Document Detail

Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe.
MedLine Citation:
PMID:  17879143     Owner:  NLM     Status:  MEDLINE    
OBJECTIVE: Intravenous enzyme replacement therapy (ERT) with recombinant alpha-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.
J Cox-Brinkman; R G M Timmermans; F A Wijburg; W E Donker; A T van de Ploeg; J M F G Aerts; C E M Hollak
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Publication Detail:
Type:  Journal Article; Multicenter Study     Date:  2007-09-21
Journal Detail:
Title:  Journal of inherited metabolic disease     Volume:  30     ISSN:  1573-2665     ISO Abbreviation:  J. Inherit. Metab. Dis.     Publication Date:  2007 Nov 
Date Detail:
Created Date:  2007-12-06     Completed Date:  2008-01-08     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  7910918     Medline TA:  J Inherit Metab Dis     Country:  Netherlands    
Other Details:
Languages:  eng     Pagination:  984     Citation Subset:  IM    
Department of Pediatrics, Division of Metabolic Diseases, G8-205, Academic Medical Center, PO Box 22660, 1100 DD, Amsterdam, The Netherlands.
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MeSH Terms
Child, Preschool
Enzymes / therapeutic use*
Home Infusion Therapy / methods*
Home Nursing
Mucopolysaccharidosis I / therapy*
Patient Satisfaction
Quality of Life
Self Care
Reg. No./Substance:

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