Tomek Szczudlo, VP, Global Medical Affairs, Franchise Head, Solid Tumors at Daiichi Sankyo, discusses the tenosynovial giant cell tumors (TGCT) Observational Platform Project (TOPP) registry, data from which was presented at the Connective Tissue Oncology Society 2021 Annual Meeting.
TGCT are a group of rare, benign tumors that involve the synovium, bursae and tendon sheath. While these tumors are benign, they can grow and cause extreme damage to the surrounding tissues. Joint pain, swelling, and decreased range in motion are all common symptoms of TGCT but these may be overlooked as being caused by more common conditions. The typical treatment for TGCT is surgical removal of the tumor but other options, such as the FDA-approved CSF1R inhibitor, pexidartinib, can be effective.
As Szczudlo explains, the TOPP registry was a multinational, multicenter, prospective observational study involving 12 tertiary sarcoma centers in 7 European countries, as well as 2 US sites. This study enrolled for 2 years adult patients with diagnosed primary or recurrent cases of diffuse-type TGCT. At baseline, patient demographic and clinical characteristics were collected.
At the Connective Tissue Oncology Society annual meeting (CTOS 2021), two presentations were given based on data from the TOPP registry (Abstract #1818870 and Abstract #1077220). As Szczudlo explains, at baseline 79/176 patients were off-treatment without a plan to receive medical therapy or surgery while 45 patients being treated or planning to be treated with medical therapy only, and 39 patients had surgery or were planning to have surgery only.
Patient reported outcomes were measured. In general, pain scores and other patient reported outcomes were worse at years 1 and 2 in patients who had to change their treatment strategy. Another important finding was that patients who remained off-treatment or who received systemic treatment only from year 1 to year 2 had, on average, higher quality of life scores than those who received surgery in year 2. Mr. Szczudlo emphasizes that this data demonstrates the benefit of keeping TGCT patients on the same treatment course as well as involving multiple specialists in the management of the disease. He also notes that the general stability in patient reported outcomes from baseline to year 2 when patients remained on the same treatment further demonstrates the chronic nature of TGCT symptoms.
To learn more about TGCT, visit our TGCT Learning Center here.
