Real-world effectiveness of burosumab versus oral phosphate and active vitamin D in adults with X-linked hypophosphatemia (XLH): a 3-year follow-up of the XLH Disease Monitoring Program
Authors' conclusion
In this real-world analysis of adults with XLH over a 3-yr follow-up, burosumab treatment was associated with improved serum phosphate and patient-reported outcomes versus Pi/D.
Study context
- Population
- Adults with XLH in the XLH Disease Monitoring Program (DMP)
- Participants
- 54 vs 55
- Exposure
- Burosumab vs oral phosphate and active vitamin D
- Burosumabn = 54
- Pi/Dn = 55reference
- Outcome
- Change from baseline in serum phosphate
- Follow-up
- Year 3
Methodological detailsreal-world, Americas-based, inverse probability of treatment weighting, 3-yr follow-up
- Real-world
- Americas-based
- Inverse probability of treatment weighting
- 3-yr follow-up
Main finding
As reported in the abstract
Burosumab
0.6
mean · SD 0.8 · mg/dL
Pi/D
0.1
mean · SD 0.7 · mg/dL
P < 0.001
Observational data: associations, not effects.
Further findings
Serum phosphate above lower limit of normal (2.5 mg/dL) · year 1
P < 0.001
Serum phosphate above lower limit of normal (2.5 mg/dL) · year 3
P < 0.001
| Outcome | Burosumab | Pi/D | P value |
|---|---|---|---|
| Change from baseline in serum phosphate · year 1Mean, mg/dL | 0.8 (SD 0.8) | 0.1 (SD 0.7) | P < 0.001 |
| Change from baseline in WOMAC pain · year 1Mean | −6.3 (SD 27.7) | 2.1 (SD 22.4) | P = 0.055 |
| Change from baseline in WOMAC physical function · year 1Mean | −6.4 (SD 19.1) | 0.1 (SD 29.0) | P = 0.122 |
| Change from baseline in WOMAC stiffness · year 1Mean | −11.1 (SD 32.1) | −2.3 (SD 33.7) | P = 0.073 |
| Change from baseline in WOMAC pain · year 3Mean | −9.8 (SD 22.6) | 3.3 (SD 24.1) | P < 0.001 |
| Change from baseline in WOMAC physical function · year 3Mean | −10.0 (SD 25.7) | 3.3 (SD 24.7) | P = 0.001 |
| Change from baseline in WOMAC stiffness · year 3Mean | −15.2 (SD 29.5) | −3.7 (SD 31.4) | P = 0.016 |
Original publication
Real-world effectiveness of burosumab versus oral phosphate and active vitamin D in adults with X-linked hypophosphatemia (XLH): a 3-year follow-up of the XLH Disease Monitoring Program. J Bone Miner Res. Published 23 September 2026. doi:10.
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