Amyotrophic lateral sclerosis & frontotemporal degeneration最新文献

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Impact of apathy over the course of disease in amyotrophic lateral sclerosis. 在肌萎缩性侧索硬化症中冷漠对病程的影响。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-04-29 DOI: 10.1080/21678421.2025.2495020
Ratko Radakovic, Debbie Gray, Ana Paula Trucco, Allan Bregola, Eneida Mioshi, Helen Copsey, David Dick, Judith Newton, Shuna Colville, Suvankar Pal, Siddharthan Chandran, Zachary Simmons, Sharon Abrahams
{"title":"Impact of apathy over the course of disease in amyotrophic lateral sclerosis.","authors":"Ratko Radakovic, Debbie Gray, Ana Paula Trucco, Allan Bregola, Eneida Mioshi, Helen Copsey, David Dick, Judith Newton, Shuna Colville, Suvankar Pal, Siddharthan Chandran, Zachary Simmons, Sharon Abrahams","doi":"10.1080/21678421.2025.2495020","DOIUrl":"10.1080/21678421.2025.2495020","url":null,"abstract":"<p><p><i>Objective</i>: Apathy is a common syndrome in amyotrophic lateral sclerosis (ALS), particularly Initiation apathy (lack of motivation for self-generated thoughts and/or actions). The aim was to determine how apathy subtypes change over time, and their impact on individuals' quality of life (QoL), caregiver-wellbeing and burden or strain. <i>Methods</i>: Forty-nine people living with ALS (pwALS) and their caregiver participated in interviews at three time-points (3-month intervals). They completed the Dimensional Apathy Scale (DAS), and assessments of depression, anxiety and emotional lability, cognitive-behavioral functioning and functional disability. PwALS QoL, caregiver burden or strain, caregiver-wellbeing and care-related QoL were measured. <i>Results</i>: At baseline, Initiation apathy was most common (38.8%, <i>N</i> = 19) followed by Emotional apathy (16.3%, <i>N</i> = 8). Lower caregiver-wellbeing was observed in Initiation apathy (<i>p</i> < 0.05) and Mixed-emotional apathy (<i>p</i> < 0.001) groups, where only Initiation apathy had higher caregiver burden or strain (<i>p</i> < 0.05) than those with no apathy. Over three visits (<i>N</i> = 31), there was an increase in Initiation apathy (<i>p</i> < 0.01) and Executive apathy (<i>p</i> < 0.05) over time. While controlling for functional disability, only increasing Emotional apathy was associated with increasing caregiver burden or strain (<i>p</i> < 0.05), decreasing caregiver-wellbeing (<i>p</i> < 0.001), and decreasing care-related QoL (<i>p</i> < 0.05). <i>Conclusion</i>: Initiation and Emotional apathy were variably associated with higher levels of caregiver burden or strain and decreased caregiver-wellbeing in ALS. As ALS progresses, Initiation and Executive apathy increased, while Emotional apathy has been shown to impact care-related QoL, caregiver-wellbeing and burden or strain. This has implications for understanding the progression of apathy subtypes and the interplay of caregiver-wellbeing, QoL, burden, or strain.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"516-525"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144058366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
ALSUntangled #78: Zinc. ALSUntangled #78:锌。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-03-14 DOI: 10.1080/21678421.2025.2476688
Benjamin Helmold, George Nathaniel, Paul Barkhaus, Tulio Bertorini, Mark Bromberg, Andrew Brown, Gregory T Carter, Vincent Chang, Jesse Crayle, Keelie Denson, Jonathan Glass, Terry Heiman-Patterson, Esther Hobson, Carlayne Jackson, Sartaj Jhooty, Elise Mallon, Nicholas Maragakis, Javier Mascias Cadavid, Christopher Mcdermott, Gary Pattee, Kaitlyn Pierce, Olivia Wang, Paul Wicks, Richard Bedlack
{"title":"ALSUntangled #78: Zinc.","authors":"Benjamin Helmold, George Nathaniel, Paul Barkhaus, Tulio Bertorini, Mark Bromberg, Andrew Brown, Gregory T Carter, Vincent Chang, Jesse Crayle, Keelie Denson, Jonathan Glass, Terry Heiman-Patterson, Esther Hobson, Carlayne Jackson, Sartaj Jhooty, Elise Mallon, Nicholas Maragakis, Javier Mascias Cadavid, Christopher Mcdermott, Gary Pattee, Kaitlyn Pierce, Olivia Wang, Paul Wicks, Richard Bedlack","doi":"10.1080/21678421.2025.2476688","DOIUrl":"10.1080/21678421.2025.2476688","url":null,"abstract":"<p><p>ALSUntangled reviews alternative and off-label treatments for people living with amyotrophic lateral sclerosis (PALS). In this review, we assess the utilization of dietary zinc supplements for modulating ALS pathology and progression. Studies in mouse models of ALS have demonstrated that high-dose zinc supplementation may be harmful, but moderate doses could potentially be beneficial. Clinical data is limited, and only one trial has explored zinc supplementation within PALS. This study reported potential benefits in slowing ALS progression but lacked statistical analyses and failed to report quantitative evidence. Numerous case reports from individual patients at varying doses have demonstrated no benefit. Zinc supplements at moderate doses are generally low cost and not associated with severe complications, but further research is required to determine the safety and efficacy of zinc supplementation within PALS. Therefore, we cannot at this time, endorse zinc supplementation to slow ALS progression.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"599-603"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143626930","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Time-to-event prediction in ALS using a landmark modeling approach, using the ALS Natural History Consortium dataset. 使用里程碑建模方法的ALS事件时间预测,使用ALS自然历史联盟数据集。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-04-02 DOI: 10.1080/21678421.2025.2482943
David Schneck, Andres Arguedas, Annette Xenopoulos-Oddsson, Ximena Arcila-Londono, Christian Lunetta, James Wymer, Nicholas Olney, Kelly Gwathmey, Senda Ajroud-Driss, Ghazala Hayat, Terry Heiman-Patterson, Federica Cerri, Christina Fournier, Jonathan Glass, Alex Sherman, Mark Fiecas, David Walk
{"title":"Time-to-event prediction in ALS using a landmark modeling approach, using the ALS Natural History Consortium dataset.","authors":"David Schneck, Andres Arguedas, Annette Xenopoulos-Oddsson, Ximena Arcila-Londono, Christian Lunetta, James Wymer, Nicholas Olney, Kelly Gwathmey, Senda Ajroud-Driss, Ghazala Hayat, Terry Heiman-Patterson, Federica Cerri, Christina Fournier, Jonathan Glass, Alex Sherman, Mark Fiecas, David Walk","doi":"10.1080/21678421.2025.2482943","DOIUrl":"10.1080/21678421.2025.2482943","url":null,"abstract":"<p><strong>Background and objectives: </strong>Times to clinically relevant events are a valuable outcome in observational and interventional studies, complementing linear outcomes such as functional rating scales and biomarkers. In ALS, there are several clinically relevant events. We developed dynamic prediction models for several of these times to events that can be used for clinical trial modeling and personal planning.</p><p><strong>Methods: </strong>Landmark time-to-event analysis was implemented to determine the effect of patient characteristics on disease progression. Longitudinal data from 1557 participants in the ALS Natural History Consortium dataset were used. Five outcomes in the ALS disease progression were considered: loss of ambulation, loss of speech, gastrostomy, noninvasive ventilation (NIV) use, and continuous NIV use. Covariates in our models include age at diagnosis, sex, onset location, riluzole use, diagnostic delay, ALSFRS-R scores at the landmark time, and ALSFRS-R rates of change from baseline. Internal and external validation techniques were used.</p><p><strong>Results: </strong>For each of our models and landmark times, we present risk prediction intervals for random sets of patient characteristics. We demonstrate our models' application for an individual's personal predicted time-to-event. Our internal and external validation metrics indicate good concordance and overall performance. The time to loss of speech models perform the best for each metric in terms of both internal and external validation.</p><p><strong>Discussion: </strong>Landmarking is an efficient, individualized risk prediction model that is intuitive for both clinicians and patients. Importantly, landmarking can be used for clinical trial modeling, personal planning, and development of real-world evidence of the impacts of treatment interventions.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"417-425"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143765909","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Further development of a patient-reported outcome measure to assess the impact of oral secretion problems in people living with MND. 进一步发展患者报告的结果测量,以评估口腔分泌物问题对MND患者的影响。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-03-10 DOI: 10.1080/21678421.2025.2469721
Sarah L Boddy, Rebecca M Simpson, Stephen J Walters, Theresa Walsh, Christopher J McDermott
{"title":"Further development of a patient-reported outcome measure to assess the impact of oral secretion problems in people living with MND.","authors":"Sarah L Boddy, Rebecca M Simpson, Stephen J Walters, Theresa Walsh, Christopher J McDermott","doi":"10.1080/21678421.2025.2469721","DOIUrl":"10.1080/21678421.2025.2469721","url":null,"abstract":"<p><p><i>Objective:</i> Oral secretion problems are common yet poorly managed in people living with MND (plwMND). A validated patient-reported outcome for measuring saliva symptoms in this patient group would facilitate better monitoring of individuals. This study aimed to assess the validity, reliability and sensitivity to change of a revised version of the clinical saliva score for MND (CSS-MNDr). <i>Methods:</i> Data were collected as part of a longitudinal, observational saliva management study. The CSS-MNDr, ALS Functional Rating Scale, a Global Rating of Change questionnaire and saliva-specific modified Likert scale were completed at each study visit, each of which probed the severity of saliva symptoms. Construct validity, test-retest reliability and sensitivity of the CSS-MNDr were assessed and the minimal important difference of the instrument was estimated. <i>Results:</i> The CSS-MNDr showed excellent test-retest reliability (intraclass correlation coefficient >0.9). Construct validity showed the CSS-MNDr performed as expected, with bulbar-onset participants scoring significantly higher than those who reported limb-onset across all visits (group mean scores). Strong correlation of total scores with the ALSFRS-R saliva question was demonstrated (-0.8), with the thick subscore correlating less well (-0.5). A minimal important difference in the range of -2.5 to -3.6 over 3 months was estimated for worsening symptoms. <i>Conclusions:</i> The CSS-MNDr has been validated as a reliable patient reported outcome for measuring saliva problems in plwMND. With separate scores for thick and thin secretion problems, the CSS-MNDr is the most comprehensive tool for assessing salivary problems in plwMND reported to date.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"507-515"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143598350","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Respiratory measurements, respiratory symptoms, and quality of life in ALS: results from the REVEALS study. ALS患者的呼吸测量、呼吸症状和生活质量:来自reveal研究的结果
IF 2.8
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-03-06 DOI: 10.1080/21678421.2025.2471421
Deirdre Murray, James Rooney, Dara Meldrum, Ammar Al-Chalabi, Tommy M Bunte, Theresa Chiwera, Mutahhara Choudhury, Adriano Chio, Lauren Fenton, Jennifer Fortune, Lindsay Maidment, Umberto Manera, Christopher J McDermott, Myrte Meyjes, Rachel Tattersall, Maria Claudia Torrieri, Philip Van Damme, Elien Vanderlinden, Claire Wood, Leonard H Van Den Berg, Orla Hardiman
{"title":"Respiratory measurements, respiratory symptoms, and quality of life in ALS: results from the REVEALS study.","authors":"Deirdre Murray, James Rooney, Dara Meldrum, Ammar Al-Chalabi, Tommy M Bunte, Theresa Chiwera, Mutahhara Choudhury, Adriano Chio, Lauren Fenton, Jennifer Fortune, Lindsay Maidment, Umberto Manera, Christopher J McDermott, Myrte Meyjes, Rachel Tattersall, Maria Claudia Torrieri, Philip Van Damme, Elien Vanderlinden, Claire Wood, Leonard H Van Den Berg, Orla Hardiman","doi":"10.1080/21678421.2025.2471421","DOIUrl":"10.1080/21678421.2025.2471421","url":null,"abstract":"<p><p><i>Objective</i>: Progressing respiratory weakness throughout the course of amyotrophic lateral sclerosis (ALS) is clinically associated with distressing symptoms, including dyspnea, orthopnea, and difficulty clearing secretions. Fatigue, poor sleep, and reduced quality of life are also considered to be associated with declining respiratory function. Respiratory measurements guide prescription of interventions, which aim to alleviate symptoms. The relationships between respiratory measurements and patient reported symptoms are currently unclear. <i>Method</i>: The REVEALS study was a longitudinal, observational, multisite study of decline in respiratory function in people with ALS attending six European centers. Respiratory measures (forced and slow vital capacity (F/SVC), sniff nasal inspiratory pressure (SNIP), and peak cough flow) were collected, as were the presence of respiratory symptoms and simple quality of life, fatigue and sleep measures. We used Bayesian's multivariate models to explore the associations of the respiratory measures with outcome variables. <i>Results</i>: Two hundred and eighty participants completed in-person assessments over a median of 8 (IQR 2.3, 14.1) months, with 974 data collection timepoints. The probability of reporting symptoms including dyspnea, orthopnea, and difficulty clearing secretions increased with decreasing respiratory measurement scores. The probability of reporting moderately low quality of life and moderate fatigue also increased with decreasing test scores, but reported sleep quality was not associated with respiratory scores. <i>Conclusion</i>: Respiratory weakness in people with ALS was associated with symptoms including dyspnea, orthopnea, and difficulty clearing secretions. The probability of reporting symptoms increased incrementally as respiratory weakness increased, supporting the use of both respiratory measurements and the presence of symptoms in making decisions about clinical interventions.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"467-477"},"PeriodicalIF":2.8,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143569013","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The contralateral co-movement test in a Chinese population with amyotrophic lateral sclerosis. 中国肌萎缩性侧索硬化症患者的对侧联合运动试验。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-02-24 DOI: 10.1080/21678421.2025.2467959
Jieying Wu, Can Sun, Yingsheng Xu, Dongsheng Fan, Shan Ye
{"title":"The contralateral co-movement test in a Chinese population with amyotrophic lateral sclerosis.","authors":"Jieying Wu, Can Sun, Yingsheng Xu, Dongsheng Fan, Shan Ye","doi":"10.1080/21678421.2025.2467959","DOIUrl":"10.1080/21678421.2025.2467959","url":null,"abstract":"<p><strong>Introduction: </strong>Mirror movements (MMs) are often overlooked in patients with amyotrophic lateral sclerosis (ALS). Although the contralateral co-movement (COMO) test can be used to evaluate MMs in patients with ALS, it lacks a systematic evaluation. The aim of this study was to validate the effectiveness of the Chinese version of the COMO test in a Chinese ALS population.</p><p><strong>Methods: </strong>We prospectively enrolled 173 patients with ALS as the disease group and 28 healthy individuals as controls. All participants were evaluated using the Chinese version of the COMO test. Univariate analysis and multiple linear regression were used to compare differences between groups. Subgroup analysis of the COMO scores was performed based on different disease characteristics.</p><p><strong>Results: </strong>The COMO score in the ALS group was significantly greater (5.00% [1.67-10.00]) than that in the healthy control group (1.67% [0.00-3.33]). After adjusting for confounders, this difference remained significant. Multivariate linear analysis suggested that the upper motor neuron (UMN) score independently predicted the COMO score (<i>P</i> < 0.001). The COMO score was not affected by different onset regions or lateralizations. Propensity score matching revealed no significant difference in COMO scores between uninvolved limb segments and the corresponding limb segments in other patients. The Cronbach's α of the Chinese COMO test was 0.621.</p><p><strong>Conclusion: </strong>The Chinese COMO test can serve as a potential tool for assessing MMs in Chinese patients with ALS. The UMN score is a factor influencing the COMO score. The COMO test can provide objective evidence for ALS characteristics and the severity of UMN damage.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"426-435"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143484627","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Minimum clinically important difference for drug effectiveness in an area of patient-oriented therapeutic goals in amyotrophic lateral sclerosis. 在肌萎缩性侧索硬化症以患者为导向的治疗目标领域中,药物有效性的最小临床重要差异。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-03-11 DOI: 10.1080/21678421.2025.2475893
Adriano Chiò, Juliette Foucher, Kelly G Gwathmey, Caroline Ingre
{"title":"Minimum clinically important difference for drug effectiveness in an area of patient-oriented therapeutic goals in amyotrophic lateral sclerosis.","authors":"Adriano Chiò, Juliette Foucher, Kelly G Gwathmey, Caroline Ingre","doi":"10.1080/21678421.2025.2475893","DOIUrl":"10.1080/21678421.2025.2475893","url":null,"abstract":"<p><p><i>Objective:</i> In this review, we will examine the more common endpoints incorporated in randomized controlled trials (RCTs) and their strength of evidence, focusing on the definition of what constitutes a clinically meaningful change. We will also reflect on the perspective of patients and their families regarding the design of RCTs in amyotrophic lateral sclerosis (ALS). <i>Methods:</i> Authors performed a scoping review of the literature around clinical meaningfulness in the ALS field and the minimum clinically important difference to deem a treatment effective. <i>Results:</i> The use of survival as an RCT endpoint, as well as the ALS functional rating scale-revised slope, has been criticized, and their relevance for patients remains debated. Biomarkers are promising alternatives as surrogate endpoints, but currently, only cerebrospinal fluid and plasma neurofilaments have emerged as reliable and sensitive biomarkers of disease progression. Incorporating patients' preferences and priorities for their care when treatments are selected is important to minimize the burden of care and limit the potential harms of overtreatment. Patients' interest in and acceptance of a new therapy is also determined by its impact on their quality of life. <i>Discussion and conclusion:</i> While scientifically sound trials must be conducted, this must be balanced with patient expectations of limiting trial burden, duration and placebo usage. An important approach in uniting these diverging needs is the inclusion of people with ALS and their organizations to advise in the design and execution of clinical trials, facilitating the design of RCTs more focused on patients' expectations while retaining a high scientific rigor.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"389-398"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606731","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Amyotrophic lateral sclerosis and lovastatin: a promising treatment perspective. 肌萎缩性侧索硬化症和洛伐他汀:一个有希望的治疗前景。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-02-15 DOI: 10.1080/21678421.2025.2463943
Zain Ali Nadeem, Sophia Ahmed
{"title":"Amyotrophic lateral sclerosis and lovastatin: a promising treatment perspective.","authors":"Zain Ali Nadeem, Sophia Ahmed","doi":"10.1080/21678421.2025.2463943","DOIUrl":"10.1080/21678421.2025.2463943","url":null,"abstract":"","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"595-596"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426603","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Self-assessment of amyotrophic lateral sclerosis functional rating scale on the patient's smartphone proves to be non-inferior to clinic data capture. 肌萎缩性侧索硬化症功能评定量表在患者智能手机上的自我评估证明不逊于临床数据采集。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-02-22 DOI: 10.1080/21678421.2025.2468404
Laura Steinfurth, Torsten Grehl, Ute Weyen, Dagmar Kettemann, Robert Steinbach, Annekathrin Rödiger, Julian Grosskreutz, Susanne Petri, Matthias Boentert, Patrick Weydt, Sarah Bernsen, Bertram Walter, René GüNTHER, Paul Lingor, Jan Christoph Koch, Petra Baum, Jochen H Weishaupt, Johannes Dorst, Yasemin Koc, Isabell Cordts, Maximilian Vidovic, Jenny Norden, Peggy Schumann, Péter Körtvélyessy, Susanne Spittel, Christoph Münch, André Maier, Thomas Meyer
{"title":"Self-assessment of amyotrophic lateral sclerosis functional rating scale on the patient's smartphone proves to be non-inferior to clinic data capture.","authors":"Laura Steinfurth, Torsten Grehl, Ute Weyen, Dagmar Kettemann, Robert Steinbach, Annekathrin Rödiger, Julian Grosskreutz, Susanne Petri, Matthias Boentert, Patrick Weydt, Sarah Bernsen, Bertram Walter, René GüNTHER, Paul Lingor, Jan Christoph Koch, Petra Baum, Jochen H Weishaupt, Johannes Dorst, Yasemin Koc, Isabell Cordts, Maximilian Vidovic, Jenny Norden, Peggy Schumann, Péter Körtvélyessy, Susanne Spittel, Christoph Münch, André Maier, Thomas Meyer","doi":"10.1080/21678421.2025.2468404","DOIUrl":"10.1080/21678421.2025.2468404","url":null,"abstract":"<p><strong>Objective: </strong>To investigate self-assessment of the amyotrophic lateral sclerosis functional rating scale-revised (ALSFRS-R) using the patient's smartphone and to analyze non-inferiority to clinic assessment.</p><p><strong>Methods: </strong>In an observational study, ALSFRS-R data being remotely collected on a mobile application (App-ALSFRS-R) were compared to ALSFRS-R captured during clinic visits (clinic-ALSFRS-R). ALS progression rate (ALSPR)-as calculated by the monthly decline of ALSFRS-R-and its intrasubject variability (ALSPR-ISV) between ratings were used to compare both cohorts. To investigate non-inferiority of App-ALSFRS-R data, a non-inferiority margin was determined.</p><p><strong>Results: </strong>A total of 691 ALS patients using the ALS-App and 1895 patients with clinic assessments were included. Clinical characteristics for the App-ALSFRS-R and clinic-ALSFRS-R cohorts were as follows: Mean age 60.45 (SD 10.43) and 63.69 (SD 11.30) years (<i>p</i> < 0.001), disease duration 38.7 (SD 37.68) and 56.75 (SD 54.34) months (<i>p</i> < 0.001) and ALSPR 0.72 and 0.59 (<i>p</i> < 0.001), respectively. A paired sample analysis of ALSPR-ISV was applicable for 398 patients with clinic as well as app assessments and did not show a significant difference (IQR 0.12 [CI 0.11, 0.14] vs 0.12 [CI 0.11, 0.14], <i>p</i> = 0.24; Cohen's d = 0.06). CI of IQR for App-ALSFRS-R was below the predefined non-inferiority margin of 0.15 IQR, demonstrating non-inferiority.</p><p><strong>Conclusions: </strong>Patients using a mobile application for remote digital self-assessment of the ALSFRS-R revealed younger age, earlier disease course, and faster ALS progression. The finding of non-inferiority of App-ALSFRS-R assessments underscores, that data collection using the ALS-App on the patient's smartphone can serve as additional source of ALSFRS-R in ALS research and clinical practice.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"495-506"},"PeriodicalIF":0.0,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143477181","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Exploring oculomotor challenges in amyotrophic lateral sclerosis: a comprehensive review. 探索肌萎缩性侧索硬化症的动眼肌挑战:全面回顾。
Amyotrophic lateral sclerosis & frontotemporal degeneration Pub Date : 2025-08-01 Epub Date: 2025-05-09 DOI: 10.1080/21678421.2025.2501690
Dongchao Shen, Anfeng Liu, Xunzhe Yang, Qing Liu, Mingsheng Liu, Liying Cui
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引用次数: 0
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