2024
United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use
Habib A, Klink A, Muppidi S, Parthan A, Sader S, Balanean A, Gajra A, Nowak R, Howard J. United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use. Journal Of Neurology 2024, 271: 6114-6126. PMID: 39052039, PMCID: PMC11377470, DOI: 10.1007/s00415-024-12569-w.Peer-Reviewed Original ResearchMinimal symptom expression’Minimal manifestation statusGeneralized myasthenia gravisEculizumab initiationEculizumab treatmentTotal scoreMG-ADLPrednisone dosageMyasthenia gravisMethodsA retrospective chart reviewComplement C5 inhibitor eculizumabSteroid-sparing effectAssociated with sustained improvementOpen-label extensionEffectiveness of eculizumabImmunosuppressive therapy useConcomitant medication useRetrospective chart reviewNonsteroidal immunosuppressive therapyMyasthenia Gravis-ActivitiesTreatment-refractoryC5 inhibitor eculizumabDiscontinue prednisoneImmunosuppressive therapySymptom expression
2023
Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open‐label extension
Nowak R, Breiner A, Bril V, Allen J, Khan S, Levine T, Jacobs D, Sahagian G, Siddiqi Z, Xu J, Macias W, Benatar M, Adams L, Genge A, Habib A, Hinton J, Holmlund T, Jacobs D, Lange D, Nicolle M, Phan H, Silvestri N, Small G, Yegiaian S. Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open‐label extension. Annals Of Clinical And Translational Neurology 2023, 11: 194-206. PMID: 38062618, PMCID: PMC10791011, DOI: 10.1002/acn3.51946.Peer-Reviewed Original ResearchAnti-acetylcholine receptor antibodiesGeneralized myasthenia gravisTotal immunoglobulin GMyasthenia gravisImmunoglobulin G subclassesReceptor antibodiesOpen-label extension studyG subclassesImmunoglobulin GMyasthenia Gravis ActivitiesPatient-administered therapyPreliminary clinical benefitsQuantitative Myasthenia GravisOpen-label extensionPlacebo-controlled trialPhase 2a trialWeekly subcutaneous injectionsReceptor monoclonal antibodyMyasthenia Gravis QualityStudy support further investigationSupport further investigationMyasthenia Gravis CompositeEligible patientsPrimary endpointSecondary endpoints
2019
P.026 Response to eculizumab in patients with myasthenia gravis recently treated with chronic intravenous immunoglobulin
Genge A, Jacob S, Murai H, Utsugisawa K, Nowak R, Wiendl H, Fujita K, O’Brien F, Howard J. P.026 Response to eculizumab in patients with myasthenia gravis recently treated with chronic intravenous immunoglobulin. Canadian Journal Of Neurological Sciences / Journal Canadien Des Sciences Neurologiques 2019, 46: s20-s21. DOI: 10.1017/cjn.2019.126.Peer-Reviewed Original ResearchIntravenous immunoglobulinMyasthenia gravisAnti-acetylcholine receptor antibody-positive refractoryRefractory myasthenia gravisOpen-label extensionPlacebo-controlled studyMeaningful clinical improvementExperienced exacerbationRescue therapyClinical improvementEighteen patientsMore patientsSubgroup analysisEculizumabPatientsPlaceboExacerbationRelevant improvementGravisPhase 3Status changesInterim dataImmunoglobulinPrevious reportsMeaningful responsesA.01 Relieving the burden of myasthenia gravis: eculizumab reduces exacerbation, hospitalization and rescue therapy rates
Siddiqi Z, Howard J, Jacob S, Guptill J, Meisel A, Wiendl H, Murai H, Utsugisawa K, Nowak R, Fujita K, O’Brien F, Patra K, Muppidi S. A.01 Relieving the burden of myasthenia gravis: eculizumab reduces exacerbation, hospitalization and rescue therapy rates. Canadian Journal Of Neurological Sciences / Journal Canadien Des Sciences Neurologiques 2019, 46: s8-s8. DOI: 10.1017/cjn.2019.83.Peer-Reviewed Original ResearchPre-study yearOpen-label extensionHealthcare resource utilizationMyasthenia gravisEculizumab treatmentDisease burdenSignificant healthcare resource utilizationAnti-acetylcholine receptorExacerbations/hospitalizationsGreater disease burdenPoisson regression modelsMG crisisTherapy ratesRescue therapyResponsive patientsTherapy useTherapy usageClinical endpointsExacerbationHospitalizationPatientsMeaningful benefitREGAIN studyPatient recordsEvent rates