Off-label mesenchymal stromal cell treatment in two infants with severe bronchopulmonary dysplasia: clinical course and biomarkers profile

Abstract

Background: Bronchopulmonary dysplasia (BPD) is the most prevalent sequelae of premature birth, for which therapeutic options are currently limited. Mesenchymal stromal cells (MSCs) are a potential therapy for prevention or reversal of BPD.

Series of cases: We report on two infants with severe BPD in whom off-label treatment with repeated intravenous doses of allogeneic bone marrow-derived MSCs were administered. We analyzed the temporal profile of serum and tracheal cytokines and growth factors as well as safety, tolerability and clinical response. The administration of repeated intravenous doses of MSCs in two human babies with severe and advanced BPD was feasible and safe and was associated with a decrease of pro-inflammatory molecules and lung injury biomarkers. Both patients were at very advanced stages of BPD with very severe lung fibrosis and did not survive the disease.

Conclusions: MSCs are a promising therapy for BPD, but they should be administered in early stages of the disease.

Keywords: bronchopulmonary dysplasia; chronic lung disease of the newborn; mesenchymal stromal cells; molecular biomarkers; preterm newborns; pulmonary vascular disease; regenerative medicine; very low–birth weight infants.