{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,10]],"date-time":"2026-03-10T01:21:05Z","timestamp":1773105665180,"version":"3.50.1"},"reference-count":12,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2023,7,26]],"date-time":"2023-07-26T00:00:00Z","timestamp":1690329600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100008373","name":"Takeda Pharmaceutical Company","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008373","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Internal Medicine Journal"],"published-print":{"date-parts":[[2024,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Enzyme replacement therapy (ERT) has revolutionised the management of patients with Gaucher disease (GD). In 2018, we published the safety and efficacy of rapid 10\u2010min infusion of velaglucerase alfa in previously treated patients, mostly on low\u2010dose therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To improve quality of life (QoL) for patients needing lifelong bi\u2010weekly infusions by introducing a 10\u2010min infusion instead of 1 h per label in patients naive to ERT and on high\u2010dose therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Fifteen naive patients were enrolled; all received bi\u2010weekly infusions of 60 units\/kgBW velaglucerase alfa; the infusion rate was gradually reduced in the hospital, followed by home infusions. Each infusion was followed for safety. Efficacy parameters were assessed every 3 months. Patient\u2010reported outcome questionnaires were collected at baseline and follow\u2010up.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Ten\u2010minute rapid infusions were well tolerated without related severe adverse events (SAEs). Two patients experienced a non\u2010related SAE and another a possibly related AE. In three patients, the infusion rate was increased to 30 or 60 min (two because of suboptimal response and one because of AE). Two patients dropped out because of an unwillingness to attend follow\u2010up visits during the COVID\u201019 pandemic. All 13 remaining patients reached the 24\u2010month end\u2010point. The platelet counts increased by a median (range) of 68.38% (12.5\u2013300%) and the lyso\u2010Gb1 levels decreased by 62.6% (32.9\u201389.9%).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Home therapy with rapid infusion of high\u2010dose velaglucerase alfa was a safe, effective and preferable alternative for patients with GD na\u00efve to treatment. We believe that shortening the infusion time improves the QoL of patients with GD who have a lifelong commitment to intravenous therapy.<\/jats:p><\/jats:sec>","DOI":"10.1111\/imj.16179","type":"journal-article","created":{"date-parts":[[2023,7,26]],"date-time":"2023-07-26T13:19:30Z","timestamp":1690377570000},"page":"398-403","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Rapid home therapy infusion of velaglucerase alfa in na\u00efve patients with Gaucher disease"],"prefix":"10.1111","volume":"54","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5894-6742","authenticated-orcid":false,"given":"Michal","family":"Becker\u2010Cohen","sequence":"first","affiliation":[{"name":"Gaucher Unit Shaare Zedek Medical Centre  Jerusalem 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