Intermediate-dose idebenone and quality of life in Friedreich ataxia.

Idebenone has been used as therapy for Friedreich ataxia for more than a decade. Although several studies have assessed the influence of therapy on neurologic or cardiac function, there is a paucity of data surrounding patient-reported outcome measures. In an observational study of the effect of intermediate-dose idebenone (20 mg/kg per day) on quality of life and neurologic function measures, seven patients with Friedreich ataxia were assessed using the Pediatric Quality of Life Inventory, the International Cooperative Ataxia Rating Scale, and an Activities of Daily Living Scale before initiation of idebenone therapy and after 1 year of therapy. Physical scores on the Pediatric Quality of Life Inventory were universally worse after 1 year, and correlated with decreased activities of daily living scores. Despite worsening physical scores, there was a trend toward improved total, emotional, social, and school components of quality of life scores after 1 year of idebenone therapy. There was no statistically significant change in Pediatric Quality of Life Inventory scores between baseline and 1 year of idebenone therapy. Functional ability, as measured by activities of daily living scores, appeared to have the most influence on the perception of physical quality of life, which may be important in planning future therapeutic trials.