included complete remission (CR/CRi), measurable residual disease (MRD) negativity, event-free survival (EFS), relapse-free survival (RFS), overall survival (OS), cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), infections, and prolonged cytopenias. Publications reporting the same cohort were considered longitudinal evidence to avoid double counting.

Results: The evidence demonstrates high initial response rates. In the pivotal ELIANA trial, 81% of infused patients achieved CR/CRi within 3 months, all with MRD-negative remission; 12-month EFS and OS were 50% and 76%, respectively. Long-term follow-up showed 5-year OS of 55%. Real-world data confirmed high remission rates, while pooled safety analyses reported CRS in 79%, infections in 42%, prolonged cytopenias in 40%, and neurologic events in 36%. More recent pediatric studies evaluating CD19 CAR-T and CD19/CD22 dual-target strategies continue to demonstrate high MRD-negative remission rates, while highlighting relapse, antigen escape, and neurotoxicity as persistent challenges.

Conclusion: CAR-T therapy provides substantial antileukemic activity and clinically meaningful survival in pediatric relapsed/refractory B-ALL. Continued prospective studies are needed to optimize patient selection, CAR-T design, toxicity management, and strategies to prevent relapse.

Ana Laura Tibolla Marti is a sixth-semester medical student at the Salesian Auxilium Catholic University Center in Araçatuba, São Paulo. She has had two papers on pediatric oncology published at highly reputable conferences.