TURALIO® Risk Evaluation and Mitigation Strategy (tREMS) program: 5-year retrospective hepatic safety assessment

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Abstract
To assess hepatic safety data of pexidartinib in adult patients with tenosynovial giant cell tumor (TGCT) from the retrospective 5-year evaluation of the TURALIO® Risk Evaluation and Mitigation Strategy (tREMS) Program. We analyzed data collected from healthcare professionals, patients, pharmacies, and distributors who participated in the tREMS Program from August 2019 to June 2024. For the current reporting period, June 2022 to June 2024, of the 524 patients enrolled in the tREMS with ≥ 1 dispense of pexidartinib, 24 (4.6%) had adverse events or laboratory abnormalities suggestive of serious and potentially fatal liver injury, with a total of 45/735 (6.1%) patients over 5 years. One (0.2%) patient in the current period had confirmed vanishing bile duct syndrome (VBDS) and was recovering at last follow-up. Another patient from the previous reporting period had unconfirmed VBDS and was alive with unknown recovery status at last follow-up. For the current reporting period and cumulative 5-year tREMS assessment, the reported frequency and pattern of liver injury were consistent with the phase 3 ENLIVEN trial. The tREMS Program effectively mitigates the risk of hepatotoxicity through careful monitoring and early intervention. Pexidartinib remains a viable treatment option for TGCT when managed according to recommended guidelines.
Funding Information
  • Daiichi Sankyo, Inc

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