Muscle & Nerve最新文献_第9页

MGBase: A Global, Observational Registry for Collaborative Research in Myasthenia Gravis. MGBase：重症肌无力合作研究的全球观察性注册。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-09-01 Epub Date: 2025-06-04 DOI: 10.1002/mus.28450

Katherine A Buzzard, Anneke Van der Walt, Helmut Butzkueven, Wen Wen Zhang, Carolina Barnett-Tapia, Gary Cutter, Jeannine M Heckmann, Pamela Farr, Carolyn Tran, Charlotte Sartori, Dusko Stupar, Rein More, Linda Sim, Alison Le, Qingxiao Tang, Stephen W Reddel

{"title":"MGBase: A Global, Observational Registry for Collaborative Research in Myasthenia Gravis.","authors":"Katherine A Buzzard, Anneke Van der Walt, Helmut Butzkueven, Wen Wen Zhang, Carolina Barnett-Tapia, Gary Cutter, Jeannine M Heckmann, Pamela Farr, Carolyn Tran, Charlotte Sartori, Dusko Stupar, Rein More, Linda Sim, Alison Le, Qingxiao Tang, Stephen W Reddel","doi":"10.1002/mus.28450","DOIUrl":"10.1002/mus.28450","url":null,"abstract":"Introduction/aims: Patient registries are valuable tools for outcomes research in rare diseases such as myasthenia gravis (MG). Existing MG registries are limited by factors including a lack of geographical scope. MGBase has been designed as a global, observational registry aimed at studying clinical practice outcomes in MG.Methods: MGBase was developed with the support of the independent MSBase Foundation. An international scientific leadership group (SLG) established a minimum dataset and outcome measures. Data are entered on a purpose-designed platform in real time and held in a web-based registry. Members can request access to the global dataset for investigator-driven substudies.Results: MGBase data collection commenced in October 2021. From inception until April 2024, 565 patients from 16 clinics and 8 countries were enrolled. The cohort is 56% female, with a mean age of 57 (SD19) years at the last visit and a median disease duration of 5 (IQR 1.8, 10.8) years. Seventy-six percent of patients are acetylcholine receptor antibody positive (AChR ab+) and 7% have antibodies to muscle-specific kinase (MuSK ab+). At diagnosis, 33% of patients had ocular MG. Immunotherapy was used in 87% of patients. A minority of patients (7%) required three or more concurrent immunotherapies. Thymectomy was performed in 24% of patients.Discussion: MGBase is a global registry for collaborative research in MG. Interim analysis of registry data shows disease characteristics similar to those previously published. As global enrollments increase, the registry will generate clinical practice evidence of treatment outcomes, safety, and disease prognostic markers.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"424-432"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338016/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144226027","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Idiopathic Inflammatory Myopathies and Malignancy Screening: A Survey of Current Practices Amongst Canadian Neurologists and Rheumatologists. 特发性炎性肌病和恶性肿瘤筛查：加拿大神经科医生和风湿病学家当前实践的调查。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-09-01 Epub Date: 2025-06-28 DOI: 10.1002/mus.28463

Maria Jekielek, Rosane Nisenbaum, Ophir Vinik, Charles D Kassardjian

{"title":"Idiopathic Inflammatory Myopathies and Malignancy Screening: A Survey of Current Practices Amongst Canadian Neurologists and Rheumatologists.","authors":"Maria Jekielek, Rosane Nisenbaum, Ophir Vinik, Charles D Kassardjian","doi":"10.1002/mus.28463","DOIUrl":"10.1002/mus.28463","url":null,"abstract":"Introduction/aims: Although the need for malignancy screening in idiopathic inflammatory myopathies (IIM) is generally accepted, data to guide the choice and timing of investigations are limited. Our aim was to characterize the gaps and uncertainties amongst Canadian neurologists and rheumatologists with respect to malignancy screening in IIM.Methods: An online survey consisting of 18 multiple-choice questions related to malignancy screening practices was distributed to adult neurologists and rheumatologists practising in Canada, and survey responses were described and compared between groups.Results: Of 69 participants, the majority (95.7%) performed malignancy screening. However, there was variability in practice including delegation and choice of screening tests, influence of patient-specific factors, and timing of repeat testing relative to original testing. Only 18.2% of respondents were confident in their malignancy screening practices. The most significant perceived knowledge gap was lack of consensus or guidelines on choice and frequency of malignancy screening (92.8%). Compared with neurologists, rheumatologists saw a higher number of IIM patients per year (72.5% vs. 41.4% reported five or more, p = 0.009), were more likely to consider patient risk factors and order more investigations, while neurologists were more likely to repeat testing.Discussion: Variability and knowledge gaps exist amongst neurologists and rheumatologists with regard to malignancy screening in IIM patients. The identified variability and lack of confidence may lead to lack of standardization of care, and potentially either under- or over-investigating of IIM patients for malignancy. Further research is required to better understand the optimal choice of tests and timing of repeat investigations.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"502-508"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338006/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144529001","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Serum Biomarkers for Traumatic Peripheral Nerve Injury: A Systematic Review. 外伤性周围神经损伤的血清生物标志物：系统综述。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-09-01 Epub Date: 2025-07-02 DOI: 10.1002/mus.28467

Amanda M Faust, Stephen J DeMartini, Christopher J Dy, David M Brogan

{"title":"Serum Biomarkers for Traumatic Peripheral Nerve Injury: A Systematic Review.","authors":"Amanda M Faust, Stephen J DeMartini, Christopher J Dy, David M Brogan","doi":"10.1002/mus.28467","DOIUrl":"10.1002/mus.28467","url":null,"abstract":"Diagnosing the severity of a peripheral nerve injury or compression, and assessing its potential for recovery, remain clinical challenges. An objective and easily quantifiable serum-based biomarker that reflects axonal health would be a valuable diagnostic adjunct in the treatment of patients with peripheral nerve pathology. We therefore performed a review of the published literature using search strategies created for serum biomarkers and traumatic peripheral nerve injuries with both standardized index terms (i.e., MeSH, Emtree) and relevant key words. This yielded a total of 4626 combined results. Data was extracted from 12 studies (6 animal studies, 6 human studies). The biomarkers identified in this review ranged from axonal specific markers like neurofilament light chain (NfL) to more generalized markers such as lipid profiles and cytokines. The biomarker in this review deemed most promising for future use in the diagnosis and prognostication of peripheral nerve injury is serum NfL given its specificity for the nervous system and its dynamic behavior after nerve injury in animal models. Further work assessing its sensitivity and specificity to traumatic and compressive peripheral nerve injury may lay the foundation for widespread clinical use.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"383-392"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144540878","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}

引用次数: 0

Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen-Treated ALS. 血清心肌肌钙蛋白T水平作为肌萎缩侧索硬化症治疗反应的标志物。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-09-01 Epub Date: 2025-06-09 DOI: 10.1002/mus.28453

Sarah Bernsen, Rachel Fabian, Yasemin Koc, Peggy Schumann, Peter Körtvélyessy, Sergio Castro-Gomez, Thomas Meyer, Patrick Weydt

{"title":"Serum Cardiac Troponin T Levels as a Therapy Response Marker in Tofersen-Treated ALS.","authors":"Sarah Bernsen, Rachel Fabian, Yasemin Koc, Peggy Schumann, Peter Körtvélyessy, Sergio Castro-Gomez, Thomas Meyer, Patrick Weydt","doi":"10.1002/mus.28453","DOIUrl":"10.1002/mus.28453","url":null,"abstract":"Introduction/aims: Cardiac troponin T (cTnT) levels are elevated in the majority of persons with amyotrophic lateral sclerosis (ALS) and increase over time. Neurofilament light chain (NfL) is an established therapy response biomarker in ALS as superoxide dismutase1 (SOD1)-ALS patients treated with the antisense oligonucleotide tofersen show a decrease in NfL. In this study, we assess cTnT levels in SOD1-ALS at baseline and during tofersen treatment.Methods: cTnT was analyzed at baseline and during tofersen treatment in 23 SOD1-ALS patients at two specialized ALS centers in Germany and compared to a control cohort of 74 ALS patients without SOD1 variants.Results: cTnT levels increased in the control ALS cohort over time (p < 0.0001) but not in the tofersen group (p = 0.36). Creatine kinase (CK) and CK-MB levels did not show significant changes over time. The median monthly increase of cTnT was 0.045 points (IQR 0.02-0.08) in the control ALS cohort and 0.01 points (IQR -0.01-0.03) in the tofersen group (p = 0.0013). A significantly lower fold change in cTnT levels was observed in the tofersen-treated cohort (median 1.2; IQR 0.77-1.59) relative to the control group (median 1.89; IQR 1.35-2.75) (p = 0.0003). Nine (39%) patients treated with tofersen experienced a reduction in cTnT levels.Discussion: In this study, we describe a response signal of cTnT to tofersen treatment, which supports the value of cTnT as an independent biomarker in ALS. These results contribute to the notion that cTnT may provide additional value as a progression and treatment response biomarker in ALS complementary to NfL and warrant further investigation.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"509-514"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338008/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144258613","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Focal Neurogenic Muscle Hypertrophy and Focal Neurogenic Myositis. 局灶性神经源性肌肉肥大和局灶性神经源性肌炎。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-08-28 DOI: 10.1002/mus.70008

Kevin J Felice, Charles H Whitaker, William J Pesce, Qian Wu

{"title":"Focal Neurogenic Muscle Hypertrophy and Focal Neurogenic Myositis.","authors":"Kevin J Felice, Charles H Whitaker, William J Pesce, Qian Wu","doi":"10.1002/mus.70008","DOIUrl":"https://doi.org/10.1002/mus.70008","url":null,"abstract":"Focal neurogenic muscle hypertrophy (F-NMH) and focal myositis from neurogenic causes (F-NM) are terms used to describe the paradoxical changes in skeletal muscle following injury to peripheral motor axons. Since the first clinicopathologic description in 1978, over 100 patients with F-NMH and F-NM have been reported. We reviewed the medical records and clinicopathological data on our series of 6 patients from 2007 to 2025, and then expanded the review to include all patients reported in case studies or series with well-defined F-NMH or F-NM based electrodiagnostic studies, muscle histopathology, or both. Inclusive of our 6 patients, 94 patients were reviewed. Most presented with unilateral calf muscle hypertrophy followed in frequency by involvement of the trapezius, biceps brachii, tibialis anterior, and tensor fascia lata. Radiculopathy was the most common injury site. Electrodiagnostic studies showed a mixture of ongoing and chronic changes of denervation and reinnervation. Complex repetitive discharges were observed in 50%. Magnetic resonance imaging showed focal muscle hypertrophy with variable signal changes. Muscle biopsies showed mixed myopathic and neurogenic changes with 39% having inflammation. During the period of observation, 37 improved including 7 without treatment, and none worsened. Botulinum toxin and oral prednisone were the most efficacious treatments. F-NMH is a rare, non-progressive, and seemingly benign paradoxical reaction to peripheral nerve injury. The cause is unknown but likely multifactorial based on clinical observations and experimental studies. Muscle inflammation is not a consistent finding, does not appear to alter the clinical course or prognosis, and may be an epiphenomenon.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-08-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962154","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}

引用次数: 0

Serum Creatine Kinase and Transaminase Levels in Duchenne and Becker Muscular Dystrophies. 杜氏和贝克尔肌营养不良症患者血清肌酸激酶和转氨酶水平。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-08-01 Epub Date: 2025-05-11 DOI: 10.1002/mus.28431

Marie Rohlenová, Kateřina Machová, Jana Baranová, Lenka Mokrá, Livie Mensová, Radim Mazanec, Lenka Juříková, Jan Staněk, Petra Fuchsová, Barbora Lauerová, Markéta Kumhera, Jana Haberlová

{"title":"Serum Creatine Kinase and Transaminase Levels in Duchenne and Becker Muscular Dystrophies.","authors":"Marie Rohlenová, Kateřina Machová, Jana Baranová, Lenka Mokrá, Livie Mensová, Radim Mazanec, Lenka Juříková, Jan Staněk, Petra Fuchsová, Barbora Lauerová, Markéta Kumhera, Jana Haberlová","doi":"10.1002/mus.28431","DOIUrl":"10.1002/mus.28431","url":null,"abstract":"Introduction/aims: The distinction between hepatic and muscular origin of transaminases in Duchenne (DMD) and Becker (BMD) muscular dystrophy is challenging. We aimed to describe the relations between creatine kinase (CK), lactate dehydrogenase (LDH), myoglobin, and alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma glutamyltransferase (GGT), and their evolution with age, the severity of the disease, and its complications (corticosteroid therapy, cardiac ejection fraction, body mass index [BMI]).Methods: Multiple regression analysis of the studied markers was performed in three cohorts: 1. Retrospective data of 212 DMD and BMD boys available in the national registry of muscular dystrophies (REaDY), and 2. Cross-sectional study supplemented by retrospective data of A. 64 DMD boys and B. 18 adults with either BMD or B/DMD carriers.Results: The estimation of normal levels of transaminases from CK levels was: ALT [UI/L] = 228.508 + 0.011*CK [UI/L] ± 341.822for DMD, and ALT [UI/L] = 124.498 + 0.011*CK [UI/L] ± 341.822for BMD. AST [UI/L] = 167.436 + 0.012*CK [UI/L]-6.323*Age ± 249.804 for both (all p < 0.001). In the smaller cohort, ALT was best predicted by LDH and physical performance, whereas AST was defined by CK and LDH combined (all p < 0.001). In DMD, all AST, ALT, CK, LDH, and myoglobin decreased with age (p < 0.001), while GGT increased with age and BMI (both p < 0.001).Discussion: Transaminases are dependent on CK and LDH in dystrophinopathy. There is an increase in GGT in DMD connected to BMI.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"240-249"},"PeriodicalIF":3.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144019063","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}

引用次数: 0

Body Composition in Children With Spinal Muscular Atrophy Types 2 and 3 Receiving Nusinersen Treatment: A Longitudinal Cohort Study. 接受Nusinersen治疗的2型和3型脊髓性肌萎缩症儿童的体成分：一项纵向队列研究

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-08-01 Epub Date: 2025-05-22 DOI: 10.1002/mus.28439

Yue Yan, Xinli Zou, Dongming Zhou, Danhui Zhu, Yijie Feng, Yicheng Yu, Jianing Jin, Qi Long, Yuzhi Chen, Fei Chen, Feng Gao, Shanshan Mao

{"title":"Body Composition in Children With Spinal Muscular Atrophy Types 2 and 3 Receiving Nusinersen Treatment: A Longitudinal Cohort Study.","authors":"Yue Yan, Xinli Zou, Dongming Zhou, Danhui Zhu, Yijie Feng, Yicheng Yu, Jianing Jin, Qi Long, Yuzhi Chen, Fei Chen, Feng Gao, Shanshan Mao","doi":"10.1002/mus.28439","DOIUrl":"10.1002/mus.28439","url":null,"abstract":"Introduction/aims: Body composition was reported as a biomarker of muscle atrophy in the natural history of children with spinal muscular atrophy (SMA). We aimed to analyze longitudinal changes in the body composition of patients receiving nusinersen treatment and to investigate its correlation with functional motor assessments.Methods: This cohort study enrolled patients with SMA types 2 and 3 receiving nusinersen treatment for 14 months. Data including fat-free mass (FFM), muscle mass (MM) and their indexes, and Hammersmith Functional Motor Scale Expanded (HFMSE) scores at baseline and at 6, 10, and 14 months were analyzed.Results: Forty-six patients with SMA type 2 and 3, median age at initial treatment 7.1(4.6-10.4) years, were included, with 25 males and 21 females. Compared with the baseline, FFM, MM, and indexes of both significantly increased during 14 months (p < 0.05) while body fat mass index showed no significant change (p = 0.18). Significant increases were noted in HFMSE scores (p < 0.001), which were moderately correlated with FFM index and MM index. The associations between HFMSE and muscle-related variables were strong in children who achieved walking at baseline.Discussion: Body composition changes in muscle-related variables were found in children with SMA types 2 and 3 who received nusinersen treatment. Higher muscle mass was associated with better motor function.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"331-335"},"PeriodicalIF":3.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120346","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}

引用次数: 0

Educating the Next Generation of Neuromuscular and Electrodiagnostic Practitioners: Challenges and Opportunities. 培养下一代神经肌肉和电诊断从业者：挑战和机遇。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-08-01 Epub Date: 2025-05-27 DOI: 10.1002/mus.28443

Lawrence R Robinson

{"title":"Educating the Next Generation of Neuromuscular and Electrodiagnostic Practitioners: Challenges and Opportunities.","authors":"Lawrence R Robinson","doi":"10.1002/mus.28443","DOIUrl":"10.1002/mus.28443","url":null,"abstract":"Today's neuromuscular (NM) and electrodiagnostic medicine (EDM) trainees learn very differently from those of times past. There are a variety of new tools available to them, with nearly ubiquitous access to information, technology, and social media, along with high expectations regarding immediate access to information. At the same time, they require assistance honing their reflective and critical thinking skills. Educators are encouraged to develop explicit goals of training and to consider new teaching methods such as the flipped classroom and the one-minute preceptor. Use of humor in education, when appropriate, can help with information retention and managing stress and emotion in the healthcare team. Despite inherent barriers, educators need to fail learners who do not meet expectations to benefit both learners and patients. Self-assessment examinations (SAEs) can provide very valuable feedback for both individuals and programs, as well as the field. Those who perform poorly on SAEs have a low chance of passing a subsequent certification examination. Performance on the electrodiagnostic SAE improves with increasing numbers of patient studies, not leveling out until roughly 300-400 studies have been completed; this suggests a possible benchmark for training. Poor performance on initial certification examinations is strongly correlated with the risk of subsequent state medical board disciplinary action; such individuals will want to make a special effort to stay informed and up to date. After initial certification examinations, ongoing longitudinal assessment programs can combine formative and summative assessments for both learning and certification.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"212-216"},"PeriodicalIF":3.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144151291","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}

引用次数: 0

Efgartigimod Is an Effective Treatment for Triple-Seronegative Generalized Myasthenia Gravis: A Report of Three Patients. 埃夫加替莫德是治疗三血清阴性全身性重症肌无力的有效药物——附3例报告。

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-08-01 Epub Date: 2025-05-20 DOI: 10.1002/mus.28441

Jingyi Shen, Chi Ma, Ying Zhu, Ruixia Zhu

{"title":"Efgartigimod Is an Effective Treatment for Triple-Seronegative Generalized Myasthenia Gravis: A Report of Three Patients.","authors":"Jingyi Shen, Chi Ma, Ying Zhu, Ruixia Zhu","doi":"10.1002/mus.28441","DOIUrl":"10.1002/mus.28441","url":null,"abstract":"Introduction/aims: Efgartigimod has been approved for treating acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR-Ab+ gMG). The aim of this study was to report the therapeutic effect of efgartigimod in three patients with triple-seronegative (triple-SN) gMG.Methods: The medical records of three patients with triple-SN gMG who received efgartigimod at a dose of 10 mg/kg weekly for four consecutive weeks (one treatment cycle) were retrospectively reviewed. The patients were evaluated using the quantitative MG (QMG) score and the MG activities of daily living (MG-ADL) scale.Results: The first patient, who was refractory to conventional immunosuppressive therapies, demonstrated symptom improvement with a QMG reduction of 20 and an ADL reduction of 10 points following efgartigimod therapy. The second patient with a QMG score of 26 and an ADL score of 19, who showed a poor response to immunoglobulin therapy, achieved rapid symptom control after two cycles of efgartigimod treatment by week 10 (QMG: 2, ADL:1). The third patient, diagnosed with MG acute exacerbation (QMG: 6, ADL:5), responded favorably to efgartigimod therapy by week 4 (QMG:0, ADL:0).Discussion: These patient reports provide preliminary evidence supporting the effectiveness of efgartigimod as a potential alternative treatment for patients with triple-SN gMG who do not respond adequately to traditional therapies. However, multicenter randomized controlled trials are necessary to further evaluate the efficacy and safety of efgartigimod in this patient population.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"327-330"},"PeriodicalIF":3.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144102257","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}

引用次数: 0

Frequency of Guillain-Barré Syndrome During the COVID-19 Pandemic: A Multicenter Study. COVID-19大流行期间格林-巴勒综合征的发生率：一项多中心研究

IF 3.1 3区医学

Muscle & Nerve Pub Date : 2025-08-01 Epub Date: 2025-05-05 DOI: 10.1002/mus.28430

Toshiyuki Hayashi, Tatsushi Toda, Atsuro Chiba, Nobutaka Hattori, Masahiro Sonoo, Hideto Nakajima, Takanori Yokota, Yasuyuki Iguchi, Hidetomo Murakami, Haruhisa Kato, Osamu Kano, Kazuo Kitagawa, Satoshi Kitagawa, Hiroshi Nagayama, Kazumi Kimura

{"title":"Frequency of Guillain-Barré Syndrome During the COVID-19 Pandemic: A Multicenter Study.","authors":"Toshiyuki Hayashi, Tatsushi Toda, Atsuro Chiba, Nobutaka Hattori, Masahiro Sonoo, Hideto Nakajima, Takanori Yokota, Yasuyuki Iguchi, Hidetomo Murakami, Haruhisa Kato, Osamu Kano, Kazuo Kitagawa, Satoshi Kitagawa, Hiroshi Nagayama, Kazumi Kimura","doi":"10.1002/mus.28430","DOIUrl":"10.1002/mus.28430","url":null,"abstract":"Introduction/aims: The incidence of Guillain-Barré syndrome (GBS) during the COVID-19 pandemic varies according to country. We investigated the changes in the number of patients with GBS and their characteristics who were reported in Tokyo in 2020 at the beginning of the COVID-19 pandemic, compared to those two years prior.Methods: Patients with GBS who were admitted between January 2018 and December 2020 to hospitals in Tokyo were retrospectively included. We collected data on preceding infections, clinical symptoms, spinal fluid test results, nerve conduction study results, and anti-glycolipid antibody levels.Results: Thirty-four of 57 hospitals (59.6%) participated in this survey and reported 330 patients with GBS over three years. The number of patients in 2020 (n = 71) was significantly lower than in 2018 (n = 124) and 2019 (n = 135) (p = 0.0014). The number of patients with antecedent gastroenteritis (p = 0.03) or upper respiratory tract infections (p = 0.0003) in 2020 was significantly lower than the averages in 2018 and 2019. Analysis by subtype showed that the number of patients with Miller Fisher syndrome (p = 0.0009) and Bickerstaff brainstem encephalitis (p = 0.04) significantly decreased in 2020. The number of patients seropositive for anti-glycolipid antibodies, especially anti-GQ1b antibodies, decreased in 2020 (p = 0.0002).Discussion: Infection control strategies during the COVID-19 pandemic resulted in fewer patients with preceding infections in Tokyo, which contributed to a decrease in the overall number of reported patients with GBS. Precautions for infection may reduce the frequency of patients with GBS.","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"224-229"},"PeriodicalIF":3.1,"publicationDate":"2025-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144028374","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}

引用次数: 0