Closing the gap between acceptable and ideal in catheterisation for paediatric and congenital heart disease-A global view.

In recent issues of the Journal of the Society for Cardiovascular Angiography and Interventions and the Journal of the American College of Cardiology: Cardiovascular Interventions, Holzer and colleagues presented an Expert Consensus Document titled: "PICS / AEPC / APPCS / CSANZ / SCAI / SOLACI: Expert consensus statement on cardiac catheterization for pediatric patients and adults with congenital heart disease." This Expert Consensus Document is a massively important contribution to the community of paediatric and congenital cardiac care. This document was developed as an Expert Consensus Document by the Pediatric and Congenital Interventional Cardiovascular Society, the Association for European Paediatric and Congenital Cardiology, the Asia-Pacific Pediatric Cardiac Society, the Cardiac Society of Australia and New Zealand, the Society for Cardiovascular Angiography and Interventions, and the Latin American Society of Interventional Cardiology, as well as the Congenital Cardiac Anesthesia Society and the American Association of Physicists in Medicine.As perfectly stated in the Preamble of this Expert Consensus Document, "This expert consensus document is intended to inform practitioners, payors, hospital administrators and other parties as to the opinion of the aforementioned societies about best practices for cardiac catheterisation and transcatheter management of paediatric and adult patients with congenital heart disease, with added accommodations for resource-limited environments." And, the fact that the authorship of this Expert Consensus Document includes global representation is notable, commendable, and important.This Expert Consensus Document has the potential to fill an important gap for this patient population. National guideline documents for specific aspects of interventions in patients with paediatric heart disease, including training guidelines, do exist. However, this current Expert Consensus Document authored by Holzer and colleagues provides truly globally applicable standards on cardiac catheterisation for both paediatric patients and adults with congenital heart disease (CHD).Our current Editorial provides different regional perspectives from senior physicians dedicated to paediatric and congenital cardiac care who are practicing in Europe, the Asia-Pacific region, Latin America, Australia/New Zealand, and North America. Establishing worldwide standards for cardiac catheterisation laboratories for children and adults with CHD is a significant stride towards improving the quality and consistency of care. These standards should not only reflect the current state of medical knowledge but should also be adaptable to future advancements, ultimately fostering better outcomes and enhancing the lives of individuals affected by CHD worldwide.Ensuring that these standards are accessible and adaptable across different healthcare settings globally is a critical step. Given the variability in resources and infrastructure globally, the need exists for flexibility and tailoring to implement recommendations.The potential impact of the Expert Consensus Document and its recommendations is likely significant, but heterogeneity of healthcare systems will pose continuing challenges on healthcare professionals. Indeed, this heterogeneity of healthcare systems will challenge healthcare professionals to finally close the gap between acceptable and ideal in the catheterisation of patients with paediatric and/or congenital heart disease.

Paediatric aortic valve replacement using decellularized allografts: a multicentre update following 143 implantations and five-year mean follow-up.

OBJECTIVES: Decellularized aortic homografts (DAH) were introduced in 2008 as a further option for paediatric aortic valve replacement (AVR). METHODS: Prospective, multicentre follow-up of all paediatric patients receiving DAH for AVR in 8 European centres. RESULTS: A total of 143 DAH were implanted between February 2008 and February 2023 in 137 children (106 male, 74%) with a median age of 10.8 years (interquartile range 6.6-14.6). Eighty-four (59%) had undergone previous cardiac operations and 24 (17%) had undergone previous AVR. The median implanted DAH diameter was 21 mm (interquartile range 19-23). The median operation duration was 348 min (227-439) with a median cardiopulmonary bypass time of 212 min (171-257) and a median cross-clamp time of 135 min (113-164). After a median follow-up of 5.3 years (3.3-7.2, max. 15.2 years), the primary efficacy end-points peak gradient (median 14 mmHg, 9-28) and regurgitation (median 0.5, interquartile range 0-1, grade 0-3) showed good results but an increase over time. Freedom from death/explantation/endocarditis/bleeding/thromboembolism at 5 years were 97.8 ± 1.2/88.7 ± 3.3/99.1 ± 0.9/100 and 99.2 ± 0.8%, respectively. Freedom from death/explantation/endocarditis/bleeding/thromboembolism at 10 years were 96.3 ± 1.9/67.1 ± 8.0/93.6 ± 3.9/98.6 ± 1.4 and 86.9 ± 11.6%, respectively. In total, 21 DAH were explanted. Seven were replaced by a mechanical AVR, 1 Ross operation was performed and a re-do DAH was implanted in 13 patients with no redo mortality. The calculated expected adverse events were lower for DAH compared to cryopreserved homograft patients (mean age 8.4 years), and in the same range as for Ross patients (9.2 years) and mechanical AVR (13.0 years). CONCLUSIONS: This large-scale prospective analysis demonstrates excellent mid-term survival using DAH with adverse event rates comparable to paediatric Ross procedures.

Direct comparison of the diagnostic performance of growth differentiation factor 8 in pediatric versus adult heart failure.

INTRODUCTION: Growth differentiation factor 8 (GDF-8, myostatin) has been proposed for the management of adult heart failure (HF). Its potential role in pediatric HF patients is unknown. We sought to investigate its diagnostic performance in adult versus pediatric HF. METHODS: GDF-8 was measured prospectively in pediatric and adult HF patients and in matching controls. HF was defined as the combination of typical symptoms and impaired left ventricular systolic function. Diagnostic performance for the detection of HF was evaluated by receiver operating characteristic (ROC) analysis. RESULTS: We enrolled 137 patients with HF (85 pediatric) and 67 healthy controls (47 pediatric). Neither pediatric nor adult HF patients had significantly different GDF-8 levels compared to the reference groups (3.53 vs 3.46 ng/mL, p = 0.334, and 6.87 vs 8.15 ng/mL, p = 0.063, respectively), but pediatric HF patients had significantly lower GDF-8 levels compared to adult patients (p < 0.001). ROC analysis showed no significant improvement adding GDF-8 to NT-proBNP, age and sex (area under the curve (AUC): 0.870 vs 0.868, p = 0.614) in children and neither in addition to age nor sex in adult HF patients (AUC: 0.74 vs 0.62, p = 0.110). CONCLUSION: GDF-8 did not accurately differentiate between HF patients and normal comparators in neither adults nor in children.

Assessment for learning of paediatric cardiology trainees in 41 centres from 19 European countries.

BACKGROUND: Limited data exist on how trainees in paediatric cardiology are assessed among countries affiliated with the Association of European Paediatric and Congenital Cardiology. METHODS: A structured and approved questionnaire was circulated to educationalists/trainers in 95 Association for European Paediatric and Congenital Cardiology training centres. RESULTS: Trainers from 46 centres responded with complete data in 41 centres. Instructional design included bedside teaching (41/41), didactic teaching (38/41), problem-based learning (28/41), cardiac catheterisation calculations (34/41), journal club (31/41), fellows presenting in the multidisciplinary meeting (41/41), fellows reporting on echocardiograms (34/41), clinical simulation (17/41), echocardiography simulation (10/41), and catheterisation simulation (3/41). Assessment included case-based discussion (n = 27), mini-clinical evaluation exercise (mini-CEX) (n = 12), directly observed procedures (n = 12), oral examination (n = 16), long cases (n = 11), written essay questions (n = 6), multiple choice questions (n = 5), and objective structured clinical examination (n = 2). Entrustable professional activities were utilised in 10 (24%) centres. Feedback was summative only in 17/41 (41%) centres, formative only in 12/41 (29%) centres and a combination of formative and summative feedback in 10/41 (24%) centres. Written feedback was provided in 10/41 (24%) centres. Verbal feedback was most common in 37/41 (90 %) centres. CONCLUSION: There is a marked variation in instructional design and assessment across European paediatric cardiac centres. A wide mix of assessment tools are used. Feedback is provided by the majority of centres, mostly verbal summative feedback. Adopting a programmatic assessment focusing on competency/capability using multiple assessment tools with regular formative multisource feedback may promote assessment for learning of paediatric cardiology trainees.

Adult congenital heart disease training in Europe: current status, disparities and potential solutions.

OBJECTIVES: This study aimed to determine the status of training of adult congenital heart disease (ACHD) cardiologists in Europe. METHODS: A questionnaire was sent to ACHD cardiologists from 34 European countries. RESULTS: Representatives from 31 of 34 countries (91%) responded. ACHD cardiology was recognised by the respective ministry of Health in two countries (7%) as a subspecialty. Two countries (7%) have formally recognised ACHD training programmes, 15 (48%) have informal (neither accredited nor certified) training and 14 (45%) have very limited or no programme. Twenty-five countries (81%) described training ACHD doctors 'on the job'. The median number of ACHD centres per country was 4 (range 0-28), median number of ACHD surgical centres was 3 (0-26) and the median number of ACHD training centres was 2 (range 0-28). An established exit examination in ACHD was conducted in only one country (3%) and formal certification provided by two countries (7%). ACHD cardiologist number versus gross domestic product Pearson correlation coefficient=0.789 (p<0.001). CONCLUSION: Formal or accredited training in ACHD is rare among European countries. Many countries have very limited or no training and resort to 'train people on the job'. Few countries provide either an exit examination or certification. Efforts to harmonise training and establish standards in exit examination and certification may improve training and consequently promote the alignment of high-quality patient care.

Premature closure of the arterial duct presenting with right heart failure of the fetus and ductal aneurysm postnatally.

In utero idiopathic constriction of the arterial duct is a rare condition with only a handful reported cases. Ductal aneurysms with thrombus formations on the other hand are significantly more common. We report a case of a term infant who presented with right heart failure due to premature ductal closure and postnatal severe respiratory distress. Subsequent diagnostics revealed paresis of left laryngeal nerve and obstruction of the left pulmonary artery secondary to a ductal aneurysm. Consequently, surgical intervention was considered necessary. Post-operatively, right ventricular function and hoarseness resolved slowly.

Macitentan in the Young-Mid-term Outcomes of Patients with Pulmonary Hypertensive Vascular Disease treated in a Pediatric Tertiary Care Center.

BACKGROUND: Pulmonary hypertension (PH) is a severe hemodynamic condition with high morbidity and mortality. Approved targeted therapies are limited for pediatric subjects, and treatments are widely adopted from adult algorithms. Macitentan is a safe and effective drug used for adult PH, but data on pediatric patients are limited. In this prospective single-center study, we investigated mid- and long-term effects of macitentan in children with advanced pulmonary hypertensive vascular disease. METHODS: Twenty-four patients were enrolled in the study for treatment with macitentan. Efficacy was determined by echo parameters and brain natriuretic peptide levels (BNP) at 3 months and 1 year. For detailed analysis, the entire cohort was subgrouped into patients with congenital heart disease-related PH (CHD-PH) and non-CHD-PH patients, respectively. RESULTS: Mean age of the patients was 10.7 ± 7.6 years; median observation period was 36 months. Twenty of 24 patients were on additional sildenafil and/or prostacyclins. Two of 24 patients discontinued because of peripheral edema. Within the entire cohort, BNP levels and all echo measures such as right ventricular systolic pressure (RVSP), right ventricular end-diastolic diameter (RVED), tricuspid annular plane systolic excursion (TAPSE), pulmonary velocity time integral (VTI), and pulmonary artery acceleration time (PAAT) improved significantly after 3 months (p ≤ 0.01), whereas in the long term significant improvement persisted for BNP levels (-16%), VTI (+14%) and PAAT (+11%) (p < 0.05). By subgroup analysis, non-CHD PH patients showed significant improvements in BNP levels (-57%) and all echo measures (TAPSE +21%, VTI +13%, PAAT +37%, RVSP -24%, RVED -12%) at 3 months (p ≤ 0.01), whereas at 12 months, improvements persisted (p < 0.05) except for RVSP and RVED (nonsignificant). In CHD-PH patients, none of the measures changed (nonsignificant). 6-MWD (distance walked in 6 minutes) slightly increased but was not statistically evaluated. CONCLUSION: Data presented herein account for the largest cohort of severely affected pediatric patients receiving macitentan. Overall, macitentan was safe and associated with significant beneficial effects and sustained positive signals after 1 year, albeit in the long term disease progression remains a major concern. Our data suggest limited efficacy in CHD-related PH, whereas favorable outcomes were mainly driven by improvements in patients with PH not related to CHD. Larger studies are needed to verify these preliminary results and to prove efficacy of this drug in different pediatric PH entities.

Aortic Valve Repair in Pediatric Patients: 30 Years Single Center Experience.

BACKGROUND: Valve repair is the procedure of choice for congenital aortic valve disease. With increasing experience, the surgical armamentarium broadened from simple commissurotomy to more complex techniques. We report our 30-year experience with pediatric aortic valve repair. METHODS: A retrospective chart review of all patients aged less than 18 years who underwent aortic valve repair from May 1985 to April 2020 was conducted. Mortality was cross-checked with the national health insurance database (96% complete mortality follow-up in April 2020). Primary study endpoints were survival and incidence of reoperations. RESULTS: From May 1985 until April 2020, 126 patients underwent aortic valve repair at a median age of 1.8 years (interquartile range, 0.2-10). Early mortality was 5.6% (7 of 126). All early deaths occurred in neonates with critical aortic stenosis undergoing commissurotomy. No early deaths were observed after 2002. Kaplan-Meier estimated survival was 90.8% (95% CI, 84.0-94.8) at 10 years, 86.9% (95% CI, 78.7-92.2) at 20 years, and 83.5% (95% CI, 71.7-90.6) at 30 years. The cumulative incidence of aortic valve replacement was 37% (95% CI, 27.7-46.3) at 10 years, 62.2% (95% CI, 50.1-72.1) at 20 years, and 67.4% (51.2-79.2) at 30 years. Nine patients had undergone re-repair of the aortic valve. The majority of valve replacements were Ross procedures. CONCLUSIONS: Our results support a repair-first strategy for patients with congenital heart disease and underline that aortic valve reconstruction can be a successful long-term solution. Longevity did not differ between aortic valve commissurotomy and complex aortic valve reconstruction.

Autosomal recessive cutis laxa type Ib-Successful redo aortic root and arch replacement.

We present an adolescent girl with a highly stenotic ascending aortic conduit of her former during infancy corrected giant aneurysm. Genetic testing determined autosomal recessive cutis laxa type-Ib as the underlying connective tissue disorder. Re-do valve sparing root and arch replacement gained excellent restoration of the aorta; 1-year-follow-up was uneventful.

Long-term outcomes after surgical repair of subvalvular aortic stenosis in pediatric patients.

Objectives: Subvalvular aortic stenosis (SAS) can occur as discrete or tunnel-like obstruction of the left ventricular outflow tract and as progressive disease often leads to aortic valve regurgitation. We report our 30-year single-center experience after surgical repair of SAS. Methods: A retrospective chart review of all patients aged < 18 years, who underwent surgical repair of SAS from May 1985 to April 2020, was conducted. Mortality was cross-checked with the national health insurance database (93.8% complete mortality follow-up in April 2020). Survival and competing risks analysis were used to analyze the primary endpoints survival and incidence of reoperations. Results: From May 1985 until April 2020 103 patients (median age 5.5 years) underwent surgical repair of SAS. Survival was 90.8% at 10 years and 88.7% at 20 and 30 years. Age < 1 year at time of surgery, Shone's complex, mitral stenosis and concomitant mitral valve surgery were associated with mortality. The cumulative incidence of reoperation for SAS was 21.6% at 10 years, 28.2% at 20 and 30 years. The incidence of reoperation for SAS did not differ between the myectomy, membrane resection and combined myectomy and membrane resection groups. The cumulative incidence of reoperation on the aortic valve was 13.5% at 20 years. Conclusion: Recurrence rate of SAS is not to be neglected, though surgical repair of subaortic stenosis has good long-term results. Patients who needed a combined membrane resection and septal myectomy are not more prone to recurrence than patients who underwent solitaire myectomy or membrane resection.

[Intergenerational care of patients with tachycardiac arrhythmias]. / Generationenübergreifende Betreuung von Patienten mit tachykarden Rhythmusstörungen.

Within the last decades the treatment options for patients with arrhythmias have developed from a purely conservative drug treatment to a really curative treatment with removal of the arrhythmogenic substrate by continuously more sophisticated possibilities in the sense of electrophysiological techniques and ablation. Parallel to this, special outpatient departments of cardiac rhythmology for the care of patients with ion channelopathies have also become established in pediatric cardiology centers. Their task is the intergenerational care of whole families, with the aim of primarily preventing malignant arrhythmias by appropriate counselling and guidance.

Discordant Post-natal Patterns in Fetuses With Heterotaxy Syndrome: A Retrospective Single-Centre Series on Outcome After Fetal Diagnosis.

Objective: Cardiac and extra-cardiac anomalies in 46 pre-natally diagnosed cases of heterotaxy were compared to post-natal anatomical patterns in order to reveal discordant findings. Second, the outcome of these fetuses was evaluated. Methods: Fetuses with heterotaxy, diagnosed in a tertiary referral centre, were analysed retrospectively. Based on the foetal abdominal situs view, right atrial isomerism (RAI) and left atrial isomerism (LAI) were defined as foetal sub-types. Post-natally, discordant anatomical patterns for broncho-pulmonary branching, atrial appendage morphology, and splenic status were further clarified with CT scans. In summary, the spectrum of pre-natally and post-natally detected cardiac and extra-cardiac anomalies is systematically reviewed. Necessary surgical interventions and mid-long-term outcomes were compared between the two sub-types in surviving infants. Results: A total of 46 fetuses with heterotaxy were included; LAI was diagnosed in 29 (63%) fetuses and RAI was diagnosed in 17 (37%) fetuses. Extra-cardiac anomalies were noted in 35% of fetuses. Seven out of the 29 fetuses (24%) with LAI had atrio-ventricular block (AVB) and four of these cases presented with hydrops. Twenty nine out of the 46 participating fetuses (63%) were live births, with 62% in the LAI group and 65% in the RAI group. Five fetuses were lost to follow-up. At the age of 1 year, the overall survival of live births [estimate (95% CI)] was 67% (48; 92%) in patients with LAI and 55% (32; 94%) in patients with RAI. At the age of 5 years, the estimates were 67% (48; 92%) in the LAI group and 46% (24-87%) in the RAI group. The median survival (first quartile; third quartile) was 11.1 (0.1; 14) years for patients with LAI and 1.3 (0.09; NA) years for patients with RAI. Of 17 children who had undergone cardiac surgery, five (29%) children achieved a bi-ventricular repair and 12 (70%) children achieved a uni-ventricular palliation. Three were primarily palliated, but converted to bi-ventricular thereafter. Foetal subtype definition of heterotaxy based on the abdominal situs and post-natal thoracic imaging studies showed a discordant pattern of broncho-pulmonary branching and atrial appendage anatomy in 40% of our live-born children. Conclusion: Heterotaxy is a rare and complex condition with significant morbidity and mortality related to severe cardiac and extra-cardiac associations. Accurate pre-natal diagnosis can help identify the fetuses at risk and allow for timely intervention in a multi-disciplinary setting. Further studies are warranted to shed light on the exact sub-type definition in fetuses with heterotaxy and the presence of discordant post-natal patterns.

First-in-man use of the EXCOR Venous Cannula for combined cavopulmonary and systemic ventricular support in Fontan circulation failure.

BACKGROUND AND AIM: The Berlin Heart EXCOR system has been developed for mechanical circulatory support (MCS) of pediatric patients with terminal heart failure. A recently introduced iteration of the system (EXCOR Venous Cannula, Berlin Heart GmbH, Berlin, Germany) is dedicated to support patients with univentricular physiologies by facilitating implantation of the EXCOR device into the Fontan pathway. CASE PRESENTATION: We report the worldwide first successful implantation of the EXCOR Venous Cannula in a biventricular support concept for a 12-year-old boy (140 cm, 42.7 kg, body surface area 1.29 m2, Pedimacs Level 2) with severe systemic ventricle dysfunction and failing Fontan circulation. Surgery comprised of standard Berlin Heart EXCOR implantation to support the failing ventricle (12 mm apex / staged 12/9 mm arterial cannula / 50 ml ventricle). Cannulation for subpulmonary EXCOR support was achieved by performing a total cavopulmonary connection takedown with subsequent anastomosis of a staged 12/9 mm outflow cannula to the pulmonary artery and implantation of a 14/18 mm EXCOR Venous Cannula as subpulmonary inflow graft, which was connected to the superior vena cava and Fontan tunnel using GORE-TEX grafts. In the postoperative course, cardiac output and central venous pressures rapidly improved with hepatic and renal functions restoring to age- and condition-specific norm values. CONCLUSION: The Berlin Heart EXCOR Venous Cannula is the first system for standardized mechanical support of Fontan circulatory failure. In our patient, subpulmonary support restoring a biventricular circulation combined with systemic MCS normalized hemodynamics and reversed end-organ dysfunction.

SARS-CoV-2 Associated Pediatric Inflammatory Multisystem Syndrome With a High Prevalence of Myocarditis - A Multicenter Evaluation of Clinical and Laboratory Characteristics, Treatment and Outcome.

Introduction: Pediatric inflammatory multisystem syndrome - temporally associated with SARS-CoV-2 infection (PIMS -TS) comprises a new disease entity having emerged after the COVID-19 outbreak in 2019. Materials and Methods: For this multicenter, retrospective study children between 0 and 18 years with PIMS-TS between March 2020 and May 2021 were included, before availability of vaccination for children. Frequent SARS-CoV-2 variants at that period were the wildtype virus, alpha, beta and delta variants. Inclusion criteria were according to the PIMS-TS criteria, proposed by the Royal College of Pediatrics and WHO. Study aim was to review their clinical, laboratory and echocardiographic data with a focus on cardiac involvement. Results: We report 45 patients, median age 9 years, 64% male. SARS-CoV-2 antibodies were positive in 35/41 (85%). PIMS occurrence followed local COVID-19 peak incidence periods with a time lag. The most common symptoms at presentation were fever (98%), abdominal pain (89%) and rash (80%). Fever history of > 5 days was associated with decreased left ventricular function (p = 0.056). Arterial hypotension and cardiac dysfunction were documented in 72% patients, increased brain natriuretic peptide in 96% and increased cardiac troponin in 64% of the children. Echocardiography revealed mitral valve regurgitation (64%), coronary abnormalities (36%) and pericardial effusions (40%). Increased NT-proBNP was significantly associated with the need of inotropics (p < 0.05), which were necessary in 40% of the patients. Treatment comprised intravenous immunoglobulin (93%), systemic steroids (84%) and acetylsalicylic acid (100%; 26/45 started with high dosages). For insufficient response to this treatment, five (11%) children received the interleukin-1 receptor antagonist anakinra. All patients were discharged with almost resolved cardiac signs. Conclusion: Our analysis of non-vaccinated children with PIMS-TS demonstrates that a considerable number have associated myocarditis requiring intensive care and inotropic support. Most children showed adequate response to intravenous immunoglobulin and steroids and good recovery. Further evaluation of pediatric patients with COVID-19 associated diseases is required to evaluate the impact of new virus variants.

Paediatric and adult congenital cardiology education and training in Europe.

BACKGROUND: Limited data exist on training of European paediatric and adult congenital cardiologists. METHODS: A structured and approved questionnaire was circulated to national delegates of Association for European Paediatric and Congenital Cardiology in 33 European countries. RESULTS: Delegates from 30 countries (91%) responded. Paediatric cardiology was not recognised as a distinct speciality by the respective ministry of Health in seven countries (23%). Twenty countries (67%) have formally accredited paediatric cardiology training programmes, seven (23%) have substantial informal (not accredited or certified) training, and three (10%) have very limited or no programme. Twenty-two countries have a curriculum. Twelve countries have a national training director. There was one paediatric cardiology centre per 2.66 million population (range 0.87-9.64 million), one cardiac surgical centre per 4.73 million population (range 1.63-10.72 million), and one training centre per 4.29 million population (range 1.63-10.72 million population). The median number of paediatric cardiology fellows per training programme was 4 (range 1-17), and duration of training was 3 years (range 2-5 years). An exit examination in paediatric cardiology was conducted in 16 countries (53%) and certification provided by 20 countries (67%). Paediatric cardiologist number is affected by gross domestic product (R2 = 0.41). CONCLUSION: Training varies markedly across European countries. Although formal fellowship programmes exist in many countries, several countries have informal training or no training. Only a minority of countries provide both exit examination and certification. Harmonisation of training and standardisation of exit examination and certification could reduce variation in training thereby promoting high-quality care by European congenital cardiologists.

Copeptin: Prognostic Relevance as a Perioperative Marker in Pediatric Cardiac Surgery.

BACKGROUND: Copeptin is a cleavage product of vasopressin. This study aimed to figure out if copeptin would be a suitable biomarker in patients with congenital heart disease in the postoperative course. METHODS: The primary outcome endpoint of this study was the change in copeptin concentration perioperatively in patients with congenital heart disease after surgery, with the use of a cardiopulmonary bypass. Three blood samples were taken from 81 patients up to 6 years of age in order to evaluate changes in copeptin concentration. RESULTS: Significant increase of copeptin concentration was shown between the first and second blood draws as well as between the first and third blood draws (Ps < .001). Additionally, positive and significant correlations (r ≥ .27) between the cardiopulmonary bypass times, The Society of Thoracic Surgeons and European Association for Cardio-Thoracic Surgery mortality category, the inotropic score, the duration of mechanical ventilation, the length of stay at the intensive care unit (ICU), the length of stay at the hospital, and the preoperative as well as the ICU copeptin levels were found. CONCLUSIONS: Copeptin showed a tendency to predict the clinical outcome of patients after congenital heart surgery. Patients with higher copeptin levels underwent more complex procedures, had longer cardiopulmonary bypass times, required more catecholamine support, needed longer time of invasive ventilation, and had longer overall stay and ICU stay.

Initial Angiotensin Receptor Blocker Response in Young Marfan Patients Decreases After 3 Years of Treatment.

Marfan syndrome is caused by mutations of the fibrillin-1 gene, which weakens the connective tissue integrity. Since 2003, bioavailability regulations of TGF-ß through fibrillin alterations have been presumed of being the culprit mechanisms for aortic aneurysm development. We present the analysis of our single-center Marfan children and adolescents cohort to assess the influence of age, sex, degree of cardiovascular involvement and dosage on losartan effectivity. This prospective longitudinal registered echocardiographical investigation (EudraCT 2009-016139-36) of 49 patients with an average follow-up of 72 months focused on aortic root z-scores, elasticity, and yearly progression rates. The 33 patients under medication with losartan showed an aortic root z-score reduction during the first 36 months compared to 22 patients without medication presenting constant mild progression. Yet, results diminished under losartan thereafter, adding up to similar progressions over 72 months in both groups (0.07 ± 0.10/year versus 0.04 ± 0.11/year). Although male patients exhibited higher root z-scores, progression with and without medication was comparable to females and not age-dependent. In conclusion, losartan evoked a significant aortic root z-score regression in young Marfan patients over the first 3 years, but this effect mitigated thereafter. The initial improvement concurred with ameliorated elasticity; lower stiffness levels predicted better clinical outcome, but likewise only up to 36 months. Sex differences in dilatation severity were observed but neither age nor sex had significant influence on progression rates. Losartan dosages were gradually increased in more severely affected patients and provided an equal rate of root progression over 72 months in comparison to patients under losartan treatment with lesser baseline dilatation severity.

Percutaneous treatment of a huge patent ductus venosus and severe portal vein hypoplasia using a Figulla Flex II atrial septal defect occluder in a 2-year-old infant: a case report.

BACKGROUND: Intra- or extrahepatic porto-caval shunts (PCSs) can account for multiorgan dysfunction with pulmonary arterial hypertension and portosystemic encephalopathy as the most serious consequences of bypass of the hepatic circulation. The ductus venosus (DV) represents a rare foetal PCS and might be persistently patent in newborns after birth. Treatment strategies include surgical ligation and percutaneous device closure. The degree of portal vein hypoplasia limits therapy making liver transplantation the only option in some of them. CASE SUMMARY: In a newborn female patient a huge persistently patent DV, known already prenatally, resulted in severe secondary portal vein hypoplasia. She presented with hyperammonaemia, elevated liver enzymes, and pulmonary hypertension. With only diminutive portal venous branches and exceedingly high portal venous pressures during test-occlusion of the DV, shunt closure was not possible. At the age of 2 years more favourable portal venous pressures allowed transcatheter device closure with a nitinol atrial septal defect occlusion device. Pulmonary artery pressures and ammonia levels normalized after the procedure without any signs of portal hypertension. DISCUSSION: The case highlights the importance of meticulous imaging using balloon occlusion angiography of PCSs like the DV, to search for intrahepatic portal veins. Moreover, portal vein pressure during test-occlusion can identify patients amenable for surgical or endovascular shunt closure. Occlusion devices licensed for other indications like atrial septal defect closure can be used safely in huge PCS vessels in a one-step or staged procedure. Optimal timing of the intervention should be tailored to the patient's needs.

A guide to echocardiographic assessment in children and adolescents with pulmonary hypertension.

While the current definition of pulmonary hypertension (PH) is still based on haemodynamic variables, transthoracic echocardiography is the most important diagnostic clinical tool for the first assessment and evaluation of a patient, in whom PH is suspected. In addition, it is the most important clinical modality in long term follow-up and the utility of echocardiography has widely been demonstrated in patients with PH. Echocardiography not only reveals the underlying cardiac morphology and diagnosis of any associated cardiac defects. In most patients with PH right ventricular (RV) pressure estimation is feasible. In addition, ventricular systolic and diastolic function, as well as ventricular-ventricular interactions of both ventricles can be assessed by using echocardiography. Maximizing the use of echocardiography by reporting several measures to gain information and quantitatively describe the parameters, that are linked to prognosis, seem particularly appealing in these children, in whom other advanced imaging modalities requiring anaesthesia is associated with a considerable risk. Herein we provide a practical approach and a concise and clinically applicable echocardiographic guidance and present basic variables, which should be obtained at any assessment. Moreover, we present additional advanced echocardiographic measures, that can be applied in a research or clinical setting when progressive PH needs a deeper insight to assess heart function, estimation of pulmonary artery pressures among others, by echocardiography. Finally, clinically relevant studies in view of the prognostic properties with a focus on the most important echocardiographic variables in pediatric PH are summarized.

Characteristics, management, and outcome of pediatric patients with post-transplant lymphoproliferative disease-A 20 years' experience from Austria.

BACKGROUND: Management of pediatric post-transplantation lymphoproliferative disorder (PTLD) after hematopoietic stem cell (HSCT) and solid organ transplantation (SOT) is challenging. AIM: This study of 34 PTLD patients up to 19-years old diagnosed in Austria from 2000 to 2018 aimed at assessing initial characteristics, therapy, response, and outcome as well as prognostic markers of this rare pediatric disease. METHODS AND RESULTS: A retrospective data analysis was performed. Types of allografts were kidney (n = 12), liver (n = 7), heart (n = 5), hematopoietic stem cells (n = 4), lungs (n = 2), multi-visceral (n = 2), small intestine (n = 1), and vessels (n = 1). Eighteen/34 were classified as monomorphic PTLD, with DLBCL accounting for 15 cases. Polymorphic disease occurred in nine, and non-destructive lesions in six cases. One patient had a non-classifiable PTLD. Thirteen/34 patients are surviving event-free in first remission (non-destructive, n = 4/6; polymorphic, n = 4/9; monomorphic, n = 6/18). Fourteen/34 patients lacked complete response to first-line therapy, of whom seven died. Four/34 patients relapsed, of whom two died. In 3/34 patients, death occurred as a first event. The 5-year overall and event-free survival rates were 64% ± 9% and 35% ± 9% for the whole cohort. Among all parameters analyzed, only malignant disease as the indication for transplantation had a significantly poor influence on survival. CONCLUSIONS: This study shows PTLD still to be a major cause of mortality following SOT or HSCT in children. A continued understanding of the molecular biology of the disease shall allow to decrease treatment intensity for lower risk patients and to identify patients who may benefit from newer therapy approaches to improve outcome and decrease morbidity.