A case report recently published in Pediatric Transplantation describes an 18-year-old young man with Duchenne muscular dystrophy (DMD) and end-stage heart failure who successfully underwent a heart transplant.
The patient was followed for 3.5 years after the transplant and has maintained very good heart function with few complications.
The patient was originally diagnosed with DMD at 10 years old after experiencing muscle weakness beginning at eight years of age. At age 16, he was diagnosed with dilated cardiomyopathy. He then presented at an adult heart transplant center two years later with symptoms of heart failure and respiratory difficulty.
Clinical tests confirmed heart dysfunction with New York Heart Association (NYHA) Class 4 heart failure, the most severe stage of heart failure in the NYHA classification system. His left heart ventricle was extremely weak, and the right ventricle had mildly decreased function. Nevertheless, the adult transplant center declined his case due to his DMD diagnosis.
Read more about DMD prognosis
The center also refused to offer a ventricular assist device (VAD), which helps the heart pump blood, because both the patient’s right and left ventricles were weakened and would likely both need support.
After being transferred to an advanced pediatric cardiac center, the patient received a left ventricular assist device (LVAD). He required one revision surgery 28 days later and was discharged 83 days following the original surgery.
The patient received physical therapy multiple times each week. While he was initially able to walk with a cane, he transitioned to using a wheelchair by the time he left the hospital. Otherwise, he experienced no bleeding complications, infections or other adverse events from the LVAD.
“His prolonged ICU hospitalization, immobility, and sequential cardiac surgeries (including his VAD revision) may have contributed to his decline,” noted the authors.
The patient requested to be placed on a heart transplant waitlist around six months after receiving the LVAD. The transplant was performed 140 days after he joined the waitlist, and he left the hospital after 16 days.
In the 3.5 years since receiving his transplant, the patient has had no hospital readmissions and has experienced almost no complications with the new heart. His respiratory and skeletal muscle function have both declined, however, and he requires full-time care for most of his daily activities.
“Despite his functional decline, the patient self-reports an excellent quality of life and is satisfied with the decision to proceed with heart transplantation,” the authors wrote.
Sign up here to get the latest news, perspectives, and information about DMD sent directly to your inbox. Registration is free and only takes a minute.
