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.
Part 1 - Monitoring heart disease in Duchenne and carrier moms/daughters
Part 2 - Interventions