Afif El-Khuffash1, Neidín Bussmann2, Colm R Breatnach2, Aisling Smith2, Elizabeth Tully3, Joanna Griffin4, Naomi McCallion5, John David Corcoran5, Elena Fernandez6, Claudia Looi6, Brian Cleary7, Orla Franklin8, Patrick J McNamara9. 1. Department of Neonatology, The Rotunda Hospital, Dublin, Ireland; Department of Pediatrics, The Royal College of Surgeons in Ireland, Dublin, Ireland. Electronic address: afifelkhuffash@rcsi.com. 2. Department of Neonatology, The Rotunda Hospital, Dublin, Ireland. 3. Department of Obstetrics & Gynecology, The Royal College of Surgeons in Ireland, Dublin, Ireland. 4. Department of Research & Academic Affairs, The Rotunda Hospital, Dublin, Ireland. 5. Department of Neonatology, The Rotunda Hospital, Dublin, Ireland; Department of Pediatrics, The Royal College of Surgeons in Ireland, Dublin, Ireland. 6. Department of Pharmacy, The Rotunda Hospital, Dublin, Ireland. 7. Department of Pharmacy, The Rotunda Hospital, Dublin, Ireland; School of Pharmacy, Royal College of Surgeons in Ireland, Dublin, Ireland. 8. Department of Pediatric Cardiology, Our Lady's Children's Hospital Crumlin, Ireland. 9. Division of Neonatology, Stead Family Children's Hospital, Iowa City, IA; Department of Pediatrics, University of Iowa, Iowa City, IA; Department of Cardiology, University of Iowa, Iowa City, IA.
Abstract
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 eitheribuprofen 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).
RCT Entities:
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).
Authors: Megan Barcroft; Christopher McKee; Darren P Berman; Rachel A Taylor; Brian K Rivera; Charles V Smith; Jonathan L Slaughter; Afif El-Khuffash; Carl H Backes Journal: Clin Perinatol Date: 2022-01-21 Impact factor: 3.430
Authors: Ömer Erdeve; Emel Okulu; Yogen Singh; Richard Sindelar; Mehmet Yekta Oncel; Gianluca Terrin; Giovanni Boscarino; Ali Bülbül; Hannes Sallmon; Begüm Atasay; Fahri Ovalı; Ronald I Clyman Journal: Turk Arch Pediatr Date: 2022-03