Enzyme replacement therapy for mucopolysaccharidoses: opinions of patients and families.
J Pediatr
; 152(5): 723-7, 2008 May.
Article
in En
| MEDLINE
| ID: mdl-18410781
ABSTRACT
OBJECTIVES:
To assess the opinions of individuals with mucopolysaccharidoses (MPS) and their parents regarding the use of enzyme replacement therapy (ERT). STUDYDESIGN:
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.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Sulfatases
/
Family
/
Patient Acceptance of Health Care
/
Mucopolysaccharidoses
Type of study:
Qualitative_research
Aspects:
Patient_preference
Limits:
Adult
/
Child
/
Female
/
Humans
/
Male
Country/Region as subject:
America do norte
/
Oceania
Language:
En
Journal:
J Pediatr
Year:
2008
Document type:
Article
Affiliation country:
Australia