Transcript Duchenne Muscular Dystrophy: CardiacManagement

Duchenne Muscular Dystrophy:
Cardiac Management
Introduction
• Aim: early detection and treatment of deterioration in
heart muscle function
• Cardiac disease most often manifests as
– Cardiomyopathy and/or
– Cardiac arrhythmia
• Progressive cardiomyopathy a major source of DMD/BMD
morbidity – warrants further study
– Clear onset of disease in myocardium before clinical symptoms
– Surveillance and proactive management are key: a cardiologist
must be in the care team
• Family should have a copy of the latest assessments to
show attending doctors
Surveillance: Baselines
• Baseline evaluation of cardiac function at diagnosis or by 6
years at the latest, especially if possible without sedation
– Clinical judgement should be used for patients under 6 requiring
sedation
– Incidence of echocardiograph abnormalities is low in children
under 8-10 years, but where they do exist can affect clinical
decision-making
– Baseline echocardiogram also allows for screening for
anatomical abnormalities(e.g. atrial/ventricular septal defects,
patent ductus arteriosis) which may affect long-term
cardiovascular function
• Minimal evaluation should include, but is not limited to,
and electrocardiogram (ECG) and echocardiogram
Surveillance: Annual Evaluation
• Evaluation at least once every 2 years to the age
of 10.
• Annual complete cardiac assessments should
begin at 10, or at onset of cardiac signs or
symptoms if these occur earlier
• Increased surveillance (at least every 6 months)
required if non-invasive cardiac tests show
abnormalities of ventricular function.
– Drug treatment should be initiated, irrespective of the
age at which they are detected
Treatment
• First-line therapy: angiotension converting
enzyme (ACE) inhibitors
• Beta-blockers and diuretics also appropriate, and
should follow published guidelines for
management of heart failure
• Recent clinical trial evidence supports the
prophylactic treatment of cardiomyopathy with
ACE inhibitors prior to signs of abnormal
functioning: further studies awaited to allow firm
recommendations.
Abnormalities (1)
• Abnormalities of cardiac rhythm should be
promptly investigated with Holder or event
monitor recording, and should be treated
• Sinus tachycardia is common in DMD, but also
noted in systolic dysfunction.
– New onset in absence of clear cause should prompt
assessment including that of left-ventricular function
• Those on steroids need additional cardiovascular
attention, especially for hypertension which may
necessitate an adjustment of steroid dose [TLN
table 2 part 1]
Abnormalities (2)
• Systemic arterial hypertension should be treated
• Prevention of systemic thromboembolic events
by anticoagulation therapy can be considered in
severe cardiac dysfunction, but inappropriate in
earlier cardiac dysfunction
• Usefulness of internal cardiac defibrillator not
established
• With generally improved fitness of DMD patients,
cardiac transplants may require consideration in
future
References & Resources
• The Diagnosis and Management of Duchenne
Muscular Dystrophy, Bushby K et al, Lancet
Neurology 2010 9 (1) 77-93 & Lancet
Neurology 2010 9 (2) 177-189
– Particularly references, p186-188
• The Diagnosis and Management of Duchenne
Muscular Dystrophy: A Guide for Families
• TREAT-NMD website: www.treat-nmd.eu
• CARE-NMD website: www.care-nmd.eu