A Patient-Reported Outcome Measure of Communication Difficulties in Friedreich Ataxia: COMATAX.

Abstract Background and Objectives: Friedreich ataxia (FA) causes progressive impairment of communication due to gradual deterioration of speech, associated with impaired hearing, socio-cognitive and language skills. There is an urgent need to investigate the impact of this multiparametric alteration on patients’ lives. Therefore, the COMunication and ATAXia measure (COMATAX) was developed and validated in French and German. Methods: In the PROFA study (NCT05943002), we conducted focus groups and cognitive interviews with patients with FA, professionals and caregivers to elaborate relevant items of communication disabilities. Subsequently, the measure was validated in 93 patients with FA via a mobile health app. For validation, distribution properties, reliability (Cronbach’s alpha) and validity (correlations with VHI-30, SSQ-12, SARA total score, SARA speech item, Speech rate and GAA repeats; known-groups validity by age, sex, and self-rated health/wellbeing, disease-severity, hearing function, daily activities, and exploratory factor analysis) were calculated. An IRT analysis was performed to assess item characteristics. Results: COMATAX consists of 17 items (five response options each) and an 18th question asking about the most bothersome symptom. We observed high internal consistency for the total COMATAX scale (α = 0.897), strong correlations with VHI-30 ( r =.894), SSQ-12 ( r = −.531), moderate with SARA score ( r =.498), SARA speech item ( r =.416), Speech rate ( r =-.354), weak with GAA repeat length (r GAA1 =-0.207) and the ability to distinguish between different subgroups (disease severity, self-rated health, wellbeing, hearing function, and daily activities). Most items showed good discrimination of communication ability. Conclusion: The COMATAX is a valid and reliable patient-reported communication disability measure, useful in future therapeutic trials in FA.

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Journal
The Cerebellum
Published
2026-05-23
DOI
https://doi.org/10.1007/s12311-026-02020-3
Primary Topic
Genetic Neurodegenerative Diseases
Type
article
Field-Weighted Citation Impact
0.00

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article

A Patient-Reported Outcome Measure of Communication Difficulties in Friedreich Ataxia: COMATAX.

Rania Hilab, Fanny Bertrand, Brittany Humphries, Lucie Pierron et al.
The Cerebellum
Genetic Neurodegenerative Diseases
article

A Patient-Reported Outcome Measure of Communication Difficulties in Friedreich Ataxia: COMATAX.

Rania Hilab, Fanny Bertrand, Brittany Humphries, Lucie Pierron, Sylvia Boesch, Maresa Buchholz, Dorota Sarwinska, Andreas Nadke, Arif Iskandar, Vivian Maas, Almut T. Bischoff, Victoire Monier, Elisabetta Indelicato, Jörg B. Schulz, Marcus Grobe-Einsler, Kathrin Reetz, Stéphanie Borel, Thomas Klopstock, Katrin Feldmann, Mariana Atencio, Feng Xie, Sabrina Sayah, Claire Ewenczyk, Elodie Petit, Anna Heinzmann, Chloé Diot, Stéphan Rouillon, Thomas Klockgether, Zofia Fleszar, Bernhard Michalowsky, Alexandra Durr, Jennifer Faber, Alix Vallancien
article en

Abstract

Abstract Background and Objectives: Friedreich ataxia (FA) causes progressive impairment of communication due to gradual deterioration of speech, associated with impaired hearing, socio-cognitive and language skills. There is an urgent need to investigate the impact of this multiparametric alteration on patients’ lives. Therefore, the COMunication and ATAXia measure (COMATAX) was developed and validated in French and German. Methods: In the PROFA study (NCT05943002), we conducted focus groups and cognitive interviews with patients with FA, professionals and caregivers to elaborate relevant items of communication disabilities. Subsequently, the measure was validated in 93 patients with FA via a mobile health app. For validation, distribution properties, reliability (Cronbach’s alpha) and validity (correlations with VHI-30, SSQ-12, SARA total score, SARA speech item, Speech rate and GAA repeats; known-groups validity by age, sex, and self-rated health/wellbeing, disease-severity, hearing function, daily activities, and exploratory factor analysis) were calculated. An IRT analysis was performed to assess item characteristics. Results: COMATAX consists of 17 items (five response options each) and an 18th question asking about the most bothersome symptom. We observed high internal consistency for the total COMATAX scale (α = 0.897), strong correlations with VHI-30 ( r =.894), SSQ-12 ( r = −.531), moderate with SARA score ( r =.498), SARA speech item ( r =.416), Speech rate ( r =-.354), weak with GAA repeat length (r GAA1 =-0.207) and the ability to distinguish between different subgroups (disease severity, self-rated health, wellbeing, hearing function, and daily activities). Most items showed good discrimination of communication ability. Conclusion: The COMATAX is a valid and reliable patient-reported communication disability measure, useful in future therapeutic trials in FA.

The CerebellumVol. 25(4)
Centre National de la Recherche Scientifique (FR), Innsbruck Medical University (AT), Inserm (FR), Universitätsmedizin Greifswald (DE), University Hospital Bonn (DE), German Center for Neurodegenerative Diseases (DE), Sorbonne Université (FR), Association Française de Chirurgie (FR), Assistance Publique – Hôpitaux de Paris (FR), Centre for Movement Disorders (CA), Friedrich Baur Stiftung (DE), Jülich Aachen Research Alliance (DE), Hertie Institute for Clinical Brain Research (DE), Institut du Cerveau (FR), Impact (CA), University of Tübingen (DE), Ludwig-Maximilians-Universität München (DE), RWTH Aachen University (DE), McMaster University (CA)
Agence Nationale de la Recherche, Bundesministerium für Ernährung und Landwirtschaft
Openalex Percentile: Top 8%
Genetic Neurodegenerative Diseases
DOIOpen Access PDFOpenAlex
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