Open AccessENLIVEN study: Pexidartinib for tenosynovial giant cell tumor (TGCT)
Author(s) -
William D. Tap
Publication year - 2020
Publication title -
future oncology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.857
H-Index - 72
eISSN - 1744-8301
pISSN - 1479-6694
DOI - 10.2217/fon-2020-0307
Subject(s) - medicine , tendon sheath , pigmented villonodular synovitis , dermatology , clinical trial , liver function , synovitis , surgery , tendon , arthritis
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. Clinical Trial Registration: NCT02371369 ( ClinicalTrials.gov )