Advancing Future Amyotrophic Lateral Sclerosis Medicines by Incorporating The Patient Voice Into Patient-Centered Holistic Measurement Strategies for Clinical and Real-World Studies: Results from Targeted Literature Reviews.

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-05-12 DOI:10.1007/s40120-025-00740-y

Diana Rofail, Michael Chladek, Betsy Williams, Nick Patel, William B Nowell, Stella Karantzoulis, Oren Levy

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引用次数: 0

Abstract

Introduction: This analysis sought to understand the patient experience in amyotrophic lateral sclerosis (ALS) and to assess whether commonly used clinical outcome assessments (COAs) reliably and validly capture that experience.

Methods: Two targeted literature reviews were conducted to identify and describe key concepts potentially important to patients (signs, symptoms, impacts), and identify commonly used COAs in ALS. Insights gained were used to map target COAs to concepts identified as potentially relevant to patients and their caregivers. COAs of interest were further examined to evaluate evidence of their validity and reliability within ALS.

Results: Forty-three articles were identified for concept extraction. Signs and symptoms were identified across multiple themes: motor; non-motor; respiratory; cognitive; and behavioral. Patient impacts were identified across multiple themes: physical; functional; emotional; social; and other aspects of well-being. Caregiver impacts were identified across four themes: general; emotional; social; and physical. Of 236 unique COAs identified, 6 were found to provide the greatest coverage of potentially important concepts. Closer examination of these showed some evidence gaps supporting content validity and/or psychometric properties.

Conclusions: Several concepts related to ALS were identified that are relevant to patients in their daily lives. We identified and reviewed COAs commonly used in assessing these concepts, and found gaps in their content validity and/or psychometric properties. These findings suggest the need for further testing/refinement of existing tools, and the opportunity to use other instruments alongside those most frequently used (e.g., ALSFRS-R) to comprehensively capture the patient experience of ALS in future clinical trial and real-world studies.

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通过将患者声音纳入临床和现实世界研究中以患者为中心的整体测量策略，推进未来肌萎缩性侧索硬化药物：目标文献综述的结果。

本分析旨在了解肌萎缩性侧索硬化症（ALS）患者的经历，并评估常用的临床结果评估（coa）是否可靠和有效地捕捉到这种经历。方法：进行两项有针对性的文献综述，以确定和描述对患者潜在重要的关键概念（体征、症状、影响），并确定ALS中常用的coa。获得的见解用于将目标coa映射到确定为与患者及其护理人员潜在相关的概念。进一步检查感兴趣的coa以评估其在ALS中的有效性和可靠性的证据。结果：共识别43篇文章进行概念提取。在多个主题中确定了体征和症状：运动；非机动车;呼吸系统;认知;和行为。在多个主题中确定了患者的影响：身体；功能;情感;社会;以及幸福的其他方面。在四个主题中确定了照顾者的影响：一般；情感;社会;和物理。在确定的236个独特coa中，发现有6个提供了最大范围的潜在重要概念。更仔细的检查显示了一些支持内容效度和/或心理测量特性的证据差距。结论：确定了与ALS相关的几个概念，这些概念与患者的日常生活有关。我们确定并回顾了通常用于评估这些概念的coa，并发现了其内容有效性和/或心理测量特性的差距。这些发现表明，需要进一步测试/改进现有工具，并有机会在最常用的工具（如ALSFRS-R）之外使用其他工具，以便在未来的临床试验和实际研究中全面捕捉ALS患者的体验。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

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.