What is the clinical evidence from this study?

Study design: Cohort. Population: Duchenne Muscular Dystrophy (n=40). Intervention: Duchenne Muscular Dystrophy vs. Healthy volunteers. Primary outcome: Left ventricular ejection fraction (LVEF) (p=<0.001).

May 21, 2010Open Access

Myocardial inflammation in Duchenne Muscular Dystrophy as a precipitating factor for heart failure: a prospective study

Q: What are the key findings of this study?

Duchenne Muscular Dystrophy patients had significantly lower left ventricular ejection fraction compared to healthy controls (53.9% vs 63%, p<0.001), and those with CMR-documented myocardial inflammation experienced rapid progression to heart failure.

Key Result

Duchenne Muscular Dystrophy patients had significantly lower left ventricular ejection fraction compared to healthy controls (53.9% vs 63%, p<0.001), and those with CMR-documented myocardial inflammation experienced rapid progression to heart failure.

Study Design

Type

Cohort (n=40)

Multicenter

Structured PICO

Does myocardial inflammation documented by CMR predict rapid progression to heart failure in patients with Duchenne Muscular Dystrophy?

Population

40 male adolescents (20 with Duchenne Muscular Dystrophy and 20 healthy controls) aged 15-18 years, followed for 2 years to evaluate myocardial inflammation as a precipitating factor for heart failure.

Exposure

Evaluation of myocardial inflammation using cardiovascular magnetic resonance imaging (CMR) and myocardial biopsy in positive cases.

Comparator

Age-matched healthy volunteers.

Outcome

Deterioration of left ventricular function and progression to heart failure over 2 years.surrogate

Myocardial inflammation detected by CMR in patients with Duchenne Muscular Dystrophy is associated with rapid deterioration of left ventricular function and progression to heart failure.

Main Result

Absolute Event Rate: 53.9% vs 63%

p-value: p=<0.001

Limitations

Small number of examined patients
Small number of myocardial biopsies taken from intraventricular septum and not from the gadolinium late enhanced areas
Patients were selected due to recent onset of possible cardiac involvement and do not reflect the real incidence of myocarditis in the DMD population

Abstract

BACKGROUND: In patients with Duchenne Muscular Dystrophy (DMD), the absent or diminished dystrophin leads to progressive skeletal muscle and heart failure. We evaluated the role of myocardial inflammation as a precipitating factor in the development of heart failure in DMD. METHODS: 20 DMD patients (aged 15-18 yrs) and 20 age-matched healthy volunteers were studied and followed-up for 2 years. Evaluation of myocarditis with cardiovascular magnetic resonance imaging (CMR) was performed using STIR T2-weighted (T2W), T1-weighted (T1W) before and after contrast media and late enhanced images (LGE). Left ventricular volumes and ejection fraction were also calculated. Myocardial biopsy was performed in patients with positive CMR and immunohistologic and polymerase chain reaction (PCR) analysis was employed. RESULTS: In DMD patients, left ventricular end-diastolic volume (LVEDV) was not different compared to controls. Left ventricular end-systolic volume (LVESV) was higher (45.1 +/- 6.6 vs. 37.3 +/- 3.8 ml, p < 0.001) and left ventricular ejection fraction (LVEF) was lower (53.9 +/- 2.1 vs. 63 +/- 2.4%, p < 0.001). T2 heart/skeletal muscle ratio and early T1 ratio values in DMD patients presented no difference compared to controls. LGE areas were identified in six DMD patients. In four of them with CMR evidence of myocarditis, myocardial biopsy was performed. Active myocarditis was identified in one and healing myocarditis in three using immunohistology. All six patients with CMR evidence of myocarditis had a rapid deterioration of left ventricular function during the next year. CONCLUSIONS: DMD patients with myocardial inflammation documented by CMR had a rigorous progression to heart failure.

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