Pexidartinib is the first approved medication in the USA for people with
tenosynovial giant cell tumor (TGCT). The
drug was approved based on the ENLIVEN study, which looked at
pexidartinib (brand name, Turalio™), a medication taken by mouth (orally) for people with TGCT (also known as
giant cell tumor of the tendon sheath [GCTTS] and
pigmented villonodular synovitis [PVNS]) who are not able to have surgery because of the location and/or the size of the
tumor. The study showed that
pexidartinib is effective in treating people with TGCT because it shrunk the size of their
tumors and improved their symptoms and their ability to function. In general, people treated with
pexidartinib had side effects that were mostly mild that went away
after treatment with
pexidartinib was stopped. The most common side effects were hair color changes and tiredness (
fatigue).
Pexidartinib was also associated with liver problems (or hepatotoxicity), which started within the first 2 months of treatment. Due to the risk of liver problems, which may be severe and potentially life threatening, the researchers closely monitored participants' blood liver function tests before, during, and after participants in the study took
pexidartinib. To read the full Plain Language Summary of this article, click on the View Article button above and download the PDF. Clinical Trial Registration: NCT02371369 (ClinicalTrials.gov).