Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life.

Evaluate the impact of vosoritide on health-related quality of life in children with achondroplasia. Participants received vosoritide (15 μg/kg/day) in an extension trial (NCT03424018) after having participated in a placebo-controlled trial (NCT03197766). The population comprised 119 participants (mean [SD] age 9.7 [2.6] years). Mean treatment duration was 4 (0.78) years. At year 3, the largest mean (SD) changes were observed in the QoLISSY physical score (5.99 [19.41], caregiver-reported; 6.32 [20.15], self-reported) and social score (2.85 [8.29] and 6.76 (22.64), respectively). Changes were greatest in participants with ≥1 SD increase in height Z-score (physical: 11.36 [19.51], caregiver-reported [n=38]; 8.48 [21.83], self-reported [n=28]) (social: 5.84 [15.45] and 9.79 [22.80], respectively). To determine how domain scores may change with age in untreated persons, models were produced using observational/untreated-person data. A 1-year increase in age was associated with a change of 0.16 (SE, 0.55) and 0.16 (0.50), for caregiver-reported physical and social domain scores, respectively. Self-reported scores changed by 1.45 (0.71) and 1.92 (0.77), respectively. These data suggest that after 3 years of treatment vosoritide demonstrates a positive effect on physical and social functioning among children with achondroplasia, particularly in children with a more pronounced change in height Z-score.

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