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dc.contributor.authorCox, Timothyen
dc.contributor.authorAmato, Dominicken
dc.contributor.authorHollak, Carla EMen
dc.contributor.authorLuzy, Cecileen
dc.contributor.authorSilkey, Mariabethen
dc.contributor.authorGiorgino, Rubenen
dc.contributor.authorSteiner, Robert Den
dc.contributor.authorMiglustat, Maintenance Study Groupen
dc.date.accessioned2013-01-29T06:25:52Z
dc.date.available2013-01-29T06:25:52Z
dc.date.issued2012-12-27en
dc.identifier.issn1750-1172
dc.identifier.urihttp://www.dspace.cam.ac.uk/handle/1810/244214
dc.description.abstractAbstract Background Previous studies have provided equivocal data on the use of miglustat as maintenance therapy in Gaucher disease type 1. We report findings from a clinical trial evaluating the effects of miglustat treatment in patients with stable type 1 Gaucher disease after enzyme therapy. Methods Adult type 1 Gaucher disease patients stabilized during at least 3 years of previous enzyme therapy were included in this 2-year, prospective, open-label non-inferiority study. The primary endpoint was percent change from baseline in liver volume. Secondary endpoints included changes in spleen volume, hemoglobin concentration and platelet count. Results Forty-two patients were enrolled (mean±SD age, 45.1±12.7 years; previous enzyme therapy duration 9.5±4.0 years). Median (range) exposure to miglustat 100 mg t.i.d. was 658 (3–765) days. Twenty-one patients discontinued treatment prematurely; 13 due to adverse events, principally gastrointestinal. The upper 95% confidence limit of mean percent change in liver volume from baseline to end of treatment was below the non-inferiority margin of 10% (–1.1%; 95%CI −6.0, 3.9%). Mean (95%CI) changes in spleen volume, hemoglobin concentration and platelet count were 102 (24,180) mL, –0.95 (−1.38, –0.53) g/dL and −44.1 (–57.6, –30.7) ×109/L, respectively. Conclusions The primary efficacy endpoint was met; overall there was no change in liver volume during 24 months of miglustat therapy. Several patients showed a gradual deterioration in some disease manifestations, suggesting that miglustat could maintain clinical stability, but not in all patients. Miglustat demonstrated a predictable profile of safety and tolerability that was consistent with that reported in previous clinical trials and experience in clinical practice. Trial registration Clinicaltrials.gov identifier NCT00319046
dc.languageEnglishen
dc.language.isoen
dc.titleEvaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority studyen
dc.typeArticle
dc.date.updated2013-01-29T06:25:53Z
dc.description.versionRIGHTS : This article is licensed under the BioMed Central licence at http://www.biomedcentral.com/about/license which is similar to the 'Creative Commons Attribution Licence'. In brief you may : copy, distribute, and display the work; make derivative works; or make commercial use of the work - under the following conditions: the original author must be given credit; for any reuse or distribution, it must be made clear to others what the license terms of this work are.en
dc.rights.holderTimothy M Cox et al.; licensee BioMed Central Ltd.
prism.publicationDate2012en
dcterms.dateAccepted2012-12-07en
rioxxterms.versionofrecord10.1186/1750-1172-7-102en
rioxxterms.licenseref.urihttp://www.rioxx.net/licenses/all-rights-reserveden
rioxxterms.licenseref.startdate2012-12-27en
dc.contributor.orcidCox, Timothy [0000-0002-4951-9941]
dc.identifier.eissn1750-1172
rioxxterms.typeJournal Article/Reviewen
pubs.funder-project-idMEDICAL RESEARCH COUNCIL (MR/K015338/1)


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