P3446Left-ventricular assist device as a destination therapy in Duchenne cardiomyopathy: are we ready to change the natural history?

Abstract Background Heart failure is becoming the most prominent cause of death among patients with Duchenne Muscular Dystrophy (DMD) and end-stage cardiomyopathy (CMP). Implantation of left ventricular assist devices (LVAD) as destination therapy (DT) in this group of patients is still matter of debate. No current data are available on long-term follow- up of young patients. Purpose The aim of this study was to evaluate the long-term outcome of LVAD in DMD end stage heart failure (HF) compared to a group treated optimal medical treatment (OMT) with inotropes Methods All patients affected by DMD patients presenting with end-stage HF receiving OMT or LVAD have been reviewed. All data about characteristics of HF at presentation including assessment of nutritional status and respiratory function, left ventricular ejection fraction, brain natriuretic peptide, serum sodium, in-hospital treatment and long term follow up have been collected. OMT group was constituted by 4 patients matching implantation VAD criteria but not eligible or refusing treatment. Survival was represented by Kaplan Meier analysis. Results A total of 12 DMD patients with end-stage CMP were considered for this analysis. 8 have been implanted with LVAD during the period from 2011 until mid-2017 and compared with 4 treated with OMT. Mean systolic pressure was 103 versus 91 mmHg in those not receiving LVAD (p=0.2), and left ventricular ejection fraction at the time of presentation was 16‰ for LVAD group and 22‰ for the second group (p=0.1). LVADs improved survival throughout follow-up for patients undergoing baseline inotropic infusions (P=0.0014); for the LVAD group versus the OMT group, 1-month survival was 100‰ and 67‰ respectively and 2-month survival was 100‰ versus 0‰. For LVAD group survival was 86‰ at one year, 71‰ at 2 years and 48‰ at 5 years from implantation. Analysis on short and long-term follow-up showed significant improvement of survival in DMD patients treated with LVAD (log rank<0.001), with a 5-year increase in life expectancy. Conclusions LVAD improved outcomes in patients with LVAD when compared to those without. Long-term follow-up was similar to DT LVAD adult population. Further studies are necessary to confirm these data.