Clinical trial NCT05100212: umBilical Or Adult Donor Red Blood Cells in Extremely Low Gestational Age Neonates and Retinopathy of Prematurity (BORN)
Official title: Umbilical or Adult Donor Red Blood Cells to Transfuse Extremely Low Gestational Age Neonates. A Randomized Trial to Assess the Effect on Retinopathy of Prematurity Severity.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Retinopathy of Prematurity
Lead sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Sponsor class: OTHER
Collaborators: name: Fresenius Kabi
First posted: 2021-10-29
Enrollment: 146
Sex: ALL
Minimum age (years): 24
Maximum age (years): 27
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Interventional, randomized, controlled, double-blind, with an adaptive design to evaluate safety and efficacy of allogeneic CB-RBC transfusions.
Primary purpose: PREVENTION

Brief summary: Extremely low gestational age neonates (ELGAN, i.e., born before 28 gestation weeks) are among the most heavily transfused pediatric patients. In this clinical setting, repeated red blood cell (RBC) transfusions independently predict a poor outcome, with a higher risk for mortality and morbidity. Recent studies from our own and other groups highlighted a close association between low levels of fetal hemoglobin (HbF) and severity of retinopathy of prematurity (ROP) and bronchopulmonary dysplasia (BPD), two disabilities that frequently complicate preterm birth. This association is not surprising, considering that 1) preterm neonates have a highly immature antioxidant reserve and both ROP and BPD rely on the oxidative damage as underlying mechanism; 2) in comparison with HbA, HbF is endowed with higher oxygen affinity, greater redox potential, higher tetrameric stability, and higher ability to generate unbound nitric oxide, all functions potentially protective in presence of an oxidative challenge; 3) in normal prenatal life, developing organ and tissues are exposed exclusively to HbF until last weeks of gestation; 4) in preterm neonates, the switch of the synthesis from HbF to HbA occurs around their due date, i.e., several weeks after the premature birth; 5) when preterm neonates receive transfusions, their tissues are abruptly exposed to high levels of HbA. We have recently run a pilot trial demonstrating as a proof-of-concept that transfusing cord blood red blood cell concentrates (CB-RBC) effectively prevents or restrains the HbF loss consequent to adult donor standard transfusions (A-RBC). This study explores the hypothesis that transfusing CB-RBCs instead of A-RBC may lower the incidence of severe ROP in ELGANs needing transfusions.


Eligibility criteria: Inclusion Criteria:

* gestational age (GA) at birth between 24+0 and 27+6 weeks
* signed informed consent of parents.

Exclusion Criteria:

One or more of the following:

* maternal-fetal immunization
* hydrops fetalis
* major congenital malformations associated or not with genetic syndromes
* previous transfusions
* hemorrhage at birth
* congenital infections
* health care team deeming it inappropriate to approach the infant's family for informed consent.

Primary outcomes:
measure: Retinopathy of prematurity
timeFrame: up to the age of 40 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Firenze
country: Italy
facility: Azienda Ospedaliero Universitaria Careggi
city: Foggia
country: Italy
facility: Ospedale Casa Sollievo della Sofferenza, San Giovanni Rotondo,
city: Milan
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
city: Naples
country: Italy
facility: Ospedale Evangelico Villa Betania
city: Napoli
country: Italy
facility: AORN Santobono-Pausilipon
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico S. Matteo
city: Pisa
country: Italy
facility: Azienda Ospedaliero Universitaria Pisana
city: Reggio Calabria
country: Italy
facility: Azienda Ospedaliera Bianchi Melacrino Morelli
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario A.Gemelli IRCCS
city: Torino
country: Italy
facility: Città della Salute e della Scienza
Study officials:
affiliation: Fondazione Policlinico Gemelli IRCCS
name: Luciana Teofili, MD
role: PRINCIPAL_INVESTIGATOR
References: