Burosumab prevents further height deficit in toddlers affected by XLH.

ABSTRACT:

Background

X-linked hypophosphatemia (XLH) is a rare disease caused by PHEX variants. Besides rickets, XLH leads to disproportionately short stature, which develops during the first months of life. Burosumab afforded minimal improvement of growth in children above the age of 4 years. No data are available on growth, including body mass index, of XLH children who started burosumab at a very young age, i.e., between 1 and 4 years.

Methods

We performed a prospective follow-up of growth and other XLH-related outcomes in XLH children who started burosumab before the age of 4 years. We compared these children 1:2 with a historical cohort of XLH children who started vitamin D analogs and phosphate supplements before the age of 4 years.

Results

We included 15 children treated

SUBMITTER: Sala E 

PROVIDER: S-EPMC12538273 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature