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A randomized, phase 2 clinical trial of lithium carbonate in Machado-Joseph disease
Movement disorders, 2014-04, Vol.29 (4), p.568-573
Saute, Jonas Alex Morales
de Castilhos, Raphael Machado
Monte, Thais Lampert
Schumacher-Schuh, Artur Francisco
Donis, Karina Carvalho
D'Ávila, Rui
Souza, Gabriele Nunes
Russo, Aline Dutra
Furtado, Gabriel Vasata
Gheno, Tailise Conte
de Souza, Diogo Onofre Gomes
Portela, Luis Valmor Cruz
Saraiva-Pereira, Maria-Luiza
Camey, Suzi Alvez
Torman, Vanessa Bielefeld Leotti
de Mello Rieder, Carlos Roberto
Jardim, Laura Bannach
2014
Volltextzugriff (PDF)
Details
Autor(en) / Beteiligte
Saute, Jonas Alex Morales
de Castilhos, Raphael Machado
Monte, Thais Lampert
Schumacher-Schuh, Artur Francisco
Donis, Karina Carvalho
D'Ávila, Rui
Souza, Gabriele Nunes
Russo, Aline Dutra
Furtado, Gabriel Vasata
Gheno, Tailise Conte
de Souza, Diogo Onofre Gomes
Portela, Luis Valmor Cruz
Saraiva-Pereira, Maria-Luiza
Camey, Suzi Alvez
Torman, Vanessa Bielefeld Leotti
de Mello Rieder, Carlos Roberto
Jardim, Laura Bannach
Titel
A randomized, phase 2 clinical trial of lithium carbonate in Machado-Joseph disease
Ist Teil von
Movement disorders, 2014-04, Vol.29 (4), p.568-573
Ort / Verlag
United States: Blackwell Publishing Ltd
Erscheinungsjahr
2014
Quelle
MEDLINE
Beschreibungen/Notizen
ABSTRACT 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]). 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. 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). 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. © 2014 International Parkinson and Movement Disorder Society
Sprache
Englisch
Identifikatoren
ISSN: 0885-3185
eISSN: 1531-8257
DOI: 10.1002/mds.25803
Titel-ID: cdi_proquest_miscellaneous_1627984939
Format
–
Schlagworte
Adult
,
Ataxia
,
clinical trial
,
Confidence intervals
,
Double-Blind Method
,
Drug therapy
,
Enzyme Inhibitors - adverse effects
,
Enzyme Inhibitors - therapeutic use
,
Female
,
Humans
,
Lithium
,
Lithium Carbonate - adverse effects
,
Lithium Carbonate - therapeutic use
,
Machado-Joseph disease
,
Machado-Joseph Disease - drug therapy
,
Male
,
Middle Aged
,
Movement disorders
,
polyglutamine disorders
,
spinocerebellar ataxias
,
Treatment Outcome
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