Abstract
Background
The patient-reported outcome measure of ataxia (PROM-Ataxia) is the first patient-reported questionnaire specifically developed for use in patients with cerebellar ataxia. The scale was recently designed and validated in English language, and it consists of 70 items encompassing all aspects associated with the patient experience, including physical and mental health and their consequences on activities of daily living. The aim of the study was to translate and culturally adapt into Italian the PROM-Ataxia questionnaire, before assessing its psychometric properties.
Methods
We translated and culturally adapted into Italian the PROM-Ataxia following the ISPOR TCA Task Force guidelines. The questionnaire was field tested via cognitive interviews with users.
Results
The Italian patients found that the questionnaire was complete, and no significant contents related to the physical, mental, and functional dimensions were missing. Some items were found redundant or ambiguous. Most of the identified issues pertained to semantic equivalence, and a few to conceptual and normative equivalence, while the questionnaire did not contain any idiomatic expression.
Conclusions
The translation and cultural adaptation of the PROM-Ataxia questionnaire in the Italian patient population represent the pre-requisite for the subsequent psychometric validation of the scale. This instrument may be valuable for cross-country comparability that would allow the merging of the data in collaborative multinational research studies.
This is a preview of subscription content, log in via an institution to check access.
Similar content being viewed by others
Data availability
Data supporting these findings are available in the supplementary files.
References
van Gaalen J, van de Warrenburg BPC (2012) A practical approach to late-onset cerebellar ataxia: putting the disorder with lack of order into order. Pract Neurol 12:14–24
Witek N, Hawkins J, Hall D (2021) Genetic ataxias: update on classification and diagnostic approaches. Curr Neurol Neurosci Rep 21(3):13
Jacobi H, du Montcel ST, Bauer P et al (2015) Long- term disease progression in spinocerebellar ataxia types 1, 2, 3, and 6: a longitudinal cohort study. Lancet Neurol 14(11):1101–1108
Schmitz-Hubsch T, du Montcel ST, Baliko L et al (2006) Scale for the assessment and rating of ataxia: development of a new clinical scale. Neurology 66:1717–1720
Bürk K, Mälzig U, Wolf S et al (2009) Comparison of three clinical rating scales in Friedreich ataxia (FRDA). Mov Disord 24(12):1779–1784
Jacobi H, Rakowicz M, Rola R et al (2013) Inventory of non-ataxia signs (INAS): validation of a new clinical assessment instrument. Cerebellum 12(3):418–428
Perez-Lloret S, van de Warrenburg B, Rossi M et al (2021) Members of the MDS rating scales review committee. Assessment of ataxia rating scales and cerebellar functional tests: critique and recommendations. Mov Disord 36(2):283–297
Food and Drug Administration (FDA). Guidance for industry- patient-reported outcome measures: use in medical product development to support labeling claims. 2009. https://www.fda.gov/media/77832/download. Accessed June 2022.
Deshpande PR, Rajan S, Sudeepthi BL, Nazir CPA (2011) Patient-reported outcomes: a new era in clinical research. Perspect Clin Res 2(4):137–144
Black N (2013) Patient reported outcome measures could help transform health care. BMJ 346:f167
Maas RPPWM, van de Warrenburg BPC (2021) Exploring the clinical meaningfulness of the scale for the assessment and rating of ataxia: a comparison of patient and physician perspectives at the item level. Parkinsonism Relat Disord 91:37–41
Cano SJ, Riazi A, Schapira AH, Cooper JM, Hobart JC (2009) Friedreich’s ataxia impact scale: a new measure striving to provide the flexibility required by today’s studies. Mov Disord 24:984–992
Schmitz-Hubsch T, Coudert M, Giunti P et al (2010) Self-rated health status in spinocerebellar ataxia–results from a European multicenter study. Mov Disord 25(5):587–595
Brusse E, Brusse-Keizer MG, Duivenvoorden HJ, van Swieten JC (2011) Fatigue in spinocerebellar ataxia: patient self-assessment of an early and disabling symptom. Neurology 76(11):953–959
Tai G, Yiu EM, Corben LA, Delatycki MB (2015) A longitudinal study of the Friedreich ataxia impact scale. J Neurol Sci 352(1–2):53–57
Lo RY, Figueroa KP, Pulst SM et al (2016) Depression and clinical progression in spinocerebellar ataxias. Parkinsonism Relat Disord 22:87–92
Jacobi H, Tezenas du Montcel S, Bauer P et al (2018) Long-term evolution of patient-reported outcome measures in spinocerebellar ataxias. J Neurol 265(9):2040–2051
Schmahmann JD, Pierce S, MacMore J, L’Italien GJ (2021) Development and validation of a patient-reported outcome measure of ataxia. Mov Disord 36(10):2367–2377
Wild D, Grove A, Martin M, Eremenco S, McElroy S, Verjee-Lorenz A, Erikson P, ISPOR Task Force for Translation and Cultural Adaptation (2005) Principles of good practice for the translation and cultural adaptation process for patient-reported outcomes (PRO) measures: report of the ISPOR task force for translation and cultural adaptation. Value Health 8(2):94–104
Denzin NK, Lincoln YS (eds) (2010) The SAGE handbook of qualitative research, 2nd edn. Sage, Thousand Oaks
Klockgether T, Lüdtke R, Kramer B et al (1998) The natural history of degenerative ataxia: a retrospective study in 466 patients. Brain 121(Pt 4):589–600
Collins D (2003) Pretesting survey instruments: an overview of cognitive methods. Qual Life Res 12(3):229–238
Guillemin F, Bombardier C, Beaton D (1993) Cross-cultural adaptation of health-related quality of life measures: literature review and proposed guidelines. J Clin Epidemiol 46:1417–1432
Jenkins CR, Dillman DA (1997) Towards a theory of self-administered questionnaire design. In: Lyberg L, Biemer P, Collins M, de Leeuw E, Schwartz N, Trewin D (eds) Survey measurement and process quality. Wiley, New York, pp p165-196
Harrison C, Loe BS, Lis P, Sidey-Gibbons C (2020) Maximizing the potential of patient-reported assessments by using the open-source concerto platform with computerized adaptive testing and machine learning. J Med Internet Res 22(10):e20950
Acknowledgements
We thank to authors of the original version of the PROM-Ataxia questionnaire (Schmahmann JD, Pierce S, MacMore J, and L’Italien G.). In particular, we are deeply indebted to Professor Jeremy Schmahmann for his valuable support and contribution during the translation process. We are also very grateful to the patients who generously contributed in this work.
Funding
The study was supported by the Italian Ministry of Health (Grant RF-2019–12368918 to CM, and RRC).
Author information
Authors and Affiliations
Contributions
The study conception and design were performed by Anna Castaldo, Mariangela Farinotti, Alessandra Solari, and Caterina Mariotti. Material preparation, data collection, and analysis were performed by Anna Castaldo, Mariangela Farinotti, Mario Fichera, Lorenzo Nanetti, and Filippo Fortuna, MD. The first draft of the manuscript was written by Caterina Mariotti and Alessandra Solari, and all authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.
Corresponding author
Ethics declarations
Ethics approval
The study was approved by the Ethics Committees of the Fondazione IRCCS Istituto Neurologico Carlo Besta, Italy. All procedures followed the Declaration of Helsinki.
Informed consent
Informed consent was obtained from all individual participants included in the study.
Competing interests
CM is a site principal investigator on clinical trials sponsored by F. Hoffmann-La Roche Ltd; Reata Pharmaceuticals; Novartis, and Prilenia Therapeutics, and received consultancy fees for advisory board from Reata Swiss International, and Vico Therapeutics. AS is the principal investigator of a clinical trial granted by the Foundation of the Italian Multiple Sclerosis Society (FISM); she received consultancy fees for advisory board from Almirall and Merck Serono. The authors report no conflicts of interest related to this article. CM is an unpaid member of the Steering Committee of the EFACTS (European Friedreich Ataxia Consortium for Translational Studies) and of the Executive Committee of the European Huntington Disease Network (EHDN). C.M., L.N., and M.Fi. are members of the European Reference Network for Rare Neurological Disorders (ERN-RND), Project ID No. 739510.
Additional information
Publisher's note
Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Supplementary Information
Below is the link to the electronic supplementary material.
10072_2023_6714_MOESM1_ESM.docx
Supplementary file 1. Translation grid. The translation grid reports challenging phrases, uncertainties and rationale of panel decisions. The grid also contains queries sent to the scale author, and his responses. (DOCX 144 KB)
10072_2023_6714_MOESM2_ESM.docx
Supplementary file 2. Cognitive debriefing guide. Methodology and questions to explore the patient's understanding of each part of the questionnaire. (DOCX 37 KB)
10072_2023_6714_MOESM3_ESM.docx
Supplementary file 3. Audit trail of the cognitive debriefing. The audit trail summarizes comments and suggestions of the patients during the cognitive debriefing. (DOCX 70 KB)
10072_2023_6714_MOESM4_ESM.docx
Supplementary file 4. Italian Version of PROM-ATAXIA. Final Italian translated version of the PROM-ATAXIA questionnaire (DOCX 54 KB)
Rights and permissions
Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.
About this article
Cite this article
Castaldo, A., Farinotti, M., Fichera, M. et al. Italian cross-cultural adaptation of the patient-reported outcome measure of ataxia. Neurol Sci 44, 2773–2779 (2023). https://doi.org/10.1007/s10072-023-06714-y
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1007/s10072-023-06714-y