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Rapidly progressive heart failure requiring transplantation in muscular dystrophy: a need for frequent screening

Justin M Pick1, Zachary D Ellis2, Juan C Alejos3

  • 11Children's Hospital of Orange County,Orange,California,United States of America.

Cardiology in the Young
|July 11, 2017
PubMed

Insights

Fukuyama congenital muscular dystrophy (FCMD) can rapidly worsen heart function, even with mild skeletal muscle weakness. Early cardiac screening is vital for FCMD patients to monitor for severe cardiomyopathy and potential heart transplant needs.

Area of Science:

  • Neurology
  • Cardiology
  • Genetics

Background:

  • Fukuyama congenital muscular dystrophy (FCMD) is a rare genetic neuromuscular disorder.
  • It affects skeletal and cardiac muscles, leading to progressive weakness.

Observation:

  • A 14-year-old boy with FCMD exhibited mild skeletal myopathy.
  • He simultaneously presented with severe cardiomyopathy, necessitating a heart transplant within a year due to rapid cardiac function decline.

Findings:

  • In FCMD, cardiomyopathy progression can outpace skeletal muscle deterioration.
  • The severity of cardiac involvement may not correlate with skeletal muscle symptoms.

Implications:

  • FCMD patients require consistent cardiac monitoring, irrespective of their skeletal muscle status.
  • This highlights the critical need for proactive cardiological screening in managing FCMD to prevent severe cardiac events and guide timely interventions like heart transplantation.

Keywords:
Fukuyama congenital muscular dystrophydilated cardiomyopathyleft ventricular systolic function

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