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.

Follow-up echocardiographic changes in children and youth aged <25 years with latent rheumatic heart disease: A systematic review and meta-analysis of global data.

OBJECTIVES: To estimate progression, regression and persistence rates for borderline and mild-definite latent RHD in children and youth diagnosed at age < 25 years. METHODS: A review was conducted in accordance with Preferred Reporting Items for Systematic reviews and Meta-Analysis guidelines. Electronic databases were searched for latent RHD echocardiography follow-up studies which used World Heart Federation diagnostic criteria. A meta-analysis of outcomes was conducted for borderline and mild-definite disease subcategories. RESULTS: Data for 1618 individuals from 12 studies were included. For borderline cases, 48.51% regressed (95%CI 45.10-51.93), 13.99% progressed (95%CI 9.72-18.25), and 38.61% had persistent (unchanged) disease at follow-up (95%CI 29.68-47.54). For mild-definite cases, 34.01% regressed (95%CI 28.88-39.15), 8.06% progressed (95%CI 3.65-16.90), and 60.23% had persistent disease (95%CI 55.08-67.38). CONCLUSIONS: Borderline and mild-definite latent RHD show variable evolution following initial diagnosis. While 8% of mild-definite and 14% borderline cases had signs of disease progression at follow-up, a third of mild-definite and half of borderline cases had disease regression, even with sub adequate antibiotic prophylaxis. The significant variability between study cohorts suggests latent RHD natural history is likely variable between different endemic regions globally. Future research is needed to identify those individuals who would most benefit from antibiotic prophylaxis and determine regional natural history of latent RHD.

Shear-wave elastography for monitoring Fontan-associated liver disease: A prospective cohort study.

The spectrum of Fontan-associated liver disease (FALD) varies from abnormal liver function tests to fibrosis and even cirrhosis. In this prospective study, we evaluated the role of shear-wave elastography (SWE) in predicting the presence of advanced FALD. Forty-eight patients (30 males, 13.9 [6-21] years) with a Fontan circulation were evaluated at 8.3 (2.1-18.7) years since the Fontan surgery. The median liver stiffness measurement (LSM) value was higher than values in normal children at 15.4 (9.5-38.7) kPa. The LSMs had a weak but significant correlation with age at the time of LSM (r = 0.25, p = 0.01) and duration post-Fontan surgery (r = 0.31, p = 0.02). It had a poor correlation with the concomitant aspartate transaminase-to-platelet ratio index (r = 0.1, p = 0.39). No difference in the elastography values between children with and without ultrasound evidence of advanced liver disease (17.7 [interquartile range, IQR: 4] vs. 16.1 [IQR: 6], p = 0.62] was observed. Further studies are required to determine the precise role of SWE as a noninvasive marker of liver fibrosis in FALD.

Australian National Standards of Care for Childhood-onset Heart Disease (CoHD Standards). 1st Edition.

These first Australian National Standards of Care for Childhood-onset Heart Disease (CoHD Standards) have been developed to inform the healthcare requirements for CoHD services and enable all Australian patients, families and carers impacted by CoHD (paediatric CoHD and adult congenital heart disease [ACHD]) to live their best and healthiest lives. The CoHD Standards are designed to provide the clarity and certainty required for healthcare services to deliver excellent, comprehensive, inclusive, and equitable CoHD care across Australia for patients, families and carers, and offer an iterative roadmap to the future of these services. The CoHD Standards provide a framework for excellent CoHD care, encompassing key requirements and expectations for whole-of-life, holistic and connected healthcare service delivery. The CoHD Standards should be implemented in health services in conjunction with the National Safety and Quality Health Service Standards developed by the Australian Commission on Safety and Quality in Health Care. All healthcare services should comply with the CoHD Standards, as well as working to their organisation's or jurisdiction's agreed clinical governance framework, to guide the implementation of structures and processes that support safe care.

Implementing neurodevelopmental follow-up care for children with congenital heart disease: A scoping review with evidence mapping.

AIM: To identify and map evidence describing components of neurodevelopmental follow-up care for children with congenital heart disease (CHD). METHOD: This was a scoping review of studies reporting components of neurodevelopmental follow-up programmes/pathways for children with CHD. Eligible publications were identified through database searches, citation tracking, and expert recommendations. Two independent reviewers screened studies and extracted data. An evidence matrix was developed to visualize common characteristics of care pathways. Qualitative content analysis identified implementation barriers and enablers. RESULTS: The review included 33 studies. Twenty-one described individual care pathways across the USA (n = 14), Canada (n = 4), Australia (n = 2), and France (n = 1). The remainder reported surveys of clinical practice across multiple geographical regions. While heterogeneity in care existed across studies, common attributes included enrolment of children at high-risk of neurodevelopmental delay; centralized clinics in children's hospitals; referral before discharge; periodic follow-up at fixed ages; standardized developmental assessment; and involvement of multidisciplinary teams. Implementation barriers included service cost/resourcing, patient burden, and lack of knowledge/awareness. Multi-level stakeholder engagement and integration with other services were key drivers of success. INTERPRETATION: Defining components of effective neurodevelopmental follow-up programmes and care pathways, along with enhancing and expanding guideline-based care across regions and into new contexts, should continue to be priorities. WHAT THIS PAPER ADDS: Twenty-two different neurodevelopmental follow-up care pathways/programmes were published, originating from four countries. Twelve additional publications described broad practices for neurodevelopmental follow-up across regions Common attributes across eligibility, service structure, assessment processes, and care providers were noted. Studies reported programme acceptability, uptake, cost, and effectiveness. Implementation barriers included service cost/resourcing, patient burden, and lack of knowledge/awareness.

Utility of routine chest radiographs after chest drain removal in paediatric cardiac surgical patients-a retrospective analysis of 1076 patients.

OBJECTIVES: Chest drains are routinely placed in children following cardiac surgery. The purpose of this study was to determine the incidence of a clinically relevant pneumothorax and/or pleural effusion after drain removal and to ascertain if a chest radiograph can be safely avoided following chest drain removal. METHODS: This single-centre retrospective cohort study included all patients under 18 years of age who underwent cardiac surgery between January 2015 and December 2019 with the insertion of mediastinal and/or pleural drains. Exclusion criteria were chest drain/s in situ ≥14 days and mortality prior to removal of chest drain/s. A drain removal episode was defined as the removal of ≥1 drains during the same episode of analgesia ± sedation. All chest drains were removed using a standard protocol. Chest radiographs following chest drain removal were reviewed by 2 investigators. RESULTS: In all, 1076 patients were identified (median age: 292 days, median weight: 7.8 kg). There were 1587 drain removal episodes involving 2365 drains [mediastinal (n = 1347), right pleural (n = 598), left pleural (n = 420)]. Chest radiographs were performed after 1301 drain removal episodes [mediastinal (n = 1062); right pleural (n = 597); left pleural (n = 420)]. Chest radiographs were abnormal after 152 (12%) drain removal episodes [pneumothorax (n = 43), pleural effusion (n = 98), hydropneumothorax (n = 11)]. Symptoms/signs were present in 30 (2.3%) patients. Eleven (<1%) required medical management. One required reintubation and 2 required chest drain reinsertion. CONCLUSIONS: The incidence of clinically significant pneumothorax/pleural effusion following chest drain removal after paediatric cardiac surgery is low (<1%). Most patients did not require reinsertion of a chest drain. It is reasonable not to perform routine chest radiographs following chest drain removal in most paediatric cardiac surgical patients.

Systematic Review of Instruments Assessing Psychosocial Adaptation and Outcomes Among Families of Children With Congenital Heart Disease.

OBJECTIVE: This systematic review identified instruments quantitatively assessing psychosocial adaptation and outcomes in families of children with congenital heart disease (CHD) and evaluated instrument psychometrics. METHODS: Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines and a prospectively registered protocol, electronic databases (CINAHL, Embase, PubMed/MEDLINE, PsycINFO, and SCOPUS) were searched from inception until June 20, 2021 for peer-reviewed articles published in English, reporting quantitative data on psychosocial outcomes among parents/caregivers, siblings, or family system. Instrument characteristics and psychometrics were extracted, and adapted COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) criteria were applied to assess instrument quality. Descriptive statistics and narrative synthesis were used for analysis. RESULTS: Overall, 108 articles reporting on 107 distinct samples across 26 countries met inclusion. Across those articles, 40 instruments assessed psychological functioning or distress, 12 assessed coping, 11 assessed quality of life constructs, 10 assessed parenting stress/caregiver burden, 10 assessed family functioning/impact, 10 assessed stress appraisal, 5 assessed sibling psychosocial outcomes, and 2 assessed couple relationship satisfaction/strain. Applying COSMIN criteria to available data on original instrument development articles/manuals for English language instruments (n = 54), 67% scored a positive property evidence rating for content validity, 39% for internal consistency, 4% for test-retest reliability, and 9% for responsiveness (longitudinal validity). CONCLUSIONS: Studies vary widely in instruments used to assess psychosocial adaptation and outcomes among families of children with CHD. Instrument selection informed by robust key psychometrics, increased psychometric reporting, development of both a "toolkit" approach and a comprehensive CHD-specific family instrument are among key recommendations.

Early Experience With the Bentley BeGraft Aortic Stent for the Management of Aortic Arch Pathology in the Paediatric Population.

BACKGROUND: Aortic arch pathology in older children is often treated preferentially with stenting. Both bare metal and covered stents have been utilised, with potential advantages of covered stents. The search for the ideal covered stent continues. METHODS: Retrospective review of all paediatric patients undergoing treatment of aortic arch pathology utilising the Bentley BeGraft Aortic stent (BeGraft Aortic, Bentley InnoMed, Hechingen, Germany) from June 2017 to May 2021. Outcome measures were procedural success, complications, medium-term patency and need for re-intervention. RESULTS: Fourteen (14) stents were placed in 12 children (seven males). Indications were coarctation of the aorta in 10 and aneurysm in two. Median age was 11.8 years (8.7-16.6 years) and median weight 42.5 kg (24.8-84 kg). Median coarctation narrowing of 4 mm (range 1-9 mm), improved to 11 mm (range 9-15 mm). The median coarctation gradient improved from 32 mmHg (range 11-42 mmHg) to 7 mmHg (range 0-14 mmHg). Both aneurysms were successfully occluded. There was no mortality or major morbidity. In one patient balloon rupture occurred requiring a second balloon for full inflation and one patient had a minor access site bleed. Follow-up median was 28 months (range 13-65 months). One patient underwent repeat balloon dilation for increased blood pressure gradient at 47 months post implant and a second patient additional stent insertion for a mid-stent aneurysm at 65 months. CONCLUSION: The Bentley BeGraft Aortic stent can be safely deployed in children for the treatment of aortic arch pathology. Medium-term patency is acceptable. Longer term follow-up in larger series will be required to assess stent performance.

Clinical stakeholder preferences for paediatric cardiac surgery outcome reporting in Australia and New Zealand.

BACKGROUND: Outcome reporting is an essential element of quality assurance. Evaluation of the information needs of stakeholders of outcome reporting is limited. This study aimed to examine stakeholder preferences for the content, format, and dissemination of paediatric cardiac surgery performance data in Australia and New Zealand. METHODS: Semi-structured interviews were completed with a purposive sample of Queensland stakeholders to evaluate their attitudes and expectations regarding reporting of paediatric cardiac surgery outcomes. The interviews were audio-recorded and transcribed. Two researchers used an interpretive description approach to analyse the transcripts qualitatively. RESULTS: Nineteen stakeholders were interviewed including fifteen clinicians, four parents, one hospital administrator, and one consumer advocate were interviewed. Mortality was highlighted as the area of greatest interest in reports by clinical and consumer groups. The majority preferred hospital rather than individual/clinician-level reporting. Annual reports were preferred by clinicians who requested reports be distributed electronically. CONCLUSIONS: The evidence generated from outcome reporting in paediatric cardiac surgery is highly desired by clinicians, administrators, parents, families, and advocacy groups. Clinical users prefer information to assist in clinical decision-making, while families seek personalised information at crucial time points in their clinical journey.

The Cost of Neurodevelopmental Disability: Scoping Review of Economic Evaluation Methods.

The provision of effective care models for children with neurodevelopmental delay or disability can be challenging in resource constrained healthcare systems. Economic evaluations have an important role in informing resource allocation decisions. This review systematically examined the scope and methods of economic models evaluating interventions for supporting neurodevelopment among children with common neurodevelopmental disorders and identified methods of economic models and presented policy implications. This scoping review employed the Arksey and O'Malley framework and aligned with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses extension for Scoping Reviews (PRISMA-ScR). Four electronic databases were systematically searched to identify eligible model-based economic evaluations of neurodevelopmental care models published since 2000. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist was used to assess quality of reporting. Data were systematically extracted, tabulated, and qualitatively synthesised across diagnostic categories. Searches identified 1431 unique articles. Twelve studies used a decision analytic model to evaluate care for neurodevelopmental disorders and were included in the review. Included studies focused on attention-deficit/hyperactivity disorder (ADHD, n=6), autism spectrum disorder (ASD, n=3), cerebral palsy (n=2), and dyslexia (n=1). The most used decision analytic modelling approach was a Markov model (n=6), followed by a decision tree (n=3), and a combination of decision tree and Markov model (n=3). Most studies (n=7) adopted a societal perspective for reporting costs. None of the reviewed studies modelled impact on families and caregivers. Four studies reported cost-savings, three identified greater quality of life, and three identified cost increases.

Vocal cord dysfunction after pediatric cardiac surgery: A prospective implementation study.

Objective: To determine the incidence, outcomes, and evaluate diagnostic modalities for postoperative vocal cord dysfunction (VCD) following cardiothoracic surgery in children. Methods: A prospective mixed-methods study using principles of implementation science was completed. All patients undergoing surgery involving the aortic arch, ductus, or ligamentum arteriosum and vascular rings from September 2019 to December 2020 were enrolled. Patients underwent speech pathology assessment, laryngeal ultrasound, and flexible direct laryngoscopy. Results: Ninety-five patients were eligible for inclusion. The incidence of VCD ranged from 18% to 56% and varied according to procedure group. VCD occurred in 42% of neonates. Repair of hypoplastic aortic arch was associated with increased risk of VCD (57%; P = .002). There was no significant difference in duration of intubation, pediatric intensive care unit stay, or hospital stay. Forty percent children were able to achieve full oral feeding. Children with VCD were more likely to require nasogastric supplementary feeding at discharge (60% vs 36%; P = .044). Sixty-eight percent of patients demonstrated complete resolution of VCD at a median of 97 days postoperatively. Laryngeal ultrasound and speech pathology assessment combined had a sensitivity of 91% in comparison to flexible direct laryngoscopy. Conclusions: VCD occurred in one-third and resolved in two-thirds of patients at a median of 3 months following cardiac surgery. Aortic arch repair carried the highest risk of VCD. VCD adversely influenced feeding. Forty percent of patients achieved full oral feeding before discharge. VCD did not delay intensive care unit or hospital discharge. Speech pathology assessment and laryngeal ultrasound combined was reliable for diagnosis in most patients and was more patient friendly than flexible direct laryngoscopy.

Integrated guidance to enhance the care of children and adolescents with familial hypercholesterolaemia: Practical advice for the community clinician.

Familial hypercholesterolaemia (FH) is a highly penetrant monogenic disorder present from birth that markedly elevates plasma low-density lipoprotein (LDL)-cholesterol (LDL-C) concentration and, if untreated, leads to premature atherosclerosis and coronary artery disease (CAD). At a prevalence of 1:250 individuals, with over 90% undiagnosed, recent estimates suggest that there are approximately 22 000 children and adolescents with FH in Australia and New Zealand. However, the overwhelming majority remain undetected and inadequately treated until adulthood or after their first cardiac event. The guidance in this paper aims to increase awareness about paediatric FH and provide practical advice for the diagnosis and management of FH in children and adolescents. Recommendations are given on the detection, diagnosis, assessment and management of FH in children and adolescents. Recommendations are also made on genetic testing, including counselling and the potential for universal screening programmes. Practical guidance on management includes treatment of non-cholesterol risk factors, and safe and appropriate use of LDL-C lowering therapies, including statins, ezetimibe, PCSK9 inhibitors and lipoprotein apheresis. Models of care for FH need to be adapted to local and regional health care needs and available resources. Targeting the detection of FH as a priority in children and young adults has the potential to alter the natural history of atherosclerotic cardiovascular disease and recognise the promise of early detection for improving long-term health outcomes. A comprehensive implementation strategy, informed by further research, including assessments of cost-benefit, will be required to ensure that this new guidance benefits all families with or at risk of FH.

Corrigendum to Synopsis of an integrated guidance for enhancing the care of familial hypercholesterolaemia: An Australian perspective [American Journal of Preventive Cardiology 6 (2021) 100151].

[This corrects the article DOI: 10.1016/j.ajpc.2021.100151.].

Fontan operation at less than 3 years of age is not a risk factor for long-term failure.

OBJECTIVES: The age at which the Fontan operation is performed varies globally. Over the last decade, the median age of patients having the Fontan in Australia and New Zealand has been 4.6 years, including 6% of patients younger than 3 years. Long-term outcomes of an early Fontan operation are unclear and are described in this study. METHODS: Patients from the Australian and New Zealand Fontan Registry were grouped by age at Fontan. A Fontan before 3 years (early Fontan) was compared to the combined second and third quartiles by age at surgery in the Registry (3.6-6.1 years; control). Outcomes included Fontan failure (death, transplant, New York Heart Association functional group III/IV heart failure, Fontan takedown or conversion, protein losing enteropathy and plastic bronchitis), arrhythmias, thromboembolism and reinterventions. RESULTS: A total of 191 patients who had early Fontan operations were compared to 781 controls. Profound or progressive cyanosis was noted more frequently in the early than in the control group (63% vs 23%; P < 0.001). The early group was followed up for a median 22.1 years. The incidence of long-term failure was similar between the 2 groups (early, 1.08 failures per 100 patient-years of follow-up vs control, 0.99; log-rank P = 0.79). Adjusted for risk factors, early age at Fontan was not a risk factor for long-term failure [hazard ratio (HR) 1.16, 95% confidence interval (CI) 0.77-1.76; P = 0.48], new-onset arrhythmia (HR 0.93, 95% CI 0.63-1.39; P = 0.73), thromboembolism (HR 0.50, 95% CI 0.28-0.91; P = 0.024) or reintervention (HR 1.08, 95% CI 0.80-1.45; P = 0.62). CONCLUSIONS: Having the Fontan operation at an early age was not a risk factor for short- or long-term adverse outcomes in our cohort.

The "Super-Fontan" Phenotype: Characterizing Factors Associated With High Physical Performance.

Background: People with a Fontan circulation usually have moderately impaired exercise performance, although a subset have high physical performance ("Super-Fontan"), which may represent a low-risk phenotype. Methods: People with a "Super-Fontan" phenotype were defined as achieving normal exercise performance [≥80% predicted peak oxygen uptake (VO2) and work rate] during cardiopulmonary exercise testing (CPET) and were identified from the Australian and New Zealand Fontan Registry. A Fontan control group that included people with impaired exercise performance (<80% predicted VO2 or work rate) was also identified based on a 1:3 allocation ratio. A subset of participants were prospectively recruited and completed a series of physical activity, exercise self-efficacy, and health-related quality of life questionnaires. Results: Sixty CPETs ("Super-Fontan", n = 15; control, n = 45) were included. A subset ("Super-Fontan", n = 10; control, n = 13) completed a series of questionnaires. Average age was 29 ± 8 years; 48% were males. Exercise capacity reflected by percent predicted VO2 was 67 ± 17% in the entire cohort. Compared to the "Super-Fontan" phenotype, age at Fontan completion was higher in controls (4.0 ± 2.9 vs. 7.2 ± 5.3 years, p = 0.002). Only one (7%) person in the "Super-Fontan" group had a dominant right ventricle compared to 15 (33%) controls (p = 0.043). None of those in the "Super-Fontan" group were obese, while almost a quarter (22%) of controls were obese based on body mass index (p = 0.046). Lung function abnormalities were less prevalent in the "Super-Fontan" group (20 vs. 70%, p = 0.006). Exercise self-efficacy was greater in the "Super-Fontan" group (34.2 ± 3.6 vs. 27.9 ± 7.2, p = 0.02). Self-reported sports participation and physical activity levels during childhood and early adulthood were higher in the "Super-Fontan" group (p < 0.05). The total average time spent participating in structured sports and physical activity was 4.3 ± 2.6 h/wk in the "Super-Fontan" group compared to 2.0 ± 3.0 h/wk in controls, p = 0.003. There were no differences in self-reported current total physical activity score or health-related quality of life between groups (p ≥ 0.05). Conclusions: The "Super-Fontan" phenotype is associated with a healthy weight, lower age at Fontan completion, better exercise self-efficacy, and higher overall levels of sport and physical activity participation during physical development.

Synopsis of an integrated guidance for enhancing the care of familial hypercholesterolaemia: an Australian perspective.

INTRODUCTION: Familial hypercholesterolaemia (FH) is a common, heritable and preventable cause of premature coronary artery disease, with significant potential for positive impact on public health and healthcare savings. New clinical practice recommendations are presented in an abridged guidance to assist practitioners in enhancing the care of all patients with FH. MAIN RECOMMENDATIONS: Core recommendations are made on the detection, diagnosis, assessment and management of adults, children and adolescents with FH. There is a key role for general practitioners (GPs) working in collaboration with specialists with expertise in lipidology. Advice is given on genetic and cholesterol testing and risk notification of biological relatives undergoing cascade testing for FH; all healthcare professionals should develop skills in genomic medicine. Management is under-pinned by the precepts of risk stratification, adherence to healthy lifestyles, treatment of non-cholesterol risk factors, and appropriate use of low-density lipoprotein (LDL)-cholesterol lowering therapies, including statins, ezetimibe and proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors. Recommendations on service design are provided in the full guidance. POTENTIAL IMPACT ON CARE OF FH: These recommendations need to be utilised using judicious clinical judgement and shared decision making with patients and families. Models of care need to be adapted to both local and regional needs and resources. In Australia new government funded schemes for genetic testing and use of PCSK9 inhibitors, as well as the National Health Genomics Policy Framework, will enable adoption of these recommendations. A broad implementation science strategy is, however, required to ensure that the guidance translates into benefit for all families with FH.

Essentials of a new clinical practice guidance on familial hypercholesterolaemia for physicians.

Familial hypercholesterolaemia (FH) is a common, heritable and preventable cause of premature coronary artery disease. New clinical practice recommendations are presented to assist practitioners in enhancing the care of all patients with FH. Core recommendations are made on the detection, diagnosis, assessment and management of adults, children and adolescents with FH. Management is under-pinned by the precepts of risk stratification, adherence to healthy lifestyles, treatment of non-cholesterol risk factors and appropriate use of low-density lipoprotein (LDL)-cholesterol-lowering therapies including statins, ezetimibe and proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors. The recommendations need to be utilised using judicious clinical judgement and shared decision-making with patients and families. New government-funded schemes for genetic testing and use of PCSK9 inhibitors, as well as the National Health Genomics Policy Framework, will enable adoption of the recommendations. However, a comprehensive implementation science and practice strategy is required to ensure that the guidance translates into benefit for all families with FH.

The Gore Septal Occluder (GSO) for Multiple Indications in Children - An Intention to Treat Analysis.

OBJECTIVE: To assess the safety and efficacy of all uses of the Gore septal occluder (GSO) (WL Gore and Associates, Flagstaff, AZ, USA) in a tertiary children's hospital. BACKGROUND: The GSO is widely used in atrial septal communication closure in the adult population. Usage in the paediatric population is expanding. Device design potentially confers some benefits in this population. METHODS: Single centre retrospective data review over 6 years to July 2019 in a tertiary paediatric cardiac centre. All instances of intended GSO use were identified. Outcome measures were closure rates and procedural complications. RESULTS: Fifty-four (54) patients with median age 7.9 years (3-17 years) and weight 26.5 kg (12.6-76 kg) underwent attempted GSO implantation. Indications were atrial septal defect (ASD) with volume loading (39); abolition of right to left shunt (8); stroke prevention (3) and Fontan fenestration (4). Successful GSO deployment in 46/50 (92%) of the ASD/PFO group and 100% of the Fontan group. Occlusion rate was assessed at 24 hours and at medians of 1.4 months (IQR 1.1 mo), 9 months (IQR 8.3 mo) and 25.4 months (IQR 28.1 mo). Complete occlusion documented at these points was 91%, 87%, 93% and 98% respectively. Major complications prevented GSO implantation in four patients. Two (2) patients had transient rhythm disorder. There was no incidence of erosion, further arrhythmia, venous access complications or death during follow-up. CONCLUSIONS: The GSO can be safely deployed in both the atrial septum and Fontan fenestration with a low rate of complications. Trivial residual shunt is present in a number of cases early after deployment, although improves with time. Delivery sheath size was not a cause of significant complications in this patient cohort.

Balloon dilatation versus surgical valvotomy for congenital aortic stenosis: a propensity score matched study.

BACKGROUND: Balloon valvuloplasty and surgical aortic valvotomy have been the treatment mainstays for congenital aortic stenosis in children. Choice of intervention often differs depending upon centre bias with limited relevant, comparative literature. OBJECTIVES: This study aims to provide an unbiased, contemporary matched comparison of these balloon and surgical approaches. METHODS: Retrospective analysis of patients with congenital aortic valve stenosis who underwent balloon valvuloplasty (Queensland Children's Hospital, Brisbane) or surgical valvotomy (Royal Children's Hospital, Melbourne) between 2005 and 2016. Patients were excluded if pre-intervention assessment indicated ineligibility to either group. Propensity score matching was performed based on age, weight, and valve morphology. RESULTS: Sixty-five balloon patients and seventy-seven surgical patients were included. Overall, the groups were well matched with 18 neonates/25 infants in the balloon group and 17 neonates/28 infants in the surgical group. Median age at balloon was 92 days (range 2 days - 18.8 years) compared to 167 days (range 0 days - 18.1 years) for surgery (rank-sum p = 0.08). Mean follow-up was 5.3 years. There was one late balloon death and two early surgical deaths due to left ventricular failure. There was no significant difference in freedom from reintervention at latest follow-up (69% in the balloon group and 70% in the surgical group, p = 1.0). CONCLUSIONS: Contemporary analysis of balloon aortic valvuloplasty and surgical aortic valvotomy shows no difference in overall reintervention rates in the medium term. Balloon valvuloplasty performs well across all age groups, achieving delay or avoidance of surgical intervention.