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Table 1 Kinase-inhibitors and other targeted agents in pediatric malignancies

From: Precision medicine in pediatric oncology

Genomic alteration Target structure Medication Example pediatric tumor
ALK mutation/fusion ALK Crizotinib Neuroblastoma
Embryonal tumors
MYCN amplification AURKA Alisertib Neuroblastoma
BRAF mutations/fusions BRAF Vemurafenib Melanoma
Dabrafenib Langerhans-cell histiocytosis
glioma
FGFR1/2/3 fusion, amplification, mutation FGFR Dovitinib Rhabdomyosarcoma
Ponatinib Ewing Sarcoma
N/KRAS mutation MEK Trametinib Melanoma
PTPN11 mutation Selumetinib Glioblastoma
Juvenile myelomonocytic leukemia
BRCA1/2 mutations PARP1 Olaparib Osteosarcoma
EWSR1-FLI fusion Rucaparib Ewing sarcoma
ATM mutation
loss of PTEN PI3K/mTOR Everolimus Sarcoma
PIK3CA mutations Temsirolimus
Rapamycin
PTCH1 mutation SMO Vismodegib Medulloblastoma
FLT3 mutation Multikinases Sorafenib Acute myeloid leukemia
or internal tandem duplication
VEGF Receptor Multikinases Pazopanib, Sarcoma
Expression of cKit and PDGF receptor Regorafinib Ewing sarcoma