Perceived Impact of Vosoritide on Health-Related Quality of Life in Children with Selected Genetic Causes of Short Stature: A Qualitative Study of Children and Caregivers
Authors' conclusion
Genetic short stature significantly impacts HRQoL, as reported by both children and their caregivers. Both children and caregivers noted emotional, physical, and social impacts as well as impacts on daily activities. Vosoritide treatment was associated with meaningful improvements in physical impacts and impacts on daily activities.
Study context
- Population
- Children receiving vosoritide for >2 years in a clinical trial and their caregivers; 12 hypochondroplasia, 4 NPR2 mutations, 2 ACAN mutations, 2 Noonan syndrome20 families
- Participants
- 21 vs 14
- Exposure
- Caregivers vs Children
- Caregiversn = 21
- Childrenn = 14
- Outcome
- Changes in impacts on daily activities after starting vosoritide
Methodological detailscross-sectional, remote, interview study, thematic analysis with grounded theory-guided coding
- Cross-sectional
- Remote
- Interview study
- Thematic analysis with grounded theory-guided coding
Main finding
As reported in the abstract
Caregivers
74%
Children
100%
- Improved ability to reach things
- 12 caregivers and 14 children
- Being more independent/needing less assistance
- 9 caregivers and 5 children
Observational data: associations, not effects.
Original publication
Perceived Impact of Vosoritide on Health-Related Quality of Life in Children with Selected Genetic Causes of Short Stature: A Qualitative Study of Children and Caregivers. Horm Res Paediatr. Published 4 September 2026. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →