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LVAD as Destination Therapy for Patients with Duchenne Muscular Dystrophy

Antonio Amodeo, MD
Bambino Gesù Pediatric Hospital
Rome, Italy

Typically, patients with Duchenne muscular dystrophy (DMD) are not considered candidates for heart transplantation because of the global nature of their skeletal muscle disease with high likelihood of respiratory failure. An alternative treatment for end-stage heart failure in dystrophinopathies we have explored at the Bambino Gesù Children's Hospital in Rome is the use of left ventricular assist devices (LVAD) as destination therapy (DT). To date we have treated seven DMD patients with a Jarvik 2000 LVAD as destination therapy (DT). The main advantage of this device in DMD is related to the positioning of the power cable exit site from the retroauricular area. For wheelchair-dependent patients we believe this provides a lower risk of infection compared with an abdominal driveline exit site.

Accurate and appropriate selection of DMD patients suitable for LVAD is important. This group of patients presents significant challenge with every single step characterized by a fine balance between the risks and benefits, and different variables play an important role. This class of patients requires a series of clinical and surgical precautions and maneuvers that are possible only in centers with a high level of experience with DMD patients, especially in the postoperative phase. Our experience shows that postoperative care can be extremely challenging and is often burdened by unexpected complications. The presence of comorbidities such as severe kyphoscoliosis and respiratory muscle weakness may increase intraoperative risks as well as the risk of postoperative complications. We strongly suggest the use of early postoperative non-invasive ventilation because it allows for the reduction of postoperative respiratory insufficiency. Furthermore, particular care for even routine maneuvers like chest tube placement is warranted due to the potential risk of abdominal organ damage with chest wall deformity and abnormally elevated diaphragms. Finally, postoperative anticoagulation management can be challenging in patients with DMD.

Despite these features we have observed satisfactory outcomes for our patients with DMD who received DT-LVAD therapy. All patients survived to hospital discharge and resumed normal activities. At median follow-up time of 25 months (range 11-50 months) we had 3 late deaths. One patient died after 45 months for sepsis due to Staphylococcus aureus lung infection, one patient with tracheostomy died after 28 months in a peripheral hospital for tracheal bleeding due to an inappropriate otorhinolaryngology maneuver, and one patient died after 15 months after cerebral hemorrhage. Our longest surviving patient is over 4 years from LVAD placement.

In summary, the prolonged life expectancy of DMD patients up to the third or fourth decade of life incurs the problem of DCM being the main cause of death. Preoperative patient selection and an accurate surgical strategy with multidisciplinary postoperative management are mandatory to ensure good early and midterm results. According to the basic philosophy of palliative care, which is to achieve the best quality of life for patients even when their illness cannot be cured, the use of VAD as DT may be a palliative, time-limited therapy for the treatment of patients with no other therapeutic options. ■

Disclosure Statement: The author has no conflicts of interest to disclose.

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