Movement Disorders (revue)

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A randomized, phase 2 clinical trial of lithium carbonate in Machado-Joseph disease.

Identifieur interne : 000700 ( PubMed/Checkpoint ); précédent : 000699; suivant : 000701

A randomized, phase 2 clinical trial of lithium carbonate in Machado-Joseph disease.

Auteurs : Jonas Alex Morales Saute [Brésil] ; Raphael Machado De Castilhos ; Thais Lampert Monte ; Artur Francisco Schumacher-Schuh ; Karina Carvalho Donis ; Rui D'Ávila ; Gabriele Nunes Souza ; Aline Dutra Russo ; Gabriel Vasata Furtado ; Tailise Conte Gheno ; Diogo Onofre Gomes De Souza ; Luis Valmor Cruz Portela ; Maria-Luiza Saraiva-Pereira ; Suzi Alvez Camey ; Vanessa Bielefeld Leotti Torman ; Carlos Roberto De Mello Rieder ; Laura Bannach Jardim

Source :

RBID : pubmed:24399647

English descriptors

Abstract

Because lithium exerts neuroprotective effects in preclinical models of polyglutamine disorders, our objective was to assess the safety and efficacy of lithium carbonate (0.5-0.8 milliequivalents per liter) in patients with Machado-Joseph disease (spinocerebellar ataxia type 3 [MJD/SCA3]).

DOI: 10.1002/mds.25803
PubMed: 24399647


Affiliations:


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pubmed:24399647

Le document en format XML

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<div type="abstract" xml:lang="en">Because lithium exerts neuroprotective effects in preclinical models of polyglutamine disorders, our objective was to assess the safety and efficacy of lithium carbonate (0.5-0.8 milliequivalents per liter) in patients with Machado-Joseph disease (spinocerebellar ataxia type 3 [MJD/SCA3]).</div>
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<AbstractText Label="BACKGROUND" NlmCategory="BACKGROUND">Because lithium exerts neuroprotective effects in preclinical models of polyglutamine disorders, our objective was to assess the safety and efficacy of lithium carbonate (0.5-0.8 milliequivalents per liter) in patients with Machado-Joseph disease (spinocerebellar ataxia type 3 [MJD/SCA3]).</AbstractText>
<AbstractText Label="METHODS" NlmCategory="METHODS">For this phase 2, single-center, double-blind, parallel, placebo-controlled trial (ClinicalTrials.gov identifier NCT01096082), 62 patients who had MJD/SCA3 with a disease duration ≤10 years and an independent gait were randomly assigned (1:1) to receive either lithium or placebo.</AbstractText>
<AbstractText Label="RESULTS" NlmCategory="RESULTS">After 24 weeks, 169 adverse events were reported, including 50.3% in the lithium group (P = 1.00; primary safety outcome). Sixty patients (31 in the placebo group and 29 in the lithium group) were analyzed for efficacy (intention-to-treat analysis). Mean progression between groups did not differ according to scores on the Neurological Examination Score for the Assessment of Spinocerebellar Ataxia (NESSCA) after 48 weeks (-0.35; 95% confidence interval, -1.7 to 1.0; primary efficacy outcome). The lithium group exhibited minor progression on the PATA speech-rate (P = 0.002), the nondominant Click Test (P = 0.023), the Spinocerebellar Ataxia Functional Index (P = 0.003), and the Composite Cerebellar Functional Score (P = 0.029).</AbstractText>
<AbstractText Label="CONCLUSIONS" NlmCategory="CONCLUSIONS">Lithium was safe and well tolerated, but it had no effect on progression when measured using the NESSCA in patients with MJD/SCA3. This slowdown in secondary outcomes deserves further clarification.</AbstractText>
<CopyrightInformation>© 2014 International Parkinson and Movement Disorder Society.</CopyrightInformation>
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<name sortKey="De Souza, Diogo Onofre Gomes" sort="De Souza, Diogo Onofre Gomes" uniqKey="De Souza D" first="Diogo Onofre Gomes" last="De Souza">Diogo Onofre Gomes De Souza</name>
<name sortKey="Donis, Karina Carvalho" sort="Donis, Karina Carvalho" uniqKey="Donis K" first="Karina Carvalho" last="Donis">Karina Carvalho Donis</name>
<name sortKey="Furtado, Gabriel Vasata" sort="Furtado, Gabriel Vasata" uniqKey="Furtado G" first="Gabriel Vasata" last="Furtado">Gabriel Vasata Furtado</name>
<name sortKey="Gheno, Tailise Conte" sort="Gheno, Tailise Conte" uniqKey="Gheno T" first="Tailise Conte" last="Gheno">Tailise Conte Gheno</name>
<name sortKey="Jardim, Laura Bannach" sort="Jardim, Laura Bannach" uniqKey="Jardim L" first="Laura Bannach" last="Jardim">Laura Bannach Jardim</name>
<name sortKey="Monte, Thais Lampert" sort="Monte, Thais Lampert" uniqKey="Monte T" first="Thais Lampert" last="Monte">Thais Lampert Monte</name>
<name sortKey="Portela, Luis Valmor Cruz" sort="Portela, Luis Valmor Cruz" uniqKey="Portela L" first="Luis Valmor Cruz" last="Portela">Luis Valmor Cruz Portela</name>
<name sortKey="Russo, Aline Dutra" sort="Russo, Aline Dutra" uniqKey="Russo A" first="Aline Dutra" last="Russo">Aline Dutra Russo</name>
<name sortKey="Saraiva Pereira, Maria Luiza" sort="Saraiva Pereira, Maria Luiza" uniqKey="Saraiva Pereira M" first="Maria-Luiza" last="Saraiva-Pereira">Maria-Luiza Saraiva-Pereira</name>
<name sortKey="Schumacher Schuh, Artur Francisco" sort="Schumacher Schuh, Artur Francisco" uniqKey="Schumacher Schuh A" first="Artur Francisco" last="Schumacher-Schuh">Artur Francisco Schumacher-Schuh</name>
<name sortKey="Souza, Gabriele Nunes" sort="Souza, Gabriele Nunes" uniqKey="Souza G" first="Gabriele Nunes" last="Souza">Gabriele Nunes Souza</name>
<name sortKey="Torman, Vanessa Bielefeld Leotti" sort="Torman, Vanessa Bielefeld Leotti" uniqKey="Torman V" first="Vanessa Bielefeld Leotti" last="Torman">Vanessa Bielefeld Leotti Torman</name>
</noCountry>
<country name="Brésil">
<region name="Rio Grande do Sul">
<name sortKey="Saute, Jonas Alex Morales" sort="Saute, Jonas Alex Morales" uniqKey="Saute J" first="Jonas Alex Morales" last="Saute">Jonas Alex Morales Saute</name>
</region>
</country>
</tree>
</affiliations>
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