Validation of the shortened Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS).

ABSTRACT:

Background

The rare disease, Hunter Syndrome (mucopolysaccharidosis type II; MPS II), characterized by iduronate-2-sulfatase deficiency, has heterogeneous symptoms often including cognitive impairment (CI). To evaluate physical functioning and daily activity limitations of patients with MPS II, the multidomain shortened Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) questionnaire was previously developed and preliminarily validated. Here we gather data in a dedicated prospective longitudinal observational study using direct responses to the shortened instrument and assess its psychometric properties further.

Methods

Interview data were collected from eligible self-reporting patients (≥ 12 years of age) or caregivers of patients using respecti

SUBMITTER: Mattera M 

PROVIDER: S-EPMC6225555 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature