- |||||||||| Review, Journal, Tumor Mutational Burden: Genomic alterations in thymoma-molecular pathogenesis? (Pubmed Central) - Jan 16, 2021
TCs, however, display a higher mutational burden, with frequent mutations of TP53 and epigenetic regulatory genes and loss of CDKN2A. The knowledge of molecular alterations in TETs fosters the understanding of their pathogenesis and provides guidance for further studies that may lead to the development of targeted therapies.
- |||||||||| Review, Journal: Autoimmune disorders and paraneoplastic syndromes in thymoma. (Pubmed Central) - Jan 16, 2021
Whenever suitable and backed by the limited available evidence, the perspectives from both the thymoma and the affected organ/tissue will be highlighted. Specific issues addressed are the prognostic significance of thymectomy on myasthenia gravis and other thymoma-associated AID/PND and further the impact and safety of immunotherapies on AID and PND relating to thymomas.
- |||||||||| Clinical, Review, Journal: When to suspect a thymoma: clinical point of view. (Pubmed Central) - Jan 16, 2021
A clinical diagnosis, especially if the first manifestation is a thymoma-associated paraneoplastic disease, is not always easy and should be supported by an appropriate imaging in order to guide the proper management for each patient. In this article, we would like to give an overview of the local and systemic clinical manifestations, which can be found in patients affected by thymoma.
- |||||||||| Journal, IO biomarker: Signaling pathways of genetic variants and miRNAs in the pathogenesis of myasthenia gravis. (Pubmed Central) - Jan 16, 2021
These five key genes containing SNPs or targeted by miRNAs were found to be involved in negative regulation of T cell differentiation. We speculate that SNPs cause the genes to be defective or the miRNAs to downregulate the factors that subsequently negatively regulate regulatory T cells and trigger the onset of MG.
- |||||||||| Journal: Neuro-Ophthalmologic Complications of Coronavirus Disease 2019 (COVID-19). (Pubmed Central) - Jan 15, 2021
These symptoms and signs may be the result of a range of pathophysiological mechanisms throughout the course from acute illness to recovery phase. Optic nerve dysfunction, eye movement abnormalities and visual field defects have been described.
- |||||||||| Clinical, Review, Journal: Outcome Measures in Clinical Trials of Patients With Myasthenia Gravis. (Pubmed Central) - Jan 12, 2021
We highlight the choice of outcome measures in published and ongoing trials, and we denote whether trial efficacy was reached on these outcomes. We discuss advantages and limitations of the individual scales, and discuss some of the unresolved factors relating to outcome assessments in MG.
- |||||||||| Trial completion, Enrollment change, Trial primary completion date: OVEMP in Myasthenia (clinicaltrials.gov) - Jan 11, 2021
P=N/A, N=96, Completed,
We discuss advantages and limitations of the individual scales, and discuss some of the unresolved factors relating to outcome assessments in MG. Recruiting --> Completed | N=200 --> 96 | Trial primary completion date: Dec 2019 --> Dec 2020
- |||||||||| Opdivo (nivolumab) / Ono Pharma, BMS
Journal, Checkpoint inhibition: Neurologic complications of immune checkpoint inhibitors. (Pubmed Central) - Jan 8, 2021
Various immunobiologic triggers have been proposed to explain why certain patients might develop neurologic irAEs and are also briefly discussed.Expert Opinion: All providers who care for patients with cancer should be made aware of common neurologic irAEs and able to recognize when prompt evaluation and consultation with appropriate specialists are indicated. Symptoms suggestive of encephalitis, myasthenia-gravis or an acute polyradiculopathy such as Guillain-Barre Syndrome (GBS) in patients exposed to these agents warrant immediate attention with a low threshold for hospitalization to expedite work-up and monitor for severe and/or life-threatening manifestations.
- |||||||||| Review, Journal: Double vision in Parkinson's Disease: a systematic review. (Pubmed Central) - Jan 8, 2021
The acute onset of diplopia should always result in tests to exclude other diseases. Tailored interventions should be developed, leading to improved patient safety, quality of life, and quality of care.
- |||||||||| Journal: Cerebellar ataxia and exercise intolerance in Erdheim-Chester disease. (Pubmed Central) - Jan 8, 2021
Tailored interventions should be developed, leading to improved patient safety, quality of life, and quality of care. In patients with myasthenia-like symptoms who test negatively for myasthenia gravis, neurodegenerative diseases, and disorders of the hypothalamus, a diagnosis of ECD should be taken into consideration.
- |||||||||| Trial completion date, Trial primary completion date: Neurology Inpatient Clinical Education Trial (clinicaltrials.gov) - Jan 8, 2021
P=N/A, N=80, Active, not recruiting,
In patients with myasthenia-like symptoms who test negatively for myasthenia gravis, neurodegenerative diseases, and disorders of the hypothalamus, a diagnosis of ECD should be taken into consideration. Trial completion date: Dec 2020 --> Dec 2021 | Trial primary completion date: Oct 2020 --> Oct 2021
- |||||||||| botulism antitoxin heptavalent (NP-018) / Emergent Biosolutions
[VIRTUAL] Wound botulism in black TAR heroin injecting users () - Jan 6, 2021 - Abstract #WSMRF2021WSMRF_498;
If there is high clinical suspicion, a prompt administration of botulism antitoxin heptavalent (BAT) should be considered...The history of drug injection and signs of infection especially around the drug injecting sites further increase the suspicion. The timely administration of BAT can significantly lessen the duration and severity of wound botulism.
- |||||||||| Biomarker, Clinical, Journal: Paediatric myasthenia gravis: Prognostic factors for drug free remission. (Pubmed Central) - Jan 6, 2021
Complete drug free remission is not uncommon in paediatric MG and several factors appear to influence this outcome with antibody status being the most important. These factors can be easily evaluated at diagnosis, and may help to determine whose patients are likely to require more intensive treatments.
- |||||||||| Journal: Emerging therapies for autoimmune myasthenia gravis: Towards treatment without corticosteroids. (Pubmed Central) - Jan 6, 2021
This problem is exacerbated by the tendency of most clinical trials to select patients with a stable, but severe disease. Future trials should: select patients with weakness and fatigability that is completely explained by their myasthenia gravis, use a design that avoids the exclusion of patients with recent changes in medication, and explore the possibilities to completely avoid the use of corticosteroids.
- |||||||||| Review, Journal: Update on immune-mediated therapies for myasthenia gravis. (Pubmed Central) - Jan 5, 2021
Herein, we discuss these advances and the potential for these newer therapies to significantly influence the current treatment paradigms. While these therapies provide exciting new options with rapid efficacy, there are anticipated challenges to their use, especially in terms of a dramatic increase in cost of care for some patients with myasthenia gravis.
- |||||||||| Review, Journal: Snake three-finger α-neurotoxins and nicotinic acetylcholine receptors: molecules, mechanisms and medicine. (Pubmed Central) - Jan 5, 2021
The pharmacology of snake α-3FNTxs including their mechanisms of neuromuscular blockade, variations in reversibility of nAChR interactions, specificity for nAChR subtypes or for distinct ligand-binding interfaces within a subtype and the role of α-3FNTxs in neurotoxic envenomation are also detailed. Lastly, a reconciliation of structure-function relationships between α-3FNTx and nAChRs, derived from historical mutational and biochemical studies and emerging atomic level structures of nAChR models in complex with α-3FNTxs is discussed.
- |||||||||| Journal, Adverse events: Diagnosis and management of immune-related adverse effects of immune checkpoint therapy in the emergency department. (Pubmed Central) - Jan 5, 2021
We also suggest a workflow for the recognition and treatment of emergencies arising from serious immune-related adverse effects, including but not limited to colitis, adrenal crisis, myocarditis, pneumonitis, myasthenic crisis, diabetic ketoacidosis, bullous pemphigus, and hemophagocytic lymphohistiocytosis. Rapid advances in cancer therapy are bringing new diagnostic and therapeutic challenges to emergency providers, and therefore it is crucial to raise awareness and provide guidelines for the management of new treatment-related toxicities.
- |||||||||| Soliris (eculizumab) / Alexion Pharma, Ultomiris IV (ravulizumab IV) / Alexion Pharma
[VIRTUAL] Formation of multivalent complexes in the presence of more than one conventional antibody to complement factor C5 () - Jan 5, 2021 - Abstract #BSHI2020BSH-I_303;
This highlights the importance of rigorous functional characterization of such complexes and incorporation of monitoring and mitigation processes to assess and prevent their potential formation in patients. Other methods of avoiding multivalent complexes would include switching among antibodies recognizing and binding the same epitope, such as switching from eculizumab to ravulizumab, or switching to a monovalent C5 antagonist which is unable to promote the formation of multivalent immune complexes.
- |||||||||| Clinical, Journal: Prognosis in Pediatric Myasthenia Gravis. (Pubmed Central) - Jan 3, 2021
Other methods of avoiding multivalent complexes would include switching among antibodies recognizing and binding the same epitope, such as switching from eculizumab to ravulizumab, or switching to a monovalent C5 antagonist which is unable to promote the formation of multivalent immune complexes. Investigators from Oxford John Radcliff Hospital and Great Ormond Street Hospital for Children performed a retrospective study of myasthenia patients diagnosed before the age of 16 years.
- |||||||||| Review, Journal: Immunoregulatory Cells in Myasthenia Gravis. (Pubmed Central) - Jan 3, 2021
There is an inverse correlation between the frequency of circulating IL-10-producing B cells and clinical status in MG patients. Taken together, both functional and numerical defects in various populations of immunoregulatory cells in EAMG and human MG have been demonstrated, but how they relate to pathogenesis and whether these cells can serve as biomarkers of disease activity in humans deserve further exploration.
- |||||||||| Plasmapheresis Indications : TTP, HUS, Cryoglobulinemia, Hyperviscosity, Myasthenia gravis, Goodpastures, Catastrophic APL Syn, Chr inflamm demyelinating polyneuropathy, Removal of drugs/toxins, Alveolar hemorrhage, ANCA vasculitis (questionable) https://t.co/8TBExIx0Kx (Twitter) - Jan 2, 2021
- |||||||||| Yervoy (ipilimumab) / Ono Pharma, BMS
Journal, Adverse events, Checkpoint inhibition: Diagnosis and Differential Diagnosis of Neurological Adverse Events during Immune Checkpoint Inhibitor Therapy. (Pubmed Central) - Jan 1, 2021
Since the approval of the first ICI, ipilimumab, for treatment of advanced melanoma by the Food and Drug Administration (FDA) in 2011, the spectrum of indications and approved ICIs has grown, rapidly...Patients with malignancy are predominantly not treated by neurologists. Because of the complexity of neurological symptoms, detailed neurological investigations in specialized institutions are necessary in patients with new neurological symptoms and need to be critically discussed with treating oncologists.
- |||||||||| Journal: Myasthenia Gravis Associated With SARS-CoV-2 Infection. (Pubmed Central) - Dec 31, 2020
Because of the complexity of neurological symptoms, detailed neurological investigations in specialized institutions are necessary in patients with new neurological symptoms and need to be critically discussed with treating oncologists. No abstract available
- |||||||||| Journal: Frequency and features of myasthenia gravis developing after thymectomy. (Pubmed Central) - Dec 30, 2020
Reductions ≤1.5 mg/day from the maintenance dosage should be considered for patients with late-onset disease. Our results suggest that approximately 11% of patients with TAMG were PostTx MG, and presurgical assessment of anti-AChR antibody titer or electrophysiological testing may predict PostTx MG development; however, no difference in clinical manifestation and prognosis is observed between PreTx MG and PostTx MG.
- |||||||||| Clinical, Journal: Chronic Stress, Depression and Personality Type in Patients with Myasthenia Gravis. (Pubmed Central) - Dec 30, 2020
We found a significant association of MG severity with depression and chronic stress, as well as with female gender. These findings should raise awareness that the long-term management of MG should address depression and potential stress, and consider behavioural management to prevent stress-related immune imbalance.
- |||||||||| Review, Journal: Immunopathology of Autoimmune Myasthenia Gravis: Implications for Improved Testing Algorithms and Treatment Strategies. (Pubmed Central) - Dec 29, 2020
We propose a comprehensive "reflex testing," algorithm for the presence of MG autoantibodies, and foresee that in the near future, the convenience and specificity of novel assays will permit the clinicians to consider them into routine systematic testing, thus stimulating laboratories to make these tests available. Moreover, adopting treatment driven testing algorithms will be crucial to identify subgroups of patients potentially benefiting from novel immunotherapies for MG.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: A new era for monoclonal antibodies with applications in neurology (Review). (Pubmed Central) - Dec 29, 2020
These innovative therapies are more effective and can treat previously untreatable diseases. As better understanding of the pathogenic mechanisms of neurological diseases is gained, additional mAbs are expected to be developed at a lower cost and with better safety profile compared with current treatment options.
- |||||||||| Retrospective data, Journal: Long Term Follow-Up on Pediatric Cases With Congenital Myasthenic Syndromes-A Retrospective Single Centre Cohort Study. (Pubmed Central) - Dec 29, 2020
Our findings and comparison with the literature- suggest a better treatment-response and less severe progression of symptoms present in patients suffering from mutations in CMS-genes directly associated with receptor deficiency, while patients with defects leading to synaptopathy and presynaptic defects tend to have worse outcomes. Assessment of affected muscular groups and clinical symptoms by CMS-ST may be a useful tool for optimal therapeutic management of the patients, especially for future clinical studies.
- |||||||||| Soliris (eculizumab) / Alexion Pharma
Review, Journal: Complement Inhibition for the Treatment of Myasthenia Gravis. (Pubmed Central) - Dec 29, 2020
Eculizumab, an antibody directed towards C5, has recently been approved for the treatment of AChR antibody-positive gMG...For such purpose, there is a critical need to identify possible biomarkers predictive of therapeutic response, a field not yet sufficiently explored in MG. This review aims to give an overview of the complement cascade involvement in MG, the evolution of complement-inhibiting therapies and possible biomarkers useful to tailor and monitor complement-directed therapies.
- |||||||||| Bridion (sugammadex) / Merck (MSD)
[VIRTUAL] Sugammadex: drug utilization evaluation (DUE) () - Dec 26, 2020 - Abstract #ASHP2020ASHP_2961;
Further price analysis may be required, but the data collected from this analysis can lead to less overuse and ultimately reduce financial burden. The non-restricted use of sugammadex can contribute significantly to increased costs associated with anesthesia.
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