Plasma idebenone monitoring in Friedreich's ataxia patients during a long-term follow-up

Despite the growing interest and the potential benefits of idebenone as a repurposed drug for different orphan conditions, data regarding its monitoring are scarce. This study reports plasma idebenone values in a cohort of Friedreich's ataxia (FRDA) patients during a long-term follow-up. Taking advantage of this, cardiological and neurological status were also assessed together with idebenone values and genetic background. A long-term follow-up retrospective study in 27 FRDA patients with a disease onset at the pediatric age treated with idebenone by compassionate use is reported. Plasma idebenone was measured by HPLC with electrochemical detection. Median plasma idebenone values increased when doses were increased, but apparently linearity was lost in the highest dose group. Marked intraindividual and interindividual differences were observed among patients. We did not find a consistent positive effect after analysis of paired data at the beginning and the end of the study. We only found a correlation between some cardiological measures and the duration of idebenone therapy at high doses, but with uncertain significance. The large variations observed among the different individuals involved in this study should be considered for optimization of individual dosage regimens.

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