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Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy

Doris G Leung1, Daniel A Herzka, W Reid Thompson

  • 1Hugo W. Moser Research Institute, Kennedy Krieger Institute, Baltimore, MD; Departments of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD.

Annals of Neurology
|July 22, 2014
PubMed

Insights

Sildenafil did not improve cardiac function in adults with Duchenne and Becker muscular dystrophies (DBMD). The study found no significant benefits, suggesting sildenafil is unlikely to be an effective treatment for DBMD cardiomyopathy.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Duchenne and Becker muscular dystrophies (DBMD) are genetic disorders caused by dystrophin mutations.
  • Adults with DBMD are at risk for life-threatening cardiomyopathy.
  • Phosphodiesterase 5 (PDE5) inhibitors have shown promise in improving cardiac function in DBMD mouse models.

Purpose of the Study:

  • To evaluate the efficacy of the PDE5 inhibitor sildenafil in treating cardiomyopathy in adult patients with DBMD.
  • To assess the impact of sildenafil on cardiac function, skeletal muscle function, and quality of life.

Main Methods:

  • A randomized, double-blind, placebo-controlled trial was conducted.
  • Adults with DBMD and cardiomyopathy (ejection fraction ≤ 50%) received either sildenafil (20mg 3x daily) or placebo for 6 months, followed by 6 months of open-label sildenafil.
  • The primary endpoint was the change in left ventricular end-systolic volume (LVESV) measured by cardiac MRI.

Main Results:

  • An interim analysis showed no statistically significant difference in outcome measures between the sildenafil and placebo groups.
  • A higher percentage of subjects on sildenafil experienced worsening LVESV compared to placebo, although this was not statistically significant.
  • The study was terminated early due to safety concerns and lack of efficacy.

Conclusions:

  • Sildenafil is unlikely to improve cardiac function in adults with DBMD.
  • Further research with larger sample sizes is needed to definitively assess the role of PDE5 inhibitors in DBMD cardiomyopathy.
  • Caution is advised when interpreting results due to the small sample size and early termination.
Abstract

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