{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,31]],"date-time":"2026-08-31T10:44:46Z","timestamp":1788173086161,"version":"build-2803163510"},"reference-count":32,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,8,17]],"date-time":"2024-08-17T00:00:00Z","timestamp":1723852800000},"content-version":"am","delay-in-days":290,"URL":"http:\/\/www.elsevier.com\/open-access\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2023,11,1]],"date-time":"2023-11-01T00:00:00Z","timestamp":1698796800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2023,11]]},"DOI":"10.1016\/j.ymgme.2023.107667","type":"journal-article","created":{"date-parts":[[2023,7,26]],"date-time":"2023-07-26T02:32:44Z","timestamp":1690338764000},"page":"107667","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":5,"title":["Patient reported outcomes of patients with Gaucher disease type 1 treated with eliglustat in real-world settings: The ELIPRO study"],"prefix":"10.1016","volume":"140","author":[{"given":"Fabrice","family":"Camou","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Audrey","family":"Lagadec","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Coutinho","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marc G.","family":"Berger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B\u00e9reng\u00e8re","family":"Cador-Rousseau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francis","family":"Gaches","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nadia","family":"Belmatoug","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"2","key":"10.1016\/j.ymgme.2023.107667_bb0005","doi-asserted-by":"crossref","DOI":"10.3390\/ijms18020441","article-title":"A review of Gaucher disease pathophysiology, clinical presentation and treatments","volume":"18","author":"Stirnemann","year":"2017","journal-title":"Int. J. Mol. Sci."},{"issue":"9645","key":"10.1016\/j.ymgme.2023.107667_bb0010","doi-asserted-by":"crossref","first-page":"1263","DOI":"10.1016\/S0140-6736(08)61522-6","article-title":"Phenotype, diagnosis, and treatment of Gaucher\u2019s disease","volume":"372","author":"Grabowski","year":"2008","journal-title":"Lancet."},{"key":"10.1016\/j.ymgme.2023.107667_bb0015","series-title":"CERDELGATM 84 mg capsules Summary of Product Characteristics","year":"2018"},{"key":"10.1016\/j.ymgme.2023.107667_bb0020","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1016\/j.ejim.2016.07.011","article-title":"Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe","volume":"37","author":"Belmatoug","year":"2017","journal-title":"Eur. J. Intern. Med."},{"issue":"9985","key":"10.1016\/j.ymgme.2023.107667_bb0025","doi-asserted-by":"crossref","first-page":"2355","DOI":"10.1016\/S0140-6736(14)61841-9","article-title":"Eliglustat compared with imiglucerase in patients with Gaucher\u2019s disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial","volume":"385","author":"Cox","year":"2015","journal-title":"Lancet."},{"issue":"3","key":"10.1016\/j.ymgme.2023.107667_bb0030","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1016\/j.ymgme.2017.12.001","article-title":"Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: the phase 3, randomized, double-blind EDGE trial","volume":"123","author":"Charrow","year":"2018","journal-title":"Mol. Genet. Metab."},{"issue":"7","key":"10.1016\/j.ymgme.2023.107667_bb0035","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1001\/jama.2015.459","article-title":"Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial","volume":"313","author":"Mistry","year":"2015","journal-title":"JAMA."},{"issue":"1\u20132","key":"10.1016\/j.ymgme.2023.107667_bb0040","doi-asserted-by":"crossref","first-page":"S97","DOI":"10.1016\/j.ymgme.2016.11.243","article-title":"Long-term results of ENGAGE: a phase 3, randomized, double blind, placebo-controlled, multi center study investigating the efficacy and safety of eliglustat in adults with type 1 Gaucher disease|Cochrane library","volume":"120","author":"Mistry","year":"2017","journal-title":"Mol. Genet. Metab."},{"issue":"11","key":"10.1016\/j.ymgme.2023.107667_bb0045","doi-asserted-by":"crossref","first-page":"1170","DOI":"10.1002\/ajh.24877","article-title":"Outcomes after 18 months of eliglustat therapy in treatment-naive adults with Gaucher disease type 1: the phase 3 ENGAGE trial","volume":"92","author":"Mistry","year":"2017","journal-title":"Am. J. Hematol."},{"issue":"17","key":"10.1016\/j.ymgme.2023.107667_bb0050","doi-asserted-by":"crossref","first-page":"2375","DOI":"10.1182\/blood-2016-12-758409","article-title":"Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy","volume":"129","author":"Cox","year":"2017","journal-title":"Blood."},{"issue":"9","key":"10.1016\/j.ymgme.2023.107667_bb0055","doi-asserted-by":"crossref","first-page":"1038","DOI":"10.1002\/ajh.25875","article-title":"Real-world effectiveness of eliglustat in treatment-naive and switch patients enrolled in the international collaborative Gaucher group Gaucher registry","volume":"95","author":"Mistry","year":"2020","journal-title":"Am. J. Hematol."},{"key":"10.1016\/j.ymgme.2023.107667_bb0060","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1016\/j.bcmd.2016.10.008","article-title":"Management goals for type 1 Gaucher disease: an expert consensus document from the European working group on Gaucher disease","volume":"68","author":"Biegstraaten","year":"2018","journal-title":"Blood Cells Mol. Dis."},{"issue":"1","key":"10.1016\/j.ymgme.2023.107667_bb0065","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1186\/s13023-021-02163-y","article-title":"Development and validation of Gaucher disease type 1 (GD1)-specific patiet-reported outcome measures (PROMs) for clinical monitoring and for clinical trials","volume":"17","author":"Elstein","year":"2022","journal-title":"Orphanet. J. Rare Dis."},{"issue":"5","key":"10.1016\/j.ymgme.2023.107667_bb0070","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1111\/j.1751-7176.2008.07572.x","article-title":"Predictive validity of a medication adherence measure in an outpatient setting","volume":"10","author":"Morisky","year":"2008","journal-title":"J. Clin. Hypertens (Greenwich)."},{"issue":"3","key":"10.1016\/j.ymgme.2023.107667_bb0075","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.jclinepi.2010.09.002","article-title":"Improving the measurement of self-reported medication nonadherence: response to authors","volume":"64","author":"Morisky","year":"2011","journal-title":"J. Clin. Epidemiol."},{"issue":"1","key":"10.1016\/j.ymgme.2023.107667_bb0080","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1097\/00005650-198601000-00007","article-title":"Concurrent and predictive validity of a self-reported measure of medication adherence","volume":"24","author":"Morisky","year":"1986","journal-title":"Med. Care"},{"issue":"20","key":"10.1016\/j.ymgme.2023.107667_bb0085","doi-asserted-by":"crossref","first-page":"4095","DOI":"10.1182\/blood-2010-06-293902","article-title":"Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz 112638) treatment: 2-year results of a phase 2 study","volume":"116","author":"Lukina","year":"2010","journal-title":"Blood."},{"key":"10.1016\/j.ymgme.2023.107667_bb0090","series-title":"EuroQol--a new facility for the measurement of health-related quality of life. Health policy (Amsterdam, Netherlands)","first-page":"199","volume":"16(3)","year":"1990"},{"key":"10.1016\/j.ymgme.2023.107667_bb0095","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1186\/s13023-015-0280-3","article-title":"Evaluation of disease burden and response to treatment in adults with type 1 Gaucher disease using a validated disease severity scoring system (DS3)","volume":"10","author":"Weinreb","year":"2015","journal-title":"Orphanet. J. Rare Dis."},{"key":"10.1016\/j.ymgme.2023.107667_bb0100","series-title":"26th Scientific Plenary Meeting of the EuroQol Group - Proceedings:1\u201319 \u00a9 2011","article-title":"Valuing EQ-5D using Time Trade-Off in France","author":"Chevalier","year":"2011"},{"issue":"5","key":"10.1016\/j.ymgme.2023.107667_bb0105","doi-asserted-by":"crossref","first-page":"631","DOI":"10.1111\/j.1524-4733.2010.00727.x","article-title":"General population reference values for the French version of the EuroQol EQ-5D health utility instrument","volume":"13","author":"Perneger","year":"2010","journal-title":"Value Health"},{"issue":"5","key":"10.1016\/j.ymgme.2023.107667_bb0110","doi-asserted-by":"crossref","first-page":"708","DOI":"10.1016\/j.jval.2012.02.008","article-title":"Interim scoring for the EQ-5D-5L: mapping the EQ-5D-5L to EQ-5D-3L value sets","volume":"15","author":"van Hout","year":"2012","journal-title":"Value Health"},{"key":"10.1016\/j.ymgme.2023.107667_bb0115","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1186\/1750-1172-7-77","article-title":"The French Gaucher\u2019s disease registry: clinical characteristics, complications and treatment of 562 patients","volume":"7","author":"Stirnemann","year":"2012","journal-title":"Orphanet. J. Rare Dis."},{"issue":"9","key":"10.1016\/j.ymgme.2023.107667_bb0120","doi-asserted-by":"crossref","first-page":"1156","DOI":"10.1002\/ajh.26276","article-title":"Clinical outcomes after 4.5years of eliglustat therapy for Gaucher disease type 1: phase 3 ENGAGE trial final results","volume":"96","author":"Mistry","year":"2021","journal-title":"Am. J. Hematol."},{"issue":"1","key":"10.1016\/j.ymgme.2023.107667_bb0125","first-page":"59","article-title":"New medication adherence scale versus pharmacy fill rates in hypertensive seniors","volume":"15","author":"Krousel-Wood","year":"2009","journal-title":"Am. J. Manag. Care"},{"issue":"2480","key":"10.1016\/j.ymgme.2023.107667_bb0130","doi-asserted-by":"crossref","first-page":"909","DOI":"10.53738\/REVMED.2004.62.2480.0909","article-title":"Revue M\u00e9dicale Suisse\u202f: Am\u00e9liorer l\u2019observance m\u00e9dicamenteuse","volume":"62","author":"Amati","year":"2004","journal-title":"Rev. M\u00e9d. Suisse."},{"issue":"21","key":"10.1016\/j.ymgme.2023.107667_bb0135","doi-asserted-by":"crossref","first-page":"6265","DOI":"10.3390\/jcm11216265","article-title":"Real-life experience with oral eliglustat in patients with Gaucher disease previously treated with enzyme replacement therapy","volume":"11","author":"Isataiti","year":"2022","journal-title":"J. Clin. Med."},{"key":"10.1016\/j.ymgme.2023.107667_bb0140","doi-asserted-by":"crossref","first-page":"39","DOI":"10.2147\/PPA.S24752","article-title":"A literature review to explore the link between treatment satisfaction and adherence, compliance, and persistence","volume":"6","author":"Barbosa","year":"2012","journal-title":"Patient Prefer. Adher."},{"key":"10.1016\/j.ymgme.2023.107667_bb0145","doi-asserted-by":"crossref","DOI":"10.1007\/s10198-022-01559-2","article-title":"Population norms in France with EQ-5D-5L: health states, value indexes, and VAS","author":"Gautier","year":"2023","journal-title":"Eur. J. Health Econ."},{"issue":"1","key":"10.1016\/j.ymgme.2023.107667_bb0150","doi-asserted-by":"crossref","first-page":"128","DOI":"10.1186\/s13023-019-1085-6","article-title":"Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1","volume":"14","author":"Peterschmitt","year":"2019","journal-title":"Orphanet. J. Rare Dis."},{"issue":"5","key":"10.1016\/j.ymgme.2023.107667_bb0155","doi-asserted-by":"crossref","first-page":"688","DOI":"10.1016\/S0002-9394(00)00754-6","article-title":"A comparison of observational studies and randomized, controlled trials","volume":"130","author":"Benson","year":"2000","journal-title":"Am J. Ophthalmol."},{"issue":"25","key":"10.1016\/j.ymgme.2023.107667_bb0160","doi-asserted-by":"crossref","first-page":"1887","DOI":"10.1056\/NEJM200006223422507","article-title":"Randomized, controlled trials, observational studies, and the hierarchy of research designs","volume":"342","author":"Concato","year":"2000","journal-title":"N. Engl. J. Med."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719223002974?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719223002974?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,12,26]],"date-time":"2024-12-26T17:13:32Z","timestamp":1735233212000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719223002974"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,11]]},"references-count":32,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2023,11]]}},"alternative-id":["S1096719223002974"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2023.107667","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2023,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Patient reported outcomes of patients with Gaucher disease type 1 treated with eliglustat in real-world settings: The ELIPRO study","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2023.107667","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 Published by Elsevier Inc.","name":"copyright","label":"Copyright"}],"article-number":"107667"}}