Eliglustat and cardiac comorbidities in Gaucher disease: a pharmacogenomic approach to safety and efficacy

This study represents the first real-world clinical evidence evaluating Eliglustat's cardiac safety in a high-risk GD1 population. Unlike prior theoretical concerns derived from <i>in vitro</i> ion channel studies, our findings demonstrate that Eliglustat does not induce clinically significant cardiac events when administered according to pharmacogenomic guidelines. The misinformation regarding Eliglustat's cardiotoxicity, largely driven by speculative interpretations rather than clinical data, is effectively countered by our findings, which show no significant QT prolongation or arrhythmias over a median treatment duration of 8 years.

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Publication Details

Journal
Frontiers in Medicine
Published
2025-03-17
DOI
https://doi.org/10.3389/fmed.2025.1535099
Citations
3
Primary Topic
Lysosomal Storage Disorders Research
Type
article
Field-Weighted Citation Impact
3.49
Controls
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article

Eliglustat and cardiac comorbidities in Gaucher disease: a pharmacogenomic approach to safety and efficacy

Noor Ul Ain, Audrey Ruan, Aaron S. Burton, Pramod K. Mistry et al.
3 citations
Frontiers in Medicine
Lysosomal Storage Disorders Research
3.49
article

Eliglustat and cardiac comorbidities in Gaucher disease: a pharmacogenomic approach to safety and efficacy

Noor Ul Ain, Audrey Ruan, Aaron S. Burton, Pramod K. Mistry, Armaan Saith, Ruhua Yang
article en
3 citations

Abstract

This study represents the first real-world clinical evidence evaluating Eliglustat's cardiac safety in a high-risk GD1 population. Unlike prior theoretical concerns derived from <i>in vitro</i> ion channel studies, our findings demonstrate that Eliglustat does not induce clinically significant cardiac events when administered according to pharmacogenomic guidelines. The misinformation regarding Eliglustat's cardiotoxicity, largely driven by speculative interpretations rather than clinical data, is effectively countered by our findings, which show no significant QT prolongation or arrhythmias over a median treatment duration of 8 years.

Frontiers in MedicineVol. 12
Yale New Haven Hospital (US), Yale University (US)
Openalex Percentile: Top 9%
Lysosomal Storage Disorders Research
3.49
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