Dramatic improvement in mitochondrial cardiomyopathy following treatment with idebenone

被引：41

作者：

Lerman-Sagie, T

Rustin, P

Lev, D

Yanoov, M

Leshinsky-Silver, E

Sagie, A

Ben-Gal, T

Munnich, A

机构：

[1] Wolfson Med Ctr, Metab Neurogenet Serv, Mitochondrial Dis Clin, IL-58100 Holon, Israel

[2] Wolfson Med Ctr, Inst Physiol Hyg, IL-58100 Holon, Israel

[3] Rabin Med Ctr, Dept Cardiol, Petah Tikva, Israel

[4] Tel Aviv Univ, Sackler Fac Med, IL-69978 Tel Aviv, Israel

[5] Hop Necker Enfants Malad, Unite Rech Handicaps Genet Enfant, Paris, France

来源：

JOURNAL OF INHERITED METABOLIC DISEASE | 2001年 / 24卷 / 01期

关键词：

D O I：

10.1023/A:1005642302316

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Idebenone, a synthetic analogue of coenzyme Q(10), has been shown to improve cardiac function in patients with Friedreich ataxia and a deficiency of respiratory chain complexes I-III. We describe a woman with severe combined right and left heart failure due to a mitochondrial cardiomyopathy. The patient underwent an endomyocardial biopsy as part of an evaluation for cardiac transplantation. It showed severely decreased respiratory complex activities dependent on CoQ, pointing to CoQ depletion. Following idebenone treatment there was a dramatic improvement in her clinical status with resolution of the heart failure.

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页码：28 / 34

页数：7

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