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.
References:
Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Mohnike K, Prada CE, Kubota T, Arundel P, Leiva-Gea A, Rowell R, Low A, Sabir I, Huntsman-Labed A, Day J Genet Med. 2024. doi: 10.1016/j.gim.2024.101274.
Epub ahead of print.
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