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Updated: Feb 26, 2026

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Everolimus to treat aggressive retinal astrocytic hamartoma in tuberous sclerosis complex
Nambi Nallasamy1, Michael I Seider2, Sridharan Gururangan3
1Department of Ophthalmology, Duke University, Durham, North Carolina.
Abstract:
Retinal astrocytic hamartomas (RAH) are the most frequent ocular manifestation of tuberous sclerosis complex and are usually indolent, requiring only observation. We report an aggressive RAH subtype in a child unresponsive to anti-VEGF and laser therapy. Treatment with systemic everolimus was well-tolerated and significantly reduced ocular (and nonocular) tumor size and fluid exudation.
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