Measurement Properties of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia.

IF 3.9 3区 医学 Q1 CLINICAL NEUROLOGY Neurology and Therapy Pub Date : 2025-04-01 Epub Date: 2025-01-13 DOI:10.1007/s40120-024-00708-4
Michele H Potashman, Evan Popoff, Lauren C Powell, Melissa Wolfe Beiner, Ainsley Mackenzie, Vlad Coric, Sub Subramony, Matthis Synofzik, Jeremy Schmahmann, Gilbert L'Italien
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Abstract

Introduction: The Friedreich Ataxia Rating Scale-Activities of Daily Living (FARS-ADL) is a valid, highly utilized measure for assessing ADL impacts in patients with Friedreich ataxia. We provide evidence of the psychometric validity of the FARS-ADL in two cohorts of patients with spinocerebellar ataxia (SCA).

Methods: Using data from a cohort of real-world subjects with SCA (recruited at Massachusetts General Hospital [MGH]; n = 33) and a phase 3 trial of troriluzole in adults with SCA (NCT03701399 [Study 206]; n = 217), comprising a subset of patients with the SCA3 genotype (n = 89), the psychometric measurement properties and minimal change thresholds of the FARS-ADL were examined.

Results: Ceiling effects for the FARS-ADL were absent within the MGH cohort while floor effects were observed for eight of nine items. Excellent internal consistency reliability was observed (αtotal = 0.88; αitems-removed = 0.86-0.87), and item-to-total correlations were acceptable (r = 0.55-0.89 per item). Convergent and divergent validity were supported with strong correlations demonstrated between FARS-ADL and scales measuring similar concepts (Neuro-QOL [Upper], Neuro-QOL [Lower], PROM-ADL, PROM-PHYS, and FARS-FUNC; all P < 0.001) and weaker correlations shown between measures of differing constructs. A two- to three-point threshold for meaningful changes was supported as 0.5 × SD = 2.43, SEM = 2.19. Mean changes from baseline for subjects classified as "improved," "no change," or "deteriorated" were -0.54, 0.22, and 1.47, respectively. Similar trends were observed in the Study 206 all-SCA and SCA3 cohorts.

Conclusion: Psychometric evaluation showed that the FARS-ADL performed well on analyses examining the reliability and validity of the measure and can detect meaningful changes in patients with SCA, including those with SCA3.

Trial registration: ClinicalTrials.gov identifier, NCT03701399 (Study 206).

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弗里德赖希共济失调评定量表在脊髓小脑性共济失调患者中的测量特性。
简介:弗里德赖希共济失调评定量表-日常生活活动(FARS-ADL)是一种有效的、被广泛使用的评估弗里德赖希共济失调患者ADL影响的方法。我们在两组脊髓小脑性共济失调(SCA)患者中提供了FARS-ADL心理测量效度的证据。方法:使用来自马萨诸塞州总医院[MGH]招募的真实世界SCA受试者队列的数据;n = 33)和成人SCA患者的三期试验(NCT03701399 [Study 206];n = 217),包括SCA3基因型患者亚组(n = 89),研究了FARS-ADL的心理测量特性和最小变化阈值。结果:在MGH队列中,FARS-ADL的上限效应不存在,而在9个项目中有8个项目存在下限效应。具有良好的内部一致性信度(αtotal = 0.88;αitems-removed = 0.86-0.87), item-to-total相关性是可以接受的(r = 0.55-0.89 / item)。FARS-ADL与测量类似概念的量表(neuroqol [Upper], neuroqol [Lower], PROM-ADL, PROM-PHYS和FARS-FUNC)之间存在强相关性,证明了收敛效度和发散效度的支持;结论:心理测量评估显示,fas - adl在检验该测量的信度和效度的分析中表现良好,可以检测SCA患者(包括SCA3患者)的有意义的变化。试验注册:ClinicalTrials.gov识别码,NCT03701399 (Study 206)。
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来源期刊
Neurology and Therapy
Neurology and Therapy CLINICAL NEUROLOGY-
CiteScore
5.40
自引率
8.10%
发文量
103
审稿时长
6 weeks
期刊介绍: Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Neurology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research. Rapid Publication The journal’s rapid publication timelines aim for a peer review decision within 2 weeks of submission. If an article is accepted, it will be published online 3-4 weeks from acceptance. These rapid timelines are achieved through the combination of a dedicated in-house editorial team, who closely manage article workflow, and an extensive Editorial and Advisory Board who assist with rapid peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model, this allows for the rapid and efficient communication of the latest research and reviews to support scientific discovery and clinical practice. Open Access All articles published by Neurology and Therapy are open access. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning that authors will always have a personal point of contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. We also encourage pre-submission enquiries and are always happy to provide a confidential assessment of manuscripts. Digital Features and Plain Language Summaries Neurology and Therapy offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by key summary points, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article. The journal also provides the option to include various types of digital features including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations. All additional features are peer reviewed to the same high standard as the article itself. If you consider that your paper would benefit from the inclusion of a digital feature, please let us know. Our editorial team are able to create high-quality slide decks and infographics in-house, and video abstracts through our partner Research Square, and would be happy to assist in any way we can. For further information about digital features, please contact the journal editor (see ‘Contact the Journal’ for email address), and see the ‘Guidelines for digital features and plain language summaries’ document under ‘Submission guidelines’. For examples of digital features please visit our showcase page https://springerhealthcare.com/expertise/publishing-digital-features/ Publication Fees Upon acceptance of an article, authors will be required to pay the mandatory Rapid Service Fee of €5250/$6000/£4300. The journal will consider fee discounts and waivers for developing countries and this is decided on a case-by-case basis. Peer Review Process Upon submission, manuscripts are assessed by the editorial team to ensure they fit within the aims and scope of the journal and are also checked for plagiarism. All suitable submissions are then subject to a comprehensive single-blind peer review. Reviewers are selected based on their relevant expertise and publication history in the subject area. The journal has an extensive pool of editorial and advisory board members who have been selected to assist with peer review based on the afore-mentioned criteria. At least two extensive reviews are required to make the editorial decision, with the exception of some article types such as Commentaries, Editorials and Letters which are generally reviewed by one member of the Editorial Board. Where reviews conflict, an Editorial Board Member will be contacted for further advice and a presiding decision. Manuscripts are then either accepted, rejected or authors are required to make major or minor revisions (both reviewer comments and editorial comments may need to be addressed. Once a revised manuscript is re-submitted, it is assessed along with the responses to reviewer comments and if it has been adequately revised, it will be accepted for publication. Accepted manuscripts are then copyedited and typeset by the production team before online publication. Appeals against decisions following peer review are considered on a case-by-case basis and should be sent to the journal editor, and authors are welcome to make rebuttals against individual reviewer comments, if appropriate. Preprints We encourage posting of preprints of primary research manuscripts on preprint servers, authors'' or institutional websites, and open communications between researchers whether on community preprint servers or preprint commenting platforms. Posting of preprints is not considered prior publication and will not jeopardize consideration in our journals. Please see here for further information on preprint sharing: https://www.springer.com/gp/authors-editors/journal-author/journal-author-helpdesk/submission/1302#c16721550 Copyright Neurology and Therapy is published under the Creative Commons Attribution-Noncommercial License, which allows users to read, copy, distribute, and make derivative works for non-commercial purposes from the material, as long as the author of the original work is cited. The author assigns the exclusive right to any commercial use of the article to Springer. For more information about the Creative Commons Attribution-Noncommercial License, click here: http://creativecommons.org/licenses/by-nc/4.0. Contact For more information about the journal, including pre-submission enquiries, please contact managing editor Lydia Alborn at lydia.alborn@springer.com.
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