Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe
Objective: Intravenous enzyme replacement therapy (ERT) with recombinant α-l-iduronidase may ameliorate the non-neurological symptoms in patients with mucopolysaccharidosis type I (MPS I). Since home-based ERT for Gaucher and Fabry diseases has been reported to be safe and successful, we investigate...
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| Veröffentlicht in: | Journal of inherited metabolic disease 2007-11, Vol.30 (6), p.984-984 |
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| container_title | Journal of inherited metabolic disease |
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| creator | Cox-Brinkman, J Timmermans, R. G. M Wijburg, F. A Donker, W. E van de Ploeg, A. T Aerts, J. M. F. G Hollak, C. E. M |
| description | Objective: Intravenous enzyme replacement therapy (ERT) with recombinant α-l-iduronidase may ameliorate the non-neurological symptoms in patients with mucopolysaccharidosis type I (MPS I). Since home-based 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. Setting: This two-centre study included 17 ERT-treated 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-hospital administration of ERT and after a self- or home nurse-supported home setting was arranged. Results: Thirteen out of 17 patients transferred to home treatment with a median time to transfer of 13 months (range 7-40 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-associated reactions were observed, both within the first 3 months of in-hospital administration of ERT. All patients except one developed antibodies against the recombinant enzyme, but this was not associated with the development of hypersensitivity reactions. Conclusion: ERT for MPS I applied at home is safe and might alleviate the burden of life-long intravenous treatment in these patients. |
| doi_str_mv | 10.1007/s10545-007-0686-8 |
| format | Article |
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G. M ; Wijburg, F. A ; Donker, W. E ; van de Ploeg, A. T ; Aerts, J. M. F. G ; Hollak, C. E. M</creator><creatorcontrib>Cox-Brinkman, J ; Timmermans, R. G. M ; Wijburg, F. A ; Donker, W. E ; van de Ploeg, A. T ; Aerts, J. M. F. G ; Hollak, C. E. M</creatorcontrib><description>Objective: Intravenous enzyme replacement therapy (ERT) with recombinant α-l-iduronidase may ameliorate the non-neurological symptoms in patients with mucopolysaccharidosis type I (MPS I). Since home-based 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. Setting: This two-centre study included 17 ERT-treated 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-hospital administration of ERT and after a self- or home nurse-supported home setting was arranged. Results: Thirteen out of 17 patients transferred to home treatment with a median time to transfer of 13 months (range 7-40 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-associated reactions were observed, both within the first 3 months of in-hospital administration of ERT. All patients except one developed antibodies against the recombinant enzyme, but this was not associated with the development of hypersensitivity reactions. Conclusion: ERT for MPS I applied at home is safe and might alleviate the burden of life-long intravenous treatment in these patients.</description><identifier>ISSN: 0141-8955</identifier><identifier>EISSN: 1573-2665</identifier><identifier>DOI: 10.1007/s10545-007-0686-8</identifier><identifier>PMID: 17879143</identifier><identifier>CODEN: JIMDDP</identifier><language>eng</language><publisher>Dordrecht: Dordrecht : Springer Netherlands</publisher><subject>Adolescent ; Adult ; Biological and medical sciences ; Carbohydrates (enzymatic deficiencies). Glycogenosis ; Child ; Child, Preschool ; Enzyme Therapy ; Errors of metabolism ; Home Infusion Therapy - methods ; Home Nursing ; Hospitals ; Humans ; Infant ; Medical genetics ; Medical sciences ; Metabolic diseases ; Mucopolysaccharidosis I - therapy ; Patient Satisfaction ; Quality of Life ; Safety ; Self Care</subject><ispartof>Journal of inherited metabolic disease, 2007-11, Vol.30 (6), p.984-984</ispartof><rights>2007 SSIEM</rights><rights>2008 INIST-CNRS</rights><rights>SSIEM and Springer 2007</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c429A-cbd7a79c07e8490146ea0507aa74015d6776fba8e471f5020082b360df4a43bb3</citedby></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1007%2Fs10545-007-0686-8$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1007%2Fs10545-007-0686-8$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,776,780,1411,27901,27902,45550,45551</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=19912480$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/17879143$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Cox-Brinkman, J</creatorcontrib><creatorcontrib>Timmermans, R. G. M</creatorcontrib><creatorcontrib>Wijburg, F. A</creatorcontrib><creatorcontrib>Donker, W. E</creatorcontrib><creatorcontrib>van de Ploeg, A. T</creatorcontrib><creatorcontrib>Aerts, J. M. F. G</creatorcontrib><creatorcontrib>Hollak, C. E. M</creatorcontrib><title>Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe</title><title>Journal of inherited metabolic disease</title><addtitle>J Inherit Metab Dis</addtitle><description>Objective: Intravenous enzyme replacement therapy (ERT) with recombinant α-l-iduronidase may ameliorate the non-neurological symptoms in patients with mucopolysaccharidosis type I (MPS I). Since home-based 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. Setting: This two-centre study included 17 ERT-treated 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-hospital administration of ERT and after a self- or home nurse-supported home setting was arranged. Results: Thirteen out of 17 patients transferred to home treatment with a median time to transfer of 13 months (range 7-40 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-associated reactions were observed, both within the first 3 months of in-hospital administration of ERT. All patients except one developed antibodies against the recombinant enzyme, but this was not associated with the development of hypersensitivity reactions. Conclusion: ERT for MPS I applied at home is safe and might alleviate the burden of life-long intravenous treatment in these patients.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Biological and medical sciences</subject><subject>Carbohydrates (enzymatic deficiencies). Glycogenosis</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Enzyme Therapy</subject><subject>Errors of metabolism</subject><subject>Home Infusion Therapy - methods</subject><subject>Home Nursing</subject><subject>Hospitals</subject><subject>Humans</subject><subject>Infant</subject><subject>Medical genetics</subject><subject>Medical sciences</subject><subject>Metabolic diseases</subject><subject>Mucopolysaccharidosis I - therapy</subject><subject>Patient Satisfaction</subject><subject>Quality of Life</subject><subject>Safety</subject><subject>Self Care</subject><issn>0141-8955</issn><issn>1573-2665</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2007</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>BENPR</sourceid><recordid>eNqFkM1v1DAQxS0EotvCH8AFokrlFhg7dmwfV-Wji4o4QM_WxJmwqfKFnagKfz1edqVKXDjN0-j33oweY684vOMA-n3koKTKk8yhNGVunrANV7rIRVmqp2wDXPLcWKXO2HmM9wBgjVLP2RnXRlsuiw2rb8aesjkQzj0Nc_bQzvuMht9r2gaaOvT0dz_vKeC0Zs0Ysn7x4zR2a0Tv9xjaeoxtzOZ1omyXJdUQxrbqKMOhziI29II9a7CL9PI0L9jdp48_rm_y22-fd9fb29xLYbe5r2qN2nrQZKRNz5eEoEAjaglc1aXWZVOhIal5o0AAGFEVJdSNRFlUVXHB3h5zpzD-WijOrm-jp67DgcYlutKCUMaoBF7-A96PSxjSb07wVE2hZZEgfoR8GGMM1LgptD2G1XFwh_7dsX93kIf-nUme16fgpeqpfnScCk_A1QnA6LFrAg6-jY-ctVxIA4kzR-6h7Wj9_2X3Zff1A1gltsn65mhtcHT4M6T4u-8CeAFcC7BQFn8AyyKnQg</recordid><startdate>200711</startdate><enddate>200711</enddate><creator>Cox-Brinkman, J</creator><creator>Timmermans, R. 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Glycogenosis</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Enzyme Therapy</topic><topic>Errors of metabolism</topic><topic>Home Infusion Therapy - methods</topic><topic>Home Nursing</topic><topic>Hospitals</topic><topic>Humans</topic><topic>Infant</topic><topic>Medical genetics</topic><topic>Medical sciences</topic><topic>Metabolic diseases</topic><topic>Mucopolysaccharidosis I - therapy</topic><topic>Patient Satisfaction</topic><topic>Quality of Life</topic><topic>Safety</topic><topic>Self Care</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Cox-Brinkman, J</creatorcontrib><creatorcontrib>Timmermans, R. G. M</creatorcontrib><creatorcontrib>Wijburg, F. A</creatorcontrib><creatorcontrib>Donker, W. E</creatorcontrib><creatorcontrib>van de Ploeg, A. T</creatorcontrib><creatorcontrib>Aerts, J. M. F. G</creatorcontrib><creatorcontrib>Hollak, C. E. 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Setting: This two-centre study included 17 ERT-treated 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-hospital administration of ERT and after a self- or home nurse-supported home setting was arranged. Results: Thirteen out of 17 patients transferred to home treatment with a median time to transfer of 13 months (range 7-40 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-associated reactions were observed, both within the first 3 months of in-hospital administration of ERT. 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| ispartof | Journal of inherited metabolic disease, 2007-11, Vol.30 (6), p.984-984 |
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| source | MEDLINE; Springer Nature - Complete Springer Journals; Wiley Online Library Journals Frontfile Complete |
| subjects | Adolescent Adult Biological and medical sciences Carbohydrates (enzymatic deficiencies). Glycogenosis Child Child, Preschool Enzyme Therapy Errors of metabolism Home Infusion Therapy - methods Home Nursing Hospitals Humans Infant Medical genetics Medical sciences Metabolic diseases Mucopolysaccharidosis I - therapy Patient Satisfaction Quality of Life Safety Self Care |
| title | Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe |
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