Pediatric X-linked hypophosphatemia: burosumab shows promise

  • Carpenter TO & al.
  • N Engl J Med
  • 24 May 2018

  • curated by Emily Willingham, PhD
  • Clinical Essentials
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Takeaway

  • In children with X-linked hypophosphatemia, burosumab led to improved renal phosphate reabsorption, growth, serum phosphorus levels, and function, along with reduced disease severity and less pain.

Why this matters

Key results

  • Improvements seen with burosumab include:
    • Thacher rickets severity score, baseline to week 40: decreased from 1.9 to 0.8 (dosing every 2 weeks); from 1.7 baseline to 1.1 (dosing every 4 weeks; P<.001 both>
    • Mean serum phosphorus: increased after the first dose, in normal range by week 6 in more than half of patients (both dose groups); stable levels maintained to week 64, dosing every 2 weeks.
    • Increased renal phosphate reabsorption and height z-scores.
    • Improved pain/comfort and function scores in both dose groups.
  • No clinically significant safety findings.

Study design

  • Open-label, phase 2 trial.
  • 52 children: 26 dosing every 2 weeks, 26 dosing every 4 weeks.
  • Dose adjusted to serum phosphorus level.
  • Primary endpoint: change, baseline to 40, 64 weeks.
  • Funding: Ultragenyx Pharmaceutical, Kyowa Hakko Kirin. 

Limitations

  • No active control group; trial comparing drug to conventional therapy is ongoing.

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