2014
Impact of velaglucerase alfa on bone marrow burden score in adult patients with type 1 Gaucher disease: 7-Year follow-up
Elstein D, Haims AH, Zahrieh D, Cohn GM, Zimran A. Impact of velaglucerase alfa on bone marrow burden score in adult patients with type 1 Gaucher disease: 7-Year follow-up. Blood Cells Molecules And Diseases 2014, 53: 56-60. PMID: 24581483, DOI: 10.1016/j.bcmd.2014.02.006.Peer-Reviewed Original ResearchConceptsBone marrow burdenBMB scoreVelaglucerase alfaLumbar spinePhase I/II trialLong-term extension studyGaucher diseaseType 1 Gaucher diseaseTotal score decreaseBone marrow infiltrationBone marrow burden scoreT2-weighted magnetic resonance imagesAssessable patientsII trialAdult patientsMarrow involvementMarrow infiltrationProspective studyBurden scoreGD patientsMedian changeLarge cohortScore decreaseDose reductionPatients
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
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