{"title":"Clinical outcomes and economic impact of a digital telemedicine intervention in patients with functional motor disorders: a single-blind, randomised controlled trial.","authors":"Marialuisa Gandolfi, Stefano Landi, Angela Sandri, Ilaria Antonella Di Vico, Christian Geroin, Zoe Menaspà, Gianluca Maistri, Melania Fasoli, Federico Schena, Michele Tinazzi, Chiara Leardini","doi":"10.1136/jnnp-2025-336437","DOIUrl":"https://doi.org/10.1136/jnnp-2025-336437","url":null,"abstract":"<p><strong>Background: </strong>Functional motor disorders (FMD) cause long-term disability and economic burden. There is a need for multidisciplinary interventions to manage both motor and non-motor symptoms. We aim to evaluate the clinical and economic effects of integrating digital telemedicine into multidisciplinary FMD management.</p><p><strong>Methods: </strong>This single-blind, randomised controlled trial involved patients with FMD. They were randomly assigned to receive 5-day multidisciplinary rehabilitation with either a digital telemedicine or a standard care programme. The digital telemedicine group received remote management and wearable sensors. A blinded evaluator assessed primary and secondary outcomes at baseline, post-treatment, 12-week and 24-week follow-ups. The primary outcomes were changes in motor symptoms. The secondary outcomes were changes in non-motor symptoms, quality of life (QoL) and mobility in unsupervised settings. The incremental cost-effectiveness ratio (ICER) and quality-adjusted life years (QALYs) were calculated at 24 weeks.</p><p><strong>Results: </strong>Of the total of 62 patients, half made up the digital telemedicine group (n=31, 40.82% female, mean age 42.55±12.65 years) and half the standard care group (n=31, 59.18% female, mean age 43.77±14.44 years). The mental QoL score for the standard care group was lower (p=0.045) and declined compared with the digital telemedicine group (p=0.034), with lower scores at follow-up (p=0.03). At 24 weeks, the ICER (€5503/QALY) showed that digital telemedicine, despite higher initial costs, yielded 0.037 additional QALYs and reduced healthcare use during follow-up.</p><p><strong>Conclusions: </strong>Despite similar improvements in motor symptoms in both groups, digital telemedicine offers an effective adjunct to maintain mental health QoL and reduces healthcare costs in the long-term management of FMD.</p><p><strong>Trial registration number: </strong>ClinicalTrials.gov ID: NCT05345340.</p>","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":""},"PeriodicalIF":7.5,"publicationDate":"2025-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145006228","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}
Antoine Gavoille, Mikail Nourredine, Fabien Rollot, Romain Casey, Guillaume Mathey, Anne Kerbrat, Jonathan Ciron, Jérôme De Sèze, Bruno Stankoff, Elisabeth Maillart, Aurelie Ruet, Pierre Labauge, Arnaud Kwiatkowski, Helene Zephir, Caroline Papeix, Gilles Defer, Christine Lebrun-Frenay, Thibault Moreau, David-Axel Laplaud, Eric Berger, Pierre Clavelou, Eric Thouvenot, Olivier Heinzlef, Jean Pelletier, Abdullatif Al Khedr, Olivier Casez, Bertrand Bourre, Abir Wahab, Laurent Magy, Solène Moulin, Jean-Philippe Camdessanché, Ines Doghri, Mariana Sarov, Karolina Hankiewicz, Corinne Pottier, Amélie Dos Santos, Eric Manchon, Maia Tchikviladze, Muriel Rabilloud, Fabien Subtil, Sandra Vukusic
{"title":"Target trial emulation to replicate randomised clinical trials using registry data in multiple sclerosis.","authors":"Antoine Gavoille, Mikail Nourredine, Fabien Rollot, Romain Casey, Guillaume Mathey, Anne Kerbrat, Jonathan Ciron, Jérôme De Sèze, Bruno Stankoff, Elisabeth Maillart, Aurelie Ruet, Pierre Labauge, Arnaud Kwiatkowski, Helene Zephir, Caroline Papeix, Gilles Defer, Christine Lebrun-Frenay, Thibault Moreau, David-Axel Laplaud, Eric Berger, Pierre Clavelou, Eric Thouvenot, Olivier Heinzlef, Jean Pelletier, Abdullatif Al Khedr, Olivier Casez, Bertrand Bourre, Abir Wahab, Laurent Magy, Solène Moulin, Jean-Philippe Camdessanché, Ines Doghri, Mariana Sarov, Karolina Hankiewicz, Corinne Pottier, Amélie Dos Santos, Eric Manchon, Maia Tchikviladze, Muriel Rabilloud, Fabien Subtil, Sandra Vukusic","doi":"10.1136/jnnp-2025-336762","DOIUrl":"https://doi.org/10.1136/jnnp-2025-336762","url":null,"abstract":"<p><p>BackgroundTarget trial emulation (TTE) offers a formal framework for causal inference using observational data, but its validity must be evaluated in each research domain by replicating randomised clinical trials (RCTs). We aimed to replicate eight RCTs evaluating the efficacy of disease-modifying therapies (DMTs) in multiple sclerosis (MS) using French registry data.</p><p><strong>Methods: </strong>This multicentre, retrospective, observational study was conducted using data extracted in December 2023 from the <i>Observatoire Français de la Sclérose en Plaques</i> (OFSEP) database. For each emulated trial, patients were included when they initiated one of the DMT evaluated in the corresponding RCT and met its inclusion criteria. Clinical outcomes were the annualised relapse rate and 3-month confirmed Expanded Disability Status Scale progression. Radiological outcomes were new/enlarged T2-lesions and new gadolinium-enhanced T1-lesions on a brain MRI. A targeted maximum likelihood estimator was used to estimate the treatment effect adjusted for confounding factors between groups and corrected for censoring and missing outcome assessment.</p><p><strong>Results: </strong>14 111 patients were included in eight emulated trials: ASSESS (fingolimod vs glatiramer acetate), BEYOND (interferon beta vs glatiramer acetate), CONFIRM (dimethyl fumarate (DMF) vs glatiramer acetate), OPERA (ocrelizumab vs interferon beta), REGARD (interferon beta vs glatiramer acetate), RIFUND-MS (rituximab vs DMF), TENERE (teriflunomide vs interferon beta) and TRANSFORMS (fingolimod vs interferon beta). Treatment effects estimated in emulated trials were concordant with RCT findings in seven of eight trials for relapse rate, and in all six trials assessing disability progression. Radiological outcomes were more challenging to replicate; concordance was achieved in three of five trials for new T2-lesions, and one of four trials for new gadolinium-enhanced T1-lesions.</p><p><strong>Conclusion: </strong>The combined use of a TTE methodology and high-quality registry data is a valid tool to evaluate treatment effectiveness in MS.</p>","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":""},"PeriodicalIF":7.5,"publicationDate":"2025-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145000784","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}
Alberto Calvi, Francesc Vivó Pascual, Elisabeth Solana, Elisabet Lopez-Soley, Baris Kanber, Salut Alba-Arbalat, Maria Sepulveda, Eugenia Martínez-Hernández, José Maria Cabrera-Maqueda, Elianet Fonseca, Santiago Medrano-Martorell, Albert Saiz, Yolanda Blanco, Pablo Villoslada, Ferran Prados Carrasco, Eloy Martinez-Heras, Sara Llufriu
{"title":"Diffusivity anisotropy signature of the slowly expanding lesions predicts progression independent of relapse activity in multiple sclerosis.","authors":"Alberto Calvi, Francesc Vivó Pascual, Elisabeth Solana, Elisabet Lopez-Soley, Baris Kanber, Salut Alba-Arbalat, Maria Sepulveda, Eugenia Martínez-Hernández, José Maria Cabrera-Maqueda, Elianet Fonseca, Santiago Medrano-Martorell, Albert Saiz, Yolanda Blanco, Pablo Villoslada, Ferran Prados Carrasco, Eloy Martinez-Heras, Sara Llufriu","doi":"10.1136/jnnp-2025-335884","DOIUrl":"https://doi.org/10.1136/jnnp-2025-335884","url":null,"abstract":"<p><strong>Background: </strong>Slowly expanding lesions (SELs) in multiple sclerosis (MS) are markers of chronic active lesions and seem to trigger disability. This study aimed to analyse spatial features of SELs through diffusion MRI and their clinical impact on progression independent of relapse activity (PIRA).</p><p><strong>Methods: </strong>An observational study of MS subjects prospectively followed since 2011; inclusion required at least three longitudinal T1/T2-weighted and diffusion-weighted MRIs. Subjects followed clinical assessments, using Multiple Sclerosis Functional Composite (MSFC) and Expanded Disability Status Scale. At MRI, lesions were categorised using non-linear deformation as definite or possible SELs, and non-SELs. Fractional anisotropy (FA) was extracted from each lesion core and perilesional area. Differences in FA values across core and perilesional areas by SEL category were assessed using the Mann-Whitney test. Associations with PIRA and clinical outcomes were evaluated using mixed-effects, logistic and Cox regression models.</p><p><strong>Results: </strong>130 subjects underwent MRI (median 25 months) and clinical assessments (median follow-up 9.2 years), of which 29 (22%) developed PIRA. Of 4811 lesions, 8% were definite SELs. Definite SELs exhibited FA decline over time in core and perilesional areas compared with other lesions. Longitudinal core FA reductions within definite SELs were associated with worse MSFC z-score evolution (β=0.03, 95% CI 0.01 to 0.05, p=0.003), higher odds for PIRA (OR=0.01, 95% CI 0.01 to 0.12, p=0.001) and predicted faster time to reach first PIRA event (HR=0.03, 95% CI 0 to 0.49, p=0.015).</p><p><strong>Conclusions: </strong>Definite SELs show distinct greater microstructural damage and are associated with PIRA, making their FA signature a potential predictor of MS progression.</p>","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":""},"PeriodicalIF":7.5,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957674","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}
Vasilis-Spyridon Tseriotis, Georgina Arrambide, Edgar Carnero Contentti, Carmen Tur, Mike P Wattjes, Rosa Cortese, Dimos-Dimitrios Mitsikostas, Nikolaos Grigoriadis, Antonis Adamou, David-Dimitris Chlorogiannis, Eleftherios Beltsios, Melinda Magyari, Thomas Clement Truelsen, Letizia Leocani, Xavier Montalban
{"title":"MRI lesion distribution criteria for MS, NMOSD and MOGAD differentiation: a systematic review and meta-analysis.","authors":"Vasilis-Spyridon Tseriotis, Georgina Arrambide, Edgar Carnero Contentti, Carmen Tur, Mike P Wattjes, Rosa Cortese, Dimos-Dimitrios Mitsikostas, Nikolaos Grigoriadis, Antonis Adamou, David-Dimitris Chlorogiannis, Eleftherios Beltsios, Melinda Magyari, Thomas Clement Truelsen, Letizia Leocani, Xavier Montalban","doi":"10.1136/jnnp-2025-336694","DOIUrl":"https://doi.org/10.1136/jnnp-2025-336694","url":null,"abstract":"<p><strong>Background: </strong>Multiple sclerosis (MS), neuromyelitis optica spectrum disorder (NMOSD) and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) can share similar features, posing diagnostic challenges. In this study, we identified sets of conventional MRI lesion distribution criteria proposed for disease differentiation and investigated their clinical utility.</p><p><strong>Methods: </strong>We searched five electronic databases for English-written and peer-reviewed diagnostic accuracy studies that included brain MRI at least. Hierarchical and univariate random-effects logistic regression models were employed for diagnostic accuracy meta-analysis. Heterogeneity was explored with subgroup analyses. Certainty of evidence was assessed using the GRADEpro tool.</p><p><strong>Results: </strong>Three sets of criteria ('Matthews', 'Cacciaguerra', 'MS lesion checklist') were investigated in 11 studies (2008 patients; MS, n=1037; NMOSD, n=842; MOGAD, n=129), with low applicability concerns. Overall pooled sensitivity and specificity of the Matthews brain MRI criteria (MS vs seropositive-NMOSD differentiation) were 0.92 (0.86 to 0.96) and 0.85 (0.79 to 0.90), respectively, with higher diagnostic values in non-Caucasian populations and during follow-up. Pooled sensitivity and specificity of the Cacciaguerra brain-spinal cord criteria (seropositive-NMOSD vs MS differentiation) were 0.96 (0.76 to 0.99) and 0.83 (0.71 to 0.90), respectively. The MS lesion checklist (MS vs NMOSD/MOGAD differentiation) had lower diagnostic accuracy measures (sensitivity, specificity: 0.74, 0.79, respectively). The Matthews criteria provided the strongest moderate certainty evidence and also showed high pooled diagnostic accuracy for MS versus seronegative-NMOSD (sensitivity: 0.93 (0.84 to 0.97)); specificity: 0.90 (0.80 to 0.95)) and for MS versus MOGAD differentiation (sensitivity: 0.86 (0.81 to 0.90); specificity: 0.87 (0.76 to 0.93)).</p><p><strong>Conclusions: </strong>Lesion distribution criteria can accurately discriminate between MS, NMOSD and MOGAD. Further optimised validation studies, and revisions or extensions may support sustained implementation.</p><p><strong>Prospero registration number: </strong>CRD42023472178.</p>","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":""},"PeriodicalIF":7.5,"publicationDate":"2025-08-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957653","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}
{"title":"Moving past NfL? Multibiomarker models for ALS prognosis and stratification.","authors":"Andrea Malaspina","doi":"10.1136/jnnp-2025-336814","DOIUrl":"10.1136/jnnp-2025-336814","url":null,"abstract":"","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":""},"PeriodicalIF":7.5,"publicationDate":"2025-08-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144883021","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}
Jiaze Sun, Joke De Vocht, Daphne Stam, Chih-Hao Lien, Yun-An Huang, Nikita Lamaire, Maarten Laroy, Kristof Vansteelandt, Ann D'Hondt, Maarten J A Van Den Bossche, Rik Vandenberghe, Ronald R Peeters, Stefan Sunaert, Philip van Damme, Mathieu Vandenbulcke, Jan Van den Stock
{"title":"Neural correlates of memory deficits in premanifest <i>C9orf72</i>-repeat expansions.","authors":"Jiaze Sun, Joke De Vocht, Daphne Stam, Chih-Hao Lien, Yun-An Huang, Nikita Lamaire, Maarten Laroy, Kristof Vansteelandt, Ann D'Hondt, Maarten J A Van Den Bossche, Rik Vandenberghe, Ronald R Peeters, Stefan Sunaert, Philip van Damme, Mathieu Vandenbulcke, Jan Van den Stock","doi":"10.1136/jnnp-2024-335169","DOIUrl":"10.1136/jnnp-2024-335169","url":null,"abstract":"<p><strong>Background: </strong>The premanifest stage in carriers of hexanucleotide repeat expansions in the <i>C9orf72</i> gene (C9RE) is associated with memory impairment. The present study examines whether the impairment is general across domains or disproportionately affects specific stimulus categories such as socioemotional events, and its underlying functional neuroanatomy.</p><p><strong>Methods: </strong>This task-based fMRI-study included 21 premanifest C9RE (preC9RE) carriers and 24 controls. Participants encoded stimuli of (emotional and neutral) faces and houses, followed by a recognition task. Using univariate and multivoxel pattern analyses at whole-brain level and region-of-interest level, we investigated the neural change during encoding and retrieval processes, as well as the neural pattern similarity between encoding and retrieval.</p><p><strong>Results: </strong>Compared with controls, the preC9RE group demonstrated poorer performance in memorising faces (U=104, p=0.002), while their ability to memorise houses remained intact. The preC9RE group exhibited distinct neural patterns in the anterior insula during face encoding compared with the controls (accuracy>0.765, p<0.05). During face retrieval, the preC9RE group showed an increased neural response to encoded faces versus new faces in the right anterior insula (U=394, p=0.015). Individuals with preC9RE exhibited reduced encoding-retrieval neural similarity in the salience network specifically related to face stimuli (U=120, p=0.023).</p><p><strong>Conclusions: </strong>The findings reveal functional changes in the salience network related to impaired social memory at the premanifest stage of C9RE. The findings further underscore the high potential of multidimensional neural response patterns as a sensitive biomarker for neurodegenerative functional changes, and the salience network as biomarker for C9RE disease staging.</p>","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":"861-869"},"PeriodicalIF":7.5,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573271","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}
Simon Fandler-Höfler, Kanishk Kaushik, Benedetta Storti, Slaven Pikija, Dermot Mallon, Gareth Ambler, Payam Tabaee Damavandi, Larysa Panteleienko, Isabella Canavero, Marianne A A van Walderveen, Ellis S van Etten, Jacopo Cosimo DiFrancesco, Christian Enzinger, Thomas Gattringer, Anna Bersano, Marieke J H Wermer, Gargi Banerjee, David J Werring
{"title":"Clinical-radiological presentation and natural history of iatrogenic cerebral amyloid angiopathy.","authors":"Simon Fandler-Höfler, Kanishk Kaushik, Benedetta Storti, Slaven Pikija, Dermot Mallon, Gareth Ambler, Payam Tabaee Damavandi, Larysa Panteleienko, Isabella Canavero, Marianne A A van Walderveen, Ellis S van Etten, Jacopo Cosimo DiFrancesco, Christian Enzinger, Thomas Gattringer, Anna Bersano, Marieke J H Wermer, Gargi Banerjee, David J Werring","doi":"10.1136/jnnp-2024-335164","DOIUrl":"10.1136/jnnp-2024-335164","url":null,"abstract":"<p><strong>Background: </strong>We aimed to describe neuroimaging features, clinical profiles and long-term outcomes in patients with iatrogenic cerebral amyloid angiopathy (iCAA).</p><p><strong>Methods: </strong>We performed a systematic literature search for case series of iCAA and included individual patients and their longitudinal clinical and neuroimaging data in this pooled cohort study. Patients meeting a modified version of the Queen Square criteria for iCAA were included. Baseline and follow-up MRIs were centrally analysed for markers of CAA using validated rating scales.</p><p><strong>Results: </strong>We included 51 patients (68.6% male, median age at presentation 48 years), 51.0% with probable and 49.0% with possible iCAA. We evaluated 219 MRIs acquired over a median follow-up time of 3.7 years (IQR 1.8-6.4). There were 43 symptomatic intracerebral haemorrhages (ICH) in 24 patients during follow-up, a rate of 16.7 per 100 patient-years.Patients with previous supratentorial brain surgery had an ipsilateral-dominant distribution and spread of haemorrhagic markers on MRI. 14/51 (27.5%) patients had transient inflammatory changes (cortical or parenchymal oedema, sulcal hyperintensities). Haemorrhagic markers progressed during follow-up. In addition to 43 symptomatic ICH, 36 asymptomatic ICH (mostly smaller intragyral haemorrhages) were detected on follow-up scans. Besides numerous lobar microbleeds (median 16 at baseline, 53 at last follow-up), deep microbleeds were present in 19.6% of patients at baseline and 44.4% at follow-up. Severe perivascular spaces in centrum semiovale were common at baseline (64.7%) and follow-up (95.6%).</p><p><strong>Conclusions: </strong>Patients with iCAA appear to have distinctive MRI characteristics, which might differentiate iCAA from other CAA subtypes and provide new insights into underlying disease mechanisms.</p>","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":"832-841"},"PeriodicalIF":7.5,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143408667","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}
Uros Klickovic, Luca Zampedri, Nick Zafeiropoulos, Oliver J Ziff, Christopher Dj Sinclair, Stephen Wastling, Magdalena Dudziec, Jodie Allen, Karin Trimmel, Robin S Howard, Andrea Malaspina, Nikhil Sharma, Katie Cl Sidle, Sachit Shah, Christian Nasel, Tarek A Yousry, Linda Greensmith, Jasper M Morrow, John S Thornton, Pietro Fratta
{"title":"Muscle MRI quantifies disease progression in amyotrophic lateral sclerosis.","authors":"Uros Klickovic, Luca Zampedri, Nick Zafeiropoulos, Oliver J Ziff, Christopher Dj Sinclair, Stephen Wastling, Magdalena Dudziec, Jodie Allen, Karin Trimmel, Robin S Howard, Andrea Malaspina, Nikhil Sharma, Katie Cl Sidle, Sachit Shah, Christian Nasel, Tarek A Yousry, Linda Greensmith, Jasper M Morrow, John S Thornton, Pietro Fratta","doi":"10.1136/jnnp-2024-335571","DOIUrl":"10.1136/jnnp-2024-335571","url":null,"abstract":"<p><strong>Background and objectives: </strong>Quantitative and operator-independent biomarkers of disease progression are urgently needed in amyotrophic lateral sclerosis (ALS) research. We assess the potential of skeletal muscle MRI as a sensitive and reliable outcome measure for future ALS clinical trials.</p><p><strong>Methods: </strong>In this longitudinal cohort study, muscle MRI of head-neck, upper and lower limb regions, alongside clinical and functional assessments, were acquired at three time points over the individual maximum observation period (iMOP) of 1 year in 20 patients with ALS and 16 healthy controls. Quantitative MRI parameters cross-sectional area (CSA), volume (VOL), fat fraction, functional rest muscle area and water T2 (T<sub>2m</sub>) were correlated with changes in clinical disease severity (functional rating scales and myometry).</p><p><strong>Results: </strong>Among 20 patients with ALS, 17 completed follow-up. Progressive muscle atrophy (CSA, VOL) was observed at hand (rs=0.66), head-neck (partial η²=0.47) and lower-limb level (thighs: η²=0.56, calves: η²=0.54) over iMOP. MRI changes correlated with leg muscle strength (knee extension: r=0.77; plantar flexion: r=0.78), hand grip strength (r=0.71) and functional rating scales (r=0.68).</p><p><strong>Interpretation: </strong>Our findings demonstrate the effectiveness of muscle MRI as a sensitive neuroimaging biomarker of disease progression in ALS, highlighting its potential application in clinical trials.</p>","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":"908-911"},"PeriodicalIF":7.5,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12418527/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143710338","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}
Marina Romozzi, Lucia Di Nardo, Vincenzo Trigila, Giovanni Cuffaro, Gustavo Savino, Luigi Francesco Iannone, Stanislao Rizzo, Catello Vollono, Paolo Calabresi
{"title":"CGRP increase in tear fluid of migraine patients is reversed by anti-CGRP monoclonal antibodies.","authors":"Marina Romozzi, Lucia Di Nardo, Vincenzo Trigila, Giovanni Cuffaro, Gustavo Savino, Luigi Francesco Iannone, Stanislao Rizzo, Catello Vollono, Paolo Calabresi","doi":"10.1136/jnnp-2025-335868","DOIUrl":"10.1136/jnnp-2025-335868","url":null,"abstract":"","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":"914-916"},"PeriodicalIF":7.5,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248330","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}
{"title":"Ending nuclear weapons, before they end us.","authors":"Chris Zielinski","doi":"10.1136/jnnp-2025-336653","DOIUrl":"10.1136/jnnp-2025-336653","url":null,"abstract":"","PeriodicalId":16418,"journal":{"name":"Journal of Neurology, Neurosurgery, and Psychiatry","volume":" ","pages":"e1"},"PeriodicalIF":7.5,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12418553/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144078522","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}