title: Everolimus (RAD001): first systemic treatment for subependymal giant cell astrocytoma associated with tuberous sclerosis complex.
abstract: Everolimus (RAD001), a mTOR inhibitor, was initially used as an immunosuppressant in organ transplant patients; however, it also has significant antineoplastic properties. In patients with subependymal giant cell astrocytomas (SEGAs) associated with tuberous sclerosis complex who are not candidates for surgery, single-agent everolimus has demonstrated the ability to significantly reduce SEGA volume with good tolerability. In the Phase III, randomized, placebo-controlled trial, everolimus was associated with a SEGA response rate of 35% compared with 0% in the placebo group. The most common adverse events in clinical trials were stomatitis/mouth ulceration and upper respiratory tract infections, and most adverse events were grade 1 or 2; grade 4 events were rare.
authors.affiliation: Department of Neurology & Epileptology, The Children's Memorial Health Institute, 04-730, Warsaw, Poland. sergiusz.jozwiak@gmail.com
authors.forename: Sergiusz
authors.initials: S
authors.lastname: Józwiak
authors.forename: Karen
authors.initials: K
authors.lastname: Stein
authors.forename: Katarzyna
authors.initials: K
authors.lastname: Kotulska
mesh_terms: D000818:Animals; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000493:pharmacokinetics / Q000627:therapeutic use*; D001254:Astrocytoma / Q000150:complications / Q000188:drug therapy*; D002986:Clinical Trials as Topic; D000068338:Everolimus; D006801:Humans; D019226:Oral Ulcer / Q000139:chemically induced; D020123:Sirolimus / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000737:chemistry / Q000493:pharmacokinetics / Q000494:pharmacology / Q000627:therapeutic use; D013280:Stomatitis / Q000139:chemically induced; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors; D014402:Tuberous Sclerosis / Q000188:drug therapy* / Q000209:etiology
chemical_list: D000970:Antineoplastic Agents; D000068338:Everolimus; C546842:MTOR protein, human; D058570:TOR Serine-Threonine Kinases; D020123:Sirolimus