{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T17:28:28Z","timestamp":1772213308173,"version":"3.50.1"},"reference-count":15,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2015,2,5]],"date-time":"2015-02-05T00:00:00Z","timestamp":1423094400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100005646","name":"Actelion Pharmaceuticals","doi-asserted-by":"publisher","award":["IS3"],"award-info":[{"award-number":["IS3"]}],"id":[{"id":"10.13039\/100005646","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2015,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Following approval in the EU in 2002 and the USA in 2003, an Intensive Safety Surveillance Scheme (IS<jats:sup>3<\/jats:sup>) was initiated to educate prescribers on the appropriate use of miglustat for the treatment of type I Gaucher disease (GD1), and to actively solicit safety\u2010relevant information. This report summarises data from all patients enrolled in IS<jats:sup>3<\/jats:sup> between its initiation in 2003 and its closure in October 2012.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>The IS<jats:sup>3<\/jats:sup> was a prospective observational drug registry with a secure internet\u2010based data capture system. All patients receiving miglustat at participating sites received standard medical care according to routine medical practice. Data on patient and disease characteristics were collected at patient enrolment, subsequent follow\u2010up visits and treatment discontinuation (if applicable). Data were summarised using descriptive statistics.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>During the 9\u2009years of IS<jats:sup>3<\/jats:sup>, 407 patients were enrolled at 111 sites across 15 European countries. Approximately half (<jats:italic>n<\/jats:italic>\u2009=\u2009202) had GD1, and half had other diseases (mainly Niemann\u2013Pick disease type C (NP\u2010C), for which miglustat was approved in Europe in 2009). In total, 368 patients had data from at least one follow\u2010up visit, 192 of whom had GD1. IS<jats:sup>3<\/jats:sup> provided data from 798 patient\u2010years exposure to miglustat. Safety\u2010relevant data were consistent with earlier published 5\u2010year findings from IS<jats:sup>3<\/jats:sup>, the safety profile reported for miglustat in GD1 clinical trials and other published information on GD1 manifestations.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Overall, the results of this long\u2010term safety surveillance programme were in line with the well\u2010known, documented safety profile of miglustat. Copyright \u00a9 2015 John Wiley &amp; Sons, Ltd.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pds.3760","type":"journal-article","created":{"date-parts":[[2015,2,5]],"date-time":"2015-02-05T19:08:54Z","timestamp":1423163334000},"page":"329-333","source":"Crossref","is-referenced-by-count":15,"title":["Results from a 9\u2010year Intensive Safety Surveillance Scheme (IS<sup>3<\/sup>) in miglustat (Zavesca<sup>\u00ae<\/sup>)\u2010treated patients"],"prefix":"10.1002","volume":"24","author":[{"given":"Monika","family":"Brand","sequence":"first","affiliation":[{"name":"Actelion Pharmaceuticals Ltd  Allschwil Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Audrey","family":"Muller","sequence":"additional","affiliation":[{"name":"Actelion Pharmaceuticals Ltd  Allschwil Switzerland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonathan","family":"Alsop","sequence":"additional","affiliation":[{"name":"Numerus Ltd  Wokingham UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ivo N.","family":"van Schaik","sequence":"additional","affiliation":[{"name":"Academic Medical Center  Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bruno","family":"Bembi","sequence":"additional","affiliation":[{"name":"Regional Coordinator Centre for Rare Diseases Azienda Ospedaliera Santa Maria della Misericordia di Udine  Udine Italy"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Derralynn","family":"Hughes","sequence":"additional","affiliation":[{"name":"Royal Free and University College Medical School  London UK"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2015,2,5]]},"reference":[{"key":"e_1_2_8_2_1","unstructured":"Actelion.Miglustat prescribing information.2014;http:\/\/www.zavesca.com\/pdf\/ZAVESCA-Full-Prescribing-Information.pdf 2009. 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