Upfront treatment with mTOR inhibitor everolimus in pediatric low-grade gliomas: A single-center experience

Antonella Cacchione, Mariachiara Lodi, Andrea Carai, Evelina Miele, Marco Tartaglia, Giacomina Megaro, Giada Del Baldo, Iside Alessi, Giovanna Stefania Colafati, Alessia Carboni, Luigi Boccuto, Francesca Diomedi Camassei, Giuseppina Catanzaro, Agnese Po, Elisabetta Ferretti, Lucia Pedace, Simone Pizzi, Valentina Folgiero, Marco Pezzullo, Tiziana CorsettiDomitilla Elena Secco, Maria Giuseppina Cefalo, Franco Locatelli, Angela Mastronuzzi

Risultato della ricerca: Contributo in rivistaArticolo in rivista


Pediatric low-grade gliomas (pLGGs) are the most frequent brain tumor in children. Adjuvant treatment, consisting in chemotherapy and radiotherapy, is often necessary if a complete surgical resection cannot be obtained. Traditional treatment approaches result in a significant long-term morbidity, with a detrimental impact on quality of life. Dysregulation of the mitogen-activated protein kinase (MAPK) pathway is the molecular hallmark of pLGGs and hyperactivation of the downstream mammalian target of rapamycin (mTOR) pathway is frequently observed. We report clinical and radiological results of front-line treatment with everolimus in 10 consecutive patients diagnosed with m-TOR positive pLGGs at the Bambino Gesù Children's Hospital in Rome, Italy. Median duration of treatment was 19 months (range from 13-60). Brain magnetic resonance imaging showed stable disease in 7 patients, partial response in 1 and disease progression in 2. Therapy-related adverse events were always reversible after dose reduction or temporary treatment interruption. To the best of our knowledge, this is the first report of everolimus treatment for chemo- and radiotherapy-naïve children with pLGG. Our results provide preliminary support, despite low sample size, for the use of everolimus as target therapy in pLGG showing lack of progression with a manageable toxicity profile.
Lingua originaleEnglish
pagine (da-a)2522-2534
Numero di pagine13
RivistaInternational Journal of Cancer
Stato di pubblicazionePubblicato - 2021


  • everolimus
  • pediatric low-grade gliomas
  • mTOR
  • MAPK pathway


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