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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice
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Effects of cardiac medications on ventricular function in patients with Duchenne muscular dystrophy-related

Alexander P Murphy1, Anna Johnson2, Volker Straub1

  • 1The John Walton Muscular Dystrophy Research Centre, Institute of Translational and Clinical Research, Newcastle University, Newcastle upon Tyne, UK.

Muscle & Nerve
|May 29, 2021
PubMed
Abstract

Insights

Angiotensin-converting enzyme-inhibitor (ACEi) and beta-blocker (BB) therapy stabilized left ventricular function in Duchenne muscular dystrophy (DMD) patients. Early initiation of this cardiac medication combination is crucial for long-term benefits in DMD cardiac dystrophinopathy.

Area of Science:

  • Cardiology
  • Genetics
  • Pharmacology

Background:

  • Duchenne muscular dystrophy (DMD) can lead to progressive cardiac dystrophinopathy.
  • International guidelines recommend angiotensin-converting enzyme-inhibitor (ACEi) and beta-blocker (BB) therapy for cardiac involvement in DMD.
  • Previous studies indicated a progressive decline in left ventricular function in DMD patients.

Purpose of the Study:

  • To audit the changes in left ventricular function over 8 years in DMD patients treated with ACEi/BB therapy.
  • To evaluate the effectiveness of a combined ACEi/BB therapy in stabilizing cardiac function in DMD.
  • To assess the impact of ACEi/BB therapy initiation timing on left ventricular function.

Main Methods:

  • Retrospective audit of serial echocardiographic measures of left ventricular fractional shortening.
  • Inclusion of data from 104 genetically confirmed DMD patients.
  • Analysis of data from 4 years before and 4 years after initiating ACEi/BB therapy.

Main Results:

  • Left ventricular fractional shortening decline slowed from 1.5%/year to 0.9%/year after initiating ACEi/BB therapy.
  • The beneficial response to therapy was not affected by age at detection of left ventricular dysfunction or fractional shortening percentage at initiation.
  • Steroid therapy was associated with a later age of detection of left ventricular dysfunction.

Conclusions:

  • The study supports international recommendations for using ACEi/BB therapy in DMD.
  • Combination therapy with ACEi and BB effectively stabilizes heart function in DMD patients.
  • Initiating cardiac medication upon first detection of left ventricular impairment is vital for optimal long-term outcomes.
Keywords:
ACE inhibitorsbeta-blockersdystrophinopathyheart failureprognosis

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