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@ARTICLE{Schubert:180249,
author = {N. A. Schubert and C. Y. Chen and A. Rodríguez and J.
Koster and M. Dowless and S. M. Pfister$^*$ and D. J.
Shields and L. F. Stancato and G. Vassal and H. N. Caron and
M. L. van den Boogaard and A. G. Henssen$^*$ and J. J.
Molenaar},
title = {{T}arget actionability review to evaluate {CDK}4/6 as a
therapeutic target in paediatric solid and brain tumours.},
journal = {European journal of cancer},
volume = {170},
issn = {0014-2964},
address = {Amsterdam [u.a.]},
publisher = {Elsevier},
reportid = {DKFZ-2022-01200},
pages = {196 - 208},
year = {2022},
abstract = {Childhood cancer is still a leading cause of death around
the world. To improve outcomes, there is an urgent need for
tailored treatment. The systematic evaluation of existing
preclinical data can provide an overview of what is known
and identify gaps in the current knowledge. Here, we applied
the target actionability review (TAR) methodology to assess
the strength and weaknesses of available scientific
literature on CDK4/6 as a therapeutic target in paediatric
solid and brain tumours by structured critical
appraisal.Using relevant search terms in PubMed, a list of
original publications investigating CDK4/6 in paediatric
solid tumour types was identified based on relevancy
criteria. Each publication was annotated for the tumour type
and categorised into separate proof-of-concept (PoC) data
modules. Based on rubrics, quality and experimental outcomes
were scored independently by two reviewers. A third reviewer
evaluated and adjudicated score discrepancies. Scores for
each PoC module were averaged for each tumour type and
visualised in a heatmap matrix in the publicly available R2
data portal.This CDK4/6 TAR, generated by analysis of 151
data entries from 71 publications, showed frequent genomic
aberrations of CDK4/6 in rhabdomyosarcoma, osteosarcoma,
high-grade glioma, medulloblastoma, and neuroblastoma.
However, a clear correlation between CDK4/6 aberrations and
compound efficacy is not coming forth from the literature.
Our analysis indicates that several paediatric indications
would need (further) preclinical evaluation to allow for
better recommendations, especially regarding the dependence
of tumours on CDK4/6, predictive biomarkers, resistance
mechanisms, and combination strategies. Nevertheless, our
TAR heatmap provides support for the relevance of CDK4/6
inhibition in Ewing sarcoma, medulloblastoma, malignant
peripheral nerve sheath tumour and to a lesser extent
neuroblastoma, rhabdomyosarcoma, rhabdoid tumour and
high-grade glioma. The interactive heatmap is accessible
through R2 $[r2platform.com/TAR/CDK4_6].$},
keywords = {CDK4/6 (Other) / Cell cycle inhibitors (Other) / Paediatric
oncology (Other) / Preclinical research (Other) / Systematic
review (Other) / Targeted therapy (Other)},
cin = {B062 / HD01 / BE01},
ddc = {610},
cid = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331 /
I:(DE-He78)BE01-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:35671543},
doi = {10.1016/j.ejca.2022.04.028},
url = {https://inrepo02.dkfz.de/record/180249},
}