Literature DB >> 21832228

The natural history of spinocerebellar ataxia type 1, 2, 3, and 6: a 2-year follow-up study.

H Jacobi1, P Bauer, P Giunti, R Labrum, M G Sweeney, P Charles, A Dürr, C Marelli, C Globas, C Linnemann, L Schöls, M Rakowicz, R Rola, E Zdzienicka, T Schmitz-Hübsch, R Fancellu, C Mariotti, C Tomasello, L Baliko, B Melegh, A Filla, C Rinaldi, B P van de Warrenburg, C C P Verstappen, S Szymanski, J Berciano, J Infante, D Timmann, S Boesch, S Hering, C Depondt, M Pandolfo, J-S Kang, S Ratzka, J Schulz, S Tezenas du Montcel, T Klockgether.   

Abstract

OBJECTIVE: To obtain quantitative data on the progression of the most common spinocerebellar ataxias (SCAs) and identify factors that influence their progression, we initiated the EUROSCA natural history study, a multicentric longitudinal cohort study of 526 patients with SCA1, SCA2, SCA3, or SCA6. We report the results of the 1- and 2-year follow-up visits.
METHODS: As the primary outcome measure we used the Scale for the Assessment and Rating of Ataxia (SARA, 0-40), and as a secondary measure the Inventory of Non-Ataxia Symptoms (INAS, 0-16) count.
RESULTS: The annual increase of the SARA score was greatest in SCA1 (2.18 ± 0.17, mean ± SE) followed by SCA3 (1.61 ± 0.12) and SCA2 (1.40 ± 0.11). SARA progression in SCA6 was slowest and nonlinear (first year: 0.35 ± 0.34, second year: 1.44 ± 0.34). Analysis of the INAS count yielded similar results. Larger expanded repeats and earlier age at onset were associated with faster SARA progression in SCA1 and SCA2. In SCA1, repeat length of the expanded allele had a similar effect on INAS progression. In SCA3, SARA progression was influenced by the disease duration at inclusion, and INAS progression was faster in females.
CONCLUSIONS: Our study gives a comprehensive quantitative account of disease progression in SCA1, SCA2, SCA3, and SCA6 and identifies factors that specifically affect disease progression.

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Year:  2011        PMID: 21832228      PMCID: PMC3174068          DOI: 10.1212/WNL.0b013e31822e7ca0

Source DB:  PubMed          Journal:  Neurology        ISSN: 0028-3878            Impact factor:   9.910


  26 in total

1.  SCA6 is caused by moderate CAG expansion in the alpha1A-voltage-dependent calcium channel gene.

Authors:  O Riess; L Schöls; H Bottger; D Nolte; A M Vieira-Saecker; C Schimming; F Kreuz; M Macek; A Krebsová; T Klockgether; C Zühlke; F A Laccone
Journal:  Hum Mol Genet       Date:  1997-08       Impact factor: 6.150

2.  Spinocerebellar ataxia type 2. Genotype and phenotype in German kindreds.

Authors:  L Schöls; S Gispert; M Vorgerd; A M Menezes Vieira-Saecker; P Blanke; G Auburger; G Amoiridis; S Meves; J T Epplen; H Przuntek; S M Pulst; O Riess
Journal:  Arch Neurol       Date:  1997-09

3.  Autosomal dominant cerebellar ataxia type I. Clinical and molecular study in 36 Italian families including a comparison between SCA1 and SCA2 phenotypes.

Authors:  A Filla; G De Michele; G Campanella; A Perretti; L Santoro; L Serlenga; M Ragno; O Calabrese; I Castaldo; G De Joanna; S Cocozza
Journal:  J Neurol Sci       Date:  1996-10       Impact factor: 3.181

4.  The natural history of degenerative ataxia: a retrospective study in 466 patients.

Authors:  T Klockgether; R Lüdtke; B Kramer; M Abele; K Bürk; L Schöls; O Riess; F Laccone; S Boesch; I Lopes-Cendes; A Brice; R Inzelberg; N Zilber; J Dichgans
Journal:  Brain       Date:  1998-04       Impact factor: 13.501

5.  Spinocerebellar ataxia type 6. Molecular and clinical features of 35 Japanese patients including one homozygous for the CAG repeat expansion.

Authors:  R Matsumura; N Futamura; Y Fujimoto; S Yanagimoto; H Horikawa; A Suzumura; T Takayanagi
Journal:  Neurology       Date:  1997-11       Impact factor: 9.910

6.  Cloning of the gene for spinocerebellar ataxia 2 reveals a locus with high sensitivity to expanded CAG/glutamine repeats.

Authors:  G Imbert; F Saudou; G Yvert; D Devys; Y Trottier; J M Garnier; C Weber; J L Mandel; G Cancel; N Abbas; A Dürr; O Didierjean; G Stevanin; Y Agid; A Brice
Journal:  Nat Genet       Date:  1996-11       Impact factor: 38.330

7.  Autosomal dominant cerebellar ataxia: phenotypic differences in genetically defined subtypes?

Authors:  L Schöls; G Amoiridis; T Büttner; H Przuntek; J T Epplen; O Riess
Journal:  Ann Neurol       Date:  1997-12       Impact factor: 10.422

8.  Autosomal dominant cerebellar ataxia (SCA6) associated with small polyglutamine expansions in the alpha 1A-voltage-dependent calcium channel.

Authors:  O Zhuchenko; J Bailey; P Bonnen; T Ashizawa; D W Stockton; C Amos; W B Dobyns; S H Subramony; H Y Zoghbi; C C Lee
Journal:  Nat Genet       Date:  1997-01       Impact factor: 38.330

9.  Spinocerebellar ataxia type 6: genotype and phenotype in German kindreds.

Authors:  L Schöls; R Krüger; G Amoiridis; H Przuntek; J T Epplen; O Riess
Journal:  J Neurol Neurosurg Psychiatry       Date:  1998-01       Impact factor: 10.154

10.  Age at onset variance analysis in spinocerebellar ataxias: a study in a Dutch-French cohort.

Authors:  Bart P C van de Warrenburg; Harrie Hendriks; Alexandra Dürr; Martin C A van Zuijlen; Giovanni Stevanin; Agnès Camuzat; Richard J Sinke; Alexis Brice; Berry P H Kremer
Journal:  Ann Neurol       Date:  2005-04       Impact factor: 10.422

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  75 in total

1.  Serum neurofilament light is increased in multiple system atrophy of cerebellar type and in repeat-expansion spinocerebellar ataxias: a pilot study.

Authors:  Carlo Wilke; Friedemann Bender; Stefanie N Hayer; Kathrin Brockmann; Ludger Schöls; Jens Kuhle; Matthis Synofzik
Journal:  J Neurol       Date:  2018-05-08       Impact factor: 4.849

2.  Long-term evolution of patient-reported outcome measures in spinocerebellar ataxias.

Authors:  Heike Jacobi; Sophie Tezenas du Montcel; Peter Bauer; Paola Giunti; Arron Cook; Robyn Labrum; Michael H Parkinson; Alexandra Durr; Alexis Brice; Perrine Charles; Cecilia Marelli; Caterina Mariotti; Lorenzo Nanetti; Lidia Sarro; Maria Rakowicz; Anna Sulek; Anna Sobanska; Tanja Schmitz-Hübsch; Ludger Schöls; Holger Hengel; Laszlo Baliko; Bela Melegh; Alessandro Filla; Antonella Antenora; Jon Infante; José Berciano; Bart P van de Warrenburg; Dagmar Timmann; Sandra Szymanski; Sylvia Boesch; Wolfgang Nachbauer; Jun-Suk Kang; Massimo Pandolfo; Jörg B Schulz; Audrey Tanguy Melac; Alhassane Diallo; Thomas Klockgether
Journal:  J Neurol       Date:  2018-06-29       Impact factor: 4.849

3.  Longitudinal study of cognitive and psychiatric functions in spinocerebellar ataxia types 1 and 2.

Authors:  Roberto Fancellu; Dominga Paridi; Chiara Tomasello; Marta Panzeri; Anna Castaldo; Silvia Genitrini; Paola Soliveri; Floriano Girotti
Journal:  J Neurol       Date:  2013-12       Impact factor: 4.849

4.  Comparing speech characteristics in spinocerebellar ataxias type 3 and type 6 with Friedreich ataxia.

Authors:  Bettina Brendel; Matthis Synofzik; Hermann Ackermann; Tobias Lindig; Theresa Schölderle; Ludger Schöls; Wolfram Ziegler
Journal:  J Neurol       Date:  2014-09-30       Impact factor: 4.849

Review 5.  Ataxia.

Authors:  Sheng-Han Kuo
Journal:  Continuum (Minneap Minn)       Date:  2019-08

6.  SCA 6 with Writer's Cramp: The Phenotype Expanded.

Authors:  Diana Angelika Olszewska; Richard Walsh; Tim Lynch
Journal:  Mov Disord Clin Pract       Date:  2015-08-26

7.  The ever expanding spinocerebellar ataxias. Editorial.

Authors:  Antoni Matilla-Dueñas
Journal:  Cerebellum       Date:  2012-12       Impact factor: 3.847

8.  Progression of Gait Ataxia in Patients with Degenerative Cerebellar Disorders: a 4-Year Follow-Up Study.

Authors:  Mariano Serrao; Giorgia Chini; Carlo Casali; Carmela Conte; Martina Rinaldi; Alberto Ranavolo; Christian Marcotulli; Luca Leonardi; Gaia Fragiotta; Fabiano Bini; Gianluca Coppola; Francesco Pierelli
Journal:  Cerebellum       Date:  2017-06       Impact factor: 3.847

9.  Inventory of Non-Ataxia Signs (INAS): validation of a new clinical assessment instrument.

Authors:  H Jacobi; M Rakowicz; R Rola; R Fancellu; C Mariotti; P Charles; A Dürr; M Küper; D Timmann; C Linnemann; L Schöls; O Kaut; C Schaub; A Filla; L Baliko; B Melegh; J-S Kang; P Giunti; B P C van de Warrenburg; R Fimmers; T Klockgether
Journal:  Cerebellum       Date:  2013-06       Impact factor: 3.847

Review 10.  Polyglutamine spinocerebellar ataxias - from genes to potential treatments.

Authors:  Henry L Paulson; Vikram G Shakkottai; H Brent Clark; Harry T Orr
Journal:  Nat Rev Neurosci       Date:  2017-08-17       Impact factor: 34.870

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