Abstract
Background: Tenosynovial giant cell tumor refers to a group of rarely occurring tumors that are formed in the joints, which are characterized by pain, swelling, and limitation of movement of the joint. Surgery is the main treatment strategy, but the tumor is likely to recur, especially in pigmented villonodular synovitis, which is the diffuse-type giant cell tumor. Pexidartinib was approved in August 2019 by the Food and Drug Administration (FDA) with a brand name TURALIO as the first systemic approved therapy for patients having Tenosynovial Giant Cell Tumors (TGCT).
Objective: In this review, different aspects pertaining to pexidartinib have been summarized, including the pathophysiology of TGCT, chemistry, pharmacokinetics and pharmacodynamics of pexidartinib. Special attention is given to various reported clinical trials of pexidartinib.
Methods: A comprehensive literature search was conducted in the relevant databases to identify studies published in this field during recent years.
Conclusion: Pexidartinib acts by inhibiting the Colony-Stimulating Factor (CSF1)/CSF1 receptor pathway, which leads to the inhibition of the cell lines proliferation and promotes the autophosphorylation process of the ligand-induced CSF1 receptor. Pexidartinib emerged as a potential drug candidate for the treatment of TGCT.
Keywords: Tenosynovial giant cell tumor, CSF, CSF1 receptors, pexidartinib, clinical trials, cell line proliferation.
Graphical Abstract