Journal Article

Denosumab treatment for progressive skull base giant cell tumor of bone in a 14 year old female – a case report and literature review

Kager, Leo, Vardevanyan, Hovhannes, Karamyan, Nerses, Avagyan, Armen, Bardakhchyan, Samvel, Harutyunyan, Lilit, Tamamyan, Gevorg, Papyan, Ruzanna, Zohrabyan, Davit, Mkhitaryan, Sergey, Iskanyan, Samvel, Danielyan, Samvel, Safaryan, Liana, Sargsyan, Lilit, Hakobyan, Lusine

2017

Overview

Background Giant cell tumor of bone (GCT) is a rare primary bone tumor, which can metastasize and undergo malignant transformation. The standard treatment of GCT is surgery. In patients with unresectable or metastatic disease, additional therapeutic options are available. These include blocking of the receptor activator of NF-kappa B ligand (RANKL) signaling pathway, which plays a role in the pathogenesis of GCT of bone, via the anti-RANKL monoclonal antibody denosumab. Case Presentation Herein we report on a female teenager who presented in a very poor clinical condition (cachexia, diplopia, strabismus, dysphonia with palsy of cranial nerves V, VI, VIII, IX, X, XI and XII) due to progressive disease, after incomplete resection and adjuvant radiotherapy, of a GCT which affected the cervical spine (C1 and C2) as well as the skull base; and who had an impressive clinical response to denosumab therapy. To the best of our knowledge, this is the youngest patient ever reported with a skull base tumor treated with denosumab. Conclusion In situations when surgery can be postponed and local aggressiveness of the tumor does not urge for acute surgical intervention, upfront use of denosumab in order to reduce the tumor size might be considered. Principally, the goal of denosumab therapy is to reduce tumor size as much as possible, with the ultimate goal to make local surgery (or as in our case re-surgery) amenable. However, improvement in quality of life, as demonstrated in our patient, is also an important aspect of such targeted therapies.

• Publisher: BioMed Central,Springer Nature B.V,BMC
• Subject: Adolescent / Antineoplastic Agents - therapeutic use / Bone cancer / Bone Neoplasms - diagnostic imaging / Bone Neoplasms - drug therapy / Bones / Case Report / Case reports / Denosumab / Denosumab - therapeutic use / Dysphagia / Endoscopy / Female / Giant cell tumor of bone / Giant Cell Tumor of Bone - diagnostic imaging / Giant Cell Tumor of Bone - drug therapy / Humans / Immunotherapy / Ligands / Maternal and Child Health / Medicine / Medicine & Public Health / Monoclonal antibodies / Neoplasm Invasiveness / Neoplasm Staging / NMR / Nuclear magnetic resonance / Ostomy / Pain / Patients / Pediatrics / Quality of Life / Radiation therapy / Skull base / Skull Base - diagnostic imaging / Skull Base - drug effects / Surgery / Treatment Outcome / Tumors / Vertebrae