The impact of Hunter syndrome (mucopolysaccharidosis type II) on health-related quality of life.

ABSTRACT:

Background

Hunter syndrome (mucopolysaccharidosis type II (MPS II)) is a rare metabolic disease that can severely compromise health, well-being and life expectancy. Little evidence has been published on the impact of MPS II on health-related quality of life (HRQL). The objective of this study was to describe this impact using the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) questionnaire and a range of standard validated questionnaires previously used in paediatric populations.

Methods

Clinical and demographic characteristics collected in a clinical trial and responses to four HRQL questionnaires completed both by patients and parents prior to enzyme replacement treatment were used. The association between questionnaire scores and clinical

SUBMITTER: Raluy-Callado M 

PROVIDER: S-EPMC3722040 | biostudies-literature | 2013 Jul

REPOSITORIES: biostudies-literature