Safety and Efficacy of Moderate-dose Denosumab in Fibrous Dysplasia: Observational Results From a Phase 2 Clinical Trial
Authors' conclusion
Moderate-dose denosumab may provide clinical benefits comparable to the high-dose regimen in adults with FD while potentially lowering associated risks. However, discrepancies in the duration of efficacy are an important potential safety concern.
Study context
- Population
- Adults with fibrous dysplasia (FD), clinical research center8 high-dose
- Participants
- 6 vs 8
- Exposure
- Moderate-dose denosumab vs high-dose denosumab
- Moderate-dosen = 6
- High-dosen = 8reference
- Outcome
- Bone turnover markers, 18F-NaF PET/CT, lesion biopsies
Methodological detailssingle-centre, protocol amendment, Protocol amended to restart moderate-dose denosumab if clinically indicated after 8-month posttreatment observation
- Single-centre
- Protocol amendment
- Protocol amended to restart moderate-dose denosumab if clinically indicated after 8-month posttreatment observation
Main finding
As reported in the abstract
| Group | P1NP change | CTX change |
|---|---|---|
| Moderate-dose | −82% | −86% |
| High-dose | −91% | −86% |
P1NP change
Moderate-dose−82%
High-dose−91%
CTX change
Moderate-dose−86%
High-dose−86%
Observational data: associations, not effects.
Further findings
Lesion histology (sequential biopsies)
- Progressive lesional mineralization and reduced cellularity with moderate dose in 1 subject
BTM course on moderate dose
- Sustained decline in BTM
- 4 subjects
Safety
Rebound between doses on moderate dose
- Severely affected subjects with rebound between doses
- 2
- Recurrent hypercalcemia
- 1 subject
Original publication
Safety and Efficacy of Moderate-dose Denosumab in Fibrous Dysplasia: Observational Results From a Phase 2 Clinical Trial. J Clin Endocrinol Metab. Published 13 December 2024. doi:10.
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