Key result
Ticagrelor cuts recurrent stroke risk ~34% in CYP2C19 LOF carriers.
Why the study?
Despite accumulating evidence that pharmacogenomic variations significantly influence responses to stroke medications, genetic testing has not yet been widely integrated into stroke care.
Does pharmacogenomic testing improve the efficacy and safety of stroke pharmacotherapy?
RCT (n=6,412)
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Yes
Does pharmacogenomic testing improve the efficacy and safety of stroke pharmacotherapy?
Effect estimate: RR 0.66 (95% CI 0.48-0.99)
p-value: p=0.009
Pharmacogenomic testing for CYP2C19, VKORC1, CYP2C9, SLCO1B1, and ABCG2 can optimize stroke pharmacotherapy, particularly in East Asian populations with high frequencies of actionable variants.
May inform CYP2C19-guided ticagrelor selection in East Asian stroke patients; leaves open routine adoption pending prospective trials.
Pharmacogenomic variations may significantly influence responses to commonly prescribed stroke medications. Despite accumulating evidence, genetic testing has not yet been widely integrated into stroke care. This review summarizes current evidence and provides practical guidance for clinical implementation. Pharmacogenomic studies and clinical guidelines related to antiplatelet agents, anticoagulants, and statins were reviewed, with particular emphasis on East Asian populations. Substantial evidence supports genotype-guided use of clopidogrel (CYP2C19), warfarin (CYP2C9, VKORC1, CYP4F2), and statins (SLCO1B1, ABCG2). For aspirin, PTGS1/2 and PEAR1 variants have been investigated; however, current data remain insufficient for clinical application. Regarding direct oral anticoagulants (DOACs), candidate genes such as ABCB1 and CES1 demonstrate pharmacokinetic associations, though robust clinical outcome data are lacking. Distinct allele frequencies in East Asians—such as higher prevalence of CYP2C19 and ABCG2 variants—underscore the need for population-specific strategies. Beyond single-gene approaches, polygenic risk scores, pharmacogenomic panels, and integration with multi-omics data and artificial intelligence represent promising directions for personalized therapy. Pharmacogenomic testing can enhance stroke pharmacotherapy, particularly in populations with high frequencies of actionable variants. Broader implementation requires rapid testing platforms, clinician education, tailored clinical guidelines, and real-world validation of aspirin, DOACs, and multi-gene approaches. Future research should expand population-specific studies and integrate pharmacogenomics within the broader framework of precision medicine to ensure equitable clinical benefit.
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Lee et al. (2026) conducted an RCT in Minor ischemic stroke or high-risk transient ischemic attack (n=6,412). Ticagrelor vs. Clopidogrel was evaluated on Risk of recurrent stroke (RR 0.66, 95% CI 0.48-0.99, p=0.009). Ticagrelor reduced the risk of recurrent stroke by 34% among CYP2C19 LOF carriers in the CHANCE-2 trial.
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