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dc.contributor.authorCox, Timothy
dc.contributor.authorDrelichman, G
dc.contributor.authorCravo, R
dc.contributor.authorBalwani, M
dc.contributor.authorBurrow, TA
dc.contributor.authorMartins, AM
dc.contributor.authorLukina, E
dc.contributor.authorRosenbloom, B
dc.contributor.authorGoker-Alpan, O
dc.contributor.authorWatman, N
dc.contributor.authorEl-Beshlawy, A
dc.contributor.authorKishnani, PS
dc.contributor.authorPedroso, ML
dc.contributor.authorGaemers, SJM
dc.contributor.authorTayag, R
dc.contributor.authorPeterschmitt, MJ
dc.date.accessioned2017-04-20T17:40:17Z
dc.date.available2017-04-20T17:40:17Z
dc.date.issued2017-02-06
dc.identifier.issn0006-4971
dc.identifier.urihttps://www.repository.cam.ac.uk/handle/1810/263735
dc.description.abstractIn the phase 3 trial of eliglustat in patients with Gaucher disease type 1 already stabilized with enzyme therapy (ENCORE), at one year, eliglustat was non-inferior to imiglucerase enzyme therapy in maintaining stable platelet counts, hemoglobin concentrations, and spleen and liver volumes. After this primary analysis period, patients entered a long-term extension phase in which all received eliglustat. Duration on eliglustat ranged from 2 to 5 years, depending on timing of enrollment (which spanned 2 years), treatment group to which patients were randomized, and whether they lived in the United States when commercial eliglustat became available. Here we report long-term safety and efficacy of eliglustat for 157 patients who received eliglustat in the ENCORE trial; data are available for 46 patients who received eliglustat for 4 years. Mean hemoglobin concentration, platelet count, and spleen and liver volumes remained stable for up to 4 years. Year to year, all four measures remained collectively stable (composite endpoint relative to baseline values) in ≥85% of patients, as well as individually in ≥92%. Mean bone mineral density Z-scores (lumbar spine and femur) remained stable and were maintained in the healthy reference range throughout. Eliglustat was well-tolerated over 4 years; 4 (2.5%) patients withdrew due to adverse events that were considered related to the study drug. No new or long-term safety concerns were identified. Clinical stability assessed by composite and individual measures was maintained in adults with Gaucher disease type 1 treated with eliglustat who remained in the ENCORE trial for up to 4 years.
dc.description.sponsorshipThe ENCORE trial was funded and conducted by Sanofi Genzyme.
dc.languageeng
dc.language.isoen
dc.publisherAmerican Society of Hematology
dc.subjectGaucher disease type 1
dc.subjecteliglustat
dc.subjectsubstrate reduction therapy
dc.subjectacid β-glucosidase deficiency
dc.subjectimiglucerase
dc.subjectenzyme replacement therapy
dc.titleEliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy
dc.typeArticle
prism.publicationDate2017
prism.publicationNameBlood
dc.identifier.doi10.17863/CAM.9100
dcterms.dateAccepted2017-01-27
rioxxterms.versionofrecord10.1182/blood-2016-12-758409
rioxxterms.versionAM
rioxxterms.licenseref.urihttp://www.rioxx.net/licenses/all-rights-reserved
rioxxterms.licenseref.startdate2017-02-06
dc.contributor.orcidCox, Timothy [0000-0002-4951-9941]
dc.identifier.eissn1528-0020
rioxxterms.typeJournal Article/Review
pubs.funder-project-idMedical Research Council (MR/K025570/1)
pubs.funder-project-idMedical Research Council (MR/K015338/1)
rioxxterms.freetoread.startdate2018-02-06


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