Tenosynovial giant cell tumor (TGCT) is a potentially aggressive and debilitating disease characterized by overexpression of colony-stimulating factor-1 (CSF-1) in neoplastic synovial cells. Although rare, TGCT is nevertheless more common than previously appreciated, and the prevalence is relatively high given that many patients are young when the disease is diagnosed and will live with the disease for decades. Patients face a long and often difficult journey marked by multiple surgeries and surgical morbidity, decreases in physical function, and negative effects on quality of life and work productivity.
There is a high unmet need for effective approaches to treating TGCT beyond surgery. The development of next-generation, potent inhibitors of the CSF-1/CSF-1 receptor axis have demonstrated dramatic and durable responses; in a randomized, phase 3 clinical trial, pexidartinib demonstrated a robust tumor response in patients with advanced TGCT, leading to the first-ever US Food & Drug Administration (FDA) approval of a treatment for this condition.
This activity will review clinical advances and emerging research in TGCT, including diagnosis, treatment approaches, newly FDA-approved therapy, recent clinical trial data, and the relevance and integration of these new data into current practice.