Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment

RMD OpenPublished Open-label extension of a phase 3 trial

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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