Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia – open-label trial
Article first published online: 14 MAY 2013
© 2013 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd
Acta Neurologica Scandinavica
Volume 129, Issue 1, pages 32–40, January 2014
How to Cite
Triple therapy with Deferiprone, Idebenone and Riboflavin in Friedreich's ataxia – Open-label trial. Acta Neurol Scand 2014: 129: 32–40. © 2013 John Wiley & Sons A/S. Published by John Wiley & Sons Ltd., , , , , , .
- Issue published online: 3 DEC 2013
- Article first published online: 14 MAY 2013
- Manuscript Accepted: 9 APR 2013
- Friedreich's ataxia;
The objective of the study was to test the efficacy, safety and tolerability of triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia (FRDA) patients in a clinical pilot study.
Patients and methods
Patients included in this study were 10 males and three females, 14–61 years of age (average 30.2 ± 12.1), diagnosed with FRDA with normal ventricular function. Patients were treated with triple therapy with deferiprone at 5–25 mg/kg/day, idebenone at 10–20 mg/kg/day and riboflavin at 10–15 mg/kg/day for 15–45 months. The efficacy of this triple therapy was assessed by change from baseline on the scale for the assessment and rating of ataxia (SARA) and by the change from baseline in echocardiogram parameters.
Four patients discontinued due to adverse events (AEs) related with deferiprone. The annual worsening rate (AWR) was estimated in this series as 0.96 (CI 95%: 0.462–1.608) SARA score, whereas AWR for our FRDA cohort was estimated as 2.05 ± 1.23 SARA score. LVMI only decreased by 6.5 g/m2 (6.2%) at the end of the first year of therapy. LVEF remained stable, except in case of three patients.
Our results seem to indicate some uncertain benefit on the neurological and heart functions of this triple therapy in FRDA.