Everolimus to treat aggressive retinal astrocytic hamartoma in tuberous sclerosis complex. Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus Nallasamy, N. n., Seider, M. I., Gururangan, S. n., Mruthyunjaya, P. n. 2017; 21 (4): 328–31


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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