- |||||||||| Keytruda (pembrolizumab) / Merck (MSD)
Severe irAEs in patients with immune checkpoint inhibitor therapy: Experience in a Mexican cancer center. () - Apr 28, 2022 - Abstract #ASCO2022ASCO_1791;
The predominant treatment was pembrolizumab monotherapy (56%), with an average of 5 applications after those who presented irAEs...70% of these patients were managed with three boluses of methylprednisolone and subsequently prednisone 1 mg/kg as maintenance... Among the 125 patients who received an immune checkpoint inhibitor in our cancer center in 2021, the most prevalent serious irAE was myocarditis, with a higher incidence than that reported by other centers, and a prolonged therapeutic response was found in the patients who presented it.
- |||||||||| Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
Enrollment change, Trial completion date, Trial primary completion date: Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (clinicaltrials.gov) - Apr 25, 2022
P2, N=80, Recruiting,
Our study shows that HHD, AF and IHD increase the risk of NIV failure in MC receiving ventilation. N=40 --> 80 | Trial completion date: Dec 2027 --> Jun 2033 | Trial primary completion date: Mar 2026 --> Jan 2028
- |||||||||| Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
Journal: Efgartigimod alfa (Vyvgart) for myasthenia gravis. (Pubmed Central) - Apr 25, 2022
N=40 --> 80 | Trial completion date: Dec 2027 --> Jun 2033 | Trial primary completion date: Mar 2026 --> Jan 2028 No abstract available
- |||||||||| Review, Journal, Checkpoint inhibition: Exploring the Mechanisms Underlying the Cardiotoxic Effects of Immune Checkpoint Inhibitor Therapies. (Pubmed Central) - Apr 24, 2022
This is further complicated by the identification of multiple co-stimulatory and co-inhibitory signals, as well as the association of myocarditis with antibody-driven myasthenia gravis and myositis IRAEs. In this review, we focus on the recent advances in unraveling the complexity of the mechanisms driving ICI cardiotoxicity and discuss novel therapeutic strategies for directly targeting specific underlying mechanisms to reduce IRAEs and improve outcomes.
- |||||||||| prednisone / Generic mfg.
Journal, HEOR: Quality of Life in Myasthenia Gravis and Correlation of MG-QOL15 with Other Functional Scales. (Pubmed Central) - Apr 24, 2022
We recruited 45 patients with generalised MG who were starting immunomodulatory treatment with intravenous immunoglobulins and prednisone for the first time...The questionnaire MG-QOL15 correlated positively with other clinical measures. As MG is a fluctuating condition, and some symptoms are difficult to examine, we direct physicians toward the use of scales and questionnaires composed of items perceived by the patient.
- |||||||||| Preclinical, Review, Journal: Current Progress in the Creation, Characterization, and Application of Human Stem Cell-derived in Vitro Neuromuscular Junction Models. (Pubmed Central) - Apr 22, 2022
The use of stem cells for in vitro NMJ models is still in progress and requires further optimization in order to yield reliable, reproducible results. The objective of this review is (1) to outline the current progress towards fully PSC-derived in vitro co-culture models of the human NMJ and (2) to discuss future directions and challenges that must be overcome in order to create reproducible fully PSC-derived models that can be used for developmental studies, disease modeling, and drug testing.
- |||||||||| Clinical, Journal: Positive acetylcholine receptor antibody in non-myasthenic patients. (Pubmed Central) - Apr 22, 2022
Elevated titers of AChR binding antibody can sometimes be found in nonmyasthenic patients. Combined analysis of clinical presentation, AChR binding antibody titer, and AChR-modulating antibody results can be helpful in confirming an MG diagnosis.
- |||||||||| methotrexate / Generic mfg.
Journal, PD(L)-1 Biomarker, IO biomarker: Pathogenesis of follicular thymic hyperplasia associated with rheumatoid arthritis. (Pubmed Central) - Apr 22, 2022
These results suggest that FTH should be considered in patients with RA treated with methotrexate. The pathogenesis of FTH-RA includes GC expansion and increased numbers of memory B cells, follicular helper T cells, and myoid cells, indicating humoral immunity activation.
- |||||||||| Observational data, Retrospective data, Journal: Differences in immunophenotypes between myasthenia gravis patients with and without thyroid antibodies. (Pubmed Central) - Apr 22, 2022
The immunophenotypes of MG patients with and without TAb positivity were significantly different, suggesting that their sensitivity to immunotherapy may be different. Further studies focused on differences between TAb positive and TAb negative MG patients in their responses to specific immunotherapies are needed to support our exploratory findings.
- |||||||||| Retrospective data, Journal: Autoantibody profile in myasthenia gravis patients with a refractory phase. (Pubmed Central) - Apr 22, 2022
In this study, patients with refractory MG were more likely than those with nonrefractory MG to be DSN; and refractory DSNMG patients had worse MGFA classes in their recent visit compared with anti-AChR positive refractory patients. Refractory DSNMG patients may represent a distinct group that requires more individualized and targeted treatment approaches.
- |||||||||| Poster Tour: Rare and Orphan Diseases (In-person) - Apr 21, 2022 - Abstract #ISPOR2022ISPOR_1913;
Available data support the use of IVIg-C as an effective and safe treatment for this severely ill subgroup of patients during a relapse. Posters featured in this tour: EE15: Economic Burden of Transfusion-Dependent Beta-Thalassemia in the United States EE48: Cost-Effectiveness of Alternative Diagnostic Testing Pathways with Whole Exome Sequencing (WES) in a Rare Disease Patient Population: The Canadian Care-for-Rare SOLVE (SOLVE) Multi-Centre Observational Cohort EE500: Comparison of Healthcare Resource Utilization and Costs in Patients with and without Bullous Pemphigoid: A Retrospective Analysis of US Health Insurance Claims Data HSD67: The Burden of Systemic Glucocorticoid (GC) Use in Anti-Neutrophilic Cytoplasmic Autoantibody (ANCA)-Associated Vasculitis Patients HSD85: Real-World Usage Patterns and Costs of IVIG Treatment in Adults with Generalized Myasthenia Gravis in the United States PCR102: Leveraging Social Media for Patient Experience Insights in Rare Disease
- |||||||||| Journal: Neurogenerative Disease Diagnosis in Cepstral Domain Using MFCC with Deep Learning. (Pubmed Central) - Apr 21, 2022
Although the 1D CNN had the maximum accuracy of 93.11% on test data, model training produced overfitting and 2D CNN, which generalized the data better and had lower train and validation loss despite having an accuracy of 84.17% on test data. Also, 2D CNN outperforms state-of-the-art studies in the field, implying that a model trained on handcrafted features is better for speech processing than a model that extracts features directly.
- |||||||||| Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Muscle-specific Tyrosine Kinase Chimeric Autoantibody Receptor T Cells (MuSK-CAART): A Precision Cellular Immunotherapy for Antigen-specific B Cell Depletion in MuSK Myasthenia Gravis (Salon G) - Apr 20, 2022 - Abstract #ASGCT2022ASGCT_1100;
MuSK myasthenia gravis therapy with steroids and the anti-CD20 monoclonal antibody rituximab aims to suppress or eliminate antibody-producing B cells, although disease often relapses due to incomplete B cell depletion and even transient B cell depletion risks serious infections...Toxicology studies in the NSG xenograft model and screens of a high-throughput human membrane proteome array and primary human cells did not identify MuSK-CAART off-target cytotoxic interactions. These data in part formed the basis of an investigational new drug application for MuSK-CAART for the treatment of MuSK autoantibody-positive myasthenia gravis, which has been allowed to proceed by the FDA.
- |||||||||| Comirnaty (tozinameran) / Pfizer, Fosun Pharma, BioNTech, Spikevax (elasomeran) / Moderna, Takeda
Journal: Flare of myasthenia gravis induced by COVID-19 vaccines. (Pubmed Central) - Apr 20, 2022
Our study expands the spectrum of spindle cell thymoma by demonstrating the existence of cases that are predominantly composed of lymphocyte-rich elements and lack areas with a pure (lymphocyte poor) spindle cell morphology. No abstract available
- |||||||||| Journal: COMPARATIVE ANALYSIS OF CURRENT SURGICAL APPROACHES TO THYMIC TUMORS TREATMENT. (Pubmed Central) - Apr 20, 2022
All patients were performed thymectomy by videothoracoscopic method, there were no postoperative crises of myasthenia gravis and other complications in any case. Research results demonstrate high efficiency minimally invasive thymus surgery in clinic practice, which be of help to optimal management and allows to reduce the socio-economic loading on health service.
- |||||||||| prednisone / Generic mfg.
Clinical, Review, Journal: Laryngeal myasthenia gravis following influenza vaccination: a case report and literature review. (Pubmed Central) - Apr 19, 2022
The patient received pyridostigmine oral administration (60 mg/d) and steroid therapy (Prednisone, oral, 60 mg/d)...Influenza vaccination might cause an unexpected abnormal autoimmune response in MG as a very rare event. Further research is needed to assess the possible causal relationship between the influenza vaccine and neurological complications, also in addition to the safety of the vaccine.
- |||||||||| Review, Journal: Adaptive Remodeling of the Neuromuscular Junction with Aging. (Pubmed Central) - Apr 19, 2022
The NMJ also shows plasticity in response to a number of neuromuscular diseases such as amyotrophic lateral sclerosis (ALS) and Lambert-Eaton myasthenic syndrome (LEMS). Here, the structural and functional degradation of the NMJ associated with aging and disease is described, along with the measures that might be taken to effectively mitigate, if not fully prevent, that degeneration.
- |||||||||| Review, Journal: Myasthenia Gravis: An Acquired Interferonopathy? (Pubmed Central) - Apr 19, 2022
Some systemic autoimmune diseases also share common features with interferonopathies. This review aims to analyze the pathogenic role of IFN-I in these diseases as compared to AChR-MG in order to determine if AChR-MG could be an acquired interferonopathy.
- |||||||||| tacrolimus oral / Generic mfg.
Journal: Efficacy and Safety of Tacrolimus Therapy for a Single Chinese Cohort With Very-Late-Onset Myasthenia Gravis. (Pubmed Central) - Apr 19, 2022
Regarding the 13 patients, coadministering Wuzhi capsules the tacrolimus concentration increased from 2.75 (1.4-3.8) ng/ml to 5.95 (5.1-7.0) ng/ml (p = 0.012)...Tacrolimus monotherapy without corticosteroids can be used as an initial and maintenance treatment for VLOMG. Wuzhi capsules work well in elevating tacrolimus concentrations in this population.
- |||||||||| Vaxzevria (ChAdOx1-S recombinant) / University of Oxford, Emergent Biosolutions, Jenner Institute, Vaccitech, AstraZeneca
Journal: New-Onset Myasthenia Gravis After ChAdOx1 nCOV-19 Vaccine Inoculation. (Pubmed Central) - Apr 19, 2022
Higher MG-QOL-15 scores were a risk factor for anxiety and depression in late-onset MG, and women with late-onset MG were more likely to have anxiety and depression than men were. No abstract available
- |||||||||| Soliris (eculizumab) / AstraZeneca, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Observational data, Retrospective data, Journal: Eculizumab versus rituximab in generalised myasthenia gravis. (Pubmed Central) - Apr 15, 2022
This retrospective, observational study provides the first real-world evidence supporting the use of eculizumab for the treatment of refractory, anti-AChR-ab positive MG. Nonetheless, the risk of MC remained high and prompts the need for intensified monitoring and further research effort aimed at this vulnerable patient cohort.
- |||||||||| Journal: The CMS19 disease model specifies a pivotal role for collagen XIII in bone homeostasis. (Pubmed Central) - Apr 15, 2022
In the Col13a1 mice, where ColXIII is synthesized but the ectodomain shedding is prevented due to a mutation in a protease recognition sequence, the cortical bone mass decreased as well with age and the cephalometric analyses revealed significant craniofacial abnormalities but no clear phenotypical pattern. To conclude, our data indicates an intrinsic role for ColXIII, particularly the soluble form, in the upkeep of bone with aging and suggests the possibility of previously undiscovered bone pathologies in patients with CMS19.
- |||||||||| Journal: Analysis of nAChR Autoantibodies Against Extracellular Epitopes in MG Patients. (Pubmed Central) - Apr 15, 2022
Our results reveal that these autoantibodies can be divided into distinct groups, based on their target, with probably different impacts on disease severity. Although our findings are based on a small sample group of patients, they strongly support that additional analysis of the specificity of the autoantibodies of patients with MG could serve as a valuable tool for the clinicians' decision on the treatment strategy to be followed.
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