This study aimed to use cardiac magnetic resonance imaging (CMR) to evaluate the efficacy and safety of bisoprolol therapy for boys with Duchenne muscular dystrophy(DMD) and preserved ejection fraction. On top of angiotensin-converting enzyme inhibitor (ACEI) , half of the participants will receive bisoprolol in combination, while the other half will not receive any beta-blocker.
By the age of 20 years, almost all the patients with Duchenne muscular dystrophy(DMD) have experienced dilated cardiomyopathy (DCM), a condition that contributes significantly to their morbidity and mortality. Studies have shown ACEI to be an effective therapy for DMD boys with early cardiomyopathy. Although bisoprolol has been demonstrated as an effective neurohumoral drug for adult patients with DCM, few data exist concerning its safety and efficacy for the patients with DMD. The investigators hypothesize that the treatment with bisoprolol in addition to background ACEI therapy for 12 months in participants with DMD and early myocardial damage evident with late gadolinium enhancement and preserved ejection fraction, would blunt decline in left ventricular systolic performance.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
42
Bisoprolol was initiated at a dose of 1.25 mg every 24hr. At subsequent biweekly visits, the bisoprolol dose was increased 1.25mg progressively until a daily dose of 0.2mg/kg or the maximum tolerated dose (The rest heart rate \<75bpm and systolic blood pressure \<90mmHg) is achieved. If the participant feel dizziness or the rest heart rate below 60 bpm or systolic blood pressure below 85mmHg or there is any new onset contraindication to bisoprolol, the doctor will decide to return back to the previous dose or stop bisoprolol. Each time the dose is increased, the medication is administered in an outpatient clinic setting, with assessment of the participant's heart rate, blood pressure, symptoms and ECG. Once reach the target dose, the patients will be followed up every 8 weeks in an face-to-face or video visit. After 12 months , the participants will repeat cardiac MR besides heart rate, blood pressure, symptoms, ECG and brain natriuretic peptide(BNP), echocardiography records.
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
RECRUITINGCalculate the change of left ventricle global longitudinal strain in cardiac MR
Calculate the change of left ventricle global longitudinal strain in cardiac MR from baseline to 12months for each patients
Time frame: baseline and 12 months
Calculate the change of left ventricular ejection fraction in cardia MR
Calculate the change of left ventricular ejection fraction in cardia MR from baseline to 12months for each patients
Time frame: baseline and 12 months
Calculate the change of ventricle late gadolinium enhancement area in cardia MR
Calculate the change of ventricle late gadolinium enhancement area in cardia MR from baseline to 12months for each patients
Time frame: baseline and 12 months
Calculate the change of the level of high-sensitivity cardiac troponin I
Calculate the change of the level of high-sensitivity cardiac troponin I from baseline to 6months and 12months for each patients
Time frame: baseline and 6months, 12 months
Calculate the change of the level of NT-proBNP
Calculate the change of the level of NT-proBNP from baseline to 6months and 12months for each patients
Time frame: baseline and 6months, 12 months
Calculate the change of E/A ratio assessed by echocardiography
Calculate the change of diastolic dysfunction (E/A ratio) assessed by echocardiography from baseline to 12months for each patients
Time frame: baseline and 12 months
Change of the resting heart rate
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Calculate the change of the resting heart rate from baseline to 6months and 12months for each patients
Time frame: baseline and 6months, 12 months