Extracardiac Fontan Fenestration Device Closure with Amplatzer Vascular Plug II and Septal Occluder: Procedure Results and Medium-Term Follow-Up.

The objective of this study was to determine medium-term morbidity and mortality of patients who have undergone device closure of an extracardiac Fontan fenestration with an Amplatzer Vascular Plug II (AVPII) or Septal Occluder (ASO). A secondary objective was to compare medium-term morbidity and mortality between these patients and other fenestrated Fontan patients. A retrospective chart review was performed on patients who underwent an extracardiac fenestrated Fontan procedure between 1992 and 2015 at Cardinal Glennon Children's Medical Center. Procedural and follow-up data were obtained and compared between those who underwent fenestration closure and those who did not. Additional outcome measures included whether the fenestration had spontaneously closed, morbidity and mortality, oxygen saturations, and hemodynamics pre- and post-closure. Fifty-nine of 118 patients (50%) with a fenestrated Fontan underwent 60 device closures of the fenestration. Thirty-two (53%) of these were with the AVPII and 28 (47%) with the ASO. There was one device embolization. At a median follow-up of 3.9 years, five patients suffered morbidity, including 2 with arrhythmias, 1 with plastic bronchitis, 1 with protein losing enteropathy, and 1 with stroke. There were no cardiopulmonary deaths in this group. Twenty-three of 118 patients (19%) had spontaneous closure. There was no difference in morbidity and mortality between patients who underwent percutaneous fenestration closure and those who either had spontaneous closure or a persistently patent fenestration. Device closure of Fontan fenestrations is a safe and effective procedure with minimal morbidity and mortality comparable to other patients with fenestrations.

Rare occurrence of Tetralogy of Fallot in dizygotic twins conceived via in vitro fertilisation.

An increased incidence of CHD has been noted in twin gestations and in infants conceived using assisted reproductive technologies. However, CHD in these populations remains understudied and the mechanisms underlying these phenomena remain unclear. We present the case of twins conceived via in vitro fertilisation both with Tetralogy of Fallot and additional cardiac and extracardiac malformations.

Heart transplantation in children with intellectual disability: An analysis of the UNOS database.

Heart transplantation in children with intellectual disability (ID) is an issue of debate due to the shortage of available donor organs. We sought to perform the first large-scale retrospective cohort study describing the prevalence and outcomes of heart transplantation in this population. The United Network of Organ Sharing database was queried from 2008 to 2015 for pediatric patients (age <19 years) receiving first, isolated heart transplant. Recipients were divided into three subgroups: definite ID, probable ID, and no ID. The chi-square test was used to compare patients' baseline characteristics. Kaplan-Meier and Cox proportional hazard regression analyses were used to estimate the association between ID and death-censored graft failure and patient survival. Over the study period, 565 pediatric patients with definite (131) or probable (434) ID received first heart transplant, accounting for 22.4% of all first pediatric heart transplants (n=2524). Recipients with definite ID did not significantly differ from those without ID in terms of gender, ethnicity, ischemia time, severity of pretransplant condition (waitlist status, mechanical ventilation, inotrope dependence, ECMO, VAD, PVRI, infection prior to transplant), or incidents of acute rejection within the first year. ID was associated with prolonged waitlist time (P<.001). Graft and patient survival at 3 years was equivalent between children with and without ID (P=.811 and .578, respectively). We conclude that intellectual disability is prevalent in children receiving heart transplants, with 22.4% of recipients over the study period having definite or probable ID. ID does not appear to negatively affect transplantation outcomes. Future studies are needed to assess long-term outcomes of transplantation in this population.

Recommendations for utilization of the paracorporeal lung assist device in neonates and young children with pulmonary hypertension.

The management of decompensating critically ill children with severe PH is extremely challenging and requires a multidisciplinary approach. Unfortunately, even with optimal care, these children might continue to deteriorate and develop inadequate systemic perfusion and at times cardiac arrest secondary to a pulmonary hypertensive crisis. Tools to support these children are limited, and at times, the team should proceed with offering extracorporeal support, especially in newly diagnosed patients who have not benefitted from medical therapy prior to their acute deterioration, in patients with severe pulmonary venous disease and in patients with alveolar capillary dysplasia. Currently, the only approved mode for extracorporeal support in pediatric patients with PH eligible for lung transplantation is ECMO. To decrease the risks associated with ECMO, and offer potential for increased duration of support, extubation, and rehabilitation, we transitioned four small children with refractory PH from ECMO to a device comprising an oxygenator interposed between the PA and LA. This work describes in great detail our experience with this mode of support with emphasis on exclusion criteria, the implantation procedure, and the post-implantation management.

Lung transplantation for pulmonary fibrosis in dyskeratosis congenita: Case Report and systematic literature review.

BACKGROUND: Dyskeratosis congenita (DC) is a progressive, multi-system, inherited disorder of telomere biology with high risks of morbidity and mortality from bone marrow failure, hematologic malignancy, solid tumors and pulmonary fibrosis. Hematopoietic stem cell transplantation (HSCT) can cure the bone marrow failure, but it does not eliminate the risks of other complications, for which life-long surveillance is required. Pulmonary fibrosis is a progressive and lethal complication of DC. CASE PRESENTATION: In this report, we describe a patient with DC who developed pulmonary fibrosis seven years after HSCT for severe aplastic anemia, and was successfully treated with bilateral lung transplantation. We also performed a systematic literature review to understand the burden of pulmonary disease in patients with DC who did or did not receive an HSCT. Including our patient, we identified 49 DC patients with pulmonary disease (12 after HSCT and 37 without HSCT), and 509 with no reported pulmonary complications. CONCLUSION: Our current case and literature review indicate that pulmonary morbidity is one of the major contributors to poor quality of life and reduced long-term survival in DC. We suggest that lung transplantation be considered for patients with DC who develop pulmonary fibrosis with no concurrent evidence of multi-organ failure.

Successful subxyphoid hybrid approach for placement of a Melody percutaneous pulmonary valve.

A variety of complex congenital heart defects in the pediatric population involve placement of a right ventricle to pulmonary artery conduit as part of surgical repair. With the advent of percutaneous pulmonary valve implantation (PPVI), patients may avoid the risks of serial surgical reinterventions as the PPVI acts to prolong the life of a previously placed conduit. As the experience with PPVI is growing, new challenges arise from complicated anatomy and severe conduit stenosis. We present a case of a 16-year-old male who underwent successful pulmonary valve placement with a Melody valve via a subxyphoid hybrid approach after an unsuccessful attempt at percutaneous placement.

Pediatric lung transplantation.

OPINION STATEMENT: Lung transplantation in children is an effective treatment for end-stage pulmonary disease after all medical therapy has failed. It requires a huge investment in resources and absolute commitment on the part of the parents and patients. In spite of all these efforts and expense, the results with pediatric lung transplantation are the worst of all solid organ transplants. Much lies ahead to overcome the obstacles faced to improve this therapy.

The Failed Bidirectional Glenn Shunt: Risk Factors for Poor Outcomes and the Role of Early Reoperation.

Background: Bidirectional Glenn shunt (BDG) failure carries high morbidity and mortality but the clinical factors associated with failure and the optimal management strategy are understudied. Methods: A total of 217 patients undergoing BDG at our institution between 1989 and 2020 were retrospectively reviewed and categorized as success or failure. Failure was defined as the need for reoperation (BDG takedown, reoperation for correction of cardiac defect, and/or transplantation) at any time postoperatively; operative mortality (death attributable to BDG malfunction occurring during the index hospitalization for BDG or within 30 days of discharge); or late mortality (death directly attributable to BDG malfunction occurring prior to Fontan or next-stage palliation). Univariate and binary logistic regression analyses were performed. Results: BDG failure occurred in 14 (6.5%) patients. Univariate predictors were: hypoplastic left heart syndrome (P = .037), right ventricular (RV) dominance (P = .010), greater pre-BDG pulmonary vascular resistance (PVR) (P = .012), concomitant atrioventricular valve repair (P = .020), prolonged pleural drainage (P = .001), intensive care unit (P<.001) and hospital (P = .002) stays, and extracorporeal membrane oxygenation (ECMO) requirement (P<.001). Multivariate predictors were: RV dominance (P = .002), greater PVR (P = .041), ICU (P<.001) and hospital (P = .020) stays, and need for ECMO (P<.001). As many as 10 of 14 (71%) patients with BDG failure died. Reoperation was performed for 10 patients with BDG failure. Five reoperation patients survived until discharge, with four patients alive at last follow-up (mean 7.9 years). Survivors underwent reoperation earlier than nonsurvivors (36 vs. 94 days). Conclusions: BDG failure carries high mortality, but preoperative predictors and postoperative indicators of failure exist. Early BDG takedown and insertion of aorta-pulmonary shunt may allow survival.

Lung transplantation for pulmonary hypertension in children.

Lung and heart-lung transplantation are accepted treatments for children with end-stage pulmonary vascular disease. This is a review of the current literature and our own experience with lung and heart-lung transplantation for children with pulmonary hypertension of a variety of causes. I reviewed the pertinent literature and our lung transplant database to acquire information and data regarding this subject. The patients include those at St. Louis Children's Hospital as well as those reported from other institutions. The major operative complications include those related to the surgical procedure itself (vascular and airway anastomotic stenoses) and those related to graft dysfunction. The 3- and 5-yr survival is approximately 60% and 50%, respectively, for children undergoing lung transplantation for pulmonary hypertension.Although these survival statistics are somewhat poor, transplantation remains the only viable alternative for children with end-stage pulmonary vascular disease failing to respond to medical therapy.

Biventricular assist devices as a bridge to heart transplantation in small children.

BACKGROUND: Experience with the use of biventricular assist device (BiVAD) support to bridge small children to heart transplantation is limited. METHODS AND RESULTS: We used BIVAD support (Berlin EXCOR) in 9 pediatric heart transplant candidates from 4/05 to 7/07. The median patient age was 1.7 years (12 days to 17 years). The median patient weight was 9.4 kg (3 to 38 kg). All children were supported with multiple intravenous inotropes+/-mechanical ventilation (6) or ECMO (3) before BiVAD implantation. All had significant right ventricular dysfunction. The median pulmonary vascular resistance index (Rpi) was 6.0 WU/m(2). Eight patients were successfully bridged to heart transplantation after a median duration of BiVAD support of 35 days (1 to 77 days). One death occurred after 10 days of support from perioperative renal failure in a 3 kg infant. Five patients required at least 1 blood pump change. One patient had a driveline infection requiring treatment. There were no acute neurological complications, no thromboembolic events, and no bleeding complications. In 2 patients with Rpi >10 WU/m(2) unresponsive to pulmonary vasodilator therapy, Rpi dropped to 1.4 and 4.6 WU/m(2), after 33 and 41 days of support, respectively. All 8 survivors underwent successful heart transplantation. Of 5 patients supported >30 days, 3 developed an extremely elevated (>90%) panel reactive antibody by ELISA that was not confirmed by other methods; none had a positive donor-specific retrospective crossmatch. There was 1 episode of rejection (with hemodynamic compromise) in the 8 transplanted patients. Rpi was normal (<3 WU/m(2)) without pulmonary vasodilators in all patients within 3 months after transplant. There have been no deaths after transplant with a median follow-up of 19 months. CONCLUSIONS: BiVAD support can effectively be used in small children as a bridge to heart transplantation and can be accomplished with low mortality and morbidity. BiVAD support may offer an additional means to reverse extremely elevated pulmonary vascular resistance. Surveillance for HLA antibody sensitization during BiVAD support may be complicated by the development of non-HLA antibodies which may not reflect true HLA presensitization.

Hypoplastic left heart associated with scimitar syndrome.

A child with the unique combination of hypoplastic left heart syndrome (HLHS) and scimitar syndrome is presented. Her HLHS was diagnosed in utero, and her scimitar syndrome was discovered during her immediate newborn period. She underwent a successful Norwood operation complicated by supraventricular tachycardia given her Wolf-Parkinson-White syndrome. She has also undergone successful Glenn shunt and at this writing is thriving.

Infectious, malignant, and autoimmune complications in pediatric heart transplant recipients.

OBJECTIVE: To review clinical courses of pediatric heart transplant survivors after 5 years from transplantation for infections, lymphoproliferative, and autoimmune diseases. STUDY DESIGN: A total of 71 patients were examined in 2 groups, infant recipients (underwent transplant <1 year of age, n = 38) and older recipients (underwent transplant >1 year, n = 33). All patients received comparable immunosuppression. Calculated occurrence rates were reported as means per 10 years of follow-up with SEs. Differences were examined by using Poisson regression. RESULTS: Infant recipients had significantly higher (P < .001) occurrence rates of severe (mean, 2.04 +/- 0.5) and chronic infections (mean, 4.58 +/- 0.67) compared with older recipients (means, 0.37 +/- 0.19 and 1.87 +/- 0.70, respectively). Types of infections were similar to those in the general population with extremely rare opportunistic infections; however, they were more severe and resistant to treatment. Autoimmune disorders occurred at a frequency comparable with lymphoproliferative diseases and were observed in 7 of 38 infants (18%). Most common were autoimmune cytopenias. CONCLUSIONS: Infant heart transplant recipients who survive in the long term have higher occurrence rates of infections compared with older recipients. Autoimmune disorders are a previously unrecognized morbidity in pediatric heart transplantation.

A novel mutation of the MYH7 gene in a patient with hypertrophic cardiomyopathy.

Goel N, Huddleston CB, Fiore AC. A novel mutation of the MYH7 gene in a patient with hypertrophic cardiomyopathy. Turk J Pediatr 2018; 60: 315-318. Hypertrophic cardiomyopathy (HCM) is a genetic disorder characterized by asymmetric cardiac hypertrophy due to inherited mutations in genes that encode sarcomeric proteins. MYH7, which encodes ß-myosin heavy chain, is among the most commonly mutated genes in patients affected by HCM. We aimed to identify the specific mutation responsible for HCM in a six-month old Caucasian patient. NextGen DNA sequencing revealed a novel p.Ala1328Thr (A1328T) mutation of MYH7 in the affected patient as well as his asymptomatic father and asymptomatic brother. The clinical details of this mutation are described for the first time in this report. The genetic variant affects a residue that is highly conserved across species. Theoretical analysis suggests that A1328T is very likely deleterious to ß-myosin heavy chain protein structure and function. Furthermore, this novel mutation was not observed with any significant frequency in approximately 6,500 healthy individuals of European and African American ancestry in the NHLBI Exome Sequencing Project, underlining the potential pathogenicity of this variant.

Type A Aortic Dissection After the Ross Procedure.

We describe a case of type A aortic dissection in a 25-year-old woman who had undergone a Ross procedure for aortic valve endocarditis 13 years previously. She was pregnant and noted to have significant enlargement of the aortic root during the latter portion of the third trimester of her pregnancy. An echocardiogram after delivery demonstrated new aortic valve insufficiency in addition to an aortic root diameter of 6.5 cm. A computed tomography angiogram demonstrated a type A aortic dissection involving only the very proximal portion of the ascending aorta. At operation, the dissection was found to be limited to the pulmonary autograft. This was repaired using a valve-sparing technique. Her postoperative course was uneventful, and the aortic valve has shown only trace insufficiency at 3 years of follow-up.

Lower incidence of bronchiolitis obliterans in pediatric liver-lung transplant recipients with cystic fibrosis.

BACKGROUND: Simultaneous liver-lung transplantation is an infrequent but technically feasible procedure in patients with end-stage lung disease and advanced liver disease. We characterize the outcomes of pediatric patients who underwent this procedure at our institution. METHODS: We performed a retrospective, case-control study and reviewed the medical records of all patients referred to our transplant program from its inception. Seven patients were listed for simultaneous liver-lung transplant. The five patients who survived to transplant were matched to 13 controls who underwent isolated bilateral sequential lung transplant for underlying diagnosis, age at time of transplant, gender, and era of transplant. Outcome measures included patient and graft survival, occurrence of bronchiolitis obliterans (BO), and episodes of rejection. RESULTS: Of the five study patients who underwent liver-lung transplant, one died of multiorgan failure 11 days after transplant compared with 9 of 13 controls who died. The median survival for the study patients was 89 months (range, 0-112 months) compared with the controls, who had a median survival of 34 months (range, 0-118 months). The remaining four patients had bronchiolitis obliterans syndrome scores of 0 compared with 5 of 13 control patients (P=0.02). The rate of acute rejection per 100 patient days was 0.012 for the study patients compared with 0.11 for the controls (P=0.025). CONCLUSIONS: Simultaneous liver-lung transplantation is a technically feasible procedure with excellent long-term outcomes. The surviving study subjects remain free from bronchiolitis obliterans syndrome. These results suggest that the transplanted liver may bestow immunologic privilege to the lung allograft.