B-ALL Complexity: Is Targeted Therapy Still A Valuable Approach for Pediatric Patients?

Stefano Ratti, Annalisa Lonetti, Matilde Y Follo, Francesca Paganelli, Alberto M Martelli, Francesca Chiarini, Camilla Evangelisti

Research output: Contribution to journalReview articlepeer-review


B-cell acute lymphoblastic leukemia (B-ALL) is a hematologic malignancy that arises from the clonal expansion of transformed B-cell precursors and predominately affects childhood. Even though significant progresses have been made in the treatment of B-ALL, pediatric patients' outcome has to be furtherly increased and alternative targeted treatment strategies are required for younger patients. Over the last decade, novel approaches have been used to understand the genomic landscape and the complexity of the molecular biology of pediatric B-ALL, mainly next generation sequencing, offering important insights into new B-ALL subtypes, altered pathways, and therapeutic targets that may lead to improved risk stratification and treatments. Here, we will highlight the up-to-date knowledge of the novel B-ALL subtypes in childhood, with particular emphasis on altered signaling pathways. In addition, we will discuss the targeted therapies that showed promising results for the treatment of the different B-ALL subtypes.

Original languageEnglish
Pages (from-to)1-27
Number of pages27
Issue number12
Publication statusPublished - Nov 24 2020


  • childhood
  • B-ALL
  • signaling pathway
  • target therapy


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