{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T07:41:48Z","timestamp":1791013308113,"version":"4.1.0"},"reference-count":18,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2009,6,8]],"date-time":"2009-06-08T00:00:00Z","timestamp":1244419200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmacoepidemiology and Drug"],"published-print":{"date-parts":[[2009,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Purpose<\/jats:title><jats:p>Miglustat (Zavesca\u00ae) is an orally\u2010available substrate reduction therapy (SRT) for treatment of mild\u2010to\u2010moderate type 1 Gaucher disease (GD1) in adult patients unsuitable for enzyme replacement therapy (ERT). Miglustat has not been evaluated in children with GD1, and is not used during pregnancy and breast\u2010feeding. A non\u2010interventional, prospective, web\u2010based safety surveillance programme was initiated at the time of the European launch of miglustat in 2003, and is ongoing. We report the first 5 years of collected data, focusing on neurological manifestations.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Data were collected on 122 GD1 patients between March 2003 and April 2008, representing 244 patient\u2010years cumulative miglustat post\u2010authorisation experience. The electronically\u2010captured data collected from participating physicians includes patient demographics, prior and current therapies, baseline disease manifestations and concurrent conditions, disease severity, duration of miglustat exposure, and safety\u2010relevant information.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Mean (SD) age at baseline was 46.1 (16.5) years. At baseline, bone disease and neurological manifestations were reported in 55.6 and 28.6% of patients, respectively; the latter included peripheral neuropathy (7.2%) and a wide variety of neurological symptoms and signs. In addition, 23.2% had other health conditions relevant to neurological status. During the reporting period, new neurological manifestations were reported in 23 (18.9%) patients, principally tremor. Thirty\u2010five (28.7%) patients discontinued treatment, predominantly for gastrointestinal (GI) disturbances (11.5%), two\u2010thirds of which occurred during the first 6 months.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The safety profile of miglustat in GD1 patients included in the safety surveillance programme is overall consistent with that reported in the registration and other clinical trials, and no new safety finding was identified. Copyright \u00a9 2009 John Wiley &amp; Sons, Ltd.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pds.1779","type":"journal-article","created":{"date-parts":[[2009,6,8]],"date-time":"2009-06-08T07:48:17Z","timestamp":1244447297000},"page":"770-777","source":"Crossref","is-referenced-by-count":75,"title":["Miglustat (Zavesca\u00ae) in type 1 Gaucher disease: 5\u2010year results of a post\u2010authorisation safety surveillance programme"],"prefix":"10.1002","volume":"18","author":[{"given":"Carla E. M.","family":"Hollak","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Derralynn","family":"Hughes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivo N.","family":"van Schaik","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barbara","family":"Schwierin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bruno","family":"Bembi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2009,8,18]]},"reference":[{"key":"e_1_2_1_2_2"},{"key":"e_1_2_1_3_2"},{"key":"e_1_2_1_4_2"},{"key":"e_1_2_1_5_2"},{"key":"e_1_2_1_6_2"},{"key":"e_1_2_1_7_2","doi-asserted-by":"publisher","DOI":"10.1177\/106002809603000411"},{"key":"e_1_2_1_8_2"},{"key":"e_1_2_1_9_2","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-4615-0065-0_14"},{"key":"e_1_2_1_10_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0021-9258(17)37202-2"},{"key":"e_1_2_1_11_2","unstructured":"Miglustat Summary of Product Characteristics.http:\/\/www.emea. europa.eu\/humandocs\/Humans\/EPAR\/zavesca\/zavesca.htm(accessed 31 March 2009)."},{"key":"e_1_2_1_12_2"},{"key":"e_1_2_1_13_2","doi-asserted-by":"publisher","DOI":"10.1097\/00005792-199211000-00002"},{"key":"e_1_2_1_14_2"},{"key":"e_1_2_1_15_2"},{"key":"e_1_2_1_16_2","first-page":"792","article-title":"Report of the Spanish Gaucher's disease registry: clinical and genetic characteristics","volume":"85","author":"Giraldo P","year":"2000","journal-title":"Haematologica"},{"key":"e_1_2_1_17_2"},{"key":"e_1_2_1_18_2","unstructured":"Committee for Medicinal Products for Human Use. Guideline on risk management systems for medicinal products for human use. EMEA\/CHMP\/96268\/2005http:\/\/www.emea.europa.eu\/pdfs\/human\/euleg\/9626805en.pdf(accessed 12 December2008)."},{"key":"e_1_2_1_19_2","unstructured":"Managing the risks from medical product use: creating a risk management framework. Report to the FDA Commissioner from the Task Force on Risk Management.1999.http:\/\/www.fda.gov\/oc\/tfrm\/executivesummary.pdf(accessed 12 December 2008)."}],"container-title":["Pharmacoepidemiology and Drug Safety"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fpds.1779","content-type":"unspecified","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/pds.1779","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,11,14]],"date-time":"2023-11-14T22:27:38Z","timestamp":1700000858000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/pds.1779"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,8,18]]},"references-count":18,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2009,9]]}},"alternative-id":["10.1002\/pds.1779"],"URL":"https:\/\/doi.org\/10.1002\/pds.1779","archive":["Portico"],"relation":{},"ISSN":["1053-8569","1099-1557"],"issn-type":[{"value":"1053-8569","type":"print"},{"value":"1099-1557","type":"electronic"}],"subject":[],"published":{"date-parts":[[2009,8,18]]}}}