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 a Renata HEJNOVÁ

Základní údaje

Originální název

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

Autoři

KÝR, Michal (203 Česká republika, garant, domácí), Adam SVOBODNÍK (203 Česká republika, domácí), Radka ŠTĚPÁNOVÁ (203 Česká republika, domácí) a Renata HEJNOVÁ (203 Česká republika, domácí)

Vydání

Cancers, BASEL, MDPI, 2021, 2072-6694

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30204 Oncology

Stát vydavatele

Švýcarsko

Utajení

není předmětem státního či obchodního tajemství

Odkazy

URL

Impakt faktor

Impact factor: 6.575

Kód RIV

RIV/00216224:14110/21:00123627

Organizační jednotka

Lékařská fakulta

DOI

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

UT WoS

000718311900001

Klíčová slova česky

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

Klíčová slova anglicky

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

Štítky

14110321, 14110516, Excelence Science, FN Brno, MU, RIV, rivok

Příznaky

Recenzováno

Změněno: 8. 2. 2022 09:01, Mgr. Tereza Miškechová

Anotace

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”.

Návaznosti

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

Název: CZECRIN_PRO PACIENTY - zavádění inovativních moderních terapií

Investor: Ministerstvo školství, mládeže a tělovýchovy ČR, CZECRIN_PRO PACIENTY - zavádění inovativních moderních terapií, PO 1 Posilování kapacit pro kvalitní výzkum

MUNI/A/1701/2020, interní kód MU

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

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

90128, velká výzkumná infrastruktura

Název: CZECRIN III

Citovat

KÝR, Michal, Adam SVOBODNÍK, Radka ŠTĚPÁNOVÁ a Renata HEJNOVÁ. N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review. Cancers. BASEL: MDPI, 2021, roč. 13, č. 21, s. 1-18. ISSN 2072-6694. Dostupné z: 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 a Renata HEJNOVÁ. N-of-1 Trials in Pediatric Oncology: From a Population-Based Approach to Personalized Medicine-A Review. \textit{Cancers}. BASEL: MDPI, 2021, roč.~13, č.~21, s.~1-18. ISSN~2072-6694. Dostupné z: https://dx.doi.org/10.3390/cancers13215428.

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