ABSTRACT
A recent phase 1/2 study demonstrated that treatment with the mammalian target of rapamycin inhibitor everolimus reduced subependymal giant cell astrocytoma volume by 30% in 75% of the patients, all of whom were poor candidates for surgical resection. Of the enrolled patients, 4 had had previous surgery to remove subependymal giant cell astrocytoma, and the outcomes for these patients were retrospectively analyzed and are presented here. All 4 experienced over 50% initial reduction in the volume of their subependymal giant cell astrocytoma after 2 to 3 years of therapy with everolimus. Although the volume of 1 patient's subependymal giant cell astrocytoma returned to baseline volume 36 months after initiating everolimus, they have remained asymptomatic with no recurrent hydrocephalus. Further surgery has been avoided in all cases to date. This course of treatment offers a new and welcome option for these difficult-to-treat patients.
