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

Research output: Contribution to journalArticle

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.
Original languageEnglish
Pages (from-to)9112-9115
Number of pages4
JournalEuropean Review for Medical and Pharmacological Sciences
Volume24
DOIs
Publication statusPublished - 2020

Keywords

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

Fingerprint

Dive into the research topics of 'Duchenne muscular dystrophy: Preliminary experience with sacubitril-valsartan in patients with asymptomatic left ventricular dysfunction'. Together they form a unique fingerprint.

Cite this