Howard Rutman, MD, vice president of medical affairs at Daiichi Sankyo, Inc. talks about the two most common treatment options for tenosynovial giant cell tumors (TGCT) – surgery or systemic treatment with pexidartinib (Turalio).
TGCT are a group of rare, benign tumors that involve the synovium, bursae and tendon sheath. Although these tumors are benign, they can grow and cause extreme damage to surrounding areas. Symptoms can include pain, swelling, and limitation of movement of the joint.
Pexidartinib is the first systemic therapy approved for TCGT patients. The approval of pexidartinib was based on a durable overall response rate (ORR) seen in a clinical trial for TCGT patients who could not receive surgical treatment – the typical treatment option for TCGT. After 25 weeks of pexidartinib treatment, ORR was 38% with a 15% complete response rate and a 23% partial response rate. None of the patients receiving placebo in the trial had a response.
Pexidartinib comes with a Boxed Warning for a potentially fatal liver injury and the drug is only available through a Risk Evaluation and Mitigation (REM) Strategy Program, as confirmed by Dr. Rutman.
For more information about TGCT, visit https://checkrare.com/tenosynovial-giant-cell-tumor-tgct/