RESUMO
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.
Assuntos
Displasia Broncopulmonar , Transplante de Células-Tronco Mesenquimais , Células-Tronco Mesenquimais , Cordão Umbilical , Humanos , Displasia Broncopulmonar/terapia , Feminino , Transplante de Células-Tronco Mesenquimais/métodos , Masculino , Células-Tronco Mesenquimais/citologia , Recém-Nascido , Cordão Umbilical/citologia , Seguimentos , Administração Intravenosa , Idade Gestacional , Recém-Nascido PrematuroRESUMO
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.
Assuntos
Hiperglicinemia não Cetótica/diagnóstico , Hipertensão Pulmonar/etiologia , Edema Pulmonar/induzido quimicamente , Vasodilatadores/efeitos adversos , Feminino , Humanos , Hiperglicinemia não Cetótica/complicações , Hiperglicinemia não Cetótica/mortalidade , Hiperglicinemia não Cetótica/fisiopatologia , Hipertensão Pulmonar/tratamento farmacológico , Hipertensão Pulmonar/patologia , Hipertensão Pulmonar/fisiopatologia , Lactente , Pulmão/patologia , Masculino , Edema Pulmonar/diagnóstico por imagem , Edema Pulmonar/mortalidade , Radiografia , Insuficiência Respiratória/etiologia , Estudos RetrospectivosRESUMO
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.