Description

Savarirayan et al. assessed the impact of vosoritide on growth-associated health-related quality of life (HRQoL) in children with achondroplasia. Data were gathered using the Quality of Life in Short Stature Youth (QoLISSY) questionnaire. Vosoritide improved self-reported and caregiver-reported QoLISSY physical and social scores, with the greatest increases being seen in participants with a ≥1 SD increase in height Z-score.

A total of 121 children aged 5 to <18 years were enrolled in the 111-301 study. Of these, 119 proceeded to the 111-302 open-label extension study. All participants received vosoritide at a daily dose of 15 µg/kg. At Year 3, improvements were noted in QoLISSY physical and social scores, with the greatest improvements observed in participants with ≥1 standard deviation increase in height Z-score. These results indicate that there are quality of life benefits related to improved height deficit in children with achondroplasia treated with vosoritide.