Real-World Outcomes of Vosoritide Treatment in Chinese Children with Achondroplasia: The Retrospective Cohort COREV Study.
Vosoritide treatment in Chinese children with achondroplasia showed a significant improvement in height SDS and annual growth velocity, but the clinical relevance of these changes should be carefully considered.
Where it sits
this study against the rest of the vosoritide corpusSummary and findings
This study evaluated the effectiveness and safety of vosoritide in 26 Chinese children diagnosed with achondroplasia. Height standard deviation score (SDS) improved from -4.7 ± 0.1 at baseline to -4.3 ± 0.2 at 12 months, with a significant increase in annual growth velocity reaching 8.7 ± 1.4 cm/year after 18 months. Adverse reactions were monitored during treatment.
Abstract
<h4>Introduction</h4>This study aimed to evaluate the effectiveness and safety of vosoritide in Chinese children with achondroplasia (ACH) in a real-world setting.<h4>Methods</h4>A total of 26 children diagnosed with ACH and treated with vosoritide were enrolled in the study. Clinical indicators such as height, sitting height, and body mass index (BMI), as well as laboratory indicators including bone age and insulin-like growth factor level, were collected and analyzed before and after treatment. Improvements in height standard deviation score (SDS), sitting height/height ratio, and BMI were evaluated. Statistical differences were analyzed across different age and sex groups. Adverse reactions during the treatment were also monitored.<h4>Results</h4>Among the 17 patients assessed at 12 months, height SDS (based on general population growth charts) improved from - 4.7 ± 0.1 at baseline to - 4.3 ± 0.2 at 12 months (P < 0.001). With continued treatment, annual growth velocity (AGV) significantly increased, reaching 8.7 ± 1.4 cm/year in 12 patients who remained under follow-up after 18 months of treatment. Furthermore, BMI SDS exhibited a continuous decline. The sitting height/height ratio decreased during the treatment, indicating an optimization of body proportions. Additionally, growth improvement was found in both age (below five years old and above five years old) and sex (male and female) groups. Except for the total procollagen I intact N-terminal (TP1NP), which significantly increased at 12 months of treatment compared to baseline, other biochemical indicators (including insulin-like growth factor, alkaline phosphatase, and osteocalcin) showed no significant differences after the treatment.<h4>Conclusion</h4>Vosoritide exerts significant growth-promoting effects and favorable safety in Chinese children with ACH. It improves height SDS and body proportions, demonstrating consistent effectiveness across various age groups and sex groups. Early diagnosis and timely initiation of vosoritide treatment can significantly alter the growth trajectory of these children.
Background
This study addresses the real-world effectiveness of vosoritide in treating achondroplasia, a common form of dwarfism caused by genetic mutations. Prior studies have suggested potential benefits of vosoritide in promoting growth in children with this condition, but real-world data are limited. Understanding its impact in a clinical setting is essential for evaluating its utility in practice.
Methods
This retrospective cohort study included 26 children diagnosed with achondroplasia who received vosoritide treatment. Clinical indicators such as height, sitting height, and BMI were measured before and after treatment. The primary outcome was the change in height SDS, with secondary outcomes including annual growth velocity and other biochemical markers.
Results
Height SDS improved significantly from -4.7 ± 0.1 at baseline to -4.3 ± 0.2 at 12 months (P < 0.001). Annual growth velocity reached 8.7 ± 1.4 cm/year in 12 patients after 18 months. Other indicators, such as BMI SDS, showed a continuous decline, while TP1NP significantly increased at 12 months.
Interpretation
The findings suggest that vosoritide may have a statistically significant effect on growth in children with achondroplasia, although the clinical significance of a change in height SDS from -4.7 to -4.3 is uncertain. The small sample size and limited follow-up duration may confound the results, limiting the ability to generalize these findings. Practitioners should consider these limitations when interpreting the results.
Key findings
- Height SDS improved from -4.7 ± 0.1 at baseline to -4.3 ± 0.2 at 12 months (P < 0.001).
- Annual growth velocity reached 8.7 ± 1.4 cm/year in 12 patients after 18 months.
- BMI SDS exhibited a continuous decline.
- Sitting height/height ratio decreased during treatment.
- Total procollagen I intact N-terminal (TP1NP) significantly increased at 12 months compared to baseline.
Limitations
- small n=26
- limited follow-up duration of 18 months for some outcomes
- retrospective cohort design may introduce bias
- no control group for comparison