N-of-1 Trials in Pediatric Oncology: From a Population-Based
Approach to Personalized Medicine-A Review

J 2021

N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review

KÝR, Michal, Adam SVOBODNÍK, Radka ŠTĚPÁNOVÁ and Renata HEJNOVÁ

Basic information

Original name

N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review

Authors

KÝR, Michal (203 Czech Republic, guarantor, belonging to the institution), Adam SVOBODNÍK (203 Czech Republic, belonging to the institution), Radka ŠTĚPÁNOVÁ (203 Czech Republic, belonging to the institution) and Renata HEJNOVÁ (203 Czech Republic, belonging to the institution)

Edition

Cancers, BASEL, MDPI, 2021, 2072-6694

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30204 Oncology

Country of publisher

Switzerland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 6.575

RIV identification code

RIV/00216224:14110/21:00123627

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.3390/cancers13215428

UT WoS

000718311900001

Keywords (in Czech)

small samples; N-of-1; rare diseases; personalized treatment; pediatric oncology; design; statistical analysis

Keywords in English

small samples; N-of-1; rare diseases; personalized treatment; pediatric oncology; design; statistical analysis

Abstract

V originále

Pediatric oncology is a critical area where the more efficient development of new treatments is urgently needed. The speed of approval of new drugs is still limited by regulatory requirements and a lack of innovative designs appropriate for trials in children. Childhood cancers meet the criteria of rare diseases. Personalized medicine brings it even closer to the horizon of individual cases. Thus, not all the traditional research tools, such as large-scale RCTs, are always suitable or even applicable, mainly due to limited sample sizes. Small samples and traditional versus subject-specific evidence are both distinctive issues in personalized pediatric oncology. Modern analytical approaches and adaptations of the paradigms of evidence are warranted. We have reviewed innovative trial designs and analytical methods developed for small populations, together with individualized approaches, given their applicability to pediatric oncology. We discuss traditional population-based and individualized perspectives of inferences and evidence, and explain the possibilities of using various methods in pediatric personalized oncology. We find that specific derivatives of the original N-of-1 trial design adapted for pediatric personalized oncology may represent an optimal analytical tool for this area of medicine. We conclude that no particular N-of-1 strategy can provide a solution. Rather, a whole range of approaches is needed to satisfy the new inferential and analytical paradigms of modern medicine. We reveal a new view of cancer as continuum model and discuss the “evidence puzzle”.

Links

CZ.02.1.01/0.0/0.0/16_013/0001826, interní kód MU
(CEP code: EF16_013/0001826)

Name: CZECRIN_PRO PACIENTY - zavádění inovativních moderních terapií

Investor: Ministry of Education, Youth and Sports of the CR, Priority axis 1: Strengthening capacities for high-quality research

MUNI/A/1701/2020, interní kód MU

Name: Personalizovaná léčba v dětské onkologii: na cestě k „liquid dynamic medicine“ a „N-of-1 clinical trials“

Investor: Masaryk University

90128, large research infrastructures

Name: CZECRIN III

Citovat

KÝR, Michal, Adam SVOBODNÍK, Radka ŠTĚPÁNOVÁ and Renata HEJNOVÁ. N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review. Cancers. BASEL: MDPI, 2021, vol. 13, No 21, p. 1-18. ISSN 2072-6694. Available from: https://dx.doi.org/10.3390/cancers13215428.

@article{1819140,
   author = {Kýr, Michal and Svobodník, Adam and Štěpánová, Radka and Hejnová, Renata},
   article_location = {BASEL},
   article_number = {21},
   doi = {http://dx.doi.org/10.3390/cancers13215428},
   keywords = {small samples; N-of-1; rare diseases; personalized treatment; pediatric oncology; design; statistical analysis},
   language = {eng},
   issn = {2072-6694},
   journal = {Cancers},
   title = {N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review},
   url = {https://www.mdpi.com/2072-6694/13/21/5428/htm},
   volume = {13},
   year = {2021}
}

TY  - JOUR
ID  - 1819140
AU  - Kýr, Michal - Svobodník, Adam - Štěpánová, Radka - Hejnová, Renata
PY  - 2021
TI  - N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review
JF  - Cancers
VL  - 13
IS  - 21
SP  - 1-18
EP  - 1-18
PB  - MDPI
SN  - 20726694
KW  - small samples
KW  - N-of-1
KW  - rare diseases
KW  - personalized treatment
KW  - pediatric oncology
KW  - design
KW  - statistical analysis
UR  - https://www.mdpi.com/2072-6694/13/21/5428/htm
N2  - Pediatric oncology is a critical area where the more efficient development of new treatments is urgently needed. The speed of approval of new drugs is still limited by regulatory requirements and a lack of innovative designs appropriate for trials in children. Childhood cancers meet the criteria of rare diseases. Personalized medicine brings it even closer to the horizon of individual cases. Thus, not all the traditional research tools, such as large-scale RCTs, are always suitable or even applicable, mainly due to limited sample sizes. Small samples and traditional versus subject-specific evidence are both distinctive issues in personalized pediatric oncology. Modern analytical approaches and adaptations of the paradigms of evidence are warranted. We have reviewed innovative trial designs and analytical methods developed for small populations, together with individualized approaches, given their applicability to pediatric oncology. We discuss traditional population-based and individualized perspectives of inferences and evidence, and explain the possibilities of using various methods in pediatric personalized oncology. We find that specific derivatives of the original N-of-1 trial design adapted for pediatric personalized oncology may represent an optimal analytical tool for this area of medicine. We conclude that no particular N-of-1 strategy can provide a solution. Rather, a whole range of approaches is needed to satisfy the new inferential and analytical paradigms of modern medicine. We reveal a new view of cancer as continuum model and discuss the “evidence puzzle”.
ER  -

KÝR, Michal, Adam SVOBODNÍK, Radka ŠTĚPÁNOVÁ and Renata HEJNOVÁ. N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review. \textit{Cancers}. BASEL: MDPI, 2021, vol.~13, No~21, p.~1-18. ISSN~2072-6694. Available from: https://dx.doi.org/10.3390/cancers13215428.

Detailed Information on Publication Record