Abstracts from American Heart Association Scientific Sessions 2003,

Orlando, 9-12 november 2003

 

Perindopril Prevents the Onset and Progression of Left Ventricular Dysfunction in Children with Duchenne Muscular Dystrophy

Henri-Marc Becana, Pitie-Salpetriere Hospital, Paris, France; Christophe Meune, Cochin Hospital, Paris, France; Guy Lerebours, Pitie-Salpetriere Hospital, Paris, France; Jean-Yves Devaux, Denis Duboc, Cochin Hospital, Paris, France; The French Working Group on Heart Involvement in Myopathies

Background: Duchenne Muscular disease (DMD) is an inherited X-linked disease due to the absence of dystrophin and clinically characterized by progressive muscle weakness and constant myocardial involvement responsible for sudden death or end-stage heart failure in 40% of patients between the age of 15 and 30. The aim of this study was to evaluate the preventive effect of early ACEI treatment on left ventricular (LV) dysfunction in young patients with DMD and normal LVEF at inclusion. Methods: In phase I, children with genetically proven DMD and radionuclide ejection fraction (EF) superior than 55% were enrolled in a multicentric, controlled, randomized, double blind trial of perindopril 2-4 mg/day versus placebo for 3 years. In phase II, all patients then received open-label perindopril for 24 more months. Radionuclide LVEF was performed at 0, 36 and 60 months. Student's t-tests and chi-square analysis were used for comparisons. Results: 60 children asymptomatic for heart disease were included in the phase I study (10.6±1.2 years, EF 65.0±5.4%)(31 in the perindropil group and 29 in the placebo group) and 46 in the phase II study (n=23 in both groups). No adverse effect related to treatment was documented. At the end of phase I, LVEF remained stable in both groups (from 64.8±5.3% to 59.6±8.5% in the perindopril group and 65.5±5.4% to 64.5±9.9% in the placebo group, p=0.114). However, at 60 months, 6 patients in the control group (26%) had a LVEF of less than 45, versus one in the perindopril group (4%)(p=0.0319). Conclusions: Early treatment with ACEI perindopril is well tolerated in these young patients and delay onset of LVEF deterioration. This preventive efficacy of ACEI has to be evaluated in other groups of patients genetically exposed to develop LV dysfunction.

 

Impaired Strain Rate Measurements in Patients With Duchenne Muscular Dystrophy

Nickolaos Giatrakos, Cardiology dept, Hammersmith Hospital, NHLI, ICSM, London, United Kingdom; Maria Kinali, Dubowitz Neuromuscular Centre, Department of Paediatrics, Hammersmith Hospital, ICSM, London, UK, London, United Kingdom; George Koutroulis, Cardiology dept, Hammersmith Hospital, NHLI, ICSM, London, United Kingdom; Francesco Muntoni, Dubowitz Neuromuscular Centre, Department of Paediatrics, Hammersmith Hospital, ICSM, London, UK, London, United Kingdom; Petros Nihoyannopoulos; Cardiology dept, Hammersmith Hospital, NHLI, ICSM, London, United Kingdom

Background: Patients with Duchenne muscular dystrophy (DMD) develop dilated cardiomyopathy at the later stages of the disease. Strain rate (SR) has been used to study myocardial function in ischeamia and cardiomyopathies. The aim of this study was to investigate the usefulness of SR for the early detection of cardiac involvement in young, asymptomatic patients with DMD.
Methods: We studied 53 patients with genetically confirmed diagnosis of DMD (mean age 8.7±2.8 years) without clinical symptoms from the cardiovascular system and normal conventional echocardiographic studies, and compared with 22 normal controls matched for age (mean age 8.5±2.5 years). We used the HDI 5000 (Philips Medical Systems) to acquire from the parasternal long axis the colour M-mode tissue Doppler (TDI) of the posterior wall of the LV. Images were digitally stored for offline analysis with dedicated software HDI-lab (Philips Medical Systems). We calculated the SR using the formula SR=Ua-Ub/d where U the velocities of the endocardium a and epicardium b, and d the distance of a and b at each time point.
Results: There was no significant difference for the parameters from conventional echocardiographic studies between the two groups. The velocities derived from the TDI, mean velocity at systole (26,99±7,12mm/sec vs. 33,4±7,3mm/sec, p<0,000), at early diastole (-45,79±13,93mm/sec vs.-60,46±7,58mm/sec, p<0,000) and late diastole (-10,93±3,41mm/sec vs.-13,32±6,4mm/sec, p<0,02) were significantly different in patients with DMD when compared with controls. SR was found to be significantly lower at systole (1,78±0,75s-1 vs. 2,82±0,5s-1, p<0,000) and early diastole (-5,17±1,98s-1 vs.-9,02±1,25s-1, p<0,000) but not at late diastole (-1,52±0,84s-1vs.-1.6±0,46s-1, p<0,568) in patients with DMD.
Conclusions: Estimation of SR of the posterior wall of the LV showed systolic and diastolic dysfunction at early stages in asymptomatic patients with DMD and when the conventional echocardiography is still normal. Estimation of SR could be a sensitive method to investigate the pathophysiology of the disease and identify early impairment of the cardiac function.