Real-world effectiveness of burosumab vs oral phosphate and active vitamin D in adults with X-linked hypophosphatemia
Authors' conclusion
In this real-world analysis of adults with XLH, burosumab treatment was associated with improved biochemical parameters, pain, physical function, and mobility compared with Pi/D.
Study context
- Population
- Adults with X-linked hypophosphatemia (XLH) in the prospective, Americas-based XLH Disease Monitoring Program
- Participants
- 65 vs 74
- Exposure
- Burosumab vs oral phosphate/active vitamin D
- Burosumabn = 65
- Pi/Dn = 74reference
- Outcome
- Change from baseline in serum phosphate
- Follow-up
- Year 1 visit
Methodological detailsreal-world data, inverse probability of treatment weighting
- Real-world data
- Inverse probability of treatment weighting
Main finding
As reported in the abstract
Burosumab
0.78
mean · SE 0.08 · mg/dL
Pi/D
0.15
mean · SE 0.14 · mg/dL
P < 0.001
Observational data: associations, not effects.
Further findings
| Outcome | Burosumab | Pi/D | P value |
|---|---|---|---|
| Change from baseline in serum active vitamin D · year 1 visitInverse probability of treatment weighting · mean, pg/mL | 19.41 (SE 3.39) | 5.49 (SE 3.43) | P = 0.011 |
| Change from baseline in PTH · year 1 visitMean, pg/mL | −13.82 (SE 5.00) | 11.79 (SE 8.10) | P = 0.006 |
| Change from baseline in WOMAC pain · year 1 visitMean | −7.50 (SE 2.34) | 4.47 (SE 3.23) | P = 0.004 |
| Change from baseline in WOMAC physical function · year 1 visitMean | −5.68 (SE 1.96) | 6.77 (SE 4.85) | P = 0.006 |
| Change from baseline in WOMAC total · year 1 visitMean | −7.78 (SE 2.06) | 3.15 (SE 3.37) | P = 0.005 |
| Change from baseline in PROMIS PF · year 1 visitMean | 1.51 (SE 0.73) | −1.64 (SE 1.11) | P = 0.018 |
| Change from baseline in Timed Up and Go · year 1 visitMean, s | −1.19 (SE 0.42) | 0.55 (SE 0.43) | P = 0.011 |
| Change from baseline in WOMAC stiffness · year 1 visitTrend towards improvement with burosumab · mean | −10.16 (SE 2.85) | −1.79 (SE 3.68) | P = 0.086 |
Original publication
Real-world effectiveness of burosumab vs oral phosphate and active vitamin D in adults with X-linked hypophosphatemia. J Bone Miner Res. Published 2 May 2025. doi:10.
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