Coenzyme Q(10)-responsive ataxia: 2-year-treatment follow-up.
Identifieur interne : 002C99 ( Ncbi/Curation ); précédent : 002C98; suivant : 002D00Coenzyme Q(10)-responsive ataxia: 2-year-treatment follow-up.
Auteurs : Merce Pineda [Espagne] ; Raquel Montero ; Asuncion Aracil ; Mar M. O'Callaghan ; Ana Mas ; Carmen Espinos ; Dolores Martinez-Rubio ; Francesc Palau ; Placido Navas ; Paz Briones ; Rafael ArtuchSource :
- Movement disorders : official journal of the Movement Disorder Society [ 1531-8257 ] ; 2010.
English descriptors
- KwdEn :
- Adolescent, Adult, Ataxia (drug therapy), Ataxia (metabolism), Child, Child, Preschool, Female, Humans, Longitudinal Studies, Male, Neurologic Examination (methods), Statistics, Nonparametric, Time Factors, Treatment Outcome, Ubiquinone (analogs & derivatives), Ubiquinone (deficiency), Ubiquinone (therapeutic use), Vitamins (therapeutic use), Young Adult.
- MESH :
- chemical , analogs & derivatives : Ubiquinone.
- chemical , deficiency : Ubiquinone.
- drug therapy : Ataxia.
- metabolism : Ataxia.
- methods : Neurologic Examination.
- chemical , therapeutic use : Ubiquinone, Vitamins.
- Adolescent, Adult, Child, Child, Preschool, Female, Humans, Longitudinal Studies, Male, Statistics, Nonparametric, Time Factors, Treatment Outcome, Young Adult.
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.
DOI: 10.1002/mds.23129
PubMed: 20629161
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pubmed:20629161Le document en format XML
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<author><name sortKey="Montero, Raquel" sort="Montero, Raquel" uniqKey="Montero R" first="Raquel" last="Montero">Raquel Montero</name>
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<author><name sortKey="Espinos, Carmen" sort="Espinos, Carmen" uniqKey="Espinos C" first="Carmen" last="Espinos">Carmen Espinos</name>
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<author><name sortKey="Martinez Rubio, Dolores" sort="Martinez Rubio, Dolores" uniqKey="Martinez Rubio D" first="Dolores" last="Martinez-Rubio">Dolores Martinez-Rubio</name>
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<author><name sortKey="Palau, Francesc" sort="Palau, Francesc" uniqKey="Palau F" first="Francesc" last="Palau">Francesc Palau</name>
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<author><name sortKey="Briones, Paz" sort="Briones, Paz" uniqKey="Briones P" first="Paz" last="Briones">Paz Briones</name>
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<series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title>
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<term>Ataxia (metabolism)</term>
<term>Child</term>
<term>Child, Preschool</term>
<term>Female</term>
<term>Humans</term>
<term>Longitudinal Studies</term>
<term>Male</term>
<term>Neurologic Examination (methods)</term>
<term>Statistics, Nonparametric</term>
<term>Time Factors</term>
<term>Treatment Outcome</term>
<term>Ubiquinone (analogs & derivatives)</term>
<term>Ubiquinone (deficiency)</term>
<term>Ubiquinone (therapeutic use)</term>
<term>Vitamins (therapeutic use)</term>
<term>Young Adult</term>
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<keywords scheme="MESH" type="chemical" qualifier="analogs & derivatives" xml:lang="en"><term>Ubiquinone</term>
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<keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Ataxia</term>
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<term>Child</term>
<term>Child, Preschool</term>
<term>Female</term>
<term>Humans</term>
<term>Longitudinal Studies</term>
<term>Male</term>
<term>Statistics, Nonparametric</term>
<term>Time Factors</term>
<term>Treatment Outcome</term>
<term>Young Adult</term>
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<front><div type="abstract" xml:lang="en">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.</div>
</front>
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