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.
Authors
- Noor Ul Ain (ORCID: https://orcid.org/0000-0003-3097-2997)
- Audrey Ruan
- Aaron S. Burton
- Pramod K. Mistry (ORCID: https://orcid.org/0000-0003-3447-6421)
- Armaan Saith
- Ruhua Yang
Institutions
- Yale New Haven Hospital (US)
- Yale University (US)
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