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

Kód výsledku v IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00159816%3A_____%2F21%3A00074836" target="_blank" >RIV/00159816:_____/21:00074836 - isvavai.cz</a>

Nalezeny alternativní kódy

RIV/65269705:_____/21:00074836 RIV/00216224:14110/21:00123627

Výsledek na webu

<a href="https://www.mdpi.com/2072-6694/13/21/5428" target="_blank" >https://www.mdpi.com/2072-6694/13/21/5428</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.3390/cancers13215428" target="_blank" >10.3390/cancers13215428</a>

Jazyk výsledku

angličtina

Název v původním jazyce

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

Popis výsledku v původním jazyce

Simple Summary: Clinical trials in pediatric oncology and personalized medicine are challenging due to the rarity of the disease, the low prevalence, and the ever-improving treatment outcomes. Many of the methods designed for small numbers and approaches used in classical population studies are not suitable for personalized pediatric oncology. There has been a change of perspective on the whole issue of rare diseases and personalized medicine. For example, a shift from a population to an individual perspective, generalizing from the individual to the population, using repeated measures and a within-subject design instead of parallel groups, exploring the variability instead of suppressing it, etc. N-of-1 should be understood as a whole range of approaches that fit the new inferential, evidential and analytical paradigms of modern medicine.&lt;/p&gt; 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 &quot;evidence puzzle&quot;.&lt;/p&gt;

Název v anglickém jazyce

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

Popis výsledku anglicky

Simple Summary: Clinical trials in pediatric oncology and personalized medicine are challenging due to the rarity of the disease, the low prevalence, and the ever-improving treatment outcomes. Many of the methods designed for small numbers and approaches used in classical population studies are not suitable for personalized pediatric oncology. There has been a change of perspective on the whole issue of rare diseases and personalized medicine. For example, a shift from a population to an individual perspective, generalizing from the individual to the population, using repeated measures and a within-subject design instead of parallel groups, exploring the variability instead of suppressing it, etc. N-of-1 should be understood as a whole range of approaches that fit the new inferential, evidential and analytical paradigms of modern medicine.&lt;/p&gt; 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 &quot;evidence puzzle&quot;.&lt;/p&gt;

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30204 - Oncology

Projekt

Výsledek vznikl pri realizaci vícero projektů. Více informací v záložce Projekty.

Návaznosti

P - Projekt vyzkumu a vyvoje financovany z verejnych zdroju (s odkazem do CEP)<br>I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Rok uplatnění

2021

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

Cancers

ISSN

2072-6694

e-ISSN

—

Svazek periodika

13

Číslo periodika v rámci svazku

21

Stát vydavatele periodika

CH - Švýcarská konfederace

Počet stran výsledku

18

Strana od-do

5428

Kód UT WoS článku

000718311900001

EID výsledku v databázi Scopus

2-s2.0-85117926065