Randomised trial of genetic testing and targeted intervention to prevent the development and progression of Paget’s disease of bone
Primary result · New bone lesionsResult — go to the resultOdds ratio 0.41 95% CI 0.00–3.43Authors' conclusion
Genetic testing for pathogenic SQSTM1 variants coupled with intervention with ZA is well tolerated and has favourable effects on the progression of early PDB.
Study context
- Population
- Individuals at increased risk of paget's disease of bone (PDB) because of pathogenic SQSTM1 variants180 (81%) completed
- Participants
- 222 individuals
- Comparison
- Zoledronic acid 5 mg vs placebo
- Primary endpoint
- New bone lesions assessed by radionuclide bone scan
Methodological detailsplacebo-controlled, Median follow-up 84 months (range 0-127), PDB lesions at baseline: 9 (8.1%) zoledronic acid (ZA) vs 12 (10.8%) placebo
- Placebo-controlled
- Median follow-up 84 months (range 0-127)
- PDB lesions at baseline: 9 (8.1%) ZA vs 12 (10.8%) placebo
Primary result
New bone lesions
Odds ratio
0.41
95% CI 0.00–3.43
P = 0.25
Events by treatment group
ZA
0
events
Placebo
2
events
As reported in the abstract
Secondary outcomes
Events
| Outcome | ZA | Placebo | OR (95% CI) | P value |
|---|---|---|---|---|
| Poor outcome (lesions new, unchanged or progressing) | — not stated in the abstract | 8 | 0.08 (0.00–0.42) | P = 0.003 |
| Participants with lesions at study end | 1 | 11 | ||
| Rescue therapy with ZA for symptomatic disease | — not stated in the abstract | 1 |
— not stated in the abstract
Biochemical markers of bone turnover
- Significantly reduced in the ZA group
Safety
Adverse events (number and severity)
- Did not differ between groups
Original publication
Randomised trial of genetic testing and targeted intervention to prevent the development and progression of Paget’s disease of bone. Ann Rheum Dis. Published 12 March 2024. doi:10.
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