Long-term efficacy of imatinib mesylate in patients with advanced Tenosynovial Giant Cell Tumor

Verspoor, F. G. M., Mastboom, M. J. L., Hannink, G., Maki, R. G., Wagner, A., Bompas, E., Desai, J., Italiano, A., Seddon, B. M., van der Graaf, W. T. A., Blay, J. Y., Brahmi, M., Eberst, L., Stacchiotti, S., Mir, O., van de Sande, M. A. J., Gelderblom, H., Cassier, P. A. (October 2019) Long-term efficacy of imatinib mesylate in patients with advanced Tenosynovial Giant Cell Tumor. Sci Rep, 9 (1). p. 14551. ISSN 2045-2322

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URL: https://www.ncbi.nlm.nih.gov/pubmed/31601938
DOI: 10.1038/s41598-019-51211-y


Tenosynovial giant cell tumors (TGCT), are rare colony stimulating factor-1(CSF-1)-driven proliferative disorders affecting joints. Diffuse-type TGCT often causes significant morbidity due to local recurrences necessitating multiple surgeries. Imatinib mesylate (IM) blocks the CSF-1 receptor. This study investigated the long term effects of IM in TGCT. We conducted an international multi-institutional retrospective study to assess the activity of IM: data was collected anonymously from individual patients with locally advanced, recurrent or metastatic TGCT. Sixty-two patients from 12 institutions across Europe, Australia and the United States were identified. Four patients with metastatic TGCT progressed rapidly on IM and were excluded for further analyses. Seventeen of 58 evaluable patients achieved complete response (CR) or partial response (PR). One- and five-year progression-free survival rates were 71% and 48%, respectively. Thirty-eight (66%) patients discontinued IM after a median of 7 (range 1-80) months. Reported adverse events in 45 (78%) patients were among other edema (48%) and fatigue (50%), mostly grade 1-2 (89%). Five patients experienced grade 3-4 toxicities. This study confirms, with additional follow-up, the efficacy of IM in TGCT. In responding cases we confirmed prolonged IM activity on TGCT symptoms even after discontinuation, but with high rates of treatment interruption and additional treatments.

Item Type: Paper
Subjects: diseases & disorders > cancer
diseases & disorders
diseases & disorders > neoplasms
diseases & disorders > cancer > drugs and therapies
diseases & disorders > cancer > drugs and therapies > Immunotherapy
diseases & disorders > cancer > metastasis
CSHL Authors:
Communities: CSHL labs > Maki lab
Depositing User: Matthew Dunn
Date: 10 October 2019
Date Deposited: 18 Oct 2019 20:06
Last Modified: 05 Feb 2024 20:50
PMCID: PMC6786996
URI: https://repository.cshl.edu/id/eprint/38608

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