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01-09-2018 | Original Communication

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

Published in: Journal of Neurology | Issue 9/2018

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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Title: 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
Publication date: 01-09-2018
Publisher: Springer Berlin Heidelberg
Published in: Journal of Neurology / Issue 9/2018
Print ISSN: 0340-5354
Electronic ISSN: 1432-1459
DOI: https://doi.org/10.1007/s00415-018-8954-0

At a glance: The STEP trials

Springer Medicine

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

Abstract

Introduction

Methods

Results

Conclusions

At a glance: The STEP trials

Springer Medicine

Abstract

Introduction

Methods

Results

Conclusions

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