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AbstractPulmonary hypertension is a complex and progressive condition that is either idiopathic or heritable, or associated with one or multiple health conditions, with or without congenital or acquired cardiovascular disease. Recent developments have tremendously increased the armamentarium of diagnostic and therapeutic approaches in children and young adults with pulmonary hypertension that is still associated with a high morbidity and mortality. These modalities include non-invasive imaging, pharmacotherapy, interventional and surgical procedures, and supportive measures. The optimal, tailored diagnostic and therapeutic strategies for pulmonary hypertension in the young are rapidly evolving but still face enormous challenges: Healthcare providers need to take the patient’s age, development, disease state, and family concerns into account when initiating advanced diagnostics and treatment. Therefore, there is a need for guidance on core and advanced medical training in paediatric pulmonary hypertension. The Association for European Paediatric and Congenital Cardiology working group “pulmonary hypertension, heart failure and transplantation” has produced this document as an expert consensus statement; however, all recommendations must be considered and applied in the context of the local and national infrastructure and legal regulations.

IMPORTANCE: Sudden cardiac death (SCD) is the most common mode of death in childhood hypertrophic cardiomyopathy (HCM), but there is no validated algorithm to identify those at highest risk. OBJECTIVE: To develop and validate an SCD risk prediction model that provides individualized risk estimates. DESIGN, SETTING, AND PARTICIPANTS: A prognostic model was developed from a retrospective, multicenter, longitudinal cohort study of 1024 consecutively evaluated patients aged 16 years or younger with HCM. The study was conducted from January 1, 1970, to December 31, 2017. EXPOSURES: The model was developed using preselected predictor variables (unexplained syncope, maximal left-ventricular wall thickness, left atrial diameter, left-ventricular outflow tract gradient, and nonsustained ventricular tachycardia) identified from the literature and internally validated using bootstrapping. MAIN OUTCOMES AND MEASURES: A composite outcome of SCD or an equivalent event (aborted cardiac arrest, appropriate implantable cardioverter defibrillator therapy, or sustained ventricular tachycardia associated with hemodynamic compromise). RESULTS: Of the 1024 patients included in the study, 699 were boys (68.3%); mean (interquartile range [IQR]) age was 11 (7-14) years. Over a median follow-up of 5.3 years (IQR, 2.6-8.3; total patient years, 5984), 89 patients (8.7%) died suddenly or had an equivalent event (annual event rate, 1.49; 95% CI, 1.15-1.92). The pediatric model was developed using preselected variables to predict the risk of SCD. The model’s ability to predict risk at 5 years was validated; the C statistic was 0.69 (95% CI, 0.66-0.72), and the calibration slope was 0.98 (95% CI, 0.59-1.38). For every 10 implantable cardioverter defibrillators implanted in patients with 6% or more of a 5-year SCD risk, 1 patient may potentially be saved from SCD at 5 years. CONCLUSIONS AND RELEVANCE: This new, validated risk stratification model for SCD in childhood HCM may provide individualized estimates of risk at 5 years using readily obtained clinical risk factors. External validation studies are required to demonstrate the accuracy of this model's predictions in diverse patient populations.

AbstractAims:Most reports on the outcome of children who present with heart failure, due to heart muscle disease, are from an era when ventricular assist devices were not available. This study provides outcome data for the current era where prolonged circulatory support can be considered for most children.Methods & Results:Data was retrieved on 100 consecutive children, who presented between 2010 – 2016, with a first diagnosis of unexplained heart failure. Hospital outcome was classified as either death, transplantation, recovery of function or persistent heart failure. Median age at presentation was 24 months and 58% were < 5 years old. Hospital mortality was 12% and 59% received a heart transplant. Most, 79%, of the transplants were carried out on patients with a device. Recovery of function was observed in 18% and 10% stabilised on oral therapy. Eighty-four percent of the deaths occurred in the <5 year old group. Shorter duration of support was associated with survival (34 days in survivors versus 106 in non-survivors, p = 0.01) and 72% were on an assist device at time of death.Conclusion:Heart failure in children who require referral to a transplant unit is a serious illness with a high chance of either transplantation or death. Modifications in assist devices will be required to improve safety, especially for children < 5 years old where the donor wait may be prolonged. The identification of children who may recover function requires further study.

Heart transplantation is a standard treatment for selected paediatric patients with end-stage heart disease. With improvement in surgical techniques, organ procurement and preservation strategies, immunosuppressive drugs, and more sophisticated monitoring strategies, survival following transplantation has increased over time. However, rejection, infection, renal failure, post-transplant lymphoproliferative disease and post-transplant cardiac allograft vasculopathy still preclude long-term survival. Therefore, continued multidisciplinary scientific efforts are needed for future gains. This review focuses on the current status, outcomes and ongoing challenges including patient selection, indications and contraindications, national and international survivals, post-transplant complications and quality of life.

AbstractAdvanced medical and surgical treatment of heart failure and management of patients following heart transplantation is an emerging area. Treatment options at various levels are becoming available in an increasing number of countries. This rapidly evolving field involves a complex multi-disciplinary approach with a number of complementary medical and surgical strategies, including pharmacotherapy, structural cardiac interventions, electrophysiological optimisation, mechanical circulatory support, and heart transplantation. Furthermore, the importance of psycho-social support and care of patients and their families cannot be overstated. The aforementioned challenges and dynamics of new developments require guidance for core and advanced medical training in heart failure and transplantation. The Association for European Paediatric and Congenital Cardiology working group “pulmonary hypertension, heart failure and transplantation” has produced this document as an expert consensus statement; however, all recommendations must be considered and applied in the context of the local and national infrastructure and legal regulations.

Objectives: Mechanical circulatory support for pediatric heart failure patients with the Berlin Heart EXCOR ventricular assist system is the only approved and established bridging strategy for recovery or heart transplantation. In recent years, the burden of thromboembolic events has led to modifications of the recommended antithrombotic therapy. Therefore, we aimed to assess modifications of antithrombotic practice among the European EXCOR Pediatric Investigator Group members.Methods: We sent a questionnaire assessing seven aspects of antithrombotic therapy to 18 European hospitals using the EXCOR device for children. Returned questionnaires were analyzed and identified antithrombotic strategies were descriptively compared to “Edmonton protocol” recommendations developed for the US EXCOR pediatric approval study.Results: Analysis of 18 received surveys revealed substantial deviations from the Edmonton protocol, including earlier start of heparin therapy at 6–12 h postoperatively and in 50% of surveyed centers, monitoring of heparin effectiveness with aPTT assay, administering vitamin K antagonists before 12 months of age. About 39% of centers use higher international normalized ratio targets, and platelet inhibition is changed in 56% including the use of clopidogrel instead of dipyridamole. Significant inter-center variability with multiple deviations from the Edmonton protocol was discovered with only one center following the Edmonton protocol completely.Conclusion: Current antithrombotic practice among European EXCOR users representing the treatment of more than 600 pediatric patients has changed over time with a trend toward a more aggressive therapy. There is a need for systematic evidence-based evaluation and harmonization of developmentally adjusted antithrombotic management practices in prospective studies toward revised recommendations.

AbstractA 20-month-old girl presented with severe dilated cardiomyopathy and decompensated congestive cardiac failure. Despite escalating inotropic and mechanical ventilation support, she required placement on extracorporeal membrane oxygenation and transfer to the transplant centre in Newcastle, England. She was placed on biventricular assist device and then Berlin Heart but failed to show any recovery of ventricular function. She underwent orthotopic heart transplantation at 2 years of age. She developed bacterial endocarditis with Enterococcus faecalisresulting in severe aortic valve regurgitation requiring aortic valve replacement with a 19 mm On-X valve (Airtivion) 11 days after her transplant. Given the size of the donor heart, it was possible to implant a 19-mm valve in this 12 kg child with minimal risk of patient prosthesis mismatch. She was anticoagulated with warfarin (On-X valve INR 2-3 for first 3 months; INR 1.5-2.0 thereafter). Although she suffered several other post-operative complications, including malabsorption, nasojejunal feeding, liver dysfunction, vertebral fractures, renal impairment and renal calcification, and need for repeat opening of her tracheostomy site following her initial decannulation, her aortic valve function has remained stable.

AbstractBackgroundSildenafil is increasingly being used in the management of pulmonary arterial hypertension in the newborn. Its role in patients with congenital cardiac disease is less well defined and as yet has only been reported sporadically.AimPresent our experience with sildenafil treatment in patients with a failing Fontan circulation.Patients and methodsRetrospective review of 13 symptomatic patients after Fontan palliation who received treatment with sildenafil between January, 2006 and July, 2008.ResultsThree patients suffered from protein-losing enteropathy, four patients presented with bronchial casts, two had severe cyanosis after fenestrated Fontan procedure, two had prolonged chylous effusions, one had a previous failure of Fontan and take-down, and one patient had arrhythmias and end-stage cardiac failure requiring conversion to an extra-cardiac Fontan. Sildenafil was used in the dosage of 1?2 milligrams per kilogram 3?4 times per day. Protein-losing enteropathy and ?-1-antitrypsin levels improved in all three patients on sildenafil treatment. One of these patients had a concomitant catheter creation of a fenestration, as did two patients presenting with bronchial casts and both patients with persistent chylous effusions. All four patients with bronchial casts and two patients with cyanosis improved significantly on sildenafil treatment. Chylous effusions decreased after sildenafil and stent enlargement of a fenestration. There were no significant side effects requiring sildenafil withdrawal over a treatment period ranging from 2 months to 2 years.ConclusionsSildenafil can be used safely and effectively in the treatment of patients with a failing Fontan circulation.

AimThe aim of this audit was to establish whether immunosuppression was being prescribed correctly and whether target levels were being reached during the first six weeks post-transplant.1 2MethodThe standards were discussed and agreed, due to an absence of standardised local or national written protocols, with the lead paediatric cardiothoracic transplant consultant and a specialist transplant liaison nurse. The paediatric transplant database provided a list of patients between October 2016 and July 2018, from which paediatric cardiac transplant patients were included in this audit. All data were collected retrospectively, for the first six weeks post-transplant, from patient’s electronic records.ResultsTwenty-three patients were included in the audit; fifteen males and eight females and the mean age was 6 years old. The standards for the timing and dosing of the first ciclosporin dose were met for 87% and 78% of patients respectively. Six patients (26%) had a ciclosporin level within the target range by day 4 post-transplant, for the remaining seventeen patients the average was day 9 post-transplant. The mean levels remained within this range or slightly above after day 9. Azathioprine or mycophenolate was started within 7 days of transplant in 6 patients (23%). Four patients (17%) had documented episodes of rejection; in one patient all other standards were met and in the other three only one additional standard was not met. Nineteen patients (83%) did not have a documented episode of rejection.ConclusionsPost-transplant management is individualised based on multiple factors such as clinical conditions e.g. renal/liver impairment and whether other agents such as ATG or steroids are being used.1The lack of documentation around the treatment decisions made it difficult to explain deviations from standards in this audit. Ciclosporin standards were not met completely but were most likely unfeasible due to a narrow target range and the time between first dose and level monitoring. There did not appear to be a clear association between standards not being met and episodes of rejection. It would be beneficial to repeat this as a larger, prospective audit using revised standards.ReferencesCostanzo MR, Costanzo MR, Dipchand A, et al. The international society of heart and lung transplantation guidelines for the care of heart transplant recipients. J Heart Lung Transplant2010;29:914–56.Sandimmun Concentrate for Solution for Infusion 50 mg/ml - Summary of Product Characteristics (SmPC) - (eMC) [Internet]. [cited 2018 Sep 2]. Available from: https://www.medicines.org.uk/emc/product/1036

ABO-incompatible heart transplantation increases donor availability in young children and is evolving into standard of care in children younger than 2 years. Previous smaller studies suggest similar outcomes to ABO-compatible heart transplantation, but persisting alterations of the immune system in ABO-incompatible recipients might increase the risk of some infections or benefit the graft owing to reduced HLA reactivity. We aimed to assess long-term outcomes in young children after they received ABO-incompatible or ABO-compatible heart transplantation.