CONGRESS ABSTRACT

INTRODUCTION Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder associated with early and progressive degeneration of skeletal, smooth, and cardiac muscles, with progressive loss of ambulation, respiratory failure, and dilated cardiomyopathy. To date, there is limited data in the literature regarding the impact of different classes of cardioactive drugs on left ventricular dysfunction associated with DMD. Specifically, the only drugs shown to have a prognostic impact are corticosteroids and ACE inhibitors, whose use is recommended from the age of 10. However, most DMD patients experience a progressive decline in left ventricular function starting in the second decade of life. The scarcity of data in the literature, combined with the complexity of therapeutic management, often prevents the implementation of optimal heart failure therapy. AIMS To evaluate whether treatment with SGLT inhibitors (dapagliflozin) is well-tolerated and effective in patients with DMD and asymptomatic left ventricular systolic dysfunction. MATERIALS AND METHODS From 2022 to 2024, we enrolled 23 non-ambulatory patients with DMD (mean age 25±4.28) and a left ventricular ejection fraction ranging from 22% to 40% on echocardiography, without symptoms of heart failure. All patients were already on optimal medical therapy with Sacubitril/Valsartan or ACEi/ARB, beta-blockers, and anti-aldosterone agents at the maximum tolerated dose, according to the 2021 ESC heart failure guidelines. Therapy with dapagliflozin was introduced at the standard dose of 10 mg/day, with baseline renal function assessed using cystatin C. Clinical and echocardiographic follow-up was performed at 6 and 12 months. RESULTS At baseline assessment, the mean left ventricular ejection fraction (LVEF) was 31%. A significant improvement in LVEF was observed after 6 months (35%; p<0.005) and at 12 months (38%; p<0.005). A significant reduction in heart failure biomarker levels (NT-proBNP) was observed at 6 months (mean baseline value 703 pg/mL vs 374 pg/mL at 6 months, p=0.004); however, this reduction was not maintained at 12 months. Renal function, assessed using cystatin C, remained stable during follow-up. In one patient, the drug was discontinued due to poor tolerance. CONCLUSIONS Our data suggest that, in patients with DMD and  reduced ejection fraction, therapy with dapagliflozin may improve left ventricular function, with a satisfactory safety profile.