Luciana Teofili, Patrizia Papacci, Carlo Dani, Francesco Cresi, Giulia Remaschi, Claudio Pellegrino, Maria Bianchi, Giulia Ansaldi, Maria Francesca Campagnoli, Barbara Vania, Domenico Lepore, Fabrizio Gaetano Saverio Franco, Marco Fabbri, Roberta Penta de Vera d’ Aragona, Anna Molisso, Enrico Beccastrini, Antonella Dragonetti, Lorenzo Orazi, Tina Pasciuto, Iolanda Mozzetta, Antonio Baldascino, Emanuela Locatelli, Caterina Giovanna Valentini, Carmen Giannantonio, Brigida Carducci, Sabrina Gabbriellini, Roberto Albiani, Elena Ciabatti, Nicola Nicolotti, Silvia Baroni, Alessandro Mazzoni, Federico Genzano Besso, Francesca Serrao, Velia Purcaro, Alessandra Coscia, Roberta Pizzolo, Genny Raffaeli, Stefania Villa, Isabella Mondello, Alfonso Trimarchi, Flavia Beccia, Stefano Ghirardello, Giovanni Vento
Fondazione Policlinico A. Gemelli IRCCS. Università Cattolica del Sacro Cuore. Azienda Ospedaliero Universitaria Careggi. Città della Salute e della Scienza. Università di Torino. Azienda Ospedaliero Universitaria Pisana. Ospedale Santobono Pausilipon. Ospedale Evangelico Villa Betania. Polo Nazionale Ipovisione IAPB Italia Onlus. Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico. Università di Milano. Azienda Ospedaliera Bianchi Melacrino Morelli. Fondazione IRCCS Policlinico S. Matteo.
Italy
Italian Journal of Pediatrics
Ital J Pediatr 2024; 50:
DOI: 10.1186/s13052-024-01714-w
Abstract
Background: Preterm infants are at high risk for retinopathy of prematurity (ROP), with potential life-long visual impairment. Low fetal hemoglobin (HbF) levels predict ROP. It is unknown if preventing the HbF decrease also reduces ROP.
Methods: BORN is an ongoing multicenter double-blinded randomized controlled trial investigating whether transfusing HbF-enriched cord blood-red blood cells (CB-RBCs) instead of adult donor-RBC units (A-RBCs) reduces the incidence of severe ROP (NCT05100212). Neonates born between 24 and 27 + 6 weeks of gestation are enrolled and randomized 1:1 to receive adult donor-RBCs (A-RBCs, arm A) or allogeneic CB-RBCs (arm B) from birth to the postmenstrual age (PMA) of 31 + 6 weeks. Primary outcome is the rate of severe ROP at 40 weeks of PMA or discharge, with a sample size of 146 patients. A prespecified interim analysis was scheduled after the first 58 patients were enrolled, with the main purpose to evaluate the safety of CB-RBC transfusions.
Results: Results in the intention-to-treat and per-protocol analysis are reported. Twenty-eight patients were in arm A and 30 in arm B. Overall, 104 A-RBC units and 49 CB-RBC units were transfused, with a high rate of protocol deviations. A total of 336 adverse events were recorded, with similar incidence and severity in the two arms. By per-protocol analysis, patients receiving A-RBCs or both RBC types experienced more adverse events than non-transfused patients or those transfused exclusively with CB-RBCs, and suffered from more severe forms of bradycardia, pulmonary hypertension, and hemodynamically significant patent ductus arteriosus. Serum potassium, lactate, and pH were similar after CB-RBCs or A-RBCs. Fourteen patients died and 44 were evaluated for ROP. Ten of them developed severe ROP, with no differences between arms. At per-protocol analysis each A-RBC transfusion carried a relative risk for severe ROP of 1.66 (95% CI 1.06-2.20) in comparison with CB-RBCs. The area under the curve of HbF suggested that HbF decrement before 30 weeks PMA is critical for severe ROP development. Subsequent CB-RBC transfusions do not lessen the ROP risk.
Conclusions: The interim analysis shows that CB-RBC transfusion strategy in preterm neonates is safe and, if early adopted, might protect them from severe ROP.
Category
Class V. Pulmonary Hypertension Associated with Hematological, Systemic, Metabolic and Other Disorders
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication
Article Access
Free PDF File or Full Text Article Available Through PubMed or DOI: Yes