Targeted therapies in pediatric B-Cell acute lymphoblastic leukemia: mechanisms, efficacy, and future directions
Targeted therapies improve progression‑free survival and overall response rates in pediatric B‑cell acute lymphoblastic leukemia.
Acute lymphoblastic leukemia (ALL) is the most common hematologic malignancy in children. In this systematic review, we comprehensively analyze targeted therapies—monoclonal antibodies, antibody‑drug conjugates, tyrosine kinase inhibitors, proteasome inhibitors, and CAR‑T‑cell immunotherapy—used in pediatric B‑cell ALL. Across 21 clinical studies, targeted approaches have shown higher progression‑free survival and overall response rates, particularly in relapsed or refractory disease. CD19‑directed CAR‑T‑cell therapy and bispecific antibodies such as blinatumomab achieved high remission rates in early‑phase trials, while tyrosine kinase inhibitors (imatinib, dasatinib) benefit BCR‑ABL1‑positive patients. Despite promising efficacy, long‑term safety data and access barriers remain challenges.
Improved targeted therapies offer more precise, less toxic treatment options for children with high‑risk or relapsed B‑cell ALL, potentially enhancing survival and quality of life while reducing systemic chemotherapy‑related morbidity.
Bizonyítékszint: Klinikai vizsgálat. Formális klinikai vizsgálati eredmény.
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