Development and validation of parent-reported gastrointestinal health scale in MECP2 duplication syndrome.

ABSTRACT:

Background/aims

We aimed to develop a validated patient-reported Gastrointestinal Health Scale (GHS) specific to MECP2 Duplication Syndrome (MDS) to be used in clinical trials.

Methods

MDS parents completed a Gastrointestinal Health Questionnaire (GHQ) to investigate the most relevant and important items associated with gastrointestinal problems in MECP2-related disorders. Item reduction was executed according to EORTC guidelines. We performed reliability and validity studies for the finalized scale.

Results

A total of 106 surveys were eligible for item reduction and validation processes. The initial 55 items were reduced to 38 items based on parent responses, expert opinion, and initial confirmatory factor analysis (CFA). The final MDS-specific GHS included 38 items

SUBMITTER: Pehlivan D 

PROVIDER: S-EPMC10854118 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature