[Successful idebenone therapy of hypertrophic cardiomyopathy in Friedreich ataxia].
Severe hypertrophic cardiomyopathy in a 15 years old child with Friedreich ataxia was treated with idebenone on the basis of a preliminary study reported in the literature. After 3 months of treatment the muscle thickness and mass and idices of diastolic function on echocardiogram and ischaemic signs on ECG changed significantly.


CONCLUSIONS
Our data proves the preliminary results, so idebenobe treatment is very effective in the disease, where a deficiency of frataxin is involved in the regulation of mitochondral iron content which is responsible for myocardial injury. We suggest the widespread use of idebenone to treat patients with Friedreich ataxia and hypertrophic cardiomyopathy to improve the fatal prognosis of this type of cardiomyopathy.
Kádár
Krisztina
K
Gottsegen György Országos Kardiológiai Intézet, Gyermekszív Központ.
D000293:Adolescent; D000975:Antioxidants / Q000627:therapeutic use*; D016227:Benzoquinones / Q000627:therapeutic use*; D002312:Cardiomyopathy, Hypertrophic / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000378:metabolism / Q000503:physiopathology; D002648:Child; D004452:Echocardiography; D004562:Electrocardiography; D005260:Female; D005621:Friedreich Ataxia / Q000150:complications*; D006801:Humans; D033862:Iron-Binding Proteins / Q000378:metabolism; D008929:Mitochondria, Heart / Q000378:metabolism; D016896:Treatment Outcome; D014451:Ubiquinone / Q000031:analogs & derivatives; D000096923:Frataxin
D000975:Antioxidants; D016227:Benzoquinones; D033862:Iron-Binding Proteins; D014451:Ubiquinone; D000096923:Frataxin; C036619:idebenone