Left ventricular assist device support as destination therapy in pediatric patients with end-stage heart failure

Highlights

• Destination therapy is used in lieu of heart transplantation in certain children.

• Long-term outcomes of destination therapy are still unknown.

• The future will not include application of this technology to smaller and younger patients.

Abstract

The development of ventricular assist devices to sustain the circulation represents one of the great achievements in the treatment of heart failure. Though early (1st generation) pulsatile devices required that patients remain hospitalized while on support, newer 2nd and 3rd generation continuous-flow (CF) devices have allowed for hospital discharge, expanding their use beyond bridge to transplantation to include permanent support. This indication, referred to as “destination therapy” is emerging as a viable alternative to heart transplantation in adults, and more recently, children. Though no formal indications exist for destination therapy in children, it may be considered in lieu of transplantation in patients with non-cardiac life-limiting comorbidities, severe pulmonary hypertension, obesity (BMI > 35 kg/m²), recurrent malignancy, or if it is their preference. One group for whom DT may be most appropriate is those with advanced dystrophinopathies for whom post-transplant outcomes remain poor. Outpatient management of the pediatric destination therapy patient requires close monitoring by a multidisciplinary team of physicians, nurses, pharmacists, social workers, nutritionists, and psychologists. Because destination therapy in children remains quite rare, little is known about long-term outcomes in this population, though studies of children supported as outpatients as a bridge to transplantation suggest that survival is excellent and functional status improves post-implantation. As ventricular assist devices continue to develop, the future will not only include application of this technology to a more diverse group of patients, but the additional challenge of supporting patients for increasingly longer durations, managing late complications, and facilitating improvements in quality of life.

Introduction

The pursuit of the artificial heart dates back to at least 1937, when Vladimir Demikhov first used a machine to support the circulation in a dog for over 5 h [1]. In 1952, approximately 15 years after the work of Demikhov, Dr. Forest Dodrill is credited with the first successful mechanical circulatory support in a human [2]. Predating Dr. Christian Barnard's historic first heart transplant in 1967, the original intended use of these devices were for either permanent replacement for the failing heart or temporary surgical support. Since then, with the advent of heart transplant outcomes outpacing those of ventricular assist devices (VADs), there has been a conceptual shift in the utility of VADs to be used primarily as a bridge to transplantation (BTT), rather than as a definitive therapy. In recent years, however, with dramatic advancements in both the longevity and quality of life of VAD patients, we are once again returning to first principles and using mechanical support for long-term purposes. This indication, referred to as “destination therapy” (DT) is emerging as a viable alternative to heart transplantation in adults, and more recently, children. This manuscript describes the contemporary approach to DT in children with advanced heart failure, with specific emphasis on candidate selection, outpatient management, adverse events, and patient outcomes. Perspectives on future directions are also discussed.