Prospective Trial of a Pediatric Ventricular Assist Device

Texas Children's Hospital and Baylor College of Medicine, Houston, Texas 77030, USA. .
New England Journal of Medicine (Impact Factor: 55.87). 08/2012; 367(6):532-41. DOI: 10.1056/NEJMoa1014164
Source: PubMed


Options for mechanical circulatory support as a bridge to heart transplantation in children with severe heart failure are limited.
We conducted a prospective, single-group trial of a ventricular assist device designed specifically for children as a bridge to heart transplantation. Patients 16 years of age or younger were divided into two cohorts according to body-surface area (cohort 1, <0.7 m(2); cohort 2, 0.7 to <1.5 m(2)), with 24 patients in each group. Survival in the two cohorts receiving mechanical support (with data censored at the time of transplantation or weaning from the device owing to recovery) was compared with survival in two propensity-score-matched historical control groups (one for each cohort) undergoing extracorporeal membrane oxygenation (ECMO).
For participants in cohort 1, the median survival time had not been reached at 174 days, whereas in the matched ECMO group, the median survival was 13 days (P<0.001 by the log-rank test). For participants in cohort 2 and the matched ECMO group, the median survival was 144 days and 10 days, respectively (P<0.001 by the log-rank test). Serious adverse events in cohort 1 and cohort 2 included major bleeding (in 42% and 50% of patients, respectively), infection (in 63% and 50%), and stroke (in 29% and 29%).
Our trial showed that survival rates were significantly higher with the ventricular assist device than with ECMO. Serious adverse events, including infection, stroke, and bleeding, occurred in a majority of study participants. (Funded by Berlin Heart and the Food and Drug Administration Office of Orphan Product Development; number, NCT00583661.).

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Available from: Patricia Massicotte, Jan 15, 2014
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    • "Little is known with regards to the long-term outcomes of pediatric MCS patients. In the first multi-institutional prospective trial of a pediatric VAD (Berlin Heart EXCOR) reported by Fraser et al. [13]., 92% had a favorable outcome (transplant, recovery or alive on device) at around 6 months for both cohorts (<0.7 m2 and 0.7 to 1.5 m2). This was significantly better than matched ECMO groups. "
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    ABSTRACT: Mechanical circulatory support (MCS) in the pediatric heart failure population has a limited history especially for infants, and neonates. It has been increasingly recognized that there is a rapidly expanding population of children diagnosed and living with heart failure. This expanding population has resulted in increasing numbers of children with medically resistant end-stage heart failure. The traditional therapy for these children has been heart transplantation. However, children with heart failure unlike adults do not have symptoms until they present with end-stage heart failure and therefore, cannot safely wait for transplantation. Many of these children were bridged to heart transplantation utilizing extracorporeal membranous oxygenation as a bridge to transplant which has yielded poor results. As such, industry, clinicians, and the government have refocused interest in developing increasing numbers of MCS options for children living with heart failure as a bridge to transplantation and as a chronic therapy. In this review, we discuss MCS options for short and long-term support that are currently available for infants and children with end-stage heart failure.
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    • "For adolescents and young adults, larger devices are available that can effectively bridge patients to transplant [Jefferies and Morales, 2012]. Recent advances in technology have led to the development of MCS strategies for infants and children in the form of the Berlin EXCOR device which has received FDA approval for widespread use [Fraser et al., 2012]. This device can be used for an extended period of time even in the setting of neutropenia [Dedieu et al., 2013]. "
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    ABSTRACT: Barth syndrome (BTHS) is an X-linked recessive disorder that is typically characterized by cardiomyopathy (CMP), skeletal myopathy, growth retardation, neutropenia, and increased urinary levels of 3-methylglutaconic acid (3-MGCA). There may be a wide variability of phenotypes amongst BTHS patients with some exhibiting some or all of these findings. BTHS was first described as a disease of the mitochondria resulting in neutropenia as well as skeletal and cardiac myopathies. Over the past few years, a greater understanding of BTHS has developed related to the underlying genetic mechanisms responsible for the disease. Mutations in the TAZ gene on chromosome Xq28, also known as G4.5, are responsible for the BTHS phenotype resulting in a loss-of-function in the protein product tafazzin. Clinical management of BTHS has also seen improvement. Patients with neutropenia are susceptible to life-threatening bacterial infections with sepsis being a significant concern for possible morbidity and mortality. Increasingly, BTHS patients are suffering from heart failure secondary to their CMP. Left ventricular noncompaction (LVNC) and dilated CMP are the most common cardiac phenotypes reported and can lead to symptoms of heart failure as well as ventricular arrhythmias. Expanded treatment options for end-stage myocardial dysfunction now offer an opportunity to change the natural history for these patients. Herein, we will provide a current review of the genetic and molecular basis of BTHS, the clinical features and management of BTHS, and potential future directions for therapeutic strategies. © 2013 Wiley Periodicals, Inc.
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