Individualization of Treatment Improves the Survival of Children With High-Risk Solid Tumors: ...

@article{1623956,
   author = {Kýr, Michal and Polášková, Kristýna and Kuttnerová, Zuzana and Merta, Tomáš and Neradil, Jakub and Berkovcová, Jitka and Horký, Ondřej and Ježová, Marta and Veselská, Renata and Klement, Giannoula Lakka and Valík, Dalibor and Štěrba, Jaroslav},
   article_location = {Lausanne},
   article_number = {JUL 17 2019},
   doi = {http://dx.doi.org/10.3389/fonc.2019.00644},
   keywords = {cancer; children; personalized medicine; targeted therapy; comparative effectiveness research; clinical trials; metronomic},
   language = {eng},
   issn = {2234-943X},
   journal = {Frontiers in Oncology},
   title = {Individualization of Treatment Improves the Survival of Children With High-Risk Solid Tumors: Comparative Patient Series Analysis in a Real-Life Scenario},
   url = {http://dx.doi.org/10.3389/fonc.2019.00644},
   volume = {9},
   year = {2019}
}

TY  - JOUR
ID  - 1623956
AU  - Kýr, Michal - Polášková, Kristýna - Kuttnerová, Zuzana - Merta, Tomáš - Neradil, Jakub - Berkovcová, Jitka - Horký, Ondřej - Ježová, Marta - Veselská, Renata - Klement, Giannoula Lakka - Valík, Dalibor - Štěrba, Jaroslav
PY  - 2019
TI  - Individualization of Treatment Improves the Survival of Children With High-Risk Solid Tumors: Comparative Patient Series Analysis in a Real-Life Scenario
JF  - Frontiers in Oncology
VL  - 9
IS  - JUL 17 2019
SP  - 1-11
EP  - 1-11
PB  - Frontiers Media S.A.
SN  - 2234943X
KW  - cancer
KW  - children
KW  - personalized medicine
KW  - targeted therapy
KW  - comparative effectiveness research
KW  - clinical trials
KW  - metronomic
UR  - http://dx.doi.org/10.3389/fonc.2019.00644
L2  - http://dx.doi.org/10.3389/fonc.2019.00644
N2  - Introduction: The individualization of treatment is attractive, especially in children with high-risk cancer. In such a rare and very heterogeneous group of diseases, large population-based clinical randomized trials are not feasible without international collaboration. We therefore propose comparative patient series analysis in a real-life scenario. Methods: Open cohort observational study, comparative analysis. Seventy patients with high-risk solid tumors diagnosed between 2003 and 2015 and in whom the treatment was individualized either empirically or based on biomarkers were analyzed. The heterogeneity of the cohort and repeated measurements were advantageously utilized to increase effective sample size using appropriate statistical tools. Results: We demonstrated a beneficial effect of empirically given low-dose metronomic chemotherapy (HR 0.46 for relapses, p = 0.017) as well as various repurposed or targeted agents (HR 0.15 for deaths, p = 0.004) in a real-life scenario. However, targeted agents given on the basis of limited biological information were not beneficial. Conclusions: Comparative patient series analysis provides institutional-level evidence for treatment individualization in high-risk pediatric malignancies. Our findings emphasize the need for a comprehensive, multi omics assessment of the tumor and the host as well whenever molecularly driven targeted therapies are being considered. Low-dose metronomic chemotherapy or local control of the disease may be a more rational option in situations where targeted treatment cannot be justified by robust evidence and comprehensive biological information. "Targeted drugs" may be given empirically with a realistic benefit expectation when based on robust rationale.
ER  - 

KÝR, Michal, Kristýna POLÁŠKOVÁ, Zuzana KUTTNEROVÁ, Tomáš MERTA, Jakub NERADIL, Jitka BERKOVCOVÁ, Ondřej HORKÝ, Marta JEŽOVÁ, Renata VESELSKÁ, Giannoula Lakka KLEMENT, Dalibor VALÍK a Jaroslav ŠTĚRBA. Individualization of Treatment Improves the Survival of Children With High-Risk Solid Tumors: Comparative Patient Series Analysis in a Real-Life Scenario. \textit{Frontiers in Oncology}. Lausanne: Frontiers Media S.A., 2019, roč.~9, JUL 17 2019, s.~1-11. ISSN~2234-943X. Dostupné z: https://dx.doi.org/10.3389/fonc.2019.00644.