Real-world non-interventional post-authorization safety study of long-term use of burosumab in children and adolescents with X-linked hypophosphatemia: first interim analysis.

ABSTRACT:

Background

X-linked hypophosphatemia (XLH) is a rare, progressive disorder characterized by excess fibroblast growth factor 23 (FGF23), causing renal phosphate-wasting and impaired active vitamin D synthesis. Burosumab is a recombinant human monoclonal antibody that inhibits FGF23, restoring patient serum phosphate levels. Safety data on long-term burosumab treatment are currently limited.

Objectives

This post-authorization safety study (PASS) aims to monitor long-term safety outcomes in children and adolescents (1-17 years) treated with burosumab for XLH. This first interim analysis reports the initial PASS safety outcomes.

Design

A 10-year retrospective and prospective cohort study.

Methods

This PASS utilizes International XLH Registry (NCT03193476) data, whi

SUBMITTER: Boot AM 

PROVIDER: S-EPMC11102685 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature