Real-world effectiveness of burosumab across age groups: X-linked hypophosphatemia (XLH) Disease Monitoring Program
Main finding · Serum phosphate z-score, change from baselineResult — go to the resultChange from baseline 1.4Authors' conclusion
This analysis demonstrated the real-world effectiveness of burosumab for XLH, which appears evident across age groups.
Study context
- Population
- Individuals with XLH in the XLH Disease Monitoring Program, incl. age groups excluded from clinical trials (<1, 13–17, and ≥65 years)
- Participants
- 139 individuals
- Exposure
- Burosumab: Y1 and Y3 vs baseline
- Burosumabn = 139
- Outcome
- Serum phosphate z-score, change from baseline
- Follow-up
- Y1 and Y3
Methodological detailsreal-world, long-term-outcomes program, stratified by age group
- Real-world
- Long-term-outcomes program
- Stratified by age group
Main finding
As reported in the abstract
Change from baseline
1.4
P < 0.0001 for each timepoint
Change by group
Burosumab
1.4
mean · SD 1.1
Observational data: associations, not effects.
Further findings
Pediatric participants: serum alkaline phosphatase z-scores, Rickets Severity Scores, pain interference, fatigue, physical function mobility · Y1 and Y3
- Serum alkaline phosphatase z-scores, Rickets Severity Scores and patient-reported pain interference declined significantly at Y1 and Y3
- Non-significant changes in fatigue and physical function mobility
Adults: PRO measures of pain, stiffness and physical function · Y1 and Y3
- Significant improvements in PRO measures of pain, stiffness and physical function; higher proportions achieving minimal clinically important differences at Y3 vs Y1
Age sub-groups
- Trends in overall cohorts generally consistent across age sub-groups, with statistical significance reached for many endpoints
Original publication
Real-world effectiveness of burosumab across age groups: X-linked hypophosphatemia (XLH) Disease Monitoring Program. J Clin Endocrinol Metab. Published 4 April 2026. doi:10.
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