ramipril versus carvedilol in duchenne and becker patients

Conditions: Duchenne Muscular Dystrophy; Becker Muscular Dystrophy

Summary: data on preventive therapy in duchenne muscular dystrophy (dmd) and becker muscular dystrophy (bmd) affected individuals without cardiac involvement are very limited and currently lacking regard both ace-inhibitors and beta-blockers in becker muscular dystrophy and for the latter even in duchenne muscular dystrophy patients. thus, the study aim is to compare the efficacy of carvedilol vs ramipril on myocardial tissue properties and heart function, performing cmr and myocardial ultrasound tissue characterisation analysis.

this protocol represent an open randomized and prospective trial, designed to answer the specific question regarding the role of the cardioprotective therapy in duchenne muscular dystrophy and becker muscular dystrophy patients. in this light, cmr could provide relevant data, reinforcing the scientific background, to start early (particularly in bmd patients in whom this is still a debated question) a cardioprotective treatment with carvedilol or ramipril.finally,this clinical trial will clarify whether a preventive therapy may be helpful on the clinical outcome, both in reducing myocardial fibrosis and preventing the progression towards the cardiomyopathy.

Eligibility criteria:
inclusion criteria: 1. immunohystochemical and molecular diagnosis of duchenne and becker muscular dystrophy. 2. not evidence of clinical cardiomyopathy,normal 2d-echocardiography with normal systolic,wmsi = 1) and diastolic function. 3. dmd patients treated with steroid therapy. 4. all dmd and bmd patients are not treated with cardiological therapy (ace-inhibitors, arbs or beta-blockers). 5. written informed consent to study participation (with serial visit, cmr and echocardiographic study) is required from all patients themselves, as well as their parent or guardian and healthy-control subjects. exclusion criteria: 1. failure to obtain informed consent from patients, parents or guardians. 2. any controindications to carvedilol or ramipril treatment (bronchial asthma, diabetes, any degree of renal failure (all patients are required to have a normal creatinine level and clearance). 3. in bmd patients ecg changes suggestive of ischemic heart disease, left bundle-branch block, atrial flutter/fibrillation, ventricular arrhythmias, any degree of atrioventricular block and left ventricular (lv) hypertrophy. aspecific st changes will be not considered as electrocardiographic exclusion criteria both in dmd and bmd patients. 4. in bmd patients exclusion criteria will be also hypertension and valvular heart disease other than trivial. 5. dmd and bmd patients requiring ventilatory (non-invasive or invasive) assistance. 6. presence of systolic and/or diastolic dysfunction detected by 2d-echocardiography. 7. presence of any contraindications to cmr (including any history of claustrophobia). 8. patients under the age of 2 years. 9. renal failure, even mild. 10. patient unable or unwilling to attend the follow-up and tests, in the opinion of local study principal investigator, (children not willing to perform cmr will not be enrolled).

Keywords: duchenne becker muscular dystrophy cardioprotective therapy cardiac magnetic resonance ultrasound tissue characterization