Hypoplastic Left Heart Syndrome With Low Birth Weight or Prematurity: What Is the Optimal Approach?

BACKGROUND: Hypoplastic left heart syndrome with low birth weight or prematurity comprises a high-risk population with no optimal treatment pathway. Using the Pediatric Health Information System, we compared management approaches across the United States. METHODS: We analyzed neonates (≤30 days) with birth weight <2500 grams or gestational age <36 weeks between 2012 and 2021. Four strategies were identified: Norwood procedure, ductus arteriosus stent + pulmonary artery banding, pulmonary artery banding + prostaglandin infusion, or comfort care. Outcomes included hospital survival, discharge disposition, staged palliation completion, and 1-year transplant-free survival. RESULTS: Of 383 infants identified, 36.4% (n = 134) received comfort care, 43.9% (n = 165) Norwood, 12.4% (n = 49) ductal stent + pulmonary artery bands, and 8.8% (n = 34) pulmonary artery bands + prostaglandins. Neonates receiving comfort care had the lowest gestational age (35 weeks; interquartile range [IQR], 31.5-37 weeks) and birth weight (2.0 kg; IQR, 1.5-2.3 kg); 24.6% (33 of 134) had chromosomal anomalies. Infants undergoing primary Norwood had the highest birth weight (2.4 kg; IQR, 2.2-2.5 kg) and gestational age (37 weeks; IQR, 35-38 weeks). Glenn palliation was performed in 66.1% (109 of 165) compared with ductal stent + pulmonary artery band in 18.4% (9 of 49) and pulmonary artery band + prostaglandins in 35.3% (12 of 34). Only 11.3% (6 of 53) born <2 kg survived to 1 year, all after Norwood. Primary Norwood yielded higher hospital and 1-year transplant-free survival than hybrid strategies. CONCLUSIONS: Comfort care is routinely provided, particularly for infants with low birth weight, gestational age, or chromosomal anomalies. Primary Norwood offered the lowest hospital and 1-year mortality and highest palliation completion rates; birth weight was the most important factor determining 1-year survival.

Outcomes of Treatment for Infective Endocarditis Following Transcatheter Pulmonary Valve Replacement.

BACKGROUND: Recipients of transcatheter pulmonary valve replacement (TPVR) have shown increased risk of infective endocarditis (IE). Little is known about the outcomes of different management strategies, particularly surgery, for IE after TPVR. METHODS: We queried the Pediatric Health Information System database for cases of IE after TPVR performed from 2010-2020. We described patient demographics, hospital courses, admission complications, and treatment outcomes based on therapy offered, surgical or medical only. We compared outcomes of initial therapy. Data are expressed as median or percent. RESULTS: Sixty-nine cases of IE were identified, accounting for 98 related hospital admissions; 29% of patients recorded IE-related readmissions. Of those readmitted after initial medical therapy only, 33% had relapse IE. Rates of surgery were 22% during initial admission and 36% overall. Likelihood of surgical intervention increased with each subsequent admission. Renal and respiratory failure were more common in those given initial surgery. Mortality rate was 4.3% overall and 8% in the surgical cohort. CONCLUSION: Initial medical therapy may result in relapses/readmissions and possible delay of surgical therapy, which appears to be most effective for treatment of IE. For those treated only medically, a more aggressive course of therapy may be more likely to prevent relapse. Mortality following surgical therapy for IE after TPVR appears higher than reported for surgical pulmonary valve replacement generally.

Midterm Outcomes of Heart Transplantation in Children With Genetic Disorders.

BACKGROUND: Many congenital heart diseases (CHD) are associated with genetic defects. Children with complex CHD often have heart failure requiring heart transplant. Given the broad spectrum of genetic pathologies and dearth of transplants performed in these children, little is known regarding their outcomes. METHODS: We conducted a retrospective review of heart transplants performed at a high-volume center from 2007 to 2021. Patients were separated into pathogenic molecular and copy number variants, aneuploidies, and variants of uncertain significance, and compared with patients without known genetic diagnoses. Variables included genetic diagnoses, bridge-to-transplant approach, preoperative comorbidities, operative characteristics, and postoperative complications. Outcomes included intensive care unit-free days to 28 days, hospital mortality, survival, rejection, retransplantation, and educational status at latest follow-up. RESULTS: In all, 223 patients received transplants over the study period: 9.9% (22 of 223) had pathogenic molecular variants; 4.5% (10 of 223) had copy number variants; 1.8% (4 of 223) had aneuploidies; and 9% (20 of 223) had variants of uncertain significance. The most common anomalies were Turner syndrome (n = 3) and 22q11.2 deletion syndrome (n = 2). Children with aneuploidies had higher rates of hepatic dysfunction and hypothyroidism, whereas children with pathogenic copy number variants had higher rates of preoperative gastrostomy and stroke. Children with aneuploidies were intubated longer after transplant, with greater need for reintubation, and had the fewest intensive care unit-free days. Mortality and mean survival did not differ. At median follow-up of 4.4 years (range, 1.9 to 8.8), 89.7% of survivors (26 of 29) with pathogenic anomalies were attending or had graduated school. CONCLUSIONS: Despite more preoperative comorbidities, midterm outcomes after heart transplant in children with genetic syndromes and disorders are promising.

Evolution of Ventricular Assist Device Support Strategy in Children With Univentricular Physiology.

BACKGROUND: Since 2012, we have supported 18 children with single ventricle (SV) physiology on ventricular assist devices (VADs) as a bridge to decision, transplantation, or recovery. We provide a detailed report of our cumulative surgical experience and lessons learned from these patients. METHODS: We reviewed all SV-VADs between March 2012 and April 2020. Implanted SV-VADs intended for short-term support were excluded. Demographic and clinical data included palliation stage at the time of VAD implantation, cannulation configuration, device type, duration of support, circuit and device interventions, postoperative support, anticoagulation strategy, complications, mortality, and 1-year survival postdischarge. RESULTS: Five SV newborns without prior surgical palliation, 8 infants post-Norwood/hybrid procedure, 4 infants post-Glenn, and 1 infant post-Fontan were initially supported with either continuous-flow (n = 13 of 18, 72%) or pulsatile-flow (n = 5 of 18, 28%) devices. Three (17%) of 18 transitioned to another device during support. Before VAD conversion, 9 (50%) of 18 were supported by extracorporeal membrane oxygenation. Outcomes included 7 (39%) of 18 who transplanted, 2 (11%) of 18 who recovered, and 9 (50%) of 18 who died before discharge. Of these deaths, 2 occurred after transplantation and 2 after explantation, and 5 had redirection of care while on support secondary to previously undiagnosed pulmonary venoocclusive disease (n = 2) or severe neurologic events (n = 3). Overall, 6 (33%) of 18 experienced neurologic injury. At last follow-up, 9 (50%) of 18 children were alive (median 1.2 [interquartile range, 0.8-4.3] years postexplantation/transplantation). CONCLUSIONS: Our experience shows that SV children, including newborns, can be successfully bridged to desired endpoints with proper patient selection and using specific cannulation strategies. Continuing utilization of this strategy is warranted for future children requiring VAD support.

Improved Outcomes of Infant Lung Transplantation Over 3 Decades.

BACKGROUND: Lung transplantation is the definitive surgical treatment for end-stage lung disease. However, infants comprise less than 5% of pediatric cases. This study sought to provide an overview of infant lung transplantation outcomes over the past 3 decades by using linked United Network for Organ Sharing (UNOS) and Pediatric Health Information System (PHIS) data. METHODS: Infants undergoing lung transplantation from 1989 to 2020 in UNOS were reviewed. UNOS and PHIS records for patients who underwent lung transplantation from 1995 to 2020 were linked using date of birth, sex, and date of surgery ± 3 days. The study assessed underlying diagnoses, pretransplant and posttransplant extracorporeal membrane oxygenation support, retransplant-free survival to discharge, hospital experience (≥1 annual transplant for ≥4 years in a 5-year period), operative decade, bronchiolitis obliterans syndrome, long-term survival, and functional status at latest follow-up. RESULTS: A total of 112 lung transplants were performed in 109 infants over 31 years. Of these, 21 patients died before discharge, and 2 underwent repeat transplantation during the same admission. The study linked 80.6% (83 of 103) of UNOS and PHIS records. Hospital survival was lower for infants with idiopathic pulmonary hypertension and those who underwent transplant procedures at less experienced centers. All 7 infants requiring postoperative extracorporeal membrane oxygenation support died. Median freedom from bronchiolitis obliterans syndrome was 8.1 years (interquartile range, 4.6 to 11.6 years). After discharge, median survival was 10.3 years (interquartile range, 6.3 to 14.4 years), with improved 10-year survival for those patients who underwent transplantation from 2010 to 2020 (87.3%) vs 2000 to 2009 (52.4%; P = .098) and 1989 to 1999 (34.1%; P = .004). A total of 84.6% (33 of 39) of survivors had minor or no restrictions at latest follow-up. CONCLUSIONS: Carefully selected infants experience promising short- and long-term outcomes after lung transplantation.

Pulmonary-to-Systemic Arterial Shunt to Treat Children With Severe Pulmonary Hypertension.

BACKGROUND: The placement of a pulmonary-to-systemic arterial shunt in children with severe pulmonary hypertension (PH) has been demonstrated, in relatively small studies, to be an effective palliation for their disease. OBJECTIVES: The aim of this study was to expand upon these earlier findings using an international registry for children with PH who have undergone a shunt procedure. METHODS: Retrospective data were obtained from 110 children with PH who underwent a shunt procedure collected from 13 institutions in Europe and the United States. RESULTS: Seventeen children died in-hospital postprocedure (15%). Of the 93 children successfully discharged home, 18 subsequently died or underwent lung transplantation (20%); the mean follow-up was 3.1 years (range: 25 days to 17 years). The overall 1- and 5-year freedom from death or transplant rates were 77% and 58%, respectively, and 92% and 68% for those discharged home, respectively. Children discharged home had significantly improved World Health Organization functional class (P < 0.001), 6-minute walk distances (P = 0.047) and lower brain natriuretic peptide levels (P < 0.001). Postprocedure, 59% of children were weaned completely from their prostacyclin infusion (P < 0.001). Preprocedural risk factors for dying in-hospital postprocedure included intensive care unit admission (hazard ratio [HR]: 3.2; P = 0.02), mechanical ventilation (HR: 8.3; P < 0.001) and extracorporeal membrane oxygenation (HR: 10.7; P < 0.001). CONCLUSIONS: A pulmonary-to-systemic arterial shunt can provide a child with severe PH significant clinical improvement that is both durable and potentially free from continuous prostacyclin infusion. Five-year survival is comparable to children undergoing lung transplantation for PH. Children with severely decompensated disease requiring aggressive intensive care are not good candidates for the shunt procedure.

A comparison of cardiopulmonary bypass versus extracorporeal membrane oxygenation: Does intraoperative circulatory support strategy affect outcomes in pediatric lung transplantation?

OBJECTIVE: Data on adult lung transplantation suggest perioperative benefits of intraoperative extracorporeal membrane oxygenation (ECMO) compared to cardiopulmonary bypass (CPB). Information regarding their pediatric counterparts, however, is limited. This study compares outcomes of intraoperative ECMO versus CPB in pediatric lung transplantation. METHODS: We reviewed all pediatric lung transplants at our institution from 2014 to 2019 and compared those supported intraoperatively on ECMO (n = 13) versus CPB (n = 22), plus a conditional analysis excluding re-transplantations (ECMO [n = 13] versus CPB [n = 20]). We evaluated survival, surgical times, intraoperative transfusions, postoperative support, complications, and duration of hospitalization. RESULTS: Total time on ECMO support was significantly less than that of CPB support (P = .018). Intraoperatively, the ECMO group required fewer transfusions of fresh-frozen plasma (8.9 [5.8-22.3] vs 16.6 [11.4-39.0] mL/kg, P = .049) and platelets (4.2 [0.0-6.7] vs 8.0 [3.5-14.0] mL/kg, P = .049). When excluding re-transplantations, patients on ECMO required fewer packed red blood cells intraoperatively (12.6 [2.1-30.7] vs 28.2 [14.0-54.0] mL/kg, P = .048). There were no differences in postoperative support requirements, complications, or mortality at one, six, and twelve months. CONCLUSIONS: Intraoperative ECMO support during pediatric lung transplantation appears to decrease intraoperative transfusion requirements when compared to CPB. Data from additional institutions may strengthen these observations.

Comparison of Management Strategies for Neonates With Symptomatic Tetralogy of Fallot.

BACKGROUND: Neonates with tetralogy of Fallot and symptomatic cyanosis (sTOF) require early intervention. OBJECTIVES: This study sought to perform a balanced multicenter comparison of staged repair (SR) (initial palliation [IP] and subsequent complete repair [CR]) versus primary repair (PR) treatment strategies. METHODS: Consecutive neonates with sTOF who underwent IP or PR at ≤30 days of age from 2005 to 2017 were retrospectively reviewed from the Congenital Cardiac Research Collaborative. The primary outcome was death. Secondary outcomes included component (IP, CR, PR) and cumulative (SR): hospital and intensive care unit lengths of stay; durations of cardiopulmonary bypass, anesthesia, ventilation, and inotrope use; and complication and reintervention rates. Outcomes were compared using propensity score adjustment. RESULTS: The cohort consisted of 342 patients who underwent SR (IP: surgical, n = 256; transcatheter, n = 86) and 230 patients who underwent PR. Pre-procedural ventilation, prematurity, DiGeorge syndrome, and pulmonary atresia were more common in the SR group (p ≤0.01). The observed risk of death was not different between the groups (10.2% vs 7.4%; p = 0.25) at median 4.3 years. After adjustment, the hazard of death remained similar between groups (hazard ratio: 0.82; 95% confidence interval: 0.49 to 1.38; p = 0.456), but it favored SR during early follow-up (<4 months; p = 0.041). Secondary outcomes favored the SR group in component analysis, whereas they largely favored PR in cumulative analysis. Reintervention risk was higher in the SR group (p = 0.002). CONCLUSIONS: In this multicenter comparison of SR or PR for management of neonates with sTOF, adjusted for patient-related factors, early mortality and neonatal morbidity were lower in the SR group, but cumulative morbidity and reinterventions favored the PR group, findings suggesting potential benefits to each strategy.

Does Ascending Aorta Size Affect Norwood Outcomes in Hypoplastic Left Heart With Aortic Atresia?

BACKGROUND: Conflicting data exist regarding the impact of ascending aorta size on outcomes after the Norwood procedure. Results from multi-institutional studies have largely relied on heterogeneous populations undergoing this surgery for different anatomic defects. Using data from the Single Ventricle Reconstruction Trial, we analyzed the impact of preoperative ascending aortic diameter on Norwood outcomes for patients with aortic atresia variants of hypoplastic left heart syndrome. METHODS: Neonates with aortic atresia and no ventricular septal defect were included and classified into four groups, based on their baseline ascending aorta echocardiographic measurements: less than or equal to 1.5 mm, 1.6 to 1.9 mm, 2.0 to 3.9 mm, and greater than or equal to 4.0 mm. Outcomes included 14-day mortality, transplant-free survival at 1 and 14 months, need for extracorporeal membrane oxygenation, length of ventilation, intensive care, and hospital stay, intensive care unit (ICU)-free days, right ventricular function, and incidence of recoarctation by 14 months. RESULTS: Overall, 292 patients were analyzed. Median length of ICU stay was significantly longer for infants with small aortas, and ICU-free days were significantly lower. There was no difference in length of mechanical ventilation or hospitalization between groups. Long-term right ventricular function and tricuspid regurgitation did not differ. Aortic arch recoarctation incidence was higher in patients with small aortic diameters. Patients with aortas less than or equal to 1.5 mm had decreased 30-day transplant-free survival. CONCLUSIONS: Infants with aortic atresia variants of hypoplastic left heart syndrome and baseline ascending aortic diameter less than or equal to 1.5 mm appear to suffer the greatest morbidity and mortality early after Norwood procedure. These infants also experienced longer stays in the ICU and higher rates of recoarctation. Ascending aortic diameter does not appear to affect long-term ventricular function.

Symptom persistence after vascular ring repair in children.

PURPOSE: Vascular rings are often diagnosed after evaluation for swallowing and breathing difficulties. Data regarding symptoms following vascular ring repair is sparse. We sought to determine whether symptoms persist using chart review and a survey. METHODS: Sixty-three patients underwent open vascular ring repair from July 2007 to May 2018. Data regarding vascular anatomy, demographics, pre- and postoperative symptoms, and chromosomal abnormalities were collected. Freedom from reoperation, 30-day mortality, and complications were assessed. Patient families were contacted for a symptom focused survey. RESULTS: The median age of surgical intervention was 14.4 months (IQR 5.8-34.7 months) for single aortic arches with an aberrant subclavian artery (SAA), and 5.3 months (IQR 1.3-10.1 months) for double aortic arches (DAA) (Table). Prior to surgery, all but two SAA were symptomatic. There was no operative mortality. Three patients required re-exploration for chylothorax, and three required late aortopexy. At last follow-up, 45% (18/40) SAA and 65% (15/23) DAA had post-operative symptoms. Fourteen patient families completed the symptom survey (10 SAA, 4 DAA). Five SAA had breathing and swallowing symptoms, and 3 SAA and 3 DAA had breathing difficulties. CONCLUSIONS: Open vascular ring repair remains a safe repair. However, further investigation of the persistent symptoms in these patients is merited. STUDY TYPE / LEVEL OF EVIDENCE: Retrospective Comparative Study, Level III.