title: [Response to everolimus in patients with giant cell astrocytoma associated to tuberous sclerosis complex].
abstract: BACKGROUND
  Subependymal giant cell astrocytomas (SEGA) appear in 5-20% of patients with tuberous sclerosis complex (TSC) and are the most common brain tumours in TSC. They are benign tumours, of a glioneural stock, that develop mainly in the first two decades of life, generally close to the foramen of Monro, and can trigger hydrocephalus and intracranial hypertension. It is one of the leading causes of death in TSC. Recently mTOR inhibitors have proved to be a therapeutic alternative to surgical excision. AIM. To describe our experience of using everolimus to treat patients with SEGA and TSC.
  
  
  METHODS
  We conducted a prospective study of the responses of patients with TSC and at least one SEGA undergoing growth.
  
  
  RESULTS
  Three females and three males with a mean age of 12.3 years received treatment. One patient had previously undergone surgery due to SEGA with hydrocephalus. The maximum mean diameter of the SEGA on beginning treatment was 15.3 mm (range: 11.3-24.8 mm). Treatment was established with everolimus, 2.5 mg/day administered orally in patients with a body surface area < 1.2 m2, and 5 mg/day in patients with a body surface area > 1.2 m2. Two patients presented hypertriglyceridemia; one, anorexia; another, a mouth ulcer; and one, amenorrhoea. The mean reduction in the volume of the SEGA at three months of treatment was 46%, and the reduction remained steady in later control examinations (6-25 months).
  
  
  CONCLUSIONS
  Treatment with everolimus reduces the size of SEGA associated with TSC with an adequate safety profile, and constitutes an alternative to surgery in certain cases.
authors.forename: M Elena
authors.initials: ME
authors.lastname: Mateos-González
authors.affiliation: Hospital Universitario Reina Sofia. IMIBIC. CIBERER-ISCIII. , Cordoba, Espana.
authors.forename: Eduardo
authors.initials: E
authors.lastname: López-Laso
authors.forename: Josefina
authors.initials: J
authors.lastname: Vicente-Rueda
authors.forename: Rafael
authors.initials: R
authors.lastname: Camino-León
authors.forename: Joaquín A
authors.initials: JA
authors.lastname: Fernández-Ramos
authors.forename: M Auxiliadora
authors.initials: MA
authors.lastname: Baena-Gómez
authors.forename: M José
authors.initials: MJ
authors.lastname: Peña-Rosa
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000568:Amenorrhea / Q000139:chemically induced; D000855:Anorexia / Q000139:chemically induced; D001254:Astrocytoma / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D002648:Child; D000068338:Everolimus; D005260:Female; D015726:Giant Cells / Q000473:pathology; D006801:Humans; D015228:Hypertriglyceridemia / Q000139:chemically induced; D008297:Male; D009363:Neoplasm Proteins / Q000037:antagonists & inhibitors; D011446:Prospective Studies; D020123:Sirolimus / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D013281:Stomatitis, Aphthous / Q000139:chemically induced; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors* / Q000378:metabolism; D016896:Treatment Outcome; D014402:Tuberous Sclerosis / Q000150:complications* / Q000235:genetics; D000077005:Tuberous Sclerosis Complex 2 Protein; D047368:Tumor Burden / Q000187:drug effects; D025521:Tumor Suppressor Proteins / Q000235:genetics / Q000378:metabolism
chemical_list: D009363:Neoplasm Proteins; D000077005:Tuberous Sclerosis Complex 2 Protein; D025521:Tumor Suppressor Proteins; D000068338:Everolimus; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases; D020123:Sirolimus