Long-term outcomes after a randomized phase II trial of nerve growth factor eye drops for optic pathway gliomas: four-year follow-up findings in children.
J NeurooncolSep 1, 2026 (epub)
Antonio Ruggiero, Giorgio Attinà, Giorgio Placidi et al.
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In 30 seconds
This study evaluated long-term outcomes in children with optic pathway gliomas (OPG) who previously participated in a randomized phase II trial of murine nerve growth factor (mNGF) eye drops. Fifteen of the original 18 patients were monitored over four years, revealing no significant changes in visual function or neuroradiological parameters, indicating stable disease and favorable safety of mNGF treatment.
Key findings
- No statistically significant changes in visual acuity, VEP amplitude, or PhNR amplitude were observed.
- Brain MRI showed stable disease in all patients throughout the four-year follow-up.
- The study indicates favorable long-term safety and tolerability of mNGF eye drops.
Why it matters
Understanding the long-term effects of treatments for optic pathway gliomas is crucial for managing visual outcomes in affected children. This study provides insights into the safety and stability of mNGF treatment over an extended period.