Development and Validation of a Patient‐Reported Outcome Measure of Ataxia

ABSTRACT Background Assessment of cerebellar ataxia has been confined to rating scales, gait laboratories, and wearable sensors agnostic to patient input. Objectives The objective of this study was to develop a Patient‐Reported Outcome Measure of Ataxia. Methods (1) The conceptual framework, item po...

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Bibliographic Details
Published in:Movement disorders 2021-10, Vol.36 (10), p.2367-2377
Main Authors: Schmahmann, Jeremy D., Pierce, Samantha, MacMore, Jason, L'Italien, Gilbert J.
Format: Article
Language:English
Subjects:
Activities of daily living
Ataxia
Cerebellar ataxia
Cerebellum
clinical biomarker
Clinical trials
Cognitive ability
Gait
Mental health
Movement disorders
patient experience
Patients
Quality of life
symptoms
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Summary:ABSTRACT Background Assessment of cerebellar ataxia has been confined to rating scales, gait laboratories, and wearable sensors agnostic to patient input. Objectives The objective of this study was to develop a Patient‐Reported Outcome Measure of Ataxia. Methods (1) The conceptual framework, item pool development, and domain selection were developed using online surveys completed by 147 ataxia patients. Responses generated the 70‐item Patient‐Reported Outcome Measure of Ataxia, scored on a 0–4 Likert scale. (2) Cognitive debrief in 17 patients grouped by ataxia severity assessed content validity, readability, and comprehension. (3) Psychometric validation by 78 anonymized ataxia patients included test–retest reliability, responsiveness to ataxia severity, internal consistency (Cronbach's alpha), and item–total score correlations. (4) Validation was tested against measures of ataxia and quality of life in 20 patients. (5) Items were rank‐ordered to develop the Patient‐Reported Outcome Measure of Ataxia Short Form. Results Three thousand eight hundred fifty‐five symptoms were grouped into 3 domains (physical, activities of daily living, mental health) and 14 subdomains. The Patient‐Reported Outcome Measure of Ataxia was comprehensible, important, and relevant. Internal consistency, reliability, and test–retest reliability were high. Scores were responsive to ataxia severity stages 1, 2, and 3: mean ± standard deviation 81.0 ± 37.0, 129.6 ± 32.0, and 151.1 ± 41.3, respectively (r = 0.58, P 
ISSN:0885-3185
1531-8257
DOI:10.1002/mds.28670