Effects of Burosumab Treatment on Mineral Metabolism in Children and Adolescents With X-linked Hypophosphatemia
Authors' conclusion
In this real-world setting, 12 months of burosumab treatment was equally effective in normalizing serum ALP in adolescents and children, despite persistent mild hypophosphatemia in one-half of patients, suggesting that complete normalization of serum phosphate is not mandatory for substantial improvement of rickets in these patients. Adolescents appear to require lower weight-based burosumab dosage than children.
Study context
- Population
- Children and adolescents with X-linked hypophosphatemia (XLH) treated with burosumab, hospital clinics; prior oral phosphate and active vitamin D in 88%
- Participants
- 93 children
- Exposure
- Burosumab in children vs adolescents with XLH
- Childrenn = 65reference
- Adolescentsn = 28
- Outcome
- Z scores for serum phosphate, ALP and TmP/GFR
- Follow-up
- 12 months
Methodological detailsreal-world
- Real-world
Main finding
As reported in the abstract
Serum phosphate within normal range, both groups
≈42%
TmP/GFR within normal range, both groups
≈27%
Alkaline phosphatase (ALP) within normal range, both groups
≈80%
Observational data: associations, not effects.
Further findings
Final weight-based burosumab dose · 12 months · lower with Adolescents
P < 0.01
Baseline Z scores vs healthy children
- Serum phosphate (hypophosphatemia)
- −4.4 SD
- TmP/GFR (reduced)
- −6.5 SD
- ALP (elevated)
- 2.7 SD
- Each vs healthy children, irrespective of age
- P < 0.001
Original publication
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