Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial.
Identifieur interne : 000E36 ( PubMed/Curation ); précédent : 000E35; suivant : 000E37Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial.
Auteurs : Caterina Mariotti [Italie] ; Roberto Fancellu ; Serena Caldarazzo ; Lorenzo Nanetti ; Daniela Di Bella ; Massimo Plumari ; Giuseppe Lauria ; Maria D. Cappellini ; Lorena Duca ; Alessandra Solari ; Franco TaroniSource :
- Movement disorders : official journal of the Movement Disorder Society [ 1531-8257 ] ; 2012.
English descriptors
- KwdEn :
- Adult, Antioxidants (therapeutic use), Dose-Response Relationship, Drug, Double-Blind Method, Erythropoietin (therapeutic use), Female, Friedreich Ataxia (drug therapy), Friedreich Ataxia (metabolism), Humans, Iron-Binding Proteins (metabolism), Male, Recombinant Proteins (therapeutic use), Statistics, Nonparametric, Time Factors, Treatment Outcome, Ubiquinone (analogs & derivatives), Ubiquinone (therapeutic use), Young Adult.
- MESH :
- chemical , analogs & derivatives : Ubiquinone.
- chemical , metabolism : Iron-Binding Proteins.
- chemical , therapeutic use : Antioxidants, Erythropoietin, Recombinant Proteins, Ubiquinone.
- drug therapy : Friedreich Ataxia.
- metabolism : Friedreich Ataxia.
- Adult, Dose-Response Relationship, Drug, Double-Blind Method, Female, Humans, Male, Statistics, Nonparametric, Time Factors, Treatment Outcome, Young Adult.
Abstract
Friedreich ataxia is a rare disease caused by GAA-trinucleotide-repeat expansions in the frataxin gene, leading to marked reduction of qualitatively normal frataxin protein. Recently, human recombinant erythropoietin was reported to increase frataxin levels in patients with Friedreich ataxia.
DOI: 10.1002/mds.24066
PubMed: 22411849
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Cappellini</name></author><author><name sortKey="Duca, Lorena" sort="Duca, Lorena" uniqKey="Duca L" first="Lorena" last="Duca">Lorena Duca</name></author><author><name sortKey="Solari, Alessandra" sort="Solari, Alessandra" uniqKey="Solari A" first="Alessandra" last="Solari">Alessandra Solari</name></author><author><name sortKey="Taroni, Franco" sort="Taroni, Franco" uniqKey="Taroni F" first="Franco" last="Taroni">Franco Taroni</name></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2012">2012</date><idno type="doi">10.1002/mds.24066</idno><idno type="RBID">pubmed:22411849</idno><idno type="pmid">22411849</idno><idno type="wicri:Area/PubMed/Corpus">000E36</idno><idno type="wicri:Area/PubMed/Curation">000E36</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial.</title><author><name sortKey="Mariotti, Caterina" sort="Mariotti, Caterina" uniqKey="Mariotti C" first="Caterina" last="Mariotti">Caterina Mariotti</name><affiliation wicri:level="1"><nlm:affiliation>SOSD Genetics of Neurodegenerative and Metabolic Diseases, Fondazione IRCCS, Istituto Neurologico Carlo Besta, Milan, Italy. mariotti.c@istituto-besta.it</nlm:affiliation><country xml:lang="fr">Italie</country><wicri:regionArea>SOSD Genetics of Neurodegenerative and Metabolic Diseases, Fondazione IRCCS, Istituto Neurologico Carlo Besta, Milan</wicri:regionArea></affiliation></author><author><name sortKey="Fancellu, Roberto" sort="Fancellu, Roberto" uniqKey="Fancellu R" first="Roberto" last="Fancellu">Roberto Fancellu</name></author><author><name sortKey="Caldarazzo, Serena" sort="Caldarazzo, Serena" uniqKey="Caldarazzo S" first="Serena" last="Caldarazzo">Serena Caldarazzo</name></author><author><name sortKey="Nanetti, Lorenzo" sort="Nanetti, Lorenzo" uniqKey="Nanetti L" first="Lorenzo" last="Nanetti">Lorenzo Nanetti</name></author><author><name sortKey="Di Bella, Daniela" sort="Di Bella, Daniela" uniqKey="Di Bella D" first="Daniela" last="Di Bella">Daniela Di Bella</name></author><author><name sortKey="Plumari, Massimo" sort="Plumari, Massimo" uniqKey="Plumari M" first="Massimo" last="Plumari">Massimo Plumari</name></author><author><name sortKey="Lauria, Giuseppe" sort="Lauria, Giuseppe" uniqKey="Lauria G" first="Giuseppe" last="Lauria">Giuseppe Lauria</name></author><author><name sortKey="Cappellini, Maria D" sort="Cappellini, Maria D" uniqKey="Cappellini M" first="Maria D" last="Cappellini">Maria D. Cappellini</name></author><author><name sortKey="Duca, Lorena" sort="Duca, Lorena" uniqKey="Duca L" first="Lorena" last="Duca">Lorena Duca</name></author><author><name sortKey="Solari, Alessandra" sort="Solari, Alessandra" uniqKey="Solari A" first="Alessandra" last="Solari">Alessandra Solari</name></author><author><name sortKey="Taroni, Franco" sort="Taroni, Franco" uniqKey="Taroni F" first="Franco" last="Taroni">Franco Taroni</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="2012" type="published">2012</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Adult</term><term>Antioxidants (therapeutic use)</term><term>Dose-Response Relationship, Drug</term><term>Double-Blind Method</term><term>Erythropoietin (therapeutic use)</term><term>Female</term><term>Friedreich Ataxia (drug therapy)</term><term>Friedreich Ataxia (metabolism)</term><term>Humans</term><term>Iron-Binding Proteins (metabolism)</term><term>Male</term><term>Recombinant Proteins (therapeutic use)</term><term>Statistics, Nonparametric</term><term>Time Factors</term><term>Treatment Outcome</term><term>Ubiquinone (analogs & derivatives)</term><term>Ubiquinone (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="metabolism" xml:lang="en"><term>Iron-Binding Proteins</term></keywords><keywords scheme="MESH" type="chemical" qualifier="therapeutic use" xml:lang="en"><term>Antioxidants</term><term>Erythropoietin</term><term>Recombinant Proteins</term><term>Ubiquinone</term></keywords><keywords scheme="MESH" qualifier="drug therapy" xml:lang="en"><term>Friedreich Ataxia</term></keywords><keywords scheme="MESH" qualifier="metabolism" xml:lang="en"><term>Friedreich Ataxia</term></keywords><keywords scheme="MESH" xml:lang="en"><term>Adult</term><term>Dose-Response Relationship, Drug</term><term>Double-Blind Method</term><term>Female</term><term>Humans</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">Friedreich ataxia is a rare disease caused by GAA-trinucleotide-repeat expansions in the frataxin gene, leading to marked reduction of qualitatively normal frataxin protein. Recently, human recombinant erythropoietin was reported to increase frataxin levels in patients with Friedreich ataxia.</div></front></TEI><pubmed><MedlineCitation Owner="NLM" Status="MEDLINE"><PMID Version="1">22411849</PMID><DateCreated><Year>2012</Year><Month>03</Month><Day>13</Day></DateCreated><DateCompleted><Year>2012</Year><Month>07</Month><Day>11</Day></DateCompleted><DateRevised><Year>2012</Year><Month>11</Month><Day>15</Day></DateRevised><Article PubModel="Print-Electronic"><Journal><ISSN IssnType="Electronic">1531-8257</ISSN><JournalIssue CitedMedium="Internet"><Volume>27</Volume><Issue>3</Issue><PubDate><Year>2012</Year><Month>Mar</Month></PubDate></JournalIssue><Title>Movement disorders : official journal of the Movement Disorder Society</Title><ISOAbbreviation>Mov. Disord.</ISOAbbreviation></Journal><ArticleTitle>Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial.</ArticleTitle><Pagination><MedlinePgn>446-9</MedlinePgn></Pagination><ELocationID EIdType="doi" ValidYN="Y">10.1002/mds.24066</ELocationID><Abstract><AbstractText Label="BACKGROUND" NlmCategory="BACKGROUND">Friedreich ataxia is a rare disease caused by GAA-trinucleotide-repeat expansions in the frataxin gene, leading to marked reduction of qualitatively normal frataxin protein. Recently, human recombinant erythropoietin was reported to increase frataxin levels in patients with Friedreich ataxia.</AbstractText><AbstractText Label="METHODS" NlmCategory="METHODS">We performed a 6-month, randomized placebo-controlled, double-blind, dose-response pilot trial to assess the safety and efficacy of erythropoietin in increasing frataxin levels. Sixteen adult patient with Friedreich ataxia were randomly assigned to erythropoietin (n = 11) or matching placebo (n = 5). All patients continued Idebenone treatment (5 mg/kg/day). Treatment consisted of a 6-month scaling-up phase, in which erythropoietin was administered intravenously at the following doses: 20,000 IU every 3 weeks, 40,000 IU every 3 weeks, and 40,000 IU every 2 weeks.</AbstractText><AbstractText Label="RESULTS" NlmCategory="RESULTS">Erythropoietin treatment was safe and well tolerated, but did not result in any significant hematological, clinical, or biochemical effects in Friedreich ataxia patients.</AbstractText><CopyrightInformation>Copyright © 2012 Movement Disorder Society.</CopyrightInformation></Abstract><AuthorList CompleteYN="Y"><Author ValidYN="Y"><LastName>Mariotti</LastName><ForeName>Caterina</ForeName><Initials>C</Initials><AffiliationInfo><Affiliation>SOSD Genetics of Neurodegenerative and Metabolic Diseases, Fondazione IRCCS, Istituto Neurologico Carlo Besta, Milan, Italy. mariotti.c@istituto-besta.it</Affiliation></AffiliationInfo></Author><Author ValidYN="Y"><LastName>Fancellu</LastName><ForeName>Roberto</ForeName><Initials>R</Initials></Author><Author ValidYN="Y"><LastName>Caldarazzo</LastName><ForeName>Serena</ForeName><Initials>S</Initials></Author><Author ValidYN="Y"><LastName>Nanetti</LastName><ForeName>Lorenzo</ForeName><Initials>L</Initials></Author><Author ValidYN="Y"><LastName>Di Bella</LastName><ForeName>Daniela</ForeName><Initials>D</Initials></Author><Author ValidYN="Y"><LastName>Plumari</LastName><ForeName>Massimo</ForeName><Initials>M</Initials></Author><Author ValidYN="Y"><LastName>Lauria</LastName><ForeName>Giuseppe</ForeName><Initials>G</Initials></Author><Author ValidYN="Y"><LastName>Cappellini</LastName><ForeName>Maria D</ForeName><Initials>MD</Initials></Author><Author ValidYN="Y"><LastName>Duca</LastName><ForeName>Lorena</ForeName><Initials>L</Initials></Author><Author ValidYN="Y"><LastName>Solari</LastName><ForeName>Alessandra</ForeName><Initials>A</Initials></Author><Author ValidYN="Y"><LastName>Taroni</LastName><ForeName>Franco</ForeName><Initials>F</Initials></Author></AuthorList><Language>eng</Language><PublicationTypeList><PublicationType UI="D016428">Journal Article</PublicationType><PublicationType UI="D016449">Randomized Controlled Trial</PublicationType><PublicationType UI="D013485">Research Support, Non-U.S. Gov't</PublicationType></PublicationTypeList><ArticleDate DateType="Electronic"><Year>2012</Year><Month>01</Month><Day>04</Day></ArticleDate></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="D000975">Antioxidants</NameOfSubstance></Chemical><Chemical><RegistryNumber>0</RegistryNumber><NameOfSubstance UI="D033862">Iron-Binding Proteins</NameOfSubstance></Chemical><Chemical><RegistryNumber>0</RegistryNumber><NameOfSubstance UI="D011994">Recombinant Proteins</NameOfSubstance></Chemical><Chemical><RegistryNumber>0</RegistryNumber><NameOfSubstance UI="C098527">frataxin</NameOfSubstance></Chemical><Chemical><RegistryNumber>11096-26-7</RegistryNumber><NameOfSubstance UI="D004921">Erythropoietin</NameOfSubstance></Chemical><Chemical><RegistryNumber>1339-63-5</RegistryNumber><NameOfSubstance UI="D014451">Ubiquinone</NameOfSubstance></Chemical><Chemical><RegistryNumber>HB6PN45W4J</RegistryNumber><NameOfSubstance UI="C036619">idebenone</NameOfSubstance></Chemical></ChemicalList><CitationSubset>IM</CitationSubset><MeshHeadingList><MeshHeading><DescriptorName MajorTopicYN="N" UI="D000328">Adult</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D000975">Antioxidants</DescriptorName><QualifierName MajorTopicYN="N" UI="Q000627">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D004305">Dose-Response Relationship, Drug</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D004311">Double-Blind Method</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D004921">Erythropoietin</DescriptorName><QualifierName MajorTopicYN="Y" UI="Q000627">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D005260">Female</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D005621">Friedreich Ataxia</DescriptorName><QualifierName MajorTopicYN="Y" UI="Q000188">drug therapy</QualifierName><QualifierName MajorTopicYN="Y" UI="Q000378">metabolism</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D006801">Humans</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D033862">Iron-Binding Proteins</DescriptorName><QualifierName MajorTopicYN="Y" UI="Q000378">metabolism</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D008297">Male</DescriptorName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D011994">Recombinant Proteins</DescriptorName><QualifierName MajorTopicYN="N" UI="Q000627">therapeutic use</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="N" UI="Q000031">analogs & derivatives</QualifierName><QualifierName MajorTopicYN="N" UI="Q000627">therapeutic use</QualifierName></MeshHeading><MeshHeading><DescriptorName MajorTopicYN="N" UI="D055815">Young Adult</DescriptorName></MeshHeading></MeshHeadingList></MedlineCitation><PubmedData><History><PubMedPubDate PubStatus="received"><Year>2011</Year><Month>4</Month><Day>1</Day></PubMedPubDate><PubMedPubDate PubStatus="revised"><Year>2011</Year><Month>10</Month><Day>26</Day></PubMedPubDate><PubMedPubDate PubStatus="accepted"><Year>2011</Year><Month>11</Month><Day>14</Day></PubMedPubDate><PubMedPubDate PubStatus="aheadofprint"><Year>2012</Year><Month>1</Month><Day>4</Day></PubMedPubDate><PubMedPubDate PubStatus="entrez"><Year>2012</Year><Month>3</Month><Day>14</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="pubmed"><Year>2012</Year><Month>3</Month><Day>14</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate><PubMedPubDate PubStatus="medline"><Year>2012</Year><Month>7</Month><Day>12</Day><Hour>6</Hour><Minute>0</Minute></PubMedPubDate></History><PublicationStatus>ppublish</PublicationStatus><ArticleIdList><ArticleId IdType="doi">10.1002/mds.24066</ArticleId><ArticleId IdType="pubmed">22411849</ArticleId></ArticleIdList></PubmedData></pubmed></record>Pour manipuler 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