ECEESPE2025 Poster Presentations Growth Axis and Syndromes (91 abstracts)
Evaluation of growth velocity in patients with achondroplasia treated with vosoritide
Yasemin Denkboy Öngen 1 , Hatice Nursoy 1 , Munever Uluba 1 & Erdal Eren 1
1Bursa Uludag University, Department of Pediatric Endocrinology, Bursa, Türkiye
JOINT327
Introduction: Achondroplasia, the most common skeletal dysplasia, is associated with severe short stature. Vosoritide is the first treatment for achondroplasia that targets the underlying pathophysiology, a C-type natriuretic peptide (CNP) analog that binds to its receptor on chondrocytes and promotes growth by inhibiting the ERK1/2-MAPK pathway. This study aimed to evaluate the growth velocity of vosoritide treatment in achondroplasia cases of various ages.
Method: Eleven patients had been evaluated. Annual growth velocities were calculated. Descriptive statistics were performed using the IBM SPSS 29. 0. 2. 0 statistical package program (IBM Corp. Released 2023. IBM SPSS Statistics for Windows, Version 29. 0. 2. 0 Armonk, NY: IBM Corp. ).
Results: Vosoritide treatment had been started on eleven patients with achondroplasia (15 mg/kg/dose subcutaneously daily). All patients had c. 1138G>A heterozygous variant in the FGFR3 gene. The mean age at the start of treatment was 7. 01±3. 40 years (2. 84-12. 56 years). The mean duration of vosoritide treatment was determined as 1. 13±0. 51 years (0. 33-2. 08 years). The calculated mean annual growth velocity was 6. 11±2. 41 cm (2. 73-11. 62). Patients’ characteristics are shown in Table 1.
| Patient number | Gender | Age at the start of treatment | Duration of vosoritide treatment (years) | Calculated mean annual growth velocity | Baseline height SDS | Height SDS at last visit | Target height SDS | Tanner stage at the start of treatment | Tanner stage at last visit |
| 1 | M | 2. 91 | 0. 58 | 2. 73 | -4. 59 | -4. 59 | -0. 35 | 1 | 1 |
| 2 | M | 6. 49 | 0. 83 | 8. 19 | -4. 78 | -4. 30 | -0. 07 | 1 | 1 |
| 3 | F | 4. 11 | 2. 08 | 6. 40 | -4. 54 | -4. 22 | -1. 47 | 1 | 1 |
| 4 | M | 12. 56 | 0. 83 | 4. 53 | -4. 75 | -4. 66 | -0. 62 | 1 | 2 |
| 5 | M | 7. 83 | 1. 00 | 7. 73 | -2. 90 | -2. 46 | -0. 73 | 1 | 1 |
| 6 | F | 3. 89 | 1. 20 | 5. 05 | -4. 58 | -4. 76 | -0. 18 | 1 | 1 |
| 7 | M | 2. 84 | 1. 75 | 5. 30 | -4. 72 | -4. 88 | -0. 69 | 1 | 1 |
| 8 | F | 11. 07 | 1. 42 | 6. 44 | -5. 87 | -6. 08 | -0. 60 | 3 | 4 |
| 9 | F | 9. 81 | 0. 33 | 11. 62 | -1. 26 | -1. 44 | 0. 74 | 1 | 2 |
| 10 | M | 9. 50 | 1. 42 | 4. 11 | -7. 48 | -7. 31 | -0. 92 | 1 | 1 |
| 11 | F | 6. 09 | 1. 08 | 5. 11 | -5. 22 | -5. 04 | 0. 82 | 1 | 1 |
Conclusion: Vosoritide has previously been shown to increase linear growth in patients with achondroplasia. In our study, we found a wide range in annual growth velocity with vosoritide, from 2. 76 cm to 11. 62 cm. A longer-term evaluation with more patients should be conducted to determine what causes this difference (age, puberty, treatment duration, target height, baseline height, etc. ).
Keywords Achondroplasia, C-type natriuretic peptide, short stature, skeletal dysplasia, vosoritide.
Volume 110
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Endocrine Abstracts
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