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Long-term evolution of patient-reported outcome measures in spinocerebellar ataxias.
Jacobi, Heike; du Montcel, Sophie Tezenas; Bauer, Peter; Giunti, Paola; Cook, Arron; Labrum, Robyn; Parkinson, Michael H; Durr, Alexandra; Brice, Alexis; Charles, Perrine; Marelli, Cecilia; Mariotti, Caterina; Nanetti, Lorenzo; Sarro, Lidia; Rakowicz, Maria; Sulek, Anna; Sobanska, Anna; Schmitz-Hübsch, Tanja; Schöls, Ludger; Hengel, Holger; Baliko, Laszlo; Melegh, Bela; Filla, Alessandro; Antenora, Antonella; Infante, Jon; Berciano, José; van de Warrenburg, Bart P; Timmann, Dagmar; Szymanski, Sandra; Boesch, Sylvia; Nachbauer, Wolfgang; Kang, Jun-Suk; Pandolfo, Massimo; Schulz, Jörg B; Melac, Audrey Tanguy; Diallo, Alhassane; Klockgether, Thomas.
  • Jacobi H; German Center for Neurodegenerative Diseases (DZNE), Bonn, Germany. heike.jacobi@med.uni-heidelberg.de.
  • du Montcel ST; Department of Neurology, Heidelberg University Hospital, Im Neuenheimer Feld 400, 69120, Heidelberg, Germany. heike.jacobi@med.uni-heidelberg.de.
  • Bauer P; Sorbonne Universités, Université Pierre et Marie Curie (UPMC) Univ Paris 06, UMRS 1136, INSERM U 1136, Institut Pierre Louis d'Epidémiologie et de Santé Publique, 75013, Paris, France.
  • Giunti P; AP-HP, Biostatistics Unit, Groupe Hospitalier Pitié-Salpêtrière, 75013, Paris, France.
  • Cook A; Institute of Medical Genetics and Applied Genomics, University of Tübingen, Tübingen, Germany.
  • Labrum R; CENTOGENE AG, Rostock, Germany.
  • Parkinson MH; Department of Molecular Neuroscience, UCL, Institute of Neurology, London, UK.
  • Durr A; Department of Molecular Neuroscience, UCL, Institute of Neurology, London, UK.
  • Brice A; Neurogenetic Laboratory, National Hospital of Neurology and Neurosurgery, UCL, London, UK.
  • Charles P; Department of Molecular Neuroscience, UCL, Institute of Neurology, London, UK.
  • Marelli C; INSERM, U 1127, 75013, Paris, France.
  • Mariotti C; CNRS, UMR 7225, 75013, Paris, France.
  • Nanetti L; Sorbonne Universités, UPMC Univ Paris 06, UMRS_1127, 75013, Paris, France.
  • Sarro L; Institut du Cerveau et de la Moelle épinière, ICM, 75013, Paris, France.
  • Rakowicz M; Département de Génétique, APHP, Hôpital de la Pitié-Salpêtrière, 75013, Paris, France.
  • Sulek A; INSERM, U 1127, 75013, Paris, France.
  • Sobanska A; CNRS, UMR 7225, 75013, Paris, France.
  • Schmitz-Hübsch T; Sorbonne Universités, UPMC Univ Paris 06, UMRS_1127, 75013, Paris, France.
  • Schöls L; Institut du Cerveau et de la Moelle épinière, ICM, 75013, Paris, France.
  • Hengel H; Département de Génétique, APHP, Hôpital de la Pitié-Salpêtrière, 75013, Paris, France.
  • Baliko L; Département de Génétique, APHP, Hôpital de la Pitié-Salpêtrière, 75013, Paris, France.
  • Melegh B; Service de Neurologie-CMRR, CHRU Gui de Chauliac, 80, av. A. Fliche, 34295, Montpellier Cedex 05, France.
  • Filla A; SOSD Genetics of Neurodegenerative and Metabolic Diseases, Fondazione-IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
  • Antenora A; SOSD Genetics of Neurodegenerative and Metabolic Diseases, Fondazione-IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
  • Infante J; SOSD Genetics of Neurodegenerative and Metabolic Diseases, Fondazione-IRCCS Istituto Neurologico Carlo Besta, Milan, Italy.
  • Berciano J; Department of Clinical Neurophysiology, Institute of Psychiatry and Neurology, Warsaw, Poland.
  • van de Warrenburg BP; Department of Genetics, Institute of Psychiatry and Neurology, Warsaw, Poland.
  • Timmann D; Department of Clinical Neurophysiology, Institute of Psychiatry and Neurology, Warsaw, Poland.
  • Szymanski S; Charité Universitätsmedizin Berlin, NeuroCure Clinical Research Center, Berlin, Germany.
  • Boesch S; Department of Neurodegeneration and Hertie-Institute for Clinical Brain Research, University of Tübingen and Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE), 72076, Tübingen, Germany.
  • Nachbauer W; Department of Neurodegeneration and Hertie-Institute for Clinical Brain Research, University of Tübingen and Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE), 72076, Tübingen, Germany.
  • Kang JS; Department of Neurology, Zala County Hospital, Zrinyi M. Str. 1, Zalaegerszeg, 8900, Hungary.
  • Pandolfo M; Department of Medical Genetics, Medical School, and Szentagothai Research Center, University of Pécs, Pécs, Hungary.
  • Schulz JB; Department of Neuroscience, and Reproductive and Odontostomatological Sciences, Federico II University Naples, Naples, Italy.
  • Melac AT; Department of Neuroscience, and Reproductive and Odontostomatological Sciences, Federico II University Naples, Naples, Italy.
  • Diallo A; Service of Neurology, University Hospital Marqués de Valdecilla (IDIVAL), University of Cantabria (UC) and Centro de Investigación Biomédica en Red de Enfermedades Neurodegenerativas (CIBERNED), Santander, Spain.
  • Klockgether T; Service of Neurology, University Hospital "Marqués de Valdecilla (IDIVAL)", "Centro de Investigación Biomédica en Red de Enfermedades Neurodegenerativas (CIBERNED)", University of Cantabria (UC), Santander, Spain.
J Neurol ; 265(9): 2040-2051, 2018 Sep.
Article en En | MEDLINE | ID: mdl-29959555
ABSTRACT

INTRODUCTION:

To study the long-term evolution of patient-reported outcome measures (PROMs) in the most common spinocerebellar ataxias (SCAs), we analyzed 8 years follow-up data of the EUROSCA Natural History Study, a cohort study of 526 patients with SCA1, SCA2, SCA3 and SCA6.

METHODS:

To assess the functional capacity in daily living, we used the functional assessment (part IV) of the Unified Huntington's Disease Rating Scale (UHDRS-IV), for health-related quality of life the visual analogue scale of the EuroQol Five Dimensions Questionnaire (EQ-5D VAS), and for depressive symptoms the Patient Health Questionnaire (PHQ-9). Severity of ataxia was assessed using the Scale for the Assessment and Rating of Ataxia (SARA) and neurological symptoms other than ataxia with the Inventory of Non-Ataxia Signs (INAS).

RESULTS:

UHDRS-IV [SCA1 - 1.35 (0.12); SCA2 - 1.15 (0.11); SCA3 - 1.16 (0.11); SCA6 - 0.99 (0.12)] and EQ-5D [SCA1 - 2.88 (0.72); SCA2 - 1.97 (0.49); SCA3 - 2.06 (0.55); SCA6 - 1.03 (0.57)] decreased linearly, whereas PHQ-9 increased [SCA1 0.15 (0.04); SCA2 0.09 (0.03); SCA3 0.06 (0.04); SCA6 0.07 (0.04)] during the observational period. Standard response means (SRMs) of UHDRS-IV (0.473-0.707) and EQ-5D VAS (0.053-0.184) were lower than that of SARA (0.404-0.979). In SCA1, higher SARA scores [- 0.0288 (0.01), p = 0.0251], longer repeat expansions [- 0.0622 (0.02), p = 0.0002] and the presence of cognitive impairment at baseline [- 0.5381 (0.25), p = 0.0365] were associated with faster UHDRS-IV decline. In SCA3, higher INAS counts were associated with a faster UHDRS-IV decline [- 0.05 (0.02), p = 0.0212]. In SCA1, PHQ-9 progression was faster in patients with cognitive impairment [0.14 (0.07); p = 0.0396].

CONCLUSIONS:

In the common SCAs, PROMs give complementary information to the information provided by neurological scales. This underlines the importance of PROMs as additional outcome measures in future interventional trials.
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Calidad de Vida / Actividades Cotidianas / Ataxias Espinocerebelosas Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Aged / Female / Humans / Male / Middle aged Idioma: En Año: 2018 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Search on Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Calidad de Vida / Actividades Cotidianas / Ataxias Espinocerebelosas Tipo de estudio: Observational_studies / Prognostic_studies / Risk_factors_studies Límite: Aged / Female / Humans / Male / Middle aged Idioma: En Año: 2018 Tipo del documento: Article