{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T01:37:36Z","timestamp":1790386656125,"version":"4.1.0"},"reference-count":21,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2005,10,24]],"date-time":"2005-10-24T00:00:00Z","timestamp":1130112000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2005,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Type 1 Gaucher disease (GD) is a progressive lysosomal storage disorder due to an autosomal recessive deficiency of glucocerebrosidase. Clinical manifestations include anemia, thrombocytopenia, hepatosplenomegaly, and bone and pulmonary disease. Intravenous enzyme replacement (ERT) with imiglucerase is the accepted standard for treatment of symptomatic patients. More than 3,500 patients worldwide have received ERT with well\u2010documented beneficial effects on the hematological, visceral, skeletal, and pulmonary manifestations, and with resultant improvement in health\u2010related quality of life. Miglustat, an imino sugar that reversibly inhibits glucosylceramide synthase and reduces intracellular substrate burden, is an oral treatment for patients with type 1 GD that was recently approved in the United States for symptomatic patients with mild to moderate clinical manifestations for whom ERT is not an option. Because responses to miglustat are slower and less robust than those observed with ERT, and because miglustat is associated with significant side effects, clinicians who care for patients with GD should become familiar with the limited indications for miglustat use and the circumstances when it may be prescribed appropriately. This review article and position statement represents the current opinion of American physicians with extensive expertise in GD regarding patient management in the context of the availability of standard imiglucerase treatment and the recent introduction of miglustat. Am. J. Hematol. 80:223\u2013229, 2005. \u00a9 2005 Wiley\u2010Liss, Inc.<\/jats:p>","DOI":"10.1002\/ajh.20504","type":"journal-article","created":{"date-parts":[[2005,10,24]],"date-time":"2005-10-24T15:47:32Z","timestamp":1130168852000},"page":"223-229","source":"Crossref","is-referenced-by-count":81,"title":["Guidance on the use of miglustat for treating patients with type 1 Gaucher disease"],"prefix":"10.1002","volume":"80","author":[{"given":"Neal J.","family":"Weinreb","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John A.","family":"Barranger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Charrow","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory A.","family":"Grabowski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henry J.","family":"Mankin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pramod","family":"Mistry","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2005,10,24]]},"reference":[{"key":"e_1_2_1_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0065-2660(08)60202-3"},{"key":"e_1_2_1_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.05.053"},{"key":"e_1_2_1_4_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.01.050"},{"key":"e_1_2_1_5_2","doi-asserted-by":"publisher","DOI":"10.1212\/01.WNL.0000130191.31669.48"},{"key":"e_1_2_1_6_2","doi-asserted-by":"publisher","DOI":"10.1053\/j.seminhematol.2004.07.009"},{"key":"e_1_2_1_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0002-9343(02)01150-6"},{"key":"e_1_2_1_8_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V93.6.2081.406k07_2081_2088"},{"key":"e_1_2_1_9_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V82.4.1107.1107"},{"key":"e_1_2_1_10_2","doi-asserted-by":"publisher","DOI":"10.1517\/13543784.12.2.273"},{"key":"e_1_2_1_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/0009-3084(80)90057-2"},{"key":"e_1_2_1_12_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0021-9258(18)47132-3"},{"key":"e_1_2_1_13_2","doi-asserted-by":"publisher","DOI":"10.2174\/1389200013338414"},{"key":"e_1_2_1_14_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(00)02161-9"},{"key":"e_1_2_1_15_2","unstructured":"European Public Assessment Report (EPAR). Zavesca [miglustat]. Committee for Proprietary Medicinal Products EPAR. CPMP\/3795\/02. London England: The European Agency for the Evaluation of Medicinal Products;2002.http:\/\/www.emea.eu.int\/humandocs\/Humans\/EPAR\/zavesca\/zavesca.htm."},{"key":"e_1_2_1_16_2","unstructured":"U.S. FDA Medical Review.http:\/\/www.fda.gov\/cder\/foi\/nda\/2003\/21\u2013348_Zavesca.htm."},{"key":"e_1_2_1_17_2","doi-asserted-by":"publisher","DOI":"10.1023\/A:1025902113005"},{"key":"e_1_2_1_18_2","doi-asserted-by":"publisher","DOI":"10.1098\/rstb.2003.1272"},{"key":"e_1_2_1_19_2","doi-asserted-by":"publisher","DOI":"10.1023\/B:BOLI.0000045756.54006.17"},{"key":"e_1_2_1_20_2","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1006\/bcmd.2002.0497","article-title":"Low\u2010dose N\u2010butyldeoxynojirimycin (OGT 918) for type I Gaucher disease","volume":"28","author":"Heitner R","year":"2002","journal-title":"Blood Cell Mol Dis"},{"key":"e_1_2_1_21_2","unstructured":"Canadian Coordinating Office for Health Technology Assessment. 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