Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients.
case_series · Level IV
Where this comes from
- Record sourced from PubMed, PMID 39624115.
- Also identified by DOI 10.1016/j.bonr.2024.101814 and PMC identifier 11609550.
- Licence recorded as CC BY.
- The licence permits redistribution, so the abstract is shown in full and the full text is available from the publisher.
Abstract
X-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020. We describe three adults with persistent XLH symptoms who received burosumab treatment in a real-world setting. Patients report improvements in pain, mobility, physical function, energy, fatigue, and mental wellbeing through patient-reported outcome measures, enriched with further detail from written testimonials.