Tiopronin safety in cystinuria: first real-world pharmacovigilance analysis using the FDA Adverse Event Reporting System (FAERS): an EAU YAU and Endourology sections review
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Summary
This summary is machine-generated.Tiopronin is a key treatment for cystinuria, but real-world safety data are limited. This study identified new safety concerns, especially in children, highlighting the need for better monitoring and education.
Area Of Science
- Pharmacovigilance
- Rare genetic disorders
- Pediatric pharmacology
Background
- Cystinuria is a rare genetic disorder causing recurrent kidney stones, primarily affecting children.
- Tiopronin is the main pharmacologic treatment for cystinuria when conservative measures fail.
- Real-world safety data for tiopronin are scarce, necessitating further investigation.
Purpose Of The Study
- To evaluate the adverse-event profile of tiopronin using real-world data.
- To identify known and novel safety signals associated with tiopronin use.
- To assess the safety of tiopronin, particularly in the pediatric population.
Main Methods
- Utilized the FDA Adverse Event Reporting System (FAERS) database from Q1 2014 to Q1 2025.
- Included deduplicated cases where tiopronin was the primary suspect drug.
- Employed MedDRA coding and multiple disproportionality algorithms for signal detection.
Main Results
- Identified 1,838 unique cases, with nearly 70% involving pediatric patients.
- Detected significant safety signals in seven System Organ Classes and 67 Preferred Terms.
- Observed unlabelled safety signals including hyposmia, skin atrophy, breath odor, and incorrect dosage, with the latter being critical in children.
Conclusions
- This pharmacovigilance study is the first FAERS-based analysis of tiopronin safety.
- Identified both known and novel safety signals, emphasizing challenges in pediatric dosing.
- Stressed the need for enhanced pharmacovigilance, accurate treatment oversight, and improved patient/clinician education.
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