ESPE Abstracts

Aim and Method: Achondroplasia is the most common form of short-limbed dwarfism, characterized by mutations in the FGFR3 gene. Vosoritide, a C-type natriuretic peptide analog, has shown promise in promoting linear growth in children with achondroplasia. This study aims to present the real-life experiences of using vosoritide in children diagnosed with achondroplasia.

Method and Results: A prospective study was conducted on children with achondroplasia receiving vosoritide treatment. Primary outcomes measured included changes in annual growth rate and anthropometric parameters. The clinical characteristics of 14 children (8 males) diagnosed with achondroplasia, with a current mean age of 7.98±3.94 years, were examined. All patients were treated with vosoritide at a dose of 15 mcg/kg/day for a median duration of 0.76 (0-1.31) years, 11 patients completed 6 months and 8 patients completed 9 months of treatment. A statistically significant improvement in the annual growth velocity (GV) standard deviation score (SDS) was detected in the patients. The median GV-SDS values(min-max) changed from -1.92 (-3.87-0.36) pre-treatment to -0.68 (-2.17 to 0.98) post-treatment (P = 0.0029). Additionally, there was no significant difference in upper/lower segment SDS and the arm span-height difference SDS with treatment. This suggests that the growth induced by vosoritide was proportional, maintaining the typical body proportions for children with achondroplasia.

Other anthropometric parameter evaluations are presented in Table 1.

In this patient group, treatment with vosoritide resulted in significant increase in GVSDS and sitting height SDS. However significant improvement in height SDS seems to require longer time.

Summary of Results: Vosoritide appears to be effective in increasing height velocity without changing body proportions in children with achondroplasia based on our real-life clinical experience.