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Follicular helper (Tfh), peripheral helper (Tph), and regulatory (Treg) T cells are involved in myasthenia gravis (MG) pathogenesis, an autoimmune disorder arising from autoantibodies targeting neuromuscular junction proteins. This study explores the impact of low-dose IL-2 on Tfh, Tph, and Treg cells in vitro in MG. Acetylcholine-receptor antibody-positive MG (AChR-MG), muscle-specific kinase antibody-positive MG (MuSK-MG) patients, and healthy controls (HC) were studied. Blood cells were cultured with/without IL-2 and compared by the ratios of IL-2 stimulated/unstimulated cultures. In both AChR-MG and MuSK-MG patients, CD25+FoxP3+Tregs were lower, while CXCR5+PD-1+ or ICOS+Tfh and CXCR5-PD-1+ or ICOS+Tph cells were higher compared with HC. Among the MG group, the FoxP3+ Treg cells in AChR-MG patients were even lower compared with MuSK-MG patients. In vitro IL-2 stimulation increased Tregs in all groups while decreasing PD-1+/ICOS+Tfh and PD-1+/ICOS+Tph populations. The fold-increase ratio of Tregs and the fold-decrease ratio of PD-1+ or ICOS+Tfh and ICOS+Tph cells in AChR-MG and MuSK-MG patients were greater than in HCs. Low-dose IL-2 treatment may balance Tfh, Tph, and Treg cells in MG patients, offering a potential opportunity for disease modulation.

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Background: Muscle-specific tyrosine kinase (MuSK) autoantibody-positive (Ab+) generalised myasthenia gravis (gMG) is a rare and frequently severe subtype of gMG. Objectives: To assess the efficacy and safety of rozanolixizumab in the subgroup of patients with MuSK Ab+ gMG in the MycarinG study. Design: A randomised, double-blind, placebo-controlled phase III study. Methods: Patients with acetylcholine receptor (AChR) Ab+ or MuSK Ab+ gMG (aged ⩾18 years, Myasthenia Gravis Foundation of America Disease Class II-IVa, Myasthenia Gravis Activities of Daily Living [MG-|ADL] score ⩾3.0 [non-ocular symptoms], Quantitative Myasthenia Gravis score ⩾11.0) were randomly assigned (1:1:1) to receive once-weekly subcutaneous infusions of rozanolixizumab 7 mg/kg, rozanolixizumab 10 mg/kg or placebo for 6 weeks, followed by an 8-week observation period. Randomisation was stratified by AChR and MuSK autoantibody status. The primary study endpoint was change from baseline to Day 43 in MG-ADL score. Treatment-emergent adverse events (TEAEs) were also assessed. Results: Overall, 200 patients were randomised, of whom 21 had MuSK Ab+ gMG and received rozanolixizumab 7 mg/kg (n = 5), 10 mg/kg (n = 8) or placebo (n = 8). In patients with MuSK Ab+ gMG, reductions from baseline to Day 43 in MG-ADL scores were observed: rozanolixizumab 7 mg/kg least squares mean (LSM) change (standard error), -7.28 (1.94); 10 mg/kg, -4.16 (1.78); and placebo, 2.28 (1.95). Rozanolixizumab 7 mg/kg LSM difference from placebo was -9.56 (97.5% confidence interval: -15.25, -3.87); 10 mg/kg, -6.45 (-11.03, -1.86). TEAEs were experienced by four (80.0%), five (62.5%) and three (37.5%) patients with MuSK Ab+ gMG receiving rozanolixizumab 7 mg/kg, 10 mg/kg and placebo, respectively. No patients experienced serious TEAEs. No deaths occurred. Conclusion: This subgroup analysis of adult patients with MuSK Ab+ gMG enrolled in the MycarinG study supports the use of rozanolixizumab as an effective treatment option for patients with gMG who have MuSK autoantibodies. Trial registration: ClinicalTrials.gov: NCT03971422 (https://clinicaltrials.gov/study/NCT03971422); EU Clinical Trials Register: EudraCT 2019-000968-18 (https://www.clinicaltrials|register.eu/ctr-search/trial/2019-000968-18/GB).

Rozanolixizumab improved symptoms in people with anti-muscle-specific tyrosine kinase antibody-positive generalised myasthenia gravis in the MycarinG clinical study Myasthenia gravis is a rare, chronic autoimmune disease affecting the communication between nerves and muscles. People with the disease experience fluctuating muscle weakness and fatigue, leading to problems with mobility, speaking, swallowing and breathing. The disease is called generalised when muscles other than those that move the eyes and eyelids are affected. It is caused by antibodies that attack a person's own cells. Most people with the disease have antibodies against acetylcholine receptors (AChRs). However, some have antibodies against the muscle-specific tyrosine kinase (MuSK) protein and can experience more severe symptoms compared with people who have anti-AChR antibodies. Standard treatments for myasthenia gravis do not always work for people with anti-MuSK antibodies. The MycarinG study looked at whether rozanolixizumab was better than a placebo at treating the symptoms of adults with generalised myasthenia gravis and anti-AChR or anti-MuSK antibodies. Assessments measured disease severity and myasthenia gravis symptoms, such as physical fatigue, and how they affected daily activities. The study also looked at whether people receiving rozanolixizumab had any side effects. Here, we look at the group of people with anti-MuSK antibodies who took part in the MycarinG study. In total, 21 of the 200 people in the study had anti-MuSK antibodies. The symptoms of myasthenia gravis improved more in people with anti-MuSK antibodies who received rozanolixizumab than in those who received placebo. Common side effects with rozanolixizumab included headache, diarrhoea and feeling sick. No serious side effects were seen, and no patients died. The results show that rozanolixizumab is an effective treatment for people with generalised myasthenia gravis who have anti-MuSK antibodies. The results in this group of people are consistent with those seen in all people who took part in the study (with either antibody type).

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The THαß host immunological pathway contributes to the response to infectious particles (viruses and prions). Furthermore, there is increasing evidence for associations between autoimmune diseases, and particularly type 2 hypersensitivity disorders, and the THαß immune response. For example, patients with systemic lupus erythematosus often produce anti-double stranded DNA antibodies and anti-nuclear antibodies and show elevated levels of type 1 interferons, type 3 interferons, interleukin-10, IgG1, and IgA1 throughout the disease course. These cytokines and antibody isotypes are associated with the THαß host immunological pathway. Similarly, the type 2 hypersensitivity disorders myasthenia gravis, Graves' disease, graft-versus-host disease, autoimmune hemolytic anemia, immune thrombocytopenia, dermatomyositis, and Sjögren's syndrome have also been linked to the THαß pathway. Considering the potential associations between these diseases and dysregulated THαß immune responses, therapeutic strategies such as anti-interleukin-10 or anti-interferon α/ß could be explored for effective management.

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INTRODUCTION: Myasthenia gravis (MG) is an autoimmune disorder. Microvesicle-derived miRNAs have been implicated in autoimmune diseases. However, the role of microvesicle-derived miR-29a-3p in MG remains poorly understood. This study aimed to investigate the therapeutic effect and mechanism of miR-29a-3p derived from stem cell microvesicles (MVs) on experimental autoimmune myasthenia gravis (EAMG) rats. METHODS: EAMG was induced in rats by injection of the subunit of the rat nicotinic anti-acetylcholine receptor (AChR) R97-116 peptide.Besides the control group, EAMG rats were randomly allocated into the EAMG model group, MV group, MV-NC-agomir group, and MV- miR-29a-3p-agomir group. RESULTS: Our results found that BMSCs-MV promoted miR-29a-3p expression in gastrocnemius of EAMG rats. Bone marrow mesenchymal stem cells (BMSCs) derived microvesicle miR-29a-3p improved the hanging ability and swimming time of EMGA rats and weakened the degree of muscle fiber atrophy. Furthermore, microvesicles from miR-29a-3p overexpressing BMSCs reduced the content of AchR-Ab in the serum of EAMG rats. BMSC-derived microvesicle miR-29a-3p further suppressed the expression of IFN-γ and enhanced the IL-4 and IL-10 in the serum of EAMG rats by restoring the Th17/Treg cells balance. DISCUSSION: BMSCs-derived microvesicle miR-29a-3p improved the stability of rat myasthenia gravis by regulating Treg/Th17 cells. It may be an effective treatment for MG.

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Introduction: In this study, we aimed to investigate the clinical effects of COVID-19 infection and vaccines on Myasthenia gravis (MG) during the pandemic. Methods: A total of 141 MG patients between April 2020 and December 2021 were retrospectively analyzed. Data including demographic and clinical characteristics of patients, COVID-19 test results, and vaccine types (mRNA-BNT162b2 and/or inactivated-CoronaVac) were recorded. All patients were followed by face-to-face interviews and/or phone calls. Worsening MG symptoms after COVID-19 infection or vaccines were noted. Results: A total of 60 patients were diagnosed with COVID-19, and reverse transcriptase-polymerase chain reaction test results were COVID-19 positive in 54 (90%) patients. Twenty-eight (46.7%) patients had lung involvement, while 20(33.3%) patients were followed in the ward. Twelve (20%) patients were followed in the intensive care unit, and two of them (3.3%) died. Both deceased patients were unvaccinated. The most common symptoms were fatigue (78.3%), and 13(21.7%) patients were asymptomatic. Of the patients, 96(68%) received at least one dose BNT162b2 or CoronaVac, while 30.4% of the patients received ≥3 doses of vaccines. The local skin irritation and fatigue rate was significantly higher with BNT162b2 vaccine than CoronaVac (p<0.001 and p=0.004, respectively). No serious side effect was observed with either vaccine. Five patients had worsening MG symptoms after vaccination during a six-week follow-up. None of the patients experienced myasthenic crises. Conclusion: Our study results suggest that COVID-19 infection affects MG similar to the general population and does not lead to worsening MG symptoms. Both mRNA and inactivated vaccines with proven efficacy can be used safely in MG patients.

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This article deals with peripheral neuroimmunological diseases and briefly outlines the currently most important aspects and treatment developments. Idiopathic inflammatory myopathies have different mechanisms of development, manifestations and prognoses. New classification systems and more specific treatment concepts have been developed. The IIMs include different subgroups. These entities can have specific autoantibodies. Diagnostically, a muscle biopsy is generally desirable for a precise diagnosis and is essential in unclear cases. Primary systemic vasculitides can be divided into different groups based on the predominant pattern of involvement, while secondary vasculitides and single organ vasculitides are also differentiated. Vasculitic myopathy cannot be equated with myositis and a reliable distinction is currently only possible by a muscle biopsy. Treatment concepts should be developed on an interdisciplinary basis. Chronic inflammatory demyelinating polyneuropathy is the most frequent immune-mediated neuropathy and is characterized by a predominant demyelination of the motor and sensory nerves. The disease course runs in phases or is progressive and leads to significant disability and reduction in quality of life, despite current standard treatment. Novel treatment approaches are currently undergoing clinical trials. Myasthenia gravis, with the leading symptom of exercise-induced muscle weakness, is caused by autoantibodies against structures of the neuromuscular endplate. Autoantibody testing is the most important pillar in the diagnosis and is now also increasingly guiding treatment decisions. Overall, peripheral neuroimmunological diseases represent a heterogeneous group. Increasing knowledge of the pathophysiology is the key to numerous developments in diagnostics and treatment, which could lead to far-reaching practical changes in the future.

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Ocular myasthenia gravis (OMG) is a neuromuscular disease characterized by the production of autoantibodies against post-synaptic proteins at the neuromuscular junction (NMJ). An 18-year-old male who had symptoms of drooping eyelids and double vision was diagnosed with ocular myasthenia gravis on investigations and examinations. Treatment was initiated with a tablet of pyridostigmine 60 mg twice daily per oral for two weeks, followed by three times daily for four weeks. The patient demonstrated significant improvement in ptosis and diplopia. There are still a considerable number of challenges in the diagnosis and treatment of ocular myasthenia gravis, with the typical treatment involving acetylcholinesterase inhibitors and immunosuppressants.

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Background: Dyslipidemia has been implicated in autoimmunity; however, its association with myasthenia gravis (MG) prognosis is unclear. We aimed to investigate the correlation between baseline lipid profiles and risk of MG worsening. Methods: This 7-year retrospective cohort study conducted at a Chinese hospital included 264 adult patients with MG. Data on baseline lipids, 1-year worsening, and covariates, including demographics, MG characteristics, comorbidities, and treatments were extracted. Results: Univariate and multivariate logistic regression analyses failed to show a significant association between the risk of 1-year MG worsening and any of the seven blood lipid-related indicators. However, the subsequent non-linear analysis revealed an inflection point in the risk curve of ln[lipoprotein(a)], at 4.06 (58 nmol/L). The lipoprotein(a) levels on the left side of the inflection point presented a positive significant correlation with the risk of MG worsening (relative risk [RR]: 6.06, 95 % confidence interval [CI]: 1.00-38.57), whereas those on the right side of the inflection point demonstrated no significant correlation (RR: 0.86, 95 % CI: 0.55-1.34). Conclusions: Except for lipoprotein(a) levels being associated with worsening of myasthenia gravis, most lipid parameters were not associated with changes in the clinical course and severity of myasthenia gravis.we observed that lower levels of lipoprotein(a) were associated with a better prognosis in the interval 7-58 nml/L, whereas beyond this interval this was not observed, suggesting dyslipidemia may impact MG prognosis. Further studies are required to validate these findings.

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This editorial aimed to consolidate the current evidence in literature on the association between myasthenia gravis (MG) and cardiac involvement, focusing on the impact of thymoma, antistriational antibodies, and late-onset MG. Additionally, the study aimed to explore the influence of genetic differences among populations on the association with cardiac disease. We conducted a review of existing literature in PubMed and Google Scholar to find relevant studies on cardiac involvement in MG. We created search criteria using a combination of free text words, including MG, antistriational antibodies, thymectomy, cardiomyopathy, myocarditis, arrhythmias, autonomic dysfunction. Relevant articles published in English language were analyzed and incorporated. The findings indicate a strong association between thymoma, myasthenic crisis, antistriational antibodies, and late-onset MG with cardiac involvement. The study also revealed that genetic differences among populations influence the risk of cardiac disease and electrocardiography (ECG) abnormalities in MG patients. Autonomic dysfunctions altered cardiac autonomic response and increased susceptibility to arrhythmias and sudden cardiac death in MG patients. The study supports the significance of thymoma, antistriational antibodies, and late-onset MG as key factors associated with cardiac involvement in MG patients. It emphasizes the importance of ECG as the initial test in managing MG patients, particularly in the perioperative period, to identify and genetic testing if needed to address their cardiac risk effectively.

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Myasthenia gravis (MG) is a chronic and severe disease of the skeletal neuromuscular junction (NMJ) in which the effects of neurotransmitters are attenuated, leading to muscle weakness. In the most common forms of autoimmune MG, antibodies attack components of the postsynaptic membrane, including the acetylcholine receptor (AChR) or muscle-specific kinase (MuSK). MuSK, a master regulator of NMJ development, associates with the low-density lipoprotein-related receptor 4 (Lrp4) to form the signaling receptor for neuronal Agrin, a nerve-derived synaptic organizer. Pathogenic antibodies to MuSK interfere with binding between MuSK and Lrp4, inhibiting the differentiation and maintenance of the NMJ. MuSK MG can be debilitating and refractory to treatments that are effective for AChR MG. We show here that recombinant antibodies, derived from MuSK MG patients, cause severe neuromuscular disease in mice. The disease can be prevented by a MuSK agonist antibody, presented either prophylactically or after disease onset. These findings suggest a therapeutic alternative to generalized immunosuppression for treating MuSK MG by selectively and directly targeting the disease mechanism.

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There is a need to improve therapies in autoimmune neurologic conditions. Yet which strategic objectives are required, what are the barriers that stand before reaching them, and what are the options to address them? This article tries to summarize these objectives and their respective barriers. It discusses the difficulties in identifying molecular targets, biomarker-defined subgroups, the merits of upstream and downstream-targeted therapies, the need to develop autoreactivity-specific treatments in contrast to cell-type specific therapies, and the "evidence-bottleneck". Its focus is on autoantigen-specific autoimmunopathies in neurology. It also discusses the role of B- and T-cells in autoimmune neurology and how these can be exploited therapeutically. Finally, it argues for improved training of present and future neuroimmunologists.

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The treatment strategy of patients with locally advanced gastric cancer has undergone notable changes since immune checkpoint inhibitors (ICIs) were developed. Although ICIs are generally well-tolerated, they can also cause serious adverse events, such as autoimmune diseases. In patients with gastric cancer and without a history of immune disease, the incidence of myasthenia gravis combined with myocarditis caused by ICI treatment is rare. Furthermore, cases of gastric cancer with ocular myasthenia gravis, without limb weakness or severe dyspnea, although with urination difficulties and symptoms of third-degree atrioventricular block have not been previously reported, to the best of our knowledge. The present study describes the case of a 72-year-old male patient with locally advanced gastric cancer that was treated with chemoimmunotherapy with oxaliplatin + tigio + sintilimab. At 19 days following only one cycle of therapy, the patient developed a left eyelid weakness and difficulty in urinating, as well as diplopia. At 5 days after the symptom of eyelid weakness, a third-degree atrioventricular block occurred. Hormone therapy, a temporary pacemaker and gamma-globulin therapy were administered, and the patient was discharged 1 month later with the resolution of myasthenia gravis and the atrioventricular block. At the final follow-up (1 month after discharge), the patient had a full recovery from myasthenia gravis and arrhythmias. Although some similar cases have been previously reported, the majority of patients with limb weakness and have eventually succumbed; moreover, clinical symptoms were identified at a late stage, and the disease evolution records were not detailed. Therefore, the present study describes the case of the patient and treatment strategy, also providing detailed laboratory indicators and clinical symptom evolution. This was performed with the aim to aid future research and the treatment of immune-related diseases.

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Background Myasthenia gravis (MG) is a rare, autoantibody neuromuscular disorder characterized by fatigable weakness. Real-world evidence based on administrative and structured datasets regarding MG may miss important details related to the clinical encounter. Examination of free-text clinical progress notes has the potential to illuminate aspects of MG care. Objective The primary objective was to examine and characterize neurologist progress notes in the care of individuals with MG regarding the prevalence of documentation of clinical subtypes, antibody status, symptomatology, and MG deteriorations, including exacerbations and crises. The secondary objectives were to categorize MG deteriorations into practical, objective states as well as examine potential sources of clinical inertia in MG care. Methods We performed a retrospective, cross-sectional analysis of de-identified neurologist clinical notes from 2017 to 2022. A qualitative analysis of physician descriptions of MG deteriorations and a discussion of risks in MG care (risk for adverse effects, risk for clinical decompensation, etc.) was performed. Results Of the 3,085 individuals with MG, clinical subtypes and antibody status identified included gMG (n = 400; 13.0%), ocular MG (n = 253; 8.2%), MG unspecified (2,432; 78.8%), seropositivity for acetylcholine receptor antibody (n = 441; 14.3%), and MuSK antibody (n = 29; 0.9%). The most common gMG manifestations were dysphagia (n = 712; 23.0%), dyspnea (n = 626; 20.3%), and dysarthria (n = 514; 16.7%). In MG crisis patients, documentation of difficulties with MG standard therapies was common (n = 62; 45.2%). The qualitative analysis of MG deterioration types includes symptom fluctuation, symptom worsening with treatment intensification, MG deterioration with rescue therapy, and MG crisis. Qualitative analysis of MG-related risks included the toxicity of new therapies and concern for worsening MG because of changing therapies. Conclusions This study of neurologist progress notes demonstrates the potential for real-world evidence generation in the care of individuals with MG. MG patients suffer fluctuating symptomatology and a spectrum of clinical deteriorations. Adverse effects of MG therapies are common, highlighting the need for effective, less toxic treatments.

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Myasthenia gravis (MG) is an autoimmune disease mediated by B cells and is associated with acetylcholine receptor (AChR) and muscle-specific receptor tyrosine kinase (MuSK) antibodies in the postsynaptic membrane at the neuromuscular junction. Anti-CD20 monoclonal antibodies, such as ofatumumab demonstrated promising disease control in MG patients. We presented the rare case of a 34-year-old female with acetylcholine receptor-positive myasthenia gravis (AChR-MG), concomitant with systemic lupus erythematosus (SLE) and metastatic thyroid carcinoma, who was treated with ofatumumab and exhibited improvements during follow-up.

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Myasthenia gravis is an autoimmune disease characterized by muscle weakness and pathological fatigue due to autoaggressive phenomena with the formation of antibodies directed against various structures of the neuromuscular synapse. In most patients, the disease begins with the involvement of extraocular muscles, presenting with symptoms such as intermittent ptosis of the upper eyelid and/or binocular diplopia. In 15% of cases, clinical manifestations are limited to impairment of the levator palpebrae superioris and extraocular muscles, characteristic of the ocular form of myasthenia gravis. Specialists often encounter challenges in diagnosing this form, as serological and electrophysiological studies may be uninformative, necessitating diagnosis based on patient history and clinical picture. This literature review outlines the key aspects of the pathogenesis, clinical manifestations, methods of diagnosis and treatment of ocular myasthenia gravis.

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The complement system is a set of over 50 proteins that constitutes an essential part of the innate immune system. Complement system activation involves an organized proteolytic cascade. Overactivation of complement system activation is the main pathogenic mechanism of several diseases and contributes to the manifestations of many other conditions. This review describes the normal complement system and the role for complement dysregulation in critical illnesses, notably sepsis and acute respiratory distress syndrome. Complement activation is involved in the immune system response to pathogens but, when excessive, can contribute to tissue damage, runaway inflammation, and capillary leakage syndrome. Complement overactivation may play a key role in severe forms of coronavirus disease 2019 (COVID-19). Two diseases whose manifestations are mainly caused by complement overactivation, namely, atypical hemolytic and uremic syndrome (aHUS) and myasthenia gravis, are discussed. A diagnostic algorithm for aHUS is provided. Early complement-inhibiting therapy has been proven effective. When renal transplantation is required, complement-inhibiting drugs can be used prophylactically to prevent aHUS recurrence. Similarly, acetylcholine-receptor autoantibody-positive generalized myasthenia gravis involves complement system overactivation and responds to complement inhibition. The two main complement inhibitors used in to date routine are eculizumab and ravulizumab. The main adverse event is Neisseria infection, which is rare and preventable, but can be fatal. The complement system is crucial to health but, when overactivated, can cause or contribute to disease. Effective complement inhibitors are now available, although additional data are required to determine optimal regimens. Further research is also needed to better understand the complement system, develop advanced diagnostic tools, and identify markers that allow the personalization of treatment strategies.

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Objective: This study assesses the utility of jitter analysis with concentric needles to evaluate disease severity in myasthenia gravis (MG), correlate changes in jitter with clinical status as well as identify reasons for any discordance. Methods: We performed a retrospective chart review of 82 MG patients and extracted data on demographics, MG subtype, antibody status, clinical scales, electrophysiology, and interventions at baseline and follow-up. Results: Baseline MGII scores correlated with jitter (r = 0.25, p = 0.024) and abnormal pairs (r = 0.24, p = 0.03). After 28 months, MGII scores correlated with jitter (r = 0.31, p = 0.006), abnormal pairs (r = 0.29, p = 0.009), and pairs with blocks (r = 0.35, p = 0.001). Changes in MGII scores correlated with changes in jitter (r = 0.35, p = 0.002), abnormal pairs (r = 0.27, p = 0.014), and pairs with blocks (r = 0.36, p = 0.001). Conclusions: Concentric needle jitter analysis may have the potential to evaluate baseline and sequential disease severity in MG. Significance: This study highlights the potential for improved MG patient care through precise assessment and management using concentric needle jitter analysis to improve the accuracy of MG diagnosis and monitoring of disease activity.

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Purpose: To investigate the predictors for poor outcomes (including disease exacerbation, hospitalization and myasthenic crisis) in patients with pre-existing myasthenia gravis (MG) following Coronavirus disease 2019 (COVID-19), and to explore the potential effects of COVID-19 on inflammatory and immune responses in MG patients. Patients and Methods: This retrospective cohort study analyzed medical records of 845 MG patients who were diagnosed with COVID-19 between January 2020 to March 2023 at a single medical center. Results: Generalized MG at onset and comorbidities (chronic kidney disease and malignancy) were independent risk factors of poor outcomes. Patients achieving minimal manifestation or better status before COVID-19 had a significantly reduced risk for poor outcomes. Furthermore, patients with older onset age or anti-acetylcholine receptor antibody had a higher risk of exacerbation and hospitalization than those without. Prednisone or immunosuppressant treatment had the potential to reduce the occurrence of poor outcomes, while the duration of prednisone or immunosuppressant usage was associated with a higher risk of poor outcomes. Of the 376 MG patients with blood results available, patients with COVID-19 tended to have higher levels of leukocyte counts, neutrophil-lymphocyte-ratio, hypersensitive C-reactive protein, and Interleukin-6, as well as lower percentages of lymphocytes and regulatory T cells compared to patients without COVID-19. Conclusion: Disease severity at onset, comorbidities, and unsatisfactory control of myasthenic symptoms predicted the occurrence of poor outcomes in MG patients following COVID-19. The risk of poor outcomes was reduced in patients controlled by short-term immunosuppressive therapy. Novel coronavirus might affect inflammatory and immune responses in MG patients, particularly in altering interleukin-6 and regulatory T cell levels.

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ADAPT-SC (NCT04735432) was designed to evaluate noninferiority of subcutaneous (SC) efgartigimod PH20 to intravenous (IV) efgartigimod in participants with generalized myasthenia gravis (gMG). ADAPT-SC+ (NCT04818671) is an open-label extension study designed to assess long-term safety, tolerability, and efficacy of efgartigimod PH20 SC. Adult participants in ADAPT-SC were randomly assigned to receive a treatment cycle of 4 once-weekly administrations of efgartigimod PH20 SC 1000 â€‹mg or efgartigimod IV 10 â€‹mg/kg, followed by 7 weeks of follow-up. Primary endpoint was percentage change from baseline in total immunoglobulin G (IgG) level at week 4 (1 week after the fourth administration). Secondary efficacy endpoints assessed number and percentage of Myasthenia Gravis Activities of Daily Living (MG-ADL) and Quantitative Myasthenia Gravis (QMG) responders and mean change from baseline in total score for each measure. The primary endpoint was met, demonstrating noninferiority in total IgG reduction between efgartigimod PH20 SC 1000 â€‹mg and efgartigimod IV 10 â€‹mg/kg. Clinically meaningful improvements were seen as early as 1 week following the first administration in both treatment arms, with maximal improvements at week 4. Continued treatment cycles of efgartigimod PH20 SC in ADAPT-SC+ have demonstrated long-term safety and consistent improvements in MG-ADL total score. Findings from ADAPT-SC and ADAPT-SC+ demonstrate similar safety and efficacy as observed in the placebo-controlled ADAPT study. Collectively, these findings support noninferiority between efgartigimod PH20 SC 1000 â€‹mg and efgartigimod IV 10 â€‹mg/kg, as well as long-term safety, tolerability, and efficacy of efgartigimod PH20 SC for treatment of a broad population of patients with gMG.

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BACKGROUND: We investigated the proper timing, efficacy and safety of tacrolimus for juvenile myasthenia gravis (JMG). METHODS: We conducted a retrospective cohort study for JMG patients treated with tacrolimus at Xiangya Hospital, Central South University, Changsha, China from 2010 to 2023. The clinical information of patients with a follow-up of more than 1 year was collected. Comparisons of clinical features between groups of patients who achieved therapeutic goal and those who did not achieve therapeutic goal as well as between groups of patients treated with tacrolimus within or after 1 year from JMG onset was carried out. RESULTS: Forty-three patients were enrolled, of whom 28 achieved therapeutic goal. Tacrolimus reduced glucocorticoids (GC) dosages for the 28 cases and 15 cases discontinued GC completely. Generalized myasthenia gravis (GMG) subtype had an association with a group of patients who achieved therapeutic goal (p = 0.001). Median duration from JMG onset to tacrolimus use was 10.50 months for those who achieved therapeutic goal and 36.00 months for those who did not achieve therapeutic goal (p = 0.010). The median Myasthenia Gravis Activities of Daily Living (MG-ADL) score improved significantly (p = 0.003). The initiation of tacrolimus within 1 year of JMG onset showed an association with achievement of therapeutic goal (p = 0.026). GMG subtype showed an association with a group of patients who received tacrolimus within 1 year (p = <0.001). Tacrolimus side effects were tolerable. CONCLUSION: The provision of tacrolimus within 1 year of JMG onset is effective and safe.