{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,3]],"date-time":"2026-06-03T08:40:57Z","timestamp":1780476057416,"version":"3.54.1"},"reference-count":20,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2015,6,22]],"date-time":"2015-06-22T00:00:00Z","timestamp":1434931200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004321","name":"Shire","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004321","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2015,7]]},"abstract":"<jats:p>Gaucher disease (GD) is a lysosomal storage disorder; symptomatic patients with type 1 GD need long\u2010term disease\u2010specific therapy of which the standard of care has been enzyme replacement therapy (ERT). Thirty\u2010eight of 40 patients (aged 9\u201371 years) clinically stable on ERT with imiglucerase, safely switched to a comparable dose of velaglucerase alfa (units\/kg) during TKT034, a 12\u2010month, open\u2010label clinical study, and for 10\u201350 months in an extension study. The most common adverse events (AEs) judged to be drug\u2010related in the extension were fatigue and bone pain. No drug\u2010related serious AEs were reported. No AEs led to study withdrawal. At 24 months from baseline (baseline being TKT034 week 0), patients had generally stable hemoglobin, platelet, spleen, liver, and bone density parameters. Nevertheless, dose adjustment based on the achievement of therapeutic goals was permitted, and 10 patients, including seven patients who had platelet counts &lt;100 \u00d7 10<jats:sup>9<\/jats:sup>\/L at baseline, were given at least one 15 U\/kg\u2010dose increase during the extension. Trends indicative of improvement in platelet count and spleen volume, and decreasing levels of GD biomarkers, chitotriosidase and CCL18, were observed. Immunogenicity was seen in one patient positive for anti\u2010imiglucerase antibodies at baseline. This patient tested positive for anti\u2010velaglucerase alfa antibodies in TKT034, with low antibody concentrations, and throughout the extension study; however, the patient continued to receive velaglucerase alfa without clinical deterioration. In conclusion, clinically stable patients can be switched from imiglucerase to velaglucerase alfa ERT and maintain or achieve good therapeutic outcomes. Am. J. Hematol. 90:592\u2013597, 2015. \u00a9 2015 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/ajh.24007","type":"journal-article","created":{"date-parts":[[2015,3,17]],"date-time":"2015-03-17T01:57:42Z","timestamp":1426557462000},"page":"592-597","source":"Crossref","is-referenced-by-count":17,"title":["Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 <scp>G<\/scp>aucher disease"],"prefix":"10.1002","volume":"90","author":[{"given":"Deborah","family":"Elstein","sequence":"first","affiliation":[{"name":"Gaucher Clinic, Shaare Zedek Medical Center, affiliated with the Hebrew University\u2010Hadassah Medical School Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Atul","family":"Mehta","sequence":"additional","affiliation":[{"name":"Department of Haematology Royal Free Hospital London United Kingdom"},{"name":"Department of Haematology University College London London United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Derralynn A.","family":"Hughes","sequence":"additional","affiliation":[{"name":"Department of Haematology Royal Free Hospital London United Kingdom"},{"name":"Department of Haematology University College London London United Kingdom"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pilar","family":"Giraldo","sequence":"additional","affiliation":[{"name":"Medicina Metab\u00f3lica Hereditaria Centro de Investigaci\u00f3n Biom\u00e9dica en Red de Enfermedades Raras (CIBERER) Zaragoza Spain"},{"name":"Grupo de Estudio de Enfermedades Hematolog\u00edcas y Metabolicas, Hospital Universitario Miguel Servet Zaragoza Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joel","family":"Charrow","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Ann &amp; Robert H. Lurie Children's Hospital of Chicago Chicago Illinois"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurie","family":"Smith","sequence":"additional","affiliation":[{"name":"Center for Pediatric Genomic Medicine Children's Mercy Hospital Kansas City Missouri"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suma P.","family":"Shankar","sequence":"additional","affiliation":[{"name":"Departments of Human Genetics and Ophthalmology, School of Medicine Emory University Atlanta Georgia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas N.","family":"Hangartner","sequence":"additional","affiliation":[{"name":"Department of Biomedical, Industrial, &amp; Human Factors Engineering Wright State University Dayton Ohio"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yune","family":"Kunes","sequence":"additional","affiliation":[{"name":"Bioanalytical and BioMarker Development Shire Lexington Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nan","family":"Wang","sequence":"additional","affiliation":[{"name":"Biostatistics &amp; Statistical Programming Department Shire Lexington Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric","family":"Crombez","sequence":"additional","affiliation":[{"name":"Rare Disease Unit, Shire Lexington Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ari","family":"Zimran","sequence":"additional","affiliation":[{"name":"Gaucher Clinic, Shaare Zedek Medical Center, affiliated with the Hebrew University\u2010Hadassah Medical School Jerusalem Israel"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2015,6,22]]},"reference":[{"key":"e_1_2_6_2_1","first-page":"1065\u20131071","volume-title":"Williams Hematology","author":"Zimran A","year":"2010"},{"key":"e_1_2_6_3_1","first-page":"155\u2013173","volume-title":"Gaucher disease","author":"Mistry PK","year":"2007"},{"key":"e_1_2_6_4_1","unstructured":"Shire Human Genetic Therapies Inc.VPRIV\u2122: Highlights of prescribing information. 2010. 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