Advances in treatment for tenosynovial giant cell tumors. (2nd December 2018)

• Record Type: Journal Article
• Authors: Palmerini, Emanuela; Longhi, Alessandra; Donati, Davide; Staals, Eric L.
• Is Part Of: Expert opinion on orphan drugs. Volume 6:Number 12(2018)
• Page Start: 753
• Page End: 757
• Publication Date: 2018-12-02
• Subjects: Pigmented villonodular synovitis -- PVNS -- tenosynovial giant cell tumor -- TGCT -- treatment -- CSF1R -- colony-stimulating factor 1 (CSF1) -- surgery; Orphan drugs -- Periodicals; Rare diseases -- Periodicals; Chemotherapy -- Periodicals; 615.1
• DOI: 10.1080/21678707.2018.1549481
• Languages: English
• ISSNs: 2167-8707

Abstract

ABSTRACT: Introduction : Tenosynovial giant cell tumor (TGCT) is a benign clonal neoplastic proliferation arising from the synovium often causing pain, swelling, joint stiffness, and reduced quality of life. The optimal treatment strategy in patients with diffuse-type TGCT (dt-TGCT) is unclear. The purpose of this review is to describe recent advances in the knowledge of TGCT pathogenesis and potential therapeutic implications. Areas covered : Current treatment options for TGCT are discussed, including surgery and radiotherapy. Recent evidence that TGCT cells overexpress colony-stimulating factor 1 (CSF1), resulting in recruitment of CSF1 receptor (CSF1R) positive tumor-associated macrophage and contributing to tumor growth, has created new opportunities for systemic treatments of dt-TGCT. Results of clinical trials with CSF1R inhibitors are now available. These inhibitors include small molecules such as imatinib, nilotinib, pexidartinib, and monoclonal antibodies like emactuzumab, MCS110, and cabiralzumab. Expert opinion : TGCT impairs patients' quality of life significantly. The confirmation that the pathogenetic loop of TGCT can be inhibited through targeted agents could potentially change the therapeutic armamentarium for this condition.