Background Since the introduction of randomised controlled trials (RCT) in clinical research, there has been discussion of whether enrolled patients have worse or better outcomes than comparable nonparticipants.
Objective To investigate whether very preterm infants randomised to a placebo group in a RCT have equivalent neurodevelopmental outcomes to infants who were eligible but not randomised (eligible NR).
Methods In the course of an RCT investigating the neuroprotective effect of early high dose erythropoietin on the neurodevelopment of very preterm infants, the outcome data of 72 infants randomised to placebo were compared with those of 108 eligible NR infants. Our primary outcome measures were the mental (MDI) and psychomotor (PDI) developmental indices of the Bayley Scales of Infant Development II at 24 months corrected age. The outcomes of the two groups were considered equivalent if the confidence intervals of their mean differences fitted within our ± 5 point margin of equivalence.
Results Except for a higher socioeconomic status of the trial participants, both groups were balanced for most perinatal variables. The mean difference (90% CI) between the placebo and the eligible NR group was -2.1 (-6.1 and 1.9) points for the MDI and -0.8 (-4.2 and 2.5) points for the PDI (in favour of the placebo group). After adjusting for the socioeconomic status, maternal age and child age at follow-up, the mean difference for the MDI was -0.5 (-4.3 and 3.4) points.
Conclusions Our results indicate that the participation of very preterm infants in an RCT is associated with equivalent long-term outcomes compared to non-participating infants.