{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T07:42:37Z","timestamp":1784619757696,"version":"3.55.0"},"reference-count":0,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2007,9,21]],"date-time":"2007-09-21T00:00:00Z","timestamp":1190332800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2007,11]]},"abstract":"<jats:title>Summary<\/jats:title><jats:sec><jats:title>Objective:<\/jats:title><jats:p>Intravenous enzyme replacement therapy (ERT) with recombinant \u03b1\u2010<jats:sc>l<\/jats:sc>\u2010iduronidase may ameliorate the non\u2010neurological symptoms in patients with mucopolysaccharidosis type I (MPS I). Since home\u2010based ERT for Gaucher and Fabry diseases has been reported to be safe and successful, we investigated the feasibility and safety of home therapy in patients with MPS I.<\/jats:p><\/jats:sec><jats:sec><jats:title>Setting:<\/jats:title><jats:p>This two\u2010centre study included 17 ERT\u2010treated MPS I patients between 1 and 35 years of age. A patient was allowed to transfer to home treatment after a minimum period of 6 months of in\u2010hospital administration of ERT and after a self\u2010 or home nurse\u2010supported home setting was arranged.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p>Thirteen out of 17 patients transferred to home treatment with a median time to transfer of 13 months (range 7\u201340 months). Two patients preferred to continue ERT in the hospital, whereas for two other patients the transfer to home was hampered for practical reasons. All patients who received ERT at home were assisted by either a relative or a nurse. In total over 1000 home infusions were performed and no serious complications were observed. Two infusion\u2010associated reactions were observed, both within the first 3 months of in\u2010hospital administration of ERT. All patients except one developed antibodies against the recombinant enzyme, but this was not associated with the development of hypersensitivity reactions.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion:<\/jats:title><jats:p>ERT for MPS I applied at home is safe and might alleviate the burden of life\u2010long intravenous treatment in these patients.<\/jats:p><\/jats:sec>","DOI":"10.1007\/s10545-007-0686-8","type":"journal-article","created":{"date-parts":[[2007,9,18]],"date-time":"2007-09-18T23:16:14Z","timestamp":1190157374000},"page":"984-984","source":"Crossref","is-referenced-by-count":28,"title":["Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe"],"prefix":"10.1002","volume":"30","author":[{"given":"J.","family":"Cox\u2010Brinkman","sequence":"first","affiliation":[{"name":"Department of Pediatrics Division of Metabolic Diseases PO Box 22660 G8\u2010205, Academic Medical Center Amsterdam 1100 DD The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R. G. M.","family":"Timmermans","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine Erasmus Medical Center Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"F. A.","family":"Wijburg","sequence":"additional","affiliation":[{"name":"Department of Pediatrics Division of Metabolic Diseases PO Box 22660 G8\u2010205, Academic Medical Center Amsterdam 1100 DD The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"W. E.","family":"Donker","sequence":"additional","affiliation":[{"name":"Department of Biochemistry Academic Medical Center Amsterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A. T.","family":"van de Ploeg","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Metabolic Diseases and Genetics Erasmus Medical Center Rotterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J. M. F. G.","family":"Aerts","sequence":"additional","affiliation":[{"name":"Department of Biochemistry Academic Medical Center Amsterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C. E. M.","family":"Hollak","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, Division of Endocrinology and Metabolism Academic Medical Center Amsterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2007,9,21]]},"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/s10545-007-0686-8","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T04:43:36Z","timestamp":1694753016000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1007\/s10545-007-0686-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2007,9,21]]},"references-count":0,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2007,11]]}},"alternative-id":["10.1007\/s10545-007-0686-8"],"URL":"https:\/\/doi.org\/10.1007\/s10545-007-0686-8","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2007,9,21]]}}}