Framework for a Study of the Treatment of BPD in Newborns

Type of studyPlacebo-controlled, randomized clinical trial with no crossover so that long-term outcomes can be assessed
Entry criteriaThe entry criteria would differ according to the phase of disease studied; generally, entry criteria would focus on infants of <32 weeks' gestational age; in addition to disease, entry criteria would focus on severity and potentially would have more strict criteria for drugs such as corticosteroids that have a higher potential for harm
Exclusion criteriaExclusion criteria would include
    Newborns with major anomalies, including pulmonary, cardiac (excluding patent ductus arteriosus), airway, genetic, and lethal anomalies
    Confirmed sepsis
    Inability to comply with follow-up
    Infants expected to die within 72 h of the enrollment window
Exit criteriaTo minimize and discourage the use of open-label drugs, the pulmonary group supported stringent predefined criteria for failure
Duration of outcome assessmentThe work group supported the following parameters for outcome assessment
    Longitudinal evaluations at a minimum of 2 y of age
    Ideally, longer-term assessments at 8–10 y of age, when cognition and lung function can be assessed more accurately, would be included, particularly if the study involved steroids
    Duration would depend on drug class studied (eg, shorter for diuretics than corticosteroids)