2011
Haematological Disorders of the Liver
Mistry P, Jain D. Haematological Disorders of the Liver. 2011, 48-69. DOI: 10.1002/9781444341294.ch4.Peer-Reviewed Original ResearchLiver diseaseHaematological disordersEnd-stage liver diseasePrimary haematological disordersPrimary liver diseaseAcute liver failureMajor clinical significanceE hepatitisLiver transplantationLiver involvementLiver failureNormal physiological interactionsErythrocyte dysfunctionClinical significanceAbnormal plateletsHaematological effectsBone marrowCoagulation pathwayDiseaseDisease statesMembrane abnormalitiesStorage diseaseDisordersLiverPhysiological interactions
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