Dr. Abraham on the Clinical Utility of Pexidartinib in TGCT

John A. Abraham, MD, FACS, founder, Orthopaedic Oncology Service, Rothman Orthopaedic Institute, and attending surgeon, Orthopaedic Surgery, Fox Chase Cancer Center, discusses the clinical utility of pexidartinib (Turalio) in tenosynovial giant cell tumor (TGCT).

In August 2019, the FDA approved pexidartinib for the treatment of adult patients with symptomatic TGCT that is associated with severe morbidity or function limitations and is not responsive to improvement with surgery.

Monoclonal antibodies and small molecule inhibitors were evaluated in TGCT prior to the approval of pexidartinib, says Abraham.

The phase 3 ENLIVEN trial, which served as the basis for the regulatory decision, demonstrated a 38% overall response rate, reduction in disease volume, and significant symptom improvement with pexidartinib, Abraham explains.

However, pexidartinib is not without adverse effects, so patients should be selected based on whether the potential benefits associated with the agent outweigh the risks, Abraham says.

Moreover, although surgery remains the mainstay frontline option, pexidartinib should be considered in patients who are not candidates for surgery, have extensive disease, or have undergone multiple surgeries, concludes Abraham.