Long-Term Outcome of LVAD in Duchenne Population with End Stage Cardiomyopathy

Abstract

Purpose The aim of this study was to report the effect of long-term use of VAD in patients affected by Duchenne Muscular Distrophy and end stage cardiomyopathy (DMD-DCM). Methods We collected data of end stage DMD-DCM patients who were implanted with LVAD in our center (group 1) and the ones in regular follow-up because not eligible for LVAD implantation (group 2). Each patient of group 1 underwent extensive pre-operative assessment, in-hospital treatment and was enrolled in a dedicated DMD heart failure (HF) clinic during the post-discharge period. Patients of group 2 were followed in a dedicated DMD HF clinic or as inpatient in case of need of inotropic support. All data during long-term follow-up were recorded including echocardiography, ECG examination, laboratory blood tests, BNP dosing and assessment of nutritional status and respiratory function. Survival of the groups was represented by Kaplan Meier analysis and compared by log rank. Results A total of eight DMD-DCM patients (group 1) were implanted with LVAD and treated at our center during the period from 2011 until 2018 (mean age at implantation was 16.9 ± 2.9 years)¸whilst 4 patients (group 2) were not eligible for LVAD implantation. At the time of the last follow-up, 4 out of 8 patients were alive and 2 out of 8 patients did not have post-operative complications. The most prominent complications were bleeding and infections with one episode of acute heart failure that was successfully treated. The principal causes of 3 deaths at the median follow-up of 22 months were sepsis, tracheal bleeding, and cerebral hemorrhage. After one year from implantation we noticed a significant decrease in heart rate (p=0.002), in LV volumes and diameters (LVEDD p=0.03, LVESD p=0.02, EDV p=0.01 and ESV p=0.02) together with a significant increase in ejection fraction (p=0.0036). However, relative wall thickness did not change over time, showing an eccentric remodeling pattern before and after LVADs. Analysis on short and long-term follow-up showed significant improvement of survival in DMD patients treated with LVAD compared to the group that was not implanted (log rank Conclusion Our data showed that cardiac atrophy is persistent in Duchenne cardiomyopathy despite the improvement of LV function. LVAD improved outcomes in patients with LVAD when compared to those without.