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Background Although mortality has decreased considerably in pediatric heart transplantation, waitlist and post-transplant death rates remain notable. End-of-life focused research in this population, however, is very limited. This Pediatric Heart Transplant Society study aimed to describe the circumstances surrounding death of pediatric heart transplant patients. Methods A retrospective analysis of the multi-institutional, international, Pediatric Heart Transplant Society registry was conducted. Descriptive statistics and univariate analyses were performed to 1) describe end-of-life in pediatric pre- and post-heart transplant patients and 2) examine associations between location of death and technological interventions at end-of-life with demographic and disease factors. Results Of 9217 patients (0-18 years) enrolled in the registry between 1993 and 2018, 2804 (30%) deaths occurred; 1310 while awaiting heart transplant and 1494 post-heart transplant. The majority of waitlist deaths (89%) occurred in the hospital, primarily in ICU (74%) with most receiving mechanical ventilation (77%). Fewer post-transplant deaths occurred in the hospital (22%). Out-of-hospital death was associated with older patient age (p Conclusions ICU deaths with high use of technological interventions at end-of-life were common, particularly in patients awaiting heart transplant. In this high mortality population, findings raise challenging considerations for clinicians, families, and policy makers on how to balance quality of life amidst high risk for hospital-based death.

BACKGROUND We aimed to describe the incidence, risk factors, and clinical outcomes of pericardial effusions within 6 months after pediatric heart transplantation (HT). METHODS A single-center retrospective cohort study was performed on all pediatric HT recipients from 2004 to 2018. Logistic regression was used to identify factors associated with pericardial effusions post-HT, and survival was compared using log-rank test. RESULTS During the study period, 97 HTs were performed in 93 patients. Fifty patients (52%) had a ≥small pericardial effusion within 6 months, 16 of which were, or became, ≥moderate in size. Pericardial drain was placed in 8 patients. In univariate analysis, larger recipient body surface area (p = .01) and non-congenital heart disease (p = .002) were associated with pericardial effusion development. Donor/recipient size ratios, post-HT hemodynamics, and rejection did not correlate with pericardial effusion development. In multivariable analysis, non-congenital heart disease (adjusted odds ratio 3.3, p = .01) remained independently associated with development of pericardial effusion. There were no significant differences in post-HT survival between patients with and without ≥small (p = .68) or ≥moderate pericardial effusions (p = .40). CONCLUSIONS Pericardial effusions are common after pediatric HT. Patients with cardiomyopathy, or non-congenital heart disease, were at higher risk for post-HT pericardial effusions. Pericardial effusions increased morbidity but had no effect on mortality in our cohort. The risk factors identified may be used for anticipatory guidance in pediatric HT.

Background In a stable, inotrope-dependent pediatric patient with dilated cardiomyopathy, we evaluated the cost-effectiveness of continuous-flow VAD implantation compared to a watchful waiting approach using chronic inotropic therapy. Methods We used a state-transition model to estimate the costs and outcomes of 14-year-old (INTERMACS profile 3) patients receiving either VAD or watchful waiting. We measured benefits in terms of lifetime QALYs gained. Model inputs were taken from the literature. We calculated the ICER, or the cost per additional QALY gained, of VADs and performed multiple sensitivity analyses to test how our assumptions influenced the results. Results Compared to watchful waiting, VADs produce 0.97 more QALYs for an additional $156 639, leading to an ICER of $162 123 per QALY gained from a healthcare perspective. VADs have 17% chance of being cost-effective given a cost-effectiveness threshold of $100 000 per QALY gained. Sensitivity analyses suggest that VADs can be cost-effective if the costs of implantation decrease or if hospitalization costs or mortality among watchful waiting patients is higher. Conclusions As a bridge to transplant, VADs provide a health benefit to children who develop stable, inotrope-dependent heart failure, but immediate implantation is not yet a cost-effective strategy compared to watchful waiting based on commonly used cost-effectiveness thresholds. Early VAD support can be cost-effective in sicker patients and if device implantation is cheaper. In complex conditions such as pediatric heart failure, cost-effectiveness should be just one of many factors that inform clinical decision-making.

Heart transplant providers often focus on post-transplant outcomes when making donor decisions, potentially at the expense of higher waitlist mortality. This study aimed to assess public opinion regarding the selection of donor hearts and the balance between pre- and post-transplant risk. The authors generated a survey to investigate public opinion regarding donor acceptance. The survey was shared freely online across social media platforms in April-May 2019. A total of 718 individuals responded to the survey, with an equal distribution between patients and family members. Respondents consistently favored post-transplant outcomes over waitlist outcomes. About 83.9% of respondents favored a hospital with longer waitlist times, worse waitlist outcomes, but excellent post-transplant survival over a hospital with short waitlist times, a high waitlist survival, and inferior post-transplant survival. This preference was no different between pediatric and adult populations (P = .7), patient and family members (P = .935), or those with a pre- vs post-transplant perspective (P = .985). Patients and their family members consistently favor improved post-transplant survival over waitlist survival when considering the risks of accepting a donor organ. These findings suggest that current practice patterns of donor selection align with the opinions of patients and family members with heart failure or who have undergone heart transplantation.

Background Despite advancements in treatment and survival, pediatric organ failure and transplant populations continue to face significant risks of morbidity and mortality. Little scientific attention has been given to addressing the end-of-life care needs of this growing population of young people. This study characterized current practices, beliefs, and challenges specific to the disclosure of prognosis and end-of-life care topics among providers caring for pediatric organ failure and transplant populations. Methods This cross-sectional study included 144 healthcare providers actively caring for children, adolescents, and young adults with organ failure or solid organ transplant history. Participants completed an electronic survey measuring frequency and comfort in discussing the following topics with patients and parents: prognosis/survival statistics, re-transplantation, advance care planning (ACP), and death/dying. Descriptive statistics, two-sample t tests, and analysis of variance were used. Results Fewer than half of respondents regularly discuss prognosis/survival statistics and potential need for re-transplantation with their pediatric and young adult patients. Less than 20% of providers engage their pediatric patients in ACP discussions, and approximately 30% facilitate such discussions with young adult patients. Pediatric organ failure and transplant providers endorse a number of barriers specific to discussing these topics. Conclusion Pediatric organ failure and transplant providers do not regularly discuss prognosis or end-of-life care topics with this patient population. Communication-focused intervention research is needed to improve honest and compassionate discussion of these topics that is aligned with both patients' and parents' needs and preferences.

Autoimmune-mediated bowel disease has been reported after pediatric heart transplantation. Recognition and treatment of these patients has been difficult. We describe a patient who responded to steroids and basiliximab therapy after an inflammatory process secondary to abnormal T-cell activation. Our patient is a 28-month-old female who received a heart transplant at five wk of age. At 24 months post-transplant, she developed fever and bloody stools. Initial investigations were significant for an elevated ESR (>120) and CRP (15.2). Symptoms persisted despite bowel rest and mycophenolate discontinuation. Endoscopic evaluation revealed discontinuous ulcerative disease involving esophagus, terminal ileum, right and left colon, necessitating extensive bowel resection. She had additional airway inflammation leading to a TEF at the site of esophageal ulceration, requiring tracheostomy. Immune evaluation revealed autoimmune dysregulation that responded to parenteral methylprednisolone. Chronic basiliximab therapy allowed for successful weaning of steroids with sustained remission. She has been transitioned to sirolimus and tacrolimus maintenance immunosuppression with plans to discontinue basiliximab once off steroids. In conclusion, bowel disease in the setting of pediatric heart transplantation can be severe and refractory to traditional treatment methods. Tailoring immune therapy to activated T cells can result in remission. Basiliximab therapy was used in our patient to maintain steroid-induced remission, but long-term complications of this disease process are unknown.

Prolonged graft ischemia may be a risk factor for early rejection post-HTx, but this has not been well studied in children. Furthermore, factors moderating the association between IT and early rejection have not been investigated. From 2004 to 2012, pediatric HTx recipients (n = 2381) were identified from the UNOS database. A ROC curve determined the optimal IT discriminating patients by the presence of early rejection. Separate univariate analyses identified factors associated with: (i) early (prior to hospital discharge) rejection, and (ii) IT. A multivariable logistic regression assessed independent risk factors for early rejection. We included interaction terms to evaluate whether IT's independent risk effect on early rejection is moderated via interaction with associated factors found in univariate analysis. Longer IT was associated with an increased risk of early rejection. In multivariable analysis, IT > 3.1 hours was an independent risk factor for early rejection (AOR 1.44, P = .01). No interaction terms between IT and any associated factors were significant. Longer IT is an independent risk for early rejection in pediatric HTx recipients. Better understanding the association between IT and early rejection may identify interventions to mitigate this risk.

While VAD use in pediatric patients has previously been associated with anti-HLA antibody production, the clinical significance of these antibodies is unclear. We investigated the clinical impact of anti-HLA antibodies associated with VAD use in a large cohort of pediatric HTx recipients. From 2004 to 2011, pediatric cardiomyopathy patients post-HTx (N=1288) with pre-HTx PRA levels were identified from the United Network for Organ Sharing database. PRA levels were compared between VAD patients and those with no history of MCS. Incidence of rejection and overall survival were compared between VAD and non-MCS groups after stratification by PRA and age. VAD recipients were more likely to produce anti-HLA antibodies than non-MCS patients (25.5% vs 10.5% had PRA10%, P.0001). Sensitized VAD patients (PRA10%) had a higher incidence of rejection within 15 months of HTx compared to sensitized non-MCS patients (57.1% vs 35.9%, P=.02). There was no intergroup difference in 15-month mortality. Among pediatric cardiomyopathy patients supported with a VAD, the presence of anti-HLA antibodies prior to HTx is associated with an increased risk of rejection. The mechanism of the association between VAD-associated antibodies and early rejection is unclear and warrants further investigation.

The impact of nutritional status on HTx waitlist mortality in children is unknown, and there are conflicting data regarding the role of nutrition in post-HTx survival. This study examined the influence of nutrition on waitlist and post-HTx outcomes in children. Children 2-18 yr listed for HTx from 1997 to 2011 were identified from the OPTN database and stratified by BMI percentile. Multivariable logistic regression evaluated the influence of BMI on waitlist mortality. Cox proportional hazard regression assessed the impact of BMI on post-HTx mortality. When all 2712 patients were analyzed, BMI did not impact waitlist, one-, or five-yr mortality. However, when stratified by diagnosis, BMI 95% (AOR 1.96; 95% CI 1.24, 3.09) and BMI 1% (AOR 2.17; 95% CI 1.28, 3.68) were independent risk factors for waitlist mortality in patients with CM. BMI did not impact waitlist mortality in CHD and did not impact post-HTx outcomes, regardless of diagnosis. BMI 95% and BMI 1% are independent risk factors for waitlist mortality in patients with CM, but not CHD. This suggests differing risk factors based on disease etiology, and an individualized approach to risk assessment based on diagnosis may be warranted.

Across pediatric organ transplant populations, patient and family psychosocial functioning is associated with important health-related outcomes. Research has suggested that pediatric heart transplant recipients and their families are at increased risk for adverse psychosocial outcomes; however, recent investigation of psychosocial functioning in this population is lacking. This study aimed to provide a contemporary characterization of psychosocial functioning in pediatric heart transplant recipients and their families. Associations between psychosocial function, demographic variables, and transplant-related variables were investigated. Fifty-six parents/guardians of pediatric heart transplant recipients completed a comprehensive psychosocial screening measure during transplant follow-up clinic visits. Descriptive statistics, correlational analyses, and independent samples t tests were performed. Forty percent of pediatric heart transplant recipients and their families endorsed clinically meaningful levels of total psychosocial risk. One-third of patients presented with clinically significant psychological problems per parent report. Psychosocial risk was unassociated with demographic or transplant-related factors. Despite notable improvements in the survival of pediatric heart transplant recipients over the past decade, patients and families present with sustained psychosocial risks well beyond the immediate post-transplant period, necessitating mental health intervention to mitigate adverse impact on health-related outcomes.

We present a case of coronary ectasia and LAD coronary artery aneurysm with angiographic characteristics of Kawasaki disease in a three-yr-old girl two-yr status post-orthotopic heart transplant. Coronary anomalies were noted during initial screening coronary angiography two yr after transplant. Subsequent review of the donor echocardiogram revealed that the LMCA had been mildly dilated prior to transplant. In the absence of any symptoms consistent with Kawasaki disease in the transplant recipient, this appears to be a case of Kawasaki disease in the organ donor manifesting with coronary anomalies in the transplant recipient. The patient has done well clinically, and repeat coronary angiography has revealed partial regression of coronary anomalies. Given multiple reports in the literature of persistent abnormalities of coronary artery morphology and function after Kawasaki disease, close monitoring is warranted, with consideration of potential coronary protective medical therapies.

PLE is an important cause of morbidity and mortality in patients who have undergone Fontan palliation. While multiple PLE therapies have been reported, none has proved consistently effective. Patients who do not respond to "standard" PLE therapies face poor long-term outcomes. We report here a significant response to dopamine infusion in three patients with chronic, refractory PLE. We hypothesize that this response may be at least partially due to a dopamine effect on lymphatic receptors rather than to an augmentation of cardiac output.

The process of pediatric solid organ transplantation (SOT) places new and increased stressors on patients and family members. Measures of family functioning may predict psychological and health outcomes for pediatric patients and their families, and provide opportunity for targeted intervention. This systematic review investigated parent and family functioning and factors associated with poorer functioning in the pediatric SOT population. Thirty-seven studies were identified and reviewed. Studies featured a range of organ populations (eg, heart, liver, kidney, lung, intestine) at various stages in the transplant process. Findings highlighted that parents of pediatric SOT populations commonly report increased stress and mental health symptoms, including posttraumatic stress disorder. Pediatric SOT is also associated with increased family stress and burden throughout the transplant process. Measures of parent and family functioning were associated with several important health-related factors, such as medication adherence, readiness for discharge, and number of hospitalizations. Overall, findings suggest that family stress and burden persists post-transplant, and parent and family functioning is associated with health-related factors in SOT, highlighting family-level functioning as an important target for future intervention.