Treatment Of Myasthenia Gravis Research Articles

Abdeg technology for the treatment of myasthenia gravis: efgartigimod drug experience

ABSTRACT Introduction Myasthenia gravis is characterized by fluctuating muscle weakness that improves with rest and worsens with effort or throughout the day. Areas Covered Efgartigimod is a human IgG1–derived Fc fragment modified at five residues to increase its affinity for the neonatal Fc receptor by Abdeg technology. Thus, efgartigimod binds to the neonatal Fc receptor and decreases the levels of IgG, including autoantibodies of this isotype. For acetylcholine receptor (AChR) antibody-positive patients, efgartigimod had a higher proportion of MG-ADL responders than placebo in the first treatment cycle. The mean changes of multiple outcomes from baseline were better for efgartigimod than placebo from weeks 1 to 7 in the first treatment cycle. The decrease of IgG and AChR autoantibodies was 61.3% and 57.6% one week after the first treatment cycle ends, respectively. The most common adverse events were headache, nasopharyngitis, nausea, and diarrhea, which occurred in the same proportion in the efgartigimod and placebo groups. Urinary and upper respiratory tract infections were twice as frequent in efgartigimod-treated patients. Expert Opinion Efgartigimod was efficacious and safe for generalized myasthenia patients with AChR antibody-positive patients. These findings need to be confirmed in AChR antibody-negative patients, and long-term safety studies are currently ongoing.

Subxiphoid and subcostal arch versus unilateral video-assisted thoracic surgery approaches to thymectomy for myasthenia gravis.

Thymectomy is an important treatment for myasthenia gravis (MG). We conducted this study to compare the clinical outcomes of the recently introduced subxiphoid and subcostal arch thymectomy (SASAT) approach with those of the standard unilateral video-assisted thoracoscopic surgery (VATS). We analyzed, retrospectively, the perioperative, and long-term outcomes of 179 consecutive MG patients (age 18-65years), who underwent SASAT or unilateral VATS-extended thymectomy between July, 2012 and May, 2019. All demographic and clinical characteristics were comparable in the two groups. The median surgical time, estimated blood loss, thoracotomy conversion rate, total and chest drainage, and complications did not differ significantly between the groups. The visual analog scale (VAS) score was significantly lower in the SASAT group. Complete stable remission (CSR) was achieved in a significantly larger proportion of the SASAT group patients and was significantly higher in women than in men. The Quantitative MG score was significantly lower in the SASAT group. Patients in the MG Foundation of America Clinical Classification groups I and II achieved better remission rates than those in groups III-V. SASAT is a safe and feasible MG treatment, which may yield better outcomes than unilateral VATS and improve the quality of treatment.

Morvan syndrome presenting with agrypnia excitata in post-thymectomy myasthenia gravis: a case report

Background: Morvan syndrome is characterized by neuromyotonia, dysautonomia, and various neuropsychiatric symptoms, as well as sleep disturbances, although these are less common than neuromuscular symptoms. Herein, we report a case of Morvan syndrome with peculiar sleep disturbances, documented via polysomnography. Case Report: We present herein the case of a 67-year-old man who developed agitation and severe insomnia after undergoing a thymectomy for the treatment of myasthenia gravis, which was subsequently diagnosed as agrypnia excitata due to Morvan syndrome, based on 24-hour polysomnography. Conclusion: We presented the 24-hour polysomnographic findings of a case of agrypnia excitata in Morvan syndrome. An extended polysomnography, however, might be helpful in analyzing sleep disturbances in Morvan syndrome.

Criteria for Treatment Response in Myasthenia Gravis: Comparison Between Absolute Change and Improvement Percentage in Severity Scores.

BackgroundThe absolute change in the severity score between the baseline and pre-specified time frame (absolute criterion) was recommended as a criterion for myasthenia gravis (MG) treatment response. But heterogeneity of disease severity might dilute major changes in individual patients. The rationality of relative criterion (improvement percentage) had not been evaluated in treatment response in patients with MG.ObjectivesTo investigate the consistency between an absolute criterion and a relative criterion in the evaluation of treatment response in patients with MG.MethodsWe retrospectively analyzed the treatment response to a 3-month standardized treatment protocol with only glucocorticoid in 257 MG patients native to immunological treatments. With the commonly used absolute criterion, cut-offs of relative criteria were generated with the receiver operating characteristic (ROC) curve in the whole cohort and in patients with different degrees of baseline severity stratified by pre-treatment quantitative myasthenia gravis score (QMGS). The consistency between absolute and relative criteria was examined with Cohen's Kappa test and Venn diagrams.ResultsThe absolute and relative criteria had an overall substantial consistency (Kappa value, 0.639, p < 0.001) in the cohort. The Kappa values were substantial to almost perfect in mild and moderate groups and moderate in severe groups between the absolute and relative criteria (all p ≤ 0.001). More patients were classified as responsive with an absolute criterion while as unresponsive with a relative criterion in the moderate and severe groups.ConclusionsThe overall consistency between absolute and relative criteria was substantial in the whole cohort. The inconsistency between the two criteria was mainly from the moderate or severe patients at the baseline.

Assessment of length of stay and cost of minimally invasive versus open thymectomies in patients with myasthenia gravis in Florida.

Thymectomy has become a standard component in treatment for myasthenia gravis. The best surgical approach is still subject to debate. Minimally invasive surgery may have a lower mortality and morbidity rate, improved cosmetic results, and equivalent efficacy at improving neurologic symptoms to open approaches. We compared the perioperative outcomes and cost between the two techniques. We queried Florida Inpatient Discharge Dataset for patients who underwent thymectomy and had a primary diagnosis of non-thymomatous myasthenia gravis using International Classification of Diseases (ICD)-9 and ICD-10 codes to carry out this retrospective cohort study. The dates ranged between January 1st, 2013, to December 31st, 2018. We compared outcomes of patients who underwent minimally invasive thymectomy versus those who had open thymectomy. An open approach was used in 108 patients, whereas a minimally invasive approach was used in 40 patients. Minimally invasive surgery group had a shorter length of stay (3.0 vs. 6.0 days, P<0.001) and had a non-significant lower total cost ($18.4K vs. $22.1K, P=0.186). After adjusting for age and Elixhauser score, length of stay for minimally invasive group was 32% (P=0.01) lower compared to the open surgery group. Patients who underwent minimally invasive thymectomy for Myasthenia gravis had a significantly shorter length of stay and a lower, although not significant, overall cost.

Good outcomes after aggressive treatment of immune checkpoint inhibitor related myasthenia gravis: a case series

IntroductionMyasthenia gravis (MG) is a rare but potentially life-threatening complication of immune- checkpoint inhibitor (ICI) therapy and often co-presents with myositis and myocarditis. We present a series of six patients from a tertiary oncology center.MethodsWe reviewed The Christie Hospital database of immunotherapy-related toxicity from 2017–2020. Symptom severity was assessed using the MGFA classification.ResultsSix patients with ICI-related MG were identified. The median number of preceding immunotherapy doses was 2. Common initial presenting complaints were ptosis (100%) and diplopia (50%) with 2/6 patients showing severe (MGFA &gt; II) symptoms. Five patients had concomitant myositis and myocarditis. AChR or MuSK autoantibodies were positive in three patients. Two patients required advanced respiratory support.All patients received urgent treatment with intravenous methylprednisolone and mycophenolate. Five also received intravenous immunoglobulin and a single patient was treated additionally with abatacept and rituximab. No patient died from MG-related complications. All were successfully discharged following the acute episode.DiscussionPrevious case series of ICI-related MG have reported high mortality rates, particularly in those with overlapping myositis/myocarditis. In our cohort we demonstrate good outcomes associated with early aggressive treatment with IVIG, IVMP, and mycophenolate. An agreed national treatment protocol or clinical discussion forum would be beneficial.jamweaver75@gmail.com17

Effectiveness of modified Buzhong Yiqi decoction in treating myasthenia gravis: study protocol for a series of N-of-1 trials

BackgroundMyasthenia gravis (MG) is an acquired autoimmune disease with high heterogeneity. The disease is chronic, relapsing repeatedly and progressive with acute exacerbation occasionally. Although the treatment of MG has developed, it is still unsatisfactory and has some unexpected side effects. Traditional Chinese medicine (TCM) has shown great potential in MG treatment, including relief of muscle weakness syndrome, improvement of patient’s quality of life, and reduction of side effects of western medicine. The purpose of this study is to evaluate the effectiveness of modified Buzhong Yiqi decoction (MBYD) as an add-on therapy for MG through a small series of N-of-1 trials.MethodsSingle-centre, randomized, double-blind, 3 crossover N-of-1 trials will be conducted to enroll patients with MG diagnosed as spleen-stomach deficiency syndrome or spleen-kidney deficiency syndrome in TCM. Each N-of-1 trial has 3 cycles of two 4-week periods containing the MBYD period and placebo period. The wash-out interval of 1 week is prior to switching each period. Primary outcome: quantitative myasthenia gravis (QMG). Secondary outcomes: the following scales: myasthenia gravis composite (MGC), myasthenia gravis activities of daily living profile (MG-ADL), myasthenia gravis quality of life (MG-QOL); the level of CD4+FoxP3+Treg cells and cytokines (IL-4, IL-17A, INF-γ, TGF-β) in the peripheral blood; the alterations of the composition of gut microbiota; reduction of the side effects of western medicine.DiscussionUsed by WinBUGS software, we will conduct a hierarchical Bayesian statistical method to analyze the efficacy of MBYD in treating MG in individuals and populations. Some confounding variables such as TCM syndrome type and potential carryover effect of TCM will be introduced into the hierarchical Bayesian statistical method to improve the sensitivity and applicability of the trials, and the use of prior available information within the analysis may improve the sensitivity of the results of a series of N-of-1 trials, from both the individual and population level to study the efficacy of TCM syndrome differentiation. We assumed that this study would reveal that MBYD is effective for MG and provide robust evidence of the efficacy of TCM to treat MG.Trial registrationChinese Clinical Trial Register, ID: ChiCTR2000040477, registration on 29 November 2020.

Corticosteroid Treatment-Resistance in Myasthenia Gravis.

Chronic, high-dose, oral prednisone has been the mainstay of myasthenia gravis treatment for decades and has proven to be highly beneficial in many, toxic in some way to all, and not effective in a significant minority. No patient characteristics or biomarkers are predictive of treatment response leading to many patients suffering adverse effects with no benefit. Presently, measurements of treatment response, whether taken from clinician or patient perspective, are appreciated to be limited by lack of good correlation, which then complicates correlation to biological measures. Treatment response may be limited because disease mechanisms are not influenced by corticosteroids, limits on dosage because of adverse effects, or individual differences in corticosteroids. This review evaluates potential mechanisms that underlie lack of response to glucocorticoids in patients with myasthenia gravis.

A Comparative Study of Video-Assisted Thoracoscopic Surgery Versus Median Sternotomy Thymectomy in Myasthenia Gravis

Background: Thymectomy is a standard treatment for myasthenia gravis (MG) patients. Although there are different approaches for thymectomy available, the standard approach is still known as a median sternotomy. Currently, the video-assisted thoracoscopic (VATS) approach is increasing in popularity. It has shown benefits in less blood loss, less postoperative pain, and improvement of recovery after surgery. However, there are still controversies in the outcomes of these two approaches. Objective: To compare perioperative and neurological outcomes of thymectomy between the median sternotomy approach and the VATS approach. Materials and Methods: One hundred twenty-three patients underwent thymectomy between January 1, 2012 and December 30, 2020 and were enrolled in the present retrospective study. They were classified into two groups depending on the approach of the surgery. The perioperative and neurological outcomes were analyzed and compared between the two groups. The analyses were performed using students’ t-test, Mann-Whitney test, chi-square, or Fisher’s exact test. Results: There were 72 patients in the median sternotomy group and 51 patients in the VATS group, and no death in both groups. There were also no significant differences between the two groups regarding surgical time, postoperative pain, postoperative complications, and neurological outcomes. Intraoperative blood loss, intercostal drainage volume and duration, and length of hospital stay were significantly less in the VATS group. Complete remission was significantly higher in VATS group. The median follows up time was significantly longer in the median sternotomy group. Conclusion: The VATS approach for thymectomy had shown good outcomes, which were not inferior to the median sternotomy approach. Keywords: VATS Thymectomy; MG; Thymoma; Thymectomy; Minimally invasive thymectomy; Myasthenia gravis; Left VATS thymectomy; Subxiphoid VATS thymectomy; Anterior mediastinal tumor; Anterior mediastinal mass

Anti-complement Agents for Autoimmune Neurological Disease.

In recent years, there has been increasing recognition of the diversity of autoimmune neurological diseases affecting all levels of the nervous system. A growing understanding of disease pathogenesis has enabled us to better target specific elements of the immune system responsible for the cell dysfunction and cell destruction seen in these diseases. This is no better demonstrated than in the development of complement directed therapies for the treatment of complement mediated autoimmune neurological conditions. Herein, we describe the basic elements of the complement cascade, provide an overview of select autoimmune neurological diseases whose pathogenesis is mediated by complement, the effector system of autoantigen bound autoantibodies, and discuss the complement directed therapies trialed in the treatment of these diseases. Several complement-directed therapies have demonstrated benefit in the treatment of autoimmune neurological diseases; we also review the trials resulting in the approval of these therapies for the treatment of AChR Ab-positive myasthenia gravis (MG) and neuromyelitis spectrum disorder. Finally, on the heels of the recent successes described, we discuss possibilities for the future, including additional targeted therapies with greater ease of administration, improved risk profiles, and other possible uses for therapeutics targeting elements of the complement cascade.

Glycyrrhiza uralensis Fisch. and its active components mitigate Semen Strychni-induced neurotoxicity through regulating high mobility group box 1 (HMGB1) translocation

Semen Strychni has long been used for the treatment of rheumatoid arthritis, facioplegia and myasthenia gravis due to its anti-inflammation and anti-nociceptive properties in China. However, the fatal neurotoxicity of Semen Strychni has limited its wider clinical application. To investigate the acute toxicity induced by Semen Strychni and the detoxification of liquorice, we evaluated inflammation, oxidative stress and the translocation of high mobility group box 1 (HMGB1) in rats. As a result, there were obvious oxidative stress and inflammation in hippocampus after the Semen Strychni extracts (STR) treatment in rats. Liquorice extracts (LE) and its three active monomers – glycyrrhizic acid (GA), liquiritigenin (LIQ), isoliquiritigenin (ISL) showed the potential for mitigating STR-induced neurotoxicity. HMGB1 levels in cytoplasm and serum and the levels of two downstream receptors RAGE and TLR4 were significantly increased after STR treatment. Through using LE and the monomers, the nucleocytoplasmic transport and release of HMGB1 were inhibited. In addition, the binding between HMGB1 and TLR4 was weakened in detoxification groups comparing with the STR group. Taken together, these findings indicated that liquorice and its active components alleviated acute neurotoxicity induced by Semen Strychni partly via HMGB1-related pathway.

Current Treatment of Myasthenia Gravis

Myasthenia gravis (MG) is the most extensively studied antibody-mediated disease in humans. Substantial progress has been made in the treatment of MG in the last century, resulting in a change of its natural course from a disease with poor prognosis with a high mortality rate in the early 20th century to a treatable condition with a large proportion of patients attaining very good disease control. This review summarizes the current treatment options for MG, including non-immunosuppressive and immunosuppressive treatments, as well as thymectomy and targeted immunomodulatory drugs.

Childhood-Onset Myasthenia Gravis Patients Benefited from Thymectomy in a Long-Term Follow-up Observation.

The effect of thymectomy on the treatment of childhood-onset myasthenia gravis (CMG) remains debatable. The objective of this study was to evaluate the clinical outcome and relevant prognostic factors of thymectomy for CMG patients. A total of 32 CMG patients who underwent thymectomy before 18 years of age were included in this retrospective study. Clinical state following thymectomy was assessed by quantified myasthenia gravis (QMG) scores, myasthenia gravis-related activities of daily living (MG-ADL) scores, and Myasthenia Gravis Foundation of America postintervention status. Repeated-measures analysis of variance (ANOVA) examined the changes in postoperative scores during the 5-year follow-up. Univariate logistic regression was applied to identify factors associated with short-term (1-year postoperation) and long-term (5-year postoperation) clinical outcomes. Repeated-measures ANOVA showed that QMG scores (F = 6.737, p < 0.001) and MG-ADL scores (F = 7.923, p < 0.001) decreased gradually with time. Preoperative duration (odds ratio [OR] = 0.85, 95% confidence interval [CI]: 0.73-1.00, p = 0.043), gender (OR = 0.19, 95% CI: 0.04-0.94, p = 0.041), and MG subgroup (OR = 13.33, 95% CI: 1.43-123.99, p = 0.023) were predictors for 1-year postoperative prognosis. Shorter disease duration (OR = 0.82, 95% CI: 0.70-0.97, p = 0.018) and generalized CMG (OR = 6.11, 95% CI: 1.06-35.35, p = 0.043) were found to have more favorable long-term results. Our results suggest that thymectomy is effective in treating CMG. Thymectomy could be recommended for CMG patients, especially for patients in the early course of GMG.

Integrated chemical molecular docking with network pharmacology to study the molecular mechanism of JianPi YiQi BuSui method for treating myasthenia gravis

Treatment of myasthenia gravis (MG), a chronic autoimmune neuromuscular disorder, remains challenging due to side effects associated with modern medications which was used to treat the disease. JianPi YiQi BuSui Method (JPYQBSM) is a traditional Chinese medicine (TCM) which has exhibited pivotal MG-alleviating therapeutic benefits. However, JPYQBSM active chemical components, molecular mechanisms and therapeutic targets have not yet been comprehensively investigated, which promptedthis study. The pharmacodynamic basis and action mechanism of JPYQBSM in the treatment of MG were predicted by computer network pharmacological technology and molecular docking technology of chemical composition. JPYQBSM alleviated MG through multi-chemical component, multitarget and multi-pathway synergistic therapeutic effects. This study provides new insights into specific mechanisms underlying JPYQBSM therapeutic effects for alleviating MG.

Characteristics, treatment, and outcomes of Myasthenia Gravis in COVID-19 patients: A systematic review

ObjectiveRecent studies suggest that the clinical course and outcomes of patients with coronavirus disease 2019 (COVID-19) and myasthenia gravis (MG) are highly variable. We performed a systematic review of the relevant literature with a key aim to assess the outcomes of invasive ventilation, mortality, and hospital length of stay (HLoS) for patients presenting with MG and COVID-19. MethodsWe searched the PubMed, Scopus, Web of Science, and MedRxiv databases for original articles that reported patients with MG and COVID-19. We included all clinical studies that reported MG in patients with confirmed COVID-19 cases via RT-PCR tests. We collected data on patient background characteristics, symptoms, time between MG and COVID-19 diagnosis, MG and COVID-19 treatments, HLoS, and mortality at last available follow-up. We reported summary statistics as counts and percentages or mean±SD. When necessary, inverse variance weighting was used to aggregate patient-level data and summary statistics. ResultsNineteen studies with 152 patients (mean age 54.4 ± 12.7 years; 79/152 [52.0%] female) were included. Hypertension (62/141, 44.0%) and diabetes (30/141, 21.3%) were the most common comorbidities. The mean time between the diagnosis of MG and COVID-19 was7.0 ± 6.3 years. Diagnosis of COVID-19 was confirmed in all patients via RT-PCR tests. Fever (40/59, 67.8%) and ptosis (9/55, 16.4%) were the most frequent COVID-19 and MG symptoms, respectively. Azithromycin and ceftriaxone were the most common COVID-19 treatments, while prednisone and intravenous immunoglobulin were the most common MG treatments. Invasive ventilation treatment was required for 25/59 (42.4%) of patients. The mean HLoS was 18.2 ± 9.9 days. The mortality rate was 18/152 (11.8%). ConclusionThis report provides an overview of the characteristics, treatment, and outcomes of MG in COVID-19 patients. Although COVID-19 may exaggerate the neurological symptoms and worsens the outcome in MG patients, we did not find enough evidence to support this notion. Further studies with larger numbers of patients with MG and COVID-19 are needed to better assess the clinical outcomes in these patients.

Treatment of myasthenia gravis with the method of tonifying spleen and replenishing qi in traditional Chinese medicine: A protocol for systematic review and meta-analysis.

Background:Myasthenia gravis (MG) is an autoimmune disease that is associated with the production of autoantibodies. The symptoms of MG are mainly mediated by pathogenic antibodies directed against nicotinic acetylcholine receptors, resulting in a decrease in the number of acetylcholine receptors molecules on the postsynaptic membrane of the neuromuscular junction endplate, leading to clinical symptoms of muscle fatigue and weakness. At present, traditional Chinese medicine (TCM) treatment of MG has a better effect by tonifying spleen and replenishing qi. The purpose of this study was to systematically evaluate the efficacy and safety of TCM therapy for tonifying spleen and replenishing qi in the treatment of myasthenia gravis.Methods:We searched the following databases from their establishment until December 2021: PubMed, Cochrane Library, EMBASE, Web of Science, Springer, CNKI, Wanfang, China Biomedical Database, China Science and Technology Journal Database, Chinese Knowledge Infrastructure, China Clinical Trial Registry, and Baidu Scholars. The literature search language was limited to Chinese and English, and publication time and status were not limited. Randomized controlled trials (RCTs) were included. Two researchers independently searched and screened the articles, assessed their quality, and used the RevMan 5.4.1 software to perform a meta-analysis of the included literature.Results:This study compared the main outcome indicators: efficacy rate, recurrence rate, quality of life, and quantitative myasthenia gravis (QMG). Secondary outcome indicators were the clinical absolute score, TCM syndrome score, serum acetylcholine receptor antibody level, and electromyogram low-frequency repetitive nerve stimulation.Conclusion:This study aimed to evaluate whether the TCM method of tonifying spleen and replenishing qi is effective in the treatment of MG and to provide evidence-based data.Ethics and dissemination:The protocol of the systematic review did not require ethical approval because it did not involve human subjects. This article will be published in peer-reviewed journals and will be presented at conferences.

Advancing research in autoimmune neuromuscular disorders

A new Series on autoimmune neuromuscular disorders in The Lancet Neurology comprises three Reviews 1 Huijbers MG Marx A Plomp JJ Le Panse R Phillips WD Advances in the understanding of disease mechanisms of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022; 21: 163-175 Google Scholar , 2 Verschuuren JJGM Palace J Murai H Tannemaat M Kaminski HJ Bril V Advances and ongoing research in the treatment of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022; 21: 189-202 Google Scholar , 3 Punga AR Maddison P Heckmann JM et al. Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022; 21: 176-188 Google Scholar that altogether provide a broad overview of the current status of, and ongoing research into, the causes and treatment of myasthenia gravis and Lambert-Eaton myasthenic syndrome. The major contribution of this Series is the comprehensive information that they provide about disease mechanisms, 1 Huijbers MG Marx A Plomp JJ Le Panse R Phillips WD Advances in the understanding of disease mechanisms of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022; 21: 163-175 Google Scholar new drugs in development, 2 Verschuuren JJGM Palace J Murai H Tannemaat M Kaminski HJ Bril V Advances and ongoing research in the treatment of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022; 21: 189-202 Google Scholar and known and potential biomarkers. 3 Punga AR Maddison P Heckmann JM et al. Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disorders. Lancet Neurol. 2022; 21: 176-188 Google Scholar Advances and ongoing research in the treatment of autoimmune neuromuscular junction disordersMyasthenia gravis and Lambert-Eaton myasthenic syndrome are antibody-mediated autoimmune diseases of the neuromuscular junction that usually present with weakness in ocular muscles and in proximal muscles of the limb and trunk. Prognosis regarding muscle strength, functional abilities, quality of life, and survival is generally good. However, some patients do not respond to treatment. Symptomatic drugs, corticosteroids, and steroid-sparing immunosuppressive drugs remain the cornerstone of treatment. Full-Text PDF Advances in the understanding of disease mechanisms of autoimmune neuromuscular junction disordersMuscle weakness and fatigue are the hallmarks of autoimmune neuromuscular junction disorders. Although a plethora of immunosuppressive treatments exist, no cure is available to date and many patients are left with debilitating muscle weakness. Recent advances in the understanding of the structure and function of the neuromuscular junction, and the development of novel in vitro and in vivo models, have been instrumental in unravelling the pathophysiology of these autoimmune diseases. These advances are providing the rationale for the development of new therapeutic strategies. Full-Text PDF Epidemiology, diagnostics, and biomarkers of autoimmune neuromuscular junction disordersAutoimmune neuromuscular junction disorders are rare. However, myasthenia gravis is being increasingly recognised in people older than 50 years. In the past 5–10 years, epidemiological studies worldwide suggest an incidence of acetylcholine receptor antibody-positive myasthenia gravis of up to 29 cases per 1 million people per year. Muscle-specific tyrosine kinase antibody-positive myasthenia gravis and Lambert-Eaton myasthenic syndrome are about 20 times less common. Several diagnostic methods are available for autoimmune neuromuscular junction disorders, including serological antibody, electrophysiological, imaging, and pharmacological tests. Full-Text PDF

Myasthenia gravis-treatment and severity in nationwide cohort.

Myasthenia gravis (MG) is a rare autoimmune disorder of neuromuscular junction. MG healthcare burden has not been studied in Poland before. Data were drawn from the National Health Fund database; MG patient was defined as a person who received at least once medical service with ICD-10 code MG (G70) and at least two reimbursed prescriptions for pyridostigmine bromide or ambenonium chloride in two consecutive years. We have analyzed treatment: immunosuppression, intravenous immunoglobulins (IVIg), plasma exchange (PE), the number and length of hospitalizations (LOS), intensive care unit (ICU) care, and deaths between 2013 and 2018. In 2018, there were 9012 MG patients (F:M 1.62:1), and 30.6% had early -onset MG (<50years). 66.3% received symptomatic treatment only, 33.7%-glucocorticoids (CS) and/or other immunosuppressants (IS), 64.6%-CS only, 17.5%-azathioprine plus CS, 11%-azathioprine only, 4.6%-CS plus other IS (methotrexate, mycophenolate mofetil, cyclosporine, or tacrolimus), and 2%-other IS only. In 2018, 42.3% of patients were hospitalized at least once (mean 2.05/year), 13.7% due to MG (1.47/year). In 2018, 1.63% patients received PE, 2.33% IVIg. In 2013-2018, 2.7%-3.2% of MG patients required hospitalization in ICU. ICU mean LOS 2013-2018 was 11.5-15.0days/per patient/year. 2.1% of all MG patients had myasthenic crisis. Mean age at death was 75.7years for MG and 73.9 for general population (p=.006). All-cause mortality was higher for men (4.1%-5.1%) than for women (2.5%-3.1%), p<.01. Our findings confirm significant healthcare burden of MG, comprising a tool to plan resources needed for MG patients.

Observation on the efficacy of different methylprednisolone regimens in the treatment of myasthenia gravis.

Objective:To investigate the efficacy of different methylprednisolone regimens in the treatment of myasthenia gravis (MG).Methods:A total of 98 patients with MG admitted to Shijiazhuang People’s Hospital from December 2018 to November 2019 were randomly divided into two groups, with 49 cases in each group. Patients in the control group received high-dose methylprednisolone pulse therapy, while those in the experimental group received medium-dose periodic therapy of methylprednisolone. Anti-acetylcholine receptor antibodies (AChRab), clinical absolute scores, complement levels (C3, C4), T lymphocyte subsets (CD3+, CD4+, CD4+CD25+), cytokines [interferon-γ (INF-γ), transforming growth factor-β1 (TGF-β1), interleukin-6 (IL- 6), interleukin-18 (IL-18)], and changes in quality of life [15-item Myasthenia Gravis Quality of Life Scale (MGQOL-15) score] were compared between the two groups before treatment and one and three months after treatment. Moreover, the incidence of adverse drug reactions in the two groups during 3 months of treatment was compared.Results:After three months of treatment, ACHRAB, clinical absolute score, CD3+, CD4+, INF-γ, IL-6, IL-18 and MGQOL-15 scores in both groups were significantly decreased compared with those before treatment (p<0.05), and the scores of C3, C4, CD4+CD25+ and TGF-β1 in both groups were significantly higher than those before treatment (p<0.05), and the experimental group had more significant changes than the control group (p<0.05). During three months of treatment, the total incidence of adverse drug reactions in the experimental group was significantly lower than that in the control group (p<0.05).Conclusions:The medium-dose periodic therapy of methylprednisolone is more prominent in the long-term efficacy performance. It can improve the immunity and quality of life of patients, and it is safer and has high clinical application value.

Factors affecting minimal manifestation status induction in myasthenia gravis.

Background:Minimal manifestation status (MMS) is an important landmark in the treatment of myasthenia gravis (MG), and predictors of MMS induction have rarely been identified in previous studies.Objective:The objective of this study is to evaluate the clinical factors associated with MMS induction among patients with MG.Design:This two-step retrospective cohort study with a single center investigated the factors that may be associated with MMS induction and retested these predictors in a test cohort.Methods:A total of 388 diagnosed MG patients who visited Xiangya Hospital between 1 July 2015 and 1 July 2019 were involved. We performed detailed chart reviews and recorded all cases achieving MMS. Demographics and clinical characteristics were also collected and their relationships to achieving MMS were investigated.Results:MMS was achieved in 124 patients (50.2%), and the median time to achieve MMS was 26 months. Several factors were found to be associated with MMS induction in exploring cohort, including muscle-specific tyrosine-protein kinase receptor (MuSK) antibody positivity (adjusted hazard ratio, HR = 4.333, 95% confidence interval, CI: 1.862–10.082), isolated ocular involvement (adjusted HR = 1.95, 95% CI: 1.284–2.961), and low baseline quantitative myasthenia gravis score (QMG score; adjusted HR = 2.022, 95% CI: 1.086–3.764). These factors were then retested in the test cohort. Isolated ocular involvement or low baseline QMG scores were factors found to be beneficial for MMS induction were confirmed.Conclusion:Isolated ocular involvement and low baseline QMG score are predictors of MMS induction in MG patients.