TY - JOUR
T1 - Upfront treatment with mTOR inhibitor everolimus in pediatric low-grade gliomas: A single-center experience
AU - Cacchione, Antonella
AU - Lodi, Mariachiara
AU - Carai, Andrea
AU - Miele, Evelina
AU - Tartaglia, Marco
AU - Megaro, Giacomina
AU - Del Baldo, Giada
AU - Alessi, Iside
AU - Colafati, Giovanna Stefania
AU - Carboni, Alessia
AU - Boccuto, Luigi
AU - Diomedi Camassei, Francesca
AU - Catanzaro, Giuseppina
AU - Po, Agnese
AU - Ferretti, Elisabetta
AU - Pedace, Lucia
AU - Pizzi, Simone
AU - Folgiero, Valentina
AU - Pezzullo, Marco
AU - Corsetti, Tiziana
AU - Secco, Domitilla Elena
AU - Cefalo, Maria Giuseppina
AU - Locatelli, Franco
AU - Mastronuzzi, Angela
PY - 2021
Y1 - 2021
N2 - 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.
AB - 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.
KW - everolimus
KW - pediatric low-grade gliomas
KW - mTOR
KW - MAPK pathway
KW - everolimus
KW - pediatric low-grade gliomas
KW - mTOR
KW - MAPK pathway
UR - http://hdl.handle.net/10807/228463
U2 - 10.1002/ijc.33438
DO - 10.1002/ijc.33438
M3 - Article
SN - 0020-7136
VL - 148
SP - 2522
EP - 2534
JO - International Journal of Cancer
JF - International Journal of Cancer
ER -