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A Pilot Randomized Controlled Trial of Early Targeted Patent Ductus Arteriosus Treatment Using a Risk Based Severity
Afif El-Khuffash1, Neidín Bussmann2, Colm R Breatnach2
1Department of Neonatology, The Rotunda Hospital, Dublin, Ireland; Department of Pediatrics, The Royal College of Surgeons in Ireland, Dublin, Ireland.
Insights
Recruiting preterm infants for a patent ductus arteriosus (PDA) treatment trial using a PDA severity score (PDAsc) is feasible. While consent rates were high, PDA closure rates in the intervention group were low, necessitating improved treatment strategies.
Area of Science:
- Neonatalogy
- Pediatric Cardiology
- Clinical Trial Methodology
Background:
- Patent ductus arteriosus (PDA) is common in preterm infants.
- Effective treatment strategies for PDA are crucial to improve outcomes.
- A PDA severity score (PDAsc) was developed to guide treatment decisions.
Purpose of the Study:
- To assess the feasibility of recruiting preterm infants for a randomized controlled trial (RCT) of PDA treatment using a PDA severity score (PDAsc).
- To identify challenges in obtaining consent and protocol compliance.
- To evaluate PDA closure rates in response to treatment.
Main Methods:
- A single-center, randomized control pilot study involving 60 preterm infants (<29 weeks gestation) with a high PDAsc (≥5.0).
- Infants received either intravenous ibuprofen or a placebo.
- The study monitored consent rates, open-label treatment, and PDA closure rates. The primary outcome was chronic lung disease or death.
Main Results:
- 88% of families approached consented to participate.
- The overall PDA closure rate was 57% in the ibuprofen group versus 17% in the placebo group (P < .01).
- No significant difference was observed in the primary outcome of chronic lung disease or death between groups.
Conclusions:
- Recruitment of preterm infants for a PDA treatment RCT using a PDAsc is feasible.
- High consent rates and low rates of open-label treatment were observed.
- Future RCTs should focus on developing more effective PDA closure strategies due to low closure rates in the intervention arm.
Objectives:
To evaluate the feasibility of recruiting preterm infants to a randomized controlled trial of patent ductus arteriosus (PDA) treatment based on a PDA severity score (PDAsc) and to characterize challenges in obtaining consent, compliance with the protocol, and PDA closure rates.
Study Design:
This single-center, randomized control pilot study of 60 infants <29 weeks of gestation with a high PDAsc (≥5.0) at 36-48 hours of age receiving either ibuprofen or placebo intravenously. The study protocol did not allow for additional PDA therapy within the first 2 weeks. We reported the rate of consent, open label treatment, and PDA closure rates. The primary outcome was chronic lung disease or death.
Results:
We approached 83 families for enrollment with 73 (88%) providing consent; 13 infants had a PDAsc of <5; of the remaining infants, 30 were assigned ibuprofen and 30 received placebo. Eight infants received open label treatment in the first 2 weeks (12%). The overall PDA closure rate after treatment was 57% in the intervention group and 17% in the control group (P < .01). There was no difference in the primary clinical outcome (OR, 0.8; 95% CI, 0.3-2.1).
Conclusions:
Using a PDAsc for infant recruitment to a PDA treatment randomized controlled trial is feasible. There is a high rate of consent and relatively low rate of open-label PDA treatment. The overall PDA closure rate in the intervention arm was low placing the emphasis on devising more effective PDA closure strategies in future randomized controlled trials.
Trial Registration:
ISRCTN (13281214) and European Union Drug Regulating Authorities Clinical Trials Database (2015-004526-33).
