Is heart transplantation a real option in patients with Duchenne syndrome? Inferences from a case report

• DOI: 10.1002/ehf2.12905
• Publication Date: 2020-08-01T10:39:35Z
• Authors: Antonio Piperata; Tomaso Bottio; Giuseppe Toscano; Martina Avesani; Andrea Vianello; Gino Gerosa
• Keywords: Destination therapy; Dilated Cardiomyopathy; Vital capacity

Abstract Duchenne muscular dystrophy (DMD) is the most frequent and severe form of MD. It firstly affects skeletal muscles, causing disability, subsequently myocardium. The only two options to treat end‐stage heart failure in these patients are either a left ventricular assist device (LVAD) implantation as destination therapy or transplant. These hypotheses still controversial, data very limited. We describe case an 18‐year‐old male patient, affected by DMD wheelchair from age 11. He progressively developed dilated cardiomyopathy, 2016, at 14 years, he underwent HeartWare LVAD implantation, therapy, without post‐operative complications. has been followed up for 47 consecutive months; 30 months after infection exit site, treated antibiotics surgical toilette. Following this event, on basis patient's good general conditions willingness, we started consider transplant option. Before patient was listed, accurate workup, found higher values forced vital capacity, expiratory volume 1 s, peak flow, compared with predicted same‐age patients. have neither scoliosis nor need non‐invasive mechanical ventilation, finally, always steroids stable thoraco‐abdominal function over years. According considerations, listed In 12 February 2020, 18 no Cardiac transplantation not considered valid option patients, because shortage donor availability systemic nature disease. Considering that had already experienced LVAD‐related complication better condition than his peers, him described who successful assistance. Three months' follow‐up uneventful.