The First Allogeneic Transplantation of Human Umbilical Cord-derived Mesenchymal Stem/stromal Cells for Bronchopulmonary Dysplasia: Preliminary Outcomes in Four Vietnamese Infants

Abstract

<jats:title>Abstract</jats:title> <jats:p><jats:bold>Background: </jats:bold>Bronchopulmonary dysplasia (BPD) is a severe condition in premature infants that compromises theirlung function and necessitatesoxygen support. Despite major improvements in perinatal care minimizing the devastating effects, BPD remains the most frequent complication of extreme preterm birth. Our study reports the safety ofthe allogeneic administration of umbilical cord-derived mesenchymal stem/stromal cells (allo-UC-MSCs) and the preliminary efficacy of the treatment in four infants with established BPD.<jats:bold>Methods</jats:bold>: UC tissue was collected from a healthy donor, followed by propagation at the Stem Cell Core Facility at Vinmec Research Institute of Stem Cell and Gene Technology. UC-MSC culture was conducted under xeno-free and serum-free conditions. Four patients with established BPD were enrolled in this study between May 25, 2018, and December 31, 2018. All four patients received two intravenous doses of allo-UC-MSCs (1 million cells/kg patient body weight (PBW) per dose) with an intervening interval of 7 days. Safety and efficacy were evaluated during hospitalization and at 7 days and 1, 6 and 12 postdischargemonths.<jats:bold>Results: </jats:bold>No transplantation-associated severe adverse events or prespecified adverse events were observed in the four patients throughout the study period. At the time of this report, all patients had recovered from BPD and been weaned off of oxygen support. Chest X-rays and CT scans confirmed the dramatic reduction infibrosis.<jats:bold>Conclusions: </jats:bold>Allo-UC-MSC transplantation is safe and might improve respiratory function anddecrease lung fibrosis in preterm infants with established BPD.<jats:bold>Trial registration</jats:bold>: This preliminary study was approved by Vinmec International Hospital Ethics Board, approval number: 88/2019/QĐ-VMEC, registered 12 March 2019 - retrospectively registered.</jats:p>