Journal of the Peripheral Nervous System最新文献

{"title":"Correction to “Results From a Phase 1 Study Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of ANX005, a C1q Inhibitor, in Patients With Guillain–Barré Syndrome”","authors":"","doi":"10.1111/jns.70024","DOIUrl":"https://doi.org/10.1111/jns.70024","url":null,"abstract":"<p>Q. D. Mohammad, Z. Islam, N., Papri, et al. “Results From a Phase 1 Study Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of ANX005, a C1q Inhibitor, in Patients With Guillain–Barré Syndrome,” <i>Journal of the Peripheral Nervous System</i> 30 (2025): e70009. https://doi.org/10.1111/jns.70009</p><p>FIGURE 7 | Change in MRC from baseline for ANX005 and placebo (<i>N</i> = 26). MRC, Medical Research Council.</p><p>We apologize for this error.</p>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-05-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jns.70024","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144074633","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}

{"title":"Patient-Reported Outcome Measures for Assessing Health-Related Quality of Life in Patients With Polyneuropathies, Focusing on Guillain-Barré Syndrome and Chronic Inflammatory Demyelinating Polyneuropathy: A Systematic Review of Measurement Properties","authors":"Farah Pelouto,&nbsp;Adája E. Baars,&nbsp;Nowshin Papri,&nbsp;Juanita A. Haagsma,&nbsp;Bart C. Jacobs,&nbsp;Caroline B. Terwee","doi":"10.1111/jns.70022","DOIUrl":"https://doi.org/10.1111/jns.70022","url":null,"abstract":"<p>Guillain-Barre syndrome (GBS) and chronic inflammatory demyelinating polyneuropathy (CIDP) are immune-mediated peripheral neuropathies. Despite treatment, patients may report residual deficits, pain, and fatigue with considerable impact on quality of life. A systematic review was conducted of the methodological quality of current patient-reported outcome measures (PROMs) for measuring health-related quality of life (HRQoL) in patients with GBS and CIDP. A literature search was conducted in EMBASE, MEDLINE, Web of Science, and Google Scholar. PROMs developed to measure (aspects of) HRQoL in patients with polyneuropathy were classified using the Wilson and Cleary model. Measurement properties were evaluated in accordance with Consensus-based Standards for selection of health Measurement Instruments (COSMIN) guideline. A total of 57 articles identified 31 unique PROMs that are used for measuring HRQoL in patients with polyneuropathies. Of these, 22 measured symptom status, 19 functional status, and 4 general health perception. Eight PROMs were developed or validated in patients with GBS/CIDP. None of the PROMs demonstrated sufficient content validity for recommendation in this population. Only the Rasch-built Fatigue Severity Scale (R-FSS) performed sufficiently across all other measurement properties. The Inflammatory Rasch-built Overall Disability Scale (I-RODS) and IN-QoL are not recommended for use because of insufficient construct validity. GBS Patient Experience Questionnaire, Chronic Acquired Polyneuropathy Patient-Reported Index (CAP-PRI), Fatigue Severity Scale (FSS), R-FSS, Rotterdam Handicap Scale (RHS) and the 36-Item Short Form Health Survey (SF-36) need further validation. PROMs of good quality assessing all relevant aspects of HRQoL are required for better insight in HRQoL in patients with GBS and CIDP.</p>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jns.70022","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143949953","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}

{"title":"Advanced Hybrid Closed-Loop Insulin Delivery Is Associated With Improved Glycemic Indicators and Normalization of Small Nerve Fibre Structure in Adults With Type 1 Diabetes","authors":"Hoda Gad,&nbsp;Einas Elgassim,&nbsp;Kawsar Mohamed Ayon Mohamud,&nbsp;Najlaa Sultan Al-Naimi,&nbsp;Hamad Ali Al-Sharshani,&nbsp;Ibrahim Mohammed,&nbsp;Mariam A. Al-Malaheem,&nbsp;Dorothy J. Quadros,&nbsp;Alhanoof AlJalahma,&nbsp;Neila Lamine,&nbsp;Ahmad Yaser Alhaddad,&nbsp;Hussein Ahmed Hussein Zaky Aly,&nbsp;John-John Cabibihan,&nbsp;Abdulaziz Al-Ali,&nbsp;Kishor Kumar Sadasivuni,&nbsp;Ioannis N. Petropoulos,&nbsp;Georgios Ponirakis,&nbsp;Hamda A. Ali,&nbsp;Dabia AlMohanadi,&nbsp;Khaled Baagar,&nbsp;Rayaz A. Malik","doi":"10.1111/jns.70026","DOIUrl":"https://doi.org/10.1111/jns.70026","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Hyperglycemia is a major driver of diabetic peripheral neuropathy (DPN) in type 1 diabetes mellitus (T1DM). Advanced hybrid closed-loop (AHCL) technologies improve glycemic control and reduce glycemic variability and may improve DPN.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Patients with T1DM treated for 9.8 ± 0.32 months with the 780G SmartGuard system (<i>n</i> = 14) were compared with patients with T1DM on MDI (<i>n</i> = 20) and healthy controls (<i>n</i> = 15). Time in range (TIR), time above range (TAR), time below range (TBR), glycemic variability, and HbA_1c were evaluated, and corneal confocal microscopy (CCM) was undertaken to quantify corneal nerve fiber density (CNFD), branch density (CNBD), fiber length (CNFL), and inferior whorl length (IWL).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Participants using the 780G system had a significantly higher TIR (<i>p</i> &lt; 0.001), lower glycemic variability (<i>p</i> = 0.02), and less time in level 2 hyperglycemia (<i>p</i> = 0.01), level 1 hyperglycemia (<i>p</i> = 0.04), and level 2 hypoglycemia (<i>p</i> = 0.008) compared with the MDI group. CNFD (<i>p</i> = 0.02), CNBD (<i>p</i> = 0.04), CNFL (<i>p</i> = 0.04), and IWL (<i>p</i> &lt; 0.001) were significantly higher in the 780G group compared with the MDI group and comparable to healthy controls.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The MiniMed 780G with SmartGuard improves glycemic control and variability with an improvement in small nerve fiber morphology in patients with DPN.</p>\u0000 </section>\u0000 </div>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143944697","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":"Clinical and Electrophysiological Characterization of Diabetic Neuropathy in a Sub-Saharan African Cohort","authors":"Samuel Eric Chokote,&nbsp;Gaelle Lemdjo,&nbsp;Juan Francisco Idiaquez Rios,&nbsp;Aurelien Tejiozem Anakeu,&nbsp;Leonard Ngarka,&nbsp;Leonard N. Nfor,&nbsp;Michel K. Mengnjo,&nbsp;Wepnyu Y. Njamnshi,&nbsp;Herman Nestor Tsague Kengni,&nbsp;Ruth Joelle Ngongang,&nbsp;Gilles Simeni,&nbsp;Lylian Piameu,&nbsp;Alain Balla Nkonda,&nbsp;Faustin Yepnjio,&nbsp;Godwin Y. Tatah,&nbsp;Umapathi N. Thirugnanam,&nbsp;Alfred Kongnyu Njamnshi","doi":"10.1111/jns.70021","DOIUrl":"https://doi.org/10.1111/jns.70021","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Diabetic neuropathy (DN) is the most frequent complication of diabetes mellitus, contributing to increased morbidity and mortality. Previous clinical studies on DN in sub-Saharan Africa (sSA) have used purely clinical approaches, potentially underestimating the true magnitude of this disease. This study was designed to determine the prevalence of definite diabetic neuropathy and describe the different subtypes using objective small and large fiber function measures.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This was a hospital-based cross-sectional study that included diabetes and prediabetes patients, followed up at Jordan Medical Services, Yaoundé, Cameroon, between March 2022 and February 2023. The “Toronto Clinical Neuropathy Score” and “Douleur Neuropathique en 4” questionnaires were used for clinical evaluation. Autonomic symptoms were equally recorded. Nerve conduction studies and Sudoscan were used for electrophysiological assessments of large and small fibre functions.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Eighty-four participants were included; 91.7% had type 2 DM, 2.4% had type 1 DM, and 6% had glucose intolerance. DN was found in 73/84 (86.9%). Diabetic sensorimotor polyneuropathy (DSP) was the most frequent subtype (63.8%), followed by diabetic autonomic neuropathy (40.5%), mononeuropathy (36.9%), asymmetric axonal sensory neuropathy (4.8%) and treatment-induced neuropathy of diabetes (TIND) in 1.2% of patients. The prevalence of large and small fibre neuropathies was 38.1% and 25.0%, respectively.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The prevalence of DN and specifically DSP in our study was higher than previously described in African literature. We identified subtypes never before reported in sSA, mainly small fibre neuropathy and TIND. This may have management and policy implications.</p>\u0000 </section>\u0000 </div>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jns.70021","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143925751","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}

{"title":"Differential Effects of Visceral and Subcutaneous Adiposity on Peripheral Neuropathy","authors":"Georgios Ponirakis,&nbsp;Leeza Peerzada,&nbsp;Ioannis N. Petropoulos,&nbsp;Hoda Gad,&nbsp;Sidra Abdulshakoor,&nbsp;Jenneth M. Concepcion,&nbsp;Sara H. Khalfalla,&nbsp;Iynas S. A. Elamin,&nbsp;Abeer T. H. AlZawqari,&nbsp;Einas Elgassim,&nbsp;Areej Baraka,&nbsp;Ziyad R. Mahfoud,&nbsp;Marwa A. El Deeb,&nbsp;Nahla Afifi,&nbsp;Rayaz A. Malik","doi":"10.1111/jns.70025","DOIUrl":"https://doi.org/10.1111/jns.70025","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>Obesity increases the risk of diabetic neuropathy. This study investigates the impact of visceral (VAT) and subcutaneous adipose tissue (SAT) volume on peripheral neuropathy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A total of 302 adults from the Qatar Biobank (QBB) underwent iDXA to measure VAT and SAT volumes, intima media thickness (IMT), and peripheral neuropathy assessments using corneal confocal microscopy (CCM), vibration perception threshold (VPT), and the DN4 questionnaire.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The QBB cohort was aged 43.9 ± 12.9 years, of whom 43.7% were women, 42.1% had obesity, 17.4% had type 2 diabetes (T2D) and 10.9% had hypertension. VAT was associated with T2D, hypertension, higher HbA1c, diastolic blood pressure, triglycerides, and inflammatory markers, and lower HDL (<i>p</i> &lt; 0.0001). There were no significant associations between SAT and these cardiovascular risk factors. VAT volume was associated with lower corneal nerve inferior whorl length (IWL) (<i>p</i> &lt; 0.05) and higher VPT (<i>p</i> = 0.01), partially mediated by elevated HbA1c (<i>p</i> &lt; 0.05, <i>p</i> = 0.001) and IMT (<i>p</i> &lt; 0.0001), while its association with neuropathic symptoms was fully mediated by systolic blood pressure (<i>p</i> &lt; 0.05), T2D (<i>p</i> &lt; 0.01), and triglycerides (<i>p</i> = 0.05). SAT showed no associations with measures of neuropathy.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>VAT but not SAT is associated with peripheral neuropathy. This study underscores the need to target VAT to improve neuropathy.</p>\u0000 </section>\u0000 </div>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143925750","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":"Acquired Transthyretin Amyloidosis in Domino Liver Transplantation Treated With Gene-Silencers","authors":"Alessandro Salvalaggio,&nbsp;Alberto Cipriani,&nbsp;Luisa Frizziero,&nbsp;Manuele Marasca,&nbsp;Mario Cacciavillani,&nbsp;Chiara Briani","doi":"10.1111/jns.70023","DOIUrl":"https://doi.org/10.1111/jns.70023","url":null,"abstract":"","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-05-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143909363","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":"New Approaches Based on Serial-Block Face Electron Microscopy to Investigate the Peripheral Nervous System","authors":"Vitalijs Borisovs,&nbsp;Mario Bossi,&nbsp;Laura Matino,&nbsp;Paola Marmiroli,&nbsp;Guido Cavaletti","doi":"10.1111/jns.70019","DOIUrl":"https://doi.org/10.1111/jns.70019","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background and Aims</h3>\u0000 \u0000 <p>Serial block face scanning electron microscopy (SBF-SEM) enables automated 3D imaging of specimens with ultrastructural resolution. However, its application is often restricted due to the complex and labor-intensive nature of the processes involved. This study addresses the challenges associated with sample preparation and the final 3D reconstruction for ultrastructural analysis of peripheral nerves and dorsal root ganglia (DRG) specimens.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Specimens from the caudal nerve and DRG of mice were prepared for SBF-SEM using three different techniques: (1) manual high molecular weight staining, regarded as the gold standard, (2) automated standard transmission electron microscopy (TEM) preparation, and (3) automated uranyl-free en bloc preparation. The acquired data were processed by combining different software programs for image analysis and 3D rendering.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Upon analyzing all samples, the high molecular weight method demonstrated its superiority. Nonetheless, the two alternative methods produced high-quality images of the caudal nerve. Consequently, 3D rendering was successfully achieved for all samples using an automated approach. The investigation of DRG specimens posed greater challenges with the standard TEM preparation due to the low contrast of smaller organelles compared to the cytosol, whereas the uranyl-free protocol provided significantly improved contrast.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Interpretation</h3>\u0000 \u0000 <p>Our findings indicate that automated uranyl-free staining can effectively compete with the traditional gold standard manual and uranyl-based staining methods, albeit with some limitations. Furthermore, high-quality SBF-SEM imaging is attainable, especially in peripheral nerves, using samples prepared via the standard TEM method, thereby facilitating the analysis of previously embedded samples even if they were not specifically prepared for 3D examination.</p>\u0000 </section>\u0000 </div>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jns.70019","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143849037","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}

{"title":"Genetic and Clinical Features of 10 Families With Hereditary Sensory Neuropathies","authors":"Ke Xu,&nbsp;Zhongzheng Li,&nbsp;Mengli Wang,&nbsp;Lei Liu,&nbsp;Sen Zeng,&nbsp;Xiaobo Li,&nbsp;Wanqian Cao,&nbsp;Shunxiang Huang,&nbsp;Huadong Zhao,&nbsp;Yan Yang,&nbsp;Yongzhi Xie,&nbsp;Zhengmao Hu,&nbsp;Beisha Tang,&nbsp;Ruxu Zhang","doi":"10.1111/jns.70020","DOIUrl":"https://doi.org/10.1111/jns.70020","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background and Objectives</h3>\u0000 \u0000 <p>Hereditary sensory neuropathies (HSNs) are a group of genetically and clinically heterogeneous diseases. Our study aims to summarize the genetic and clinical features of HSNs in 10 Chinese families.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Clinical data from 10 families with HSNs were collected retrospectively. Genetic screening was performed by whole exome sequencing (WES). Repeated-primed PCR and capillary electrophoresis were performed for WES-negative patients to analyze repeat expansions in <i>RFC1</i>.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among the 10 probands with HSNs, eight cases were sporadic, and two had a positive family history. Six probands had early-onset (onset age &lt; 20 years). Seven probands presented with pure-HSNs type, and three exhibited HSNs-complex type with ataxia. Variants in the <i>NTRK1, SPTLC1, COX20, PUM1,</i> and <i>RFC1</i> genes were detected in six probands. A novel variant, c.444C&gt;A (p.N148K), in <i>NTRK1</i> was identified in an autosomal recessive inheritance family with HSAN-IV, and a novel variant, c.182dup (p.H61Qfs*31), in <i>PUM1</i> was identified in a proband with adult-onset paresthesia and mild cerebellar ataxia. Additionally, biallelic expansion of the pathogenic variant structure (AAGGG)exp repeat amplification in the <i>RFC1</i> gene was identified in a proband with sensory neuropathy, ataxia, and right vestibular hypofunction.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>The novel variants in <i>NTRK1</i> and <i>PUM1</i> expanded the genotypic spectrum of HSNs. This study highlights the associations between sensory neuropathies and other symptoms, particularly cerebellar ataxia. Given the ultra-rarity of HSNs, future multicenter studies with larger cohorts may facilitate the identification of novel variants, improve genetic diagnostic rates, and enhance disease recognition.</p>\u0000 </section>\u0000 </div>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143818391","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":"Diagnosis and Management of Multifocal Motor Neuropathy in the United Kingdom: A Multicentre Survey","authors":"Yusuf A. Rajabally,&nbsp;Christina Englezou,&nbsp;Grace Cluett,&nbsp;Roberto Bellanti,&nbsp;Simon Rinaldi,&nbsp;Mow Wei Chin,&nbsp;Robert Hadden,&nbsp;Madalina Roman,&nbsp;Channa Hewamadduma,&nbsp;Ashleigh Murray,&nbsp;Amar Elsaddig,&nbsp;Tim Lavin,&nbsp;Oliver Cousins,&nbsp;Niranjanan Nirmalananthan,&nbsp;Joumana Freiha,&nbsp;Chinar Osman,&nbsp;Matthew Evans,&nbsp;Aisling Carr,&nbsp;James K. L. Holt","doi":"10.1111/jns.70018","DOIUrl":"https://doi.org/10.1111/jns.70018","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>We conducted a survey to determine the current diagnosis and treatment of multifocal motor neuropathy (MMN) in the United Kingdom.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Demographic, diagnostic and treatment data were collected at nine UK neuroscience centres.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Ninety-five subjects were included. Mean age at diagnosis was 49.9 years (SD: 11.4). Males were more commonly affected (ratio: 1.9:1). Diagnostic delay was &gt; 1 year from the time of first neurological assessment, in &gt; 50% of subjects. Applying modified EFNS/PNS 2010 criteria, 69/95 (72.6%) had definite MMN, 10/95 (10.5%) had probable MMN, 15/95 (15.8%) had possible MMN, through treatment responsiveness in 9/15 (60%) and 1/95 (1.1%) did not meet criteria. Cerebrospinal fluid examination, anti-GM1 antibody testing and brachial plexus magnetic resonance imaging were non-contributory. Immunoglobulin response was reported in 90/92 subjects (97.8%), and 84/90 (93.3%) remained on treatment after a mean of 9.4 years, at a mean dose of 26.2 g/week (range: 4–114). Mean long-term immunoglobulin dose was 30%–60% higher than reported in neighbouring countries. Contrasting with previous reports of frequent loss of immunoglobulin response and functional decline, our physician-assessed long-term outcome was favourable (stable or improving) in 74/84 (88.1%) treated subjects.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Interpretation</h3>\u0000 \u0000 <p>MMN diagnosis and treatment in the United Kingdom are comparable to that of neighbouring countries and follow existing guidelines. Diagnostic delay after the first neurological assessment is considerable. Electrophysiology shows at least one definite/probable conduction block in nearly 90% of cases. The mean long-term immunoglobulin dose is higher in the United Kingdom than reported elsewhere, although highly variable. Whether higher doses of immunoglobulin may improve long-term outcomes requires further study.</p>\u0000 </section>\u0000 </div>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jns.70018","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143818392","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}

{"title":"Neuropathy Progression in Acquired Amyloidosis After Domino Liver Transplantation","authors":"Catarina Falcão de Campos,&nbsp;Miguel Miranda,&nbsp;Isabel Castro,&nbsp;José Castro,&nbsp;Miguel Oliveira Santos,&nbsp;Isabel Conceição","doi":"10.1111/jns.70016","DOIUrl":"https://doi.org/10.1111/jns.70016","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background and Aims</h3>\u0000 \u0000 <p>Domino liver transplantation (DLT) has been used to address the shortage of donor organs. However, recipients of liver grafts from patients with hereditary transthyretin amyloidosis (ATTRv) develop de novo transthyretin (TTR) amyloidosis. Our aim is to describe the clinical presentation of patients with acquired TTR amyloidosis and compare the rate of neuropathy progression (NP) with untreated ATTRv amyloidosis with neuropathy (ATTRv-NP) patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Patients with acquired TTR amyloidosis after DLT followed at a reference centre were evaluated. Medical records were reviewed for clinical characterization and systematic assessment of neuropathy. NP was defined as an increase in neuropathy impairment score of the lower limbs (NIS-LL) at 12 months and compared to a historical control group of untreated ATTRv amyloidosis patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Twenty-four patients with acquired ATTR amyloidosis were included. Time from DLT to neuropathy onset was 9 <span></span><math>\u0000 <semantics>\u0000 <mrow>\u0000 <mo>±</mo>\u0000 </mrow>\u0000 <annotation>$$ pm $$</annotation>\u0000 </semantics></math> 2.0 years and the majority of patients reported feet sensory changes as first symptom. Thirteen patients with ≥ 2 evaluations with 12-months interval were analysed. Almost all patients developed a neuropathic phenotype with small nerve fibre involvement. Neuropathy progression was similar to untreated ATTRv-NP patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Interpretation</h3>\u0000 \u0000 <p>Recipients from liver grafts of ATTRv patients develop de novo amyloidosis with clinical presentation and NP similar to untreated ATTRv amyloidosis patients. Study of these patients might help elucidate the pathways for ATTR fibril formation.</p>\u0000 </section>\u0000 </div>","PeriodicalId":17451,"journal":{"name":"Journal of the Peripheral Nervous System","volume":"30 2","pages":""},"PeriodicalIF":3.9,"publicationDate":"2025-04-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143793606","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}