Pimicotinib: a new agent for an orphan disease
Mené sur 94 patients atteints d'une tumeur ténosynoviale à cellules géantes, cet essai international de phase III évalue l'efficacité, du point de vue du taux de réponse objective en semaine 25, et la toxicité du pimicotinib (un inhibiteur du récepteur du facteur de stimulation des colonies 1)
Tenosynovial giant cell tumour (TGCT, formerly known as pigmented villonodular synovitis) is a rare, locally aggressive, connective tissue neoplasm affecting mostly young adults.1–4 This neoplasm is driven by the overexpression of colony-stimulating factor 1 (CSF-1), resulting from fusion genes or transcriptional deregulation, which recruit CSF-1 receptor (CSF-1R)-positive macrophages in the tumour.1 Symptoms include joint swelling, pain, functional impairment, and eventually joint destruction, with a major effect on the quality of life and work capacity of these patients.
The Lancet , commentaire, 2026