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Shahin Moledina, Great Ormond Street Hospital, UK
Mandy MacLean, University of Strathclyde, UK
Genetics of pulmonary arterial hypertension. What we can learn by studying children with and without congenital heart disease
15:00-15:15 BST
Presenter: // Wendy Chung, University of Columbia, USA
Awaiting further information
Therapeutic options in advanced PVD: Potts shunt versus lung transplantation
15:22-15:37 BST
Presenter: // Mark Grady, Washington University School of Medicine in St. Louis, USA
Awaiting further information
Stem cells and Regenerative Medicine for Neonatal Lung Disease: implications for COPD
15:45-16:00 BST
Presenter: // Bernard Thebaud, The Ottowa Hospital, Canada
Advances in perinatal medicine have enabled the survival of preterm infants at the limit of viability. As a consequence, prevention of injury to the ever more immature lung has become more challenging. The incidence of bronchopulmonary dysplasia (BPD), the chronic lung disease of prematurity, has remained unchanged over the past two decades. The corollary is that individuals born very preterm or with very low birth weight are at risk of not reaching their full airway growth potential in adolescence and early adulthood, suggesting an increased risk of chronic obstructive pulmonary disease2 and pulmonary vascular disease in later adulthood. Corticosteroids, the only current treatment to substantially reduce the incidence of BPD, are used parsimoniously, because of severe adverse effects on neurodevelopment. Next generation therapies that safely enable both, injury prevention/repair and proper organ development are required to improve respiratory outcome in extreme preterm infants and interrupt the potential link to COPD. Stem cell-based therapies offer a new hope in preventing organ damage in extreme preterm infants. Preclinical studies have demonstrated the lung protective effects of exogenous cell therapies in experimental neonatal lung injury models. Amongst the numerous repair cells proposed, mesenchymal stromal cells (MSCs), identified over 40 years ago in the bone marrow as niche cells crucial for the proper functioning of hematopoietic stem cells, have advanced to the forefront of cell-based therapies. Contrary to the initial hypothesis, cell engraftment and differentiation do not contribute to the repair mechanism. Instead, MSCs seem to orchestrate the repair process by cell-cell contact, modulation of the immune response and release of factors that promote lung growth. Fuelled by these promising preclinical studies, early phase clinical trials have begun indicating feasibility and absence of toxicity in a small number of extreme preterm infants. Nonetheless, cell-based therapy for regenerative purposes is still in its infancy. The initial enthusiasm for MSCs has been curbed because results of adult clinical trials have not matched the enticing preclinical data so far. Thus, major knowledge gaps remain and laboratory studies are needed to better understand the biology of MSCs and other putative repair cells. Technological advances will allow a better characterization of putative repair cells, the development of reliable potency assays that predict therapeutic efficacy, and enable the manufacturing of high quality, safe and efficient cell products. Well designed early phase clinical trials should be based on robust preclinical data, biological plausibility and a strong rationale. Adopting a rigorous, evidence-based approach will be critical for cell therapies to fulfil their potential in preventing/regenerating lung injury.
Assessment of operability in patients with congenital heart disease and PAH: Fallacies and pitfalls
16:08-16:23 BST
Presenter: // Erika B. Rosenzweig, Columbia University Medical Center, USA
Awaiting further information
The impact of chronic pulmonary hypertension on ventricular function in extremely premature infants
16:30-16:40 BST
Presenter: // Gabriela de Carvalho Nunes, Mcgill University Health Centre, Montreal Children’s Hospital, Canada
Awaiting further information
Improving guidance for the correctability of congenital cardiovascular shunts with increased pulmonary vascular resistance
16:45-16:55 BST
Presenter: // Ronald Day, University of Utah, Department of Pediatrics, USA
Congenital cardiovascular shunts are usually identified early in life and corrected without long-term complications associated with pulmonary hypertension. However, some shunts are associated with enough pulmonary vascular disease that correction may not improve the patient’s quality or length of life. Current guidelines for correctability based upon expert opinion. I sought to use the results of the Inhaled Nitric Oxide as a Preoperative Test I study to evaluate the accuracy of recent guidelines. The results indicate that acute vasodilator testing may help to increase the accuracy of a preoperative hemodynamic evaluation.
