Health-related quality of life of children with X-linked hypophosphatemia in Germany.

ABSTRACT:

Background

X-linked hypophosphatemia (XLH) is a rare inherited phosphate-wasting disorder associated with bone and dental complications. Health-related quality of life (HRQoL) is reduced in XLH patients on conventional treatment with phosphate supplements and active vitamin D, while information on patients treated with burosumab is rare.

Methods

HRQoL was assessed in 63 pediatric XLH patients participating in a prospective, observational study and patient registry in Germany using the KIDSCREEN-52 survey instrument and standardized qualitative interviews.

Results

The median age of the XLH patients was 13.2 years (interquartile range 10.6 - 14.6). At the time of the survey, 55 (87%) patients received burosumab and 8 (13%) conventional treatment. Forty-six patients (84%

SUBMITTER: Klein M 

PROVIDER: S-EPMC11413074 | biostudies-literature | 2024 Nov

REPOSITORIES: biostudies-literature