Giant Cell Tumor of Tendon Sheath Medication

Updated: Oct 18, 2022
  • Author: James R Verheyden, MD; Chief Editor: Harris Gellman, MD  more...
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Medication Summary

The FDA approved pexidartinib as the first systemic therapy approved for tenosynovial giant cell tumor (TGCT) in 2019. Pexidartinib is a first-in-class inhibitor of colony stimulating factor-1 receptor (CSF1R), a tyrosine kinase receptor. Approval was based on the ENLIVEN study, which demonstrated a significant overall response rate with pexidartinib as compared with placebo (38% vs 0%; P < .0001). [39]  


CSF1R Inhibitor

Class Summary

Inhibition of colony stimulating factor-1 receptor (CSF1R), a tyrosine kinase receptor, prevents interleukin-34 activation. Blocking IL-34 and the production of inflammatory mediators by macrophages and monocytes is thought to inhibit tumor cell proliferation.

Pexidartinib (Turalio)

CSF1R inhibitor. It is indicated for adults with tenosynovial giant cell tumor (TGCT) that is associated with severe morbidity or functional limitations and is not amenable to improvement with surgery. A lower dose was approved in October 2022 to reduce risk of adverse effects, including hepatotoxicity. Administer with a low-fat meal (~11-14 g total fat).