Excellent Results in the Off-Label Use of Denosumab in Pediatric Oncology: Monocentric Case Series Study
Abstract
Denosumab is a human monoclonal antibody approved by the Food and Drug Administration in 2013 for use in adults with inoperable giant cell tumor of bone (GCTB). In children, it is used as an off‐label drug in some giant cell‐rich tumors of bone (GCRTB) and giant cell granuloma (GCG). The aim of the study is to evaluate the efficacy and acute toxicity of denosumab in children. From February 2022 to December 2023, at the Istituto Giannina Gaslini, 5 patients were treated with denosumab, 2 females and 3 males. Age ranged from 8 to 17 years. Two had ABC (hemipelvis and D9), and 3 had GCG (sphenoid, jaw, and maxilla). An assay of calcium and vitamin D as well as a dental scan was performed before administering denosumab due to possible side effects such as hypocalcemia and jaw osteonecrosis. Therapy was given subcutaneously at the dose of 70 mg/m2 (weight 50 kg) at days +1, +8, +15, +28, and then monthly for 3–5 months, followed by imaging evaluation. Overall, 21 doses were administered to Patient 1, 20 doses to Patient 2, 11 doses to Patient 3, 10 doses to Patient 4, and 9 doses to Patient 5. Calcium carbonate and vitamin D were given as supportive therapy. We observed lesion volume reduction in 4 patients and radiological stability in 1 patient. Surgery was possible in 1 case thanks to the significant reduction of lesion size. A longer administration over 22 months was safe and well tolerated. No disease progression or side effects were observed. This study confirms literature…
How strong is the evidence?
Evidence Hub reads this as a case series, done in people. Its evidence level is 5 of 5 (Very low · case / narrative / animal / in vitro / in silico). Case reports, narrative reviews, and animal, cell or computer-model studies give the weakest evidence for human health.
Read the paper
Related studies
- Effectiveness of Bisphosphonates and Denosumab on Risk of Vertebral Fractures in Prostate Cancer Patients Under Androgen Deprivation Therapies: A Real-World Prospective Study
- Osteoporosis-related fractures after spinal cord injury: a retrospective study from Brazil
- Serum 25-hydroxyvitamin D, parathyroid hormone, and mortality in older men
- Lack of Association between Vitamin D Insufficiency and Cardiovascular or Fracture Risk: A UK Biobank Study
- Muscle strength and body composition are clinical indicators of osteoporosis
- Incidence of bone protection and associated fragility injuries in patients with proximal femur fractures
Go deeper in Evidence Hub
Evidence Hub adds a study data sheet (design, sample size, evidence level, key numbers), an AI summary and a chat grounded in the paper's text.