{"title":"Correction to “The Digital Frontier in Huntington's Disease: Opportunities for Clinical Trials”","authors":"","doi":"10.1002/mds.70126","DOIUrl":"https://doi.org/10.1002/mds.70126","url":null,"abstract":"","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"178 1","pages":""},"PeriodicalIF":8.6,"publicationDate":"2025-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145535248","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
<p>I read with interest the recent article by Ikezawa et al. on unilateral magnetic resonance-guided focused ultrasound (MRgFUS) pallidothalamic tractotomy for Parkinson's disease.<span><sup>1</sup></span> The authors demonstrated meaningful motor improvements (including bilateral benefits) after unilateral lesion in Forel's field H1. I wish however, to raise a constructive caution regarding the interpretation of these results. In particular, the lack of detailed lesion verification (notably the absence of diffusion tensor imaging to confirm tract-specific targeting) leaves uncertainty as to whether the clinical effects can be attributed to selective disruption of the intended pathway alone. Without advanced neuroimaging validation, one must be careful in drawing mechanistic conclusions about ‘pallidothalamic tractotomy’ in isolation.</p><p>The subthalamic region targeted in this procedure is an anatomically complex and crowded crossroads of structures and fiber pathways.<span><sup>2-5</sup></span> The classic fields of Forel in the posterior subthalamic area form a narrow ‘bottleneck’ through which multiple myelinated tracts converge to the thalamus. These include the pallidothalamic fibers (ansa lenticularis and lenticular fasciculus merging into Forel's field H1), but also the cerebellothalamic tract running just posterior to the pallidal fibers, as well as nigrothalamic projections from the substantia nigra coursing in parallel. In addition, key gray matter nuclei (zona incerta [ZI] and subthalamic nucleus) lie immediately adjacent to these tracts. Notably, the ZI lies between the lenticular and thalamic fasciculi, in extremely close proximity to the pallidothalamic bundle. Recent anatomical and surgical reviews of the posterior subthalamic area underscore this intricate anatomy and the challenge of achieving a truly selective lesion highlighting the potential for off-target effects if a lesion spreads beyond the intended bundle.<span><sup>2</sup></span> Likewise, Horisawa et al. noted that ablating the pallidothalamic zone in this region often inevitably involves or affects the ZI, making it “difficult to distinguish” which structure's disruption is actually responsible for clinical benefits.<span><sup>5</sup></span> This issue is highly pertinent to interpreting the results of Ikezawa et al., since without tract-specific imaging we cannot be certain whether their MRgFUS lesion was confined to the pallidothalamic tract as presumed, or whether it also partially lesioned adjacent fiber tracts or nearby nuclei.<span><sup>1</sup></span> In short, the subthalamic target area's complexity demands modern imaging confirmation to validate that the ‘pallidothalamic tractotomy’ is anatomically what its name implies.</p><p>The authors reasonably hypothesize that interrupting pallidal outflow fibers led to the beneficial outcomes. However, without supportive neuroimaging evidence this remains an inference. For example, one could argue that a lesion exten
{"title":"Unilateral Magnetic Resonance-Guided Focused Ultrasound (MRgFUS) Pallidothalamic Tractotomy in Parkinson's Disease: Promising Results But Uncertain Target","authors":"Mickael Aubignat MD, MSc","doi":"10.1002/mds.70131","DOIUrl":"10.1002/mds.70131","url":null,"abstract":"<p>I read with interest the recent article by Ikezawa et al. on unilateral magnetic resonance-guided focused ultrasound (MRgFUS) pallidothalamic tractotomy for Parkinson's disease.<span><sup>1</sup></span> The authors demonstrated meaningful motor improvements (including bilateral benefits) after unilateral lesion in Forel's field H1. I wish however, to raise a constructive caution regarding the interpretation of these results. In particular, the lack of detailed lesion verification (notably the absence of diffusion tensor imaging to confirm tract-specific targeting) leaves uncertainty as to whether the clinical effects can be attributed to selective disruption of the intended pathway alone. Without advanced neuroimaging validation, one must be careful in drawing mechanistic conclusions about ‘pallidothalamic tractotomy’ in isolation.</p><p>The subthalamic region targeted in this procedure is an anatomically complex and crowded crossroads of structures and fiber pathways.<span><sup>2-5</sup></span> The classic fields of Forel in the posterior subthalamic area form a narrow ‘bottleneck’ through which multiple myelinated tracts converge to the thalamus. These include the pallidothalamic fibers (ansa lenticularis and lenticular fasciculus merging into Forel's field H1), but also the cerebellothalamic tract running just posterior to the pallidal fibers, as well as nigrothalamic projections from the substantia nigra coursing in parallel. In addition, key gray matter nuclei (zona incerta [ZI] and subthalamic nucleus) lie immediately adjacent to these tracts. Notably, the ZI lies between the lenticular and thalamic fasciculi, in extremely close proximity to the pallidothalamic bundle. Recent anatomical and surgical reviews of the posterior subthalamic area underscore this intricate anatomy and the challenge of achieving a truly selective lesion highlighting the potential for off-target effects if a lesion spreads beyond the intended bundle.<span><sup>2</sup></span> Likewise, Horisawa et al. noted that ablating the pallidothalamic zone in this region often inevitably involves or affects the ZI, making it “difficult to distinguish” which structure's disruption is actually responsible for clinical benefits.<span><sup>5</sup></span> This issue is highly pertinent to interpreting the results of Ikezawa et al., since without tract-specific imaging we cannot be certain whether their MRgFUS lesion was confined to the pallidothalamic tract as presumed, or whether it also partially lesioned adjacent fiber tracts or nearby nuclei.<span><sup>1</sup></span> In short, the subthalamic target area's complexity demands modern imaging confirmation to validate that the ‘pallidothalamic tractotomy’ is anatomically what its name implies.</p><p>The authors reasonably hypothesize that interrupting pallidal outflow fibers led to the beneficial outcomes. However, without supportive neuroimaging evidence this remains an inference. For example, one could argue that a lesion exten","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"41 1","pages":"269-270"},"PeriodicalIF":7.6,"publicationDate":"2025-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://movementdisorders.onlinelibrary.wiley.com/doi/epdf/10.1002/mds.70131","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145535284","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Andrea Quattrone MD, PhD, Nicolai Franzmeier PhD, Günter U. Höglinger MD
{"title":"Reply to: Utility of Progression Rate Since Symptom Onset in Predicting Subsequent Survival in Progressive Supranuclear Palsy","authors":"Andrea Quattrone MD, PhD, Nicolai Franzmeier PhD, Günter U. Höglinger MD","doi":"10.1002/mds.70124","DOIUrl":"10.1002/mds.70124","url":null,"abstract":"","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"41 1","pages":"279-280"},"PeriodicalIF":7.6,"publicationDate":"2025-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145532098","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
<p>The search for markers of progressive supranuclear palsy (PSP) progression is important to neuroprotective clinical trial design and for patient counseling and disease management. We were therefore disappointed by the outcome of the excellent study by Quattrone et al,<span><sup>1</sup></span> “Brain Atrophy Does Not Predict Clinical Progression in Progressive Supranuclear Palsy.” The authors found that neither clinical severity nor regional magnetic resonance imaging volumes predicted individual 12-month trajectories in patients with PSP-Richardson's syndrome.</p><p>That and most other attempts to model 12-month PSP progression have used only baseline variables, but we have assessed the contribution of onset-to-baseline progression rates. First, in a pathologically verified PSP cohort, we found an association between the time from symptom onset to the first examination showing downgaze palsy (ie, scores >0 on PSP Rating Scale item 15) and overall survival duration.<span><sup>2</sup></span> Next, via logistic regression in a prospective PSP cohort, we found that survival was associated with onset age and the progression rate of downgaze palsy as reflected by downgaze palsy severity and years since clinical onset.<span><sup>3</sup></span> This conclusion was further validated in a much larger independent PSP cohort.<span><sup>4</sup></span> We also similarly calculated a progression rate for other aspects of PSP, finding that the progression rate for dysphagia for liquids (PSPRS item 13) was associated with survival duration as well.<span><sup>4, 5</sup></span></p><p>We therefore suggest that models to predict progression and survival in PSP include not only baseline findings but also progression rate of downgaze impairment and dysphagia from onset to baseline. As more compounds become candidates for advanced-phase trials, innovations in trial design using such predictive models could reduce costs and improve statistical power to detect efficacy with fewer experimental subjects or shorter timelines.<span><sup>6</sup></span></p><p>(1) Research Project: A. Conception, B. Organization, C. Execution; (2) Statistical Analysis: A. Design, B. Execution, C. Review and Critique; (3) Manuscript: A. Writing of the First Draft, B. Review and Critique.</p><p>T.X.: 1A, 1B, 1C, 3A, 3B.</p><p>L.I.G.: 1A, 1B, 1C, 3B.</p><p>TX: Stock Ownership in medically-related fields: None. Intellectual Property Rights: None.</p><p>Consultancies: CVS Caremark. Expert Testimony: None. Advisory Boards: None. Employment: The University of Chicago Medicine. Partnerships: None. Inventions: None. Contracts: None. Honoraria: Parkinson's Foundation, Ono Pharmaceutical Co., Ltd, CVS Caremark. Royalties: None. Patents: None. Grants: The Michael J. Fox Foundation for Parkinson's Research, American Parkinson's Disease Association, and NIH. Other: None. L.I.G.: Stock Ownership in medically-related fields: None. Intellectual Property Rights: None. Consultancies: AI Therapeutics, Amylyx,
{"title":"Utility of Progression Rate Since Symptom Onset in Predicting Subsequent Survival in Progressive Supranuclear Palsy","authors":"Tao Xie MD, PhD, Lawrence I. Golbe MD","doi":"10.1002/mds.70125","DOIUrl":"10.1002/mds.70125","url":null,"abstract":"<p>The search for markers of progressive supranuclear palsy (PSP) progression is important to neuroprotective clinical trial design and for patient counseling and disease management. We were therefore disappointed by the outcome of the excellent study by Quattrone et al,<span><sup>1</sup></span> “Brain Atrophy Does Not Predict Clinical Progression in Progressive Supranuclear Palsy.” The authors found that neither clinical severity nor regional magnetic resonance imaging volumes predicted individual 12-month trajectories in patients with PSP-Richardson's syndrome.</p><p>That and most other attempts to model 12-month PSP progression have used only baseline variables, but we have assessed the contribution of onset-to-baseline progression rates. First, in a pathologically verified PSP cohort, we found an association between the time from symptom onset to the first examination showing downgaze palsy (ie, scores >0 on PSP Rating Scale item 15) and overall survival duration.<span><sup>2</sup></span> Next, via logistic regression in a prospective PSP cohort, we found that survival was associated with onset age and the progression rate of downgaze palsy as reflected by downgaze palsy severity and years since clinical onset.<span><sup>3</sup></span> This conclusion was further validated in a much larger independent PSP cohort.<span><sup>4</sup></span> We also similarly calculated a progression rate for other aspects of PSP, finding that the progression rate for dysphagia for liquids (PSPRS item 13) was associated with survival duration as well.<span><sup>4, 5</sup></span></p><p>We therefore suggest that models to predict progression and survival in PSP include not only baseline findings but also progression rate of downgaze impairment and dysphagia from onset to baseline. As more compounds become candidates for advanced-phase trials, innovations in trial design using such predictive models could reduce costs and improve statistical power to detect efficacy with fewer experimental subjects or shorter timelines.<span><sup>6</sup></span></p><p>(1) Research Project: A. Conception, B. Organization, C. Execution; (2) Statistical Analysis: A. Design, B. Execution, C. Review and Critique; (3) Manuscript: A. Writing of the First Draft, B. Review and Critique.</p><p>T.X.: 1A, 1B, 1C, 3A, 3B.</p><p>L.I.G.: 1A, 1B, 1C, 3B.</p><p>TX: Stock Ownership in medically-related fields: None. Intellectual Property Rights: None.</p><p>Consultancies: CVS Caremark. Expert Testimony: None. Advisory Boards: None. Employment: The University of Chicago Medicine. Partnerships: None. Inventions: None. Contracts: None. Honoraria: Parkinson's Foundation, Ono Pharmaceutical Co., Ltd, CVS Caremark. Royalties: None. Patents: None. Grants: The Michael J. Fox Foundation for Parkinson's Research, American Parkinson's Disease Association, and NIH. Other: None. L.I.G.: Stock Ownership in medically-related fields: None. Intellectual Property Rights: None. Consultancies: AI Therapeutics, Amylyx,","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"41 1","pages":"277-278"},"PeriodicalIF":7.6,"publicationDate":"2025-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://movementdisorders.onlinelibrary.wiley.com/doi/epdf/10.1002/mds.70125","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145531217","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Rolf Pokorny, J. Michael Ryan, Khalid Abd‐Elaziz, Frans van den Berg, Eugenii Rabiner, Graham E. Searle, Manfred Schneider, Christoph Wiessner, Jean‐François Stallaert, Bruno Permanne, Anna Quattropani, Dirk Beher
<p>The importance of considering non-motor symptoms (NMS) in the assessment of patients with movement disorders is widely recognized.<span><sup>1</sup></span> In adults, symptoms such as pain, sleep disturbances, anxiety, fatigue, and cognitive dysfunction can be systematically evaluated, sometimes with validated, condition-specific scales.<span><sup>2, 3</sup></span> For example, the Pain in Dystonia Scale (PIDS) has been recently developed for pain assessment across the spectrum of adult-onset isolated dystonia.<span><sup>4</sup></span> In children, however, the evaluation of NMS remains inconsistent, fragmented, and poorly standardized.<span><sup>1</sup></span> Yet, NMS are often disabling and have substantial consequences for the quality of life of children with movement disorders and their families.<span><sup>5</sup></span> In a recent scoping review,<span><sup>6</sup></span> we provide a timely overview of NMS scales used in children with movement disorders, focusing on the three most prevalent conditions, namely dystonia, tics, and cerebral palsy (CP).<span><sup>7-9</sup></span> We identified <i>a large and heterogeneous set of instruments</i> across 382 studies. They were mostly borrowed from neurological conditions typically presenting in adulthood and other pediatric psychiatric conditions, leaving one to wonder whether they have similar accuracy and clinical relevance also in pediatric movement disorders. Here, we advocate for the development of condition-specific, developmentally appropriate, and clinically meaningful tools, and outline key priorities for achieving this goal.</p><p>A major finding of our review was the heterogeneity of NMS assessment tools in children with movement disorders.<span><sup>6</sup></span> Among the included studies, we catalogued more than 500 distinct rating instruments, and only about 60 were used in more than one study. Very few were designed for pediatric movement disorders, and full psychometric validation in the target populations was uncommon (10 studies, 7%). This leads to a paradox. Numerous instruments are available, yet their precision and disease-specific relevance are often uncertain, and disorder-specific NMS scales remain scarce. As a result, clinicians and researchers frequently default to generic measures rather than condition-sensitive tools.</p><p>For example, in the domain of sleep, 13 distinct tools were used across 24 studies, predominantly parent-reported questionnaires such as the Children's Sleep Habits Questionnaire (CSHQ), the Sleep Disturbance Scale for Children (SDSC), and the Pediatric Sleep Questionnaire (PSQ), together with objective methods (actigraphy, polysomnography).<span><sup>10-12</sup></span> Questionnaires based on child self-report appeared only sporadically (eg, Insomnia Severity Index),<span><sup>13</sup></span> and clinician-administered tools were rare and mainly cited in reviews (eg, bedtime issues, excessive daytime sleepiness, awakenings, regularity/duration
{"title":"Non-motor Symptom Scales in Pediatric Movement Disorders: A Call for Diagnostic-Specific Tools","authors":"Clément Desjardins MD, MSc, Christelle Nilles MD, Hortensia Gimeno PhD, Kathryn J. Peall MD, PhD, Davide Martino MD, PhD, Tamara Pringsheim MD, PhD, Emmanuel Roze MD, PhD","doi":"10.1002/mds.70122","DOIUrl":"10.1002/mds.70122","url":null,"abstract":"<p>The importance of considering non-motor symptoms (NMS) in the assessment of patients with movement disorders is widely recognized.<span><sup>1</sup></span> In adults, symptoms such as pain, sleep disturbances, anxiety, fatigue, and cognitive dysfunction can be systematically evaluated, sometimes with validated, condition-specific scales.<span><sup>2, 3</sup></span> For example, the Pain in Dystonia Scale (PIDS) has been recently developed for pain assessment across the spectrum of adult-onset isolated dystonia.<span><sup>4</sup></span> In children, however, the evaluation of NMS remains inconsistent, fragmented, and poorly standardized.<span><sup>1</sup></span> Yet, NMS are often disabling and have substantial consequences for the quality of life of children with movement disorders and their families.<span><sup>5</sup></span> In a recent scoping review,<span><sup>6</sup></span> we provide a timely overview of NMS scales used in children with movement disorders, focusing on the three most prevalent conditions, namely dystonia, tics, and cerebral palsy (CP).<span><sup>7-9</sup></span> We identified <i>a large and heterogeneous set of instruments</i> across 382 studies. They were mostly borrowed from neurological conditions typically presenting in adulthood and other pediatric psychiatric conditions, leaving one to wonder whether they have similar accuracy and clinical relevance also in pediatric movement disorders. Here, we advocate for the development of condition-specific, developmentally appropriate, and clinically meaningful tools, and outline key priorities for achieving this goal.</p><p>A major finding of our review was the heterogeneity of NMS assessment tools in children with movement disorders.<span><sup>6</sup></span> Among the included studies, we catalogued more than 500 distinct rating instruments, and only about 60 were used in more than one study. Very few were designed for pediatric movement disorders, and full psychometric validation in the target populations was uncommon (10 studies, 7%). This leads to a paradox. Numerous instruments are available, yet their precision and disease-specific relevance are often uncertain, and disorder-specific NMS scales remain scarce. As a result, clinicians and researchers frequently default to generic measures rather than condition-sensitive tools.</p><p>For example, in the domain of sleep, 13 distinct tools were used across 24 studies, predominantly parent-reported questionnaires such as the Children's Sleep Habits Questionnaire (CSHQ), the Sleep Disturbance Scale for Children (SDSC), and the Pediatric Sleep Questionnaire (PSQ), together with objective methods (actigraphy, polysomnography).<span><sup>10-12</sup></span> Questionnaires based on child self-report appeared only sporadically (eg, Insomnia Severity Index),<span><sup>13</sup></span> and clinician-administered tools were rare and mainly cited in reviews (eg, bedtime issues, excessive daytime sleepiness, awakenings, regularity/duration","PeriodicalId":213,"journal":{"name":"Movement Disorders","volume":"41 1","pages":"63-67"},"PeriodicalIF":7.6,"publicationDate":"2025-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://movementdisorders.onlinelibrary.wiley.com/doi/epdf/10.1002/mds.70122","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145498751","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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