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Pharmacogenomics of Antineoplastic Therapy in Children: Genetic Determinants of Toxicity and Efficacy

Zaure Dushimova1, Timur Saliev2, Aigul Bazarbayeva3

  • 1Department of Fundamental Medicine, Al-Farabi Kazakh National University, Almaty 050040, Kazakhstan.

Pharmaceutics
|February 27, 2026
PubMed

Insights

Pharmacogenomics can personalize pediatric cancer treatment by analyzing genetic variations that affect drug response. This approach aims to improve treatment effectiveness and reduce side effects in children with cancer.

Area of Science:

  • Pediatric Oncology
  • Pharmacogenomics
  • Cancer Therapeutics

Background:

  • Multimodal cancer therapy has improved survival for children, but treatment response and toxicity vary due to genetic factors affecting drug metabolism.
  • Pharmacogenomics offers a personalized approach to optimize anticancer drug efficacy and minimize adverse effects in pediatric patients.

Purpose of the Study:

  • To review current evidence on pharmacogenetic variants impacting response to major antineoplastic agents in children.
  • To discuss challenges and future directions for implementing pharmacogenomics in pediatric cancer care.

Main Methods:

  • Literature review synthesizing evidence on pharmacogenetic variants and their association with drug response in pediatric oncology.
  • Discussion of established gene-drug associations (e.g., TPMT, NUDT15) and emerging biomarkers from multi-omics studies.
  • Examination of challenges to clinical implementation and potential solutions using advanced technologies.

Main Results:

  • Key pharmacogenetic variants influencing response to thiopurines, methotrexate, anthracyclines, alkylating agents, vinca alkaloids, and platinum compounds are identified.
  • Established gene-drug associations and novel biomarkers from genome-wide and multi-omics studies are presented.
  • Challenges including cost, infrastructure, population variability, and ethics are outlined, alongside potential technological solutions.

Conclusions:

  • Pharmacogenomic testing holds significant potential to transform pediatric cancer care by enhancing drug safety and treatment precision.
  • Integration of pharmacogenomics into clinical protocols can lead to ethically grounded, personalized therapy for children with cancer.
  • Future directions involve leveraging multi-omics, systems pharmacology, and AI to accelerate clinical translation of pharmacogenomic findings.
Keywords:
DPYDNUDT15TPMTartificial intelligencechemotherapy toxicitydrug efficacygenetic polymorphismsmulti-omicspediatric oncologypersonalized therapypharmacogenomics

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