2022
Heterogeneity of Acetylcholine Receptor Autoantibody–Mediated Complement Activity in Patients With Myasthenia Gravis
Obaid AH, Zografou C, Vadysirisack DD, Munro-Sheldon B, Fichtner ML, Roy B, Philbrick WM, Bennett JL, Nowak RJ, O'Connor KC. Heterogeneity of Acetylcholine Receptor Autoantibody–Mediated Complement Activity in Patients With Myasthenia Gravis. Neurology Neuroimmunology & Neuroinflammation 2022, 9: e1169. PMID: 35473886, PMCID: PMC9128035, DOI: 10.1212/nxi.0000000000001169.Peer-Reviewed Original ResearchConceptsAChR autoantibodiesMyasthenia gravisDisease burdenComplement activityAcetylcholine receptor autoantibodiesAChR-MG patientsComplement inhibitor therapyLower autoantibody levelsClinical disease scoresSubset of patientsHealthy donor samplesCell-based assaysMost patient samplesMembrane attack complex formationModest positive associationMG subtypesAutoantibody levelsAutoantibody titersComplement membrane attack complex formationMG patientsInhibitor therapyReceptor autoantibodiesHD groupTherapeutic responseMAC formationThe clinical need for clustered AChR cell-based assay testing of seronegative MG
Masi G, Li Y, Karatz T, Pham MC, Oxendine SR, Nowak RJ, Guptill JT, O'Connor KC. The clinical need for clustered AChR cell-based assay testing of seronegative MG. Journal Of Neuroimmunology 2022, 367: 577850. PMID: 35366559, PMCID: PMC9106915, DOI: 10.1016/j.jneuroim.2022.577850.Peer-Reviewed Original ResearchConceptsSNMG patientsMyasthenia gravisAChR-specific B cellsClinical needAcetylcholine receptor autoantibodiesSeronegative MG patientsSeronegative myasthenia gravisCell-based assaysAutoantibody positivityTrial eligibilityMG patientsReceptor autoantibodiesPatientsB cellsU.S. CentersNew treatmentsAssaysGravisAutoantibodiesSerostatusAChRPositivityMyasthenia gravis complement activity is independent of autoantibody titer and disease severity
Fichtner ML, Hoarty MD, Vadysirisack DD, Munro-Sheldon B, Nowak RJ, O’Connor K. Myasthenia gravis complement activity is independent of autoantibody titer and disease severity. PLOS ONE 2022, 17: e0264489. PMID: 35290370, PMCID: PMC8923450, DOI: 10.1371/journal.pone.0264489.Peer-Reviewed Original ResearchConceptsAutoantibody titersComplement activityDisease activityMyasthenia gravisComplement pathwayAcetylcholine receptor autoantibodiesAutoimmune myasthenia gravisSecondary complement deficiencyClassical complement pathwayAChR autoantibodiesReceptor autoantibodiesClinical statusAutoimmune diseasesHealthy controlsComplement deficiencyPatients associatesStudy subjectsCandidate biomarkersDisease statusDisease severitySignificant associationDisease pathologyTitersAutoantibodiesPatients
2020
Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis
Howard JF, Nowak RJ, Wolfe GI, Freimer ML, Vu TH, Hinton JL, Benatar M, Duda PW, MacDougall JE, Farzaneh-Far R, Kaminski HJ, Barohn R, Dimachkie M, Pasnoor M, Farmakidis C, Liu T, Colgan S, Benatar M, Bertorini T, Pillai R, Henegar R, Bromberg M, Gibson S, Janecki T, Freimer M, Elsheikh B, Matisak P, Genge A, Guidon A, David W, Habib A, Mathew V, Mozaffar T, Hinton J, Hewitt W, Barnett D, Sullivan P, Ho D, Howard J, Traub R, Chopra M, Kaminski H, Aly R, Bayat E, Abu-Rub M, Khan S, Lange D, Holzberg S, Khatri B, Lindman E, Olapo T, Sershon L, Lisak R, Bernitsas E, Jia K, Malik R, Lewis-Collins T, Nicolle M, Nowak R, Sharma A, Roy B, Nye J, Pulley M, Berger A, Shabbir Y, Sachdev A, Patterson K, Siddiqi Z, Sivak M, Bratton J, Small G, Kohli A, Fetter M, Vu T, Lam L, Harvey B, Wolfe G, Silvestri N, Patrick K, Zakalik K, Duda P, MacDougall J, Farzaneh-Far R, Pontius A, Hoarty M. Clinical Effects of the Self-administered Subcutaneous Complement Inhibitor Zilucoplan in Patients With Moderate to Severe Generalized Myasthenia Gravis. JAMA Neurology 2020, 77: 582-592. PMID: 32065623, PMCID: PMC7042797, DOI: 10.1001/jamaneurol.2019.5125.Peer-Reviewed Original ResearchConceptsGeneralized myasthenia gravisSecondary end pointsDaily living scoreTolerability profileEnd pointLiving scoreMyasthenia gravisClinical effectsPlacebo-controlled phase 2 clinical trialKey secondary efficacy end pointsKey secondary end pointSecondary efficacy end pointsSevere Generalized Myasthenia GravisQuantitative Myasthenia Gravis ScorePhase 2 clinical trialAcetylcholine receptor autoantibodiesEfficacy end pointMyasthenia gravis scoreMG activityComplement component 5Disease-specific variablesQuality of lifeComplete complement inhibitionBroader populationDaily SCDifferential response to rituximab in anti-AChR and anti-MuSK positive myasthenia gravis patients: a single-center retrospective study
Litchman T, Roy B, Kumar A, Sharma A, Njike V, Nowak RJ. Differential response to rituximab in anti-AChR and anti-MuSK positive myasthenia gravis patients: a single-center retrospective study. Journal Of The Neurological Sciences 2020, 411: 116690. PMID: 32028072, DOI: 10.1016/j.jns.2020.116690.Peer-Reviewed Original ResearchConceptsMyasthenia gravisMGFA classRetrospective studyPositive myasthenia gravis patientsSingle-center retrospective studyManagement of MGAcetylcholine receptor autoantibodiesRefractory myasthenia gravisSymptom-free stateB-cell depletionMyasthenia gravis patientsClinical remissionClinical improvementDurable responsesMG patientsGravis patientsReceptor autoantibodiesMore hospitalizationsClinical symptomsExamination findingsCell depletionTreatment responsePatientsRituximabB cells
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