Vimseltinib for tenosynovial giant cell tumour
Mené sur 123 patients atteints d'une tumeur ténosynoviale à cellules géantes et non éligibles à une chirurgie, cet essai multicentrique de phase III évalue l'efficacité, du point de vue du taux de réponse objective, et la toxicité du vimseltinib (un inhibiteur de CSF1R)
Tenosynovial giant cell tumour (TGCT) is a rare, locally aggressive soft tissue tumour. TGCT is usually divided according to growth pattern into two main subtypes—localised type and diffuse type—each with different clinical and radiological characteristics. The localised type is defined as a single, well circumscribed nodule, whereas the diffuse type is characterised by multiple, poorly circumscribed, locally aggressive and invasive lesions. The 5-year recurrence-free survival rate in localised type TGCT has been reported to be 86% after primary surgery and 34% after recurrent surgery. 1 For diffuse type TGCT, the 5-year recurrence-free survival rate has been shown to be 64% after primary surgery and 25% after recurrent surgery. 2 In cases of repeated recurrence, functional impairment could be caused by the repeated surgical procedures, 2 , 3 and recurrence is more common after recurrent surgery, 1 , 2 so attempts have been made to control the disease with drugs. Pexidartinib, an inhibitor of the colony-stimulating factor 1 receptor (CSF1R), has been approved for TGCT in the USA, South Korea, and Taiwan, but not in Europe due to concerns about insufficient symptom improvement, durability of response, and liver damage.