MovDisordV3, Main, Exploration, bibRecord, 001228

A Randomized Trial of Oral Betamethasone to Reduce Ataxia Symptoms in Ataxia Telangiectasia online video

Identifieur interne : 001228 ( Main/Exploration ); précédent : 001227; suivant : 001229

A Randomized Trial of Oral Betamethasone to Reduce Ataxia Symptoms in Ataxia Telangiectasia online video

Auteurs : Raffaella Zannolli [Italie] ; Sabrina Buoni [Italie] ; Gianni Betti [Italie] ; Sara Salvucci [Italie] ; Alessandro Plebani [Italie] ; Annarosa Soresina [Italie] ; Maria C. Pietrogrande [Italie] ; Silvana Martino [Italie] ; Vincenzo Leuzzi [Italie] ; Andrea Finocchi [Italie] ; Roberto Micheli [Italie] ; Livia Nicoletta Rossi [Italie] ; Alfredo Brusco [Italie] ; Filippo Misiani [Italie] ; Alberto Fois [Italie] ; Joseph Hayek [Italie] ; Colleen Kelly [États-Unis] ; Luciana Chessa [Italie]

Source :

Movement disorders [ 0885-3185 ] ; 2012.

RBID : Pascal:12-0369642

Descripteurs français

Pascal (Inist)
- Ataxie télangiectasie, Dégénérescence, Pathologie du système nerveux, Voie orale, Bétaméthasone, Ataxie cérébelleuse, Cervelet, Neurologie.
Wicri :
- topic : Neurologie.

English descriptors

KwdEn :
- Ataxia telangiectasia, Betamethasone, Cerebellar ataxia, Cerebellum, Degeneration, Nervous system diseases, Neurology, Oral administration.

Abstract

No controlled studies exist regarding the pharmaceutical reduction of ataxia symptoms in ataxia telangiectasia (A-T). In a multicenter, double-blind, randomized, placebo-controlled crossover trial, oral betamethasone (BETA) and placebo were compared in terms of their reduction of ataxia symptoms as assessed with the International Cooperative Ataxia Rating Scale (ICARS). In this study of 13 A-T children, betamethasone reduced the ICARS total score by a median of 13 points in the intent-to-treat population and 16 points in the per-protocol population (ie, median percent decreases of ataxia symptoms of 28% and 31 %, respectively). In conclusion, Oral betamethasone could be a promising therapy to relieve ataxia symptoms in A-T patients; however, long-term effectiveness and safety must be established.

Affiliations:

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Le document en format XML

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<sourceDesc><biblStruct><analytic><title xml:lang="en" level="a">A Randomized Trial of Oral Betamethasone to Reduce Ataxia Symptoms in Ataxia Telangiectasia online video</title>
<author><name sortKey="Zannolli, Raffaella" sort="Zannolli, Raffaella" uniqKey="Zannolli R" first="Raffaella" last="Zannolli">Raffaella Zannolli</name>
<affiliation wicri:level="1"><inist:fA14 i1="01"><s1>Child Neurology and Psychiatry Unit, Azienda Ospedaliera Universitaria Senese/University of Siena, Policlinico Le Scotte</s1>
<s2>Siena</s2>
<s3>ITA</s3>
<sZ>1 aut.</sZ>
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<author><name sortKey="Buoni, Sabrina" sort="Buoni, Sabrina" uniqKey="Buoni S" first="Sabrina" last="Buoni">Sabrina Buoni</name>
<affiliation wicri:level="1"><inist:fA14 i1="01"><s1>Child Neurology and Psychiatry Unit, Azienda Ospedaliera Universitaria Senese/University of Siena, Policlinico Le Scotte</s1>
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<author><name sortKey="Betti, Gianni" sort="Betti, Gianni" uniqKey="Betti G" first="Gianni" last="Betti">Gianni Betti</name>
<affiliation wicri:level="1"><inist:fA14 i1="02"><s1>Department of Quantitative Methods, University of Siena</s1>
<s2>Siena</s2>
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<author><name sortKey="Salvucci, Sara" sort="Salvucci, Sara" uniqKey="Salvucci S" first="Sara" last="Salvucci">Sara Salvucci</name>
<affiliation wicri:level="1"><inist:fA14 i1="02"><s1>Department of Quantitative Methods, University of Siena</s1>
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<author><name sortKey="Plebani, Alessandro" sort="Plebani, Alessandro" uniqKey="Plebani A" first="Alessandro" last="Plebani">Alessandro Plebani</name>
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<author><name sortKey="Soresina, Annarosa" sort="Soresina, Annarosa" uniqKey="Soresina A" first="Annarosa" last="Soresina">Annarosa Soresina</name>
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<author><name sortKey="Pietrogrande, Maria C" sort="Pietrogrande, Maria C" uniqKey="Pietrogrande M" first="Maria C." last="Pietrogrande">Maria C. Pietrogrande</name>
<affiliation wicri:level="1"><inist:fA14 i1="04"><s1>Department of Pediatrics, University of Milan, Fondazione IRCS Ca' Granda</s1>
<s2>Milano</s2>
<s3>ITA</s3>
<sZ>7 aut.</sZ>
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<author><name sortKey="Martino, Silvana" sort="Martino, Silvana" uniqKey="Martino S" first="Silvana" last="Martino">Silvana Martino</name>
<affiliation wicri:level="3"><inist:fA14 i1="05"><s1>Department of Pediatrics, University of Torino</s1>
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<s3>ITA</s3>
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<author><name sortKey="Leuzzi, Vincenzo" sort="Leuzzi, Vincenzo" uniqKey="Leuzzi V" first="Vincenzo" last="Leuzzi">Vincenzo Leuzzi</name>
<affiliation wicri:level="3"><inist:fA14 i1="06"><s1>Department of Child Neurology and Psychiatry, University of Roma La Sapienza</s1>
<s2>Rome</s2>
<s3>ITA</s3>
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<author><name sortKey="Finocchi, Andrea" sort="Finocchi, Andrea" uniqKey="Finocchi A" first="Andrea" last="Finocchi">Andrea Finocchi</name>
<affiliation wicri:level="3"><inist:fA14 i1="07"><s1>Department of Pediatrics, Children Hospital Bambino Gesù/University of Roma Tor Vergata</s1>
<s2>Rome</s2>
<s3>ITA</s3>
<sZ>10 aut.</sZ>
</inist:fA14>
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<placeName><settlement type="city">Rome</settlement>
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<author><name sortKey="Micheli, Roberto" sort="Micheli, Roberto" uniqKey="Micheli R" first="Roberto" last="Micheli">Roberto Micheli</name>
<affiliation wicri:level="1"><inist:fA14 i1="03"><s1>Department of Pediatrics, and Istituto di Medicina Molecolare A. Nocivelli, Spedali Civili and University of Brescia</s1>
<s2>Brescia</s2>
<s3>ITA</s3>
<sZ>5 aut.</sZ>
<sZ>6 aut.</sZ>
<sZ>11 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<wicri:noRegion>Brescia</wicri:noRegion>
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<author><name sortKey="Nicoletta Rossi, Livia" sort="Nicoletta Rossi, Livia" uniqKey="Nicoletta Rossi L" first="Livia" last="Nicoletta Rossi">Livia Nicoletta Rossi</name>
<affiliation wicri:level="1"><inist:fA14 i1="04"><s1>Department of Pediatrics, University of Milan, Fondazione IRCS Ca' Granda</s1>
<s2>Milano</s2>
<s3>ITA</s3>
<sZ>7 aut.</sZ>
<sZ>12 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<wicri:noRegion>Milano</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Brusco, Alfredo" sort="Brusco, Alfredo" uniqKey="Brusco A" first="Alfredo" last="Brusco">Alfredo Brusco</name>
<affiliation wicri:level="3"><inist:fA14 i1="08"><s1>Department of Genetics Biology and Biochemistry, University of Torino, and Medical Genetics Unit "San Giovanni Battista" Hospital</s1>
<s2>Genetics, Torino</s2>
<s3>ITA</s3>
<sZ>13 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<placeName><settlement type="city">Turin</settlement>
<region type="région" nuts="2">Piémont</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Misiani, Filippo" sort="Misiani, Filippo" uniqKey="Misiani F" first="Filippo" last="Misiani">Filippo Misiani</name>
<affiliation wicri:level="1"><inist:fA14 i1="09"><s1>U.O.C Farmaceutica, Azienda Ospedaliera Universitaria Senese, University of Siena, Policlinico Le Scotte</s1>
<s2>Siena</s2>
<s3>ITA</s3>
<sZ>14 aut.</sZ>
<sZ>15 aut.</sZ>
<sZ>16 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<wicri:noRegion>Siena</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Fois, Alberto" sort="Fois, Alberto" uniqKey="Fois A" first="Alberto" last="Fois">Alberto Fois</name>
<affiliation wicri:level="1"><inist:fA14 i1="09"><s1>U.O.C Farmaceutica, Azienda Ospedaliera Universitaria Senese, University of Siena, Policlinico Le Scotte</s1>
<s2>Siena</s2>
<s3>ITA</s3>
<sZ>14 aut.</sZ>
<sZ>15 aut.</sZ>
<sZ>16 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<wicri:noRegion>Siena</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Hayek, Joseph" sort="Hayek, Joseph" uniqKey="Hayek J" first="Joseph" last="Hayek">Joseph Hayek</name>
<affiliation wicri:level="1"><inist:fA14 i1="09"><s1>U.O.C Farmaceutica, Azienda Ospedaliera Universitaria Senese, University of Siena, Policlinico Le Scotte</s1>
<s2>Siena</s2>
<s3>ITA</s3>
<sZ>14 aut.</sZ>
<sZ>15 aut.</sZ>
<sZ>16 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<wicri:noRegion>Siena</wicri:noRegion>
</affiliation>
</author>
<author><name sortKey="Kelly, Colleen" sort="Kelly, Colleen" uniqKey="Kelly C" first="Colleen" last="Kelly">Colleen Kelly</name>
<affiliation wicri:level="2"><inist:fA14 i1="10"><s1>Kelly Statistical Consulting</s1>
<s2>Carlsbad, California</s2>
<s3>USA</s3>
<sZ>17 aut.</sZ>
</inist:fA14>
<country>États-Unis</country>
<placeName><region type="state">Californie</region>
</placeName>
</affiliation>
</author>
<author><name sortKey="Chessa, Luciana" sort="Chessa, Luciana" uniqKey="Chessa L" first="Luciana" last="Chessa">Luciana Chessa</name>
<affiliation wicri:level="3"><inist:fA14 i1="11"><s1>Medical Genetic Unit, II School of Medicine, University La Sapienza</s1>
<s2>Rome</s2>
<s3>ITA</s3>
<sZ>18 aut.</sZ>
</inist:fA14>
<country>Italie</country>
<placeName><settlement type="city">Rome</settlement>
<region nuts="2">Latium</region>
</placeName>
</affiliation>
</author>
</analytic>
<series><title level="j" type="main">Movement disorders</title>
<title level="j" type="abbreviated">Mov. disord.</title>
<idno type="ISSN">0885-3185</idno>
<imprint><date when="2012">2012</date>
</imprint>
</series>
</biblStruct>
</sourceDesc>
<seriesStmt><title level="j" type="main">Movement disorders</title>
<title level="j" type="abbreviated">Mov. disord.</title>
<idno type="ISSN">0885-3185</idno>
</seriesStmt>
</fileDesc>
<profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Ataxia telangiectasia</term>
<term>Betamethasone</term>
<term>Cerebellar ataxia</term>
<term>Cerebellum</term>
<term>Degeneration</term>
<term>Nervous system diseases</term>
<term>Neurology</term>
<term>Oral administration</term>
</keywords>
<keywords scheme="Pascal" xml:lang="fr"><term>Ataxie télangiectasie</term>
<term>Dégénérescence</term>
<term>Pathologie du   système nerveux</term>
<term>Voie orale</term>
<term>Bétaméthasone</term>
<term>Ataxie cérébelleuse</term>
<term>Cervelet</term>
<term>Neurologie</term>
</keywords>
<keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Neurologie</term>
</keywords>
</textClass>
</profileDesc>
</teiHeader>
<front><div type="abstract" xml:lang="en">No controlled studies exist regarding the pharmaceutical reduction of ataxia symptoms in ataxia telangiectasia (A-T). In a multicenter, double-blind, randomized, placebo-controlled crossover trial, oral betamethasone (BETA) and placebo were compared in terms of their reduction of ataxia symptoms as assessed with the International Cooperative Ataxia Rating Scale (ICARS). In this study of 13 A-T children, betamethasone reduced the ICARS total score by a median of 13 points in the intent-to-treat population and 16 points in the per-protocol population (ie, median percent decreases of ataxia symptoms of 28% and 31 %, respectively). In conclusion, Oral betamethasone could be a promising therapy to relieve ataxia symptoms in A-T patients; however, long-term effectiveness and safety must be established.</div>
</front>
</TEI>
<affiliations><list><country><li>Italie</li>
<li>États-Unis</li>
</country>
<region><li>Californie</li>
<li>Latium</li>
<li>Piémont</li>
</region>
<settlement><li>Rome</li>
<li>Turin</li>
</settlement>
</list>
<tree><country name="Italie"><noRegion><name sortKey="Zannolli, Raffaella" sort="Zannolli, Raffaella" uniqKey="Zannolli R" first="Raffaella" last="Zannolli">Raffaella Zannolli</name>
</noRegion>
<name sortKey="Betti, Gianni" sort="Betti, Gianni" uniqKey="Betti G" first="Gianni" last="Betti">Gianni Betti</name>
<name sortKey="Brusco, Alfredo" sort="Brusco, Alfredo" uniqKey="Brusco A" first="Alfredo" last="Brusco">Alfredo Brusco</name>
<name sortKey="Buoni, Sabrina" sort="Buoni, Sabrina" uniqKey="Buoni S" first="Sabrina" last="Buoni">Sabrina Buoni</name>
<name sortKey="Chessa, Luciana" sort="Chessa, Luciana" uniqKey="Chessa L" first="Luciana" last="Chessa">Luciana Chessa</name>
<name sortKey="Finocchi, Andrea" sort="Finocchi, Andrea" uniqKey="Finocchi A" first="Andrea" last="Finocchi">Andrea Finocchi</name>
<name sortKey="Fois, Alberto" sort="Fois, Alberto" uniqKey="Fois A" first="Alberto" last="Fois">Alberto Fois</name>
<name sortKey="Hayek, Joseph" sort="Hayek, Joseph" uniqKey="Hayek J" first="Joseph" last="Hayek">Joseph Hayek</name>
<name sortKey="Leuzzi, Vincenzo" sort="Leuzzi, Vincenzo" uniqKey="Leuzzi V" first="Vincenzo" last="Leuzzi">Vincenzo Leuzzi</name>
<name sortKey="Martino, Silvana" sort="Martino, Silvana" uniqKey="Martino S" first="Silvana" last="Martino">Silvana Martino</name>
<name sortKey="Micheli, Roberto" sort="Micheli, Roberto" uniqKey="Micheli R" first="Roberto" last="Micheli">Roberto Micheli</name>
<name sortKey="Misiani, Filippo" sort="Misiani, Filippo" uniqKey="Misiani F" first="Filippo" last="Misiani">Filippo Misiani</name>
<name sortKey="Nicoletta Rossi, Livia" sort="Nicoletta Rossi, Livia" uniqKey="Nicoletta Rossi L" first="Livia" last="Nicoletta Rossi">Livia Nicoletta Rossi</name>
<name sortKey="Pietrogrande, Maria C" sort="Pietrogrande, Maria C" uniqKey="Pietrogrande M" first="Maria C." last="Pietrogrande">Maria C. Pietrogrande</name>
<name sortKey="Plebani, Alessandro" sort="Plebani, Alessandro" uniqKey="Plebani A" first="Alessandro" last="Plebani">Alessandro Plebani</name>
<name sortKey="Salvucci, Sara" sort="Salvucci, Sara" uniqKey="Salvucci S" first="Sara" last="Salvucci">Sara Salvucci</name>
<name sortKey="Soresina, Annarosa" sort="Soresina, Annarosa" uniqKey="Soresina A" first="Annarosa" last="Soresina">Annarosa Soresina</name>
</country>
<country name="États-Unis"><region name="Californie"><name sortKey="Kelly, Colleen" sort="Kelly, Colleen" uniqKey="Kelly C" first="Colleen" last="Kelly">Colleen Kelly</name>
</region>
</country>
</tree>
</affiliations>
</record>

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	Movement Disorders (revue)
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Movement Disorders (revue)

A Randomized Trial of Oral Betamethasone to Reduce Ataxia Symptoms in Ataxia Telangiectasia online video

A Randomized Trial of Oral Betamethasone to Reduce Ataxia Symptoms in Ataxia Telangiectasia online video

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