Second interim analysis of the post-authorisation safety study (PASS) of burosumab in paediatric patients with X-linked hypophosphataemia
Clicks: 4
ID: 318329
2026
Article Quality & Performance Metrics
Overall Quality
Not rated
Combines reader engagement with the AI quality analysis. This
article has not been analysed, so there is no overall score —
reader engagement is measured and shown alongside.
Reader Engagement
Steady Performance
0.9
/100
4 views
3 readers
AI Quality Assessment
Not analyzed
Readership in this journal
SteadyRanked #66 of 96 articles by views in european journal of endocrinology
Most read
Least read
Bar heights use a square-root scale.
Mint this article as an NFT
Not yet mintedCreate a permanent, verifiable on-chain record of this article on the Scimatic Network. The NFT is held in your Journament account, and you can withdraw it to your own wallet at any time.
5
SUSD
one-off · no wallet required
Abstract
OBJECTIVE: X-linked hypophosphataemia (XLH) is a rare genetic disorder characterised by defective bone and tooth mineralisation. Burosumab is a recombinant, human, anti-fibroblast growth factor 23 monoclonal antibody approved for treating XLH. Due to the rarity of the disease, additional safety data for treatment of XLH with burosumab are required. DESIGN: A post-authorisation safety study analysis of data for paediatric participants from the International X-linked Hypophosphataemia Registry. This study included standard diagnostic and monitoring clinical data at participating centres, regardless of treatment. METHODS: This was a second interim analysis performed 5 years after study initiation in the paediatric population. Primary objectives assessed safety outcomes in children and adolescents with XLH. The secondary objective compared safety outcomes with burosumab vs phosphate and/or active vitamin D. RESULTS: In total, 340 participants were treated with ≥1 dose of burosumab, and 91 were treated with phosphate and/or active vitamin D. Overall, 37.4% and 22.0% of participants in the burosumab or phosphate and/or active vitamin D cohorts, respectively, experienced ≥1 adverse event. The proportions of participants with events were similar among children and adolescents. Forty-nine (14.4%) participants reported events possibly/probably related to burosumab. No events led to withdrawal of burosumab. In both cohorts, no serious adverse events were considered related to treatment, and there were no deaths. Hospitalisations occurred in ∼71% of participants. Hyperphosphataemia, elevated parathyroid hormone, and ectopic mineralisation events were rare. CONCLUSIONS: The safety profile of burosumab was consistent with previous studies, and no new safety concerns were reported for children or adolescents.
| Reference Key |
openalex_W7165679337
Use this key to autocite in the manuscript while using
SciMatic Manuscript Manager or Thesis Manager
|
|---|---|
| Authors | Signe Sparre Beck‐Nielsen, Gema Ariceta, Annemieke M. Boot, Maria Luisa Brandi, Karine Briot, Cortezo Collantes C, Francesco Emma, Sandro Giannini, Dieter Haffner, Richard Keen, Elena Levtchenko, Ola Nilsson, Outi Mäkitie, M Zulf Mughal, Raja Padidela, Liana Tripto-Shkolnik, M. Carola Zillikens, El Mahdi Benchekroun, Eslam Eltahan, Paul Joos-Vandewalle, Malena Melogno-Klinkas, D Schnabel |
| Journal | european journal of endocrinology |
| Year | 2026 |
| DOI |
10.1093/ejendo/lvag099
|
| URL | |
| Keywords | Keywords not found |
Citations
No citations found. To add a citation, contact the admin at info@scimatic.org
Comments
No comments yet. Be the first to comment on this article.