Authors
Abdul Waheed Rajper, Charles Dharmani, Lalitha Chilakapati, Oluwatosin Fofah, Margaret J Wooddell
Published in
Patient related outcome measures. Volume 17. Issue 1. Pages 172-180. Epub Nov 05, 2026.
Abstract
To assess hepatic safety for adults with pexidartinib-treated tenosynovial giant cell tumor (TGCT) from the 6-year retrospective evaluation of the TURALIO® Risk Evaluation and Mitigation Strategy (tREMS) Program.
Retrospective hepatic safety data (08/2019-08/2025) were collected from healthcare professionals and patients in the tREMS Program.
During the current reporting period (06/2024-08/2025), 133/145 (91.7%) new patients received ≥1 pexidartinib dispense. For the cumulative 6-year reporting period, 878/929 (94.5%) patients received ≥1 pexidartinib dispense. Overall, 51/878 (5.8%) patients had adverse events or laboratory abnormalities suggesting serious and potentially fatal liver injury; of these, 47 (92.2%) discontinued pexidartinib, 3 (5.9%) interrupted doses, and 1 (2.0%) continued at the same dose. By August 2025, 34 (66.7%) events were fully resolved, 6 (11.8%) were resolving, 9 (17.6%) were unresolved, and 2 (3.9%) had unknown outcomes. There were no cases of irreversible liver injury, additional cases of vanishing bile duct syndrome, or new safety signals during the current reporting period.
The tREMS Program effectively mitigates hepatotoxicity risk through prompt intervention/reporting and timely dose modifications for adverse event management. When used according to guidelines, pexidartinib is a therapeutic option for adults with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery.
PMID:
43022703
Bibliographic data and abstract were imported from PubMed on 13 Nov 2026.
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