Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial
Identifieur interne : 000100 ( Istex/Curation ); précédent : 000099; suivant : 000101Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial
Auteurs : Giovanna Zorzi [Italie] ; Federica Zibordi [Italie] ; Luisa Chiapparini [Italie] ; Enrico Bertini [Italie] ; Lidia Russo [Italie] ; Antonio Piga [Italie] ; Filomena Longo [Italie] ; Barbara Garavaglia [Italie] ; Domenico Aquino [Italie] ; Mario Savoiardo [Italie] ; Alessandra Solari [Italie] ; Nardo Nardocci [Italie]Source :
- 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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Disord.</title><idno type="ISSN">0885-3185</idno><idno type="eISSN">1531-8257</idno><imprint><publisher>Wiley Subscription Services, Inc., A Wiley Company</publisher><pubPlace>Hoboken</pubPlace><date type="published" when="2011-08-01">2011-08-01</date><biblScope unit="volume">26</biblScope><biblScope unit="issue">9</biblScope><biblScope unit="page" from="1755">1755</biblScope><biblScope unit="page" to="1759">1759</biblScope></imprint><idno type="ISSN">0885-3185</idno></series><idno type="istex">3D9AF621150D0AD4A867415A54A22BE317647D83</idno><idno type="DOI">10.1002/mds.23751</idno><idno type="ArticleID">MDS23751</idno></biblStruct></sourceDesc><seriesStmt><idno type="ISSN">0885-3185</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>neurodegeneration with brain iron accumulation; pantothenate kinase–associated neurodegeneration; deferiprone; magnetic resonance imaging; iron</term></keywords></textClass><langUsage><language ident="en">en</language></langUsage></profileDesc></teiHeader><front><div type="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.</div><div type="abstract">Nine patients (age range, 7–39 years) completed the study.</div><div type="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.</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></record>Pour manipuler ce document sous Unix (Dilib)
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