Predictors of response to burosumab in adults with X-linked hypophosphatemia: real-world data from an Italian cohort.

ABSTRACT:

Purpose

X-linked hypophosphatemia (XLH) is a genetic disorder characterized by elevated FGF23 levels, leading to phosphate wasting and hypophosphatemia, causing skeletal and extraskeletal abnormalities. Burosumab, an antibody targeting FGF23, improves hypophosphatemia and clinical outcomes. This study evaluated the real-world efficacy of burosumab and identify predictors of treatment response.

Methods

Twenty-seven adult XLH patients (mean age 42 years; 48% female) from an Italian multicenter cohort were treated with burosumab for up to 24 weeks. Laboratory tests were evaluated at midpoints and endpoints (14 and 28 days) of the dosing interval. In a subset of patients (N = 11) followed for 48 weeks, laboratory tests and patient-reported outcomes were also assessed.

Results

SUBMITTER: Arcidiacono GP 

PROVIDER: S-EPMC12313718 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature