TY - JOUR
T1 - The Landscape of Pediatric Precision Oncology
T2 - Program Design, Actionable Alterations, and Clinical Trial Development
AU - Langenberg, Karin P S
AU - Looze, Eleonora J
AU - Molenaar, Jan J
PY - 2021/9/1
Y1 - 2021/9/1
N2 - Over the last years, various precision medicine programs have been developed for pediatric patients with high-risk, relapsed, or refractory malignancies, selecting patients for targeted treatment through comprehensive molecular profiling. In this review, we describe characteristics of these initiatives, demonstrating the feasibility and potential of molecular-driven precision medicine. Actionable events are identified in a significant subset of patients, although comparing results is complicated due to the lack of a standardized definition of actionable alterations and the different molecular profiling strategies used. The first biomarker-driven trials for childhood cancer have been initiated, but until now the effect of precision medicine on clinical outcome has only been reported for a small number of patients, demonstrating clinical benefit in some. Future perspectives include the incorporation of novel approaches such as liquid biopsies and immune monitoring as well as innovative collaborative trial design including combination strategies, and the development of agents specifically targeting aberrations in childhood malignancies.
AB - Over the last years, various precision medicine programs have been developed for pediatric patients with high-risk, relapsed, or refractory malignancies, selecting patients for targeted treatment through comprehensive molecular profiling. In this review, we describe characteristics of these initiatives, demonstrating the feasibility and potential of molecular-driven precision medicine. Actionable events are identified in a significant subset of patients, although comparing results is complicated due to the lack of a standardized definition of actionable alterations and the different molecular profiling strategies used. The first biomarker-driven trials for childhood cancer have been initiated, but until now the effect of precision medicine on clinical outcome has only been reported for a small number of patients, demonstrating clinical benefit in some. Future perspectives include the incorporation of novel approaches such as liquid biopsies and immune monitoring as well as innovative collaborative trial design including combination strategies, and the development of agents specifically targeting aberrations in childhood malignancies.
KW - precision medicine
KW - targeted therapy
KW - next-generation sequencing
U2 - 10.3390/cancers13174324
DO - 10.3390/cancers13174324
M3 - Review article
C2 - 34503139
SN - 2072-6694
VL - 13
SP - 1
EP - 19
JO - Cancers
JF - Cancers
IS - 17
M1 - 4324
ER -
