Myasthenia Gravis
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  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Long-Term Safety, Tolerability, And Efficacy of Efgartigimod in Patients with Generalized Myasthenia Gravis: Concluding Analyses from the ADAPT+ Study (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1580;    
    In all anti-AChR-Ab+ patients (n=111), consistent improvements in MG-ADL (mean[SE] change week 3 of cycle 1: -5.0[0.33]; up to 14 cycles) and QMG (-4.7[0.41]; up to 7 cycles) scores were observed during each cycle, mirroring repeatable reductions in total IgG (mean[SE] reduction: -55.9%[1.15]; up to 7 cycles) and anti-AChR autoantibody levels (-56.1%[1.43]; up to 7 cycles). Conclusions These analyses of ADAPT+ suggest long-term efgartigimod treatment is well-tolerated and results in consistent and repeatable reductions in IgG antibody levels and clinical outcomes (MG-ADL and QMG) in patients with gMG.
  • ||||||||||  Risk of Infection in Patients with Myasthenia Gravis With and Without Thymoma (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1437;    
    Rates of CMV, Zoster, UTI, and death were similar. Conclusions The rates of all non-chemotherapy related infections in patients with MG and thymoma are higher compared to MG patients without thymoma but are similar after adjusting for comorbidities and observable confounders.
  • ||||||||||  Effect of COVID-19 on Myasthenia Gravis (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1435;    
    Future, prospective studies could investigate CMAP as a biomarker of disease activity and response to treatment. Our study results revealed higher premorbid MGFA scores or use of immunosuppressant therapy did not alter a patient
  • ||||||||||  Iron Metabolism in Non-anemic Myasthenia Gravis Patients: A Cohort Study (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1433;    
    Transferrin < 2.00 g/L was an independent predictor for MMS at 12-month follow-up. Conclusions Iron metabolism parameters might be promising biomarkers for evaluating disease severity and guiding therapeutic decision in MG patients.
  • ||||||||||  Burden of Myasthenia Gravis Based on Sentiment Analysis of Patients (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1430;    
    Conclusions Digital conversations reveal a high degree of concern among patients with MG most specifically related to symptoms, life impact, misdiagnosis and treatments (for MG). Therapies that provide better symptom control could positively affect many aspects of patient
  • ||||||||||  Epidemiology of Antibody-positive Myasthenia Gravis in Sardinia, Italy (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1408;    
    Conclusions We report the highest incidence and prevalence of MG worldwide. The environmental and genetic determinants of MG risk in the Sardinian population need to be investigated.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Characterization of Early Adopters of Efgartigimod for gMG (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1275;    
    Regional distribution and primary practice setting did not differ between adopters and non-adopters, nor did reimbursement hassles as a perceived barrier to use (58%, 49%). Conclusions Analysis of efgartigamod early adopters suggests that the prescriber base is driven by specialists with a high volume of gMG patients, with comfort prescribing biologic therapies for gMG.
  • ||||||||||  Acquired Rippling Muscle Disease with Acetylcholine Receptor Antibodies (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1274;    
    Most cases of RMD/MG were heralded by RMD and may only exhibit minimal or no manifestations of MG despite AChR antibody positivity. IS therapies appear to be ineffective for RMD symptoms, although one patient had significant symptomatic benefit with IVIg.
  • ||||||||||  The impact of Myasthenia Gravis antibody status on pregnancy and neonatal outcomes (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1273;    
    Findings of transient neonatal myasthenia gravis and arthrogryposis in seronegative individuals suggests that another, currently unknown antibody or biological process may be present in the seronegative group. Future, larger studies are warranted.
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD), Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Efgartigimod for Pembrolizumab-induced Myasthenia Gravis Refractory to Standard Therapy (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1272;    
    Conclusions Current treatments for pembrolizumab-induced myasthenia gravis are corticosteroids, IVIG, and plasmapheresis. Rituximab is effective in refractory cases, although onset of action can take more than 6 weeks.2 Efgartigimod rapidly improved myasthenia gravis symptoms in both patients.
  • ||||||||||  prednisone / Generic mfg.
    Malignant Ectopic Cervical Thymoma in Myasthenia Gravis: A Case Report (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1271;    
    Therefore, the prednisone dose was lowered with no need for non-steroidal immunotherapies...Conclusions The diagnosis of ECT is challenging due to unusual locations, rare incidence, and inconclusive results of fine needle aspiration cytology (FNAC) that can lead to initial misinterpretation of a thymoma for thyroid or parathyroid lesions, as in our case. A comprehensive search and a timely diagnosis of thymomas for surgical resection are recommended.
  • ||||||||||  Post COVID-19 Myasthenia Gravis (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1238;    
    Uniquely, this case demonstrates waxing and waning symptomatology, potentially attributable to repeated exposure and/or subclinical infection with COVID-19. Further data will be helpful in delineating prognosis, risk factors for post COVID-19 development of myasthenia gravis, and potential strategies for effective management.
  • ||||||||||  nipocalimab (M281) / J&J
    Nipocalimab Dose Selection for A Phase 3 Study in Adult Patients with Generalized Myasthenia Gravis (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1237;    
    Conclusions A 30 mg/kg loading dose followed by 15 mg/kg Q2W maintenance dose was identified as optimal for the Ph3 gMG study. The predicted exposure in patients with gMG is well below the exposure from 60 mg/kg q2w dosing regimen in Ph2, which was generally safe and well-tolerated.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Efgartigimod Demonstrates Consistent Improvements in Generalized Myasthenia Gravis Patients of Shorter Disease Duration (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1234;    
    Adverse events were mostly mild-moderate and included headache, nasopharyngitis, nausea, diarrhea, and upper respiratory/urinary tract infection. Conclusions A greater percentage of patients early in their disease course (<3 years disease duration) treated with efgartigimod were responders and achieved MSE compared with placebo.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Use of Efgartigimod in Treatment of Myasthenia Gravis Relapse after Possible Recurrence of Invasive Thymoma: A Case Report. (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1230;    
    Conclusions Our patient with AChR antibody-positive, thymoma-associated, generalized MG with recurrent symptoms despite mycophenolate treatment, thymectomy and radiotherapy showed a dramatic response to initial cycles of efgartigimod therapy without significant adverse effects. We propose that efgartigimod be considered early in the management of MG patients with recurrent symptoms after thymectomy.
  • ||||||||||  Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
    A Rare Case of Thymoma-Associated Idiopathic Inflammatory Myopathy and Myasthenia Gravis (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1228;    
    Prednisone, pyridostigmine, and IVIG previously provided no relief...He has since been maintained on rituximab...Conclusions When presented with a patient with a history of recurrent thymoma and weakness of unclear etiology, MG-IM overlap syndrome should be considered in the differential diagnosis. Further study into the mechanism and appropriate management of this autoimmune dysregulation syndrome should be undertaken.
  • ||||||||||  Investigating Autoantibody Profiles in Seronegative Myasthenia Gravis (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1224;    
    Conclusions Findings from a large SNMG cohort support the clinical need to implement clustered AChR CBA testing in the evaluation of SNMG patients. Further ongoing investigations are warranted to confirm the presence of novel autoantibodies in SNMG.
  • ||||||||||  Ultomiris IV (ravulizumab IV) / AstraZeneca
    Ravulizumab for the Treatment of Generalized Myasthenia Gravis: Timing of Response (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1223;    
    P3
    Conclusions Cumulative response rates indicate that first response was achieved by almost half of patients after one ravulizumab infusion (by Week 2) and by two-thirds of patients by Week 12; however, some patients (15.8%) achieved a first response after longer treatment. Median time to first response to ravulizumab treatment in patients with AChR
  • ||||||||||  azathioprine / Generic mfg.
    Comorbidities in patients with myasthenia gravis in the USA: a retrospective claims database analysis (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1222;    
    Similarly, 6.5% of patients prescribed chronic intravenous immunoglobulin and 14.3% of patients prescribed azathioprine were diagnosed with venous thromboembolism and malignancy, respectively, within 1 year prior to treatment initiation. Conclusions This study demonstrates the high rates of comorbidities in patients with MG and the importance of considering the potential impact of these comorbidities in treatment choice discussions.
  • ||||||||||  CD8 T Cell Immune Signatures in Autoimmune Myasthenia Gravis (Both in-person and online) -  Mar 12, 2023 - Abstract #AAN2023AAN_1085;    
    Steroid treatment reduces the CD8 T cell proinflammatory phenotype. This data suggests CD8 T cells contribute to MG pathogenesis and may potentially serve as a biomarker for monitoring response to treatment.
  • ||||||||||  Review, Journal:  Airway clearance in patients with neuromuscular disease. (Pubmed Central) -  Mar 10, 2023   
    Neuromuscular disease is an umbrella term used to describe disorders that involve dysfunction of peripheral nerves, the neuromuscular junction, or skeletal muscle. Although this paper specifically reviews airway clearance pertaining to those with neuromuscular diseases (e.g., muscular dystrophy, spinal muscular atrophy, myasthenia gravis), most of its content is relevant to the management of patients with central nervous system disorders such as chronic static encephalopathy caused by trauma, metabolic or genetic abnormalities, congenital infection, or neonatal hypoxic-ischemic injury.
  • ||||||||||  tolebrutinib (SAR442168) / Sanofi
    Trial completion date, Trial termination, Trial primary completion date:  URSA: Efficacy and Safety of Tolebrutinib (SAR442168) Tablets in Adult Participants With Generalized Myasthenia Gravis (clinicaltrials.gov) -  Mar 10, 2023   
    P3,  N=6, Terminated, 
    Although this paper specifically reviews airway clearance pertaining to those with neuromuscular diseases (e.g., muscular dystrophy, spinal muscular atrophy, myasthenia gravis), most of its content is relevant to the management of patients with central nervous system disorders such as chronic static encephalopathy caused by trauma, metabolic or genetic abnormalities, congenital infection, or neonatal hypoxic-ischemic injury. Trial completion date: Dec 2026 --> Feb 2023 | Active, not recruiting --> Terminated | Trial primary completion date: Nov 2024 --> Feb 2023; For strategic reasons the Tolebrutinib MG study has been terminated