Myasthenia Gravis
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  • ||||||||||  Journal:  Developing the health state classification system for a condition-specific preference-based measure for patients with myasthenia gravis. (Pubmed Central) -  Sep 14, 2023   
    Based on a solid process of development and consultations with clinical professionals and patients, a valid MG-specific preference-based measure, MGQoL-6D, was developed. Further research will estimate the local preference weight to support the MG-related cost-utility analysis.IMPLICATION FOR REHABILITATIONA new condition-specific health state classification system (HCS) named Myasthenia gravis Quality of Life Scale (MGQoL-6D) is proposed.MGQoL-6D classifies MG health states as a combination of six dimensions with three response levels.The dimensions of the HCS are social activity, hobbies and fun activity, family needs, work performance, mobility, and emotion.The HCS and the upcoming value set of the MGQoL-6D could support the cost-effectiveness analysis of MG-related clinical and rehabilitated interventions.
  • ||||||||||  Journal:  Myasthenia gravis patients exhibiting an eyelid myotonia-like phenomenon. (Pubmed Central) -  Sep 13, 2023   
    We discuss the possible neurological pathophysiology of this disorder and suggest to name this rare ocular disorder "myotonia-like disorder of the pre-tarsal and pre-septal parts of the orbicularis oculi". This study expands our knowledge of this rare clinical feature of MG and highlights the need for increased awareness of it and further investigation of this ocular manifestation.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca
    Journal:  Starting eculizumab as rescue therapy in refractory myasthenic crisis. (Pubmed Central) -  Sep 12, 2023   
    The use of eculizumab in myasthenic crisis is still investigational, but this case report suggests that it may be a promising treatment option for patients with severe clinical condition. Ongoing clinical trials will be needed to further evaluate the safety and efficacy of eculizumab in myasthenic crisis.
  • ||||||||||  Preclinical, Journal:  Presynaptic Congenital Myasthenic Syndromes: Understanding Clinical Phenotypes through In vivo Models. (Pubmed Central) -  Sep 12, 2023   
    Finally, many presynaptic genes are expressed in the brain, justifying the presence of additional central nervous system symptoms.Several animal models have been developed to study CMS, providing the opportunity to identify disease mechanisms and test treatment options. In this review, we describe presynaptic CMS phenotypes with a focus on in vivo models, to better understand CMS pathophysiology and define new causative genes.
  • ||||||||||  Journal:  Eye Muscle MRI in Myasthenia Gravis and Other Neuromuscular Disorders. (Pubmed Central) -  Sep 12, 2023   
    Surprisingly, we found no atrophy in MG, even in patients with long-term ophthalmoplegia. This implies that even long-term ophthalmoplegia in MG does not lead to secondary structural myopathic changes precluding functional recovery.
  • ||||||||||  Journal, Adverse events, Checkpoint inhibition:  Immune checkpoint inhibitors induced side effects of the peripheral nervous system. (Pubmed Central) -  Sep 11, 2023   
    The myasthenia/myositis/myocarditis overlap syndrome deserves special attention as it carries the highest risk of mortality. The role of neurotoxic pretreatment regimens, preexisting subclinical neurological autoimmune diseases and the risk of ICI-re-challenge after irNAEs has to be further investigated.
  • ||||||||||  Journal:  Thymectomy in myasthenia gravis. (Pubmed Central) -  Sep 11, 2023   
    The role of neurotoxic pretreatment regimens, preexisting subclinical neurological autoimmune diseases and the risk of ICI-re-challenge after irNAEs has to be further investigated. Thymectomy is an effective treatment option in adult patients with early onset acetylcholine-receptor positive myasthenia gravis but uncertainty remains with regard to certain patient subgroups.
  • ||||||||||  Enrollment change, Real-world evidence, Real-world:  Descriptive Analysis of Real-world Data Collected With ME&MGopen (clinicaltrials.gov) -  Sep 11, 2023   
    P=N/A,  N=500, Recruiting, 
    Thymectomy is an effective treatment option in adult patients with early onset acetylcholine-receptor positive myasthenia gravis but uncertainty remains with regard to certain patient subgroups. N=120 --> 500
  • ||||||||||  Review, Journal:  Functional autoantibodies: Definition, mechanisms, origin and contributions to autoimmune and non-autoimmune disorders. (Pubmed Central) -  Sep 4, 2023   
    In addition, in this review article we discuss potential triggers of production of functional autoantibodies, including infections, immune deficiency and tumor development. Finally, we describe the contribution of functional autoantibodies to autoimmune diseases including autoimmune thyroid diseases, myasthenia gravis, autoimmune pulmonary alveolar proteinosis, autoimmune autonomic ganglionopathy, pure red cell aplasia, autoimmune encephalitis, pemphigus, acquired thrombotic thrombocytopenic purpura, idiopathic dilated cardiomyopathy and systemic sclerosis, as well as non-autoimmune disorders such as allograft rejection, infectious diseases and asthma.
  • ||||||||||  Journal:  Myasthenia Gravis and Ischemic Stroke: A Bidirectional Mendelian Randomization Study. (Pubmed Central) -  Aug 31, 2023   
    Finally, we describe the contribution of functional autoantibodies to autoimmune diseases including autoimmune thyroid diseases, myasthenia gravis, autoimmune pulmonary alveolar proteinosis, autoimmune autonomic ganglionopathy, pure red cell aplasia, autoimmune encephalitis, pemphigus, acquired thrombotic thrombocytopenic purpura, idiopathic dilated cardiomyopathy and systemic sclerosis, as well as non-autoimmune disorders such as allograft rejection, infectious diseases and asthma. Bidirectional MR analysis did not provide evidence to support a causal relationship between genetically predicted MG and IS, although observational studies have found such a potential link.
  • ||||||||||  An uncommon task specific dystonia masquerading as Myasthenia Gravis () -  Aug 30, 2023 - Abstract #MDSCongress2023MDS_Congress_1336;    
    Careful clinical assessment of patients is a key component of neurological diagnosis and a systematic approach is important in classification of movement disorders. This case report highlights how the task-specific element of a dystonia can be misdiagnosed as fatiguable weakness.
  • ||||||||||  D-penicillamine induced myasthenia gravis  () -  Aug 30, 2023 - Abstract #MDSCongress2023MDS_Congress_928;    
    It occurs usually 2-12 month after treatment initiation. Symptoms are usually ocular, with good response to pyridostigmine and remits within 1 year after DPA treatment cessation.
  • ||||||||||  Traveling through the telescope: a neuromuscular and a movement disorders reunion () -  Aug 30, 2023 - Abstract #MDSCongress2023MDS_Congress_800;    
    There is clinical overlap between DP and MG, involving symptoms/signs such as fatigue, motor disability, dysarthria and dysphagia. Our case report reinforces the importance of reconsider the diagnosis when unusual symptoms appear and avoid blaming it on a previously diagnosed entity.
  • ||||||||||  mycophenolate mofetil / Generic mfg.
    DaT Scan Positive Parkinson () -  Aug 30, 2023 - Abstract #MDSCongress2023MDS_Congress_796;    
    This case demonstrates the clinical challenges that are present in this overlap of neuromuscular and neurodegenerative disease in a single individual. We highlight the importance of a thorough history and physical examination, supported with objective evidence by a positive DaT scan, to elucidate the appropriate etiology of symptoms.
  • ||||||||||  Lumevoq (lenadogene nolparvovec) / GenSight, Rituxan (rituximab) / Biogen, Zenyaku Holdings, Roche
    Clinical, Journal:  Literature Commentary. (Pubmed Central) -  Aug 30, 2023   
    We highlight the importance of a thorough history and physical examination, supported with objective evidence by a positive DaT scan, to elucidate the appropriate etiology of symptoms. Mark L. Moster, Marc J. Dinkin, and Deborah I. Friedman discuss the following 6 articles:Piehl F, Eriksson-Dufva A, Budzianowska A, Feresiadou A, Hansson W, Hietala MA, H
  • ||||||||||  prednisolone / Generic mfg., carboplatin / Generic mfg., paclitaxel / Generic mfg.
    A rare case of multiorgan autoimmunity associated with thymoma: TAMA (e-Poster Hall) -  Aug 30, 2023 - Abstract #EADV2023EADV_3598;    
    It is considered necessary for disease control of TAMA to reduce the volume of thymoma as much as possible by thymectomy and chemotherapy. Narrowband-UVB is known to enhance the regulatory T cell proliferation, and it has been reported that narrowband-UVB is highly effective in acute GVHD skin lesions.
  • ||||||||||  Case of Thymoma-associated multiorgan autoimmunity and literature review (e-Poster Hall) -  Aug 30, 2023 - Abstract #EADV2023EADV_3532;    
    It may present as an erythroderma that may be reminiscent of graft-versus-host disease (GVHD), along with colitis, hepatitis and thyroiditis. Histopathologically, similar findings to those of GVHD are observed, with a characteristic absence of Langerhans cells in the epidermis.
  • ||||||||||  azathioprine / Generic mfg.
    A Case of Azathioprine-induced Hair Loss in a Patient with Myasthenia Gravis (e-Poster Hall) -  Aug 30, 2023 - Abstract #EADV2023EADV_3399;    
    The hair grew back within one and a half months of stopping the azathioprine, as well as application of minoxidil 2% solution twice daily. This case highlights the complex interactions of medication, immunosuppression, autoimmunity, and the hair cycle equilibrium, as well as anagen effluvium as a rare possible side effect of azathioprine.
  • ||||||||||  Imfinzi (durvalumab) / AstraZeneca
    Durvalumab-associated generalized morphea (e-Poster Hall) -  Aug 30, 2023 - Abstract #EADV2023EADV_2785;    
    The long-term side effects of these agents remain to be determined. The spectrum of irAEs is still being dismembered and it would be of value to identify biomarkers or unique gene profiles that help to predict them.
  • ||||||||||  Opdivo (nivolumab) / Ono Pharma, BMS
    Ptosis under Check Point Inhibitors (e-Poster Hall) -  Aug 30, 2023 - Abstract #EADV2023EADV_2718;    
    CPI-treatment comes with the risk of autoimmune adverse reactions of which myositis presents neuromuscular affections with myocarditis posing a potentially life-threatening reaction. Unilateral ptosis may be an early marker of immune-related myositis and hence myocarditis which prompts diligent diagnostic assessment and therapeutic intervention to prevent fatal outcome.
  • ||||||||||  ivermectin oral / Generic mfg.
    Journal:  Fatal Disseminated Strongyloidiasis in an Immunosuppressed Patient During COVID-19 Pandemic. (Pubmed Central) -  Aug 29, 2023   
    Clinicians working in endemic areas should be mindful of the possibility of gastric involvement in strongyloidiasis, even if symptoms are nonspecific. We recommend that high-risk individuals be screened for S. stercoralis prior to immunosuppressive therapy in endemic regions to raise awareness and prevent similar cases.
  • ||||||||||  Ultomiris (ravulizumab-cwvz) / AstraZeneca
    Trial completion, Trial completion date:  Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis (clinicaltrials.gov) -  Aug 29, 2023   
    P3,  N=175, Completed, 
    We recommend that high-risk individuals be screened for S. stercoralis prior to immunosuppressive therapy in endemic regions to raise awareness and prevent similar cases. Active, not recruiting --> Completed | Trial completion date: Dec 2023 --> May 2023
  • ||||||||||  Keytruda (pembrolizumab) / Merck (MSD)
    Journal, Adverse events, Checkpoint inhibition:  Unexpected Adverse Events of Immune Checkpoint Inhibitors. (Pubmed Central) -  Aug 26, 2023   
    One of the two experienced palmoplantar keratoderma, while the other presented with Reiter's syndrome (urethritis, conjunctivitis and arthritis). The 4th patient, an 80-year-old male with stage 4 squamous cell carcinoma of the lung, received chemo-immunotherapy (pembrolizumab) and developed myasthenia gravis.
  • ||||||||||  Trial completion date, Trial primary completion date:  The EXPLORE MG Registry for Myasthenia Gravis (clinicaltrials.gov) -  Aug 25, 2023   
    P=N/A,  N=800, Recruiting, 
    The 4th patient, an 80-year-old male with stage 4 squamous cell carcinoma of the lung, received chemo-immunotherapy (pembrolizumab) and developed myasthenia gravis. Trial completion date: Mar 2024 --> Oct 2026 | Trial primary completion date: Sep 2023 --> Sep 2026
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Liver Transplant in a Patient with PDA and Myasthenia Gravis (Exhibit Hall) -  Aug 23, 2023 - Abstract #ASA2023ASA_6034;    
    65y/o male with history of patent ductus arteriosus and severe Myasthenia Gravis (controlled with thymectomy and a novel drug Efgartigimod alfa (Vyvigard)) underwent a deceased donor liver transplant...His MG was evaluated by Endocrinology who advised a course of IVIG to prevent myasthenic crisis in the perioperative period. He successfully underwent DDLT and recovered well without any significant morbidity from his PDA or MG.
  • ||||||||||  Perioperative Management for Cardiac Ablation in a patient with Myasthenia Gravis (South: Hall F) -  Aug 23, 2023 - Abstract #ASA2023ASA_5810;    
    Our patient was a 55 yo M with a history of myasthenia gravis scheduled for cardiac ablation for atrial fibrillation. In order to circumvent the risks of neuromuscular blockade, we required compliance with pyridostigmine and performed induction, intubation and maintenance of anesthesia without use of paralytic agents requiring meticulous attention to anesthetic depth and prevention of post operative complications.