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Echocardiographic evaluation of verapamil in Friedreich's ataxia
Insights
Verapamil did not improve cardiac function in patients with hypertrophic cardiomyopathy and Friedreich's ataxia. Echocardiography showed no significant differences compared to an untreated group, indicating no benefit from this calcium antagonist.
Area of Science:
- Cardiology
- Neurology
- Genetics
Background:
- Friedreich's ataxia (FA) is a rare inherited disorder causing progressive nervous system damage.
- Cardiac involvement, specifically hypertrophic cardiomyopathy (HCM), is a common and serious complication of FA.
- Myocardial calcium overload is a hypothesized contributor to cardiac disease in FA.
Purpose of the Study:
- To evaluate the efficacy of verapamil, a calcium channel blocker, in treating established myocardial hypertrophy in patients with Friedreich's ataxia.
- To determine if verapamil can improve cardiac structure and diastolic function in this patient population.
Main Methods:
- A prospective study involving nine patients with HCM associated with FA.
- Patients received daily oral verapamil (7 mg/kg) for a mean of 24 months.
- M-mode echocardiography was used to assess cardiac parameters at baseline and end of follow-up, comparing treated patients to an untreated control group of nine patients.
Main Results:
- Verapamil treatment showed no significant changes in left ventricular wall thickness, mass index, or internal diameter.
- Key echocardiographic measures of systolic and diastolic function, including fractional shortening and wall thinning rates, remained unchanged.
- No significant differences were observed between the verapamil-treated group and the untreated control group.
Conclusions:
- Verapamil did not demonstrate a beneficial effect on established myocardial hypertrophy or cardiac function in patients with Friedreich's ataxia.
- The findings suggest that verapamil is not an effective treatment for cardiac complications in this specific patient group.
- Further research is needed to explore alternative therapeutic strategies for cardiac disease in Friedreich's ataxia.
Abstract:
Nine patients with hypertrophic cardiomyopathy associated with Friedreich's ataxia were treated with the calcium antagonist verapamil, which is known to reduce myocardial hypertrophy and improve diastolic function in patients with idiopathic hypertrophic cardiomyopathy. Daily oral doses of 7 mg/kg were given for a mean (SD) of 24 (8) months. M mode echocardiography performed at the start of the study and at the end of follow up showed no significant difference between the treated group and an untreated control group of nine patients. Verapamil produced no changes in left ventricular wall thickness, mass index, left ventricular internal diameter, fractional shortening, peak normalised lengthening rate, peak rate of septal and posterior wall thinning, and time from minimum ventricular cavity dimension to mitral valve opening. Myocardial calcium overload has been suggested as a cause of cardiac disease in Friedreich's ataxia; however, verapamil had no beneficial effect on these patients with established myocardial hypertrophy.