Retinal Structural and Microvascular Findings During Novel NPR-B-Targeted Vosoritide Therapy in Children with Achondroplasia
- 1Department of Ophthalmology, Kartal Dr. Lütfi Kırdar Kartal City Hospital, İstanbul 34865, Turkey
- 2Department of Pediatric Endocrinology, Cerrahpaşa Faculty of Medicine, İstanbul University-Cerrahpaşa, İstanbul 34098, Turkey
Abstract
Background: The aim of this study was to evaluate the short-term ocular findings during vosoritide therapy in children with achondroplasia using optical coherence tomography (OCT) and OCT angiography (OCTA). Methods: In this study, pediatric patients with genetically confirmed achondroplasia underwent standardised ophthalmologic examination, OCT, and OCTA imaging at baseline and after 12 months of vosoritide treatment. Structural parameters, including macular thickness and peripapillary retinal nerve fiber layer (RNFL), as well as vascular densities of the superficial and deep capillary plexuses and choriocapillaris, were analysed. Findings were compared longitudinally and with age- and sex-matched healthy controls. Results: A total of 12 patients and 12 controls were included. During the 12-month follow-up, nominal increases were observed in nasal parafoveal thickness and in average, superior, and nasal RNFL thickness (p < 0.05); however, none remained significant after false-discovery-rate correction. No significant longitudinal changes were detected in OCTA-derived vascular densities. However, vascular density was significantly lower in specific regions in the achondroplasia group compared to controls both before and after treatment. No clinically relevant ocular pathology was identified. Conclusions: No clinically apparent ocular abnormalities or statistically robust longitudinal changes in retinal structure or microvascular perfusion were identified over one year of vosoritide treatment in this small, preliminary cohort.
Related
- Vosoritide therapy in Brazil: Consensus on the Diagnosis, Monitoring, and Management of Achondroplasia
- Achondroplasia: Current Disease-Modifying Therapies and Genome Editing Strategies.
- The hepatic sinusoid is a site of microvascular complications of hyperglycemia
- Development of a 4D Cerebral Microvascular Imaging Platform for Mouse Stroke Model
- Suspected Microvascular Occlusion Syndrome Following Scorpion Envenomation: A Pediatric Case Report
BibTeX
@article{agackesen2026retinal,
title = {{Retinal Structural and Microvascular Findings During Novel NPR-B-Targeted Vosoritide Therapy in Children with Achondroplasia}},
author = {Anıl Ağaçkesen and Hasan Karakaş and Hande Turan and Elvan Bayramoğlu and Olcay Evliyaoğlu},
journal = {Journal of clinical medicine},
year = {2026},
month = oct,
doi = {10.3390/jcm15197661},
url = {https://europepmc.org/article/PMC/PMC13665766},
note = {CC BY},
}
RIS
TY - JOUR
TI - Retinal Structural and Microvascular Findings During Novel NPR-B-Targeted Vosoritide Therapy in Children with Achondroplasia
AU - Anıl Ağaçkesen
AU - Hasan Karakaş
AU - Hande Turan
AU - Elvan Bayramoğlu
AU - Olcay Evliyaoğlu
PY - 2026
DA - 2026/10/01
JO - Journal of clinical medicine
DO - 10.3390/jcm15197661
UR - https://europepmc.org/article/PMC/PMC13665766
AB - Background: The aim of this study was to evaluate the short-term ocular findings during vosoritide therapy in children with achondroplasia using optical coherence tomography (OCT) and OCT angiography (OCTA). Methods: In this study, pediatric patients with genetically confirmed achondroplasia underwent standardised ophthalmologic examination, OCT, and OCTA imaging at baseline and after 12 months of vosoritide treatment. Structural parameters, including macular thickness and peripapillary retinal nerve fiber layer (RNFL), as well as vascular densities of the superficial and deep capillary plexuses and choriocapillaris, were analysed. Findings were compared longitudinally and with age- and sex-matched healthy controls. Results: A total of 12 patients and 12 controls were included. During the 12-month follow-up, nominal increases were observed in nasal parafoveal thickness and in average, superior, and nasal RNFL thickness (p < 0.05); however, none remained significant after false-discovery-rate correction. No significant longitudinal changes were detected in OCTA-derived vascular densities. However, vascular density was significantly lower in specific regions in the achondroplasia group compared to controls both before and after treatment. No clinically relevant ocular pathology was identified. Conclusions: No clinically apparent ocular abnormalities or statistically robust longitudinal changes in retinal structure or microvascular perfusion were identified over one year of vosoritide treatment in this small, preliminary cohort.
SN - 2077-0383
ER -