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 (P = .034) but not in eyes in the chemotherapy group (P = .824). A moderate and weak positive correlation was found (r = 0.58 and r = 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 (r = -0.06 and r = -0.17, respectively). A negative moderate correlation was identified between RNFL thickness and BCVA at the last follow-up visit (r = -0.48 in the no treatment group and r = -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. [J Pediatr Ophthalmol Strabismus. 2022;59(2):128-135.].

中文翻译：

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1 型神经纤维瘤病化疗治疗视神经胶质瘤的视觉结果。

目的 分析 1 型神经纤维瘤病患儿视神经胶质瘤化疗后的视力结果。 方法 这是一项回顾性队列研究，纳入 2003 年至 2019 年间 58 名视神经胶质瘤患儿。诊断时和治疗时的最佳矫正视力 (BCVA)对末次随访进行分析。进行相关测试以确定最终 BCVA 是否由初始 BCVA、初始 Dodge 阶段或视网膜神经纤维层 (RNFL) 厚度稳定性预测。一线化疗方案包括长春新碱加卡铂（2003 年至 2016 年）和长春碱（自 2017 年起）。结果 24 名儿童 (41%) 接受了化疗。诊断时的中位年龄为 3 岁，61% 的儿童为女性。从第一次就诊到最后一次随访，未治疗组眼睛的平均 BCVA 有统计学意义的差异 (P = .034)，但在化疗组的眼睛中没有 (P = .824)。发现中度和弱正相关（分别为 r = 0.58 和 r = 0.29）。在最后一次随访中，未治疗组 73% 的眼睛 BCVA 保持稳定，27% 的眼睛改善，化疗组 25% 的眼睛 BCVA 恶化，62% 的眼睛保持稳定，13% 的眼睛改善团体。在最后一次随访中，BCVA 和 Dodge 阶段呈弱负相关（分别为 r = -0.06 和 r = -0.17）。在最后一次随访中发现 RNFL 厚度和 BCVA 之间存在中度负相关（无治疗组 r = -0.48，化疗组 r = -0.46）。结论 接受化疗的儿童的 BCVA 较差，尽管治疗阻止了 75% 患者的 BCVA 下降或改善，但没有严重的副作用。[J 小儿眼科斜视。2022;59(2):128-135.]。