End stage dilated cardiomyopathy (DCM) is a known complication of Duchenne muscular dystrophy. DCM leads to end stage heart failure and the premature death of patients with Duchenne. Given recent advances in respiratory care, heart failure is the cause of death in more than 40% of patients with Duchenne.

A “ventricular assist device (VAD)” is a mechanical pump that is connected to the heart in order to support its function. The type of device that would be considered in individuals with Duchenne/Becker is a “left ventricular assist device (LVAD)”. It works by assisting the ill heart in pumping blood from the left ventricle to the body. Generally VAD therapy is used as a “bridge to transplant” (after the heart is too weak to support the body and until a donor heart for transplant is found). In some patients, a VAD could be used as “destination therapy.” What this means is that the device is implanted in an individual who is not a transplant candidate with the intent that the device will be in place for the remainder of their life.

Due to progressive skeletal myopathy and limited functional capacity, cardiac transplantation has been significantly limited for patients with Duchenne. Recent advances utilizing LVAD’s as “destination therapy,” rather than as a bridge to heart transplant, are being explored as a new therapeutic option for this patient population

LVAD’s have now been placed in three patients worldwide. The first LVAD recipients were two adolescent boys in Italy and, more recently a 29-year-old man at Cincinnati Children's Hosptial Medical Center in the US. While each of these young men is reportedly doing well, the surgical procedure and life post-operatively is not without significant risk of complications.

On January 24 from 1:00-2:00, PPMD will be hosting the third in a series of four cardiac webinars. This webinar will be dedicated to the subject of LVAD use in patients with Duchenne. We are pleased to be joined by a panel of four outstanding surgeons who are all experts in the field of cardiothoracic surgery. The four members of the panel include:

• Dr. Pirooz Eghtesady - cardiothoracic surgery, St. Louis Children’s Hospital
• David Morales - Chief of Cardiovascular surgery, executive co-director of the Heart Institute, Cincinnati Children's Hosptial Medical Center
• Dr. Robert “Jake” Jaquiss  - Chief of Pediatric Cardiothoracic Surgery, Duke University
• Dr. Patrick McConnell  - Cardiothoracic Surgery, Nationwide Children’s Hospital

The topics they will cover will include:

• What is a VAD/LVAD?
• When might a VAD be considered?
• Who might be considered as a candidate?
• Pros and cons of VAD implantation
• Post-VAD life; lessons learned from the first 3 recipients

The webinar will be 50 minutes long with 10 minutes for questions. We welcome your participation. It should be a very interesting and informative hour.

Kathi Kinnett, Director of Clinical Care
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