Treatment of Children and Adults With X-Linked Hypophosphatemia With Calcitriol Alone: A Prospective, Open-Label Study
Authors' conclusion
Calcitriol monotherapy is safe and well-tolerated in XLH, with modest benefits on laboratory indices of mineral metabolism and rickets.
Study context
- Population
- Children and adults with X-linked hypophosphatemia (XLH) aged ≥4 years, not pregnant, baseline 25-hydroxyvitamin D ≥20 ng/mL and normal serum calcium; outpatient endocrinology clinics, United States
- Exposure
- Calcitriol monotherapy: 12 months vs baseline
- Outcome
- Change in serum phosphate and nephrocalcinosis (all participants) and rickets severity score (children)
- Follow-up
- 12 months
Methodological detailssingle-arm, open-label, 1-year, Data collected in a research unit
- Single-arm
- Open-label
- 1-year
- Data collected in a research unit
Main finding
As reported in the abstract
Serum phosphate, nephrocalcinosis, rickets
Rickets improved in children with open epiphyses
2 of 4
Observational data: associations, not effects.
Secondary outcomes
Height z-scores (children) · 12 months
- Height z-scores were unchanged
Cortical thickness at radius diaphysis (adults) · 12 months
- Increased cortical thickness in adults
- P = 0.012
Serum alkaline phosphatase (children) · 12 months
- Decreased in children
- P = 0.021
PTH · 12 months
- Trended lower in children
- P = 0.065
- Trended lower in adults
- P = 0.063
Safety
Participants
- Mild hypercalcemia
- 4
- Hypercalciuria
- 2
- Resolved with calcitriol titration
Original publication
Treatment of Children and Adults With X-Linked Hypophosphatemia With Calcitriol Alone: A Prospective, Open-Label Study. J Clin Endocrinol Metab. Published 24 October 2025. doi:10.
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