2021
Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results
Mistry PK, Lukina E, Turkia H, Shankar SP, Feldman H, Ghosn M, Mehta A, Packman S, Lau H, Petakov M, Assouline S, Balwani M, Danda S, Hadjiev E, Ortega A, Foster MC, Gaemers SJM, Peterschmitt MJ. Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results. American Journal Of Hematology 2021, 96: 1156-1165. PMID: 34161616, PMCID: PMC8457136, DOI: 10.1002/ajh.26276.Peer-Reviewed Original ResearchConceptsDisease type 1Gaucher disease type 1Type 1Disease manifestationsENGAGE trialOpen-label extension periodOral substrate reduction therapySpine T-scoreYears of treatmentSubstrate reduction therapyMagnitude of improvementEliglustat therapyMean hemoglobinUntreated patientsAdverse eventsTrial cohortClinical outcomesEligible adultsPlatelet countUntreated adultsSpleen volumeLiver volumeHematologic parametersReduction therapyPatients
2019
Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials
Weinreb N, Cox T, Mistry P, Charrow J, Lukina E, Foster M, Peterschmitt M. Biomarker Response to Oral Eliglustat in Adults with Gaucher Disease Type 1: Results from 4 Completed Clinical Trials. Blood 2019, 134: 4859. DOI: 10.1182/blood-2019-127337.Peer-Reviewed Original ResearchGaucher disease type 1Enzyme replacement therapySanofi GenzymeTreatment-naïve patientsMedian percent reductionMIP-1βDisease type 1Travel reimbursementOral eliglustatAdverse eventsMost patientsStable patientsClinical trialsBiomarker levelsSpeakers bureauEliglustat treatmentType 1Advisory CommitteeLong-term therapeutic goalPoor CYP2D6 metabolizersStorage of glycosphingolipidsFirst-line treatmentSubset of patientsLong-term time pointsAttrition of patients
2018
Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials
Mistry P, Charrow J, Cox T, Lukina E, Marinakis T, Foster M, Gaemers S, Peterschmitt J. Long-Term Effects of Oral Eliglustat on Skeletal Manifestations of Gaucher Disease Type 1: Results from Four Completed Clinical Trials. Blood 2018, 132: 2396. DOI: 10.1182/blood-2018-99-117289.Peer-Reviewed Original ResearchGaucher disease type 1Enzyme replacement therapyBone crisesSanofi GenzymeTreatment-naïve patientsMIP-1β levelsSpine Z-scoreSpine T-scoreENCORE trialDisease type 1Z-scoreBone diseaseT-scoreOral eliglustatBone painAdverse eventsClinical trialsNormal rangeYears of ERTAcid β-glucosidase activityOral substrate reduction therapyType 1Phase 2Principal investigatorTravel reimbursement
2011
Report on the Safety of Velaglucerase Alfa Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease and the Transition From Clinic to Home Infusions During Treatment Protocol HGT-GCB-058
Pastores G, Rosenbloom B, Weinreb N, Goker-Alpan O, Mardach R, Lipson M, Ibrahim J, Cohn G, Zahrieh D, Mistry P. Report on the Safety of Velaglucerase Alfa Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease and the Transition From Clinic to Home Infusions During Treatment Protocol HGT-GCB-058. Blood 2011, 118: 1101. DOI: 10.1182/blood.v118.21.1101.1101.Peer-Reviewed Original ResearchType 1 Gaucher's diseaseEnzyme replacement therapyTreatment-naïve patientsAdverse eventsSwitch patientsHome infusionHome therapyVelaglucerase alfaGaucher diseaseSpeakers bureauSerious AEsReplacement therapyVelaglucerase alfa enzyme replacement therapyClinical sitesInfusion-related adverse eventsCommon adverse eventsMost adverse eventsVelaglucerase alfa treatmentSerious adverse eventsSevere adverse eventsBone marrow pathologyAccumulation of glucosylceramideMononuclear phagocyte systemAdvisory CommitteeImiglucerase treatment