Idebenone treatment in paediatric and adult patients with Friedreich ataxia: long-term follow-up

Mercè Pineda, Javier Arpa, Raquel Montero, Asunción Aracil, Francisco Domínguez, Marta Galván, Anna Mas, Loreto Martorell, Cristina Sierra, Nuria Brandi, Elena García-Arumí, Miquel Rissech, Daniel Velasco, Juan A Costa, Rafael Artuch
European Journal of Paediatric Neurology: EJPN 2008, 12 (6): 470-5

BACKGROUND: Antioxidant therapy is a new therapeutical approach for patients with Friedreich ataxia.

AIMS: To assess the effectiveness of long-term idebenone treatment in Friedreich ataxia patients.

METHODS: An open-labelled prospective study. Ten paediatric patients (age range 8-18 years) and 14 adults (age range 18-46 years) with genetic diagnosis of Friedreich ataxia were treated with idebenone (5-20mg/kg/day) for 3-5 years. Neurological evolution was evaluated using the International Cooperative Ataxia Rating Scale (ICARS), and cardiological outcomes using echocardiography.

RESULTS: In paediatric patients, no significant differences were observed in ICARS scores and echocardiographic measurements when comparing baseline status and after 5 years of follow-up. Concerning adult cases, ICARS scores showed a significant increase in neurological dysfunctions during 3 years of therapy (Wilcoxon test, p=0.005), while echocardiographic measurements remained unchanged.

CONCLUSIONS: Our results indicate that longer-term idebenone treatment prevented progression of cardiomyopathy in both paediatric and adult patients, whereas its stabilizing effect on neurological dysfunction was present only in the paediatric population, mainly before puberty. This suggests that the age at which idebenone treatment is initiated may be an important factor in the effectiveness of the therapy.

Full Text Links

Find Full Text Links for this Article


You are not logged in. Sign Up or Log In to join the discussion.

Related Papers

Remove bar
Read by QxMD icon Read

Save your favorite articles in one place with a free QxMD account.


Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"