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.

Éxito de la cirugía combinada de trasplante bipulmonar y reparación de cardiopatía congénita en paciente pediátrico / Successful combined bilateral lung transplant and congenital heart disease repair in a pediatric patient

No disponible

Valve-Sparing Tetralogy of Fallot Repair With Intraoperative Dilation of the Pulmonary Valve. Mid-Term Results.

Deleterious long-term effects of chronic pulmonary regurgitation after repair of tetralogy of Fallot have become evident during the last decades. Subsequently, some groups have developed strategies to spare the pulmonary valve function at the time of repair with good early results. However, mid-term outcomes are scarce in the literature and in some cases controversial. The aim of our study is to report our results mid-term with valve-sparing repair of tetralogy of Fallot. We retrospectively reviewed patients undergoing tetralogy of Fallot repair and having preservation of the pulmonary valve with intraoperative dilation at our institution. From June 2009 through June 2017, 42 patients underwent valve-sparing tetralogy of Fallot repair. Median age and weight at surgery were 5.2 months and 7.2 kg. Median preoperative pulmonary valve diameters and Z scores by echocardiography were 6.4 mm (range 4.5-11 mm) and -2.3 (range -1.3 to -4.5). No patient died in our series. For a median follow-up of 45 months, the pulmonary valve has grown by Z score (P < 0.0001) as well as the pulmonary trunk (P= 0.00216). Significant pulmonary regurgitation has developed in 9 patients (21.4%). No patient has required reintervention/reoperation for recurrent right ventricular outflow tract obstruction. Patients with tetralogy of Fallot who had valve-sparing repair with intraoperative dilation of the pulmonary valve show good early and mid-term results with respect to right ventricular outflow tract obstruction. The pulmonary valve annulus and the pulmonary trunk grow through follow-up. Progressive development of significant pulmonary regurgitation is seen in more than 20% of patients. Long-term data with this approach and comparison with a population of patients undergoing a transannular patch repair are required to establish the real utility of this approach.