Coenzyme Q(10)-responsive ataxia: 2-year-treatment follow-up.
Identifieur interne : 002C99 ( Ncbi/Merge ); 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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O'Callaghan</name></author><author><name sortKey="Mas, Ana" sort="Mas, Ana" uniqKey="Mas A" first="Ana" last="Mas">Ana Mas</name></author><author><name sortKey="Espinos, Carmen" sort="Espinos, Carmen" uniqKey="Espinos C" first="Carmen" last="Espinos">Carmen Espinos</name></author><author><name sortKey="Martinez Rubio, Dolores" sort="Martinez Rubio, Dolores" uniqKey="Martinez Rubio D" first="Dolores" last="Martinez-Rubio">Dolores Martinez-Rubio</name></author><author><name sortKey="Palau, Francesc" sort="Palau, Francesc" uniqKey="Palau F" first="Francesc" last="Palau">Francesc Palau</name></author><author><name sortKey="Navas, Placido" sort="Navas, Placido" uniqKey="Navas P" first="Placido" last="Navas">Placido Navas</name></author><author><name sortKey="Briones, Paz" sort="Briones, Paz" uniqKey="Briones P" first="Paz" last="Briones">Paz Briones</name></author><author><name sortKey="Artuch, Rafael" sort="Artuch, Rafael" uniqKey="Artuch R" first="Rafael" last="Artuch">Rafael Artuch</name></author></analytic><series><title level="j">Movement disorders : official journal of the Movement Disorder Society</title><idno type="eISSN">1531-8257</idno><imprint><date when="2010" type="published">2010</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adolescent</term><term>Adult</term><term>Ataxia (drug therapy)</term><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></keywords><keywords scheme="MESH" type="chemical" qualifier="analogs & derivatives" xml:lang="en"><term>Ubiquinone</term></keywords><keywords scheme="MESH" type="chemical" qualifier="deficiency" xml:lang="en"><term>Ubiquinone</term></keywords><keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Ataxia</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Ataxia</term></keywords><keywords scheme="MESH" qualifier="methods" xml:lang="en"><term>Neurologic Examination</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Ubiquinone</term><term>Vitamins</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Adolescent</term><term>Adult</term><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></keywords></textClass></profileDesc></teiHeader><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></TEI><pubmed><MedlineCitation Owner="NLM" Status="MEDLINE"><PMID Version="1">20629161</PMID><DateCreated><Year>2010</Year><Month>07</Month><Day>26</Day></DateCreated><DateCompleted><Year>2010</Year><Month>11</Month><Day>04</Day></DateCompleted><DateRevised><Year>2014</Year><Month>11</Month><Day>20</Day></DateRevised><Article PubModel="Print"><Journal><ISSN IssnType="Electronic">1531-8257</ISSN><JournalIssue CitedMedium="Internet"><Volume>25</Volume><Issue>9</Issue><PubDate><Year>2010</Year><Month>Jul</Month><Day>15</Day></PubDate></JournalIssue><Title>Movement disorders : official journal of the Movement Disorder Society</Title><ISOAbbreviation>Mov. Disord.</ISOAbbreviation></Journal><ArticleTitle>Coenzyme Q(10)-responsive ataxia: 2-year-treatment follow-up.</ArticleTitle><Pagination><MedlinePgn>1262-8</MedlinePgn></Pagination><ELocationID EIdType="doi" ValidYN="Y">10.1002/mds.23129</ELocationID><Abstract><AbstractText>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.</AbstractText></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Pineda</LastName><ForeName>Merce</ForeName><Initials>M</Initials><AffiliationInfo><Affiliation>Department of Pediatric Neurology, Hospital Sant Joan de Déu, Barcelona, Spain.</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Montero</LastName><ForeName>Raquel</ForeName><Initials>R</Initials></Author><Author ValidYN="Y"><LastName>Aracil</LastName><ForeName>Asuncion</ForeName><Initials>A</Initials></Author><Author ValidYN="Y"><LastName>O'Callaghan</LastName><ForeName>Mar M</ForeName><Initials>MM</Initials></Author><Author ValidYN="Y"><LastName>Mas</LastName><ForeName>Ana</ForeName><Initials>A</Initials></Author><Author ValidYN="Y"><LastName>Espinos</LastName><ForeName>Carmen</ForeName><Initials>C</Initials></Author><Author ValidYN="Y"><LastName>Martinez-Rubio</LastName><ForeName>Dolores</ForeName><Initials>D</Initials></Author><Author ValidYN="Y"><LastName>Palau</LastName><ForeName>Francesc</ForeName><Initials>F</Initials></Author><Author ValidYN="Y"><LastName>Navas</LastName><ForeName>Placido</ForeName><Initials>P</Initials></Author><Author ValidYN="Y"><LastName>Briones</LastName><ForeName>Paz</ForeName><Initials>P</Initials></Author><Author ValidYN="Y"><LastName>Artuch</LastName><ForeName>Rafael</ForeName><Initials>R</Initials></Author></AuthorList><Language>eng</Language><PublicationTypeList><PublicationType UI="D016430">Clinical Trial</PublicationType><PublicationType UI="D016428">Journal Article</PublicationType><PublicationType UI="D013485">Research Support, Non-U.S. Gov't</PublicationType></PublicationTypeList></Article><MedlineJournalInfo><Country>United States</Country><MedlineTA>Mov Disord</MedlineTA><NlmUniqueID>8610688</NlmUniqueID><ISSNLinking>0885-3185</ISSNLinking></MedlineJournalInfo><ChemicalList><Chemical><RegistryNumber>0</RegistryNumber><NameOfSubstance UI="D014815">Vitamins</NameOfSubstance></Chemical><Chemical><RegistryNumber>1339-63-5</RegistryNumber><NameOfSubstance UI="D014451">Ubiquinone</NameOfSubstance></Chemical><Chemical><RegistryNumber>EJ27X76M46</RegistryNumber><NameOfSubstance UI="C024989">coenzyme Q10</NameOfSubstance></Chemical></ChemicalList><CitationSubset>IM</CitationSubset><MeshHeadingList><MeshHeading><DescriptorName MajorTopicYN="N" UI="D000293">Adolescent</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D000328">Adult</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D001259">Ataxia</DescriptorName><QualifierName MajorTopicYN="Y" UI="Q000188">drug therapy</QualifierName><QualifierName MajorTopicYN="N" UI="Q000378">metabolism</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D002648">Child</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D002675">Child, Preschool</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D005260">Female</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D006801">Humans</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D008137">Longitudinal Studies</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D008297">Male</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D009460">Neurologic Examination</DescriptorName><QualifierName MajorTopicYN="N" UI="Q000379">methods</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D018709">Statistics, Nonparametric</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D013997">Time Factors</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D016896">Treatment Outcome</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D014451">Ubiquinone</DescriptorName><QualifierName MajorTopicYN="Y" UI="Q000031">analogs & derivatives</QualifierName><QualifierName MajorTopicYN="N" UI="Q000172">deficiency</QualifierName><QualifierName MajorTopicYN="N" UI="Q000627">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D014815">Vitamins</DescriptorName><QualifierName MajorTopicYN="Y" UI="Q000627">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D055815">Young Adult</DescriptorName></MeshHeading></MeshHeadingList></MedlineCitation><PubmedData><History><PubMedPubDate PubStatus="entrez"><Year>2010</Year><Month>7</Month><Day>15</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="pubmed"><Year>2010</Year><Month>7</Month><Day>16</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="medline"><Year>2010</Year><Month>11</Month><Day>5</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate></History><PublicationStatus>ppublish</PublicationStatus><ArticleIdList><ArticleId IdType="doi">10.1002/mds.23129</ArticleId><ArticleId IdType="pubmed">20629161</ArticleId></ArticleIdList></PubmedData></pubmed><affiliations><list><country><li>Espagne</li></country><region><li>Catalogne</li></region><settlement><li>Barcelone</li></settlement></list><tree><noCountry><name sortKey="Aracil, Asuncion" sort="Aracil, Asuncion" uniqKey="Aracil A" first="Asuncion" last="Aracil">Asuncion Aracil</name><name sortKey="Artuch, Rafael" sort="Artuch, Rafael" uniqKey="Artuch R" first="Rafael" last="Artuch">Rafael Artuch</name><name sortKey="Briones, Paz" sort="Briones, Paz" uniqKey="Briones P" first="Paz" last="Briones">Paz Briones</name><name sortKey="Espinos, Carmen" sort="Espinos, Carmen" uniqKey="Espinos C" first="Carmen" last="Espinos">Carmen Espinos</name><name sortKey="Martinez Rubio, Dolores" sort="Martinez Rubio, Dolores" uniqKey="Martinez Rubio D" first="Dolores" last="Martinez-Rubio">Dolores Martinez-Rubio</name><name sortKey="Mas, Ana" sort="Mas, Ana" uniqKey="Mas A" first="Ana" last="Mas">Ana Mas</name><name sortKey="Montero, Raquel" sort="Montero, Raquel" uniqKey="Montero R" first="Raquel" last="Montero">Raquel Montero</name><name sortKey="Navas, Placido" sort="Navas, Placido" uniqKey="Navas P" first="Placido" last="Navas">Placido Navas</name><name sortKey="O Callaghan, Mar M" sort="O Callaghan, Mar M" uniqKey="O Callaghan M" first="Mar M" last="O'Callaghan">Mar M. O'Callaghan</name><name sortKey="Palau, Francesc" sort="Palau, Francesc" uniqKey="Palau F" first="Francesc" last="Palau">Francesc Palau</name></noCountry><country name="Espagne"><region name="Catalogne"><name sortKey="Pineda, Merce" sort="Pineda, Merce" uniqKey="Pineda M" first="Merce" last="Pineda">Merce Pineda</name></region></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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