Review of clinical trials and guidelines for children and youth with mucopolysaccharidosis: outcome selection and measurement.

ABSTRACT:

Background

To inform the development of a core outcome set (COS) for children and youth with mucopolysaccharidoses (MPS), we aimed to identify all outcomes and associated outcome measurement instruments that are reported in recent clinical trials and recommended as measurements in clinical management guidelines.

Methods

To identify English-language clinical trials and guidelines pertaining to MPS published between 2011 and mid-2021, we applied a comprehensive peer-reviewed search strategy to relevant databases and registers on May 16, 2021. Two reviewers independently screened retrieved citations and then full-text articles to determine eligibility for inclusion. From articles meeting inclusion criteria, we extracted details of the study design, population, intervention, and

SUBMITTER: Howie AH 

PROVIDER: S-EPMC11520150 | biostudies-literature | 2024 Oct

REPOSITORIES: biostudies-literature