Real-world impact of treatment on growth in children with X-linked hypophosphatemia
Authors' conclusion
Burosumab had a robust, positive association with improved growth outcomes in male and female children and adolescents with XLH. Modeling based on a median of 3.3 years of follow-up predicted burosumab can support improvement of growth that will likely result in greater adult height.
Study context
- Population
- Children aged 2–17 years with X-linked hypophosphatemia (XLH), outpatient clinicsanalysed
- Participants
- 641 children
- Exposure
- Burosumab vs oral phosphate salts and active vitamin D or no treatment
- Burosumab-treatedn = 498
- Burosumab-naïven = 143reference
- Outcome
- Growth, from retrospective and prospective height data, as a long-term estimate of the impact of treatment
Methodological detailsinterim data from 3 ongoing studies unified into APEX, retrospective and prospective height data, mixed regression model
- Interim data from 3 ongoing studies unified into APEX
- Retrospective and prospective height data
- Mixed regression model
Main finding
As reported in the abstract
Additional growth velocity
Additional growth velocity (children)
0.085 Z-score/year
P < 0.0001
children
Additional growth velocity (adolescents)
0.121 Z-score/year
P < 0.0001
adolescents
Observational data: associations, not effects.
Further findings
Enrollment age
Predicted adult height
- Modeling predicted greater adult height among burosumab-treated participants
Original publication
Real-world impact of treatment on growth in children with X-linked hypophosphatemia. J Clin Endocrinol Metab. Published 31 March 2026. doi:10.
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