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Updated: Oct 18, 2025

Pre-clinical Evaluation of Tyrosine Kinase Inhibitors for Treatment of Acute Leukemia
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Targeted Therapy in the Treatment of Pediatric Acute Lymphoblastic Leukemia-Therapy and Toxicity Mechanisms

Monika Lejman1, Kinga Kuśmierczuk2, Kinga Bednarz2

  • 1Laboratory of Genetic Diagnostics, Medical University of Lublin, A. Gębali 6, 20-093 Lublin, Poland.

International Journal of Molecular Sciences
|September 28, 2021
PubMed

Insights

Targeted therapies improve outcomes for pediatric acute lymphoblastic leukemia (ALL) with genetic mutations. This review examines the toxicity of these novel treatments in children.

Area of Science:

  • Pediatric Oncology
  • Hematology
  • Pharmacology

Background:

  • Targeted therapy and immunotherapy represent a paradigm shift in treating high-risk pediatric acute lymphoblastic leukemia (ALL).
  • These novel approaches aim to enhance treatment efficacy and reduce conventional chemotherapy doses.
  • Advances in molecular diagnostics enable precise identification of genetic abnormalities in ALL, facilitating personalized treatment strategies.

Purpose of the Study:

  • To review the existing knowledge on the toxicity of molecular-targeted drugs and immunotherapies used in pediatric ALL.
  • To highlight the potential adverse events associated with these advanced therapeutic modalities in children and adolescents.

Main Methods:

  • Comprehensive literature review of studies on targeted therapy and immunotherapy in pediatric ALL.
  • Analysis of clinical data and research focusing on the toxicological profiles of these treatments.
  • Synthesis of information regarding specific molecular alterations and their targeted agents.

Main Results:

  • Molecularly targeted drugs and immunotherapies have shown promise in improving outcomes for pediatric ALL.
  • Limited clinical data currently exists on the specific toxic events associated with these therapies in pediatric populations.
  • Identification of unique expression profiles and genetic mutations aids in risk stratification and personalized therapy selection.

Conclusions:

  • Targeted therapies offer a promising avenue for treating genetically defined pediatric ALL, potentially reducing chemotherapy burden.
  • Further clinical research is crucial to fully understand and manage the toxicity of these advanced treatments in children.
  • Personalized therapeutic strategies based on molecular profiling are key to optimizing treatment and minimizing adverse events in pediatric ALL.
Keywords:
acute lymphoblastic leukemiaimmunotherapymolecular toxicitypediatricside effectstarget therapytyrosine kinase inhibitors

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