- Burosumab is recommended as the primary therapy for pediatric patients with X-linked hypophosphatemia (XLH) presenting with rickets, elevated alkaline phosphatase (ALP), or radiologic signs of rickets.
- Long-term studies have reported benefits of reduced dental abscesses and improved quality of life in pediatric patients treated with burosumab.
- Monitoring includes serum phosphate levels but not fibroblast growth factor 23 (FGF23) levels.
- The guidelines emphasize the need for further research to develop clinical scores for evaluating treatment efficacy, optimize surgical timing, and assess long-term outcomes.
- Burosumab was contraindicated during pregnancy as a result of potential reproductive toxicity.
- Contraception was recommended for patients of childbearing potential.
New XLH Guidelines Shift Primary Therapy to Burosumab
Conexiant
January 16, 2025