2023
P1588: LONG‐TERM REAL‐WORLD EFFECTIVENESS OF ELIGLUSTAT IN TREATMENT‐NAÏVE AND SWITCH PATIENTS ENROLLED IN THE INTERNATIONAL COLLABORATIVE GAUCHER GROUP (ICGG) GAUCHER REGISTRY
Mistry P, Balwani M, Charrow J, Lorber J, Niederau C, Carwile J, Uslu S, Kishnani P. P1588: LONG‐TERM REAL‐WORLD EFFECTIVENESS OF ELIGLUSTAT IN TREATMENT‐NAÏVE AND SWITCH PATIENTS ENROLLED IN THE INTERNATIONAL COLLABORATIVE GAUCHER GROUP (ICGG) GAUCHER REGISTRY. HemaSphere 2023, 7: e2191333. PMCID: PMC10430384, DOI: 10.1097/01.hs9.0000973228.21913.33.Peer-Reviewed Original Research
2020
Real‐world effectiveness of eliglustat in treatment‐naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry
Mistry PK, Balwani M, Charrow J, Kishnani P, Niederau C, Underhill LH, McClain MR. Real‐world effectiveness of eliglustat in treatment‐naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry. American Journal Of Hematology 2020, 95: 1038-1046. PMID: 32438452, PMCID: PMC7497238, DOI: 10.1002/ajh.25875.Peer-Reviewed Original ResearchConceptsSpine Z-scoreInternational Collaborative Gaucher Group Gaucher RegistryGaucher disease type 1Treatment-naïve patientsSwitch patientsMean hemoglobinPlatelet countLiver volumeZ-scoreGaucher RegistrySpleen volumeLumbar spine Z-scoreFirst-line oral therapyCYP2D6 metabolizer phenotypeMean platelet countReal-world effectivenessClinical trial resultsDisease type 1Real-world outcomesEliglustat therapyGD1 patientsOral therapyTreatment-naïveMost patientsERT patients
2019
Two years of efficacy of oral eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher registry
Mistry P, Balwani M, Charrow J, Kishnani P, Niederau C, McClain M. Two years of efficacy of oral eliglustat in treatment-naïve and switch patients enrolled in the International Collaborative Gaucher Group Gaucher registry. Molecular Genetics And Metabolism 2019, 126: s100-s101. DOI: 10.1016/j.ymgme.2018.12.252.Peer-Reviewed Original ResearchX109/LTreatment-naïve patientsInternational Collaborative Gaucher Group Gaucher RegistryGaucher disease type 1Enzyme replacement therapySwitch patientsMean hemoglobinPlatelet countLiver volumeGaucher RegistrySpleen volumeClinical trialsOral substrate reduction therapyLong-term clinical improvementCYP2D6 metabolizer phenotypeMajor disease manifestationsDisease type 1Substrate reduction therapyKey disease parametersOral eliglustatTreatment-naïveClinical improvementChitotriosidase levelsDisease manifestationsReduction therapy
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