Everolimus to treat aggressive retinal astrocytic hamartoma in tuberous sclerosis complex

Nambi Nallasamy; Michael I Seider; Sridharan Gururangan; Prithvi Mruthyunjaya

doi:10.1016/j.jaapos.2017.04.012

Everolimus to treat aggressive retinal astrocytic hamartoma in tuberous sclerosis complex

J AAPOS. 2017 Aug;21(4):328-331. doi: 10.1016/j.jaapos.2017.04.012. Epub 2017 Jul 18.

Authors

Nambi Nallasamy¹, Michael I Seider², Sridharan Gururangan³, Prithvi Mruthyunjaya⁴

Affiliations

¹ Department of Ophthalmology, Duke University, Durham, North Carolina.
² Permanente Medical Group, San Francisco, California; University of California, San Francisco, California.
³ Preston A. Wells Center for Brain Tumor Therapy, University of Florida, Gainesville, Florida.
⁴ Department of Ophthalmology, Duke University, Durham, North Carolina; Byers Eye Institute at Stanford University, Palo Alto, California. Electronic address: [email protected].

PMID: 28733147
DOI: 10.1016/j.jaapos.2017.04.012

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.

Publication types

Case Reports

MeSH terms

Administration, Oral
Dose-Response Relationship, Drug
Drug Administration Schedule
Everolimus / administration & dosage*
Humans
Immunosuppressive Agents / administration & dosage*
Infant
Male
Retinal Diseases / drug therapy*
TOR Serine-Threonine Kinases / antagonists & inhibitors
Tuberous Sclerosis / drug therapy*

Substances

Immunosuppressive Agents
Everolimus
MTOR protein, human
TOR Serine-Threonine Kinases