Coenzyme Q10-responsive ataxia: 2-year-treatment follow-up

✍ Scribed by Merce Pineda; Raquel Montero; Asuncion Aracil; Mar M. O'Callaghan; Ana Mas; Carmen Espinos; Dolores Martinez-Rubio; Francesc Palau; Placido Navas; Paz Briones; Rafael Artuch

• Publisher: John Wiley and Sons
• Year: 2010
• Tongue: English
• Weight: 166 KB
• Volume: 25
• Category: Article
• ISSN: 0885-3185
• DOI: 10.1002/mds.23129

✦ Synopsis

We assessed the clinical outcome after coenzyme Q 10 (CoQ 10 ) therapy in 14 patients presenting ataxia classified into two groups according to CoQ 10 values in muscle (deficient or not). We performed an open-label prospective study: patients were evaluated clinically (international cooperative ataxia rating scale [ICARS] scale, MRI, and videotape registration) at baseline and every 6 months during a period of 2 years after CoQ 10 treatment (30 mg/kg/day). Patients with CoQ 10 deficiency showed a statistically significant reduction of ICARS scores (Wilcoxon test: P 5 0.018) after 2 years of CoQ 10 treatment when compared with baseline conditions. In patients without CoQ 10 deficiency, no statisti-cally significant differences were observed in total ICARS scores after therapy, although 1 patient from this group showed a remarkable clinical amelioration. Biochemical diagnosis of CoQ 10 deficiency was a useful tool for the selection of patients who are good candidates for treatment as all of them responded to therapy. However, the remarkable clinical response in 1 case without CoQ 10 deficiency highlights the importance of treatment trials for identification of patients with CoQ 10 -responsive ataxia.