Giant cell tumour of bone.
OBJECTIVE
Giant cell tumour of bone (GCT) is the most common benign bone tumour and afflicts a young population. Treatment options for patients with unresectable disease have remained fairly static for the past three decades.


RESULTS
Recent discoveries have identified a key role for the osteoclast differentiation factor, receptor activator of nuclear factor kappa B (NF-kappaB) ligand (RANKL), in the genesis of GCT. The development of the fully human monoclonal antibody to RANKL, denosumab, has led to a clinical trial in unresectable GCT. This study demonstrated an 86% response rate, with comparable evidence of clinical benefit, and was well tolerated. Other pathways that may present targets for therapy include the hypoxia-angiogenesis axis and the colony stimulating factor 1 receptor.


CONCLUSIONS
Denosumab presents a new treatment option for patients with previously untreatable GCT. The eventual role of denosumab and other targeted agents in the treatment of GCT and related disorders is currently the subject of active study.
Thomas
David M
DM
Departments of Haematology and Medical Oncology, Peter MacCallum Cancer Centre, East Melbourne, Victoria, Australia. david.thomas@petermac.org
Skubitz
Keith M
KM
D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D061067:Antibodies, Monoclonal, Humanized; D001859:Bone Neoplasms / Q000473:pathology* / Q000628:therapy*; D000069448:Denosumab; D018212:Giant Cell Tumor of Bone / Q000473:pathology* / Q000628:therapy*; D006801:Humans; D053245:RANK Ligand / Q000037:antagonists & inhibitors / Q000627:therapeutic use
D000911:Antibodies, Monoclonal; D061067:Antibodies, Monoclonal, Humanized; D053245:RANK Ligand; C505688:TNFSF11 protein, human; D000069448:Denosumab