Pediatric low-grade glioma: Targeted therapeutics and clinical trials in the molecular era

•Pediatric low-grade glioma (pLGG) is a MAPK pathway-altered disease with novel therapeutic options.•Clinical trials are vital in translation of targeted agents to influence patient care.•Targeted agents have unknown long-term toxicities and unclear impact on pLGG natural history.•Challenges include...

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Vydané v:Neoplasia (New York, N.Y.) Ročník 36; s. 100857
Hlavní autori: Manoharan, Neevika, Liu, Kevin X., Mueller, Sabine, Haas-Kogan, Daphne A., Bandopadhayay, Pratiti
Médium: Journal Article
Jazyk:English
Vydavateľské údaje: United States Elsevier Inc 01.02.2023
Neoplasia Press
Elsevier
Predmet:
BRAF
Brain Neoplasms - drug therapy
Brain Neoplasms - genetics
Child
Clinical Trials as Topic
Glioma - diagnosis
Glioma - drug therapy
Glioma - genetics
Humans
MAPK
mTOR
Original Research
pLGG
Targeted therapy
WES
Targeted therapy
IHC
pLGG
RTK
TPCV
CNS
GNT
mTOR
DNET
PFS
PLNTY
WHO
RAPNO
OS
TSC
BRAF
MAPK
FGFR
DNA
PNOC
FISH
NF1
JPA
CPK
ISSN:1476-5586, 1522-8002, 1476-5586
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Popis
Shrnutí:•Pediatric low-grade glioma (pLGG) is a MAPK pathway-altered disease with novel therapeutic options.•Clinical trials are vital in translation of targeted agents to influence patient care.•Targeted agents have unknown long-term toxicities and unclear impact on pLGG natural history.•Challenges include rapid clinical trial translation, tumor heterogeneity and need to harmonize molecular profiling techniques. pLGGs are a group of tumors for which the era of molecular diagnostics has truly shifted treatment paradigms and patient care. The discovery that this group of tumors is driven by single-gene alterations/fusions in the MAPK pathway has resulted in relatively rapid translation into targeted therapy options for patients with this often chronic disease. This translation has been facilitated through efforts of multiple collaboratives and consortia and has led to the development of clinical trials testing the role of targeted therapies in pLGG. Although these developments represent promise, many questions remain regarding these therapies including their long-term toxicities and their potential effects on the natural history of pLGG.
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SourceType-Scholarly Journals-1
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These authors contributed equally to this work.
ISSN:1476-5586
1522-8002
1476-5586
DOI:10.1016/j.neo.2022.100857