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

被引：207

作者：

Cassier, Philippe A. ^{[1
]}

Gelderblom, Hans ^{[2
]}

Stacchiotti, Silvia ^{[3
]}

Thomas, David ^{[4
]}

Maki, Robert G. ^{[5
]}

Kroep, Judith R. ^{[2
]}

van der Graaf, Winette T. ^{[6
]}

Italiano, Antoine ^{[7
]}

Seddon, Beatrice ^{[8
]}

Domont, Julien ^{[9
]}

Bompas, Emanuelle ^{[10
]}

Wagner, Andrew J. ^{[11
]}

Blay, Jean-Yves ^{[1
,12
]}

机构：

[1] Ctr Leon Berard, Dept Med, F-69008 Lyon, France

[2] Leiden Univ, Med Ctr, Dept Med Oncol, Leiden, Netherlands

[3] Natl Canc Inst, Dept Canc Med, I-20133 Milan, Italy

[4] Peter MacCallum Canc Ctr, Dept Haematol & Med Oncol, Melbourne, Vic, Australia

[5] Mem Sloan Kettering Canc Ctr, Sarcoma Program, New York, NY 10021 USA

[6] Radboud Univ Nijmegen, Med Ctr, Dept Med Oncol, NL-6525 ED Nijmegen, Netherlands

[7] Bergonie Inst, Dept Med Oncol, Bordeaux, France

[8] Univ Coll London Hosp, Dept Oncol, London, England

[9] Inst Gustave Roussy, Dept Med, Villejuif, France

[10] Rene Gauducheau Canc Ctr, Dept Med Oncol, Nantes, France

[11] Dana Farber Canc Inst, Ctr Sarcoma & Bone Oncol, Boston, MA 02115 USA

[12] Univ Lyon, Villeurbanne, France

来源：

CANCER | 2012年 / 118卷 / 06期

关键词：

tenosynovial giant cell tumor; pigmented villonodular synovitis; drug therapy; imatinib; colony-stimulating factor 1; colony-stimulating factor 1 receptor; GASTROINTESTINAL STROMAL TUMORS; TRANSLOCATION; EXPRESSION; RECEPTOR; DIFFUSE; CSF1; FMS;

D O I：

10.1002/cncr.26409

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 [肿瘤学];

摘要：

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. Cancer 2012; 118: 1649-55. VC 2011 American Cancer Society.

引用

页码：1649 / 1655

页数：7

共 18 条

[1]

DIFFUSE AND LOCALIZED TENOSYNOVIAL GIANT-CELL TUMOR AND PIGMENTED VILLONODULAR SYNOVITIS - A CLINICOPATHOLOGICAL AND FLOW CYTOMETRIC DNA ANALYSIS [J].