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Expectant Management vs Medication for Patent Ductus Arteriosus in Preterm Infants: The PDA Randomized Clinical Trial

  • Eunice Kennedy Shriver National Institute of Child Health and Human Development Neonatal Research Network
  • University of North Carolina at Chapel Hill
  • RTI International
  • University of New Mexico
  • University of Texas Health Science Center at Houston
  • Brown University
  • University of Alabama at Birmingham
  • Stanford University
  • Ohio State University
  • University of Rochester
  • University of Iowa
  • Duke University
  • Children's Hospital of Philadelphia
  • Case Western Reserve University
  • University of Utah
  • University of Pennsylvania
  • University of Texas Southwestern Medical Center
  • Sharp Healthcare
  • Emory University
  • Good Samaritan Hospital Cincinnati
  • University of Mississippi
  • University of Cincinnati
  • East Carolina University
  • Virtua Health System
  • WakeMed
  • Sanford Health
  • William P. Clements Jr. University Hospital
  • Northwestern University
  • National Institutes of Health
  • Florida Gulf Coast University

Research output: Contribution to journalArticlepeer-review

10 Scopus citations

Abstract

Importance: The management of patent ductus arteriosus (PDA) in preterm infants is controversial. Objective: To determine whether expectant management compared with active treatment of a protocol-defined PDA in preterm infants decreases the incidence of death or bronchopulmonary dysplasia (BPD). Design, Setting, and Participants: A randomized clinical trial including infants born at 22 to 28 weeks' gestation and diagnosed with a protocol-defined PDA between the age of 48 hours and 21 days at screening. The trial was conducted from December 2018 to December 2024 at 33 hospitals within the National Institute of Child Health and Human Development Neonatal Research Network. The final date of follow-up was June 2025. Interventions: Infants with PDA were randomized to expectant management (n = 242) or active treatment (n = 240; acetaminophen, ibuprofen, or indomethacin) to close the PDA. Main Outcomes and Measures: The primary outcome was death or BPD at 36 weeks' postmenstrual age. The secondary outcomes included the components of the primary outcome and other morbidities of prematurity. Results: A total of 482 infants were randomized (median gestational age, 25 weeks [IQR, 24 to 27 weeks]; median birth weight, 760 g [IQR, 620 to 935 g]). The trial was stopped for futility and safety after the 50% interim analysis for the primary outcome due to higher survival in the expectant management group. The incidence of death or BPD was 80.9% (195/241) of infants in the expectant management group vs 79.6% (191/240) of infants in the active treatment group (adjusted risk difference, 1.2% [95% CI, -5.7% to 8.1%]; P =.73). The incidence of death before 36 weeks' postmenstrual age was 4.1% (10/241) of infants in the expectant management group vs 9.6% (23/240) of infants in the active treatment group (adjusted risk difference, -5.6% [95% CI, -10.1% to -1.2%]; P =.01). Infections resulting in death occurred in 0.8% (2/241) of infants in the expectant management group vs 3.8% (9/240) of infants in the active treatment group. Conclusions and Relevance: In extremely preterm infants with a protocol-defined PDA, death or BPD did not differ between the expectant management group and the active treatment group. Survival was substantially higher with expectant management. Trial Registration: ClinicalTrials.gov

Original languageEnglish
JournalJAMA
DOIs
StateAccepted/In press - 2025

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