title: Denosumab treatment for progressive skull base giant cell tumor of bone in a 14 year old female - a case report and literature review.
abstract: 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.
  
  
  METHODS
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Samvel
authors.initials: S
authors.lastname: Bardakhchyan
authors.affiliation: Department of Pediatrics, St. Anna Children's Hospital, Medical University Vienna, Vienna, Austria.|Children's Cancer Research Institute (CCRI), Vienna, Austria.
authors.forename: Leo
authors.initials: L
authors.lastname: Kager
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Samvel
authors.initials: S
authors.lastname: Danielyan
authors.affiliation: Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Armen
authors.initials: A
authors.lastname: Avagyan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Department of Radiotherapy, National Center of Oncology, Yerevan, Armenia.
authors.forename: Nerses
authors.initials: N
authors.lastname: Karamyan
authors.affiliation: Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.|Department of Radiology, Armenian-American Wellness Center, Yerevan, Armenia.
authors.forename: Hovhannes
authors.initials: H
authors.lastname: Vardevanyan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Sergey
authors.initials: S
authors.lastname: Mkhitaryan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Ruzanna
authors.initials: R
authors.lastname: Papyan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Davit
authors.initials: D
authors.lastname: Zohrabyan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Liana
authors.initials: L
authors.lastname: Safaryan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Lilit
authors.initials: L
authors.lastname: Sargsyan
authors.affiliation: Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Lilit
authors.initials: L
authors.lastname: Harutyunyan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Lusine
authors.initials: L
authors.lastname: Hakobyan
authors.affiliation: Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia.
authors.forename: Samvel
authors.initials: S
authors.lastname: Iskanyan
authors.affiliation: Department of Oncology, Yerevan State Medical University, Yerevan, Armenia. gevorgtamamyan@gmail.com.|Yerevan State Medical University, Muratsan Hospital Complex, Clinic of Chemotherapy, Yerevan, Armenia. gevorgtamamyan@gmail.com.|Armenian Pediatric Hematology and Oncology Group, Yerevan, Armenia. gevorgtamamyan@gmail.com.
authors.forename: Gevorg
authors.initials: G
authors.lastname: Tamamyan
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001859:Bone Neoplasms / Q000000981:diagnostic imaging / Q000188:drug therapy*; D000069448:Denosumab / Q000627:therapeutic use*; D005260:Female; D018212:Giant Cell Tumor of Bone / Q000000981:diagnostic imaging / Q000188:drug therapy*; D006801:Humans; D009361:Neoplasm Invasiveness; D009367:Neoplasm Staging; D011788:Quality of Life; D019291:Skull Base / Q000000981:diagnostic imaging / Q000187:drug effects; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D000069448:Denosumab
keywords: Denosumab; Giant cell tumor of bone; Skull base