Prolia (denosumab)

This study confirms literature data about the use of denosumab in inoperable GCRTB. These results are preliminary; further studies are necessary on a larger series of cases, with a longer follow-up (3-5 years), with data collection even from other pediatric centers.

P4, N=40, Recruiting, Aarhus University Hospital

Denosumab facilitates the infiltration of CD8+ T cells into the interior of LC-BoM tissues. Based on this mechanism, we observed in both clinical cohorts and preclinical models that RANKL inhibitor can enhance the efficacy of immunotherapy in treating LC-BoM.

Management included repeat resection and denosumab therapy, achieving radiographic stability with preservation of vision in the right eye, although the optic nerve of the left eye remained atrophic. Given the diagnostic overlap between GCTB and ABC, the authors emphasize the importance of genetic testing for accurate and early diagnosis to enable timely treatment and reduce the risk of recurrence.

Patients with abnormal renal function, BRCA1 mutations, or those at high risk of developing skeletal-related events may benefit from denosumab over zoledronic acid. During denosumab treatment in patients with renal dysfunction, attention should be paid to the occurrence of hypocalcemia.

Systemic agents such as denosumab or bisphosphonates remain investigational, and radiotherapy is generally contraindicated due to malignant transformation risk...Awareness of risk factors, early imaging-based detection, and complete surgical excision are critical for optimal outcomes. Further multicenter studies are required to define surveillance protocols, validate molecular predictors, and clarify the role of systemic therapy in this challenging condition.

P4, N=100, Completed, Xi'an Honghui Hospital | Recruiting --> Completed | Trial primary completion date: Dec 2024 --> Jul 2025

Anti-osteoporosis medications (bisphosphonates, denosumab, and teriparatide) and cytoreductive agents (interferon, chemotherapeutic agents, or tyrosine kinase inhibitors) are also used. Although controlling the underlying disease is usually most effective, the benefits and risks of each therapeutic approach should be balanced if needed.

P4, N=44, Completed, National Taiwan University Hospital | Recruiting --> Completed

Among these candidate osteolytic markers, four (two COL1A1-specific products) showed decreases from baseline (p < .05) in patients on denosumab (n = 10 patients)...The range of collagen peptide fragments we discovered as a direct result of osteoclast activity indicates a complexity of bone resorption pathways not previously known, extending beyond the known proteolytic cleavage events in bone collagen proteins. Monitoring biofluid concentrations of these novel bone markers has the potential to capture multiple pathways of bone resorption activity beyond the existing assays based on cathepsin K.

One year after surgery, metastases were detected in the left axillary lymph nodes and femoral head, requiring surgical intervention and denosumab treatment...The TP53 variant is listed in the Catalogue of Somatic Variants in Cancer as a breast cancer-associated variant, whereas a splice site variant in SMARCB1 and LOH suggests a loss of SMARCB1 function. This case highlights the increased risk of malignancy in NF1 patients and underscores the need for comprehensive genomic profiling.

P=N/A, N=324, Not yet recruiting, The First Affiliated Hospital of Army Medical University​; The First Affiliated Hospital of Army Medical University