Repeated intravenous doses of human umbilical cord-derived mesenchymal stromal cells for bronchopulmonary dysplasia: results of a phase 1 clinical trial with 2-year follow-up.

BACKGROUND: Currently, there is a lack of effective treatments or preventive strategies for bronchopulmonary dysplasia (BPD). Pre-clinical studies with mesenchymal stromal cells (MSCs) have yielded encouraging results. The safety of administering repeated intravenous doses of umbilical cord tissue-derived mesenchymal stromal cells (UC-MSCs) has not yet been tested in extremely-low-gestational-age newborns (ELGANs). AIMS: to test the safety and feasibility of administering three sequential intravenous doses of UC-MSCs every 7 days to ELGANs at risk of developing BPD. METHODS: In this phase 1 clinical trial, we recruited ELGANs (birth weight ≤1250 g and ≤28 weeks in gestational age [GA]) who were on invasive mechanical ventilation (IMV) with FiO2 ≥ 0.3 at postnatal days 7-14. Three doses of 5 × 106/kg of UC-MSCs were intravenously administered at weekly intervals. Adverse effects and prematurity-related morbidities were recorded. RESULTS: From April 2019 to July 2020, 10 patients were recruited with a mean GA of 25.2 ± 0.8 weeks and a mean birth weight of 659.8 ± 153.8 g. All patients received three intravenous UC-MSC doses. The first dose was administered at a mean of 16.6 ± 2.9 postnatal days. All patients were diagnosed with BPD. All patients were discharged from the hospital. No deaths or any serious adverse events related to the infusion of UC-MSCs were observed during administration, hospital stays or at 2-year follow-up. CONCLUSIONS: The administration of repeated intravenous infusion of UC-MSCs in ELGANs at a high risk of developing BPD was feasible and safe in the short- and mid-term follow-up.

Insuficiencia cardiaca en urgencias: un reto diagnóstico / Heart failure in the emergency department: a diagnostic challenge

La insuficiencia cardiaca es una patología poco frecuente en las consultas médicas pediátricas. Las formas en las que se expresa clínicamente esta incapacidad son muy diversas, sin embargo, en el paciente adulto suelen detectarse a través de síntomas clásicos como la disnea. En el caso del paciente pediátrico este espectro de presentación es mucho más amplio e inespecífico. El porcentaje de pacientes que no son correctamente diagnosticados en la primera consulta médica asciende hasta el 50%, y muchos de ellos reciben terapia antibiótica al haber sido etiquetado de infección bacteriana. Por ello presentamos el caso de un niño de tres años diagnosticado de insuficiencia cardiaca en nuestro servicio de urgencias pediátricas

Heart failure is an uncommon pathology in medical pediatric consultations. The forms in which this disability is clinically expressed are diverse, however, in the adult patient it is often detected through classic symptoms such as dyspnea. In the case of the pediatric patient this spectrum of presentation is much wider and non-specific. The percentage of patients who are not correctly diagnosed at the first medical consultation amounts to 50%, receiving many of them antibiotic therapy after having been labelled as bacterial infection. Therefore, we present the case of a three-year-old child diagnosed with heart failure in our pediatric emergency department

Retrocardiac lung lobe in a neonate with Scimitar syndrome.

Scimitar syndrome is a rare congenital disorder which is associated with right lung developmental abnormalities, mainly hypoplastic right lung. We present the case of a neonate with a retrocardiac lung lobe that, to the best of our knowledge, has not been described yet. Multimodality imaging can establish the diagnosis and enable a correct description of this anomaly and its anatomical varieties.

Nonketotic hyperglycinemia presenting as pulmonary hypertensive vascular disease and fatal pulmonary edema in response to pulmonary vasodilator therapy.

The association of pulmonary hypertensive vascular disease with nonketotic hyperglycinemia is rare. We describe 5 infants diagnosed with nonketotic hyperglycinemia, in whom pulmonary hypertensive vascular disease was the main presenting feature, and who developed severe pulmonary edema in response to pulmonary vasodilators.