Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial
Identifieur interne : 000168 ( Istex/Corpus ); précédent : 000167; suivant : 000169Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial
Auteurs : Giovanna Zorzi ; Federica Zibordi ; Luisa Chiapparini ; Enrico Bertini ; Lidia Russo ; Antonio Piga ; Filomena Longo ; Barbara Garavaglia ; Domenico Aquino ; Mario Savoiardo ; Alessandra Solari ; Nardo NardocciSource :
- Movement Disorders [ 0885-3185 ] ; 2011-08-01.
English descriptors
Abstract
The safety and efficacy of the oral iron‐chelating agent deferiprone on magnetic resonance pallida iron concentration and on clinical status were investigated in 10 patients affected by pantothenate kinase–associated neurodegeneration.
Nine patients (age range, 7–39 years) completed the study.
A significant median reduction in globus pallidus iron content as assessed by T2* relaxometry (and calculated R2* maps; P = .008) was observed at the end of the study. None of the patients demonstrated a change in clinical status as assessed by the Burke‐Fahn and Marsden Dystonia Rating scales and by a health‐related quality‐of‐life scale. Deferiprone was well tolerated, and no serious adverse events occurred.
Future trials assessing the clinical efficacy of chelating therapy should consider early symptomatic patients and a longer treatment period. © 2011 MovementDisorder Society
Url:
DOI: 10.1002/mds.23751
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ISTEX:3D9AF621150D0AD4A867415A54A22BE317647D83Le document en format XML
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None of the patients demonstrated a change in clinical status as assessed by the Burke‐Fahn and Marsden Dystonia Rating scales and by a health‐related quality‐of‐life scale. Deferiprone was well tolerated, and no serious adverse events occurred.</div><div type="abstract">Future trials assessing the clinical efficacy of chelating therapy should consider early symptomatic patients and a longer treatment period. © 2011 MovementDisorder Society</div></front></TEI><istex><corpusName>wiley</corpusName><author><json:item><name>Giovanna Zorzi MD</name><affiliations><json:string>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string></affiliations></json:item><json:item><name>Federica Zibordi MD</name><affiliations><json:string>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string></affiliations></json:item><json:item><name>Luisa Chiapparini MD</name><affiliations><json:string>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string></affiliations></json:item><json:item><name>Enrico Bertini MD</name><affiliations><json:string>Unit of Molecular Medicine for Neuromuscular and Neurodegenerative Disorders, Ospedale Bambino Gesù, Roma, Italy</json:string></affiliations></json:item><json:item><name>Lidia Russo MD</name><affiliations><json:string>Unit of Oncology and Hematology, Ospedale Bambino Gesù, Roma, Italy</json:string></affiliations></json:item><json:item><name>Antonio Piga MD</name><affiliations><json:string>Unit of Pediatrics, Department of Clinical and Biological Sciences, University of Torino, Torino, Italy</json:string></affiliations></json:item><json:item><name>Filomena Longo MD</name><affiliations><json:string>Unit of Pediatrics, Department of Clinical and Biological Sciences, University of Torino, Torino, Italy</json:string></affiliations></json:item><json:item><name>Barbara Garavaglia PhD</name><affiliations><json:string>Neurogenetics, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string></affiliations></json:item><json:item><name>Domenico Aquino MD</name><affiliations><json:string>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string></affiliations></json:item><json:item><name>Mario Savoiardo MD</name><affiliations><json:string>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string></affiliations></json:item><json:item><name>Alessandra Solari MD</name><affiliations><json:string>Neuroepidemiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string></affiliations></json:item><json:item><name>Nardo Nardocci MD</name><affiliations><json:string>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</json:string><json:string>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, 20133 Milan, Italy</json:string></affiliations></json:item></author><subject><json:item><lang><json:string>eng</json:string></lang><value>neurodegeneration with brain iron accumulation; pantothenate kinase–associated neurodegeneration; deferiprone; magnetic resonance imaging; iron</value></json:item></subject><articleId><json:string>MDS23751</json:string></articleId><language><json:string>eng</json:string></language><originalGenre><json:string>article</json:string></originalGenre><qualityIndicators><score>5.36</score><pdfVersion>1.3</pdfVersion><pdfPageSize>612 x 810 pts</pdfPageSize><refBibsNative>true</refBibsNative><abstractCharCount>235</abstractCharCount><pdfWordCount>23617</pdfWordCount><pdfCharCount>155136</pdfCharCount><pdfPageCount>39</pdfPageCount><abstractWordCount>30</abstractWordCount></qualityIndicators><title>Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial</title><genre><json:string>article</json:string></genre><host><volume>26</volume><publisherId><json:string>MDS</json:string></publisherId><pages><total>5</total><last>1759</last><first>1755</first></pages><issn><json:string>0885-3185</json:string></issn><issue>9</issue><subject><json:item><value>Brief Report</value></json:item></subject><genre><json:string>journal</json:string></genre><language><json:string>unknown</json:string></language><eissn><json:string>1531-8257</json:string></eissn><title>Movement Disorders</title><doi><json:string>10.1002/(ISSN)1531-8257</json:string></doi></host><categories><wos><json:string>science</json:string><json:string>clinical neurology</json:string></wos><scienceMetrix><json:string>health sciences</json:string><json:string>clinical medicine</json:string><json:string>neurology & neurosurgery</json:string></scienceMetrix></categories><publicationDate>2011</publicationDate><copyrightDate>2011</copyrightDate><doi><json:string>10.1002/mds.23751</json:string></doi><id>3D9AF621150D0AD4A867415A54A22BE317647D83</id><score>0.105274945</score><fulltext><json:item><extension>pdf</extension><original>true</original><mimetype>application/pdf</mimetype><uri>https://api.istex.fr/document/3D9AF621150D0AD4A867415A54A22BE317647D83/fulltext/pdf</uri></json:item><json:item><extension>zip</extension><original>false</original><mimetype>application/zip</mimetype><uri>https://api.istex.fr/document/3D9AF621150D0AD4A867415A54A22BE317647D83/fulltext/zip</uri></json:item><istex:fulltextTEI uri="https://api.istex.fr/document/3D9AF621150D0AD4A867415A54A22BE317647D83/fulltext/tei"><teiHeader><fileDesc><titleStmt><title level="a" type="main" xml:lang="en">Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial</title></titleStmt><publicationStmt><authority>ISTEX</authority><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><pubPlace>Hoboken</pubPlace><availability><p>Copyright © 2011 Movement Disorder Society</p></availability><date>2011</date></publicationStmt><notesStmt><note type="content">*Relevant conflicts of interest/financial disclosures: Nothing to report. Full financial disclosures and author roles may be found in the online version of this article.</note></notesStmt><sourceDesc><biblStruct type="inbook"><analytic><title level="a" type="main" xml:lang="en">Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial</title><author xml:id="author-1"><persName><forename type="first">Giovanna</forename><surname>Zorzi</surname></persName><roleName type="degree">MD</roleName><note type="biography">Giovanna Zorzi and Federica Zibordi contributed equally to this work.</note><affiliation>Giovanna Zorzi and Federica Zibordi contributed equally to this work.</affiliation><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation></author><author xml:id="author-2"><persName><forename type="first">Federica</forename><surname>Zibordi</surname></persName><roleName type="degree">MD</roleName><note type="biography">Giovanna Zorzi and Federica Zibordi contributed equally to this work.</note><affiliation>Giovanna Zorzi and Federica Zibordi contributed equally to this work.</affiliation><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation></author><author xml:id="author-3"><persName><forename type="first">Luisa</forename><surname>Chiapparini</surname></persName><roleName type="degree">MD</roleName><affiliation>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation></author><author xml:id="author-4"><persName><forename type="first">Enrico</forename><surname>Bertini</surname></persName><roleName type="degree">MD</roleName><affiliation>Unit of Molecular Medicine for Neuromuscular and Neurodegenerative Disorders, Ospedale Bambino Gesù, Roma, Italy</affiliation></author><author xml:id="author-5"><persName><forename type="first">Lidia</forename><surname>Russo</surname></persName><roleName type="degree">MD</roleName><affiliation>Unit of Oncology and Hematology, Ospedale Bambino Gesù, Roma, Italy</affiliation></author><author xml:id="author-6"><persName><forename type="first">Antonio</forename><surname>Piga</surname></persName><roleName type="degree">MD</roleName><affiliation>Unit of Pediatrics, Department of Clinical and Biological Sciences, University of Torino, Torino, Italy</affiliation></author><author xml:id="author-7"><persName><forename type="first">Filomena</forename><surname>Longo</surname></persName><roleName type="degree">MD</roleName><affiliation>Unit of Pediatrics, Department of Clinical and Biological Sciences, University of Torino, Torino, Italy</affiliation></author><author xml:id="author-8"><persName><forename type="first">Barbara</forename><surname>Garavaglia</surname></persName><roleName type="degree">PhD</roleName><affiliation>Neurogenetics, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation></author><author xml:id="author-9"><persName><forename type="first">Domenico</forename><surname>Aquino</surname></persName><roleName type="degree">MD</roleName><affiliation>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation></author><author xml:id="author-10"><persName><forename type="first">Mario</forename><surname>Savoiardo</surname></persName><roleName type="degree">MD</roleName><affiliation>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation></author><author xml:id="author-11"><persName><forename type="first">Alessandra</forename><surname>Solari</surname></persName><roleName type="degree">MD</roleName><affiliation>Neuroepidemiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation></author><author xml:id="author-12"><persName><forename type="first">Nardo</forename><surname>Nardocci</surname></persName><roleName type="degree">MD</roleName><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, 20133 Milan, Italy</affiliation></author></analytic><monogr><title level="j">Movement Disorders</title><title level="j" type="abbrev">Mov. 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None of the patients demonstrated a change in clinical status as assessed by the Burke‐Fahn and Marsden Dystonia Rating scales and by a health‐related quality‐of‐life scale. Deferiprone was well tolerated, and no serious adverse events occurred.</p></abstract><abstract><p>Future trials assessing the clinical efficacy of chelating therapy should consider early symptomatic patients and a longer treatment period. © 2011 MovementDisorder Society</p></abstract><textClass xml:lang="en"><keywords scheme="keyword"><list><head>keywords</head><item><term>neurodegeneration with brain iron accumulation; pantothenate kinase–associated neurodegeneration; deferiprone; magnetic resonance imaging; iron</term></item></list></keywords></textClass><textClass><keywords scheme="Journal Subject"><list><head>article-category</head><item><term>Brief Report</term></item></list></keywords></textClass></profileDesc><revisionDesc><change when="2010-09-18">Received</change><change when="2011-03-15">Registration</change><change when="2011-08-01">Published</change></revisionDesc></teiHeader></istex:fulltextTEI><json:item><extension>txt</extension><original>false</original><mimetype>text/plain</mimetype><uri>https://api.istex.fr/document/3D9AF621150D0AD4A867415A54A22BE317647D83/fulltext/txt</uri></json:item></fulltext><metadata><istex:metadataXml wicri:clean="Wiley, elements deleted: body"><istex:xmlDeclaration>version="1.0" encoding="UTF-8" standalone="yes"</istex:xmlDeclaration><istex:document><component version="2.0" type="serialArticle" xml:lang="en"><header><publicationMeta level="product"><publisherInfo><publisherName>Wiley Subscription Services, Inc., A Wiley Company</publisherName><publisherLoc>Hoboken</publisherLoc></publisherInfo><doi registered="yes">10.1002/(ISSN)1531-8257</doi><issn type="print">0885-3185</issn><issn type="electronic">1531-8257</issn><idGroup><id type="product" value="MDS"></id></idGroup><titleGroup><title type="main" xml:lang="en" sort="MOVEMENT DISORDERS">Movement Disorders</title><title type="short">Mov. 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date="2011-03-15"></event><event type="xmlConverted" agent="Converter:JWSART34_TO_WML3G version:3.0.1 mode:FullText" date="2011-12-16"></event><event type="publishedOnlineEarlyUnpaginated" date="2011-05-06"></event><event type="publishedOnlineFinalForm" date="2011-08-09"></event><event type="firstOnline" date="2011-05-06"></event><event type="xmlConverted" agent="Converter:WILEY_ML3G_TO_WILEY_ML3GV2 version:3.8.8" date="2014-02-02"></event><event type="xmlConverted" agent="Converter:WML3G_To_WML3G version:4.1.7 mode:FullText,remove_FC" date="2014-10-31"></event></eventGroup><numberingGroup><numbering type="pageFirst">1755</numbering><numbering type="pageLast">1759</numbering></numberingGroup><correspondenceTo>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, 20133 Milan, Italy</correspondenceTo><linkGroup><link type="toTypesetVersion" href="file:MDS.MDS23751.pdf"></link></linkGroup></publicationMeta><contentMeta><countGroup><count type="figureTotal" number="1"></count><count type="tableTotal" number="2"></count><count type="referenceTotal" number="22"></count><count type="wordTotal" number="3350"></count></countGroup><titleGroup><title type="main" xml:lang="en">Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial<link href="#fn2"></link></title><title type="short" xml:lang="en">Chelating Therapy in PKAN</title></titleGroup><creators><creator xml:id="au1" creatorRole="author" affiliationRef="#af1" noteRef="#fn1"><personName><givenNames>Giovanna</givenNames><familyName>Zorzi</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au2" creatorRole="author" affiliationRef="#af1" noteRef="#fn1"><personName><givenNames>Federica</givenNames><familyName>Zibordi</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au3" creatorRole="author" affiliationRef="#af2"><personName><givenNames>Luisa</givenNames><familyName>Chiapparini</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au4" creatorRole="author" affiliationRef="#af3"><personName><givenNames>Enrico</givenNames><familyName>Bertini</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au5" creatorRole="author" affiliationRef="#af4"><personName><givenNames>Lidia</givenNames><familyName>Russo</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au6" creatorRole="author" affiliationRef="#af5"><personName><givenNames>Antonio</givenNames><familyName>Piga</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au7" creatorRole="author" affiliationRef="#af5"><personName><givenNames>Filomena</givenNames><familyName>Longo</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au8" creatorRole="author" affiliationRef="#af6"><personName><givenNames>Barbara</givenNames><familyName>Garavaglia</familyName><degrees>PhD</degrees></personName></creator><creator xml:id="au9" creatorRole="author" affiliationRef="#af2"><personName><givenNames>Domenico</givenNames><familyName>Aquino</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au10" creatorRole="author" affiliationRef="#af2"><personName><givenNames>Mario</givenNames><familyName>Savoiardo</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au11" creatorRole="author" affiliationRef="#af7"><personName><givenNames>Alessandra</givenNames><familyName>Solari</familyName><degrees>MD</degrees></personName></creator><creator xml:id="au12" creatorRole="author" affiliationRef="#af1" corresponding="yes"><personName><givenNames>Nardo</givenNames><familyName>Nardocci</familyName><degrees>MD</degrees></personName><contactDetails><email normalForm="nnardocci@istituto-besta.it">nnardocci@istituto‐besta.it</email></contactDetails></creator></creators><affiliationGroup><affiliation xml:id="af1" countryCode="IT" type="organization"><unparsedAffiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</unparsedAffiliation></affiliation><affiliation xml:id="af2" countryCode="IT" type="organization"><unparsedAffiliation>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</unparsedAffiliation></affiliation><affiliation xml:id="af3" countryCode="IT" type="organization"><unparsedAffiliation>Unit of Molecular Medicine for Neuromuscular and Neurodegenerative Disorders, Ospedale Bambino Gesù, Roma, Italy</unparsedAffiliation></affiliation><affiliation xml:id="af4" countryCode="IT" type="organization"><unparsedAffiliation>Unit of Oncology and Hematology, Ospedale Bambino Gesù, Roma, Italy</unparsedAffiliation></affiliation><affiliation xml:id="af5" countryCode="IT" type="organization"><unparsedAffiliation>Unit of Pediatrics, Department of Clinical and Biological Sciences, University of Torino, Torino, Italy</unparsedAffiliation></affiliation><affiliation xml:id="af6" countryCode="IT" type="organization"><unparsedAffiliation>Neurogenetics, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</unparsedAffiliation></affiliation><affiliation xml:id="af7" countryCode="IT" type="organization"><unparsedAffiliation>Neuroepidemiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</unparsedAffiliation></affiliation></affiliationGroup><keywordGroup xml:lang="en" type="author"><keyword xml:id="kwd1">neurodegeneration with brain iron accumulation; pantothenate kinase–associated neurodegeneration; deferiprone; magnetic resonance imaging; iron</keyword></keywordGroup><abstractGroup><abstract type="main" xml:lang="en"><title type="main">Abstract</title><section xml:id="abs1-1"><title type="main">Background:</title><p>The safety and efficacy of the oral iron‐chelating agent deferiprone on magnetic resonance pallida iron concentration and on clinical status were investigated in 10 patients affected by pantothenate kinase–associated neurodegeneration.</p></section><section xml:id="abs1-2"><title type="main">Methods:</title><p>Nine patients (age range, 7–39 years) completed the study.</p></section><section xml:id="abs1-3"><title type="main">Results:</title><p>A significant median reduction in globus pallidus iron content as assessed by T2* relaxometry (and calculated R2* maps; <i>P</i> = .008) was observed at the end of the study. None of the patients demonstrated a change in clinical status as assessed by the Burke‐Fahn and Marsden Dystonia Rating scales and by a health‐related quality‐of‐life scale. Deferiprone was well tolerated, and no serious adverse events occurred.</p></section><section xml:id="abs1-4"><title type="main">Conclusions:</title><p>Future trials assessing the clinical efficacy of chelating therapy should consider early symptomatic patients and a longer treatment period. © 2011 <i>Movement</i>Disorder Society</p></section></abstract></abstractGroup></contentMeta><noteGroup><note xml:id="fn2"><p><b>Relevant conflicts of interest/financial disclosures</b>: Nothing to report.</p><p>Full financial disclosures and author roles may be found in the online version of this article.</p></note><note xml:id="fn1"><p>Giovanna Zorzi and Federica Zibordi contributed equally to this work.</p></note></noteGroup></header></component></istex:document></istex:metadataXml><mods version="3.6"><titleInfo lang="en"><title>Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial</title></titleInfo><titleInfo type="abbreviated" lang="en"><title>Chelating Therapy in PKAN</title></titleInfo><titleInfo type="alternative" contentType="CDATA" lang="en"><title>Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial</title></titleInfo><name type="personal"><namePart type="given">Giovanna</namePart><namePart type="family">Zorzi</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation><description>Giovanna Zorzi and Federica Zibordi contributed equally to this work.</description><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Federica</namePart><namePart type="family">Zibordi</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation><description>Giovanna Zorzi and Federica Zibordi contributed equally to this work.</description><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Luisa</namePart><namePart type="family">Chiapparini</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Neuroradiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Enrico</namePart><namePart type="family">Bertini</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Unit of Molecular Medicine for Neuromuscular and Neurodegenerative Disorders, Ospedale Bambino Gesù, Roma, Italy</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Lidia</namePart><namePart type="family">Russo</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Unit of Oncology and Hematology, Ospedale Bambino Gesù, Roma, Italy</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Antonio</namePart><namePart type="family">Piga</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Unit of Pediatrics, Department of Clinical and Biological Sciences, University of Torino, Torino, Italy</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Filomena</namePart><namePart type="family">Longo</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Unit of Pediatrics, Department of Clinical and Biological Sciences, University of Torino, Torino, Italy</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Barbara</namePart><namePart type="family">Garavaglia</namePart><namePart 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type="termsOfAddress">MD</namePart><affiliation>Neuroepidemiology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation><role><roleTerm type="text">author</roleTerm></role></name><name type="personal"><namePart type="given">Nardo</namePart><namePart type="family">Nardocci</namePart><namePart type="termsOfAddress">MD</namePart><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy</affiliation><affiliation>Unit of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Via Celoria 11, 20133 Milan, Italy</affiliation><role><roleTerm type="text">author</roleTerm></role></name><typeOfResource>text</typeOfResource><genre type="article" displayLabel="article"></genre><originInfo><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><place><placeTerm type="text">Hoboken</placeTerm></place><dateIssued encoding="w3cdtf">2011-08-01</dateIssued><dateCaptured encoding="w3cdtf">2010-09-18</dateCaptured><dateValid encoding="w3cdtf">2011-03-15</dateValid><copyrightDate encoding="w3cdtf">2011</copyrightDate></originInfo><language><languageTerm type="code" authority="rfc3066">en</languageTerm><languageTerm type="code" authority="iso639-2b">eng</languageTerm></language><physicalDescription><internetMediaType>text/html</internetMediaType><extent unit="figures">1</extent><extent unit="tables">2</extent><extent unit="references">22</extent><extent unit="words">3350</extent></physicalDescription><abstract>The safety and efficacy of the oral iron‐chelating agent deferiprone on magnetic resonance pallida iron concentration and on clinical status were investigated in 10 patients affected by pantothenate kinase–associated neurodegeneration.</abstract><abstract>Nine patients (age range, 7–39 years) completed the study.</abstract><abstract>A significant median reduction in globus pallidus iron content as assessed by T2* relaxometry (and calculated R2* maps; P = .008) was observed at the end of the study. None of the patients demonstrated a change in clinical status as assessed by the Burke‐Fahn and Marsden Dystonia Rating scales and by a health‐related quality‐of‐life scale. Deferiprone was well tolerated, and no serious adverse events occurred.</abstract><abstract>Future trials assessing the clinical efficacy of chelating therapy should consider early symptomatic patients and a longer treatment period. © 2011 MovementDisorder Society</abstract><note type="content">*Relevant conflicts of interest/financial disclosures: Nothing to report. Full financial disclosures and author roles may be found in the online version of this article.</note><subject lang="en"><genre>keywords</genre><topic>neurodegeneration with brain iron accumulation; pantothenate kinase–associated neurodegeneration; deferiprone; magnetic resonance imaging; iron</topic></subject><relatedItem type="host"><titleInfo><title>Movement Disorders</title></titleInfo><titleInfo type="abbreviated"><title>Mov. 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