BMJ Neurology Open最新文献

{"title":"Spinal cord demyelination predicts neurological deterioration in patients with mild degenerative cervical myelopathy.","authors":"Abdul Al-Shawwa, Michael Craig, Kalum Ost, David Anderson, Steve Casha, W Bradley Jacobs, Nathan Evaniew, Saswati Tripathy, Jacques Bouchard, Peter Lewkonia, Fred Nicholls, Alex Soroceanu, Ganesh Swamy, Kenneth C Thomas, Stephan duPlessis, Michael Mh Yang, Julien Cohen-Adad, Nicholas Dea, Jefferson R Wilson, David W Cadotte","doi":"10.1136/bmjno-2024-000940","DOIUrl":"10.1136/bmjno-2024-000940","url":null,"abstract":"<p><strong>Background: </strong>Degenerative cervical myelopathy (DCM) is the most common form of atraumatic spinal cord injury globally. Clinical guidelines regarding surgery for patients with mild DCM and minimal symptoms remain uncertain. This study aims to identify imaging and clinical predictors of neurological deterioration in mild DCM and explore pathophysiological correlates to guide clinical decision-making.</p><p><strong>Methods: </strong>Patients with mild DCM underwent advanced MRI scans that included T2-weighted, diffusion tensor imaging and magnetisation transfer (MT) sequences, along with clinical outcome measures at baseline and 6-month intervals after enrolment. Quantitative MRI (qMRI) metrics were derived above and below maximally compressed cervical levels (MCCLs). Various machine learning (ML) models were trained to predict 6 month neurological deterioration, followed by global and local model interpretation to assess feature importance.</p><p><strong>Results: </strong>A total of 49 patients were followed for a maximum of 2 years, contributing 110 6-month data entries. Neurological deterioration occurred in 38% of cases. The best-performing ML model, combining clinical and qMRI metrics, achieved a balanced accuracy of 83%, and an area under curve-receiver operating characteristic of 0.87. Key predictors included MT ratio (demyelination) above the MCCL in the dorsal and ventral funiculi and moderate tingling in the arm, shoulder or hand. qMRI metrics significantly improved predictive performance compared to models using only clinical (bal. acc=68.1%) or imaging data (bal. acc=57.4%).</p><p><strong>Conclusions: </strong>Reduced myelin content in the dorsal and ventral funiculi above the site of compression, combined with sensory deficits in the hands and gait/balance disturbances, predicts 6-month neurological deterioration in mild DCM and may warrant early surgical intervention.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 1","pages":"e000940"},"PeriodicalIF":2.1,"publicationDate":"2025-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11792293/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143191264","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acute therapeutic effects and pathophysiology of eosinophilic granulomatosis with polyangiitis neuropathy.","authors":"Nanami Yamanaka, Yukio Takeshita, Ryota Sato, Takashi Nawata, Tatsuya Okafuji, Susumu Fujikawa, Miwako Fujisawa, Kinya Matsuo, Joe Nemoto, Yuki Mizumoto, Hideaki Nishihara, Masaya Honda, Mariko Oishi, Toshihiko Maeda, Fumitaka Shimizu, Michiaki Koga, Takashi Kanda, Masayuki Nakamori","doi":"10.1136/bmjno-2024-000938","DOIUrl":"10.1136/bmjno-2024-000938","url":null,"abstract":"<p><strong>Objective: </strong>This study investigated the effects of early treatment and pathophysiology on eosinophilic granulomatosis with polyangiitis neuropathy (EGPA-N).</p><p><strong>Methods: </strong>Twenty-six consecutive patients with EGPA-N were diagnosed and treated within a day of admission and underwent clinical analysis. Peripheral nerve recovery rates were evaluated after early treatment by identifying the damaged peripheral nerve through detailed neurological findings.</p><p><strong>Results: </strong>The eosinophil count at onset was significantly correlated with the total number of damaged nerves. There was a strong correlation between the timing of treatment and the recovery rate in patients who started treatment within 50 days, as the recovery rate did not increase after 50 days of treatment. Antineutrophil cytoplasmic antibodies (ANCA)-negative cases showed significantly higher recovery rates than ANCA-positive cases. Vasculitis was detected in 67% of ANCA-positive and 29% of ANCA-negative patients in the sural nerve and skin biopsy specimen. In addition, infiltration of eosinophils into peripheral nerve tissues was observed in 40% of ANCA-negative patients, whereas it was absent in ANCA-positive patients. Intrafascicular oedema was found in 95% of all patients.</p><p><strong>Discussion: </strong>Our results suggest three pathological pathways: (1) ischaemic peripheral nerve due to vasculitis mainly in ANCA-positive cases, (2) direct infiltration and degranulation of eosinophils in ANCA-negative cases and (3) progression of axonal ischaemia due to intrafascicular oedema in both cases. The study also found that ANCA-negative cases exhibited better responsiveness to acute-phase treatment than ANCA-positive cases. It is essential to treat patients with EGPA-N as early as possible because the patients could recover time-dependently within 50 days of the onset.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 1","pages":"e000938"},"PeriodicalIF":2.1,"publicationDate":"2025-01-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751838/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143030323","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Qualitative systematic review on the lived experience of functional neurological disorder.","authors":"Andrea Szasz, Anthony Korner, Loyola McLean","doi":"10.1136/bmjno-2024-000694","DOIUrl":"10.1136/bmjno-2024-000694","url":null,"abstract":"<p><strong>Objectives: </strong>Functional neurological disorder (FND) is a complex disorder, recently attracting much research into aetiology and treatment. However, there is limited research on the patient's lived experience. This paper addresses this gap to ask: 'What is the subjective life experience of adult patients living with FND?'</p><p><strong>Methods: </strong>From 1980 to 2020, Medline, PsycInfo, Scopus, Science Direct, PubMed, CINAHL and Embase were searched for English language qualitative adult research. The disciplines used general medicine, psychiatry, physiotherapy, nursing, neurology, psychosomatic medicine and occupational therapy. The qualitative literature search included book chapters, theses, fellowship reports and conference articles as well as peer-reviewed scientific journals.The Critical Appraisal Skills Programme tool was used to assess 33 papers, with eight papers included in the final synthesis. Nine additional papers, suggested during review, were evaluated but excluded from synthesis, though incorporated elsewhere in the paper. Two authors used an integrative immersion approach to identify the literature's main themes using line-by-line and top-down methods.</p><p><strong>Results: </strong>Eight main themes were identified: lost, body-mind dualism, preceding stressful events, relatedness, stigma, the battle or fight, the burden and losses of the illness and trust versus mistrust. From these emerged a central overarching theme of relationally regulated selves, which posits the essence of the lived experience of FND as responding to stressful experiences within a relational, regulatory context.</p><p><strong>Conclusions: </strong>The prevalent themes give valuable insight into the lived experience of FND and the impact of stressors, past and present, and the relational environment in the development of and recovery from the disorder. Further research is needed to support the formulation of the patient experience and cocreated recovery pathways.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 1","pages":"e000694"},"PeriodicalIF":2.1,"publicationDate":"2025-01-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751973/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029794","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Optimising early detection of degenerative cervical myelopathy: a systematic review of quantitative screening tools for primary care.","authors":"Sean Inzerillo, Pemla Jagtiani, Salazar Jones","doi":"10.1136/bmjno-2024-000913","DOIUrl":"10.1136/bmjno-2024-000913","url":null,"abstract":"<p><strong>Background: </strong>Early diagnosis of degenerative cervical myelopathy (DCM) is often challenging due to subtle, non-specific symptoms, limited disease awareness and a lack of definitive diagnostic criteria. As primary care physicians are typically the first to encounter patients with early DCM, equipping them with effective screening tools is crucial for reducing diagnostic delays and improving patient outcomes. This systematic review evaluates the efficacy of quantitative screening methods for DCM that can be implemented in primary care settings.</p><p><strong>Methods: </strong>A systematic search following Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines was conducted across PubMed, Embase and Cochrane Library up to July 2024 using keywords relevant to DCM screening. Studies were included if they evaluated the sensitivity and specificity of DCM screening tools applicable to primary care settings. Study quality was assessed using the Quality Assessment of Diagnostic Accuracy Studies-2 tool.</p><p><strong>Results: </strong>The search identified 14 studies evaluating 18 screening methods for DCM. Questionnaires consistently showed high diagnostic accuracy, with Youden indices exceeding 0.60, while only three out of nine conventional physical performance tests met the same threshold. Sensor-assisted tests, particularly those using advanced technology like finger-wearable gyro sensors, exhibited the highest diagnostic accuracy but present challenges related to accessibility and learning curves.</p><p><strong>Conclusion: </strong>This review highlights the potential of quantitative screening methods for early DCM detection in primary care. While questionnaires and conventional tests are effective and accessible, sensor-assisted tests offer greater accuracy but face implementation challenges. A tailored, multifaceted approach is crucial for improving outcomes. Future research should focus on validating these tools in diverse populations and standardising diagnostic criteria.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 1","pages":"e000913"},"PeriodicalIF":2.1,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11752000/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029706","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Diffusion tensor imaging in peroneal neuropathy: a prospective, single-centre study.","authors":"Christophe Oosterbos, Ahmed M Radwan, Stefan Sunaert, Sophie Hoornaert, Anais Van Hoylandt, Robin Lemmens, Tom Theys","doi":"10.1136/bmjno-2024-000876","DOIUrl":"10.1136/bmjno-2024-000876","url":null,"abstract":"<p><strong>Objective: </strong>Diffusion tensor imaging (DTI) showed promising results in diagnosing upper limb neuropathies, but its value in patients with foot drop due to peroneal neuropathy has not yet been investigated. We aim to establish reference values for DTI metrics of the healthy peroneal nerve and to evaluate differences in DTI metrics between patients and healthy controls.</p><p><strong>Methods: </strong>Diffusion-weighted images (DWI) from 22 pathological nerves, 14 asymptomatic patients' nerves and 65 healthy peroneal nerves were processed for quantitative assessment of fractional anisotropy (FA), radial diffusivity (RD), axial diffusivity and mean diffusivity. Clinical baseline and follow-up data were prospectively collected for all patients.</p><p><strong>Results: </strong>Mean patient FA values (0.40, SD 0.08) were significantly lower compared with healthy controls (mean FA 0.44, SD 0.06). Mean patient RD values (0.98 10^-3 mm²/s, SD 0.21 10^-3 mm²/s) were significantly higher compared with healthy controls (mean RD 0.85 10^-3 mm²/s, SD 0.16 10^-3 mm²/s). FA values were significantly lower in patients with severe foot drop (mean FA 0.40, SD 0.06) compared with non-severe foot drop (mean FA 0.48, SD 0.05).</p><p><strong>Conclusion: </strong>Based on these results, DTI appears to aid in the differential diagnostic process of patients with peroneal neuropathy. Future studies should focus on automation of DWI processing, confirm the results in larger patient groups and try to establish reliable cut-off values for DTI metrics.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 1","pages":"e000876"},"PeriodicalIF":2.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751924/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029696","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cognitive impairment in PSP compared with PD: assessment by clinical subtype and longitudinal change.","authors":"Takeharu Tsuboi, Hiroshi Tatsumi, Kosuke Kobayasi, Rina Hashimoto, Ikuko Aiba","doi":"10.1136/bmjno-2024-000946","DOIUrl":"10.1136/bmjno-2024-000946","url":null,"abstract":"<p><strong>Background: </strong>Longitudinal studies investigating cognitive function changes in patients with progressive supranuclear palsy (PSP) are limited. The variability of cognitive impairment across clinical subtypes of PSP remains unclear.</p><p><strong>Objective: </strong>This study aimed to compare the longitudinal changes in cognitive function between patients with PSP and Parkinson's disease (PD) and to assess differences in cognitive impairment among PSP subtypes.</p><p><strong>Methods: </strong>A retrospective observational study was conducted using neuropsychological testing data from patients with PSP and PD admitted to our hospital.</p><p><strong>Results: </strong>The study included 38 patients with PD and 41 patients with PSP (23 PSP-Richardson's syndrome, 14 PSP-progressive gait freezing (PSP-PGF), 3 PSP-Parkinsonism and 1 PSP-predominant corticobasal syndrome). At baseline, cognitive function was significantly lower in the PSP group than in the PD group. Over 12 months, patients with PSP exhibited significant declines in multiple cognitive domains, whereas no significant changes were observed in the PD group. Among PSP subtypes, PSP-RS showed a faster rate of cognitive decline than PD, while PSP-PGF demonstrated a lower progression than PSP-RS.</p><p><strong>Conclusion: </strong>PSP is associated with progressive cognitive impairment, with rates of decline varying by subtype. PSP-PGF exhibited a slower progression than PSP-RS. Clinical management should consider subtype-specific differences in cognitive prognosis to tailor treatment and care.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 1","pages":"e000946"},"PeriodicalIF":2.1,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751889/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical manifestations and outcomes of patients with intravascular large B-cell lymphoma with neurological involvement: highlighting longitudinally extensive myelopathy as a distinct feature.","authors":"Ekdanai Uawithya, Palakorn Lertsakworakul, Weerapat Owatthanapanich, Jiraporn Jitprapaikulsan","doi":"10.1136/bmjno-2024-000915","DOIUrl":"10.1136/bmjno-2024-000915","url":null,"abstract":"<p><strong>Objective: </strong>This study aimed to elucidate the clinical manifestations, laboratory findings and outcomes of patients with intravascular large B cell lymphoma (IVLBCL) with neurological involvement and to differentiate IVLBCL with and without neurological involvement.</p><p><strong>Methods: </strong>A cohort study was conducted at Siriraj Hospital, Mahidol University, Thailand, between January 2005 and September 2024. Clinical data, laboratory values and central nervous system imaging results were analysed. Categorical variables were compared using the χ² or Fisher's exact test, while continuous variables were analysed with the Mann-Whitney U test, as appropriate.</p><p><strong>Results: </strong>Of the 30 patients with IVLBCL, 10 had neurological involvement and 20 without neurological symptoms, including myelopathy (5 patients, 50%); cognitive impairment (3 patients, 30%); seizures (2 patients, 20%); optic neuropathy, hemiparesis, homonymous hemianopia, vertigo and global aphasia (each affecting 1 patient, 10%). 60% of IVLBCL with neurological involvement had systemic symptoms, including prolonged fever, anaemia, anorexia and weight loss. MRI showed hyperintense lesions in the supratentorial, infratentorial and spinal cord with the prominent findings being longitudinally extensive cord lesions (four patients, 40.0%). The median survival time of the IVLBCL with neurological involvement was 4.1 months (95% CI: 0.0 to 17.1 months), with a 1-year survival rate of 37.5% and a 2-year survival rate of 25.0%.</p><p><strong>Interpretation: </strong>This study highlights the distinct clinical, laboratory features and imaging of IVLBCL with neurological involvement and compares it to IVLBCL without neurological involvement. Early recognition of these findings is crucial for accurate diagnosis and improved patient outcomes despite the aggressive nature of IVLBCL.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"7 1","pages":"e000915"},"PeriodicalIF":2.1,"publicationDate":"2025-01-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11751980/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143029442","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Climate change and neurological diseases: report from the Hot Brain 2: Climate Change and Brain Health meeting, 2024.","authors":"James D Mills, Medine I Gulcebi, Jo Allatt, Action Amos, Jack Atkinson, Jason Berwick, Susan Clayton, Derk-Jan Dijk, Kimberly C Doell, Kristie Ebi, Candace C Fleischer, Shakoor Hajat, Candice Howarth, Oliver Jones, Mark Maslin, Lisa Page, Marina Romanello, Lisa Vanhala, Sanjay M Sisodiya","doi":"10.1136/bmjno-2024-000929","DOIUrl":"10.1136/bmjno-2024-000929","url":null,"abstract":"","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"6 2","pages":"e000929"},"PeriodicalIF":2.1,"publicationDate":"2024-12-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11683957/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142907868","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Mixed-methods process evaluation of a proactive approach to healthcare in Parkinson's disease-ParkProReakt: a protocol of a hybrid efficacy-implementation study.","authors":"Natalie Altschuck, Marlena van Munster, Johanne Stümpel, David Jose Pedrosa, Ingmar Wellach, Hannes Boebinger, Max Geraedts","doi":"10.1136/bmjno-2024-000966","DOIUrl":"10.1136/bmjno-2024-000966","url":null,"abstract":"<p><strong>Abstract: </strong></p><p><strong>Introduction: </strong>People with Parkinson's disease (PwPD) experience a wide range of motor and non-motor symptoms that have a significant impact on their health and quality of life. Effective care management for PwPD involves monitoring symptoms at home, involving specialised multidisciplinary care providers and enhancing self-management skills. This study protocol describes the process evaluation within a randomised clinical trial to assess the implementation and its impact on patient health outcomes of ParkProReakt-a proactive, multidisciplinary, digitally supported care model for community-dwelling PwPD.</p><p><strong>Methods and analysis: </strong>The hybrid efficacy-implementation study will assess key implementation outcomes using the Medical Research Council framework for complex interventions alongside a randomised controlled trial. A combination of quantitative and qualitative methods will be used to assess process data from care providers and patients. The main process outcomes are fidelity, dose, feasibility and context. Context will be analysed through semistructured interviews and focus groups using the Consolidated Framework of Implementation Research. To elucidate potential facilitators and barriers to implementation and to gain deeper insights into the efficacy outcome data, quantitative and qualitative process data will be integrated at an interpretative level using mixed methods. In addition to process evaluation, potential indirect mechanisms of impact will be measured.</p><p><strong>Ethics and dissemination: </strong>Ethical approval for this study was obtained from the responsible state medical ethics committees in Hesse and Hamburg, Germany. Results will be communicated to the funding body and disseminated through scientific publications.</p><p><strong>Trial registration: </strong>This study was registered with the German Registry for Clinical Studies (DRKS)-number: DRKS00031092.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"6 2","pages":"e000966"},"PeriodicalIF":2.1,"publicationDate":"2024-12-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11683930/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142907871","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Onset of age, site and respiratory symptoms are strongly associated with respiratory decline in sporadic amyotrophic lateral sclerosis: a long-term longitudinal study.","authors":"Shin-Ichi Terao, Yasunobu Nosaki, Atsunori Murao, Ryota Torii, Nanayo Ogawa, Naofumi Miura, Yousuke Sasaki, Gen Sobue","doi":"10.1136/bmjno-2024-000829","DOIUrl":"10.1136/bmjno-2024-000829","url":null,"abstract":"<p><strong>Objective: </strong>The objective of this study is to identify factors influencing progression of respiratory decline from the onset of neurological symptoms to respiratory failure in patients with amyotrophic lateral sclerosis (ALS).</p><p><strong>Methods: </strong>In 100 patients with sporadic ALS, %vital capacity (%VC) was continuously measured from the first visit to the respiratory endpoint (REP). Cox proportional hazards model identified factors influencing the duration from onset of ALS to REP (Onset-REP). We performed Kaplan-Meier survival curve analysis for onset-REP according to identified factors.</p><p><strong>Results: </strong>Onset sites were the upper limb (U-ALS), lower limb (L-ALS), bulbar paralysis (B-ALS) and respiratory paralysis (R-ALS) in 37, 19, 32 and 12 patients, respectively. Duration from the onset of ALS to the onset of respiratory symptoms (Onset-Rp) and REP (Onset-REP) was 16.1 (SD 12.1) and 24.9 months (SD 14.6), respectively. Multivariate analysis revealed that age at onset, site of onset, Onset-Rp and %VC decline rate significantly influenced Onset-REP duration. Elderly patients had a significantly shorter Onset-REP duration. Onset-REP duration did not significantly differ between patients with U-ALS and L-ALS, but was longer in these patients than in those with B-ALS and R-ALS. Onset-REP duration was positively associated with Onset-Rp duration. The average monthly %VC decline rate was -5.6% (SD 3.3). Age at onset, onset site and Onset-Rp duration significantly influenced the %VC decline rate.</p><p><strong>Conclusions: </strong>Our findings revealed strong and independent patient-specific factors that influence the Onset-REP duration and the %VC decline rate in patients with ALS. These could inform future clinical trials and interventions considering the respiratory function and natural history of patients with ALS.</p>","PeriodicalId":52754,"journal":{"name":"BMJ Neurology Open","volume":"6 2","pages":"e000829"},"PeriodicalIF":2.1,"publicationDate":"2024-12-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11667433/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142885648","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}