title: Everolimus Treatment for an Early Infantile Subependymal Giant Cell Astrocytoma With Tuberous Sclerosis Complex.
abstract: Subependymal giant cell astrocytomas are benign tumors often observed with tuberous sclerosis complex. These tumors are rarely diagnosed during fetal life or early infancy. Until recently, the only available treatment has been surgical resection. Current clinical research has demonstrated that everolimus can induce these tumors' regression. We report a 19-month-old boy with tuberous sclerosis complex. At 2 months of age, he presented with congenital subependymal giant cell astrocytoma that was complicated by refractory epilepsy and severe mental retardation. Treatment with everolimus was started when he was 10 months old. Three months after initiating everolimus, the tumor was significantly reduced in size, and the reduction was subsequently maintained. His seizures decreased and he showed cognitive and developmental improvement. No severe adverse events have been observed to date. Everolimus has promise as an effective alternative to surgery for subependymal giant cell astrocytomas during early infancy.
authors.affiliation: The Department of Child Neurology, Hokkaido Medical Center for Child Health and Rehabilitation, Teine-Ku, Sapporo, Japan Department of Pediatrics, Sapporo Medical University School of Medicine, Chuo-ku, Sapporo, Japan fukumura@sapmed.ac.jp.
authors.forename: Shinobu
authors.initials: S
authors.lastname: Fukumura
authors.affiliation: The Department of Child Neurology, Hokkaido Medical Center for Child Health and Rehabilitation, Teine-Ku, Sapporo, Japan Department of Pediatrics, Sapporo Medical University School of Medicine, Chuo-ku, Sapporo, Japan.
authors.forename: Toshihide
authors.initials: T
authors.lastname: Watanabe
authors.affiliation: The Department of Child Neurology, Hokkaido Medical Center for Child Health and Rehabilitation, Teine-Ku, Sapporo, Japan Department of Pediatrics, Sapporo Medical University School of Medicine, Chuo-ku, Sapporo, Japan.
authors.forename: Rumiko
authors.initials: R
authors.lastname: Takayama
authors.affiliation: The Department of Child Neurology, Hokkaido Medical Center for Child Health and Rehabilitation, Teine-Ku, Sapporo, Japan Department of Pediatrics, Sapporo Medical University School of Medicine, Chuo-ku, Sapporo, Japan.
authors.forename: Kimio
authors.initials: K
authors.lastname: Minagawa
authors.affiliation: Department of Pediatrics, Sapporo Medical University School of Medicine, Chuo-ku, Sapporo, Japan.
authors.forename: Hiroyuki
authors.initials: H
authors.lastname: Tsutsumi
mesh_terms: D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001254:Astrocytoma / Q000150:complications / Q000188:drug therapy*; D001932:Brain Neoplasms / Q000150:complications / Q000188:drug therapy*; D000068338:Everolimus / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D008279:Magnetic Resonance Imaging; D008297:Male; D014402:Tuberous Sclerosis / Q000150:complications / Q000188:drug therapy*
chemical_list: D000970:Antineoplastic Agents; D000068338:Everolimus
keywords: early infancy; everolimus; mammalian target of rapamycin; subependymal giant cell astrocytomas; tuberous sclerosis complex