Duchenne muscular dystrophy: Preliminary experience with sacubitril-valsartan in patients with asymptomatic left ventricular dysfunction

Risultato della ricerca: Contributo in rivistaArticolo in rivista

Abstract

OBJECTIVE: Duchenne muscular dystrophy (DMD) is an inherited X-linked recessive neuromuscular disease caused by mutations of the dystrophin gene, leading to early and progressive muscle deterioration and dilated cardiomyopathy. The aim of this investigation was to assess whether treatment with sacubitril/ valsartan (S/V) is well tolerated and may have beneficial effects in DMD patients with left ventricle (LV) dysfunction. PATIENTS AND METHODS: We administered S/V to 3 DMD patients (19-29 yeard old) with LV ejection fraction <35% at echocardiography but no symptoms of heart failure. All patients were on optimal medical therapy. S/V was initiated at a very low dose of 12/13 mg/die, after withdrawal of angiotensin-converting enzyme inhibitor therapy, and slowly titrated to the dose of 49/51 mg twice daily or the maximally tolerated dose. Clinical and echocardiographic follow-up was performed after 3, 6 and 12 months. RESULTS: At baseline, the LV ejection fraction was 32±1%. A significant improvement of LV ejection fraction was observed at 3 months (44.0±6.0%; p<0.05), which was maintained at 6 (45.7±5.0%) and 12 (43.3±3.2%) months (p<0.05 for both). No relevant side effects were reported throughout the period of the study. CONCLUSIONS: Our preliminary data suggest that, in DMD patients with reduced LV ejection fraction, S/V is safe and may improve LV function.
Lingua originaleEnglish
pagine (da-a)9112-9115
Numero di pagine4
RivistaEuropean Review for Medical and Pharmacological Sciences
Volume24
DOI
Stato di pubblicazionePubblicato - 2020

Keywords

  • Duchenne muscular
  • Dystrophy
  • Left ventricular dysfunction
  • Sacubitril/valsartan

Fingerprint Entra nei temi di ricerca di 'Duchenne muscular dystrophy: Preliminary experience with sacubitril-valsartan in patients with asymptomatic left ventricular dysfunction'. Insieme formano una fingerprint unica.

Cita questo