Cardioprotective medication in Duchenne muscular dystrophy: a single-centre cohort study
Janneta Kisel1,2, Emily Ballard1, Eui-Sik Suh1,2
1Lane Fox Unit, Guy's and St Thomas' NHS Trust, London, UK.
Insights
Long-term use of angiotensin-converting enzyme inhibitors (ACE-I) and beta-blockers helps preserve left ventricular ejection fraction (LVEF) in Duchenne muscular dystrophy (DMD) patients. These cardioprotective medications may prevent cardiovascular complications in DMD.
Area of Science:
- Cardiology
- Neurology
- Pharmacology
Background:
- Duchenne muscular dystrophy (DMD) is a progressive neuromuscular disorder leading to muscle wasting, impacting mobility, ventilation, and cardiac function.
- Neuromuscular cardiomyopathy is a significant cause of morbidity and mortality in DMD patients.
- Investigating cardioprotective medications like ACE-I and beta-blockers for clinical outcomes in DMD is crucial.
Purpose of the Study:
- To evaluate the impact of cardioprotective medications, specifically angiotensin-converting enzyme inhibitors (ACE-I) and beta-blockers, on clinical outcomes in patients with Duchenne muscular dystrophy.
- To assess the association between the duration of ACE-I and beta-blocker use and the decline in left ventricular ejection fraction (LVEF) in DMD patients.
Main Methods:
- Retrospective cohort study of Duchenne muscular dystrophy patients (1993-2021) at a tertiary center.
- Electronic health records were screened for demographics, comorbidities, medications, disease features, echocardiography, hospitalizations, and ventilator use.
- Analysis focused on left ventricular ejection fraction (LVEF) changes in relation to the use of ACE-I and beta-blockers.
Main Results:
- Of 68 DMD patients (mean age 27.4 years), 52 were alive; 90% required home mechanical ventilation, and 85% had DMD-associated cardiomyopathy.
- The average left ventricular ejection fraction (LVEF) declined by 3.3% over the follow-up period (P=0.002).
- Long-term use of ACE-I and beta-blockers was associated with a significantly reduced decline in LVEF compared to limited use (P=0.002 for ACE-I, P=0.02 for beta-blockers).
Conclusions:
- Long-term administration of ACE-I and beta-blockers is linked to a slower decline in LVEF in Duchenne muscular dystrophy patients.
- These cardioprotective medications may offer protection against adverse cardiovascular events in individuals with DMD.
- Consistent use of ACE-I and beta-blockers is recommended for managing cardiac function in DMD.
Background:
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder characterised by progressive muscle wasting impacting mobility, ventilation and cardiac function. Associated neuromuscular cardiomyopathy remains a major cause of morbidity and mortality. We investigated the effects of cardioprotective medications [angiotensin-converting enzyme inhibitors (ACE-I), beta-blockers] on clinical outcomes in DMD patients.
Methods:
This was a retrospective cohort study (reference: 2021/12469) of DMD patients at a tertiary centre between 1993-2021 screening the electronic records for demographics, comorbidities, medication, disease specific features, echocardiography, hospitalisations, and ventilator use.
Results:
A total of 68 patients were identified aged 27.4 (6.6) years, of which 52 were still alive. There was a difference in body mass index (BMI) between survivors and deceased patients [23.8 (5.9) vs. 19.9 (3.8) kg/m2, P=0.03]. Home mechanical ventilation (HMV) was required in 90% of patients, 85% had DMD associated cardiomyopathy. About 2/3 of all hospitalisations during the observation period were secondary to cardiopulmonary causes. The left ventricular ejection fraction (LVEF) at initial presentation was 44.8% (10.6%) and declined by 3.3% [95% confidence interval (CI): 0.4% to -7.0%] over the follow up period (P=0.002). A total of 61 patients were established on ACE-I for 75.9% (35.1%), and 62 were on beta-blockers for 73.6% (33.5%) of the follow up period. There was a significant LVEF decline in those taking ACE-I for limited periods compared to those permanently on ACE-I (P=0.002); a similar effect was recorded with beta-blockers (P=0.02).
Conclusions:
Long-term use of ACE-I and beta-blockers is associated with a reduced decline in LVEF in patients with DMD and may be protective of adverse cardiovascular ill health.
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