Abstract
AIMS: Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder, characterized by significant long-term cardiac involvement. Dilated cardiomyopathy (DCM) is the main cause of death in DMD, and angiotensin-converting enzyme inhibitors (ACEi) and beta-blockers (BB) are first-line treatments in DCM. It is unknown whether angiotensin receptor-neprilysin inhibitor (ARNi) could provide greater benefits in this setting. Our aim is to assess whether ARNi use may prevent deterioration in ejection fraction (EF) or is associated with EF improvement compared with ACEi in DMD patients with heart failure and to report the tolerability of ARNi in this group of patients. METHODS AND RESULTS: We followed 22 DMD patients, 6 of them with an EF < 40% and 16 with an EF > 40%. The first group received ARNi on top of BB, while the control group started or continued first-line therapy with ACEi ± BB. From December 2016 to December 2021, we recorded EF values at baseline and at follow-up, comparing EF changes. Median follow-up was 7 months (interquartile range 4.7-9.1). At baseline, the mean of EF (%) in the ARNi group was 31 ± 2%, while it was 59 ± 9% in the control group. At follow-up, we recorded an EF improvement in the ARNi group (38 ± 6%, P-value < 0.05). Among controls, EF at follow-up was substantially unchanged from baseline. CONCLUSIONS: Our data suggest that the use of ARNi in DMD patients with DCM and an EF < 40% might be associated with an EF improvement and a safe tolerability profile.