Muscle & Nerve最新文献

{"title":"It's Virtually Here: Telemedicine, Virtual Care, and Neuromuscular Disease.","authors":"C D Kassardjian, U Desai, S A Živković, Z Simmons, P Narayanaswami","doi":"10.1002/mus.70382","DOIUrl":"10.1002/mus.70382","url":null,"abstract":"<p><p>The COVID-19 pandemic forced a partial shift in neuromuscular clinical practice away from in-person care to remote means. However, this shift was unexpected, abrupt, reactive to the emergency of a pandemic, and disruptive to the usual manner of care, without adequate time to adapt or study the optimal manner to provide care remotely. While traditionally associated with hub-and-spoke telemedicine models, the pandemic popularized direct-to-home virtual care, mostly unsupported by on-site examiners or standardized platforms. In chronic neuromuscular disorders such as amyotrophic lateral sclerosis (ALS) and myasthenia gravis (MG), virtual care has demonstrated feasibility, patient satisfaction, and potential cost savings, particularly for geographically or functionally disadvantaged populations. Disease-specific adaptations such as the Myasthenia Gravis Core Exam and wearable-based assessments (e.g., home spirometry, grip strength monitoring, and digital outcome measures) have enabled more structured remote evaluations. However, diagnostic accuracy may be compromised by the inability to perform detailed neurological examinations or electrodiagnostic studies. Equity concerns persist due to variability in digital literacy, internet and digital device access, and socioeconomic status. Post-pandemic, patients with disabilities may continue to benefit in a combined virtual/in-person paradigm of care. More evidence is required to evaluate the impact of virtual care on neuromuscular practice quality of care and outcomes. We outline some of the questions that require further study, hoping to spur research and quality improvement investigations of these new care models.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"1318-1327"},"PeriodicalIF":2.9,"publicationDate":"2026-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148830713","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From Long-Term to Trial-Ready: 1.5-Year Follow-Up in an Established FSHD Cohort Highlights Limitations of Current Outcome Measures.","authors":"Sjan Teeselink, Sanne C C Vincenten, Amir Chaman Baz, Nicol C Voermans, Nens van Alfen, Baziel G M van Engelen, Karlien Mul","doi":"10.1002/mus.70413","DOIUrl":"https://doi.org/10.1002/mus.70413","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Identifying endpoints that are sensitive to change is crucial for clinical trial design in facioscapulohumeral muscular dystrophy (FSHD). This study adds follow-up data to a previously published 5-year natural history study in the same cohort, aiming to correlate short-term disease progression to longer term follow-up.</p><p><strong>Methods: </strong>Adult FSHD patients were reassessed 1.5 years after their 5-year visit. A subgroup underwent trimonthly assessments for 1 year following the 1.5-year visit. Disease severity scores, motor function measure (MFM), 6-min walk test (6MWT), antigravity tests (AGTs), and muscle strength by manual muscle testing (MMT) were assessed to determine change over time. Sample sizes were calculated for a hypothetical trial assuming stabilization of the disease as treatment aim.</p><p><strong>Results: </strong>Eighty-six patients completed both assessments, 30 of whom underwent trimonthly assessments. Over 1.5 years, a small decline was observed in the mean MMT score (median change: -0.2, p < 0.001), and the 6MWT (median change: -14 m, p = 0.009). In the trimonthly subgroup, individual mean MMT scores were variable between visits. Sample size estimates ranged from 16 (MMT of specific muscles) to 1824 participants (MFM) per group depending on the used clinical outcome measure and patient selection.</p><p><strong>Discussion: </strong>This study provides clinically relevant data on short-term progression in FSHD. The findings confirm the limited responsiveness of clinical outcome measures resulting in large sample size estimates, demonstrate variability of MMT, and highlight the need for multimodal or biomarker-based endpoints to enable feasible and sensitive clinical trial design.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149224469","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ultrasound-Guided Treatment of Peripheral Entrapment Mononeuropathies.","authors":"Salina Halliday, John Hermansen, John W Norbury","doi":"10.1002/mus.70425","DOIUrl":"https://doi.org/10.1002/mus.70425","url":null,"abstract":"<p><p>High-resolution neuromuscular ultrasound (US) is a valuable tool for the localization, diagnosis, and provision of conservative treatment to peripheral entrapment mononeuropathies. US-guided interventions require careful coordination of transducer and needle movement along with a detailed understanding of sonoanatomy. Preprocedural planning and positioning are instrumental in performing these interventions and should not be overlooked. Minimally invasive procedures, such as corticosteroid injections, aspiration of ganglia, orthobiologics, and hydrodissection, are useful options for the nonsurgical treatment of mononeuropathies refractory to conservative care. US is effective in determining atypical anatomy such as a bifid median nerve for improved accuracy and safety during injections, and precision based techniques such as hydrodissection may aid in physically separating the nerve from scar tissue and adhesions. The most well researched US guided treatment of entrapment neuropathy is carpal tunnel syndrome which demonstrates promising results from platelet-rich plasma, prolotherapy, hydrodissection, and percutaneous needle release. However, there is significant heterogeneity in procedure technique, injectate composition, injectate volume, and periprocedural conservative care that limits interpretation of the results and warrants continued head-to-head comparisons for research. This review highlights procedural details and outcomes for US-guided interventions of the most well-studied peripheral entrapment neuropathies. Despite many promising findings for these techniques, long-term outcomes and severe nerve compression may still benefit most from surgical decompression.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149194534","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Electrophysiologic Demyelinating Features Are Common in Vasculitic Neuropathy: A Cohort of Biopsy-Confirmed Cases.","authors":"Alexandra Maury, Guillemette Beaudonnet, Thierry Maisonobe, Christian Denier, Andoni Echaniz-Laguna, Clovis Adam, Céline Labeyrie, Edouard Berling","doi":"10.1002/mus.70434","DOIUrl":"https://doi.org/10.1002/mus.70434","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Electrophysiologic features suggestive of demyelination have been reported in vasculitic neuropathy, complicating the diagnostic process, but their frequency is unknown. We aimed to determine the prevalence of demyelinating features, including fulfillment of European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) electrophysiological criteria for demyelination in suspected CIDP, in a cohort of patients with biopsy-confirmed vasculitic neuropathy.</p><p><strong>Methods: </strong>Clinical data and nerve conduction studies from patients with biopsy-confirmed vasculitic neuropathy (definite or probable) were retrospectively analyzed in two centers in France between 2014 and 2020.</p><p><strong>Results: </strong>Sixty-three patients were included (mean age 58 ± 16 years; 30% were male), including 28 with non-systemic vasculitic neuropathy (NSVN) and 35 with systemic vasculitic neuropathy (SVN). Pain and sensory disturbances were present in all patients, whereas motor deficits occurred in 73% of patients. The predominant electrophysiological pattern was a symmetrical distal polyneuropathy (n = 26). Twenty-seven (42.9%) patients showed demyelinating NCS abnormalities, and 11 (17.5%) patients fulfilled the EAN/PNS criteria for demyelination. Motor conduction block was the most frequent demyelinating abnormality, followed by prolonged F-wave latency. Demyelinating abnormalities were present in 7.1% of NSVN patients and 25.7% of SVN patients, with no statistically significant difference between groups.</p><p><strong>Discussion: </strong>Electrophysiological patterns suggestive of demyelination are common in vasculitic neuropathy. Nevertheless, most patients presented with pain and a symmetrical distal polyneuropathy, a phenotype not typically associated with demyelinating neuropathy. Future studies integrating detailed clinical data may help identify key diagnostic features and improve recognition despite potentially misleading electrodiagnostic findings.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149169932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Statin Exposure and Survival Outcomes in Amyotrophic Lateral Sclerosis in an Asian Cohort.","authors":"Anushika Raheja, Kexin Ang, Bernett Lee, Seyed Ehsan Saffari, Eng King Tan, Crystal Jing Jing Yeo","doi":"10.1002/mus.70427","DOIUrl":"https://doi.org/10.1002/mus.70427","url":null,"abstract":"<p><strong>Introduction: </strong>Data from Asian cohorts on whether statin therapy affects amyotrophic lateral sclerosis (ALS) progression are limited. We examined whether statin exposure was associated with ALS disease duration or tracheostomy-free survival in a multiethnic Asian ALS cohort.</p><p><strong>Methods: </strong>Data from patients diagnosed with ALS between 2012 and 2025 were retrospectively reviewed. Patients were categorized as \"ever\" or \"never\" statin users for primary analyses, and as \"never,\" \"former,\" or \"current\" users at diagnosis for additional analyses. Disease duration was defined as time from symptom onset to death, and tracheostomy-free survival as time from symptom onset to tracheostomy or death without tracheostomy. Kaplan-Meier and Cox regression analyses evaluated associations between statin exposure and both outcomes. Additional analyses examined statin discontinuation after diagnosis using time-varying exposures. Sensitivity analyses excluded post-diagnosis imitators and applied 30-, 90-, and 180-day lags.</p><p><strong>Results: </strong>Among 103 ALS patients, 27 (26.2%) had ever received statins. Statin exposure was not significantly associated with disease duration (hazard ratio [HR]: 1.42, 95% CI: 0.64-3.14, p = 0.387) or tracheostomy-free survival (HR: 1.04, 95% CI: 0.50-2.18, p = 0.913). Post-diagnosis statin discontinuation was associated with shorter survival in a time-varying analysis (HR: 6.52, 95% CI: 1.48-28.74, p = 0.013), although this finding was hypothesis-generating. Sensitivity analyses did not show statistically significant associations.</p><p><strong>Discussion: </strong>In this multiethnic Asian ALS cohort, statin exposure was not significantly associated with disease duration or tracheostomy-free survival. These findings add to the evidence that statins do not need to be discontinued due to ALS diagnosis. Larger studies are needed to define this association.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149167236","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Efficacy and Safety of Tocilizumab in the Treatment of Refractory Ocular Myasthenia Gravis in Pediatric Patients.","authors":"Qing-Qing Wang, Xiang-Qi Cao, Bi-Ying Zhang, Xiao-Xi Huang, Si-Jia Hao, Ting Chang, Zhe Ruan","doi":"10.1002/mus.70435","DOIUrl":"https://doi.org/10.1002/mus.70435","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Refractory pediatric ocular myasthenia gravis (OMG) remains difficult to manage. We evaluated tocilizumab's efficacy and safety in these patients.</p><p><strong>Methods: </strong>In this single-center, retrospective analysis of prospectively collected registry data from Tangdu Hospital (China), pediatric refractory OMG patients received intravenous tocilizumab (8 mg/kg every 4 weeks) for 16 weeks as add-on therapy. Refractory disease required persistent symptoms despite adequate trials of ≥ 2 immunosuppressants, frequent relapses during glucocorticoid tapering, or intolerance of conventional oral immunosuppressants. The primary outcome was change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score from baseline to Weeks 4, 8, 12, and 16, analyzed using generalized estimating equations.</p><p><strong>Results: </strong>All 10 patients (1 male, 9 females; median age 5.5 years, range 2-11) completed 16 weeks of follow-up. Seven (70%) were positive for antiacetylcholine receptor antibody (AChR-Ab). At Week 4, mean MG-ADL score decreased from 3.4 to 2.5 (incidence rate ratio[IRR], 0.74; 95% CI, 0.54-0.99; p = 0.048), with further reduction to 1.5 at week 16 (IRR, 0.44; 95% CI, 0.24-0.80; p = 0.007). Similarly, mean Quantitative MG (QMG) score dropped from 4.7 at baseline to 3.0 at Week 4 (IRR, 0.64; 95% CI, 0.41-0.99; p = 0.048) and further to 1.9 at Week 16 (IRR, 0.40; 95% CI, 0.24-0.69; p = 0.001). By Week 16, 50% of patients achieved minimal symptom expression, and 50% reduced prednisone requirements. Tocilizumab was well-tolerated with only one transient infusion-related dizziness.</p><p><strong>Discussion: </strong>These preliminary findings suggest that tocilizumab may offer a safe and effective treatment option for children with refractory OMG.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149167804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Expanding Therapeutic Landscape in Myasthenia Gravis.","authors":"Hebatallah R Rashed","doi":"10.1002/mus.70418","DOIUrl":"https://doi.org/10.1002/mus.70418","url":null,"abstract":"<p><p>Myasthenia gravis (MG) is a rare autoimmune disorder caused by autoantibodies targeting postsynaptic proteins of the neuromuscular junction. Although conventional therapies can achieve remission or sufficient symptom control in most patients with generalized MG (gMG), approximately 10%-30% of patients remain treatment refractory with an increased risk of myasthenic crises and mortality. In addition, prolonged immunosuppression can cause undesirable side effects. Over the past decade, research has focused on identifying new therapeutic targets, which paved the way to advance the development of novel therapies. Rather than exerting broad immunosuppression, novel therapies selectively target specific components involved in the immunopathogenesis of MG, such as immunoglobulin G (IgG) production (B cell-depleting agents), IgG recycling (neonatal fragment crystallizable receptor, FcRn, blocker), and complement activation (complement inhibitors). Targeted therapies have been evolving exponentially since 2017, marking a new era in treatment strategies in gMG. They have emerged as potential steroid-sparing agents with robust efficacy and favorable safety profiles. Small cohorts have demonstrated the efficacy and tolerability of these therapies during myasthenic crisis and in patients with active thymoma. Although targeted therapies have generally comparable efficacy and safety, treatment plans should be individualized, and several factors should be considered before initiating these therapies, including pharmacological spectrum (complement inhibitors are effective only in acetylcholine receptor antibody-positive MG, while FcRn and B cell-depleting agents have broader spectra), convenience of administration, and cost-effectiveness. This monograph proposes an updated treatment algorithm in refractory gMG that integrates novel targeted therapies alongside standard immunosuppressant steroid-sparing agents.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149167198","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Duke Myasthenia Gravis Clinic Registry: IV. Does Race Affect the Outcome of Treatment of Myasthenia Gravis? A Single Center Retrospective Analysis.","authors":"Mao Liu, Chunlu Ma, Michael W Lutz, Vern C Juel, Janice M Massey, Lisa D Hobson-Webb, Shruti M Raja, Donald B Sanders","doi":"10.1002/mus.70431","DOIUrl":"https://doi.org/10.1002/mus.70431","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Few studies have investigated the role of race in the long-term outcome of patients with myasthenia gravis (MG). The aim of this study is to analyze clinical characteristics and treatment outcomes related to race in patients with MG.</p><p><strong>Methods: </strong>This is a retrospective review of data from patients seen between 1980 and 2016 and followed for at least 2 years in the Duke MG Clinic. Treatment goal (TG) was defined as achieving MGFA postintervention status of \"minimal manifestations\" or better. Multivariable logistic regression analysis and Cox proportional hazards (CPH) modeling were performed to assess the effect of race on TG by adjusting for other variables.</p><p><strong>Results: </strong>Among 820 patients (661 White, 81%; 159 Black, 19%), Black patients had a younger median age at disease onset, female predominance, higher rate of muscle-specific tyrosine kinase antibodies and thymectomy, and less non-corticosteroid immunosuppressant use. The percentage of White patients in the Registry is greater than the mean in the North Carolina census between 1980 and 2010 (81% vs. 77%, p = 0.014). Disease duration and MGFA class at first visit, thymectomy, follow-up duration, median time to TG, TG achievement and maintenance, median time to TG loss, and corticosteroid/PLEX/IVIG use were similar in the two racial groups. Neither multivariable logistic regression nor CPH modeling identified race as an independent predictor of TG.</p><p><strong>Discussion: </strong>Although racial differences were observed in patients with MG, long-term outcomes were similar in Black and White patients. Our study suggests that following treatment guidelines is important in achieving favorable MG outcomes.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149165116","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Using High-Field Magnetic Resonance Spectroscopy to Measure Muscle Glycogen in Patients With McArdle Disease.","authors":"Mads Godtfeldt Stemmerik, Gry Hatting Beha, Anna Slipsager, Vanessa Wiggermann, John Vissing","doi":"10.1002/mus.70433","DOIUrl":"https://doi.org/10.1002/mus.70433","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Patients with McArdle disease demonstrate exercise intolerance with muscle contractures and episodic rhabdomyolysis. Additionally, some patients show progressive fat replacement of paraspinal and shoulder muscles with aging. The cause of fat replacement in older McArdle patients is unknown but could relate to a higher glycogen accumulation in these muscles in line with what is found in Pompe disease. The aim of the study was to investigate if this is also the case in McArdle disease, which could imply that excess levels of glycogen accumulation are a potential driver of fat replacement of muscle.</p><p><strong>Methods: </strong>Using 7 Tesla ¹³C magnetic resonance spectroscopy, we measured glycogen noninvasively in lumbar, thigh, and calf muscles in 14 patients with McArdle disease and 19 healthy controls.</p><p><strong>Results: </strong>Patients with McArdle disease showed increased levels of glycogen in all muscles compared to controls; calf (3.2 times normal, p < 0.005), hamstrings (2.7 times normal, p < 0.005), anterior thigh compartment (2.7 times normal, p < 0.005), and lumbar muscle (2.1 times normal, p < 0.005). Glycogen was evenly distributed among muscle groups in both patients with McArdle disease and healthy controls.</p><p><strong>Discussion: </strong>Our results are in line with previous studies showing two to three times increased glycogen compared to control in muscle biopsies. We found no variability in glycogen concentration among muscles that could indicate glycogen as a predictor of muscle injury in McArdle disease.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149127968","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"One-Year Clinical Course of Patients With Amyotrophic Lateral Sclerosis Admitted to a Home Palliative Care Program: An Extension Study.","authors":"Sebastiano Mercadante, Antonino Petronaci","doi":"10.1002/mus.70429","DOIUrl":"https://doi.org/10.1002/mus.70429","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Longer term data on ALS patients receiving home palliative care are scarce. We extended follow-up of a 6-month cohort to 12 months to characterize the trajectory of function, symptoms, vital support (noninvasive ventilation [NIV], gastrostomy, and/or tracheostomy), and advance care planning (ACP).</p><p><strong>Methods: </strong>In this prospective, single-center study, 34 consecutive ALS patients in a home palliative care program (SAMOT, Palermo) were assessed at baseline, 2, 4, 6, and 12 months, using Karnofsky, ALS Functional Rating Scale-Revised (ALSFRS-R), Edinburgh Cognitive and Behavioural ALS Screen (ECAS), Edmonton Symptom Assessment Scale (ESAS), dysphagia, vital support, and advanced care planning (ACP).</p><p><strong>Results: </strong>Ten patients (29.4%) died over 12 months (70% at home); 26 completed the 12-month assessment. Karnofsky (34.4-27.7) and ALSFRS-R (27.1-21.7) declined significantly throughout (both p < 0.0005). Total ESAS rose from 15.7 to 23.7 (p < 0.0005); anxiety increased significantly between T4 and T12. NIV use rose from 20.6% to 50.0% (p = 0.014), while tracheostomy remained stable. Documented end-of-life decisions rose to 70.4% at 6 months but plateaued (69.2% at 12 months); no living wills were completed. Cognitive impairment on ECAS was present in about half of patients, unchanged over time. No patient was referred to or admitted to hospice.</p><p><strong>Discussion: </strong>Extending follow-up beyond 6 months shows continuous functional decline and rising symptom burden, and highlights late-onset anxiety and early-plateauing ACP. Despite progressive needs, this home palliative care model appeared to support most patients at home until death over 1 year, within this single-center, non-comparative cohort.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.9,"publicationDate":"2026-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149112378","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}