Plasma idebenone monitoring in Friedreich’s ataxia patients during a long-term follow-up - 09/10/21

Doi : 10.1016/j.biopha.2021.112143

Abraham J. Paredes-Fuentes ^a,^{^{1
These authors contributed equally to this work as first authors.}}, Sergi Cesar ^b,^{^{1
These authors contributed equally to this work as first authors.}}, Raquel Montero ^a, Cristina Latre ^c, Jordi Genovès ^d, Loreto Martorell ^d, Daniel Cuadras ^e, Helena Colom ^f, Mercè Pineda ^g, Maria del Mar O’Callaghan ^g, Georgia Sarquella-Brugada ^b,^h, Alejandra Darling ^g, Rafael Artuch ^a,^i,^⁎
^a Clinical Biochemistry Department, Institut de Recerca Sant Joan de Déu, Hospital Sant Joan de Déu, Esplugues de Llobregat, 08950 Barcelona, Spain
^b Arrhythmia, Inherited Cardiac Diseases and Sudden Death Unit, Hospital Sant Joan de Déu, Esplugues de Llobregat, 08950 Barcelona, Spain
^c Pharmacy Department, Hospital Sant Joan de Déu, Esplugues de Llobregat, 08950 Barcelona, Spain
^d Molecular Genetics Department, Hospital Sant Joan de Déu, Esplugues de Llobregat, 08950 Barcelona, Spain
^e Statistics Department, Institut de Recerca Sant Joan de Déu, Esplugues de Llobregat, 08950 Barcelona, Spain
^f Department of Pharmacy and Pharmaceutical Technology and Physical-Chemistry, Faculty of Pharmacy and Food Sciences, University of Barcelona, Barcelona, Spain
^g Metabolic and Ataxia Unit, Department of Paediatric Neurology, Hospital Sant Joan de Déu, Esplugues de Llobregat, 08950 Barcelona, Spain
^h Medical Sciences Department, School of Medicine, University of Girona, 17004 Girona, Spain
ⁱ Biomedical Network Research Centre on Rare Diseases (CIBERER), Instituto de Salud Carlos III, 28029 Madrid, Spain

^⁎Corresponding author at: Clinical Biochemistry Department, Institut de Recerca Sant Joan de Déu, Hospital Sant Joan de Déu, Esplugues de Llobregat, 08950 Barcelona, Spain.Clinical Biochemistry Department, Institut de Recerca Sant Joan de Déu, Hospital Sant Joan de DéuEsplugues de LlobregatBarcelona08950Spain

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Abstract

Introduction and objectives

Despite the growing interest and the potential benefits of idebenone as a repurposed drug for different orphan conditions, data regarding its monitoring are scarce. Our main goal was to report plasma idebenone values in a cohort of Friedreich’s ataxia (FRDA) patients during a long-term follow-up. Taking advantage of this, we also assessed cardiological and neurological status together with idebenone values and genetic background.

Methods

Long-term follow-up retrospective study in 27 FRDA patients with a disease onset at the paediatric age treated with idebenone by compassionate use. Plasma idebenone was measured by HPLC with electrochemical detection.

Results

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.

Conclusions

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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Highlights

•	Despite the growing interest and the potential benefits of idebenone, data regarding its monitoring are scarce.
•	We report plasma idebenone values of a cohort of 27 Friedreich’s ataxia patients during a long-term follow-up period.
•	Large interindividual and intraindividual variability in plasma idebenone concentrations was observed.
•	These large variations should be considered for optimization of individual dosage regimens.

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Abbreviations : CoQ, FRDA, HPLC, ICARS, iLV mass, IVSd, LHON, LVEF, LVPWd, RWT

Keywords : Plasma idebenone monitoring, Friedreich’s ataxia, Long-term follow-up, HPLC with electrochemical detection

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Plasma idebenone determination

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Plasma idebenone monitoring in Friedreich’s ataxia patients during a long-term follow-up - 09/10/21

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