Early Access experience with burosumab in adults with X-linked hypophosphataemia in the UK: real-world clinical and patient-reported outcomes
X-linked hypophosphataemia (XLH)
UK centres with patients enrolled in the burosumab Early Access Program are sites in this observational study. Investigators collect data from patients' medical records, including patient-reported outcomes, that were recorded during participants' normal clinical care. No additional tests or instruments were used for the purposes of this study. Key parameters that will be abstracted, if available, from medical records include: 1. Patient characteristics, demographics and relevant medical history 2. Medications, including analgesics, and treatment history 3. Serum phosphate, alkaline phosphatase, parathyroid hormone, calcium and vitamin D 4. Patient-reported outcomes including Brief Pain Inventory Short Form (BPI), Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) and EQ-5D-5L 5. Burosumab exposure and adverse events
All participants enrolled in the burosumab early access programme (EAP) will be considered for inclusion
Participants opting out will not have their clinical data collected