Echocardiography evaluation of neonatal vein of Galen aneurysmal malformation.

BACKGROUND: In neonatal vein of Galen aneurysmal malformation, vein of Galen aneurysmal malformation echocardiography remains the mainstay for early detection and explains various haemodynamic changes occurring due to a large systemic arterio-venous shunt. However, there is limited evidence of echocardiography in risk stratifying neonatal vein of Galen aneurysmal malformation vein of Galen aneurysmal malformation. The objective of this study was to identify echocardiographic parameters that could be associated with major outcomes and guide timing of neuro-intervention. METHODS: In this retrospective chart review, infants < 28 days of age with the diagnosis of vein of Galen aneurysmal malformation vein of Galen aneurysmal malformation were included. Demographic, clinical, and echocardiographic parameters were compared in neonates who survived or died with neonatal presentation. A risk algorithm model based on key echocardiographic parameters was developed to determine those who are at risk of early death. RESULTS: Of the 19 neonates included, with median birth weight 3.1 kg (IQR 2.58-3.36), nine (47%) neonates died at median age of 5 days (IQR 4-17). All neonates showed retrograde diastolic flow at the level of descending aorta by colour Doppler on the first post-natal echocardiogram at median age of 2 days (IQR 1-5.5). An aortic antegrade-to-retrograde velocity time integral ratio of < 1.5 and supra-systemic pulmonary artery pressure had 100% positive predictive value of death (p = 0.029), whereas aortic antegrade-to-retrograde velocity time integral ratio of > 1.5 and sub-systemic pulmonary artery pressure had 100% positive predictive value of survival (p = 0.029). CONCLUSION: Combination of aorta antegrade-to-retrograde velocity time integral ratio and degree of pulmonary hypertension on the first post-natal echocardiogram may help stratify the severity of disease and guide optimal timing for neuro-intervention for neonatal vein of Galen aneurysmal malformation.

Vein of Galen aneurysmal malformation: rationalizing medical management of neonatal heart failure.

Neonates who present in high output heart failure secondary to vein of Galen aneurysmal malformation can be difficult to manage medically due to the complex physiology that results from the large shunt through the malformation. Though the cardiac function is often normal, right ventricular dilation, severe pulmonary hypertension, and systemic steal can result in inadequate organ perfusion and shock. This report recommends medical management for stabilization of neonates prior to definitive management with endovascular embolization. IMPACT: Vein of Galen aneurysmal malformation (VGAM) is a rare intracranial arteriovenous malformation, which can present in the neonatal period with high output heart failure. Heart failure secondary to VGAM is often difficult to manage and is associated with high mortality and morbidity. Despite optimal medical management, many patients require urgent endovascular embolization for stabilization of their heart failure. This report offers discrete recommendations that can be used by clinicians as guidelines for the medical management of heart failure in newborns with VGAM.

Vein of Galen Aneurysmal Malformation with Anomalous Right Superior Vena Cava to the Left Atrium Leading to Atypical Clinical and Echocardiographic Findings.

Vein of Galen aneurysmal malformation (VGAM) is a rare intracranial vascular malformation which typically presents in a critically ill newborn with intractable heart failure, severe pulmonary hypertension, and right heart dilation. We report two unusual cases of neonates with VGAM and anomalous connection of right superior vena cava to the left atrium. Both neonates were diagnosed with VGAM in utero and were clinically stable after birth with dilation of the left atrium and left ventricle and no evidence of pulmonary hypertension. One case with hydrocephalus underwent transcatheter embolization at 1 week of age. The other case without hydrocephalus underwent elective transcatheter embolization at 4 months. We postulate that the presence of a right superior vena cava to the left atrium provides a physiological advantage and counters the left-to-right shunt from the arteriovenous malformation. This provides insight to a potential treatment strategy to improve outcomes in patients with severe heart failure and pulmonary hypertension secondary to VGAM.

Developmental changes of the fetal and neonatal thyroid gland and functional consequences on the cardiovascular system.

Thyroid hormones play an important role in the development and function of the cardiac myocyte. Dysregulation of the thyroid hormone milieu affects the fetal cardiac cells via complex molecular mechanisms, either by altering gene expression or directly by affecting post-translational processes. This review offers a comprehensive summary of the effects of thyroid hormones on the developing cardiovascular system and its adaptation. Furthermore, we will highlight the gaps in knowledge and provide suggestions for future research.

Management outcomes of primary pulmonary vein stenosis.

OBJECTIVES: Primary pulmonary vein stenosis (PPVS) is increasingly diagnosed in children with no prior pulmonary vein intervention history, and management is challenging. We describe characteristics of patients who underwent surgical repair of PPVS at our center, and examine factors associated with treatment failures. METHODS: A retrospective review of all patients who underwent surgical intervention for PPVS (2002-2016) was completed. Patients who had undergone prior cardiac surgery involving the pulmonary veins or atrial switch were excluded. Regression analyses were performed to examine characteristics, PPVS features, including severity score, and surgical details associated with treatment failures. RESULTS: Thirty-four children underwent initial surgical intervention for PPVS. Median age was 8.9 months (interquartile range, 5.9-18.4 months). Most patients (n = 31; 91%) had unilateral pulmonary vein involvement and the median PPVS severity score was 3.5 (interquartile range, 3-5). On competing risk analysis, 1 year following surgical repair, 9% of patients had died, 14% had undergone reintervention, and 77% were alive without reintervention; at 5 years the numbers were 9%, 30%, and 61%, respectively. Factors associated with mortality included bilateral disease and PPVS severity score >6. Bilateral disease and PPVS severity score >5 were associated with reintervention risk. CONCLUSIONS: Multidisciplinary management strategy is required for PPVS. Despite satisfactory early repair, patients continue to be at risk for recurrence and subsequent mortality, especially those with extensive primary involvement. The disappointing results underscore the need for multi-institutional collaborations to better understand this complex disease, establish management and follow-up protocols, and explore investigational treatment modalities that could modify the unfavorable outcome of this uncommon and challenging disease.

Long-Term Survival of Patients With Coarctation Repaired During Infancy (from the Pediatric Cardiac Care Consortium).

Patients who undergo coarctation repair during infancy have excellent early survival but long-term survival is unknown. We aimed to describe the long-term survival of patients with coarctation repaired during infancy and determine predictors of mortality. We performed a retrospective cohort study using data from the Pediatric Cardiac Care Consortium for patients with coarctation who underwent surgical repair before 12 months of age between 1982 and 2003. Long-term transplant-free survival was obtained by linkage with the National Death Index and the Organ Sharing Procurement Network. Kaplan Meier survival plots were constructed, and univariate and multivariable analyses were performed to determine predictors of mortality. We identified 2,424 coarctation patients who met inclusion criteria. At 20 years postoperatively, 94.5% of all patients and 95.8% of those discharged after initial operation remained alive, respectively. Significant multivariable predictors of mortality included surgical weight <2.5 kg (hazard ratio [HR] 3.70, 95% confidence interval [CI] 2.19 to 6.24), presence of a genetic syndrome (HR 2.40, 95% CI 1.13 to 5.10), and repair before 1990 (HR 1.91, 95% CI 1.09 to 3.34). None of the other factors examined including age at repair, gender, coarctation type, or surgical approach were found to be statistically significant. Over half of the deaths were due to the underlying congenital heart disease or other cardiovascular etiology. Overall long-term survival of patients who undergo coarctation repair during infancy is excellent. However, patients do experience small continued survival attrition throughout early adulthood. Ongoing monitoring of this cohort is necessary to assess late mortality risk.

Rapid Cycle Deliberate Practice Versus Reflective Debriefing for Pediatric Septic Shock Training.

OBJECTIVES: Rapid cycle deliberate practice is a simulation training method that cycles between deliberate practice and directed feedback to create perfect practice; in contrast to reflective debriefing where learners are asked to reflect on their performance to create change. The aim of this study is to compare the impact of rapid cycle deliberate practice versus reflective debriefing training on resident application and retention of the pediatric sepsis algorithm. DESIGN: Prospective, randomized-control study. SETTING: A tertiary care university children's hospital simulation room, featuring a high-fidelity pediatric patient simulator. SUBJECTS: Forty-six upper-level pediatric residents. INTERVENTIONS: Simulation training using rapid cycle deliberate practice or reflective debriefing. MEASUREMENTS AND MAIN RESULTS: Knowledge was assessed with a quiz on core sepsis management topics. The application of knowledge was assessed with a sepsis management checklist during the simulated scenario. The residents were assessed before and after the intervention and again at a follow-up session, 3-4 months later, to evaluate retention. Both groups had similar pre-intervention scores. Post-intervention, the rapid cycle deliberate practice group had higher checklist scores (rapid cycle deliberate practice 18 points [interquartile range, 18-19] vs reflective debriefing 17 points [interquartile range, 15-18]; p < 0.001). Both groups had improved quiz scores. At follow-up, both groups continued to have higher scores compared with the pre-intervention evaluation, with the rapid cycle deliberate practice group having a greater change in checklist score from pre-intervention to follow-up (rapid cycle deliberate practice 5 points [interquartile range, 3.5-7] vs reflective debriefing 3 points [interquartile range, 1.5-4.5]; p = 0.019). Both groups reported improved confidence in diagnosing and managing septic shock. CONCLUSIONS: Both rapid cycle deliberate practice and reflective debriefing are effective in training pediatric residents to apply the sepsis algorithm and in improving their confidence in the management of septic shock. The rapid cycle deliberate practice method was superior immediately post-training; however, it is unclear if this advantage is maintained over time. Both methods should be considered for training residents.

Reintervention Is Associated With Improved Survival in Pediatric Patients With Pulmonary Vein Stenosis.

OBJECTIVES: The aim of this study was to evaluate survival following catheter intervention in pediatric patients with pulmonary vein stenosis (PVS). BACKGROUND: Despite aggressive surgical and catheter intervention on PVS in children, recurrence and progression of stenosis can lead to right heart failure and death. Clinicians continue to seek effective treatment options for PVS. METHODS: A single-center, retrospective study was performed including all patients <18 years of age who underwent catheter intervention (balloon angioplasty and bare-metal stent and drug-eluting stent insertion) on PVS. Endpoints included death, vein loss, and rate of reintervention. RESULTS: Thirty patients underwent intervention (balloon angioplasty, n = 9; bare-metal stent, n = 5; drug-eluting stent, n = 16) at a median age of 6.4 months (4.3 to 9.9 months). Median follow-up duration was 30.6 months (77 days to 10.5 years). Fourteen patients (47%) died at a median of 2.0 months (0.4 to 3.2 months) following intervention. There was no association between DES placement and survival (p = 0.067). Reintervention (catheter or surgical) was associated with improved survival (p = 0.001), with a 1-year survival rate of 84% compared with 25% for no reintervention. Vein loss occurred in 34 of 58 (59%) veins at a median of 3.3 months (1.0 to 5.0 months). One-year vein survival was higher with DES implantation (p = 0.031) and with reintervention (p < 0.001). CONCLUSIONS: DES implantation at first catheter intervention appears to be associated with improved vein survival but may not result in improved patient survival. However, reintervention appears to be associated with improved patient survival and vein patency, suggesting that despite mode of treatment, frequent surveillance is important in the care of these patients.

Organ damage in high-risk patients with systemic and incomplete lupus syndromes.

The objective of this study was to characterize organ damage in lupus patients enrolled in Dallas Regional Autoimmune Disease Registry (DRADR). Retrospective chart review was carried out on 99 patients with four or more diagnostic criteria for systemic lupus erythematosus (SLE) and 15 with less than four of these criteria, who were designated as having incomplete lupus erythematosus (ILE). The majority of patients (84 %) were African American or Hispanic/Latino; mean disease duration was 9.5 years. The mean damage score was 1.57 (range 0-8), and a damage score greater than 0 was present in 64 % of the patients. The ILE group had lower mean damage scores (0.67) than the SLE group (1.67; P = 0.04), explained in part by the shorter disease duration in the ILE patients (4.33 vs. 10.24 years; P = 0.003). The most prevalent damage category was renal, present in 24 % of patients. Malignancies occurred in individuals who were significantly older than those who had renal or peripheral vascular damage (P = 0.0007). The findings confirm clinical impressions that DRADR includes a high-risk lupus population. The ILE patients have less damage but also shorter disease duration, suggesting that this might represent an earlier disease stage. These results are consistent with the hypothesis that ILE patients include a subset that is likely to experience progressive organ damage. Longitudinal study of these patients has significant likelihood of tracking the changes that are correlated with disease progression to SLE.