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Ref Type Journal Article
PMID (25617146)
Authors Lopez-Pousa A, Martin Broto J, Garrido T, Vazquez J
Title Giant cell tumour of bone: new treatments in development.
Journal Clinical & translational oncology : official publication of the Federation of
Vol 17
Issue 6
Date 2015 Jun
URL
Abstract Text Giant cell tumour of bone (GCTB) is a benign osteolytic tumour with three main cellular components: multinucleated osteoclast-like giant cells, mononuclear spindle-like stromal cells (the main neoplastic components) and mononuclear cells of the monocyte/macrophage lineage. The giant cells overexpress a key mediator in osteoclastogenesis: the RANK receptor, which is stimulated in turn by the cytokine RANKL, which is secreted by the stromal cells. The RANK/RANKL interaction is predominantly responsible for the extensive bone resorption by the tumour. Historically, standard treatment was substantial surgical resection, with or without adjuvant therapy, with recurrence rates of 20-56 %. Studies with denosumab, a monoclonal antibody that specifically binds to RANKL, resulted in dramatic treatment responses, which led to its approval by the United States Food and Drugs Administration (US FDA). Recent advances in the understanding of GCTB pathogenesis are essential to develop new treatments for this locally destructive primary bone tumour.

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
Unknown unknown bone giant cell tumor not applicable Denosumab FDA approved Actionable In a Phase II clinical trial that supported FDA approval, Xgeva (denosumab) treatment resulted in an overall objective response rate of 25% (47/187) in patients with giant cell bone tumor, with a median duration of response of 20 months among patients demonstrating response (PMID: 25617146). detail... 25617146