2019
Magnetic Resonance Imaging of Lipofibromatous Hamartoma Involving both the Median and Ulnar Nerve
Porrino JA, Wang AM, Branson B, Lischuk A, Haims A. Magnetic Resonance Imaging of Lipofibromatous Hamartoma Involving both the Median and Ulnar Nerve. PM&R 2019, 11: 900-901. PMID: 30851077, DOI: 10.1002/pmrj.12153.Peer-Reviewed Original Research
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
2010
Focal splenic lesions in type I Gaucher disease are associated with poor platelet and splenic response to macrophage‐targeted enzyme replacement therapy
Stein P, Malhotra A, Haims A, Pastores GM, Mistry PK. Focal splenic lesions in type I Gaucher disease are associated with poor platelet and splenic response to macrophage‐targeted enzyme replacement therapy. Journal Of Inherited Metabolic Disease 2010, 33: 769-774. PMID: 20683668, PMCID: PMC3008694, DOI: 10.1007/s10545-010-9175-6.Peer-Reviewed Original ResearchConceptsFocal splenic lesionsEnzyme replacement therapyGD1 patientsReplacement therapySplenic lesionsMacrophage-targeted enzyme replacement therapyPlatelet responseYears of ERTPrevalence of osteonecrosisGaucher disease type IDeterminants of responseType IPoor plateletsAvascular osteonecrosisSuboptimal responseSevere manifestationsSplenic responseIntact spleenClinical significanceSplenic parenchymaPatientsWorse thrombocytopeniaRoutine evaluationSplenomegalyTherapy