Real-world growth outcomes and safety of vosoritide in children with achondroplasia: a Brazilian single center 24 monhts experience
Authors' conclusion
Vosoritide increased linear growth in this real-world cohort, with heterogeneous response patterns over time. Height velocity increment during the first 12 months did not predict longer-term outcomes, and body proportionality remained unchanged over 24 months.
Study context
- Population
- Children with achondroplasia treated with vosoritide at a single tertiary centre in Brazil; mean age 5.92 ± 3.56 years
- Participants
- 35 children
- Exposure
- Vosoritide treatment vs baseline
- Vosoritiden = 35
- Outcome
- Growth: height SDS (WHO and achondroplasia-specific charts) and annualized HV SDS
- Follow-up
- 12 and 24 months
Methodological detailssingle-centre, real-world
- Single-centre
- Real-world
Main finding
As reported in the abstract
Change from baseline in growth
- HV SDS increase at 12 months vs baseline (p < 0.001)
- +1.50 SDS
- HV SDS increase at 24 months vs baseline (p < 0.001)
- +1.91 SDS
- Achondroplasia-specific height SDS at 12 months, trend (p = 0.060)
- +0.17 SDS
- Achondroplasia-specific height SDS at 24 months (p < 0.001)
- +0.50 SDS
- WHO-based height SDS at 24 months (p = 0.005)
- +0.30 SDS
Observational data: associations, not effects.
Further findings
Body proportionality (height/(height - sitting height) ratio)
- Remained unchanged (p = 0.134)
Predictors of response
- HV increment in first 12 months did not significantly predict height SDS gain at 24 months
- Significant age-by-time interaction (p = 0.006)
Safety
- Hypertrichosis
- 66% of patients
- Severe adverse events
- 0
Original publication
Real-world growth outcomes and safety of vosoritide in children with achondroplasia: a Brazilian single center 24 monhts experience. Horm Res Paediatr. Published 21 July 2026. doi:10.
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