Abstract 214
Background: The primary outcome and the minimum detectable treatment difference (MDTD) in RCTs strongly affect sample size and the clinical utility of the study results. Aim: To perform a systematic review of primary outcomes in recent neonatal RCTs. Methods: Medline was searched for all neonatal RCTs, published since 1993 in six high-profile journals. Results: 129 reports were eligible for review. A primary outcome could not be identified in 32 (25%) reports. A continuous (i.e. surrogate) measure was the primary outcome in 48 (37%) trials. A binary outcome was short-term in 42 (33%) trials, and long-term in 7 (5%) trials. The median (minmax) MDTD was 50% (19-89%) in short-term trials, and 40% (30-67%) in long-term trials with primary binary outcomes. Conclusions: Most authors of recently published neonatal RCTs failed to specify a primary endpoint, or chose a continuous measure (e.g. oxygenation index) rather than a clinically important outcome event (e.g. death). Trials with a primary binary outcome tend to have a short duration of follow-up and unrealistically large minimum detectable treatment differences.
Author information
Authors and Affiliations
Rights and permissions
About this article
Cite this article
Schmidt, B., Zhang, B. Do we Measure the Right Endpoints in Neonatal Randomized Clinical Trials (RCTs)?. Pediatr Res 45, 922 (1999). https://doi.org/10.1203/00006450-199906000-00232
Issue Date:
DOI: https://doi.org/10.1203/00006450-199906000-00232