| Enzyme replacement therapy for mucopolysaccharidoses: opinions of patients and families. | |
| | |
MedLine Citation:
|
PMID: 18410781 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
|
OBJECTIVES: To assess the opinions of individuals with mucopolysaccharidoses (MPS) and their parents regarding the use of enzyme replacement therapy (ERT). STUDY DESIGN: A validated questionnaire, including hypothetical clinical scenarios about ERT for MPS, was distributed to members of MPS support groups in the United States and Australia. RESULTS: The questionnaire was completed by 249 MPS support group members. Overall, 92% were in favor of ERT where MPS causes severe physical problems but does not affect intellect, and 69% were in favor of ERT where the physical limitations are mild and intellect is spared. Only 47% were in favor of ERT where severe physical and intellectual problems are well established; however, 77% were in favor of ERT in this situation if treatment begun early prolongs life and improves quality of life. CONCLUSION: Most respondents were in favor of ERT for MPS, even where it would not alter the intellectual deterioration. The medical community has a responsibility to advocate for their patients in situations where ERT is appropriate and recognize the economic burden and "family function burden" ERT can incur. |
| | |
Authors:
|
David J Coman; Ian M Hayes; Veronica Collins; Margaret Sahhar; J Ed Wraith; Martin B Delatycki |
Publication Detail:
|
Type: Journal Article; Research Support, Non-U.S. Gov't Date: 2008-02-20 |
Journal Detail:
|
Title: The Journal of pediatrics Volume: 152 ISSN: 1097-6833 ISO Abbreviation: J. Pediatr. Publication Date: 2008 May |
Date Detail:
|
Created Date: 2008-04-15 Completed Date: 2008-05-06 Revised Date: - |
Medline Journal Info:
|
Nlm Unique ID: 0375410 Medline TA: J Pediatr Country: United States |
Other Details:
|
Languages: eng Pagination: 723-7 Citation Subset: AIM; IM |
Affiliation:
|
Bruce Lefroy Centre for Genetic Health Research, Murdoch Childrens Research Institute, Victoria, Australia. |
Export Citation:
|
APA/MLA Format Download EndNote Download BibTex |
| MeSH Terms | |
Descriptor/Qualifier:
|
Adult Australia Child Family / psychology* Female Health Care Surveys Humans Male Mucopolysaccharidoses / drug therapy*, psychology Patient Acceptance of Health Care* Sulfatases / adverse effects, therapeutic use* Treatment Outcome United States |
| Chemical | |
Reg. No./Substance:
|
EC 3.1.6.-/Sulfatases |
From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
Previous Document: Effect of ascertainment and genetic features on the phenotype of Klinefelter syndrome.
Next Document: Urinary loss of erythropoietin after intravenous versus subcutaneous epoetin-beta in preterm infants...