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- Spatial Association between Myasthenia Gravis and Hazardous Waste Sites: A Statewide Planning and Research Cooperative System (SPARCS) Study (2002-2014) (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2513;
Our results demonstrated that the prevalence of MG is not significantly higher amongst residence areas with increased number of superfund sites. More investigation is needed.
- Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Refractory Myasthenia Gravis Exacerbation associated with Marginal Zone Lymphoma (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2512;
MG is an autoantibody mediated disease which may be a paraneoplastic manifestation of an underlying neoplasm, most commonly a thymoma. Refractory MG exacerbation may respond to treatment of the underlying neoplasm.
- Soliris (eculizumab) / AstraZeneca
Poor response to Eculizumab in Caucasian Patients with Treatment Refractory Generalized Myasthenia Gravis (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2511;
The exact mechanism contributing to poor clinical response to eculizumab in gMG patients remains unclear. Further studies are warranted to undermine the underlying pathogenesis.
- Novel congenital myasthenic syndrome caused by homozygous mutation in 3’-UTR of low-density lipoprotein receptor-related protein 6 (LRP6) gene (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2510;
A 3’-UTR mutation of LRP6 gene decreases its bindings to MIR-300 and MIR-let7a resulting in increased expression of LRP6 and upregulates beta-catenin directly without Wnt-protein binding. The increase of beta-catenin entering nucleus downregulates rapsyn expression.
- Measuring Adverse Event Burden in Myasthenia Gravis: Retrospective Validation of the Adverse Event Unit (AEU) (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2509;
P=N/A
We will also present data about differences in AE burden among MG treatments. Final study results will be presented.
- Opdivo (nivolumab) / Ono Pharma, BMS
Immune Checkpoint Inhibitor Associated LRP4 Positive MG (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2508;
Adverse effects of checkpoint inhibitors include myasthenia gravis which can lead to life-threatening complications at a higher rate of mortality than classical myasthenia gravis, requiring prompt diagnosis and treatment. Greater awareness of complications from this therapy can aid in faster identification, allowing for earlier intervention with discontinuation of immune checkpoint inhibitor therapy and aggressive treatments for myasthenia gravis.
- Concentric Needle Jitter Abnormalities in Patients with Ocular and Generalized MG (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2507;
Patients with MGG and normal jitter had already started treatment, and only one muscle was tested. The analysis of the second muscle showed increased sensitivity in MGO patients.
- hydroxychloroquine / Generic mfg.
Coexisting Myasthenia Gravis and Sjogren's Syndrome Patients at Virginia Commonwealth University (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2506;
In contrast to previous reports, we present 3 cases of coexisting MG and pSS, all of whom have severe disease and none of which harbor AChR binding antibodies. These 3 cases serve to expand the clinical spectrum of these rare overlap syndromes.
- 12-year trend in utilization of IVIG and plasmapheresis following acute myasthenia exacerbation and myasthenic crisis in the United States and their association with outcome. (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2504;
IVIG or PLEX use in AME and MMC increased over the last decade, but despite very strong association with decreased mortality and greater odds of home discharge, both procedures are still very underutilized in the US. Core AME/MMC treatment measures that incorporate these modalities are needed to reduce mortality.
- Real-World Resource Utilization and Productivity Loss among Patients with Myasthenia Gravis in Sweden: A Nationwide Population-based Study (Poster Hall) - Mar 6, 2022 - Abstract #AAN2022AAN_2433;
An important sub-population of patients (e.g., those with MG crisis) may be the intensive users of both inpatient and outpatient care. Future research needs to detail treatment pattern and outcomes in this population.
- Soliris (eculizumab) / AstraZeneca
Real-world effectiveness of eculizumab in generalized myasthenia gravis in Japan: 1-year post-marketing surveillance data () - Mar 6, 2022 - Abstract #AAN2022AAN_2402;
P3
Effectiveness of eculizumab for treatment of AChR Ab+ gMG was confirmed to be rapid and sustained in patients observed for 1 year in real-world clinical practice in Japan, including in patients with MGFA class V disease. No new safety signals were observed.
- Soliris (eculizumab) / AstraZeneca
Real-World Eculizumab Effectiveness on US Patient Outcomes in Myasthenia Gravis (ELEVATE): a Retrospective Chart Review () - Mar 6, 2022 - Abstract #AAN2022AAN_2401;
Results from this study suggest that treatment with eculizumab reduced patients’ treatment burden (shown by the decrease in concomitant prednisone use) and their disease burden (demonstrated by the improvement in MG-ADL score). These results support findings from the REGAIN trial and its open-label extension, and corroborate other real-world studies.
- Pathogenic Th17 Cells in Autoimmune Myasthenia Gravis Exhibit a Pro-inflammatory Transcriptomic Signature that Promotes Disease Pathogenesis () - Mar 6, 2022 - Abstract #AAN2022AAN_2399;
Th17 cells from more severe MG patients also have higher pro-inflammatory cytokines than mild patients. Future investigation is needed to determine whether the unique pathogenic Th17 cell signature could serve as a disease biomarker or target for future therapeutics.
- mycophenolate mofetil / Generic mfg., azathioprine / Generic mfg.
PROMISE-MG: A Multicenter Prospective Observational Comparative Effectiveness Study of Myasthenia Gravis Treatments () - Mar 6, 2022 - Abstract #AAN2022AAN_2291;
Those who received adequate dose/duration of these drugs achieved similar improvement to those who did not. Further analyses will evaluate secondary outcomes, adverse effects of AZT/MMF and the effective doses of these agents in real-world practice.Narayanaswami and Sanders contributed equally
- nipocalimab (M281) / J&J
Serum IgG and Autoantibody Lowering by the Anti-FcRn Monoclonal Antibody, Nipocalimab, Correlates with Improvement in MG-ADL in Generalized Myasthenia Patients () - Mar 6, 2022 - Abstract #AAN2022AAN_2224;
The results of this analysis support both the centrality of anti-neuromuscular junction protein IgG autoantibodies in the pathogenesis of gMG as well as the rapid, dose-dependent and predictable effect of nipocalimab in lowering these pathogenic autoantibodies and induction of clinical improvement in patients with gMG. In addition, the close correlation between serum IgG, anti-AChR and clinical response suggest the potential of using serial serum IgG levels as a biomarker of drug effect in management of gMG patients treated with nipocalimab; this will be further tested in the ongoing Vivacity Phase 3 gMG trial.
- Soliris (eculizumab) / AstraZeneca
Real-world experience of eculizumab treatment for generalized myasthenia gravis: initial data from a registry of patients with generalized myasthenia gravis in the USA () - Mar 6, 2022 - Abstract #AAN2022AAN_2223;
P=N/A
Initial analyses of Registry data showed that MG-ADL total score and MGFA classification improved with eculizumab, and that eculizumab was generally well tolerated. These findings provide additional evidence of the effectiveness of eculizumab for treating gMG in real-world clinical practice.
- Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
Long-term Safety, Tolerability, and Efficacy of Efgartigimod in Patients With Generalized Myasthenia Gravis: Interim Results of the ADAPT+ Study () - Mar 6, 2022 - Abstract #AAN2022AAN_2222;
This analysis suggests long-term treatment with efgartigimod was well-tolerated and efficacious, regardless of antibody status. Despite being conducted during the COVID-19 pandemic, before vaccine availability, no new safety signals were identified.
- Ultomiris IV (ravulizumab IV) / AstraZeneca
Long-term Efficacy and Safety of Ravulizumab, a Long-acting Terminal Complement Inhibitor, in Adults with Anti-Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: Results from the Phase 3 CHAMPION MG Open-label Extension () - Mar 6, 2022 - Abstract #AAN2022AAN_2219;
P3
Despite being conducted during the COVID-19 pandemic, before vaccine availability, no new safety signals were identified. Ravulizumab, administered every 8 weeks, demonstrated sustained improvements in symptoms and was well tolerated for up to 1 year in adults with AChR Ab+ gMG.
- Use of Repetitive Nerve Stimulation in the Geisinger Health System Neurophysiology Laboratory ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_2110;
The yield of RNS was relatively low. The biggest opportunity for improvement is the creation of a system-based stepwise approach for the evaluation of MG, which includes testing AChR Ab before RNS
- Pregnancy course and Musk-positive myasthenia gravis: a single center case series ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1999;
Among our patients, 4 required rescue therapy; pregnancy and delivery were not otherwise complicated by MG except for 1 patient who required C-section due to failure of delivery progression. Further study is needed to understand the mechanism and risk of worsening of MG during pregnancy or postpartum in MMG.
- A Novel Interdisciplinary Education Tool on Neuromuscular Respiratory Failure ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1952;
This educational tool is an easily distributable tool to address a training gap in critical care fellowship training. Our educational curriculum is designed for multidisciplinary use, and can be expanded to educate other physician groups, as well as advanced practice providers.
- nipocalimab (M281) / J&J
Nipocalimab’s Selective Targeting of FcRn and IgG Clearance Preserves Key Immune Functions ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1949;
P1, P2
These data suggest that nipocalimab can selectively lower IgG and IgG autoantibodies while preserving cellular immunity, complete IgM response and IgG production after neoantigen challenge. Overall, nipocalimab’s selective effect on IgG recycling provides a mechanistic rationale for potentially decreased infection risk despite substantial IgG lowering.
- Neuromuscular Manifestations of COVID-19 ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1945;
This finding may be explained by more COVID-19 patients being in the intensive care unit and bedbound for longer periods. It is worth noting that a small case series of COVID-related critical illness neuropathy and myopathy patients showed no histopathological or clinical differences compared to non-COVID patients.
- hydroxychloroquine / Generic mfg.
Management and Outcomes of Hospitalized Patients with Myasthenia Gravis Infected with COVID-19: A Literature Review ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1900;
MG patients with severe COVID-19 infections have high rates of MV (47%) but favorable outcomes with regards to mortality (10%). Unsurprisingly, treatment with AZM and HCQ was associated with higher rates of MV.
- carboplatin / Generic mfg., etoposide IV / Generic mfg.
Lambert-Eaton Myasthenic Syndrome and Paraneoplastic Cerebellar Degeneration as Harbingers of Merkel Cell Carcinoma with Unknown Primary: A Rare Case Presentation. ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1882;
Neurologic findings such as cerebellar ataxia and LEMS without clear etiology should prompt a workup for a paraneoplastic syndrome which may precede tumor diagnosis. MCC can present as occult lymph node involvement without primary cutaneous findings.
- mycophenolate mofetil / Generic mfg., azathioprine / Generic mfg.
Incidence of Skin Changes in Patients with Myasthenia Gravis Prescribed Mycophenolate mofetil or Azathioprine ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1866;
MCC can present as occult lymph node involvement without primary cutaneous findings. Based on this large MG cohort survey study we found no difference of skin lesion incidence in patients exposed to MMF or AZA compared to the non-exposed group.
- Impact of COVID-19 and vaccines in myasthenia gravis: experience from an Italian cohort ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1857;
The reduced number of adverse events in our population suggests that vaccines for SARS-cov2 are safe in myasthenic patients that could take advantage of vaccination avoiding life-threatening complications such as myasthenic crisis and COVID-19 pneumonia. The continuation of the regular and periodic clinical follow-up will provide us data on the real effectiveness of vaccine prevention in the myasthenic population
- High-Resolution Manometry is Useful for Diagnosing Myasthenia Gravis with Mild Dysphagia ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1841;
Swallowing pressure assessment with HRM during EC test may be helpful in diagnosing mild bulbar symptoms in MG patients. Further study with more patients is needed.
- Ultomiris IV (ravulizumab IV) / AstraZeneca
Efficacy and Safety of Ravulizumab, a Long-acting Terminal Complement Inhibitor, in Adults with Anti-Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: Results from the Phase 3 CHAMPION MG Study () - Mar 6, 2022 - Abstract #AAN2022AAN_1801;
P3
Further study with more patients is needed. Ravulizumab, administered every 8 weeks, provided rapid and sustained improvement in symptoms and was well tolerated in adult patients with AChR Ab+ gMG.
- Diagnostic Approach to Cranial Nerve III Palsy ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1778;
Ravulizumab, administered every 8 weeks, provided rapid and sustained improvement in symptoms and was well tolerated in adult patients with AChR Ab+ gMG. Overall, we have identified patterns that can assist in determining common or even rare causes of cranial nerve III palsy, with the hopes to help guide clinicians with their diagnostic workup.
- Soliris (eculizumab) / AstraZeneca, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
Cost-Effectiveness of Eculizumab and Efgartigimod for the Treatment of generalized Myasthenia Gravis ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1753;
Overall, we have identified patterns that can assist in determining common or even rare causes of cranial nerve III palsy, with the hopes to help guide clinicians with their diagnostic workup. Eculizumab and efgartigimod, using the annual placeholder price, exceed typical willingness-to-pay thresholds which may result in limited patient access.
- Clinical Delineation of Myasthenia Gravis in the Kingdom of Bahrain ([VIRTUAL]) - Mar 6, 2022 - Abstract #AAN2022AAN_1735;
The majority of patients had EOMG with ocular disease and AChR positivity. The clinical outcomes were favourable.
- | Journal: Newly diagnosed multiple sclerosis in a patient with ocular myasthenia gravis: A case report. (Pubmed Central) - Mar 5, 2022
When optic neuritis occurs in patients with myasthenia gravis, careful evaluation is necessary while considering the possibility that it may be the first symptom of a demyelinating central nervous system disease. Therefore, it is important to conduct shorter-interval monitoring and symptom screening for patients with neurological autoimmune diseases, such as myasthenia gravis, even if multiple sclerosis is not initially suspected, to achieve early detection of multiple sclerosis.
- || Clinical, Journal: Mass spectrometry glycophenotype characterization of ALG2-CDG in Argentinean patients with a new genetic variant in homozygosis. (Pubmed Central) - Mar 4, 2022
Previous studies reported on slightly increased concentrations of abnormal serum N-glycans in CDG-I due to defects in the mannosylation steps of dolichol-linked oligosaccharide biosynthesis. This preliminary work aims at considering serum N-glycan accumulation of high mannosylated glycoforms, such as oligomannose and hybrid type N-glycans, as potential diagnostic signals for ALG2-CDG patients.
- | Journal: Therapeutic plasma exchange in neurological diseases: Eleven years experience a tertiary care center in Turkey. (Pubmed Central) - Mar 4, 2022
This preliminary work aims at considering serum N-glycan accumulation of high mannosylated glycoforms, such as oligomannose and hybrid type N-glycans, as potential diagnostic signals for ALG2-CDG patients. TPE was safe and tolerable, with manageable complications in experienced hands.
- || Clinical, Observational data, Journal: COVID-19 pandemic year in a sample of Polish myasthenia gravis patients: an observational study. (Pubmed Central) - Mar 4, 2022
In the presented sample of Polish MG patients, favourable outcomes of COVID-19 were observed. Further studies are needed to evaluate the reliable course of COVID-19 taking into account international differences, the types of treatment applied, and the ratio of vaccinated to unvaccinated MG patients.
- | Biomarker, Retrospective data, Journal: Predictors of outcome in patients with myasthenic crisis undergoing non-invasive mechanical ventilation: A retrospective 20 year longitudinal cohort study from a single Italian center. (Pubmed Central) - Mar 4, 2022
Nine patients underwent tracheostomy which was significantly related to male gender. Comorbidities had significant effect on length of ICU .Our study confirms as predictors of prognosis in our patients male gender, older age at onset, infections as trigger, pneumonia.
- | Biomarker, Journal: The role of Serum Free Light Chain as Biomarker of Myasthenia Gravis. (Pubmed Central) - Mar 4, 2022
Comorbidities had significant effect on length of ICU .Our study confirms as predictors of prognosis in our patients male gender, older age at onset, infections as trigger, pneumonia. Increased levels of κFLC and total FLC could serve as biomarkers to support the diagnosis of MG.
- || efgartigimod SC (ARGX-113 SC) / argenx, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
Review, Journal: Efgartigimod: First Approval. (Pubmed Central) - Mar 4, 2022
Several clinical studies of intravenous and subcutaneous formulation of efgartigimod are also being investigated for other autoimmune diseases including bullous pemphigoid, chronic inflammatory demyelinating polyradiculoneuropathy, immune thrombocytopenia, autoimmune myositis and pemphigus. This article summarizes the milestones in the development of efgartigimod leading to this first approval for generalized myasthenia gravis.
- || Clinical, Review, Journal: Statin-associated myasthenia: A case report and literature review. (Pubmed Central) - Mar 3, 2022
This article summarizes the milestones in the development of efgartigimod leading to this first approval for generalized myasthenia gravis. No abstract available
- || Clinical, Journal: Tobramycine eye drops and ocular myasthenia gravis reactivation: A case report. (Pubmed Central) - Mar 3, 2022
No abstract available No abstract available
- | Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Journal, Heterogeneity: Heterogeneity in myasthenia gravis: considerations for disease management. (Pubmed Central) - Mar 3, 2022
Although reliable biomarkers predictive of clinical and biologic outcome are still lacking, recent developments promise a more effective and safe treatment. Disease subtyping according to serological testing and immunopathology is crucial to the appropriateness of clinical management.
- | Preclinical, Journal: A Targeted Complement Inhibitor CRIg/FH Protects Against Experimental Autoimmune Myasthenia Gravis in Rats via Immune Modulation. (Pubmed Central) - Mar 3, 2022
In the active EAMG model, we also found that CRIg/FH treatment remarkably reduced the serum concentration of autoantibodies and of the cytokines including IFN-γ, IL-2, IL-6, and IL-17, and upregulated the percentage of Treg cells in the spleen, which was further verified in vitro. Therefore, our findings indicate that CRIg/FH may hold the potential for the treatment of MG via immune modulation.
- | Firdapse (amifampridine) / BioMarin, Catalyst Pharma
Journal: Lambert-Eaton myasthenic syndrome in a young girl. (Pubmed Central) - Mar 3, 2022
She received plasma exchange electrophoresis, intravenous immunoglobulin, oral steroids and amifampridine with some clinical improvement. We present a case of a child with a demonstrated autoimmune propensity and no evidence of malignancy, thereby adding to the 12 previously reported cases of LEMS in children.
- |||| nipocalimab (M281) / J&J
New P2/3 trial: A Study of Nipocalimab in Children Aged 2 to Less Than 18 Years With Generalized Myasthenia Gravis (clinicaltrials.gov) - Mar 2, 2022
P2/3, N=12, Not yet recruiting, Sponsor: Janssen Research & Development, LLC
- |||||| cemdisiran (ALN-CC5) / Alnylam, Regeneron, Veopoz (pozelimab-bbfg) / Regeneron
Enrollment open, Trial primary completion date, Combination therapy, Monotherapy: NIMBLE: A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis (clinicaltrials.gov) - Mar 2, 2022
P3, N=210, Recruiting, Sponsor: Regeneron Pharmaceuticals
We present a case of a child with a demonstrated autoimmune propensity and no evidence of malignancy, thereby adding to the 12 previously reported cases of LEMS in children. Not yet recruiting --> Recruiting | Trial primary completion date: Feb 2024 --> Aug 2024
- | New trial, HEOR, Surgery: RATS: Comparison of Single Port and Two Ports Robotic Assisted Thoracic Surgery for Thymectomy (clinicaltrials.gov) - Mar 2, 2022
P=N/A, N=60, Not yet recruiting, Sponsor: Shanghai Pulmonary Hospital, Shanghai, China
- || Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
Clinical, Journal: Clinical experience with maintenance therapeutic plasma exchange in refractory generalized myasthenia gravis. (Pubmed Central) - Mar 1, 2022
Not yet recruiting --> Recruiting | Trial primary completion date: Feb 2024 --> Aug 2024 Over a period of 12 months, maintenance TPE therapy improved MG-ADL, and MGC with decreased immunosuppressant requirement, while being well-tolerated.
- ||| Stivarga (regorafenib) / Bayer
Clinical, Journal, Adverse events: Anti-PD-1 and regorafenib induce severe multisystem adverse events in microsatellite stability metastatic colorectal cancer: a case report. (Pubmed Central) - Mar 1, 2022
Though evidence of tumor shrinkage was not found, CEA levels markedly decreased. Therefore, anti-PD-1 plus regorafenib might be optional for the MSS mCRC patients which requires special caution in the clinical practice.
- | Journal: Altered expression of fragile X mental retardation-1 (FMR1) in the thymus in autoimmune myasthenia gravis. (Pubmed Central) - Mar 1, 2022
First, in relation with the higher susceptibility of females to autoimmune diseases. Second, due to the modulation of its expression by inflammatory signals that are known to be altered in MG thymuses.