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

Authors :
Cacchione, Antonella
Lodi, Mariachiara
Carai, Andrea
Miele, Evelina
Tartaglia, Marco
Megaro, Giacomina
Baldo, Giada Del
Alessi, Iside
Colafati, Giovanna Stefania
Carboni, Alessia
Boccuto, Luigi
Camassei, Francesca Diomedi
Catanzaro, Giuseppina
Po, Agnese
Ferretti, Elisabetta
Pedace, Lucia
Pizzi, Simone
Folgiero, Valentina
Pezzullo, Marco
Corsetti, Tiziana
Source :
International Journal of Cancer; May2021, Vol. 148 Issue 10, p2522-2534, 13p
Publication Year :
2021

Abstract

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 [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
00207136
Volume :
148
Issue :
10
Database :
Complementary Index
Journal :
International Journal of Cancer
Publication Type :
Academic Journal
Accession number :
150094628
Full Text :
https://doi.org/10.1002/ijc.33438