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Mayo Clinic, 200 First Street SW, Rochester, MN 55905
(Email: oliver.william{at}mayo.edu).
With the demand for pediatric donor hearts continuing to exceed a decreasing supply, the bridge to transplantation plays an ever important role in the survival of pediatric patients with intractable myocardial failure. Since the first bridge to transplant occurred in 1989 in a 9-year-old that underwent successful orthotopic heart transplantation after 12 hours of support on a Biomedicus centrifugal pump (Medtronic, Minneapolis, MN), extracorporeal membrane oxygenation or ventricular assist device (VAD) with centrifugal flow were the mainstay of circulatory support to reach transplantation. However, bridge to transplantation with extracorporeal membrane oxygenation is limited to approximately 2 weeks to attain acceptable survival and hospital discharge. It is not unusual to wait 2 to 3 months listed as 1a transplant candidate before orthotopic heart transplantation. Since the advent of the pneumatic pulsatile VAD in pediatric patients, utilization as a bridge to transplant has risen over the last decade. The increase is primarily based on the development of devices with stroke volumes of 10 mL and 20 to 30 mL to accommodate patients below 9 kg and 10 to 25 kg, respectively, and extended duration of circulatory support including a maximum of 420 days.
In this issue, Mahle and colleagues [1] become the first to do an economic analysis of this emerging therapy for refractory heart failure in pediatric patients. Their cost effectiveness analysis is a descriptive model-based economic study that included 145 children who underwent VAD implantation at 19 centers in North America during 2002 to 2007. The authors have admirably characterized the complex components of this type of cost analysis that included definitions, assumptions, corrections, and database that greatly influence the validity of such analysis. The weaknesses of the paper centered around the relatively simplistic design compared with more sophisticated analytic modeling that could have been applied. Inpatient costs associated with physician services were not in their administrative hospital database, so the authors explain that they accounted for these physician fees using CPT4-based reimbursements, but how the utilization of physician services was actually tracked was not apparent.
The risks and benefits of VAD have been well described in terms of complications, longevity, age, and types of physiologic support compared with extracorporeal membrane oxygenation in adults and to some extent in the pediatric population. The importance of this work is not only the first attempt to evaluate the economic impact of this emerging therapy, but provides a solid point of comparison for future studies and resource allocation. This is precisely shown in the sizeable mean hospital costs of $624,798 that were converted to cost per quality-adjusted life years (QALYs) used to compare treatments in terms of the financial impact. This study found VAD implementation as a bridge to transplantation in pediatric patients costs $119,937/QALY saved. Although extracorporeal membrane oxygenation was not directly compared in this study to VAD implementation, it has reported QALYs in a variety of settings well below 100,000/QALY considered by many as the benchmark for technological health expenditures. The broad aspect of this database derived from centers across North America strengthens the applicability of this study to those in the field.
Limited healthcare resources are going to require economic analysis to better evaluate therapeutic advancements and overall societal needs. With the expansion of VAD in treatment of adults with intractable myocardial failure, serial economic studies have shown reduced costs in the past decade for many reasons. Similarly, this article will be a standard to compare the future role of VAD in refractory myocardial failure in pediatric patients.
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