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Do sick newborn infants benefit from participation in a randomized clinical trial?
1Department of Pediatrics, McMaster University, Hamilton, Ontario, Canada.
Insights
Newborn infants in a clinical trial experienced shorter mechanical ventilation durations compared to eligible infants not enrolled. This suggests that participation in randomized controlled trials may benefit critically ill newborns.
Area of Science:
- Neonatal Medicine
- Clinical Trial Design
- Pediatric Critical Care
Background:
- Adults in clinical trials often show improved outcomes compared to those eligible but not enrolled.
- This study investigates if this phenomenon extends to newborn infants.
Purpose of the Study:
- To determine if newborn infants randomized to a placebo in an investigational drug trial had better outcomes than infants eligible but not randomized (eligible NR).
Main Methods:
- Prospective data collection on 76 eligible NR infants in a randomized controlled trial (RCT) of antithrombin therapy for respiratory distress syndrome.
- Comparison of outcomes between eligible NR infants and 61 infants randomized to placebo.
- Standardized use of exogenous surfactant in all infants.
Main Results:
- Infants in the placebo group had a median mechanical ventilation duration of 4.8 days, reduced from 6.2 days in the eligible NR group (P =.008).
- A trend towards less frequent and severe intraventricular hemorrhage was observed in trial participants.
- Demographic characteristics (birth weight, gestational age, sex) were similar between groups.
Conclusions:
- The findings support the hypothesis that sick newborn infants may benefit from participating in randomized controlled trials.
- Clinical trial participation may lead to improved outcomes in neonatal intensive care settings.
Background:
Adult participants in randomized controlled trials often have better outcomes than patients who are eligible but not enrolled.
Objective:
To examine whether newborn infants who were allocated to placebo in an investigational drug trial had better outcomes than infants who were eligible but not randomized (eligible NR).
Study Design:
During a randomized controlled trial of antithrombin therapy in premature infants with respiratory distress syndrome, data were collected prospectively on all 76 infants in the eligible NR group. Study outcomes were compared with those of all 61 infants who were randomized to placebo. The same exogenous surfactant was used in all patients.
Results:
In the placebo group the mean (SD) birth weight was 1201 (314) g, mean (SD) gestational age was 28.8 (2.3) weeks, and 51% were male. In infants in the eligible NR group, mean (SD) birth weight was 1141 (262) g, mean (SD) gestational age was 28.3 (2. 3) weeks, and 58% were male; 57% of infants in both groups had been exposed to steroids before birth. The median duration of mechanical ventilation was reduced from 6.2 days in the eligible NR group to 4. 8 days in the placebo group (P =.008). There was also a trend toward less frequent and less severe intraventricular hemorrhage in trial participants.
Conclusions:
These data are consistent with the hypothesis that sick newborn infants may benefit from participation in a randomized controlled trial.