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Patient-Reported Outcome Measure of Ataxia Correlates with Canonical Clinical Assessments in Chinese Spinocerebellar Ataxias

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Abstract

Spinocerebellar ataxia (SCA) patients’ reports of their own experiences are essential to the outcome evaluation in clinical trials. To better understand the health condition and well-being of ataxia population, Patient-Reported Outcome Measure of Ataxia (PROM-Ataxia) was developed. The aim of our study was to culturally adapt the PROM-Ataxia into Chinese version and assess its correlation with canonical clinical assessments. We translated the PROM-Ataxia into Chinese following the ISPOR TCA Task Force guidelines and evaluated its correlation with measures of motor ataxia, non-ataxia signs, quality of life, and mental health in 92 Chinese SCA participants. Nearly all the participants found this questionnaire complete and intelligible but some items were found repetitive or ambiguous. The total score of PROM-Ataxia from stage 0 to stage 3 was 23.24 ± 18.53, 79.11 ± 40.45, 144.30 ± 41.30, and 176.20 ± 31.74, respectively (p < 0.0001). It was strongly correlated with the Scale for the Assessment and Rating of Ataxia (SARA) (r = 0.832, p < 0.0001). Physical and activities domain of PROM-Ataxia were correlated with measures of motor ataxia, quality of life, and psychological health while mental health domain was correlated with all the clinical assessments including inventory of non-ataxia signs and cognitive assessment. We translated the PROM-Ataxia into Chinese for the first time, which allows transnational comparability in future studies. Our study validated the responsiveness of PROM-Ataxia to established clinical measures in Chinese SCA patients and implied its potential to evaluate the therapeutic effect and optimize the sensitivity of changes in clinical outcome assessments.

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Data Availability

The data that support the findings of this study and the Chinese PROM-Ataxia was available upon request to the corresponding author. The copyright on the PROM-Ataxia is owned by The Massachusetts General Hospital, including all translations, iterations and versions. There is no charge or licensing fee for using the scale for clinical and academic purposes. Charges apply only to studies conducted by industry.

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Acknowledgements

The authors express their appreciation to the team of Professor Jeremy D. Schmahmann from Massachusetts General Hospital for their development of the original version of the PROM-Ataxia and valuable support during the translation process. We also appreciate the interpreter volunteers for their contributions to the translation work of PROM-Ataxia and Professor Songjie Liao, Fengyin Liang, and Xilin Lu for their help. We are deeply grateful to the SCA patients and their families who dedicated their time and effort to our study.

Funding

This work was supported by grants from Science and Technology Program of Guangzhou (202201011228 and 2023A04J2194), National Natural Science Foundation of China (22293053), Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases (2020B1212060017), Guangdong Provincial Clinical Research Center for Neurological Diseases (2020B1111170002), Southern China International Joint Research Center for Early Intervention and Functional Rehabilitation of Neurological Diseases (2015B050501003 and 2020A0505020004), Guangdong Provincial Engineering Center for Major Neurological Disease Treatment, Guangdong Provincial Translational Medicine Innovation Platform for Diagnosis and Treatment of Major Neurological Disease, and Guangzhou Clinical Research and Translational Center for Major Neurological Diseases (201604020010).

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Author notes

  1. Huajing You and Qiong Cai have contributed equally to this work and share first authorship.

Authors and Affiliations

  1. Department of Neurology, The First Affiliated Hospital, Sun Yat-sen University, Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, National Key Clinical Department and Key Discipline of Neurology, Guangzhou, China

    Huajing You, Qiong Cai, Xunhua Li & Chao Wu

  2. China Spinocerebellar Ataxia Association, Beijing, China

    Ziyue Ouyang

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  1. Huajing You
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  2. Qiong Cai
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  3. Ziyue Ouyang
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  4. Xunhua Li
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  5. Chao Wu
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Contributions

Conceptualization: Huajing You, and Chao Wu; Methodology: Huajing You, Qiong Cai, Ziyue Ouyang, and Chao Wu; Formal analysis and investigation: Huajing You; Writing - original draft preparation: Huajing You; Writing - review and editing: Chao Wu; Supervision: Xunhua Li. All authors read and approved the final manuscript.

Corresponding author

Correspondence to Chao Wu.

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Ethics Approval

All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki Declaration and its later amendments or comparable ethical standards. The study was approved by the ethics committees of the First Affiliated Hospital, Sun Yat-sen University (Ethics approval number: [2020]507).

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Written informed consent was obtained from the parents before enrollment.

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Patients signed informed consent regarding publishing their data.

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The authors have no relevant financial or non-financial interests to disclose.

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You, H., Cai, Q., Ouyang, Z. et al. Patient-Reported Outcome Measure of Ataxia Correlates with Canonical Clinical Assessments in Chinese Spinocerebellar Ataxias. Cerebellum (2023). https://doi.org/10.1007/s12311-023-01630-5

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