Myasthenia Gravis
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  • ||||||||||  Journal:  Perianesthetic Implications and Considerations for Myasthenia Gravis. (Pubmed Central) -  May 1, 2020   
    This patient population is at high risk for respiratory failure, and therefore must be carefully assessed throughout the perioperative period to ensure that a regular spontaneous respiratory pattern is sufficient to provide adequate oxygenation. Perianesthesia providers must consider anesthetic, ventilatory, and pharmacologic implications when proposing, providing, and recovering anesthesia for the patient with MG.
  • ||||||||||  Clinical, Review, Journal, Checkpoint inhibition:  Clinical spectrum of neuromuscular complications after immune checkpoint inhibition. (Pubmed Central) -  May 1, 2020   
    Poor outcomes were more common with exacerbations of pre-existing myasthenia gravis and myocarditis overlap. Future prospective studies are needed to elucidate mechanisms and risk factors for autoimmune adverse events following immune checkpoint blockade.
  • ||||||||||  Clinical, Review, Journal:  The clinical management of neuromuscular disorders in intensive care. (Pubmed Central) -  May 1, 2020   
    They need to acquire a very specific set of skills, including both a thorough understanding of the mechanics of ventilation as well as familiarity with the diagnostic categories of genetic and of autoimmune diseases. This review of the clinical management of adult neuromuscular disease in the ICU aims to provide an overview of the most important conditions encountered in the ICU and a practical approach to their diagnosis, monitoring, and treatment.
  • ||||||||||  Firdapse (amifampridine) / BioMarin, Catalyst Pharma, Dydo Pharma
    Trial completion:  Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG (clinicaltrials.gov) -  Apr 30, 2020   
    P3,  N=70, Completed, 
    Dyspnea before thymectomy appears to associate with a poor prognosis. Active, not recruiting --> Completed
  • ||||||||||  [VIRTUAL] Investigation of immune repertoires in thymoma. () -  Apr 29, 2020 - Abstract #ASCO2020ASCO_5442;    
    Although our study is underpowered, TCR gene sequencing is efficient and may not only improve our understanding of these paraneoplastic syndromes but also identify biomarkers for their diagnosis. Our research could also potentially lead to development of targeted treatment options in the future based on TCR manipulation.
  • ||||||||||  Opdivo (nivolumab) / Ono Pharma, BMS
    [VIRTUAL] Nivolumab for thymic epithelial tumors: Case series. () -  Apr 29, 2020 - Abstract #ASCO2020ASCO_5440;    
    Low dose nivolumab may also be very toxic for T, especially in patients with known myasthenia gravis history. ICI: Immune checkpoint inhibitor, SD: Stable disease, PR: Partial response, m: Months*For adjuvant intend Research Funding: None
  • ||||||||||  Clinical, Journal:  Mathematical models: An extension of the clinician's mind. (Pubmed Central) -  Apr 29, 2020   
    Here, we focus on disorders of the neural control of eye movements, which offer many advantages to clinician scientists, providing examples of how thorny clinical mysteries became much clearer once they were formulated as models, and tested experimentally. Such an approach inevitably raises new questions and experimental tests and may suggest novel therapies.
  • ||||||||||  Journal:  103 Atypical Progressive Bulbar Palsy presenting with Dropped Head. (Pubmed Central) -  Apr 28, 2020   
    However, in all the previously reported cases, the patients had limb involvement at the time of presentation which was absent in this case, and the head drop occurred after the onset of symptoms (Lange et al, 1986; Katz et al; 1996). Dropped head syndrome can be seen in inflammatory myopathies, myasthenia gravis, facioscapulohumeral muscular dystrophy, spinal muscular atrophy, nemaline myopathy and carnitine deficiency (Umapathy et al, 2003) but ALS should also be considered in patients with atypical presentations.
  • ||||||||||  Review, Journal:  Review Analysis on Thymectomy vs Conservative Medical Management in Myasthenia Gravis. (Pubmed Central) -  Apr 28, 2020   
    We found out that rates of remission were better in post thymectomy patients than patients on various medical treatment options including corticosteroids, immunosuppressants, intravenous immunoglobulins and acetylcholinesterase inhibitors alone. Hence thymectomy is studied to be the superior treatment option than other conservative medical management options alone.
  • ||||||||||  Clinical, Journal:  Clinical utility of multigene analysis in over 25,000 patients with neuromuscular disorders. (Pubmed Central) -  Apr 28, 2020   
    Only 0.7% of these variants were later reclassified as clinically significant, most commonly in RYR1, GDAP1, SPAST, and MFN2, providing insight into the types of evidence that support VUS resolution and informing expectations of reclassification rates. These data provide guidance for clinicians using genetic testing to diagnose neuromuscular disorders and represent one of the largest studies demonstrating the utility of NGS-based testing for these disorders.
  • ||||||||||  Clinical, Journal:  Muscle Weakness in Adults: Evaluation and Differential Diagnosis. (Pubmed Central) -  Apr 26, 2020   
    Electromyography is indicated when certain diagnoses are being considered, such as amyotrophic lateral sclerosis, myasthenia gravis, neuropathy, and radiculopathy, and may also guide biopsy. If the etiology remains unclear, specialist consultation or muscle biopsy may be necessary to reach a diagnosis.
  • ||||||||||  Myasthenia gravis: From single cell signatures to cancer diagnosis  (Board Number: P430) -  Apr 25, 2020 - Abstract #IMMUNOLOGY2020IMMUNOLOGY_1152;    
    Moreover, we were able to locate T cells with the defined signature enriched in the inflamed thymus of Myasthenia gravis patients – the key organ for the induction and maintenance of the autoimmune disease. Lastly, using an unbiased pattern recognition approach, we identified lymphomas in a subset of Myasthenia gravis patients, further highlighting the potential of the applied analysis tools.
  • ||||||||||  Monovalent IgG4 autoantibodies require self-antigen driven affinity maturation to acquire pathogenic capacity  (Board Number: P419) -  Apr 25, 2020 - Abstract #IMMUNOLOGY2020IMMUNOLOGY_1139;    
    Lastly, using an unbiased pattern recognition approach, we identified lymphomas in a subset of Myasthenia gravis patients, further highlighting the potential of the applied analysis tools. These findings indicate that the autoantigen initiates the autoimmune response in MuSK MG and drives autoimmunity through the accumulation of somatic hypermutations such that monovalent IgG4 Fab-arm exchanged MG autoantibodies reach a high affinity threshold required for pathogenic capacity.
  • ||||||||||  Opdivo (nivolumab) / Ono Pharma, BMS
    Review, Journal, Checkpoint inhibition:  Immune checkpoint inhibitors-induced neuromuscular toxicity: from pathogenesis to treatment. (Pubmed Central) -  Apr 24, 2020   
    Intravenous immunoglobulin, plasma exchange and other immune-modulating treatments should be considered in more severe cases. Consideration of rechallenging with the same immunotherapy drug may be given in some cases, based on clinical picture and initial severity of irAEs.
  • ||||||||||  prednisolone / Generic mfg.
    Clinical, Journal:  Myasthenic crisis and late deep vein thrombosis following thymectomy in a patient with myasthenia gravis: A case report. (Pubmed Central) -  Apr 23, 2020   
    Additionally, patients with MG may have an increased risk of DVT possibly attributable to immune-mediated inflammation. These findings highlight the importance of perioperative avoidance of provoking factors including monitoring of stress-induced elevations in serum lactate concentration, close postoperative surveying for myasthenic crisis, and early recognition of possible thromboembolic complications in this patient population.
  • ||||||||||  sirolimus / Generic mfg., University of Cincinnati
    Clinical, Journal:  Effects of Mitophagy on Regulatory T Cell Function in Patients With Myasthenia Gravis. (Pubmed Central) -  Apr 23, 2020   
    Tregs from the MG group were subjected to rapamycin-induced culture for 48 h (Rapa group) and 3-methyladenine-induced culture for 48 h (3-MA group)...The autophagy agent 3-MA down-regulated mitophagy, which decreased the mitochondrial membrane potential and inhibitory effect of Tregs. These results reveal the possible cellular immune mechanism of Treg dysfunction in MG.
  • ||||||||||  Opdivo (nivolumab) / Ono Pharma, BMS
    Management Of Chemotherapy-induced Reversible Complete Heart Block (Poster Hall) -  Apr 22, 2020 - Abstract #RHYTHM2020RHYTHM_2338;    
    Additional study is warranted, as are preventive efforts in this high-risk population. Iatrogenic causes of CHB are common; however, nivolumab-induced CHB is a rare and serious side-effect that is potentially reversible with early recognition and aggressive treatment.
  • ||||||||||  Clinical, Journal:  Imbalance of two main circulating dendritic cell subsets in patients with myasthenia gravis. (Pubmed Central) -  Apr 22, 2020   
    In a longitudinal follow-up, we observed that circulating mDCs were significantly reduced after 1 month of therapy with a steroid and immunosuppressant, resulting in recovery of the pDC/mDC ratio. The ratio of circulating DC subsets might reflect the balance between the autoimmune response and immune tolerance of a patient, and ratio changes during treatment could be a promising marker to predict the efficacy of a specific drug used for MG patients.
  • ||||||||||  NN1213 / Novo Nordisk
    Trial suspension:  QUESST: Innovative Measures of Speech and Swallowing Dysfunction in Neurological Disorders (clinicaltrials.gov) -  Apr 22, 2020   
    P=N/A,  N=200, Suspended, 
    The ratio of circulating DC subsets might reflect the balance between the autoimmune response and immune tolerance of a patient, and ratio changes during treatment could be a promising marker to predict the efficacy of a specific drug used for MG patients. Recruiting --> Suspended
  • ||||||||||  Soliris (eculizumab) / Alexion Pharma
    Journal:  Myasthenia Gravis and Congenital Myasthenic Syndromes. (Pubmed Central) -  Apr 22, 2020   
    The diagnosis and management of patients affected by MG can be highly rewarding for any neurologist as most patients are able to live normal lives if treated appropriately. Nevertheless, future research is needed to address unresolved clinical issues, such as when and how to discontinue immunosuppressive medications in patients in remission, the role and timing of thymectomy in children, and better treatment options for refractory patients.
  • ||||||||||  Journal, Checkpoint inhibition:  Lambert-Eaton Myasthenic Syndrome, Botulism, and Immune Checkpoint Inhibitor-Related Myasthenia Gravis. (Pubmed Central) -  Apr 22, 2020   
    The diagnosis of LEMS, botulism, or immune-related MG can generally be made from the patient's history, supplemented with directed questions, a physical examination designed to demonstrate abnormalities, and laboratory and electrodiagnostic testing. Early diagnosis and carefully selected treatment not only improve outcomes of the neuromuscular disease but can affect the prognosis of underlying malignancy, when present.
  • ||||||||||  Enrollment closed:  Neurology Inpatient Clinical Education Trial (clinicaltrials.gov) -  Apr 19, 2020   
    P=N/A,  N=80, Active, not recruiting, 
    Early diagnosis and carefully selected treatment not only improve outcomes of the neuromuscular disease but can affect the prognosis of underlying malignancy, when present. Recruiting --> Active, not recruiting