. Author manuscript; available in PMC: 2012 Mar 1.

Published in final edited form as: Nat Rev Clin Oncol. 2011 Jun 28;8(9):540–549. doi: 10.1038/nrclinonc.2011.95

Table 2.

Selected challenging subtypes of childhood cancer

Targets altered and disease subtype	Frequency (%)	5-year event-free survival (%)	Potential therapy
Early T-cell precursor ALL^4,8,12	1.5	20	Nelarabine; allogenic transplantation
MLL-rearranged ALL in infants^4,17,18	2	30	FLT-3 inhibitors (lestaurtinib, midostaurin); DNA methyltransferase inhibitors (decitabine)
AML with internal tandem duplication of FLT3^4,28–30	10–15	<35	FLT-3 inhibitors; allogenic transplantation
AML with monosomy 7, 5q or t(6;9)³	3	<40	Immunotherapy; cellular therapy
Large cell and/or aplastic medulloblastoma with MYC amplification⁴⁰	10–15	<40	To be determined
Notch pathway-driven ependymoma⁴⁶	35	<20	Notch inhibitors
Diffuse intrinsic pontine glioma with PDGFR amplification⁵³	40	<5	PDGFR antagonists; PARP inhibitors
Metastatic rhabdomyosarcoma^71–76	16	<30	MET inhibitors; IGF-1R and mTOR inhibitors; FGFR-4 inhibitors; hedgehog pathway inhibitors; glycogen synthase kinase 3 (GSK-3) inhibitor; PDGFR antagonists
Gastrointestinal stromal tumor⁸⁰	<1	<20	IGF-1R inhibitors
High-risk neuroblastoma (MYCN amplification, 11q⁻ , 17q⁺, 1p⁻)⁶⁴	45	~50	ALK inhibitors; I¹³¹ or I¹²³ metaiodobenzylguanidine; aurora kinase inhibitors
Metastatic Ewing sarcoma family of tumors⁸⁵	25	<25	IGF-1R inhibitors and mTOR inhibitors

Abbreviations: ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; FGFR, fibroblast growth factor receptor; FLT, fms-related tyrosine kinase; IGF-1R, insulin-like growth factor receptor; mTOR, mammalian target of rapamycin; PARP, poly(ADP) ribose polymerase.