Early initiation of therapy with vosoritide: case report. Description of the first Italian patient affected by achondroplasia treated with vosoritide before 2 years of age - Report - MDSpire
Clinical Report: Early Treatment with Vosoritide in Achondroplasia
Background
Achondroplasia is the most common skeletal dysplasia, characterized by disproportionate short stature and a significant decrease in growth velocity during infancy. Vosoritide is the first drug approved to enhance linear growth in children with this condition.
Data Highlights
Parameter
At Birth
After 12 Months of Vosoritide
Weight
2,140 g (−1.28 SDS)
7.15 kg (−1.87 SDS)
Length
42 cm (−2.19 SDS)
67.2 cm (−0.52 SDS)
Head Circumference
33 cm (0.23 SDS)
49.5 cm (−1.10 SDS)
Key Findings
The patient was diagnosed with achondroplasia due to a de novo FGFR3 gene mutation.
Vosoritide therapy was initiated at 7 months of age.
After 12 months of treatment, the patient showed an improvement of +0.35 SD in length.
No severe adverse effects were reported during the treatment period.
The patient's growth parameters were monitored and documented at regular intervals.
Clinical Implications
Clinicians should consider the benefits of initiating therapy before the age of two, particularly in light of the rapid growth deficits characteristic of this condition.
Conclusion
This case highlights the use of vosoritide in a patient with achondroplasia, documenting the treatment initiation and growth outcomes.
