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Burosumab for Pediatric X-Linked Hypophosphatemia.

Erik A Imel1,2

  • 1Department of Medicine, Indiana University School of Medicine, 1120 West Michigan Street, CL 365, Indianapolis, IN, 46202-5111, USA. eimel@iu.edu.

Current Osteoporosis Reports
|May 10, 2021
PubMed
Summary

X-Linked hypophosphatemia (XLH) management has advanced with burosumab, a treatment improving rickets and growth in children and adults. Further research is needed to fully understand its long-term effects on various health outcomes.

Keywords:
BurosumabFGF23RicketsX-Linked hypophosphatemia

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Area of Science:

  • Medical Genetics
  • Pediatric Endocrinology
  • Bone Metabolism

Background:

  • X-Linked hypophosphatemia (XLH) is the most prevalent genetic cause of rickets.
  • XLH is characterized by elevated FGF23, leading to hypophosphatemia and significant musculoskeletal issues.
  • These complications include bone deformities, growth impairment, dental problems, and chronic pain, impacting quality of life.

Purpose of the Study:

  • To review recent advancements in managing XLH.
  • To focus on the therapeutic applications of burosumab, an FDA-approved treatment for XLH.
  • To discuss the clinical efficacy and remaining questions regarding burosumab treatment.

Main Methods:

  • Literature review of studies on XLH management.
  • Analysis of clinical trial data for burosumab in pediatric and adult XLH patients.
  • Evaluation of treatment outcomes compared to conventional therapies.

Main Results:

  • Burosumab demonstrated significant improvement in rickets and growth in children compared to conventional therapy.
  • The treatment also showed efficacy in healing pseudofractures in adults.
  • Despite positive outcomes, long-term effects on final height, nephrocalcinosis, and dental health require further investigation.

Conclusions:

  • Burosumab represents a significant advancement in XLH treatment, offering improved clinical outcomes.
  • The drug has shown promise in both pediatric and adult populations.
  • Ongoing research is crucial to address remaining uncertainties and optimize long-term management strategies for XLH.