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