TURALIO^® Risk Evaluation and Mitigation Strategy Program (tREMS): 3-year retrospective hepatic safety assessment

Area of Science:

• Hepatology
• Pharmacovigilance
• Drug Safety

Background:

• Pexidartinib (TURALIO) is a targeted therapy requiring careful safety monitoring.
• The TURALIO Risk Evaluation and Mitigation Strategy (tREMS) program was implemented to ensure safe use.
• Assessing hepatic safety is crucial due to potential liver injury risks associated with targeted therapies.

Purpose of the Study:

• To evaluate the hepatic safety data from the TURALIO Risk Evaluation and Mitigation Strategy (tREMS) Program.
• To identify and characterize any hepatic adverse events or laboratory abnormalities in patients receiving pexidartinib.
• To assess the effectiveness of risk mitigation strategies in preventing severe hepatotoxicity.

Main Methods:

• A retrospective analysis of hepatic events was conducted over a 3-year period (August 2019 to June 2022).
• Data were collected from patients enrolled and certified within the tREMS program.
• Hepatic adverse events and laboratory abnormalities suggestive of liver injury were identified and analyzed.

Main Results:

• A total of 451 patients were enrolled in the tREMS program.
• Twenty-one patients (4.7%) experienced hepatic adverse events or laboratory abnormalities.
• All identified hepatic events occurred within two months of initiating pexidartinib therapy, with no new safety signals detected.

Conclusions:

• The hepatic safety profile observed in the tREMS program is consistent with previous clinical trial data (e.g., the ENLIVEN trial).
• Proactive liver enzyme monitoring in patients treated with pexidartinib is essential.
• Early intervention, including dose adjustments and treatment discontinuation, effectively mitigates the risk of pexidartinib-induced hepatotoxicity.