Myasthenia Gravis
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  • ||||||||||  Rituxan (rituximab) / Roche
    Journal:  Cough-Variant Asthma in a Myasthenia Gravis Patient on Treatment With Steroids and Rituximab. (Pubmed Central) -  Nov 17, 2024   
    This case emphasizes how difficult it can be to diagnose respiratory symptoms in MG patients and how crucial it is to rule out CVA as a differential diagnosis, particularly in patients who have recently started immunomodulatory medication and have an allergy tendency. Fast symptom relief and positive clinical results were achieved by the efficient use of inhalation treatment.
  • ||||||||||  Neurological adverse events of ICI therapy: a single-Center ten-year experience (AUDITORIUM) -  Nov 17, 2024 - Abstract #AIOM2024AIOM_372;    
    Though rare, NirAEs are potentially lifethreatening toxicities of ICI therapy. Our long-term experience indicates that a multidisciplinary approach avoids more severe complications, and leds to clinical recovery from NirAEs in the large proportion of pts.
  • ||||||||||  Journal:  Did the speech of patients with Myasthenia Gravis decline over 4 years? (Pubmed Central) -  Nov 16, 2024   
    Despite presenting stable health status, their respiratory, phonatory, and articulatory subsystems worsened. There was no correlation between speech patterns and clinical characteristics of the disease (severity and motor scale).
  • ||||||||||  Journal:  Ocular myasthenia gravis and thyroid eye disease: double trouble. (Pubmed Central) -  Nov 15, 2024   
    In addition, further workup with orbital MRI performed due to exophthalmos and unilateral ophthalmoplegia demonstrated findings compatible with thyroid eye disease, which were further verified by antibody testing. The unusual concurrent appearance of ocular myasthenia gravis and thyroid eye disease is presented while illustrating the hallmark clinical, laboratory, and imaging findings relevant to both diseases.
  • ||||||||||  Trial completion date, Trial primary completion date:  Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Myasthenia Gravis (clinicaltrials.gov) -  Nov 15, 2024   
    P=N/A,  N=16, Recruiting, 
    The unusual concurrent appearance of ocular myasthenia gravis and thyroid eye disease is presented while illustrating the hallmark clinical, laboratory, and imaging findings relevant to both diseases. Trial completion date: Dec 2024 --> Aug 2025 | Trial primary completion date: Nov 2024 --> Apr 2025
  • ||||||||||  hydroxychloroquine / Generic mfg.
    Journal:  Oculopharyngeal muscular dystrophy, myasthenia gravis, systemic lupus erythematosus: overlap and interactions. (Pubmed Central) -  Nov 14, 2024   
    The investigation revealed a positive genetic test for OPMD and positive antibodies for acetylcholine receptor. Additionally, she has SLE, treated with hydroxychloroquine for more than 30 years.This case highlights the importance of a thoughtful anamnesis with personal and familial history and raises awareness for the rare coexistence of three pathologies with some common clinical characteristics but different treatments and management.
  • ||||||||||  Journal, Benefit-risk assessment:  Risk-Benefit Analysis of Novel Treatments for Patients with Generalized Myasthenia Gravis. (Pubmed Central) -  Nov 13, 2024   
    FcRn inhibitors and complement inhibitors assessed in this study all demonstrated clinical benefit in terms of NNT as well as an acceptable safety profile in terms of NNH. Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+
  • ||||||||||  Retrospective data, Journal:  Intensive care due to myasthenia gravis: Risk factors and prognosis. (Pubmed Central) -  Nov 13, 2024   
    Within the limitations of this meta-analysis, efgartigimod was associated with a favorable benefit-risk profile as well as a better economic value compared to ravulizumab, rozanolixizumab, and zilucoplan as treatments for anti-AChR Ab+ Our study shows an increased risk of intensive care treatment for patients with late-onset MG, female sex or thymoma, occurring usually within 6?months from diagnosis, which emphasises the importance of early treatment choices.
  • ||||||||||  Review, Journal, IO biomarker:  Immune repertoire profiling in myasthenia gravis. (Pubmed Central) -  Nov 13, 2024   
    This review delves into the generation of autoreactive TCRs and BCRs in MG and comprehensively examines the applications of immune repertoire sequencing in understanding disease pathogenesis, developing diagnostic and prognostic markers and informing targeted therapies. We also discuss the current limitations and future potential of this approach.
  • ||||||||||  Journal:  Pathogenic Th17 cells are a potential therapeutic target for tacrolimus in AChR-myasthenia gravis patients. (Pubmed Central) -  Nov 13, 2024   
    We found that TAC treatment significantly reduced Th17 and pathogenic Th17 cells, along with IL-17 levels in RG, while Th1 and Tfh cells slightly decreased without affecting Th2 or Treg subsets. This indicates that TAC's clinical benefits may be due to its inhibitory effect on the Th17 response, enhancing our insight into its immunomodulatory mechanisms in MG management.
  • ||||||||||  Journal:  Elevated serum levels of C-terminal agrin fragment in acetylcholine receptor antibody-positive myasthenia gravis. (Pubmed Central) -  Nov 13, 2024   
    Compared to healthy controls, serum CAF levels were significantly elevated in acetylcholine receptor antibody-positive MG (AChR-MG) patients, but not in muscle-specific kinase antibody-positive MG patients. In AChR-MG, baseline and post-treatment CAF levels inversely correlated with post-treatment MG activities of daily living scores, suggesting that elevated CAF levels may reflect protective mechanisms against AChR-MG pathogenesis, such as improved NMJ regeneration.
  • ||||||||||  Journal:  Elevated C1s/C1-INH in serum and plasma of myasthenia gravis patients. (Pubmed Central) -  Nov 13, 2024   
    Serum and plasma C1s-C1-INH levels were higher in MG patients than in matched healthy controls, supporting early classical pathway activation in most MG patients. These findings allow prospective validation studies of activated C1s as a putative treatment target and potential accompanying biomarker in MG.
  • ||||||||||  Journal:  Treatment of juvenile myasthenia gravis with tacrolimus: A cohort study. (Pubmed Central) -  Nov 13, 2024   
    SARS-CoV-2 vaccination had no effect on daily functioning in patients with autoimmune neuromuscular diseases, confirming its safety in these patients. The provision of tacrolimus within 1?year of JMG onset is effective and safe.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca
    Review, Journal:  Therapeutic challenges and unmet needs in the management of myasthenia gravis: an Italian expert opinion. (Pubmed Central) -  Nov 13, 2024   
    Eculizumab, a monoclonal antibody that inhibits the complement system, has been recently approved in Italy for refractory gMG...Evaluating new therapeutic options accurately is essential to find the best balance between efficacy and tolerability for each patient. Collecting real-world data on novel molecules in routine clinical practice is necessary to address unmet needs.
  • ||||||||||  Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) / argenx, Arzerra (ofatumumab) / Novartis, Genmab, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Trial completion:  Sequential Efgartigimod and Ofatumumab Therapy for Generalized Myasthenia Gravis (EUDRACT) -  Nov 10, 2024   
    P2,  N=30, Completed, 
    Collecting real-world data on novel molecules in routine clinical practice is necessary to address unmet needs. Not yet recruiting --> Completed
  • ||||||||||  Preclinical, Journal:  In vitro modulation of T cells in myasthenia gravis by low-dose IL-2. (Pubmed Central) -  Nov 7, 2024   
    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.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca, Ultomiris (ravulizumab-cwvz) / AstraZeneca
    Review, Journal:  Complement system activation: bridging physiology, pathophysiology, and therapy. (Pubmed Central) -  Nov 6, 2024   
    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.
  • ||||||||||  Descartes-08 / Cartesian Therap
    Safety and Tolerability of BCMA-Directed mRNA CAR T-Cell Therapy in Multiple Myeloma and Autoimmune Disease (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_3803;    
    P1, P2
    Clinical experience to date supports administration of mRNA CAR-T therapy in an outpatient setting and without lymphodepletion chemotherapy. Further clinical development of Descartes-08 is focused on adult and pediatric autoimmune disease, including MG (NCT04146051) and SLE (NCT06038474).
  • ||||||||||  Descartes-08 / Cartesian Therap
    Safety and Tolerability of BCMA-Directed mRNA CAR T-Cell Therapy in Multiple Myeloma and Autoimmune Disease (Halls G-H - Blood Journals Studio (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_1203;    
    P1, P2
    Clinical experience to date supports administration of mRNA CAR-T therapy in an outpatient setting and without lymphodepletion chemotherapy. Further clinical development of Descartes-08 is focused on adult and pediatric autoimmune disease, including MG (NCT04146051) and SLE (NCT06038474).