Pexidartinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor: safety and efficacy.

This article reviews the pharmacological properties, clinical efficacy, and safety of pexidartinib.Expert opinion: Pexidartinib was effective with an acceptable safety profile for advanced TGCT in phase I - III studies. The phase III trial (ENLIVEN) in unresectable TGCT met its primary endpoints of overall response - rate (ORR). These results led to FDA approval for this TGCT population. Mixed or cholestatic hepatotoxicity was observed in rare cases. For this reason, pexidartinib is currently available only through a Risk Evaluation and Mitigation Strategy (REMS) Program in the USA.TGCT significantly impairs patients' quality of life. The approval of pexidartinib has changed the therapeutic armamentarium for this condition. However, strict monitoring of liver function is warranted. PMID: 32297819 [PubMed - as supplied by publisher]
Source: Expert Review of Anticancer Therapy - Category: Cancer & Oncology Tags: Expert Rev Anticancer Ther Source Type: research