Cassier, P. A., Gelderblom, H., Stacchiotti, S., Thomas, D., Maki, R. G., Kroep, J. R., van der Graaf, W. T., Italiano, A., Seddon, B., Domont, J., Bompas, E., Wagner, A. J., Blay, J. Y. (March 2012) Efficacy of imatinib mesylate for the treatment of locally advanced and/or metastatic tenosynovial giant cell tumor/pigmented villonodular synovitis. Cancer, 118 (6). pp. 1649-55. ISSN 1097-0142 (Electronic)0008-543X (Linking)
Abstract
BACKGROUND: Pigmented villonodular synovitis (PVNS) (also known as diffuse-type giant cell tumor) and tenosynovial giant cell tumors (TGCT) are rare, usually benign neoplasms that affect the synovium and tendon sheaths in young adults. These tumors are driven by the overexpression of colony stimulating factor-1 (CSF1). CSF1 is expressed by a minority of tumor cells, which, in turn attract non-neoplastic inflammatory cells that express CSF1 receptor (CSF1R) through a paracrine effect. METHODS: Imatinib mesylate (IM) blocks CSF1R, and previous case reports indicated that it also exerts antitumor activity in PVNS. The authors conducted a multi-institutional retrospective study to assess the activity of IM in patients with locally advanced/metastatic PVNS/TGCT. RESULTS: Twenty-nine patients from 12 institutions in Europe, Australia, and the United States were included. There were 13 men, the median age was 41 years, and the most common site of disease was the knee (n = 17; 59%). Two patients had metastatic disease to the lung and/or bone. Five of 27 evaluable patients had Response Evaluation in Solid Tumor (RECIST) responses (overall response rate, 19%; 1 complete response and 4 partial responses), and 20 of 27 patients (74%) had stable disease. Symptomatic improvement was noted in 16 of 22 patients (73%) who were assessable for symptoms. Despite a high rate of symptomatic improvement and a favorable safety profile, 6 patients discontinued because of toxicity, and 4 patients decided to discontinue IM for no clear medical reason. CONCLUSIONS: IM displayed interesting activity in patients with PVNS/TGCT, providing proof of concept for targeting CSF1R in this disease. The authors concluded that the benefits of alleviating morbidity in patients with localized PVNS/TGCT must be balanced against the potential toxicity of chronic drug therapy.
| Item Type: | Paper |
|---|---|
| Uncontrolled Keywords: | Adult Aged Antineoplastic Agents/*therapeutic use Benzamides Female Fluorodeoxyglucose F18 Giant Cell Tumors/*drug therapy/pathology Humans Imatinib Mesylate Male Middle Aged Neoplasm Metastasis Piperazines/adverse effects/pharmacology/*therapeutic use Pyrimidines/adverse effects/pharmacology/*therapeutic use Receptor, Macrophage Colony-Stimulating Factor/antagonists & inhibitors Retrospective Studies Synovitis, Pigmented Villonodular/*drug therapy |
| Subjects: | diseases & disorders > cancer diseases & disorders > cancer > drugs and therapies diseases & disorders > cancer > prognosis |
| CSHL Authors: | |
| Communities: | CSHL labs > Maki lab |
| Depositing User: | Matt Covey |
| Date: | 15 March 2012 |
| Date Deposited: | 21 Oct 2016 16:43 |
| Last Modified: | 21 Oct 2016 16:43 |
| Related URLs: | |
| URI: | https://repository.cshl.edu/id/eprint/33728 |
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