Prospective Observational Non-interventional Study to Describe Characteristics and Management of Patients With Giant Cell Tumor of Bone Treated With XGEVA and Its Use in Routine Clinical Practice in France (20150360)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

  Effectiveness study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Name of medicine:
  XGEVA
• Study drug International non-proprietary name (INN) or common name:
  DENOSUMAB
• Anatomical Therapeutic Chemical (ATC) code:
  (M05BX04) denosumab

  denosumab
• Medical condition to be studied:
  Desmoid tumour

Population studied

• Age groups:
  • Paediatric Population (< 18 years)

    • Adolescents (12 to < 18 years)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
      • Adults (75 to < 85 years)
      • Adults (85 years and over)
• Estimated number of subjects:
  80

Study design details

• Main study objective:
  To describe patient and disease characteristics of giant cell tumor of bone (GCTB) patients prior to initiation of XGEVA
• Outcomes:
  Socio-demographics (age, sex, living condition, education level, employment status), clinical (localization, soft tissue extension, fracture, pulmonary metastasis, for recurrent tumor: number, localization, biopsy, MRI scanner, soft tissue extension, fracture, pulmonary metastasis) and radiological (tumor size) disease characteristics prior initiation of XGEVA, Treatment history and line of treatment at XGEVA initiation. ; Proportion of patients undergoing surgery within one year from XGEVA initiation. ; Treatment pattern of XGEVA and other treatments targeting GCTB administered over the study period. TTP, PFS, RFS. Pain score and impact on patient’s lives. ; Time from BPI-SF score prior XGEVA initiation to the first BPI-SF score deterioration. ADRs and SADRs.
• Data analysis plan:
  All analyses will be done using modified intent to treat principle: all included subjects who have received at least one dose of XGEVA treatment (mITT1). ; Categorical outcomes will be summarized by the number and percentage of patients in each category, and the corresponding 95% CI. ; Continuous data will be described by mean, standard deviation, median, Q1 and Q3 quartiles and minimum and maximum values. ; Time to event endpoints will be analyzed using Kaplan Meier estimation and described using median or rate at specific time point with a 95% CI. Follow-up will be calculated using reverse Kaplan Meier estimation. ; All BPI SF analyses will be performed in all included subjects who have received at least one dose of XGEVA and with a BPI SF score at baseline (mITT2). ; Longitudinal BPI-SF score will be studied using time to event approach and also using mixed model analysis of variance for repeated measurement or pattern mixture models (in case of missing not at random BPI SF score).