ENLIVEN study: Pexidartinib for tenosynovial giant cell tumor (TGCT)
Abstract
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)
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Financial & competing interests disclosure
William Tap, MD, of Memorial Sloan Kettering Cancer Center and Weill Cornell Medical College, New York, NY, was Principal Investigator of the ENLIVEN study. All research at Memorial Sloan Kettering is supported in part by a grant from the National Institutes of Health National Cancer Institute (#P30 CA008748). William D. Tap has served as a consultant to Eli Lilly, EMD Serono, Novartis, Eisai, Janssen, Immune Design, Adaptimmune, Daiichi Sankyo, Blueprint, Loxo, GlaxoSmithKline, Agios Pharmaceuticals, Plexxikon Pharmaceuticals, Nanno Carrier and has served on a scientific advisory board and has stock ownership in Certis Oncology Solution and Atropos Therapeutics. William D. Tap holds patents for companion diagnostic for CDK4 inhibitors- 14/854,329 pending to MSKCC/ SKI and a patent for Methods of Treating Metastatic Sarcoma Using Talimogene Laherparepvec (T-VEC) and pembrolizumab combination therapy- 62/671,625 pending to MSK/SKI. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed
Medical writing and editorial assistance in the development of this article were provided by SciStrategy Communications, Harra Feinberg, PhD, and Susan Wingeron, MBA, CMPP. Research and manuscript support were provided by Daiichi Sankyo Co., Ltd. (Tokyo, Japan).
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