NF1 is a multisystem genetic disorder characterized primarily by the growth of benign and malignant nervous system tumors. Approximately 1 in 5 NF1 patients develop a tumor in their visual system called an optic pathway glioma, causing many of them to lose their sight. Clinicians typically base their decision on whether to treat a patient with an NF1 optic pathway glioma on changes in visual acuity, which is highly dependent on patient cooperation. This becomes particularly challenging with younger patients, meaning there is a great need for a more reliable, quantitative biomarker for vision loss.
This study is an exploratory aim of the ongoing Children’s Oncology Group Phase III randomized clinical trial ACNS1831 (NCT03871257). This trial compares the treatment response of a new targeted therapy (selumetinib) to the present standard of care (carboplatin and vincristine) in children with newly diagnosed or previously untreated NF1 low-grade gliomas, including NF1 optic pathway gliomas.
The clinical study will collect three sets of OCT measurements for 60 NF1 optic pathway glioma patients enrolled in the Phase III trial: pre-treatment and after 6 and 12 months of receiving treatment. Avery and Fisher will then determine whether OCT measurements can predict treatment response prior to treatment initiation or provide early indication of treatments that are not working.