The Fontan immunophenotype and post-transplant outcomes in children: A multi-institutional study.

Background: Patients after Fontan palliation represent a growing pediatric population requiring heart transplant (HTx) and often have lymphopenia (L) and/or hypogammaglobinemia that may be exacerbated by protein-losing enteropathy (PLE, P). The post-HTx effects of this altered immune phenotype are not well studied.

Methods: In this study of the Pediatric Heart Transplant Society Registry, 106 Fontan patients who underwent HTx between 2005 and 2018 were analyzed.

Pediatric heart transplant from an incompletely treated influenza A-positive donor.

There is a shortage of pediatric donor hearts for waitlisted children, and yet nearly 50% of organs offered are not transplanted. Donor quality is often cited as a reason for declining organs offered from donors infected with influenza, presumably due to concern about disease transmission at transplant leading to severe disease. We previously described an excellent outcome after heart transplant from a donor infected with influenza B that had been treated with a complete course of oseltamivir.

Trajectory of right ventricular indices is an early predictor of outcomes in hypoplastic left heart syndrome.

Background: Children with hypoplastic left heart syndrome (HLHS) have risk for mortality and/or transplantation. Previous studies have associated right ventricular (RV) indices in a single echocardiogram with survival, but none have related serial measurements to outcomes. This study sought to determine whether the trajectory of RV indices in the first year of life was associated with transplant-free survival to stage 3 palliation (S3P).

Pediatric heart transplantation from an influenza B-positive donor.

As heart transplantation demand is increasing without subsequent growth of the donor pool, need for expansion of acceptance criteria is paramount, particularly when considering critically ill, highly sensitized patients. We present a case report of a pediatric heart transplant recipient of an organ refused by 197 prior potential recipients due to the donor being infected with influenza virus. We perform a literature review of recent solid organ transplant cases from influenza-positive donors and conclude that the donor pool may be expandable by allowing donors with treatable infections to be included.

Gain-of-function mutations in SMAD4 cause a distinctive repertoire of cardiovascular phenotypes in patients with Myhre syndrome.

Myhre syndrome is a rare, distinctive syndrome due to specific gain-of-function mutations in SMAD4. The characteristic phenotype includes short stature, dysmorphic facial features, hearing loss, laryngotracheal anomalies, arthropathy, radiographic defects, intellectual disability, and a more recently appreciated spectrum of cardiovascular defects with a striking fibroproliferative response to surgical intervention. We report four newly described patients with typical features of Myhre syndrome who had (i) a mildly narrow descending aorta and restrictive cardiomyopathy; (ii) recurrent pericardial and pleural effusions; (iii) a large persistent ductus arteriosus with juxtaductal aortic coarctation; and (iv) restrictive pericardial disease requiring pericardiectomy.

Bortezomib use in a pediatric cardiac transplant center.

Data are limited on the efficacy and safety of bortezomib for the treatment of AMR following OHT for pediatric acquired or CHD. Retrospective chart review identified patients who received bortezomib for acute (n = 3, within two wk of diagnosis) and chronic (n = 1, three months after diagnosis) AMR or as part of a desensitization regimen (n = 1). Bortezomib was associated with a 3-66% reduction in class I DSA and a 7-82% reduction in class II DSA.

Predictors of cardiac allograft vasculopathy in pediatric heart transplant recipients.

CAV remains a leading cause of late graft loss and mortality among survivors of pediatric heart transplantation. We sought to define the incidence of CAV and identify its predictors in pediatric heart transplant recipients. The OPTN/UNOS database was analyzed for pediatric recipients who underwent heart transplant between 1987 and 2011.

Orthotopic heart transplant: a therapeutic option for unresectable cardiac fibroma in infants.

Primary cardiac tumors are rare lesions in childhood, with the two most common being rhabdomyoma and fibroma. We report two infants who successfully underwent orthotopic heart transplant for massive interventricular septal cardiac fibromas. For unresectable infantile cardiac fibroma, orthotopic heart transplant may be considered a therapeutic option.

Recurrent protein-losing enteropathy and tricuspid valve insufficiency in a transplanted heart: a causal relationship?

This case report describes a toddler who developed a protein-losing enteropathy (PLE) 4 years after orthotopic heart transplantation (OHT). He was born with a hypoplastic left heart syndrome for which he underwent a successful Norwood procedure, a Hemi-Fontan palliation, and a Fontan palliation at 18 months of age. Fifteen months following the Fontan operation, he developed a PLE and Fontan failure requiring OHT.

Perioperative renal failure in pediatric heart transplant recipients: outcome and risk factors.

PRF is encountered in 10-13% of adult heart transplants. Only one study of a single center's experience with PRF has been reported in pediatric patients. This study examines the effect of PRF on pediatric heart transplant outcome using the UNOS database.

Effect of hypothermia on doxorubicin-induced cardiac myoblast signaling and cell death.

Background: Anthracyclines (AC) are useful chemotherapeutic agents whose principal limitation is cardiac toxicity, which may progress to heart failure, transplantation or even death. We have shown that this toxicity involves oxidative stress-induced activation of the DNA damage pathway. Hypothermia has been shown to be protective against other diseases involving oxidative stress but has not been studied in models of AC toxicity.

Low donor-to-recipient weight ratio does not negatively impact survival of pediatric heart transplant patients.

A major limitation to success in pediatric heart transplantation is donor organ shortage. While the use of allografts from donors larger than the recipient is accepted, the use of undersized donor grafts is generally discouraged. Using the UNOS database, we wanted to evaluate whether using smaller donor hearts affects the short- and long-term survival of pediatric heart transplant patients.

Identification and cloning of a new protein that binds the 3(') untranslated region of alpha-striated tropomyosin.

The 3' untranslated region of muscle tropomyosin (TM UTR) induces muscle differentiation when transcribed in primary fibroblasts. This sequence binds protein in extracts from cell types that differentiate upon TM UTR transcription. To identify the protein(s) bound by the TM UTR, an avian embryo fibroblast library was induced to express protein in solution and extracts from these pools were screened with electromobility shift assays using a TM UTR RNA probe.