Pexidartinib, the newly approved agent to treat patients with tenosynovial giant cell tumor (TGCT), received a category 1 recommendation from the National Comprehensive Cancer Network (NCCN) in the recent update of its Clinical Practice Guidelines in Oncology, Soft Tissue Sarcoma (Version 4.2019). A category 1 recommendation is based on a high level of evidence with uniform consensus that the intervention is appropriate.
The NCCN based its recommendation on the randomized, placebo-controlled phase 3 ENLIVEN study (NCT02371369) published in The Lancet (Tap WD, Gelderblom H, Palmerini E, et al. Lancet. 2019;394:478-487). The placebo-controlled portion of the study showed that patients treated with pexidartinib achieved a significantly higher overall response than patients in the placebo arm, 39% compared to none, respectively. The investigators identified mixed or cholestatic hepatotoxicity to be a risk of systemic therapy with the agent. Nevertheless, the “robust tumour response,” they wrote, “with improved patient symptoms and functional outcomes” establish pexidartinib as a potential treatment for TGCT in cases not amenable to improvement with surgery.