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Neurology 2003;60:1676-1679
© 2003 American Academy of Neurology
Brief Communications

Idebenone treatment in Friedreich patients: One-year-long randomized placebo-controlled trial

C. Mariotti, MD, A. Solari, MD, D. Torta, MD, L. Marano, MD, C. Fiorentini, MD and S. Di Donato, MD

From the Division of Biochemistry and Genetics (Drs. Mariotti and Di Donato) and Unit of Neuro-Epidemiology (Dr. Solari), Carlo Besta National Neurological Institute–IRCCS, Milan; and Division of Cardiology (Drs. Torta, Marano, and Fiorentini), San Paolo Hospital, University of Milan, Italy.

Address correspondence and reprint requests to Dr. Stefano Di Donato, Divisione di Biochimica e Genetica, Istituto Nazionale Neurologico Carlo Besta–IRCCS, via Celoria 11, 20133 Milano, Italy; e-mail: didonato{at}istituto-besta.it

The authors carried out a 1-year, randomized, placebo-controlled trial of idebenone in 29 patients with Friedreich ataxia. They found significant reductions of interventricular septal thickness and left ventricular mass in the idebenone group vs the placebo group, with no improvement in other heart ultrasound measures or neurologic condition. The absolute cardiac changes were modest, but the findings suggest that larger trials should assess whether idebenone reduces ventricular hypertrophy in patients with Friedreich ataxia.




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