Pexidartinib for the treatment of adult patients with symptomatic tenosynovial giant cell tumor: safety and efficacy.
Emanuela PalmeriniAlessandra LonghiDavide Maria DonatiEric Lodewijk StaalsPublished in: Expert review of anticancer therapy (2020)
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. 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.
Keyphrases
- giant cell
- phase iii
- clinical trial
- open label
- end stage renal disease
- phase ii
- newly diagnosed
- ejection fraction
- chronic kidney disease
- liver injury
- climate change
- prognostic factors
- double blind
- placebo controlled
- radiation therapy
- quality improvement
- tyrosine kinase
- squamous cell carcinoma
- randomized controlled trial
- case control
- patient reported
- smoking cessation
- combination therapy
- clinical evaluation