Pexidartinib: first approved systemic therapy for patients with tenosynovial giant cell tumor.
André B P van KuilenburgMichiel van de SandePublished in: Future oncology (London, England) (2020)
Pexidartinib is an orally administered small molecule tyrosine kinase inhibitor. Phase III ENLIVEN study results provided clinical evidence for US FDA approval for treatment of adult patients with symptomatic tenosynovial giant cell tumor associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Recommended dosage is 400 mg orally twice daily on an empty stomach. Long-term follow-up in pooled analyses showed increased response rates compared with those observed in ENLIVEN. Patients on pexidartinib also experience meaningful improvements in range of motion. Side effects associated with pexidartinib are generally manageable; however, there is a risk of potentially life-threatening mixed or cholestatic hepatotoxicity and pexidartinib has a Risk Evaluation and Mitigation Strategy (REMS) program to ensure appropriate monitoring.
Keyphrases
- giant cell
- phase iii
- small molecule
- open label
- end stage renal disease
- clinical trial
- ejection fraction
- newly diagnosed
- climate change
- physical activity
- randomized controlled trial
- drug administration
- early onset
- coronary artery bypass
- patient reported outcomes
- double blind
- phase ii
- coronary artery disease
- protein protein
- patient reported
- replacement therapy
- smoking cessation