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
2008
Correlation of MRI-Based bone marrow burden score with genotype and spleen status in Gaucher's disease.
DeMayo RF, Haims AH, McRae MC, Yang R, Mistry PK. Correlation of MRI-Based bone marrow burden score with genotype and spleen status in Gaucher's disease. American Journal Of Roentgenology 2008, 191: 115-23. PMID: 18562733, PMCID: PMC3731993, DOI: 10.2214/ajr.07.3550.Peer-Reviewed Original ResearchConceptsBone marrow burden scoreEnzyme replacement therapyBurden scoreBone marrow diseaseSpleen statusReplacement therapyGaucher diseaseN370S alleleCumulative durationPathologic findingsLiver volumeMarrow diseaseSignificant positive correlationBone marrow burdenCorrelation of MRIN370S homozygotesUntreated diseaseClinical parametersPositive correlationLiver sizeProtective roleAmeliorative effectSignificant associationGreater riskDisease