Efgartigimod improves non-AChR generalized Myasthenia Gravis: a real world experience

Neurological Sciences, Journal Year: 2025, Volume and Issue: unknown

Published: March 25, 2025

Language: Английский

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Antibodies in neurological diseases: Established, emerging, explorative

Immunological Reviews, Journal Year: 2024, Volume and Issue: unknown

Published: Oct. 1, 2024

Summary Within a few years, autoantibodies targeting the nervous system resulted in novel disease classification. For several of them, which we termed ‘established’, direct pathogenicity has been proven and now guides diagnostic pathways early immunotherapy. rapidly growing number further anti‐neuronal autoantibodies, role is less clear. Increasing evidence suggests that they could contribute to disease, by playing modulating on brain function. We therefore suggest three‐level classification neurological according degree experimentally strength clinical association: established, emerging, explorative. This may facilitate focusing constellations autoantibody‐mediated mechanisms have not assumed previously, including autoimmune psychosis dementia, cognitive impairment cancer, neurodegenerative diseases. Based recent data reviewed here, humoral autoimmunity represent an additional “super‐system” for health. The “brain antibody‐ome”, is, composition thousands shape neuronal function only but even healthy aging. Towards this concept, extensive research will elucidate from atomic level, autoantibody autoantibody. Such profiling can uncover biomarkers, enhance our understanding underlying mechanisms, identify selective therapies.

Language: Английский

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Risk–Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis

Advances in Therapy, Journal Year: 2024, Volume and Issue: 41(12), P. 4628 - 4647

Published: Oct. 29, 2024

This study used network meta-analysis (NMA) to inform and compare the number needed treat (NNT), harm (NNH), cost per improved outcome (CPIO) associated with more recently approved treatments for anti-acetylcholine receptor antibody-positive (anti-AChR Ab+) generalized myasthenia gravis (gMG). Clinical trials of neonatal Fc (FcRn) inhibitors, efgartigimod intravenous (IV) rozanolixizumab, complement ravulizumab zilucoplan, versus placebo (with background conventional treatment) were included in primary NMA efficacy safety outcomes. The outputs from NMAs estimate NNT NNH each treatment placebo. CPIO (2024 USD) was estimated a ≥ 3- or 5-point reduction baseline Quantitative Myasthenia Gravis (QMG) Gravis-Activities Daily Living (MG-ADL) scores. Sensitivity analyses performed adding PH20 subcutaneous (SC) eculizumab NMA. Efgartigimod IV had lowest achieving QMG, as well MG-ADL, whereas rozanolixizumab 3-point MG-ADL. similar across comparator treatments. among all assessed yielded results consistent analysis indicated that SC comparable values IV, higher compared other inhibitors. FcRn inhibitors this demonstrated clinical benefit terms an acceptable profile NNH. Within limitations meta-analysis, favorable benefit–risk better economic value ravulizumab, zilucoplan anti-AChR Ab+ gMG.

Language: Английский

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Towards personalized management of myasthenia gravis phenotypes: From the role of multi-omics to the emerging biomarkers and therapeutic targets

Autoimmunity Reviews, Journal Year: 2024, Volume and Issue: 23(12), P. 103669 - 103669

Published: Oct. 18, 2024

Language: Английский

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Elucidating Genetic Intersections: Co-Differentially Expressed Genes in Myasthenia Gravis and Idiopathic Inflammatory Myopathies and Their Role in Comorbid Pathogenesis

Heliyon, Journal Year: 2025, Volume and Issue: 11(1), P. e41442 - e41442

Published: Jan. 1, 2025

Myasthenia gravis (MG) and idiopathic inflammatory myopathies (IIM) are autoimmune disorders that can co-occur, complicating diagnosis treatment. The molecular mechanisms underlying this comorbidity not well understood. This study aims to identify common differentially expressed genes (co-DEGs) between MG IIM elucidate shared pathogenic pathways potential therapeutic targets. Transcriptomic data from the Gene Expression Omnibus (GEO) were analyzed using "limma" package in RStudio. Functional enrichment analyses performed Ontology (GO) Kyoto Encyclopedia of Genes Genomes (KEGG) pathways. A nomogram prediction model was developed, receiver operating characteristic (ROC) analysis used evaluate its diagnostic potential. Four co-DEGs identified IIM, associated with neurotransmitter transport ion channel regulation. model, incorporating three these co-DEGs, showed high predictive accuracy for complications, an area under ROC curve 0.94. Immune infiltration revealed distinct patterns particularly involving gamma delta T cells activated mast cells. identifies key genetic intersections providing insights into their pathogenesis highlighting Further experimental validation is required confirm findings.

Language: Английский

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A case of relapsed gAChR-positive autoimmune autonomic ganglionopathy treated by plasma exchange and mycophenolate mofetil

Frontiers in Neurology, Journal Year: 2025, Volume and Issue: 15

Published: Jan. 10, 2025

Autoimmune autonomic ganglionopathy (AAG) is a rare and acquired immune-mediated disease that leads to wide failure, mainly characterized by orthostatic hypotension, gastrointestinal dysfunction, anhidrosis poorly reactive pupils. This disorder usually associated with autoantibodies the ganglionic nicotinic acetylcholine receptor (gAChR-Ab). In this study, we describe case of gAChR-Ab-positive AAG patient two therapeutic stages. The responded well after first stage methylprednisolone pulse therapy subsequent low-dose prednisone. However, relapsed stopping oral second stage, repeated was less effective than before. Fortunately, multiple plasma exchange treatments improved patient's symptoms. end, prednisone mycophenolate mofetil provided significant improvement in during long-term follow-up. relatively neuroimmunological insidious onset confused clinical features, while it responds conventional immunotherapy, some patients may require immunotherapy. Emphasizing importance early detection treatment practice. Moreover, should be noted reduction withdrawal immunosuppressants slow cautious.

Language: Английский

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Preoperative multiclass classification of thymic mass lesions based on radiomics and machine learning

Cancer Imaging, Journal Year: 2025, Volume and Issue: 25(1)

Published: March 6, 2025

Apart from rare cases such as lymphomas, germ cell tumors, neuroendocrine neoplasms, and thymic hyperplasia, mass lesions (TMLs) are typically categorized into cysts, thymomas. However, the classification results cannot be determined in advance can only confirmed through postoperative pathology. Therefore, objective of this study is to rely on clinical parameters radiomic features extracted chest computed tomography (CT) scans facilitate preoperative TMLs. The model development specifically focused cysts thymomas, these most commonly encountered anterior mediastinal tumors practice. This retrospective included 400 participants 3 hospitals between September 2017 2024 due were classified 7 groups based ultimately etiology: including types A, AB, B1, B2, B3, C. All underwent contrast-enhanced CT scans, with senior radiologists delineating regions interest extract features. Additionally, participants' ages also collected for analysis. randomly allocated a training set validation at 7:3 ratio. A classifier models developed using data set, their performances evaluated set. exhibited good performance accuracies 0.8547. assist early diagnosis personalized treatment strategies patients

Language: Английский

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Telitacicept as an alternative to non-steroidal immunosuppressive therapies in the treatment of myasthenia gravis: a study on clinical efficacy and steroid-sparing effect

Frontiers in Immunology, Journal Year: 2025, Volume and Issue: 16

Published: March 24, 2025

Introduction Myasthenia Gravis (MG) is an autoimmune disorder characterized by impaired neuromuscular junction (NMJ) transmission. Current treatments for MG include steroids and nonsteroidal immunosuppressive therapies (NSISTs). However, approximately 20% of patients show a poor response to these therapies, which are often associated with significant side effects. Telitacicept, novel recombinant fusion protein targeting the BAFF/APRIL pathway, has shown promise in treating diseases, including MG. Methods This retrospective study compared efficacy telitacicept monotherapy (10 patients) NSISTs (16 sequential therapy (6 managing at The First Affiliated Hospital Wenzhou Medical University (July 2020-November 2024). primary endpoint was time achieve minimal symptom expression (MSE), secondary change mean daily prednisone dosage from baseline month 4. Results Among telitacicept-treated patients, 80% achieved MSE within 4 months, reduction dose (from 45.00 mg 6.25 mg, P < 0.001). In contrast, only 12.5% group MSE, no ( = 0.091). (efgartigimod followed telitacicept) maintained stable disease conditions. Conclusion Telitacicept effective inducing rapidly offers steroid-sparing effect, making it promising alternative traditional fewer effects patients.

Language: Английский

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Demographic and Geographic Trends in Myasthenia Gravis–Related Mortality in the United States, 1999–2022

Neurology, Journal Year: 2025, Volume and Issue: 104(8)

Published: March 30, 2025

The prevalence and incidence of myasthenia gravis (MG) have been increasing, globally in the United States. literature lacks data on MG-related mortality (MGRM) its trends We aimed to examine nationwide demographic geographic MGRM from 1999 2022. This retrospective population-based study used regarding deaths (MGRD) Centers for Disease Control Prevention Wide-ranging Online Data Epidemiologic Research records. International Classification Diseases (ICD) code, G70.0, was identify MG. stratified by sex, age groups (25-64 years older than 64 years), race ethnicity, geographical location. Joinpoint regression performed age-adjusted rates (AAMRs). Sensitivity analysis using MG as an underlying cause death (UCD). During period, there were 37,075 MGRD (89.6% years, 44.7% female individuals). From 2022, AAMR increased significantly 6.21 (95% CI 5.58-6.58) per 1 million population 9.51 9.14-9.88) population, with average annual percent change +2.42 1.98-2.87). increase observed regardless group, region, or ethnicity. 66.3% male individuals 29.6% over period. For aged 65 older, a concerning 82.35% 28.23 47.36. There peak during coronavirus disease 2019 pandemic (2020-2022), sensitivity revealed that trend remained consistent both UCD contributing death. rising 23-year period is warrants investigation into causes this trend. most prominent individuals. growing burden States might pose serious challenge health care future. Limitations include reliance ICD codes. Future work needs take these disparities consideration focus improving MGRM.

Language: Английский

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Perioperative management of a patient with myasthenia gravis: a case report, treatment of myasthenic crisis and literature review

EMERGENCY MEDICINE, Journal Year: 2025, Volume and Issue: 21(2), P. 189 - 198

Published: April 10, 2025

Surgical intervention, such as thymectomy, and a comprehensive approach to the treatment of patients with myasthenia gravis can improve quality duration their lives. At same time, perioperative management should be carefully selected. These require attention because risk developing myasthenic crisis complicated recovery from it. Specific care are paid administration any medications, especially neuromuscular blocking agents. Patients gravis, having pathology acetylcholine receptors, unexpec-tedly sensitive non-depolarising agents resistant depolarising succinylcholine.

Language: Английский

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