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For persons on (stable dose of) burosumab for safety & efficacy monitoring
| Serum phosphate, calcium, & creatinine, ALP, intact PTH Note: The adequacy of treatment is indicated by normal ALP, intact PTH, & normal (or near-normal) phosphate levels (using age-specific reference values).
| Every 3-6 mos in children & every 6-12 mos in adults |
| Renal ultrasound for nephrocalcinosis | At start of treatment, then every 6 mos for 1st yr, & annually thereafter |
| Lower extremity radiographs to assess skeletal response to treatment | Consider based on symptoms & physical exam findings (avoid routine radiographs & unnecessary radiation exposure; consider MRI). |
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For persons on active vitamin D analogs & phosphate therapy to identify & prevent therapeutic complications
| Serum phosphate, calcium, & creatinine, ALP, intact PTH, urinary calcium & creatinine | Every 3 mos |
| Lower extremity radiographs to assess skeletal response to treatment | Consider based on symptoms & physical exam findings (avoid routine radiographs & unnecessary radiation exposure; consider MRI). |
| Renal ultrasound exam to assess for nephrocalcinosis | At start of treatment & then consider every 1-5 yrs; however, the recommended frequency has not been well established. 1 |
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Asymptomatic adults not receiving medical therapy
| 25-hydroxyvitamin D | Consider every 6 mos depending on clinical risk factors for vitamin D deficiency |
| ALP, creatinine, calcium, PTH | Every 12 mos |
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Skeletal
| Assessment of growth & lower limb alignment (intercondylar & intermalleolar distance) | At each visit throughout childhood |
| Craniofacial exam for signs of craniosynostosis | At each visit throughout infancy |
| Clinical assessment of joint mobility & pain | At each visit &/or annually |
| Bone age measurement to evaluate growth potential | Consider based on clinical necessity (do not repeat on routine basis). |
Radiographs of painful areas to assess for calcifications, pseudofractures, &/or insufficiency fractures Note: Knee MRI has shown promise as a tool to monitor disease activity & skeletal response to treatment, which avoids radiation exposure of serial radiographs. 2
| As needed |
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Neurologic
| Eval of those w/headache &/or vertigo for Chiari I malformation |
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Dental
| Dental exam | Every 6 mos |
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Hearing
| Hearing eval | As needed |
Psychosocial /
Quality of life
| Assessment of psychosocial needs, fatigue, sleep issues, & quality of life | At each visit |
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Cardiovascular risk
| Assessment of weight, blood pressure, other cardiovascular/metabolic syndrome risk factors | At each visit as indicated |
