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Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients

Year: 2024

Type: article

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.

Source: Bone Reports

DOI: 10.1016/j.bonr.2024.101814

Authors: Julia Day, Chandrin Jayatilleke, Matthew Roy

Institutions: Bristol Royal Infirmary, Royal United Hospital, Royal United Hospital Bath NHS Trust

Language: English

Topic: Parathyroid Disorders and Treatments

Subfield: Nephrology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $2,400