The use of denosumab in rare bone diseases in adults: a systematic review from the ECTS Rare Bone Disease Action Group
Authors' conclusion
Denosumab is a feasible treatment in adults with RBDs when managed by multidisciplinary teams with knowledge of both the underlying disease and potential surgeries as well as the medical site of treatment. Denosumab discontinuation management is paramount to prevent recurrence and severe complications. The paucity of data supports the need for data collection through rare disease registries for future pertinent evidence-based recommendations.
Study context
- Population
- Adults with rare bone diseases (RBDs)
- Outcome
- Effects of denosumab in adults with RBDs
Methodological detailsTargeted evidence search for studies reporting denosumab use in RBDs in adults
- Targeted evidence search for studies reporting denosumab use in RBDs in adults
Pooled result
As reported in the abstract
- Denosumab may lead to pain reduction, lesion reduction, or bone formation
- Dosage, schedules and duration varied; 120 mg monthly or every 3 months for almost 1 year reached the desired effect in most patients
Safety
- Generally well tolerated; mild common side effects such as (asymptomatic) hypocalcemia and hypophosphatemia
- Serious adverse effects such as osteonecrosis of the jaw or atypical femoral fractures rarely reported
- Main concern: rebound effect after discontinuation, with disease recurrence in some cases
Original publication
The use of denosumab in rare bone diseases in adults: a systematic review from the ECTS Rare Bone Disease Action Group. J Clin Endocrinol Metab. Published 7 April 2026. doi:10.
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