A prospective multicenter feasibility study of a miniaturized implantable continuous flow ventricular assist device in smaller children with heart failure.

BACKGROUND: There is no FDA-approved left ventricular assist device (LVAD) for smaller children permitting routine hospital discharge. Smaller children supported with LVADs typically remain hospitalized for months awaiting heart transplant-a major burden for families and a challenge for hospitals. We describe the initial outcomes of the Jarvik 2015, a miniaturized implantable continuous flow LVAD, in the NHLBI-funded Pumps for Kids, Infants, and Neonates (PumpKIN) study, for bridge-to-heart transplant. METHODS: Children weighing 8 to 30 kg with severe systolic heart failure and failing optimal medical therapy were recruited at 7 centers in the United States. Patients with severe right heart failure and single-ventricle congenital heart disease were excluded. The primary feasibility endpoint was survival to 30 days without severe stroke or non-operational device failure. RESULTS: Of 7 children implanted, the median age was 2.2 (range 0.7, 7.1) years, median weight 10 (8.2 to 20.7) kilograms; 86% had dilated cardiomyopathy; 29% were INTERMACS profile 1. The median duration of Jarvik 2015 support was 149 (range 5 to 188) days where all 7 children survived including 5 to heart transplant, 1 to recovery, and 1 to conversion to a paracorporeal device. One patient experienced an ischemic stroke on day 53 of device support in the setting of myocardial recovery. One patient required ECMO support for intractable ventricular arrhythmias and was eventually transplanted from paracorporeal biventricular VAD support. The median pump speed was 1600 RPM with power ranging from 1-4 Watts. The median plasma free hemoglobin was 19, 30, 19 and 30 mg/dL at 7, 30, 90 and 180 days or time of explant, respectively. All patients reached the primary feasibility endpoint. Patient-reported outcomes with the device were favorable with respect to participation in a full range of activities. Due to financial issues with the manufacturer, the study was suspended after consent of the eighth patient. CONCLUSION: The Jarvik 2015 LVAD appears to hold important promise as an implantable continuous flow device for smaller children that may support hospital discharge. The FDA has approved the device to proceed to a 22-subject pivotal trial. Whether this device will survive to commercialization remains unclear because of the financial challenges faced by industry seeking to develop pediatric medical devices. (Supported by NIH/NHLBI HHS Contract N268201200001I, clinicaltrials.gov 02954497).

Predicting Stroke for Pediatric Patients Supported With Ventricular Assist Devices: A Pedimacs Report.

BACKGROUND: The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) provides detailed understanding on pediatric patients supported with ventricular assist devices (VADs). We sought to identify important variables affecting the incidence of stroke in pediatric VADs. METHODS: Between 2012 and 2022, 1463 devices in 1219 patients were reported to Pedimacs from 40 centers in patients aged <19 years at their first VAD implantation. Multiphase parametric hazard modeling was used to identify risk factors for stroke among all device types. RESULTS: Of the 1219 patients, the most common devices were implantable continuous (472 [39%]), followed by paracorporeal pulsatile (342 [28%]), and paracorporeal continuous (327 [27%]). Overall freedom from stroke at 6 months was higher in the recent era (2012-2016; 80.2% [95% CI, 77.1%-82.9%] vs 2017-2023; 87.9% [95% CI, 86.2%-89.4%], P = .009). Implantable continuous VADs had the highest freedom from stroke at 3 months (92.7%; 95% CI, 91.1%-93.9%) and 6 months (91.1%; 95% CI, 89.3%-92.6%), followed by paracorporeal pulsatile (87.0% [95% CI, 84.8%-88.9%] and 82.8% [95% CI, 79.8%-85.5%], respectively), and paracorporeal continuous (76.0% [95% CI, 71.8%-79.5%] and 69.5% [95% CI, 63.4%-74.8%], respectively) VADs. Parametric modeling identified risk factors for stoke early after implant and later. Overall, and particularly for paracorporeal pulsatile devices, early stroke risk has decreased in the most recent era (hazard ratio, 5.01). Among implantable continuous devices, cardiogenic shock was the major risk factor. For patients <10 kg, early hazard was only seen in the previous era. For congenital patients, early hazard was seen in nonimplantable device use and use of extracorporeal membrane oxygenation. CONCLUSIONS: The overall stroke rate has decreased from 20% to 15% at 6 months, with particular improvement among paracorporeal pulsatile devices. Risk factor analyses offer insights for identification of higher stroke risk subsets and further management refinements.

Evaluation and Management of Chronic Heart Failure in Children and Adolescents With Congenital Heart Disease: A Scientific Statement From the American Heart Association.

With continued medical and surgical advancements, most children and adolescents with congenital heart disease are expected to survive to adulthood. Chronic heart failure is increasingly being recognized as a major contributor to ongoing morbidity and mortality in this population as it ages, and treatment strategies to prevent and treat heart failure in the pediatric population are needed. In addition to primary myocardial dysfunction, anatomical and pathophysiological abnormalities specific to various congenital heart disease lesions contribute to the development of heart failure and affect potential strategies commonly used to treat adult patients with heart failure. This scientific statement highlights the significant knowledge gaps in understanding the epidemiology, pathophysiology, staging, and outcomes of chronic heart failure in children and adolescents with congenital heart disease not amenable to catheter-based or surgical interventions. Efforts to harmonize the definitions, staging, follow-up, and approach to heart failure in children with congenital heart disease are critical to enable the conduct of rigorous scientific studies to advance our understanding of the actual burden of heart failure in this population and to allow the development of evidence-based heart failure therapies that can improve outcomes for this high-risk cohort.

Development and validation of the Informal Supporter Readiness Inventory (ISRI).

OBJECTIVE: This article outlines the development and validation of the Informal Supporter Readiness Inventory (ISRI), based on the model developed by the present authors in (Davies, 2023). This scale assesses the readiness of informal supporters to intervene or provide support in situations of intimate partner violence (IPV). METHODS: The research followed a three-phased procedure of item development, scale development, and scale evaluation; adhering to best practice guidelines for psychometric development and validation. This process provided empirical substantiation for the domains of the Model of Informal Supporter Readiness (Davies, 2023). RESULTS: The 57-item ISRI incorporates four primary factors: normative, individual, goodman-emotional, and situational-assessment. These factors demonstrated robust internal consistency and factor structures. Additionally, the ISRI evidenced strong test-retest reliability, and both convergent and divergent validity. Although aligning closely with the Model of Informal Supporter Readiness, the scale revealed a nuanced bifurcation of situational factors into situational-emotional and situational-assessment. DISCUSSION: The ISRI offers an important advancement in IPV research by highlighting the multifaceted nature of informal supporter intervention. The findings have several implications, from tailoring individualised supportive interventions to strengthening support networks and empowering survivors. The present study's findings underscore the potential of adopting a social network-oriented approach to interventions in IPV scenarios. Applications for research and practice are discussed.

The incidence, clinical features, and management of essential infantile esotropia in the United Kingdom. A British Ophthalmology Surveillance Unit (BOSU) study.

BACKGROUND/OBJECTIVES: A national study was undertaken through the British ophthalmology surveillance unit (BOSU) to determine the incidence, presenting features and management of essential infantile esotropia (EIE) in the UK. METHODS: Data from a prospective national observational study of newly diagnosed EIE presenting to clinicians in the United Kingdom over a 12-month period were collected. Cases with a confirmed diagnosis by a clinician of a constant, non-accommodative esotropia ≥20 prism dioptres (PD), presenting at ≤12 months, with no neurological or ocular abnormalities were identified through BOSU. Follow-up data were collected at 12 months. RESULTS: A total of 57 cases were reported giving an incidence of EIE of 1 in 12,828 live births. The mean age of diagnosis and intervention were 7.05 ± 2.6 months (range 2-12) and 14.7 ± 4.9 months (range 6.5-28.1), respectively. Management was surgical in 59.6%, botulinum toxin alone in 22.8%, and 17.5% were observed. The preoperative angle of esotropia was smaller in the observation group (P = 0.04). The postoperative angle of esotropia was not statistically significant between botulinum toxin or surgery (P = 0.3), although the age of intervention was earlier in the botulinum group (P = 0.007). Early intervention (before 12 months of age) did not influence the post-intervention motor outcomes between 0 and 10 prism dioptres of esotropia (P = 0.78). CONCLUSIONS: The incidence of EIE in the UK is considerably lower than reported in other population-based studies. The preferred method of treatment was surgical with earlier intervention in those treated with botulinum toxin. An early age of intervention (<12 months) did not influence motor outcomes.

Seventh Annual Society of Thoracic Surgeons Pedimacs Report.

BACKGROUND: The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs), supported by The Society of Thoracic Surgeons, provides detailed information on pediatric patients supported with ventricular assist devices (VADs). METHODS: From September 19, 2012, to December 31, 2022, 1463 devices in 1219 patients aged <19 years were reported to the registry from 40 North American hospitals. RESULTS: Cardiomyopathy remains the most common underlying etiology (59%), followed by congenital heart disease (26%) and myocarditis (8%). Implantable continuous devices were most common (39%) type, followed by paracorporeal pulsatile (28%) and paracorporeal continuous (27%) devices. At 6 months after VAD implantation, a favorable outcome (transplant, recovery, or alive on device) was achieved in 85% of patients, which was greatest among those on implantable continuous VADs (92%) and least for paracorporeal continuous VADs (68%), although the patient population supported on these devices is different. CONCLUSIONS: This Seventh Pedimacs Report demonstrates the continued importance of VADs in the treatment of children. With the complexity of cardiac physiologies and sizes of patients, multiple types of devices are used, including paracorporeal continuous, paracorporeal pulsatile, and implantable continuous devices. The preoperative risk factors and differences in patient populations may account for some of the differences in survival observed among these devices. This report, along with other collaborative work, continues to advance the care of this challenging and vulnerable population.

Survival in Pediatric Patients With Ventricular Assist Devices: A Special Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) Report.

BACKGROUND: The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) provides detailed understanding on pediatric patients supported with ventricular assist devices (VADs). We sought to identify important variables affecting mortality in pediatric VADs. METHODS: Patients aged <19 years, from 2012 to 2021, were included. Survival analyses were performed using Kaplan-Meier. Parametric hazard modeling was used to identify risk factors for death. RESULTS: Of the 1109 patients, the most common devices were implantable continuous (IC, 448 [40%]), followed by paracorporeal pulsatile (PP, 306 [28%]), paracorporeal continuous (PC, 293 [26%]), and percutaneous (58 [5%]). Patients with percutaneous device, infants, congenital heart disease, biventricular support, and Interagency Registry for Mechanically Assisted Circulatory Support profile 1 had worse overall survival at 6 months. Positive outcome was 83% at 6 months. Consistent with their cohort composition, device type positive outcomes at 6 months were IC, 92%; PP, 84%; and PC, 69%. Parametric hazard modeling for overall survival showed an early hazard for death with biventricular support, congenital heart disease (CHD), intubation before implantation, PC device, and renal impairment, whereas a constant hazard was associated with ascites. For patients <10 kg, parametric modeling showed an early hazard for CHD, intubation, and renal impairment. Modeling in CHD patients showed an early hazard for biventricular support, renal impairment, and use of PC/PP devices. CONCLUSIONS: This multivariable analysis of the complete Pedimacs database demonstrates that illness at VAD implantation, diagnosis, and strategy of support affect survival and differ by device type. We hope this is the first step in creating a predictive tool to help providers and families have informed expectations.

Taking ACTION: A Prognostic Tool for Pediatric Ventricular Assist Device Mortality.

We sought to develop a contemporary risk assessment tool for use in pediatric ventricular assist device (VAD) candidates to estimate risk for mortality on the device using readily available preimplantation clinical data. Training and testing datasets were created from Advanced Cardiac Therapies Improving Outcomes Network (ACTION) registry data on patients supported with a VAD from 2012 to 2021. Potential risk factors for mortality were assessed and incorporated into a simplified risk prediction model utilizing an open-source, gradient-boosted decision tree machine learning library, known as random forest. Predictive performance was assessed by the area under the receiver operating characteristic curve in the testing dataset. Nine significant risk factors were included in the final predictive model which demonstrated excellent discrimination with an area under the curve of 0.95. In addition to providing a framework for establishing pediatric-specific risk profiles, our model can help inform team expectations, guide optimal patient selection, and ultimately improve patient outcomes.

Managing Myocardial Bridge and Right Ventricular Outflow Tract Obstruction in an Adolescent With Hypertrophic Cardiomyopathy.

We report a case of symptomatic myocardial bridge in an adolescent with dynamic right ventricular outflow tract obstruction and history of congenital pulmonary valve stenosis as well as hypertrophic cardiomyopathy. Definitive treatment was surgical infundibular myectomy and coronary unroofing, resulting in improvement in right ventricular outflow tract gradient and ischemic symptoms.

A systematic review of informal supporters of intimate partner violence survivors: the intimate partner violence model of informal supporter readiness.

Background: Intimate partner violence (IPV) is a serious public health issue that consists of physical, sexual, and psychological violence perpetrated by a current or former partner. Informal supporters (e.g., family and friends) of survivors are more often witness to IPV or are the first people a survivor will disclose abuse to and are more able to provide consistent ongoing support than professional services. Therefore, greater understanding of informal supporters is warranted to aid in reducing the risks experienced by survivors. This systematic review aimed to: (1) identify factors associated with either an increase or decrease in helping behaviour toward a survivor, (2), identify the most effective self-care strategies employed by informal supporters, and (3) consider the current theoretical approaches used to understand informal supporters help-giving behavioural intention. Methods: A systematic literature search was conducted following the PRISMA guidelines. The search included English language articles published between 2005 and 2021 in the databases Psych Articles, Scopus, Proquest Social Services Abstracts, and Ebscohost. Studies were included if the primary research aims explored the motivators and inhibitors of helping intention or self-care strategies of adult social network members of adult IPV survivors. Two reviewers independently screened all identified articles for inclusion suitability. Results: One hundred and twenty articles were subjected to full text screening resulting in 31 articles being identified as meeting inclusion criteria. Synthesis of the findings identified the following three key areas associated with help-giving behavioural intentions: normative factors, individual factors, and situational factors. There were no articles identified that considered self-care of informal supporters. Of the 31 articles, 22 had a theoretical underpinning. None of the utilised theories explained all three of the identified factors of help-giving behavioural intention. Conclusion: These results are integrated into a proposed Intimate Partner Violence Model of Informal Supporter Readiness (IPV-MISR), incorporating the identified factors associated with help-giving behavioural intention. This model provides a framework for conceptualising the readiness of an informal supporter to provide adequate support to IPV survivors. The model extends existing theoretical standpoints and has utility in both practice and research.

Sixth Annual Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) Report: The Society Of Thoracic Surgeons Pedimacs Annual Report.

BACKGROUND: The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs), supported by The Society of Thoracic Surgeons, provides detailed information on pediatric patients supported with ventricular assist devices (VADs). METHODS: From September 19, 2012, to December 31, 2021, there were 1355 devices in 1109 patients (<19 years) from 42 North American Hospitals. RESULTS: Cardiomyopathy was the most common underlying cause (59%), followed by congenital heart disease (25%) and myocarditis (9%). Regarding device type, implantable continuous (IC) VADs were most common at 40%, followed by paracorporeal pulsatile (PP; 28%) and paracorporeal continuous (PC; 26%). Baseline demographics differed, with the PC cohort being younger, smaller, more complex (ie, congenital heart disease), and sicker at implantation (P < .0001). At 6 months after VAD implantation, a favorable outcome (transplantation, recovery, or alive on device) was achieved in 84% of patients, which was greatest among those on IC VADs (92%) and least for PC VADs (69%). Adverse events were not uncommon, with nongastrointestinal bleeding (incidence of 14%) and neurologic dysfunction (11% [stroke, 4%]), within 2 weeks after implantation being the most prevalent. Stroke and bleeding had negative impacts on overall survival (P = .002 and P < .001, respectively). CONCLUSIONS: This Sixth Pedimacs Report demonstrates the continued evolution of the pediatric field. The complexity of cardiac physiologies and anatomic constraint mandates the need for multiple types of devices used (PC, PP, IC). Detailed analyses of each device type in this report provide valuable information to further advance the care of this challenging and vulnerable population.

Patient and Device Selection in Pediatric MCS: A Review of Current Consensus and Unsettled Questions.

The field of pediatric ventricular assist device (VAD) support has expanded significantly over the past 20 years, with one third of pediatric heart transplant recipients currently being bridged to transplant with a VAD. Despite increased pediatric VAD utilization, however, there remains little formalized guidance for patient or device selection. The population of children with advanced heart failure is quite heterogeneous, and the available data suggest that VAD outcomes vary significantly based upon patient size, anatomy, level of illness, and type of device implanted. In an effort to better understand current practice patterns and identify populations for whom there does not appear to be a consensus approach to achieving optimal VAD outcomes, the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) has surveyed clinical decision-making among member sites and conducted a review of the available literature regarding patient population-specific VAD outcomes and center-specific practices related to patient and device selection. Rather than aiming to provide clinical guidelines, this document offers an overview of contemporary approaches to patient and device selection, highlighting specific populations for whom there is not a consensus approach to achieving reliably good VAD outcomes, as these populations may benefit most from future research and quality improvement efforts directed toward identifying best practice.

Consensus Statement: Hemostasis Trial Outcomes in Cardiac Surgery and Mechanical Support.

BACKGROUND: Research evaluating hemostatic agents for the treatment of clinically significant bleeding has been hampered by inconsistency and lack of standardized primary clinical trial outcomes. Clinical trials of hemostatic agents in both cardiac surgery and mechanical circulatory support, such as extracorporeal membrane oxygenation and ventricular assist devices, are examples of studies that lack implementation of universally accepted outcomes. METHODS: A subgroup of experts convened by the National Heart, Lung, and Blood Institute and the US Department of Defense developed consensus recommendations for primary outcomes in cardiac surgery and mechanical circulatory support. RESULTS: For cardiac surgery the primary efficacy endpoint of total allogeneic blood products (units vs mL/kg for pediatric patients) administered intraoperatively and postoperatively through day 5 or hospital discharge is recommended. For mechanical circulatory support outside the perioperative period the recommended primary outcome for extracorporeal membrane oxygenation is a 5-point ordinal score of thrombosis and bleeding severity adapted from the Common Terminology Criteria for Adverse Events version 5.0. The recommended primary endpoint for ventricular assist device is freedom from disabling stroke (Common Terminology Criteria for Adverse Events AE ≥ grade 3) through day 180. CONCLUSIONS: The proposed composite risk scores could impact the design of upcoming clinical trials and enable comparability of future investigations. Harmonizing and disseminating global consensus definitions and management guidelines can also reduce patient heterogeneity that would confound standardized primary outcomes in future research.

Fifth Annual Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) Report.

BACKGROUND: The Pediatric Interagency Registry for Mechanical Circulatory Support (Pedimacs) provides detailed information on pediatric patients supported with ventricular assist devices (VADs). METHODS: From September 19, 2012, to December 31, 2020, 1229 devices in 1011 patients were reported to the registry from 47 North American Hospitals in patients aged younger than 19 years. RESULTS: Cardiomyopathy was the most common underlying etiology (58%), followed by congenital heart disease (CHD; 25%) and myocarditis (10%). The most common devices implanted were implantable continuous (IC; 419 [41%]), followed by paracorporeal pulsatile (PP; 269 [27%]), paracorporeal continuous (PC; 263 [26%]), and percutaneous (53 [5%]). Overall, at 6 months after VAD implantation, 83% had a positive outcome (transplant, explant, or alive on device). The freedom from stroke at 3 months was highest in IC VADs (93%), compared with PP VADs (84%) and with PC VADs (75%). There were differences in survival by device type, with patients on IC VADs having the best overall survival and those on PC having the lowest overall survival, though the patient populations being supported by each VAD type differed significantly from each other. CONCLUSIONS: This Fifth Pedimacs Report demonstrates the continued robust growth of VADs in the pediatric community, now with more than 1000 patients reported to the registry. The multiple available device types (PC, PP, IC) serve different populations with different pre-VAD risk profiles, which may account for differences in survival and adverse events between device types.