Efficacy of imatinib mesylate for the treatment of locally advanced and/or metastatic tenosynovial giant cell tumor/pigmented villonodular synovitis

摘要: BACKGROUND: Pigmented villonodular synovitis (PVNS) (also known as diffuse-type giant cell tumor) and tenosynovial tumors (TGCT) are rare, usually benign neoplasms that affect the synovium tendon sheaths in young adults. These driven by overexpression of colony stimulating factor-1 (CSF1). CSF1 is expressed a minority tumor cells, which, turn attract non-neoplastic inflammatory cells express receptor (CSF1R) through paracrine effect. METHODS: Imatinib mesylate (IM) blocks CSF1R, previous case reports indicated it also exerts antitumor activity PVNS. The authors conducted multi-institutional retrospective study to assess IM patients with locally advanced/metastatic PVNS/TGCT. RESULTS: Twenty-nine from 12 institutions Europe, Australia, United States were included. There 13 men, median age was 41 years, most common site disease knee (n = 17; 59%). Two had metastatic lung and/or bone. Five 27 evaluable Response Evaluation Solid Tumor (RECIST) responses (overall response rate, 19%; 1 complete 4 partial responses), 20 (74%) stable disease. Symptomatic improvement noted 16 22 (73%) who assessable for symptoms. Despite high rate symptomatic favorable safety profile, 6 discontinued because toxicity, decided discontinue no clear medical reason. CONCLUSIONS: displayed interesting PVNS/TGCT, providing proof concept targeting CSF1R this concluded benefits alleviating morbidity localized PVNS/TGCT must be balanced against potential toxicity chronic drug therapy.