Pharmacogenetics of graft-versus-host disease: a path to personalized medicine

Piotr Łacina1, Jagoda Siemaszko1, Katarzyna Bogunia-Kubik1

  • 1Laboratory of Clinical Immunogenetics and Pharmacogenetics, Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Wroclaw, Poland.

Pharmacogenomics
|August 7, 2025
PubMed

Insights

Pharmacogenetics can personalize graft-versus-host disease (GvHD) treatment after stem cell transplants. Understanding genetic variants optimizes drug selection and dosing for better GvHD management and fewer side effects.

Area of Science:

  • Hematology
  • Immunology
  • Pharmacogenetics

Background:

  • Graft-versus-host disease (GvHD) is a major complication of allogeneic hematopoietic stem cell transplantation (HSCT).
  • Current GvHD prophylaxis and treatment strategies require continuous improvement to reduce morbidity and mortality.
  • Personalized medicine approaches are increasingly important in optimizing patient care.

Purpose of the Study:

  • To review the role of pharmacogenetic variants in the prophylaxis and management of GvHD.
  • To explore how pharmacogenetics can personalize GvHD treatment strategies.
  • To highlight the need for integrating pharmacogenetics into clinical practice for GvHD.

Main Methods:

  • Literature review of pharmacogenetic studies related to GvHD.
  • Analysis of pharmacogenetic variants influencing key GvHD drugs: calcineurin inhibitors, methotrexate, mycophenolate mofetil, cyclophosphamide, and corticosteroids.
  • Synthesis of current evidence on pharmacogenetics in GvHD prophylaxis and treatment.

Main Results:

  • Pharmacogenetic variants significantly impact the efficacy and toxicity of GvHD prophylactic and therapeutic agents.
  • Specific genetic profiles can predict patient response and risk of adverse events to certain immunosuppressants.
  • Personalized drug selection and dosing based on pharmacogenetics holds promise for improved GvHD outcomes.

Conclusions:

  • Pharmacogenetics offers a powerful tool for tailoring GvHD prophylaxis and treatment, moving towards precision medicine in HSCT.
  • Further pharmacogenetic association studies are essential to expand the evidence base.
  • Clinical implementation of pharmacogenetic testing is crucial for optimizing GvHD management and patient care.

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