TITLE

Triple Therapy with Darbepoetin Alfa, Idebenone, and Riboflavin in Friedreich's Ataxia: an Open-Label Trial

AUTHOR(S)
Arpa, Javier; Sanz-Gallego, Irene; Rodríguez-de-Rivera, Francisco; Domínguez-Melcón, Francisco; Prefasi, Daniel; Oliva-Navarro, Javier; Moreno-Yangüela, Mar; Pascual-Pascual, Samuel
PUB. DATE
October 2013
SOURCE
Cerebellum;Oct2013, Vol. 12 Issue 5, p713
SOURCE TYPE
Academic Journal
DOC. TYPE
Article
ABSTRACT
Minimal objective evidence exists regarding management of Friedreich's ataxia (FRDA). Antioxidant and recombinant human erythropoietin therapies have been considered potential treatments to slow progression of FRDA in a small number of studies. The primary objective of the current study was to test the efficacy, safety, and tolerability of triple therapy-darbepoetin alfa, idebenone, and riboflavin-in FRDA in a clinical pilot study. Patients included in this study were nine females, 16 to 45 years of age (average 28 ± 8), diagnosed with FRDA with confirmed GAA repeat expansion mutations in the FXN gene and a GAA repeat ≥400 on the shorter allele. Patients had a baseline score between 8 and 28.5 (average 20.7 ± 8.3) on the scale for the assessment and rating of ataxia and 94.3 ± 27.2 g/m in left ventricular mass index (LVMI). Patients had been treated with triple therapy with 150 μg darbepoetin alfa every 2 or 3 weeks, 10-20 mg/kg/day idebenone, and 10-15 mg/kg/day riboflavin for 32 ± 19.4 months (range of 8-56 months). Triple therapy was tolerated. Although not statistically significant, improvement of ataxia was observed during the first six 4-month periods of the study. Furthermore, a small decrease in disease progression during the first 2 years of treatment was observed. Long-term statistically nonsignificant improvement of LVMI and stability of the echocardiographic parameters could be considered. Triple therapy may slow disease progression of FRDA.
ACCESSION #
90052399

 

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