Your browser doesn't support javascript.
loading
The CLN3 Disease Staging System: A new tool for clinical research in Batten disease.
Masten, Margaux C; Williams, Justin D; Vermilion, Jennifer; Adams, Heather R; Vierhile, Amy; Collins, Alyssa; Marshall, Frederick J; Augustine, Erika F; Mink, Jonathan W.
Afiliação
  • Masten MC; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Williams JD; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Vermilion J; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Adams HR; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Vierhile A; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Collins A; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Marshall FJ; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Augustine EF; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
  • Mink JW; From the Departments of Neurology (M.C.M., J.D.W., J.V., H.R.A., A.V., A.C., F.J.M., E.F.A., J.W.M.), Neuroscience (J.W.M.), and Pediatrics (J.V., H.R.A., E.F.A., J.W.M.) and Center for Health and Technology (E.F.A.), University of Rochester School of Medicine and Dentistry; and University of Roches
Neurology ; 94(23): e2436-e2440, 2020 06 09.
Article em En | MEDLINE | ID: mdl-32300063
OBJECTIVE: To develop a disease-specific staging system for CLN3 disease and to test the hypothesis that salient and discrete clinical features of CLN3 disease may be used to define disease stages by analyzed data from an 18-year-long natural history study. METHODS: A proposed staging system, the CLN3 Staging System (CLN3SS), was based on salient and clinically meaningful endpoints. The relationships between stage and age, stage and Unified Batten Disease Rating Scale (UBDRS) physical severity score, and stage and UBDRS capability impairment subscale scores were determined. We used t tests to determine whether the stages were significantly different from each other on the basis of age and scores. RESULTS: Data were analyzed from 322 evaluations in 108 individuals. There were significant differences among the stages based on age and severity scores. For individuals with longitudinal data, no individual reverted to a less severe stage over time. CONCLUSION: The CLN3SS is a disease-specific staging system that can be used to classify individuals into specific strata based on age and disease severity. The CLN3SS has potential applications in clinical trials for cohort stratification.
Assuntos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Lipofuscinoses Ceroides Neuronais Tipo de estudo: Etiology_studies / Prognostic_studies Limite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Lipofuscinoses Ceroides Neuronais Tipo de estudo: Etiology_studies / Prognostic_studies Limite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Ano de publicação: 2020 Tipo de documento: Article