Tap WD, Gelderblom H, Palmerini E, et al. - Via a phase 3 randomized trial, researchers assessed pexidartinib, a colony-stimulating factor 1 receptor inhibitor, for safety and efficacy in 174 individuals with tenosynovial giant cell tumor (TGCT) to see if it could be a feasible systemic treatment option, particularly in individuals that are not amenable to surgical resection. At week 25, the amount of individuals who accomplished overall response was greater for pexidartinib vs placebo by RECIST. Serious adverse events were seen in eight of 61 and one of the 59 individuals in the pexidartinib group and in the placebo group, respectively. The most prevalent pexidartinib-associated adverse events were hair color changes, fatigue, elevation in aspartate aminotransferase, nausea, increase in alanine aminotransferase, and dysgeusia. While mixed or cholestatic hepatotoxicity was a recognized risk. pexidartinib was concluded as the first systemic therapy to exhibit a robust tumor response in TGCT with improved individual symptoms and functional outcomes. Moreover, in cases not amenable to improvement with surgery, pexidartinib could be acknowledged as a potential treatment for TGCT correlated with severe morbidity or functional limitations.