Joint issues, which can be experienced in the form of pain, stiffness, swelling, clicking, numbness and more, can be very debilitating and have a significant impact on daily life. As joint pain and other issues can be caused by several conditions, some people may live with their symptoms for years and see multiple doctors trying to find the root cause. But unresolved joint problems could be caused by a rare condition called tenosynovial giant cell tumor, or TGCT.
TGCT, also known as pigmented villondular synovitis (PVNS) or giant cell tumor of the tendon sheath (GCT-TS), is a rare type of tumor that is typically non-malignant and occurs in or around a joint, reducing range of motion and damaging neighboring tissues. It can affect people at any age but is most often seen in those between the ages of 30 and 50 years.
TGCT symptoms such as pain, swelling and stiffness of the involved joint can come on slowly. Unfortunately, because of the rarity of TGCT, a definitive diagnosis may take several years and visits to a variety of healthcare providers.
There are two forms of TGCT — localized and diffuse. Localized TGCT is most often found in smaller joints, such as those around the hands and feet, and makes up the majority of cases. This form can usually be treated effectively with surgery.
Diffuse TGCT most commonly occurs in large joints, such as the knee, ankle and hip and may be more difficult to remove with surgery. In the U.S., the annual incidence of new diffuse cases was estimated to be about 1,300 in 2019.
“For many years, people who couldn’t be treated successfully with surgery had to cope with living with TGCT because there were no approved systemic treatments and only a few experts truly understood the ramifications of the disease,” said Howard Rutman, MD, vice president of Medical Affairs at Daiichi Sankyo, Inc. “Following the approval of Turalio, a prescription medicine used to treat certain patients with TGCT, it is critical that we continue to increase awareness and have patients see the appropriate specialists so we can help those patients.”
In August 2019, Turalio (pexidartinib) became the first and only treatment approved by the U.S. Food and Drug Administration for adult patients with symptomatic TGCT associated with severe morbidity or functional limitations and not amenable to improvement with surgery.
Turalio can cause serious side effects, including serious liver problems, which may be severe and can lead to death. The most common side effects of Turalio include changes in blood liver tests; hair color changes; tiredness; increased cholesterol level in the blood; decreased white blood cells and red blood cells; swelling in and around your eyes; rash; and loss of taste or changes in the way things taste. Turalio can cause harm to an unborn baby when administered to a pregnant woman.
Because of the risk of serious liver problems, Turalio is available only through a restricted program called the Turalio REMS program.
If you experience signs of TGCT, contact your doctor, who may be able to refer you to a specialized center where physicians are more familiar with the disease. All members of a multidisciplinary healthcare team at a specialized center are highly trained to recognize the signs and symptoms of rare diseases like TGCT.
Contact your doctor for more information about side effects of the medication.