RT Journal Article
SR Electronic
T1 Exploring how non-inferiority and equivalence are assessed in paediatrics: a systematic review
JF Archives of Disease in Childhood
JO Arch Dis Child
FD BMJ Publishing Group Ltd and Royal College of Paediatrics and Child Health
SP 1067
OP 1075
DO 10.1136/archdischild-2018-314874
VO 103
IS 11
A1 Camille Aupiais
A1 Sarah Zohar
A1 Garry Taverny
A1 Enora Le Roux
A1 Rym Boulkedid
A1 Corinne Alberti
YR 2018
UL http://adc.bmj.com/content/103/11/1067.abstract
AB 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.