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

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 s...

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Bibliographic Details
Published in:Scientific reports 2019-10, Vol.9 (1), p.14551-8, Article 14551
Main Authors: 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
Format: Article
Language:English
Subjects:
Adult
Antineoplastic Agents - therapeutic use
Australia
Colony-stimulating factor
Disease Progression
Disease-Free Survival
Edema
Europe
Female
Giant Cell Tumor of Tendon Sheath - drug therapy
Humans
Imatinib
Imatinib Mesylate - therapeutic use
Long-term effects
Macrophage colony-stimulating factor
Metastases
Metastasis
Middle Aged
Morbidity
Neoplasm Metastasis
Receptors, Colony-Stimulating Factor - antagonists & inhibitors
Retrospective Studies
Targeted cancer therapy
Treatment Outcome
Tumors
United States
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Summary: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.
ISSN:2045-2322
2045-2322
DOI:10.1038/s41598-019-51211-y