Background
Pediatric heart transplantation (HT) is resource intensive. In adults, there has been an increase in the proportion of HTs funded by public insurance, with post‐HT outcomes inferior to ...those funded by private sources. Trends in the funding of pediatric HT and outcomes in children have not been described.
Methods
We queried the United Network for Organ Sharing (UNOS) database for children (<18 years) listed for and undergoing HT between 2004 and 2021. We identified the primary payer at listing, HT, 1 year, and 1–5 years following HT. Trends were analyzed using generalized logit models. Multivariable‐extended Cox regression models were used to test the relationship between insurance type at the time of transplant and time to death or re‐transplant.
Results
There were 6382 pediatric patients who underwent transplants and had either public or private insurance at the time of transplant. The percentage of patients with public insurance at the time of HT increased over time. Public insurance at the time of HT was associated with an increased risk of death or re‐transplant beyond 2 months after HT (adjusted HR at 6 months = 1.43, 95% CI: 1.13–1.81, p = .003; adjusted HR at 9 months = 1.67, 95% CI: 1.17–2.37, p = .004).
Conclusion
There has been a statistically significant trend toward increasing public insurance for children awaiting, at the time of, and after HT. Black patients and those with public insurance at HT have worse long‐term outcomes. This study highlights ongoing disparities in pediatric HT and the need to focus efforts on achieving equitable outcomes.
As survival and neuromuscular function in Duchenne muscular dystrophy (DMD) have improved with glucocorticoid (GC) therapy and ventilatory support, cardiac deaths are increasing. Little is known ...about risk factors for cardiac and non-cardiac causes of death in DMD. A multi-center retrospective cohort study of 408 males with DMD, followed from January 1, 2005 to December 31, 2015, was conducted to identify risk factors for death. Those dying of cardiac causes were compared to those dying of non-cardiac causes and to those alive at study end. There were 29 (7.1%) deaths at a median age of 19.5 (IQR: 16.9–24.6) years; 8 (27.6%) cardiac, and 21 non-cardiac. Those living were younger 14.9 (IQR: 11.0–19.1) years than those dying of cardiac 18 (IQR 15.5–24) years,
p
= 0.03 and non-cardiac 19 (IQR: 16.5–23) years,
p
= 0.002 causes. GC use was lower for those dying of cardiac causes compared to those living 2/8 (25%) vs. 304/378 (80.4%)
, p
= 0.001. Last ejection fraction prior to death/study end was lower for those dying of cardiac causes compared to those living (37.5% ± 12.8 vs. 54.5% ± 10.8,
p
= 0.01) but not compared to those dying of non-cardiac causes (37.5% ± 12.8 vs. 41.2% ± 19.3,
p
= 0.58). In a large DMD cohort, approximately 30% of deaths were cardiac. Lack of GC use was associated with cardiac causes of death, while systolic dysfunction was associated with death from any cause. Further work is needed to ensure guideline adherence and to define optimal management of systolic dysfunction in males with DMD with hopes of extending survival.
Background Anomalous aortic origin of a coronary artery with an interarterial and intramural course (AAOCA) is a rare anomaly with increased risk of sudden cardiac death during or just after exercise ...among otherwise healthy youth. Risk stratification and management remain controversial, especially for the asymptomatic child with an anomalous right coronary artery from the left coronary sinus (ARCA). Medium-term surgical and quality-of-life (QOL) outcome data are lacking in this population. Methods We performed medical record review on 24 subjects who underwent AAOCA repair between 2001 and 2007 at The Children's Hospital of Philadelphia. QOL was prospectively assessed with age-appropriate questionnaires. Results Median age at follow-up was 18 (range, 11-25) years, median follow-up from surgery was 63 (range, 12-110) months, and 16 (67%) had ARCA. All were alive without exercise restriction. Thirteen (54%) complained of cardiac-type symptoms postoperatively, most commonly chest pain, none correlating with evidence of ischemia on testing. Of the 13 patients, 7 (54%) reported the same symptoms preoperatively; and of these, 5 had ARCA. Postoperative morbidity occurred in 16 (67%), including pericardial effusion (n = 11), wound infection (n = 2), and development of mild aortic insufficiency (n = 4). QOL questionnaires were sent to 21 subjects; 12 (57%) were returned. Average QOL was normal for all subjects. Conclusions In the medium-term after AAOCA repair, cardiac-type symptoms frequently persist and morbidity is common, but these do not impair QOL. The significance of these findings in the long-term is unknown and warrants continued follow-up.
Background Duchenne and Becker muscular dystrophy are progressive disorders associated with cardiac mortality. Guidelines recommend routine surveillance; we assess cardiac resource use and identify ...gaps in care delivery. Methods and Results Male patients, aged 1 to 18 years, with Duchenne and Becker muscular dystrophy between January 2013 and December 2017 were identified in the IBM MarketScan Research Database. The cohort was divided into <10 and 10 to 18 years of age. The primary outcome was rate of annual health care resource per person year. Resource use was assessed for place of service, cardiac testing, and medications. Adjusted incidence rate ratios (IRRs) were estimated using a Poisson regression model. Medication use was measured by proportion of days covered. There were 1386 patients with a median follow-up time of 3.0 years (interquartile range, 1.9-4.7 years). Patients in the 10 to 18 years group had only 0.40 (95% CI, 0.35-0.45) cardiology visits per person year and 0.66 (95% CI, 0.62-0.70) echocardiography/magnetic resonance imaging per person year. Older patients had higher rates of inpatient admissions (IRR, 1.46; 95% CI, 1.03-2.09), outpatient cardiology visits (IRR, 2.0; 95% CI, 1.66-2.40), cardiac imaging (IRR, 1.59; 95% CI, 1.40-1.80), and Holter monitoring (IRR, 3.33; 95% CI, 2.35-4.73). A proportion of days covered >80% for angiotensin-converting enzyme inhibitors/angiotensin receptor blockers was observed in 13.6% (419/3083) of total person years among patients in the 10 to 18 years group. Conclusions Children 10 to 18 years of age have higher rates of cardiac resource use compared with those <10 years of age. However, rates in both age groups fall short of guidelines. Opportunities exist to identify barriers to resource use and optimize cardiac care for patients with Duchenne and Becker muscular dystrophy.
To report an unusual case of simultaneous presentation of Addison's and Graves' disease in an adolescent female previously diagnosed with type 1 diabetes (T1D) and Hashimoto's.
A 15-year-old female ...with T1D and hypothyroidism presented to the emergency department with altered mental state, fever, and left arm weakness for one day. Clinical work-up revealed coexistent new-onset adrenal insufficiency and hyperthyroidism. Her clinical course was complicated by severe, life-threating multisystem organ dysfunction including neurologic deficits, acute kidney injury, and fluid overload. Thyroidectomy was ultimately performed in the setting of persistent signs of adrenal crises and resulted in rapid clinical improvement.
Endocrinopathy should be included in the differential diagnosis of altered mental status. This case additionally illustrates the challenges of managing adrenal insufficiency in the setting of hyperthyroidism and supports the use of thyroidectomy in this situation.
Rejection remains a primary cause of graft loss after heart transplant (HT). Recognizing the immunomodulation of multi-organ transplant can enhance our understanding of the mechanisms of cardiac ...rejection.
This retrospective cohort study identified patients from the UNOS database with isolated heart (H, N = 37 433), heart-kidney (HKi, N = 1516), heart-liver (HLi, N = 286), and heart-lung (HLu, N = 408) transplants from 2004 to 2019. Propensity score matching reduced baseline differences between groups. Outcomes included risk of rejection prior to transplant hospital discharge and within 1 year, and mortality within 1 year of transplant.
In the propensity score matched data, the relative risk of being treated for rejection prior to transplant hospital discharge was 61% lower for HKi (RR .39, 95% CI .29, .53) and 87% lower for HLi (RR .13, 95% CI .05, .37) compared to H. Similarly, the probability of being treated for rejection in the first year after transplant remained lower in HKi (RR .45, 95% CI .35, .57) and HLi (RR .13, 95% CI .06, .28) compared to H. The 1-year survival analysis revealed an equivalent risk of death in HKi (HR .84, 95% CI .68, 1.03) and HLi (HR 1.41, 95% CI .83, 2.41) compared to H, while HLu had a higher risk of death in the first year after transplant (HR 1.65, 95% CI 1.17, 2.33).
Recipients of HKi and HLi experience a reduced risk of rejection when compared to H, but an equivalent risk of 1 yr mortality. These findings have important implications for the future of HT medicine.
Cardiac dysfunction due to hypertension (CDHTN) in pediatrics is not well described. We aimed to describe the presentation and outcomes of pediatric CDHTN and identify clinical features associated ...with resolution of dysfunction. A single-center retrospective cohort study of patients ≤ 21 years with CDHTN from January 2005–September 2020 was performed. Patients with systolic dysfunction without another cause, blood pressure > 95th percentile, and physician judgment that dysfunction was secondary to hypertension were included. Demographics, clinical characteristics, echocardiographic findings, and outcomes were examined using Fisher’s exact and Mann–Whitney
U
tests. Multiple correspondence analysis was used to explore the relationship of resolution of dysfunction to clinical features. Thirty-four patients were analyzed at a median age of 10.9 (IQR 0.3–16.9) years. Patients were divided into groups < 1 year (
n
= 12) and ≥ 1 year (
n
= 22). Causes of hypertension were varied by age, with renovascular disease most common in infants (42%) and medical renal disease most common in older patients (77%). Echocardiography demonstrated mild LV dilation (median LV end-diastolic z-score 2.6) and mild LV hypertrophy (median LV mass z-score 2.4). Most patients (81%) had resolution of dysfunction, particularly infants (92%). One patient died and one patient was listed for heart transplant. None required mechanical circulatory support (MCS). No clinical features were statistically associated with resolution of dysfunction. Hypertension is an important but reversible cause of systolic dysfunction in children. Patients are likely to recover with low mortality and low utilization of MCS or transplantation. Further studies are needed to confirm features associated with resolution of dysfunction.
Cardiac disease results in significant morbidity and mortality in patients with muscular dystrophy (MD). Single centers have reported their ventricular assist device (VAD) experience in specific MDs ...and in limited numbers. This study sought to describe the outcomes associated with VAD therapy in an unselected population across multiple centers.
We examined outcomes of patients with MD and dilated cardiomyopathy implanted with a VAD at Advanced Cardiac Therapies Improving Outcomes Network (ACTION) centers from 9/2012 to 9/2020.
A total of 19 VADs were implanted in 18 patients across 12 sites. The majority of patients had dystrophinopathy (66%) and the median age at implant was 17.2 years (range 11.7-29.5). Eleven patients were non-ambulatory (61%) and 6 (33%) were on respiratory support pre-VAD. Five (28%) patients were implanted as a bridge to transplant, 4 of whom survived to transplant. Of 13 patients implanted as bridge to decision or destination therapy, 77% were alive at 1 year and 69% at 2 years. The overall frequencies of positive outcome (transplanted or alive on device) at 1 year and 2 years were 84% and 78%, respectively. Two patients suffered a stroke, 2 developed sepsis, 1 required tracheostomy, and 1 experienced severe right heart failure requiring right-sided VAD.
This study demonstrates the potential utility of VAD therapies in patients with muscular dystrophy. Further research is needed to further improve outcomes and better determine which patients may benefit most from VAD therapy in terms of survival and quality of life.
Abstract Background Value-based health care is a proposed driver for reimbursement under the Affordable Care Act, with value broadly defined as outcomes divided by cost. Data on value-based health ...care in pediatric heart failure are scarce. Methods and Results A retrospective analysis of the Healthcare Cost and Utilization Project Kids' Inpatient Database and Nationwide Inpatient Sample was performed for pediatric and adult cardiomyopathy and heart failure–related hospitalizations. The study included 5,689 pediatric and 473,416 adult hospitalizations. Pediatric cardiomyopathy and heart failure hospitalizations were significantly longer than adult hospitalizations (mean ± SE 16.2 ± 0.7 days vs 6.8 ± 0.1 days; P < .001). Overall mortality was greater for pediatric hospitalizations (7.7% vs 5.6%; P < .001), although it decreased over time for both pediatric and adult hospitalizations. Charges were greater for pediatric hospitalizations, both overall ($116,483 ± $5,735 vs $40,662 ± $1,419; P < .001) and for all years evaluated. Conclusions In a value-based model, pediatric cardiomyopathy and heart failure–related hospitalizations are associated with worse outcomes and greater charges than adult hospitalizations. More research is needed to understand the cost effectiveness of pediatric heart failure treatment and to reduce the burden on the health care system.
There are limited data describing the prevalence of mental health disorders (MHDOs) in patients with ventricular assist devices (VADs), or associations between MHDOs and resource use or outcomes. We ...used the Nationwide Emergency Department Sample administrative database to analyze 44,041 ED encounters for VAD-supported adults from 2010 to 2017, to assess the relationship between MHDOs and outcomes in this population. MHDO diagnoses were present for 23% of encounters, and were associated with higher charges and rates of admission, but lower mortality.
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