Myasthenia Gravis
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  • ||||||||||  Thymectomy in Myasthenic Patients with Thymoma: Killing Two Birds with One Stone () -  Dec 29, 2019 - Abstract #STSTechCon2020STS_Tech-Con_540;    
    Surgery is a cornerstone in the treatment of patients with both thymoma and Myasthenia. Extended thymectomy, as proposed by Masaoka, offers considerable oncological outcomes with a low recurrence-rate of thymoma and improved neurological results with one third of the patients reaching a complete stable remission of the neurological disease.
  • ||||||||||  efgartigimod IV (ARGX-113 IV) / argenx, Broteio
    Clinical, P2 data, Journal:  Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. (Pubmed Central) -  Dec 25, 2019   
    Efgartigimod was safe and well-tolerated. The correlation between reduction of levels of pathogenic IgG autoantibodies and disease improvement suggests that reducing pathogenic autoantibodies with efgartigimod may offer an innovative approach to treat MG.
  • ||||||||||  Clinical, Journal:  Myasthenic crisis following iodinated contrast material (iohexol) aspiration: a case report. (Pubmed Central) -  Dec 25, 2019   
    Myasthenic crisis can be precipitated by various factors and a successful recovery requires mechanical respiratory support with immunomodulatory and steroid therapy. This is the first reported case that describes the development of myasthenic crisis following iohexol-associated aspiration pneumonitis.
  • ||||||||||  Journal:  Ice Pack Test - An Useful Bedside Test to Diagnose Myasthenia Gravis. (Pubmed Central) -  Dec 23, 2019   
    Investigations that can be used to diagnose myasthenia gravis include serum acetylcholine receptor antibodies and repetitive nerve stimulation, however, these tests are not widely available. Here, we reported a case of myasthenia crisis which was diagnosed early by ice pack test and it resulted in full recovery of the patient.
  • ||||||||||  Review, Journal:  Therapeutic apheresis in neurological, nephrological and gastrointestinal diseases. (Pubmed Central) -  Dec 23, 2019   
    (category I) TPE has been used as the key therapeutic modality to reduce anti-A or anti-B antibody titers in the liver peri-transplant period with the goal of preventing rejection and facilitating graft survival. Also, plasma exchange is the first-line therapy in Wilson's disease (category I, level1C).
  • ||||||||||  Journal:  Effects of Distigmine on the Mechanical Activity of Urinary Bladder Smooth Muscle. (Pubmed Central) -  Dec 23, 2019   
    In this review article, we summarize the reported effects of distigmine on the contractile responses elicited by exogenous and endogenous ACh in isolated UBSM preparations. We also discuss the effects of distigmine on the UBSM basal tone and the contractile response of UBSM to ATP, which is co-released with ACh from parasympathetic nerve terminals.
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD)
    [VIRTUAL] DARK SIDE OF IMMUNE CHECKPOINT INHIBITOR THERAPY, BALANCING CONCURRENT MYOCARDITIS AND MULTIORGAN DYSFUNCTION (eAbstract Virtual Hall) -  Dec 22, 2019 - Abstract #ACC2020ACC_6648;    
    Here, we present a rare case of concurrent myocarditis, myositis, transaminitis and polyneuropathy manifesting as respiratory failure, dysphagia, dysphonia, muscle weakness attributed to ICI therapy.Case: 81 year old male with hypertension, metastatic lung adenocarcinoma was started on pembrolizumab and developed progressively worsening oropharyngeal dysphagia, voice hoarseness, dyspnea and weakness... This is a rare case of simultaneous ICI mediated neurological, hepatic, respiratory and cardiac adverse effects illustrating the importance of a multidisciplinary team approach.
  • ||||||||||  tacrolimus / Generic mfg.
    Journal:  Thymectomy during Myasthenic Crisis under Artificial Respiration. (Pubmed Central) -  Dec 21, 2019   
    However, a myasthenic crisis (MC) occurred, and intensive care, including emergent endobronchial intubation followed by artificial ventilation, pulse steroid therapy, high-dose intravenous immunoglobulin, and tacrolimus hydrate, was initiated...Scheduled artificial ventilation and steroid therapy together can, therefore, enable complete resection of thymoma in patients undergoing treatment for MC. While ventilation helps avert a respiratory failure, the steroid therapy temporarily reduces the tumor size, making resection easier.
  • ||||||||||  Clinical, Journal:  Incidence and Ocular Features of Pediatric Myasthenias. (Pubmed Central) -  Dec 21, 2019   
    Although relatively rare, myasthenia gravis in children has two predominant forms, CMS and JMG, both of which commonly have ocular involvement. Improvement is more likely in children with the juvenile form.
  • ||||||||||  Journal:  Myasthenic congenital myopathy from recessive mutations at a single residue in Na1.4. (Pubmed Central) -  Dec 20, 2019   
    Novel mutations in families with myasthenic congenital myopathy have been identified at p.R1460 of the sodium channel. Recessive inheritance, with experimentally established loss-of-function, is a consistent feature of sodium channel based myasthenia, whereas the mixed gain of function for p.R1460 may also cause susceptibility to myotonia.
  • ||||||||||  Opdivo (nivolumab) / Ono Pharma, BMS, Yervoy (ipilimumab) / Ono Pharma, BMS
    Review, Journal, Adverse events:  Neuromuscular adverse events associated with anti-PD-1 monoclonal antibodies: Systematic review. (Pubmed Central) -  Dec 20, 2019   
    Mortality was high in these patients, despite adequate treatment strategies including corticosteroid, IV immunoglobulins, and plasma exchange. The clinical presentation of NMDs associated with PD-1 inhibitors is often atypical, with considerable overlap between myasthenia gravis and myopathy, and cardiac/respiratory complications are common.
  • ||||||||||  Journal:  Immunoglobulin Treatment in Neuromuscular Medicine. (Pubmed Central) -  Dec 20, 2019   
    IVIg is a well-tolerated and effective treatment for these neuromuscular diseases. With this review article, we hope to increase clinicians' awareness of the indications and efficiencies of IVIg in a broad spectrum of neuromuscular diseases.
  • ||||||||||  Opdivo (nivolumab) / Ono Pharma, BMS
    Journal:  Severe Relapse of Vaccine-Induced Guillain-Barré Syndrome After Treatment With Nivolumab. (Pubmed Central) -  Dec 20, 2019   
    We present a patient who developed a severe and fatal relapse of postvaccination GBS after he was treated with nivolumab, a monoclonal antibody directed to programmed death-1 (PD-1), during a GBS treatment-related fluctuation. We recommend that caution be exercised in starting treatment with PD-1 inhibitors in the acute stage or early in the recovery period of GBS.
  • ||||||||||  Enrollment open, Real-world evidence, Real-world:  The Impact of Myasthenia Gravis in the Real World (clinicaltrials.gov) -  Dec 19, 2019   
    P=N/A,  N=2000, Recruiting, 
    We recommend that caution be exercised in starting treatment with PD-1 inhibitors in the acute stage or early in the recovery period of GBS. Not yet recruiting --> Recruiting
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD)
    Clinical, Journal:  A case of myasthenia gravis and myositis induced by pembrolizumab (Pubmed Central) -  Dec 19, 2019   
    In this case, anti-titin antibodies, one of anti-striational antibodies, was positive and correlated with severity of myasthenia gravis. With the development of immune checkpoint inhibitors for various malignancies, clinicians should closely monitor patients for important immune-related adverse events and coordinate on early treatment.
  • ||||||||||  Journal:  SHP2 inhibitor protects AChRs from effects of myasthenia gravis MuSK antibody. (Pubmed Central) -  Dec 15, 2019   
    Further researches are needed to elucidate the pathogenesis of the coexistence. Stimulating the agrin-LRP4-MuSK-DOK7 AChR clustering pathway with NSC-87877, or other drugs, could represent a novel therapeutic approach for MuSK-MG and could potentially improve other NMJ disorders with reduced AChR numbers or disrupted NMJs.
  • ||||||||||  Clinical, Journal:  Frequency and clinical features of treatment-refractory myasthenia gravis. (Pubmed Central) -  Dec 14, 2019   
    A small subgroup of patients with generalized myasthenia gravis do not respond sufficiently to standard therapies. Refractory disease has considerable implications for both patients and health care providers and highlights an unmet need for new treatment options.
  • ||||||||||  Hizentra (human immune globulin subcutaneous 20%) / CSL Behring, NN1213 / Novo Nordisk
    Trial completion:  Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis (clinicaltrials.gov) -  Dec 13, 2019   
    P2,  N=23, Completed, 
    Refractory disease has considerable implications for both patients and health care providers and highlights an unmet need for new treatment options. Active, not recruiting --> Completed
  • ||||||||||  Trial completion, Trial completion date, HEOR:  Mya-BoD: Burden of Disease in Myasthenia Gravis (clinicaltrials.gov) -  Dec 9, 2019   
    P=N/A,  N=1680, Completed, 
    N=2000 --> 600 Active, not recruiting --> Completed | Trial completion date: Dec 2019 --> Jul 2019
  • ||||||||||  Journal:  Tolerising cellular therapies: what is their promise for autoimmune disease? (Pubmed Central) -  Dec 8, 2019   
    In this review, we examine the current evidence for these three types of cellular therapy, in the context of a broader discussion around potential development pathway(s) and their likely future role. A brief overview of preclinical data is followed by a comprehensive discussion of human data.
  • ||||||||||  Soliris (eculizumab) / Alexion Pharma
    Journal:  Development and characterisation of novel anti-C5 monoclonal antibodies capable of inhibiting complement in multiple species. (Pubmed Central) -  Dec 6, 2019   
    Eculizumab, a monoclonal antibody (mAb) that binds C5 and blocks its cleavage by the convertase, is the current reference standard treatment for atypical haemolytic uraemic syndrome (aHUS) and paroxysmal nocturnal haemoglobinuria (PNH) and in clinical trials for many other diseases...The mAb 10B6 was human-specific while mAb 4G2 and 7D4 efficiently inhibited lysis by human, rabbit and rat serum, and weakly inhibited guinea pig complement; 7D4 also weakly inhibited mouse complement in vitro The rat C5-cross-reactive mAb 4G2, when administered intraperitoneally in a rat model of myasthenia gravis, effectively blocked the disease and protected muscle endplates from destruction. To our knowledge this is the first report of an anti-C5 function blocking mAb that permits preclinical studies in rats.
  • ||||||||||  bortezomib / Generic mfg.
    Enrollment change, Trial completion date, Trial termination, Trial primary completion date:  Therapy of Antibody-mediated Autoimmune Diseases by Bortezomib (TAVAB) (clinicaltrials.gov) -  Dec 3, 2019   
    P2,  N=11, Terminated, 
    To our knowledge this is the first report of an anti-C5 function blocking mAb that permits preclinical studies in rats. N=18 --> 11 | Trial completion date: Dec 2019 --> Aug 2019 | Recruiting --> Terminated | Trial primary completion date: Dec 2019 --> Aug 2019; recruitment difficulties
  • ||||||||||  Clinical, Journal:  The utility of anti-SOX2 antibodies for cancer prediction in patients with paraneoplastic neurological disorders. (Pubmed Central) -  Dec 2, 2019   
    SOX2 antibodies were detectable in 61% of patients with LEMS-SCLC, and in other paraneoplastic disorders, such as opsoclonus-myoclonus and paraneoplastic cerebellar degeneration, only when there was an underlying SCLC. SOX2 antibodies are specific (>90%) markers for SCLC, but are rarely found in patients with other tumours, whether neurological symptoms are present or not.