Patient- and proxy-reported outcomes (PROs) are an important indicator of healthcare quality and can be used to inform treatment. Despite the widescale use of PROs in adult cardiology, they are underutilised in paediatric cardiac care. This study describes a six-center feasibility and pilot experience implementing PROs in the paediatric and young adult ventricular assist device population.

The Advanced Cardiac Therapies Improving Outcomes Network (ACTION) is a collaborative learning network comprised of 55 centres focused on improving clinical outcomes and the patient/family experience for children with heart failure and those supported by ventricular assist devices. The development of ACTION’s PRO programme via engagement with patient and parent stakeholders is described. Pilot feasibility, patient/parent and clinician feedback, and initial PRO findings of patients and families receiving paediatric ventricular assist support across six centres are detailed.

Thirty of the thirty-five eligible patients (85.7%) were enrolled in the PRO programme during the pilot study period. Clinicians and participating patients/parents reported positive experiences with the PRO pilot programme. The most common symptoms reported by patients/parents in the first month post-implant period included limitations in activities, dressing change distress, and post-operative pain. Poor sleep, dressing change distress, sadness, and fatigue were the most common symptoms endorsed >30 days post-implant. Parental sadness and worry were notable throughout the entirety of the post-implant experience.

This multi-center ACTION learning network-based PRO programme demonstrated initial success in this six-center pilot study experience and yields important next steps for larger-scale PRO collection, research, and clinical intervention.

The COVID-19 pandemic created an unprecedented need for population-level clinical trials focused on the discovery of life-saving therapies and treatments. However, there is limited information on perception of research participation among perinatal populations, a population of particular interest during the pandemic.

Eligible respondents were 18 years or older, were currently pregnant or had an infant (≤12 months old), and lived in Florida within 50 miles of sites participating in the OneFlorida Clinical Research Consortium. Respondents were recruited via Qualtrics panels between April and September 2020. Respondents completed survey items about barriers and facilitators to participation and answered sociodemographic questions.

Of 533 respondents, most were between 25 and 34 years of age (n = 259, 49%) and identified as White (n = 303, 47%) and non-Hispanic (n = 344, 65%). Facebook was the most popular social media platform among our respondents. The most common barriers to research participation included poor explanation of study goals, discomforts to the infant, and time commitment. Recruitment through healthcare providers was perceived as the best way to learn about clinical research studies. When considering research participation, "myself" had the greatest influence, followed by familial ties. Noninvasive biological samples were highly acceptable. Hispanics had higher positive perspectives on willingness to participate in a randomized study (p = 0.009). Education (p = 0.007) had significant effects on willingness to release personal health information.

When recruiting women during the pregnancy and postpartum periods for perinatal studies, investigators should consider protocols that account for common barriers and preferred study information sources. Social media-based recruitment is worthy of adoption.

Complications from pulmonary hypertension are one of the leading contributors to morbidity and mortality post-cardiopulmonary bypass surgery in children with CHD. Pulmonary vasodilator therapies are commonly used post-operatively, but the optimal target patient population, therapy choice, timing of therapy initiation, and duration of therapy are not well defined.

We used PubMed and EMBASE to identify studies from 2000 to 2020 investigating the use of pulmonary vasodilator therapy post-cardiopulmonary bypass in children aged 0–18 years. To ensure eligibility criteria, studies were systematically reviewed by two independent reviewers.

We identified 26 studies of 42,971 children across four medication classes; 23 were single centre, 14 were prospective, and 11 involved randomisation (four of which employed a placebo-control arm). A disproportionate number of children were from a single retrospective study of 41,872 patients. Definitions varied, but change in pulmonary haemodynamics was the most common primary outcome, used in 14 studies. Six studies had clinical endpoints, with mortality the primary endpoint for two studies. Treatment with inhaled nitric oxide, iloprost, and sildenafil all resulted in improved haemodynamics in specific cohorts of children with post-operative pulmonary hypertension, although improved outcomes were not consistently demonstrated across all treated children. Iloprost may be a cheaper alternative to inhaled nitric oxide with similar haemodynamic response.

Studies were predominantly single-centre, a control arm was rarely used in randomised studies, and haemodynamic endpoints varied significantly. Further research is needed to reduce post-operative morbidity and mortality from pulmonary hypertension in children with CHD.

To determine the optimal antithrombotic agent choice, timing of initiation, dosing and duration of therapy for paediatric patients undergoing cardiac surgery with cardiopulmonary bypass.

We used PubMed and EMBASE to systematically review the existing literature of clinical trials involving antithrombotics following cardiac surgery from 2000 to 2020 in children 0–18 years. Studies were assessed by two reviewers to ensure they met eligibility criteria.

We identified 10 studies in 1929 children across three medications classes: vitamin K antagonists, cyclooxygenase inhibitors and indirect thrombin inhibitors. Four studies were retrospective, five were prospective observational cohorts (one of which used historical controls) and one was a prospective, randomised, placebo-controlled, double-blind trial. All included were single-centre studies. Eight studies used surrogate biomarkers and two used clinical endpoints as the primary endpoint. There was substantive variability in response to antithrombotics in the immediate post-operative period. Studies of warfarin and aspirin showed that laboratory monitoring levels were frequently out of therapeutic range (variably defined), and findings were mixed on the association of these derangements with bleeding or thrombotic events. Heparin was found to be safe at low doses, but breakthrough thromboembolic events were common.

There are few paediatric prospective randomised clinical trials evaluating antithrombotic therapeutics post-cardiac surgery; most studies have been observational and seldom employed clinical endpoints. Standardised, validated endpoints and pragmatic trial designs may allow investigators to determine the optimal drug, timing of initiation, dosing and duration to improve outcomes by limiting post-operative morbidity and mortality related to bleeding or thrombotic events.

Reflex-mediated syncope occurs in 15% of children and young adults. In rare instances, pacemakers are required to treat syncopal episodes associated with transient sinus pauses or atrioventricular block. This study describes a single centre experience in the use of permanent pacemakers to treat syncope in children and young adults.

Patients with significant pre-syncope or syncope and pacemaker implantation from 1978 to 2018 were reviewed. Data collected included the age of presentation, method of diagnosis, underlying rhythm disturbance, age at implant, type of pacemaker implanted, procedural complications and subsequent symptoms.

Fifty patients were identified. Median age at time of the first syncopal episode was 10.2 (range 0.3–20.4) years, with a median implant age of 14.9 (0.9–34.3) years. Significant sinus bradycardia/pauses were the predominant reason for pacemaker implant (54%), followed by high-grade atrioventricular block (30%). Four (8%) patients had both sinus pauses and atrioventricular block documented. The majority of patients had dual-chamber pacemakers implanted (58%), followed by ventricular pacemakers (38%). Median follow-up was 6.7 (0.4–33.0) years. Post-implant, 4 (8%) patients continued to have syncope, 7 (14%) had complete resolution of their symptoms, and the remaining reported a decrease in their pre-syncopal episodes and no further syncope. Twelve (24%) patients had complications, including two infections and eight lead malfunctions.

Paediatric patients with reflex-mediated syncope can be treated with pacing. Complication rates are high (24%); as such, permanent pacemakers should be reserved only for those in whom asystole from sinus pauses or atrioventricular block has been well documented.

Paediatric cardiac surgery on cardiopulmonary bypass induces substantial physiologic changes that contribute to post-operative morbidity and mortality. Fluid overload and oedema are prevalent complications, routinely treated with diuretics. The optimal diuretic choice, timing of initiation, dose, and interval remain largely unknown.

To guide clinical practice and future studies, we used PubMed and EMBASE to systematically review the existing literature of clinical trials involving diuretics following cardiac surgery from 2000 to 2020 in children aged 0–18 years. Studies were assessed by two reviewers to ensure that they met eligibility criteria.

We identified nine studies of 430 children across four medication classes. Five studies were retrospective, and four were prospective, two of which included randomisation. All were single centre. There were five primary endpoints – urine output, acute kidney injury, fluid balance, change in serum bicarbonate level, and required dose of diuretic. Included studies showed early post-operative diuretic resistance, suggesting higher initial doses. Two studies of ethacrynic acid showed increased urine output and lower diuretic requirement compared to furosemide. Children receiving peritoneal dialysis were less likely to develop fluid overload than those receiving furosemide. Chlorothiazide, acetazolamide, and tolvaptan demonstrated potential benefit as adjuncts to traditional diuretic regimens.

Early diuretic resistance is seen in children following cardiopulmonary bypass. Ethacrynic acid appears superior to furosemide. Adjunct diuretic therapies may provide additional benefit. Study populations were heterogeneous and endpoints varied. Standardised, validated endpoints and pragmatic trial designs may allow investigators to determine the optimal diuretic, timing of initiation, dose, and interval to improve post-operative outcomes.

Targeted drug development efforts in patients with CHD are needed to standardise care, improve outcomes, and limit adverse events in the post-operative period. To identify major gaps in knowledge that can be addressed by drug development efforts and provide a rationale for current clinical practice, this review evaluates the evidence behind the most common medication classes used in the post-operative care of children with CHD undergoing cardiac surgery with cardiopulmonary bypass.

We systematically searched PubMed and EMBASE from 2000 to 2019 using a controlled vocabulary and keywords related to diuretics, vasoactives, sedatives, analgesics, pulmonary vasodilators, coagulation system medications, antiarrhythmics, steroids, and other endocrine drugs. We included studies of drugs given post-operatively to children with CHD undergoing repair or palliation with cardiopulmonary bypass.

We identified a total of 127 studies with 51,573 total children across medication classes. Most studies were retrospective cohorts at single centres. There is significant age- and disease-related variability in drug disposition, efficacy, and safety.

In this study, we discovered major gaps in knowledge for each medication class and identified areas for future research. Advances in data collection through electronic health records, novel trial methods, and collaboration can aid drug development efforts in standardising care, improving outcomes, and limiting adverse events in the post-operative period.

Infants with moderate-to-severe CHD frequently undergo cardiopulmonary bypass surgery in childhood. Morbidity and mortality are highest in those who develop post-operative low cardiac output syndrome. Vasoactive and inotropic medications are mainstays of treatment for these children, despite limited evidence supporting their use.

To help inform clinical practice, as well as the conduct of future trials, we performed a systematic review of existing literature on inotropes and vasoactives in children after cardiac surgery using the PubMed and EMBASE databases. We included studies from 2000 to 2020, and the patient population was defined as birth – 18 years of age. Two reviewers independently reviewed studies to determine final eligibility.

The final analysis included 37 papers. Collectively, selected studies reported on 12 different vasoactive and inotropic medications in 2856 children. Overall evidence supporting the use of these drugs in children after cardiopulmonary bypass was limited. The majority of studies were small with 30/37 (81%) enrolling less than 100 patients, 29/37 (78%) were not randomised, and safety and efficacy endpoints differed widely, limiting the ability to combine data for meta-analyses.

Vasoactive and inotropic support remain critical parts of post-operative care for children after cardiopulmonary bypass surgery. There is a paucity of data for the selection and dosing of vasoactives and inotropes for these patients. Despite the knowledge gaps that remain, numerous recent innovations create opportunities to rethink the conduct of clinical trials in this high-risk population.

Preoperative mechanical ventilation is associated with morbidity and mortality following CHD surgery, but prior studies lack a comprehensive analysis of how preoperative respiratory support mode and timing affects outcomes.

We retrospectively collected data on children <18 years of age undergoing cardiac surgery at an academic tertiary care medical centre. Using multivariable regression, we examined the association between modes of preoperative respiratory support (nasal cannula, high-flow nasal cannula/noninvasive ventilation, or invasive mechanical ventilation), escalation of preoperative respiratory support, and invasive mechanical ventilation on the day of surgery for three outcomes: operative mortality, postoperative length of stay, and postoperative complications. We repeated our analysis in a subcohort of neonates.

A total of 701 children underwent 800 surgical procedures, and 40% received preoperative respiratory support. Among neonates, 243 patients underwent 253 surgical procedures, and 79% received preoperative respiratory support. In multivariable analysis, all modes of preoperative respiratory support, escalation in preoperative respiratory support, and invasive mechanical ventilation on the day of surgery were associated with increased odds of prolonged length of stay in children and neonates. Children (odds ratio = 3.69, 95% CI 1.2–11.4) and neonates (odds ratio = 8.97, 95% CI 1.31–61.14) on high-flow nasal cannula/noninvasive ventilation had increased odds of operative mortality compared to those on room air.

Preoperative respiratory support is associated with prolonged length of stay and mortality following CHD surgery. Knowing how preoperative respiratory support affects outcomes may help guide surgical timing, inform prognostic conversations, and improve risk stratification models.

Young children are particularly vulnerable to malnutrition as nutrition transition progresses. The present study aimed to document the prevalence, coexistence and correlates of nutritional status (stunting, overweight/obesity and anaemia) in Samoan children aged 24–59 months.

A cross-sectional community-based survey. Height and weight were used to determine prevalence of stunting (height-for-age Z-score <−2) and overweight/obesity (BMI-for-age Z-score >+2) based on WHO growth standards. Anaemia was determined using an AimStrip Hemoglobin test system (Hb <110 g/l).

Ten villages on the Samoan island of Upolu.

Mother–child pairs (n 305) recruited using convenience sampling.

Moderate or severe stunting was apparent in 20·3 % of children, 16·1 % were overweight/obese and 34·1 % were anaemic. Among the overweight/obese children, 28·6 % were also stunted and 42·9 % anaemic, indicating dual burden of malnutrition. Stunting was significantly less likely among girls (OR=0·41; 95 % CI 0·21, 0·79, P<0·01) than boys. Overweight/obesity was associated with higher family socio-economic status and decreased sugar intake (OR per 10 g/d=0·89, 95 % CI 0·80, 0·99, P=0·032). The odds of anaemia decreased with age and anaemia was more likely in children with an anaemic mother (OR=2·20; 95 % CI 1·22, 3·98, P=0·007). No child, maternal or household characteristic was associated with more than one of the nutritional status outcomes, highlighting the need for condition-specific interventions in this age group.

The observed prevalences of stunting, overweight/obesity and anaemia suggest that it is critical to invest in nutrition and develop health programmes targeting early childhood growth and development in Samoa.

To examine the use of vitamin D supplements during infancy among the participants in an international infant feeding trial.

Longitudinal study.

Information about vitamin D supplementation was collected through a validated FFQ at the age of 2 weeks and monthly between the ages of 1 month and 6 months.

Infants (n 2159) with a biological family member affected by type 1 diabetes and with increased human leucocyte antigen-conferred susceptibility to type 1 diabetes from twelve European countries, the USA, Canada and Australia.

Daily use of vitamin D supplements was common during the first 6 months of life in Northern and Central Europe (>80 % of the infants), with somewhat lower rates observed in Southern Europe (>60 %). In Canada, vitamin D supplementation was more common among exclusively breast-fed than other infants (e.g. 71 % v. 44 % at 6 months of age). Less than 2 % of infants in the USA and Australia received any vitamin D supplementation. Higher gestational age, older maternal age and longer maternal education were study-wide associated with greater use of vitamin D supplements.

Most of the infants received vitamin D supplements during the first 6 months of life in the European countries, whereas in Canada only half and in the USA and Australia very few were given supplementation.