Mechanical circulatory support in the Dutch National Paediatric Heart Transplantation Programme

Aims We aimed to determine whether in children with dilated cardiomyopathy repeated measurement of known risk factors for death or heart transplantation (HTx) during disease progression can identify children at the highest risk for adverse outcome. Methods and results Of 137 children we included in a prospective cohort, 36 (26%) reached the study endpoint (SE: all-cause death or HTx), 15 (11%) died at a median of 0.09 years [inter-quartile range (IQR) 0.03-0.7] after diagnosis, and 21 (15%) underwent HTx at a median of 2.9 years [IQR 0.8-6.1] after diagnosis. Median follow-up was 2.1 years [IQR 0.8-4.3]. Twenty-three children recovered at a median of 0.6 years [IQR 0.5-1.4] after diagnosis, and 78 children had ongoing disease at the end of the study. Children who reached the SE could be distinguished from those who did not, based on the temporal evolution of four risk factors: stunting of length growth (À0.42 vs. À0.02 length Z-score per year, P < 0.001), less decrease in N-terminal pro-B-type natriuretic peptide (NT-proBNP) (À0.26 vs. À1.06 2log pg/mL/year, P < 0.01), no decrease in left ventricular internal diastolic dimension (LVIDd; 0.24 vs. À0.60 Boston Z-score per year, P < 0.01), and increase in New York University Pediatric Heart Failure Index (NYU PHFI; 0.49 vs. À1.16 per year, P < 0.001). When we compared children who reached the SE with those with ongoing disease (leaving out the children who recovered), we found similar results, although the effects were smaller. In univariate analysis, NT-proBNP, length Z-score, LVIDd Z-score, global longitudinal strain (%), NYU PHFI, and age >6 years at presentation (all P < 0.001) were predictive of adverse outcome. In multivariate analysis, NT-proBNP appeared the only independent predictor for adverse outcome, a twofold higher NT-proBNP was associated with a 2.8 times higher risk of the SE (hazard ratio 2.78, 95% confidence interval 1.81-3.94, P < 0.001). Conclusions The evolution over time of NT-proBNP, LVIDd, length growth, and NYU PHFI identified a subgroup of children with dilated cardiomyopathy at high risk for adverse outcome. In this sample, with a limited number of endpoints, NT-proBNP was the strongest independent predictor for adverse outcome.

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“…However, the children that underwent HTx were in such a condition that they most likely would have died otherwise. 32…”

Section: Study Limitationsmentioning

confidence: 99%

Predicting outcome in children with dilated cardiomyopathy: the use of repeated measurements of risk factors for outcome

et al. 2021

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“…(Ann Thorac Surg 2017;104:1630-6) Ó 2017 by The Society of Thoracic Surgeons A lthough rare, heart failure in children carries an unusually high rate of morbidity and mortality [1]. Most cases of childhood end-stage heart failure are due to idiopathic dilated cardiomyopathy or congenital heart disease, with the latter patients being at the highest risk of poor outcomes [2]-a risk that is magnified in patients with a shunt-dependent, single-ventricle physiology [3].…”

mentioning

confidence: 99%

“…Options for safe and effective long-term mechanical circulatory support (MCS) as a bridge to heart transplant for these patients are limited [1,4]. Historically, extracorporeal membrane oxygenation (ECMO) has been the main approach for mechanical circulatory support [4,5].…”

mentioning

confidence: 99%

A Novel Surgical Approach to Mechanical Circulatory Support in Univentricular Infants

Gazit

Petrucci

Manning

et al. 2017

The Annals of Thoracic Surgery

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Our experience shows that long-term extracorporeal mechanical circulatory support of patients with underlying single-ventricle physiology after stage 1 palliation is feasible utilizing our technique. This approach overcomes several major challenges encountered in these patients, such as high flow requirement and stability of the cannulae, and allows extubation, rehabilitation, and at times, myocardial recovery.

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“…Following the introduction, we previously published our experience with the BH EXCOR and reported rates of serious adverse events comparable to the literature. 13 In this study, we evaluate our decade of experience with the BH EXCOR, through evaluation of the changes in waiting list mortality following its introduction in the Netherlands, as well as the occurrence of adverse events in the children supported by a VAD.…”

Section: Introductionmentioning

confidence: 99%

Pediatric Ventricular Assist Device Support in the Netherlands

Rohde

Antonides

Muslem

et al. 2020

World J Pediatr Congenit Heart Surg

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Background: This study aimed to evaluate the changes in heart transplantation (HTx) waiting list mortality following the introduction of the Berlin Heart EXCOR (BH EXCOR) in the Netherlands, as well as the occurrence of adverse events in these children. Methods: A retrospective, single-center study was conducted including all pediatric patients (18 years) awaiting HTx. Patients were grouped in two eras based on availability of the BH EXCOR in our center, era I (1998-2006; not available) and era II (2007( to July 31, 2018 available). Results: In total, 87 patients were included, 15 in era I and 72 in era II. Extracorporeal membrane oxygenator support was required in 1 (7%) patient in era I and in 13 (18%) patients in era II. Overall mortality (7/15 in era I vs 16/72 in era II; 47% vs 22%, P ¼ .06) and transplantation rates (8/15 in era I vs 47/72 in era II; 53% vs 65%, P ¼ .39) did not differ significantly. Eleven (39%) patients of the pediatric ventricular assist device (VAD) population died, with the predominant cause being cerebrovascular accidents (CVAs) in eight (29%) patients. Furthermore, 14 (50%) of the pediatric VAD patients survived to transplantation. Adverse events most frequently occurring in VAD patients included CVA in 14 (50%), mostly (68%) within 30 days after VAD implantation, and bleeding requiring rethoracotomy in 14 (50%), all within 30 days after VAD implantation. Conclusions: The introduction of the BH EXCOR has positively impacted the survival of pediatric patients with end-stage heart failure in our center. The predominant cause of death changed from end-stage heart failure in era I to CVA in era II. We emphasize the need for large prospective registry-based studies.

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Mechanical circulatory support in the Dutch National Paediatric Heart Transplantation Programme

Cited by 8 publications

References 23 publications

Predicting outcome in children with dilated cardiomyopathy: the use of repeated measurements of risk factors for outcome

Predicting outcome in children with dilated cardiomyopathy: the use of repeated measurements of risk factors for outcome

A Novel Surgical Approach to Mechanical Circulatory Support in Univentricular Infants

Pediatric Ventricular Assist Device Support in the Netherlands

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