Purpose: To analyze the visual outcomes following chemotherapy for optic pathway glioma in children with neurofibromatosis type 1.
Methods: This was a retrospective cohort study of 58 children with optic pathway glioma between 2003 and 2019. Best corrected visual acuity (BCVA) at diagnosis and at the last follow-up visit were analyzed. Correlation tests were performed to determine whether the final BCVA was predicted by the initial BCVA, initial Dodge stage, or retinal nerve fiber layer (RNFL) thickness stability. The first-line chemotherapy regimen included vincristine plus carboplatin (2003 to 2016) and vinblastine (since 2017).
Results: Twenty-four children (41%) received chemotherapy. The median age at diagnosis was 3 years, and 61% of the children were female. From the first visit to the last follow-up visit, there was a statistically significant difference in mean BCVA in eyes in the no treatment group ( = .034) but not in eyes in the chemotherapy group ( = .824). A moderate and weak positive correlation was found ( = 0.58 and = 0.29, respectively). At the last follow-up visit, BCVA remained stable in 73% and improved in 27% of eyes in the no treatment group, and BCVA worsened in 25%, remained stable in 62%, and improved in 13% of eyes in the chemotherapy group. At the last follow-up visit, BCVA and Dodge stage had a weak negative correlation ( = -0.06 and = -0.17, respectively). A negative moderate correlation was identified between RNFL thickness and BCVA at the last follow-up visit ( = -0.48 in the no treatment group and = -0.46 in the chemotherapy group).
Conclusions: Children treated with chemotherapy had worse BCVA, although the treatment arrested the decline or improved BCVA in 75% of the patients with no major side effects. .
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