Precision medicine in pediatric oncology

Molecular and Cellular Pediatrics

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

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