Coenzyme Q(10)-responsive ataxia: 2-year-treatment follow-up

Movement Disorders : Official Journal of the Movement Disorder Society
Mercé PinedaRafael Artuch

Abstract

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 = 0.018) after 2 years of CoQ(10) treatment when compared with baseline conditions. In patients without CoQ(10) deficiency, no statistically 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.

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Citations

Apr 12, 2012·Archives of Neurology·Valentina EmmanueleMichio Hirano
Jun 6, 2013·Pediatric Nephrology : Journal of the International Pediatric Nephrology Association·Fatih Ozaltin
Jun 19, 2013·Journal of Inherited Metabolic Disease·Nuria BujánAntonia Ribes
Nov 9, 2014·BMC Pediatrics·Delia YuberoRafael Artuch
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Oct 13, 2011·BioFactors·Catarina M Quinzii, Michio Hirano
Dec 3, 2014·Movement Disorders : Official Journal of the Movement Disorder Society·Raymond Y LoSheng-Han Kuo
Mar 16, 2013·Muscle & Nerve·R Brian SommervilleAlan Pestronk
Aug 18, 2017·Developmental Medicine and Child Neurology·Tjitske F LawermanUNKNOWN the Childhood Ataxia and Cerebellar Group of the European Pediatric Neurology Society
Feb 23, 2019·Current Treatment Options in Neurology·Christopher D StephenJeremy D Schmahmann
Jul 24, 2013·Journal of Neurochemistry·Katrin BürkJörg B Schulz
Mar 12, 2015·Movement Disorders : Official Journal of the Movement Disorder Society·Adolfo Ramirez-ZamoraJosé Biller
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Apr 4, 2020·Clinical Parkinsonism & Related Disorders·Divya K P, Asha Kishore

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