Abstract / Summary
Background: Neuromuscular diseases (NMDs) are frequently complicated by progressive cardiomyopathy leading to advanced heart failure. Ventricular assist device (VAD) implantation has emerged as a potential option in selected pediatric and young adult patients, but evidence on indications and outcomes is limited. We aimed to systematically review evidence on VAD implantation in children and young adults with NMDs, focusing on device strategies and clinical outcomes. Methods: Systematic review following Cochrane methodology and reported per the PRISMA guidelines. Six major biomedical databases were searched from inception to October 1, 2025. Studies including patients aged ≤25 years with NMDs undergoing VAD implantation were eligible. The primary outcome was clinical status at last reported follow-up, classified as alive on device support, alive after heart transplantation (HT), or deceased. Results: Six retrospective studies comprising 37 patients were included. Duchenne muscular dystrophy was the most common diagnosis (48.6%). Destination therapy was the most frequently reported strategy (45.9%), followed by bridge-to-transplant (37.8%) and bridge-to-decision (16.2%). At last follow-up, 75.7% were alive: 43.2% on device support and 32.4% after undergoing HT; 24.3% had died. Complications were common, most frequently bleeding and infection, whereas neurologic events were the leading cause of death. Reporting of functional outcomes and quality of life was sparse and heterogeneous. Conclusions: Evidence on VAD use in this population is limited to small retrospective cohorts. Although most patients were alive at last reported follow-up, morbidity remains substantial and patient-centered outcomes are poorly characterized. Prospective multicenter studies with standardized outcome definitions are needed.