Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment
Authors' conclusion
Continued treatment with burosumab appears necessary for sustained clinical benefit.
Study context
- Population
- Adults with X-linked hypophosphataemia who continued from a 96-week phase 3 study into a 48-week open-label extension
- Participants
- 31 adults
- Primary endpoint
- Fasting serum phosphate concentration
Methodological details48-week extension, post-hoc exploratory analysis
- 48-week extension
- Post-hoc exploratory analysis
Primary result
As reported in the abstract
Fasting serum phosphate · through the 48-week extension
- Improvements at 96 weeks maintained through the 48-week extension
Secondary outcomes
Serum dihydroxyvitamin D concentration and renal phosphate reabsorption · through the 48-week extension
- Improvements at 96 weeks were maintained through the 48-week extension
Patient-reported outcomes (PROs): stiffness and physical function (WOMAC), pain (Brief Pain Inventory short-form), fatigue (Brief Pain Inventory) · through the 48-week extension
- Improvements were maintained
Ambulatory function (6-Minute Walk Test) · through the 48-week extension
- Improvements were maintained
Laboratory efficacy tests, PROs and ambulatory function: discontinued burosumab between studies (n=7) vs received at least one dose (n=23)
- Benefits may be lost when treatment is interrupted but recover over time when treatment is reinstated
- Post-hoc exploratory analysis
Original publication
Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment. RMD Open. Published 28 February 2023. doi:10. 1136/ rmdopen-2022-002676
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