Box 5 Markers of treatment efficacy in XLH

1. Biomarkers (e.g. alkaline phosphatase levels)

2. Clinical manifestations (e.g. lower limb deformities, linear growth)

3. Imaging (e.g. pseudofracture healing, deformity analysis)

4. Patient-reported outcomes (e.g. pain)

These markers depend on the age of the person living with XLH and status of their disease [2, 7, 21]