NESSCA Validation and Responsiveness of Several Rating Scales in Spinocerebellar Ataxia Type 2

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dc.contributor.authorMonte, Thais L.
dc.contributor.authorReckziegel, Estela R.
dc.contributor.authorAugustin, Marina C.
dc.contributor.authorSilva, Amanda S. P.
dc.contributor.authorLocks-Coelho, Lucas D.
dc.contributor.authorBarsottini, Orlando [UNIFESP]
dc.contributor.authorPedroso, Jose L. [UNIFESP]
dc.contributor.authorVargas, Fernando R.
dc.contributor.authorSaraiva-Pereira, Maria-Luiza
dc.contributor.authorLeotti, Vanessa Bielefeldt
dc.contributor.authorJardim, Laura Bannach
dc.date.accessioned2019-08-19T11:50:20Z
dc.date.available2019-08-19T11:50:20Z
dc.date.issued2017
dc.description.abstractSpinocerebellar ataxia type 2 (SCA2), caused by a CAG expansion (CAGexp) at ATXN2, has a complex clinical picture. While validated ataxia scales are available, comprehensive instruments to measure all SCA2 neurological manifestations are required. This study aims to validate the Neurological Examination Score for the assessment of Spinocerebellar Ataxias (NESSCA) to be used in SCA2 and to compare its responsiveness to those obtained with other instruments. NESSCA, SARA, SCAFI, and CCFS scales were applied in symptomatic SCA2 patients. Correlations were done with age at onset, disease duration, CAGexp, and between scales. Responsiveness was estimated by comparing deltas of stable to worse patients after 12 months, according to Patient Global Impression of change, and the area under the curve (AUC) of the Receiver Operating Characteristics curve of scores range. Eighty-eight evaluations (49 patients) were obtained. NESSCA had an even distribution and correlated with disease duration (r = 0.55), SARA (r = 0.63), and CAGexp (rho = 0.32): both explained 44% of NESSCA variance. Deltas (95% CI) after 1 year in stable and worse patients were only significantly different for SARA. NESSCA, SARA, SCAFI, and CCFS AUC were 0.63, 0.81, 0.49, and 0.48, respectively. NESSCA is valid to be used in SCA2. However, the only instrument that presented good responsiveness to change in 1 year was SARA. We suggest that NESSCA can be used as a secondary outcome in future trials in SCA2 due to the burden of neurological disabilities related to disease progression.en
dc.description.affiliationHosp Clin Porto Alegre, Serv Neurol, Porto Alegre, RS, Brazil
dc.description.affiliationUniv Fed Rio Grande do Sul, Programa Posgrad Ciencias Med, Porto Alegre, RS, Brazil
dc.description.affiliationUniv Fed Rio Grande do Sul, Fac Med, Porto Alegre, RS, Brazil
dc.description.affiliationUniv Fed São Paulo, Disciplina Neurol Clin, Setor Neurol Geral & Ataxias, UNIFESP,Escola Paulista Med, São Paulo, Brazil
dc.description.affiliationFundação Oswaldo Cruz, Lab Epidemiol Malformacoes Congenitas, Rio De Janeiro, Brazil
dc.description.affiliationUniv Fed Estado Rio de Janeiro, Dept Genet & Biol Mol, Rio De Janeiro, Brazil
dc.description.affiliationHosp Clin Porto Alegre, Serv Genet Med, Porto Alegre, RS, Brazil
dc.description.affiliationHosp Clin Porto Alegre, Lab Identificacao Genet, Porto Alegre, RS, Brazil
dc.description.affiliationUniv Fed Rio Grande do Sul, Dept Bioquim, Porto Alegre, RS, Brazil
dc.description.affiliationUniv Fed Rio Grande do Sul, Dept Matemat & Estat, Porto Alegre, RS, Brazil
dc.description.affiliationUniv Fed Rio Grande do Sul, Programa Posgrad Epidemiol, Porto Alegre, RS, Brazil
dc.description.affiliationUniv Fed Rio Grande do Sul, Dept Med Interna, Porto Alegre, RS, Brazil
dc.description.affiliationInst Nacl Genet Med Populac INAGEMP, Porto Alegre, RS, Brazil
dc.description.affiliationHosp Clin Porto Alegre, Med Genet Serv, Rua Ramiro Barcelos 2350, BR-90035903 Porto Alegre, RS, Brazil
dc.description.affiliationUnifespUniv Fed São Paulo, Disciplina Neurol Clin, Setor Neurol Geral & Ataxias, UNIFESP,Escola Paulista Med, São Paulo, Brazil
dc.description.sourceWeb of Science
dc.description.sponsorshipCNPq-Conselho Nacional de Desenvolvimento Científico e Tecnológico
dc.description.sponsorshipFIPE-HCPA-Fundo de Incentivo a Pesquisa do Hospital de Clinicas de Porto Alegre
dc.description.sponsorshipINAGEMP-Instituto Nacional de Genetica Medica Populacional
dc.description.sponsorshipFAPERGS
dc.description.sponsorshipCNPq
dc.description.sponsorshipIDCNPq: 78057/2012-1
dc.description.sponsorshipIDFIPE-HCPA: GPPG HCPA 12-0357
dc.description.sponsorshipIDFIPE-HCPA: GPPG HCPA 12-0396
dc.format.extent852-858
dc.identifierhttp://dx.doi.org/10.1007/s12311-017-0855-8
dc.identifier.citationCerebellum. New York, v. 16, n. 4, p. 852-858, 2017.
dc.identifier.doi10.1007/s12311-017-0855-8
dc.identifier.issn1473-4222
dc.identifier.urihttp://repositorio.unifesp.br/handle/11600/51516
dc.identifier.wosWOS:000405033000012
dc.language.isoeng
dc.publisherSpringer
dc.rightsinfo:eu-repo/semantics/restrictedAccess
dc.subjectNeurological Examination Score for Spinocerebellar Ataxiaen
dc.subjectNESSCAen
dc.subjectSARAen
dc.subjectSCAFIen
dc.subjectSCA2en
dc.subjectSpinocerebellar ataxia type 2en
dc.titleNESSCA Validation and Responsiveness of Several Rating Scales in Spinocerebellar Ataxia Type 2en
dc.typeinfo:eu-repo/semantics/article
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