Myasthenia Gravis
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  • ||||||||||  Journal:  The clinical use of impulse oscillometry in neuromuscular diseases. (Pubmed Central) -  Sep 14, 2022   
    Clinically reported dysphagia, a decreased capability of coughing, and shortness of breath in patients with NMD make R increase in general, but X parameters, which mainly express rib cage elasticity, turn more negative. In patients with NMD, IOS monitoring may help in evaluating the regression in respiratory functions, however, future studies are needed to understand more.
  • ||||||||||  Journal:  Anemia in female patients with myasthenia gravis. (Pubmed Central) -  Sep 14, 2022   
    Long term immunosuppressive therapy can cause anemia in female MG patients. Anemia may negatively affect the quality of life of female MG patients.
  • ||||||||||  Clinical, Journal:  High-dose vitamin C therapy for symptomatic deficiency in a patient with myasthenia gravis and Crohn's disease. (Pubmed Central) -  Sep 12, 2022   
    In contrast, our patient reported a regular intake of vitamin C-containing foods, in addition to oral supplementation, but continued to have difficulty maintaining normal vitamin C levels. Scurvy should be considered for any patient with symptoms of bleeding, petechiae, or ecchymosis and, although it can typically be treated with oral vitamin C, intravenous repletion may be necessary in some cases.
  • ||||||||||  Retrospective data, Journal, Real-world evidence:  Thymectomy in myasthenia gravis: "the real world" experience beyond studies. (Pubmed Central) -  Sep 12, 2022   
    Scurvy should be considered for any patient with symptoms of bleeding, petechiae, or ecchymosis and, although it can typically be treated with oral vitamin C, intravenous repletion may be necessary in some cases. No abstract available
  • ||||||||||  RARE PRESENTATION OF A THYMOMA AT AN INFREQUENT LOCATION (Convention Center Exhibit Hall: Poster Area) -  Sep 11, 2022 - Abstract #CHEST2022CHEST_4028;    
    No abstract available Thymoma can present with features of mass effect, hormonal or autoimmune effect.
  • ||||||||||  prednisone / Generic mfg.
    WEST NILE-INDUCED MYASTHENIA GRAVIS CRISIS: AN UNEXPECTED CASE OF RESPIRATORY FAILURE (Convention Center Exhibit Hall: Rapid Area 3D) -  Sep 11, 2022 - Abstract #CHEST2022CHEST_3843;    
    The differential diagnoses for dysphagia and acute respiratory failure should be broad with a high degree of clinical suspicion for underlying neuromuscular disorders, especially in older age groups. It is prudent that providers remain vigilant about other potential rare etiologies of MG, as this disease has favorable outcomes if identified and treated appropriately.
  • ||||||||||  amphotericin b liposome / Generic mfg., voriconazole / Generic mfg.
    INVASIVE PULMONARY FUSARIOSIS WITH SUPERIMPOSED HISTOPLASMOSIS IN AN IMMUNOCOMPETENT HOST (Convention Center Exhibit Hall: Rapid Area 1C) -  Sep 11, 2022 - Abstract #CHEST2022CHEST_3578;    
    Therefore, intravenous liposomal Amphotericin B was given for 2 weeks followed by a long-course of oral Voriconazole resulting in marked improvement of symptoms, yet he remained with limited physical activity due to exertional hypoxia of less than 80%. Regardless of the immunologic status, invasive fungal pneumonia should be considered in patients with prolonged environmental exposure and non-specific chest imaging abnormalities.
  • ||||||||||  ADULT-ONSET CENTRAL HYPOVENTILATION SYNDROME: A CASE OF ONDINE'S CURSE (Convention Center Exhibit Hall: Rapid Area 3C) -  Sep 11, 2022 - Abstract #CHEST2022CHEST_3559;    
    In cases with persistent hypercapnic respiratory failure unexplained by extensive diagnostic work up, clinicians should investigate for Ondine's curse/CHS especially in patients with family history of hypercapnic respiratory failure. Prompt institution of NIV may result in decreased morbidity and mortality.
  • ||||||||||  Comirnaty (tozinameran) / Pfizer, Fosun Pharma, BioNTech
    PROGRESSIVE ASCENDING WEAKNESS AFTER THE PFIZER/BIONTECH COVID-19 VACCINE (Convention Center Exhibit Hall: Rapid Area 1B) -  Sep 11, 2022 - Abstract #CHEST2022CHEST_3298;    
    Recognizing this risk will open discussions about vaccine safety. In doing so, we can begin to formulate new parameters for post-vaccination monitoring.
  • ||||||||||  thyroxine / Generic mfg.
    CARDIAC TAMPONADE WITHOUT THE BECK TRIAD: A COMPLICATION OF SEVERE HYPOTHYROIDISM (Convention Center Exhibit Hall: Rapid Area 1B) -  Sep 11, 2022 - Abstract #CHEST2022CHEST_3296;    
    This case illustrates the importance of measuring thyroid-stimulating hormone and considering hypothyroidism as a potential cause of cardiac tamponade. Compliance with levothyroxine plays a crucial role in preventing and managing pericardial effusion in hypothyroidism.
  • ||||||||||  tremelimumab (CP-675206) / AstraZeneca, Pfizer, Imfinzi (durvalumab) / AstraZeneca
    P1 data, Journal, Combination therapy, Monotherapy:  Phase I Study of Tremelimumab Monotherapy or in Combination With Durvalumab in Japanese Patients With Advanced Solid Tumors or Malignant Mesothelioma. (Pubmed Central) -  Sep 10, 2022   
    P1
    In conclusion, these results suggested that there was no concern requiring additional precautions for the safety of nivolumab in Japanese patients with melanoma other than the safety information in the Risk Management Plan. Tremelimumab 10 mg/kg (Q4W/Q12W), tremelimumab 1 mg/kg (Q4W) plus durvalumab 20 mg/kg (Q4W/10 mg/kg Q2W), and fixed-dose tremelimumab 75 mg (Q4W) plus durvalumab 1500 mg (Q4W) were safe and tolerable.ClinicalTrials.gov Identifier: NCT02141347 (https://clinicaltrials.gov/ct2/show/NCT02141347).
  • ||||||||||  Bridion (sugammadex) / Merck (MSD)
    Retrospective data, Journal:  Perioperative Management of Patients With Myasthenia Gravis Undergoing Robotic-Assisted Thymectomy-A Retrospective Analysis and Clinical Evaluation. (Pubmed Central) -  Sep 10, 2022   
    Tremelimumab 10 mg/kg (Q4W/Q12W), tremelimumab 1 mg/kg (Q4W) plus durvalumab 20 mg/kg (Q4W/10 mg/kg Q2W), and fixed-dose tremelimumab 75 mg (Q4W) plus durvalumab 1500 mg (Q4W) were safe and tolerable.ClinicalTrials.gov Identifier: NCT02141347 (https://clinicaltrials.gov/ct2/show/NCT02141347). After careful patient selection, planning, and postoperative patient evaluation, robotic-assisted thymectomy can be performed safely without postoperative surveillance in an ICU.
  • ||||||||||  Journal, IO biomarker:  Novel pathophysiological insights in autoimmune myasthenia gravis. (Pubmed Central) -  Sep 10, 2022   
    Recent advances in the understanding of autoantibody functionalities are bringing neuroimmunologists closer to a more detailed appreciation of the mechanisms that govern MG pathology. Future investigations on the immunological heterogeneity among MG patients will be key to developing effective, individually tailored therapies.
  • ||||||||||  Journal:  Essen transition model for neuromuscular diseases. (Pubmed Central) -  Sep 7, 2022   
    The machine learning-based CT image had significant values in the prediction of different histological classification and even invasion level. The Essen Transition Model aims to close the gap in care for young patients with neuromuscular diseases during the critical transition from pediatric to adult medicine and to create a successful continuation of treatment in adulthood.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca
    Ernest Beutler Lecture and Prize (ENMCC - Hall E) -  Sep 4, 2022 - Abstract #ASH2022ASH_402;    
    Twenty years ago, the first successful trial of anti-complement therapy was performed in PNH subsequently leading in 2007 to the FDA approval of eculizumab, a C5 inhibitor...Peter Hillmen, MD, PhD, of University of Leeds School of Medicine, will describe the development of anti-complement therapy and the recent developments in complement inhibition in PNH. He will give insights into the recent development of alternative complement inhibitors and their potential roles across medicine.
  • ||||||||||  Soliris (eculizumab) / AstraZeneca
    ERNEST BEUTLER LECTURE AND PRIZE: The Complement System and Medicine: the Good, the Bad, the Future () -  Sep 4, 2022 - Abstract #ASH2022ASH_186;    
    This not only presented a dramatic change in the management of PNH but also paved the way for the approval of eculizumab in other diseases including aHUS, myasthenia gravis and neuromyelitis optica spectrum disorder. In addition, this early experience heralded the development of other complement inhibitors in many other diseases.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    Severe Transplantation-Mediated Alloimmune Thrombocytopenia After Liver Transplant Treated Successfully With Efgartigimod (Crown Ballroom) -  Sep 4, 2022 - Abstract #ACG2022ACG_4000;    
    Although TMAT generally resolves a few months after transplantation as donor lymphocytes are cleared from the circulation, initial management is challenging. Efgartigimod is a novel treatment approved by the food and drug administration for myasthenia gravis that leads to degradation of IgG by binding to the neonatal Fc receptor and should be considered for severe TMAT.
  • ||||||||||  Obstipation Masquerading as Myasthenia Gravis (Crown Ballroom) -  Sep 4, 2022 - Abstract #ACG2022ACG_3860;    
    It is hard to explain that despite his cardinal symptoms of MG, his neuropathy resolved not with immunosuppression, but with disimpaction. Figure: A.Computed tomography of the abdomen and pelvis without contrast (coronal view) showed extensive colonic distention with fecal impaction B.Computed tomography of the abdomen and pelvis without contrast (axial view) showing moderate colonic distention seen with transition point in the distal sigmoid colon with increased stool burden C.Computed tomography of the abdomen and pelvis without contrast (sagittal view) showing moderate colonic distention seen with transition point in the distal sigmoid colon with increased stool burden
  • ||||||||||  dexamethasone / Generic mfg., trimethoprim/sulfamethoxazole / Generic mfg.
    Abnormal Liver Enzymes in Thymoma-Associated Multiorgan Autoimmunity (Crown Ballroom) -  Sep 4, 2022 - Abstract #ACG2022ACG_777;    
    He had recently been hospitalized for methicillin-resistant Staphylococcus aureus aspiration pneumonia treated with trimethoprim-sulfamethoxazole (TMP-SMX)...Following multidisciplinary discussion, the patient received antithyroglobulin (ATG) and dexamethasone for TAMA...D. Liver biopsy with interlobular bile ducts surrounded and infiltrated by lymphocytes, monocytes, and a few neutrophils, reflective of a cellular type IV reaction targeting the bile duct epithelium with resultant cholestasis
  • ||||||||||  Spikevax (elasomeran) / Moderna
    Journal:  Myasthenia Gravis Presenting after Administration of the mRNA-1273 Vaccine. (Pubmed Central) -  Sep 3, 2022   
    Esophageal biopsy significant for benign squamous mucosa (top) with underlying squamous cell carcinoma (arrow) (20X, H&E) (D). We present a case of newly diagnosed myasthenia gravis after administration of the mRNA-1273 SARS-CoV-2 vaccine.mRNA-1273 vaccination against SARS-CoV-2 may exacerbate subclinical cases of myasthenia gravis.Recognition of new vaccine side effects may guide the selection of vulnerable patients.
  • ||||||||||  Journal:  Effects of 3,4-diaminopyridine on myasthenia gravis: Preliminary results of an open-label study. (Pubmed Central) -  Sep 3, 2022   
    A direct effect of 3,4-DAP on nicotinic ACh receptors (nAChRs) was excluded since human nAChRs reconstituted in an expression system, which were not affected by 3,4-DAP application. Our results suggest that 3,4-DAP may be a useful add-on therapy, especially in most severe patients or when immunosuppressive treatment has not yet reached its full effect or when significant side-effects are associated with anticholinesterase.
  • ||||||||||  Review, Journal, PD(L)-1 Biomarker, IO biomarker:  Full-length recombinant antibodies from Escherichia coli: production, characterization, effector function (Fc) engineering, and clinical evaluation. (Pubmed Central) -  Sep 3, 2022   
    Extensive engineering of the Fc domain of aglycosylated antibodies enables recruitment of various effector functions, despite the lack of N-linked glycans. This review summarizes recent research, preclinical advancements, and clinical development of E. coli-produced aglycosylated therapeutic antibodies as monoclonal, bispecific, and antibody-drug conjugates for use in autoimmune, oncology, and immuno-oncology areas.Abbreviations: ADA Anti-drug antibody; ADCC Antibody-dependent cellular cytotoxicity; ADCP Antibody-dependent cellular phagocytosis; ADC Antibody-drug conjugate; aFc Aglycosylated Fc; AMD Age-related macular degeneration aTTP Acquired thrombotic thrombocytopenic purpura; BCMA B-cell maturation antigen; BLA Biologics license application; BsAb Bispecific antibody; C1q Complement protein C1q; CDC Complement-dependent cytotoxicity; CDCC Complement-dependent cellular cytotoxicity; CDCP Complement-dependent cellular phagocytosis; CEX Cation exchange chromatography; CFPS Cell-free protein expression; CHO Chinese Hamster Ovary; CH1-3 Constant heavy chain 1-3; CL Constant light chain; DLBCL Diffuse large B-cell lymphoma; DAR Drug antibody ratio; DC Dendritic cell; dsFv Disulfide-stabilized Fv; EU European Union; EGFR Epidermal growth factor receptor; E. coli Escherichia coli; EpCAM Epithelial cell adhesion molecule; Fab Fragment antigen binding; FACS Fluorescence activated cell sorting; Fc Fragment crystallizable; FcRn Neonatal Fc receptor; FcɣRs Fc gamma receptors; FDA Food and Drug Administration; FL-IgG Full-length immunoglobulin; Fv Fragment variable; FolRαa Folate receptor alpha; gFc Glycosylated Fc; GM-CSF Granulocyte macrophage-colony stimulating factor; GPx7 Human peroxidase 7; HCL Hairy cell leukemia; HIV Human immunodeficiency virusl; HER2 Human epidermal growth factor receptor 2; HGF Hepatocyte growth factor; HIC Hydrophobic interaction chromatography; HLA Human leukocyte antigen; IBs Inclusion bodies; IgG1-4 Immunoglobulin 1-4; IP Intraperitoneal; ITC Isothermal titration calorimetry; ITP Immune thrombocytopenia; IV Intravenous; kDa Kilodalton; KiH Knob-into-Hole; mAb Monoclonal antibody; MAC Membrane-attack complex; mCRC Metastatic colorectal cancer; MM Multipl myeloma; MOA Mechanism of action; MS Mass spectrometry; MUC1 Mucin 1; MG Myasthenia gravis; NB Nanobody; NK Natural killer; nsAA Nonstandard amino acid; NSCLC Non-small cell lung cancer; P. aeruginosa Pseudomonas aeruginosa; PD-1 Programmed cell death 1; PD-L1 Programmed cell death-ligand 1; PDI Protein disulfide isomerase; PECS Periplasmic expression cytometric screening; PK Pharmacokinetics; P. pastoris Pichia pastoris; PTM Post-translational modification; Rg Radius of gyration; RA Rheumatoid arthritis; RT-PCR Reverse transcription polymerase chain reaction; SAXS Small angle X-ray scattering; scF Single chain Fv; SCLC Small cell lung cancer; SDS-PAGE Sodium dodecyl sulfate-polyacrylamide gel electrophoresis; SEC Size exclusion chromatography; SEED Strand-exchange engineered domain; sRNA Small regulatory RNA; SRP Signal recognition particle; T1/2 Half-life; Tagg Aggregation temperature; TCR T cell receptor; TDB T cell-dependent bispecific; TF Tissue factor; TIR Translation initiation region; Tm Melting temperature; TNBC Triple-negative breast cancer; TNF Tumor necrosis factor; TPO Thrombopoietin; VEGF Vascular endothelial growth factor; vH Variable heavy chain; vL Variable light chain; vWF von Willebrand factor; WT Wild type.
  • ||||||||||  tofacitinib / Generic mfg.
    Trial completion date, Trial primary completion date:  Safety And Efficacy Of Tofacitinib In Patients With Refractory Myasthenia Gravis: A Pilot Study (clinicaltrials.gov) -  Sep 2, 2022   
    P1,  N=20, Recruiting, 
    This review summarizes recent research, preclinical advancements, and clinical development of E. coli-produced aglycosylated therapeutic antibodies as monoclonal, bispecific, and antibody-drug conjugates for use in autoimmune, oncology, and immuno-oncology areas.Abbreviations: ADA Anti-drug antibody; ADCC Antibody-dependent cellular cytotoxicity; ADCP Antibody-dependent cellular phagocytosis; ADC Antibody-drug conjugate; aFc Aglycosylated Fc; AMD Age-related macular degeneration aTTP Acquired thrombotic thrombocytopenic purpura; BCMA B-cell maturation antigen; BLA Biologics license application; BsAb Bispecific antibody; C1q Complement protein C1q; CDC Complement-dependent cytotoxicity; CDCC Complement-dependent cellular cytotoxicity; CDCP Complement-dependent cellular phagocytosis; CEX Cation exchange chromatography; CFPS Cell-free protein expression; CHO Chinese Hamster Ovary; CH1-3 Constant heavy chain 1-3; CL Constant light chain; DLBCL Diffuse large B-cell lymphoma; DAR Drug antibody ratio; DC Dendritic cell; dsFv Disulfide-stabilized Fv; EU European Union; EGFR Epidermal growth factor receptor; E. coli Escherichia coli; EpCAM Epithelial cell adhesion molecule; Fab Fragment antigen binding; FACS Fluorescence activated cell sorting; Fc Fragment crystallizable; FcRn Neonatal Fc receptor; FcɣRs Fc gamma receptors; FDA Food and Drug Administration; FL-IgG Full-length immunoglobulin; Fv Fragment variable; FolRαa Folate receptor alpha; gFc Glycosylated Fc; GM-CSF Granulocyte macrophage-colony stimulating factor; GPx7 Human peroxidase 7; HCL Hairy cell leukemia; HIV Human immunodeficiency virusl; HER2 Human epidermal growth factor receptor 2; HGF Hepatocyte growth factor; HIC Hydrophobic interaction chromatography; HLA Human leukocyte antigen; IBs Inclusion bodies; IgG1-4 Immunoglobulin 1-4; IP Intraperitoneal; ITC Isothermal titration calorimetry; ITP Immune thrombocytopenia; IV Intravenous; kDa Kilodalton; KiH Knob-into-Hole; mAb Monoclonal antibody; MAC Membrane-attack complex; mCRC Metastatic colorectal cancer; MM Multipl myeloma; MOA Mechanism of action; MS Mass spectrometry; MUC1 Mucin 1; MG Myasthenia gravis; NB Nanobody; NK Natural killer; nsAA Nonstandard amino acid; NSCLC Non-small cell lung cancer; P. aeruginosa Pseudomonas aeruginosa; PD-1 Programmed cell death 1; PD-L1 Programmed cell death-ligand 1; PDI Protein disulfide isomerase; PECS Periplasmic expression cytometric screening; PK Pharmacokinetics; P. pastoris Pichia pastoris; PTM Post-translational modification; Rg Radius of gyration; RA Rheumatoid arthritis; RT-PCR Reverse transcription polymerase chain reaction; SAXS Small angle X-ray scattering; scF Single chain Fv; SCLC Small cell lung cancer; SDS-PAGE Sodium dodecyl sulfate-polyacrylamide gel electrophoresis; SEC Size exclusion chromatography; SEED Strand-exchange engineered domain; sRNA Small regulatory RNA; SRP Signal recognition particle; T1/2 Half-life; Tagg Aggregation temperature; TCR T cell receptor; TDB T cell-dependent bispecific; TF Tissue factor; TIR Translation initiation region; Tm Melting temperature; TNBC Triple-negative breast cancer; TNF Tumor necrosis factor; TPO Thrombopoietin; VEGF Vascular endothelial growth factor; vH Variable heavy chain; vL Variable light chain; vWF von Willebrand factor; WT Wild type. Trial completion date: Feb 2022 --> Feb 2023 | Trial primary completion date: Feb 2022 --> Feb 2023
  • ||||||||||  rocuronium / Generic mfg.
    Journal:  General anesthesia for treating scoliosis with congenital myasthenia syndrome: a case report. (Pubmed Central) -  Sep 1, 2022   
    As there are only a few reports on the anesthetic management of patients with congenital myasthenia syndrome, we applied the principles of managing autoimmune myasthenia gravis. The postoperative management described herein can prevent respiratory complications in patients with congenital myasthenia syndrome.
  • ||||||||||  Delayed Diagnosis Amongst gMG Patients: Results From a European Real-World Study (In-person; Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1987;    
    Physicians reported patients with diagnoses taking longer than a year experienced more fatigue, anxiety, and prolonged burden on health-related quality of life, leading to higher unnecessary health care resource utilization in patients with gMG. These findings underscore the importance of a timely diagnosis of gMG after symptom onset and the need to properly educate all stakeholders on optimal disease management strategies.
  • ||||||||||  Ultomiris IV (ravulizumab IV) / AstraZeneca, Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio
    A Matching-Adjusted Indirect Comparison of Efgartigimod Versus Ravulizumab for Generalized Myasthenia Gravis (In-person; Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1946;    
    These findings underscore the importance of a timely diagnosis of gMG after symptom onset and the need to properly educate all stakeholders on optimal disease management strategies. The MAIC suggested an improved efficacy with efgartigimod compared with ravulizumab in adult patients with gMG and AChR+.
  • ||||||||||  Vyvgart (efgartigimod alfa-fcab) / argenx, Broteio, Rituxan (rituximab) / Biogen, Zenyaku Kogyo, Roche
    An Indirect Comparison of Efgartigimod Versus Rituximab for Generalized Myasthenia Gravis (In-person; Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1780;    
    The ITC showed improved efficacy of Efgartigimod vs Rituximab. However, due to limited ESS in the MAIC and the strong assumptions underlying the Bucher’s adjusted comparison, these results should be interpreted with caution.
  • ||||||||||  Vitiligo in Italy through Administrative Healthcare Data (Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1764;    
    Vitiligo is a highly undetected and untreated disease. Although this study underestimated its prevalence, Italian administrative healthcare data catched the higher concern among women and quantified the burden on the INHS of patients with a diagnosis of vitiligo and treated with phototherapy.
  • ||||||||||  Challenges in Presenting Engagement Statistics in Real-World Digital Studies (In-person; Virtual) -  Sep 1, 2022 - Abstract #ISPOREU2022ISPOR_EU_1591;    
    Missing data can be an issue across all RWE, particularly for patient-reported data; the data must be correctly interpreted to track participants’ retention and engagement, ensuring they continue to share high-quality data. The concepts of attrition and completion should be separated to better manage digital real-world studies.