bims-hylehe 2020-05-31 papers

Issue of 2020–05–31
four papers selected by
Richard James, University of Pennsylvania

J Pediatr. 2020 Jun;pii: S0022-3476(20)30254-7. [Epub ahead of print]221 201-206.e1

Transition Readiness in Teens and Young Adults with Congenital Heart Disease: Can We Make a Difference?

Karen Uzark, Sunkyung Yu, Ray Lowery, Katherine Afton, Anji T Yetman, Jonathan Cramer, Nancy Rudd, Scott Cohen, Russell Gongwer, Michelle Gurvitz.

OBJECTIVES: To examine changes in transition readiness (knowledge, self-efficacy, self-management) over time and explore factors associated with transition readiness, including psychosocial quality of life (QOL) and health service utilization in teens/young adults with congenital heart disease.
STUDY DESIGN: In a multicenter prospective cohort study, 356 patients, age 14-27 years, completed transition readiness and QOL assessments at routine cardiology visits at baseline and 1-year follow-up.
RESULTS: Median patient age was 19.8 years at 1.03 years (IQR 0.98-1.24) following baseline transition readiness assessment. Average knowledge deficit scores decreased at follow-up (P < .0001) and self-efficacy scores increased (P < .0001). Self-management scores increased (P < .0001), but remained low (mean 57.7, 100-point scale). Information was requested by 73% of patients at baseline and was associated with greater increase in knowledge at follow-up (P = .005). Increased knowledge (P = .003) and perceived self-efficacy (P = .01) were associated with improved psychosocial QOL, but not health service utilization at follow-up. Patients who preferred face-to-face information from healthcare providers (47%) vs other information sources were more likely to request information (P < .0001). In patients <18 years old, greater agreement between teen and parental perception of teen's knowledge was associated with greater increase in patient knowledge (P = .02) and self-efficacy (P = .003).
CONCLUSION: Transition readiness assessment demonstrated improved knowledge, self-efficacy, and self-management at 1-year follow-up in teens/young adults with congenital heart disease. Improved knowledge and self-efficacy were associated with improved psychosocial QOL. Self-management remained low. Supplemental media for conveying information and greater involvement of parents may be needed to optimize transition readiness.

Keywords: adult congenital heart disease; healthcare transition; patient education; quality of life; self-management

DOI: https://doi.org/10.1016/j.jpeds.2020.02.040

J Heart Lung Transplant. 2020 Apr;pii: S1053-2498(20)30840-8. [Epub ahead of print]39(4S): S206-S207

Brighter Future for Children with Congenital Heart Disease Requiring Heart Transplantation? A UNOS Registry Analysis over the Last Three Decades.

M L Townsend, T Karamlou, G J Boyle, B Feingold, K P Daly, S Deshpande, S Auerbach, S Worley, W Liu, E Saarel, S M Amdani.

PURPOSE: Patients with congenital heart disease (CHD) account for ∼40% of pediatric heart transplants (HT) performed in the United States. Our aim was to evaluate the overall trends for waitlist and post-transplant survival for children with CHD in the last three decades and identify risk factors for waitlist and post-transplant mortality in the current era.
METHODS: Children (age <18, n= 5741) listed for HT in the UNOS database from 1990-2017 were included. Patients were divided by time at initial listing into Era 1 (1/1/90-3/12/99), Era 2 (3/13/99-12/31/08) and Era 3 (1/1/09-12/31/17). Demographic and clinical characteristics at listing, including ventricular assist device (VAD) use, were compared across the 3 eras. Kaplan-Meier curves were generated to compare survival in the waitlist and post-transplant periods. We identified risk factors for increased waitlist and post-transplant mortality by bootstrap selection method.
RESULTS: Compared to Era 1, CHD candidates in Eras 2 and 3 were older (mean age 3.5±5.4 vs 4.0±5.5 vs 4.2±5.5 years), more often of non-White race (26.7 vs 39.7 vs 41.6%), more likely on intravenous inotropes (31.1 vs 45.7 vs 46.6%) and on VAD support (1.1 vs 2.3 vs 3.2%). Compared to Era 2, those in Era 3 were less likely to have renal dysfunction (71.5 vs 54.0%) and pulmonary hypertension (27.8 vs 17.1%) or be on ECMO (14.8 vs 8.1%) or ventilator (29.8 vs 23.0%) pre-transplant. Compared to earlier Eras, children with CHD listed for HT in Era 3 had significantly improved 1-year waitlist (59.6 vs 58.4 vs 67.5%) and post-transplant survival (75.3 vs 80.6 vs 88.0%) (Figure) [p<0.05 for all]. Multivariable risk factors for waitlist and post-transplant mortality in Era 3 are shown in Table.
CONCLUSION: Children with CHD listed for HT in the U.S. have had significant improvement in waitlist and post-transplant survival particularly in the last decade. Improved candidate selection and post-transplant care may have led to these advances.

DOI: https://doi.org/10.1016/j.healun.2020.01.823

J Heart Lung Transplant. 2020 Apr;pii: S1053-2498(20)30877-9. [Epub ahead of print]39(4S): S220-S221

US Trends in Pediatric VAD Utilization - Where are We Now?

H Ahmed, J Dykes, A Power, E Profita, C Chen, J Murray, C S Almond.

PURPOSE: Pediatric ventricular assist device (VAD) therapy is a rapidly changing field where devices and applications are rapidly evolving. We sought to describe some of the most recent trends in pediatric VAD use and contemporary outcomes since UNOS began collecting VAD data on all listed patients regardless of whether they survived.
METHODS: All children ≤21 years of age listed for isolated HT between Jan 2004 and 2019 and underwent VAD placement were identified using OPTN data. Descriptive statistics were used to characterize recent trends in VAD utilization. Waitlist survival in children with cardiomyopathy, CHD and single-ventricle heart disease were estimated using the Kaplan-Meier method.
RESULTS: Overall, 2059 children met the study inclusion criteria of whom the median age was 11 (IQR 1, 17), median weight 38 (11,66) kg, 26% were black; 18.3 had congenital heart disease (CHD) including 5.1% with single-ventricle (SV) HD. Forty-nine percent were supported with a VAD at listing, 72% at transplant and 13% received a VAD but was not supported at listing or transplant. Overall, 64% received LVADs, 31% BIVADs, 2.5% RVADs 1.7% total artificial hearts (TAH). Implants of the Berlin Heart fell dipped sharply to 34 in 2015 after a peak of 63 but have rebounded since in the era of bivalirudin. As of 2018, the Heartware HVAD was the most commonly implanted VAD in children (N=61) followed by the Berlin Heart (N=48), CentriMag/PediMag (N=18) and Heartmate III (N=11). The fastest growing segment of VAD patients are single-ventricle patients (Figure).
CONCLUSION: The total number of pediatric VADs continues to grow at a brisk pace. The most recent era has seen growth of the Heartmate III and resurgence of the Berlin Heart EXCOR, after a brief decline in 2015. Single-ventricle and Heartmate III patients represent the fastest growing segment of the VAD population.

DOI: https://doi.org/10.1016/j.healun.2020.01.860

J Heart Lung Transplant. 2020 Apr;pii: S1053-2498(20)30297-7. [Epub ahead of print]39(4S): S452

Qualitative Exploration of the Pediatric Heart Failure Experience for Development of a Patient-Reported Outcome Measure.

C Chen, J L Ridgeway, F D Bocell, M L Tanenbaum, K K Hood, E Behnken, J Schmidt, S J Hanes, A Saha, B Caldwell, M Tarver, V Peiris, C S Almond, J N Johnson.

PURPOSE: No valid, reliable, and publicly available patient-reported outcome measure (PROM) exists to assess the pediatric heart failure (HF) experience. We sought to understand this experience in order to develop a PROM that can reliably capture and track outcomes for adolescent patients.
METHODS: Semi-structured individual or group interviews were conducted at two pediatric HF and transplant centers in the US. Patient ages 12-21 who had a record of symptomatic HF in the past 2 years were eligible to participate. Caregivers of participants were also eligible. Symptom experiences and their impact on daily activities were explored. Transcripts were analyzed by members of a multidisciplinary team using thematic analysis.
RESULTS: 16 patients and 9 caregivers participated. 22 completed individual interviews in-person or by phone, and 3 participated in a web-based focus group. Patient participants were 31% male, and median age was 16.5 years. 31% (n=5) had HF from cardiomyopathy and 69% (n=11) had HF due to congenital heart disease, with 7 having single ventricle physiology. 5 participants were interviewed post-transplant. Preliminary analysis revealed consistent themes across interviews. Common symptoms included fatigue, shortness of breath, and chest discomfort. Symptoms limited ability to perform daily tasks (e.g. climbing stairs at school), participate in certain extracurricular activities (e.g. sports, dance) and keep up with peers. Participants described social and emotional impacts from being treated differently by others due to surgical scars, medical devices, or functional limitations. They also reported burden of frequent medical visits and treatments, and expressed worry and uncertainty about the need for future medical interventions. Caregivers reported similar impacts on patient function and wellbeing, and they further described social exclusion by peers and anxiety about the future (e.g. limited life expectancy or career limitations).
CONCLUSION: The unique personal and social impacts of HF symptoms on adolescents and young adults may necessitate development of unique items for PRO measurement in this population. These findings will be used to draft a developmentally-relevant PROM that will subsequently be tested in this population and validated for use in clinical care and regulatory evaluation of new medical products.

DOI: https://doi.org/10.1016/j.healun.2020.01.280