Exploring how non-inferiority and equivalence are assessed in paediatrics: a systematic review.
Arch Dis Child
; 103(11): 1067-1075, 2018 11.
Article
em En
| MEDLINE
| ID: mdl-29794107
ABSTRACT
OBJECTIVE:
To review characteristics, methodology and reporting of non-inferiority and equivalence trials in the specific context of paediatrics.DESIGN:
PubMed and Cochrane databases were searched (up to September 2016) for non-inferiority/equivalence randomised controlled trials conducted in children published in high-impact-factor journals (>5.0 for general/specialist medical journals; >2.2 for paediatric journals).RESULTS:
We found that the statistical hypothesis was inconsistent with the objective in 12 (10%) of the 125 reports included. Non-inferiority (n=98) and equivalence trials (n=27) were mostly used to evaluate interventions with easier administration (45%, n=54/120) and/or better safety profile (34%, n=41/120). All the data needed for targeted sample size recalculation were available for 39 reports (31%). The margin-representing the largest difference between arms that would be clinically acceptable-was reported in 119 (95%), and 44/119 (37%) reported the method used for margin determination. The median sample size was 268 (IQR 125-531). Margins were wider in smaller trials (<125 randomised patients) than in larger trials (p=0.04/p<0.01 for binary/continuous outcomes, respectively). We did not agree with the authors' conclusions in 11% (11/103) of the reports that provided sufficient information.CONCLUSIONS:
There is still a need to improve the quality of methodology, reporting and interpretation of non-inferiority/equivalence trials in paediatrics. In particular, the margins were often not justified and the conclusion was often not supported by the design and/or the results. As researchers have to cope with small sample size and with lack of evidence, methods for non-inferiority/equivalence trials need to be used and/or developed in this vulnerable population.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Pediatria
/
Projetos de Pesquisa
/
Ensaios Clínicos Controlados Aleatórios como Assunto
/
Fidelidade a Diretrizes
/
Estudos de Equivalência como Asunto
Tipo de estudo:
Clinical_trials
/
Systematic_reviews
Limite:
Child
/
Humans
Idioma:
En
Revista:
Arch Dis Child
Ano de publicação:
2018
Tipo de documento:
Article
País de afiliação:
França